January | February | March | April | May | June | July | August | September | October | November | December |
| Company | Product | Description | Indication | Phase II status | Date | Therapeutic area |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Tibsovo (ivosidenib tablets) | IDH1 gene inhibitor | IDH1-mutated cholangiocarcinoma | Full analysis of final data from Claridhy trial in patients with previously treated disease showed an improvement in secondary endpoint of overall survival favoring Tibsovo arm vs. placebo, though statistical significance was not reached; median OS for Tibsovo was 10.3 months vs. 7.5 months for placebo (1-sided p=0.093) | 1/17/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Calquence (acalabrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Elevate-RR trial met primary endpoint of noninferior progression-free survival vs. ibrutinib (Imbruvica, Janssen Pharmaceuticals) in adults with previously treated, high-risk disease; trial also met key secondary safety endpoint, showing statistically significantly lower incidence of atrial fibrillation for study drug vs. ibrutinib | 1/25/21 | Cancer |
| Astrazeneca, of Cambridge, U.K. | Tagrisso (osimertinib) | Tyrosine kinase inhibitor; epidermal growth factor receptor antagonist | EGFR-mutated non-small-cell lung cancer | Exploratory analysis of the Adaura study showed Tagrisso reduced the risk of disease recurrence or death by 84% in patients who had been treated with prior adjuvant chemotherapy and by 77% in patients who had not had the prior therapy | 1/29/21 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | Monoclonal antibody targeting PD-1 | Advanced unresectable or metastatic esophageal squamous cell carcinoma | The Rationale 302 study met its primary endpoint with tislelizumab improving overall survival compared to chemotherapy; data to be presented at an upcoming medical conference | 1/27/21 | Cancer |
| Blue Earth Diagnostics Inc., of Burlington, Mass. | 18F-fluciclovine | PET imaging radiopharmaceutical | Recurrent brain metastases | First patient dose in Revelate trial assessing diagnostic performance in detecting recurrent brain metastases in patients previously treated with radiation therapy | 1/12/21 | Cancer |
| Cellectar Biosciences Inc., of Florham Park, N.J. | CLR-131 | Phospholipid Drug Conjugate designed to provide targeted delivery of radioisotope iodine-131 | Waldenström's macroglobulinemia | Initiated pivotal trial, designed as global, noncomparator, single-arm, expansion cohort of ongoing phase II Clover-1 trial | 1/12/21 | Cancer |
| Denovo Biopharma LLC, of San Diego | DB-102 (enzastaurin) | Protein kinase C beta inhibitor | Newly diagnosed glioblastoma | Dosed the first patient in its biomarker-guided study evaluating DB-102 in combination with temozolomide and radiation as first line therapy | 1/8/21 | Cancer |
| Genmab A/S, of Copenhagen, Denmark, and Seagen Inc., of Bothell, Wash. | Tisotumab vedotin | Antibody-drug conjugate targeting tissue factor | Recurrent or metastatic cervical cancer after 1 or 2 prior lines of systemic therapy | Started the InnovaTV 301 study testing tisotumab vedotin compared to physician’s choice single-agent chemotherapy; primary endpoint is overall survival | 1/4/21 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Margenza (margetuximab-cmkb) | HER2-targeting monoclonal antibody | Metastatic breast cancer | Pivotal Sophia trial findings in which study drug + chemotherapy showed 24% progression-free survival relative risk reduction vs. trastuzumab + chemotherapy in people with HER2-positive disease (median PFS 5.8 vs. 4.9 months) published in JAMA Oncology | 1/25/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Metastatic non-small-cell lung cancer without EGFR or ALK genomic tumor aberrations and with PD-L1 expression | In the Keynote-498 study, overall survival was 21.4 months for Keytruda plus Yervoy (ipilimumab, Bristol Myers Squibb Co.) compared to 21.9 months for Keytruda alone (p=0.74); median progression-free survival was 8.2 months for the combination vs. 8.4 months for Keytruda alone (p=0.72) | 1/29/21 | Cancer |
| Merck KGaA, of Darmstadt, Germany, and Glaxosmithkline plc, of London | Bintrafusp alfa | Bifunctional immunotherapy blocking TGF-beta and PD-L1 pathways | Stage IV non-small-cell lung cancer | Independent data monitoring committee recommended discontinuing the INTR@PID Lung 037 study because it's unlikely to meet the co-primary endpoint of progression-free survival | 1/20/21 | Cancer |
| Philogen SpA, of Siena, Italy | Nidlegy | Immunocytokines L19IL2 and L19TNF | Locally advanced, fully resectable metastatic melanoma | After review of data from 149 of the anticipated 214 patients, the data and safety monitoring board recommended continuing the study | 1/13/21 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | Anti-PD-L1 inhibitor | Hepatocellular carcinoma | Updated data from Imbrave150 study in combination with Avastin (bevacizumab) vs. sorafenib in patients with unresectable disease who have not received prior systemic therapy showed, at median follow-up of 15.6 months, reduction in risk of death by 34%; median overall survival of 19.2 months vs. 13.4 months for sorafenib | 1/12/21 | Cancer |
| Mesoblast Ltd., of Melbourne, Australia | Revascor (rexlemestrocel-L) | Allogeneic cell therapy | Chronic heart failure | Additional data from Dream-HF trial showed single dose reduced incidence of heart attacks and strokes by 60% over median follow-up of 30 months in 537 people with New York Heart Association class II or III disease (5% vs. 13%, p=0.002); those who received study drug had 68% reduction in rate of recurrent hospitalizations from non-fatal heart attacks or strokes vs. controls, with hospitalization rate of 1.90 vs. 5.95, respectively, per 100 patient-years of follow-up (p=0.0002) | 1/10/21 | Cardiovascular |
| Mesoblast Ltd., of New York | Rexlemestrocel-L | Cardioprotectant | Chronic heart failure | Additional data from Dream-HF trial showed 60% reduction in incidence of Major Adverse Cardiac Events (MACE) due to heart attacks or strokes across entire 537-patient study population, irrespective of NYHA class II or III, ischemic or non-ischemic etiology (p=0.002); 68% reduction in rate of recurrent hospitalizations from nonfatal heart attacks or strokes, with a hospitalization rate of 1.90 per 100 patient-years of follow-up vs. 5.95 per 100 patient-years of follow-up in control arm (p=0.0002); 60% reduction in cardiac death in NYHA class II patients (p=0.037) and prevention of progression to NYHA class III rate of cardiac death (p=0.004); 30% reduction in incidence of 3-point MACE (cardiac death, heart attack or stroke) across entire population (p=0.027) | 1/12/21 | Cardiovascular |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa. | Bentracimab | Monoclonal antibody fragment targeting ticagrelor | Uncontrolled major or life-threatening bleeding or patients who require urgent surgery or invasive procedure | Expanded the ongoing Reverse-it study into the EU | 1/28/21 | Cardiovascular |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | Small-molecule BET inhibitor | Heart failure | Prespecified analysis from Betonmace study published in Cardiovascular Diabetology showed significantly fewer hospitalizations for heart failure (HHF) patients with type 2 diabetes and recent history or acute coronary syndrome vs. placebo; company plans to include HHF in composite primary endpoint for upcoming registration-enabling study, Betonmace2 | 1/12/21 | Cardiovascular |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream (ARQ-151) | Phosphodiesterase type 4 inhibitor | Atopic dermatitis | Started the Integument-1 and -2 studies, which will enroll 650 patients each; primary endpoint of both studies is Investigator Global Assessment (IGA) success, defined as a Validated IGA- Atopic Dermatitis score of clear or almost clear plus a 2-grade improvement from baseline at week 4 | 1/13/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., of Tokyo | BBI-4000 | Sofpironium bromide gel | Primary axillary hyperhidrosis | Pivotal data from Japanese trial published in the Journal of Dermatology showed proportion of patients who achieved primary endpoint was 53.9% vs. 36.4% in vehicle group (p=0.003) | 1/19/21 | Dermatologic |
| Aeglea Biotherapeutics Inc., of Austin, Texas | Pegzilarginase | Human arginase | Arginase 1 deficiency | Disclosed in SEC filing that COVID-19 has created limited access environment, impacting the ability for some sites in Peace study to schedule and complete initial screening visits; company now expects to complete enrollment by end of March 2021 and anticipates top-line data in fourth quarter of 2021; to date, 22 patients (75%) have been enrolled | 1/5/21 | Endocrine/Metabolic |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Mitapivat | Pyruvate kinase R activator | Pyruvate kinase deficiency | In the Activate-T study, 37% of the 27 patients who were regularly transfused achieved a ?33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks after treatment with mitapivat (p=0.0002); 22% of the patients were transfusion-free during the 24-week fixed dose period | 1/26/21 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR gene inhibitor | Transthyretin-mediated (ATTR) amyloidosis | At 9 months, Helios-A study in people with hereditary disease with polyneuropathy met primary endpoint of change from baseline in modified Neuropathy Impairment Score and secondary endpoints of changes in quality of life assessed by Norfolk Quality of Life Questionnaire-Diabetic Neuropathy and gait speed assessed by timed 10-meter walk | 1/7/21 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | Started randomization of the 675-patient study comparing once-daily and twice-daily treatment to placebo; primary endpoint is A1c levels; secondary endpoint is change in fasting plasma glucose | 1/21/21 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | IL-23 inhibitor | Crohn's disease | In Advance and Motivate studies in adults with moderate to severe disease, both doses (600 mg and 1,200 mg) met co-primary endpoints of clinical remission measured by CDAI and endoscopic response, both at week 12 (p<0.001 for both doses on endpoints)< td> | 1/7/21 | Gastrointestinal |
| Calliditas Therapeutics AB, of Stockholm | Nefecon | Targeted release formulation of the corticosteroid budesonide | Primary IgA nephropathy | Completed enrollment in part B of the 360-patient Neflgard study | 1/21/21 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland, and Pfizer Inc., of New York | Relugolix | Oral GnRH receptor antagonist | Endometriosis | In the Spirit study, 84.8% of patients had clinically meaningful reductions in dysmenorrhea at 1 year; 73.3% of women had reductions in non-menstrual pelvic pain; drug produced an 82.8% average reduction from baseline on the Numerical Rating Scale for dysmenorrhea; plans to submit data in an NDA to the FDA in first half of 2021 | 1/26/21 | Genitourinary/Sexual Function |
| Rockwell Medical Inc., of Wixom, Mich., and Shanghai Fosun Pharmaceutical Group Co. Ltd., of Shanghai | Triferic dialysate | Iron replacement | Anemic patients with chronic kidney disease requiring hemodialysis | Enrolled first patient in the RMFPC-13 study in China | 1/13/21 | Genitourinary/Sexual Function |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | F8 gene stimulator | Hemophilia A | Ongoing Gener8-1 study in 134 adults with severe disease met primary and secondary efficacy endpoints in 1-year dataset; mean annualized bleeding rate reduced by 84% (p<0.0001) 5 and showed superiority to factor viii prophylaxis, with 80% of participants bleed-free starting at week after treatment; mean annualized infusion rate reduced by 99% (p<0.0001)< td> | 1/10/21 | Hematologic |
| Pharmaessentia Corp., of Burlington, Mass. | Ropeginterferon alfa-2b | Interferon alpha 2 ligand | Thrombocythemia | Pivotal Surpass study initiated, with plans to enroll about 160 participants, including those with high-risk essential disease, resistant to or intolerant of hydroxyurea and who did not receive prior interferon therapy; primary endpoint is patient response defined by blood count remission, improvement or non-progression in disease-related signs and symptoms, and absence of thrombotic events; top-line data expected by late 2021 | 1/7/21 | Hematologic |
| Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | Monoclonal antibody targeting interleukin-23 | Active psoriatic arthritis | In the Keepsake-1 and -2 studies, 57% and 51% of patients taking Skyrizi achieved ACR20 at week 24, respectively, compared to 34% and 27% of patients taking placebo, respectively (p<0.001 for both); 52% and 55% of patients taking skyrizi achieved pasi90, respectively, compared to 10% in both studies who received placebo (p<0.001 both)< td> | 1/5/21 | Immune |
| Kedrion Biopharma SpA, of Fort Lee, N.J. | 10% intravenous immunoglobulin | Antibodies | Primary immunodeficiency disease | Last patient treated in the Cares10 study | 1/4/21 | Immune |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab-cwvz) | Complement C5 factor inhibitor | COVID-19 infection | Enrollment paused in study of people with severe infection requiring mechanical ventilation based on independent data monitoring committee interim finding of lack of efficacy when added to standard of care | 1/13/21 | Infection |
| AM-Pharma BV, of Utrecht, the Netherlands | Recombinant alkaline phosphatase | Alkaline phosphatase stimulator | COVID-19 infection | First of up to 100 participants with confirmed infection and sepsis-associated acute kidney injury (SA-AKI) enrolled in exploratory cohort in pivotal Revival trial; main study population will include about 1,400 people with SA-AKI, with primary endpoint of improvement in 28-day all-cause mortality | 1/7/21 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Favipiravir | Antiviral | Outpatient COVID-19 | In the Preseco study, 12 out of 20 planned sites are recruiting patients | 1/28/21 | Infection |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland | Cresemba (isavuconazole) | Azole antifungal | Mycosis | Partner Asahi Kasei Pharma Corp. completed enrollment (n=103) in study in Japan of people with deep-seated infection, including invasive aspergillosis and mucormycosis; data expected in second half of 2021 | 1/7/21 | Infection |
| Dr. Reddy’s Laboratories Ltd., of Hyderabad, India | Avigan (favipiravir) | Antiviral | Moderate to severe COVID-19 | Study enrolling moderate to severe cases in Kuwait was terminated after data showed patients taking Avigan took 7 days for sustained hypoxia resolution compared to 8 days for placebo (p>0.05); patients in the subgroup with low-risk who were treated with Avigan were discharged from the hospital in 8 days compared to 11 days for placebo (p=0.0063); study of out-patient mild to moderate cases continues in North America | 1/27/21 | Infection |
| Eli Lilly and Co., of Indianapolis | Bamlanivimab and etesevimab | Monoclonal antibodies targeting SARS-CoV-2 | COVID-19 | In the Blaze-1 study, the antibodies reduced COVID-19-related hospitalizations and deaths by 70% (p=0.0004); in the Blaze-4 study, pharmacodynamic/pharmacokinetic data showed the bamlanivimab 700-mg and etesevimab 1,400-mg dose was similar to the bamlanivimab 2,800-mg and etesevimab 2,800-mg dose | 1/26/21 | Infection |
| Eli Lilly and Co., of Indianapolis, and Abcellera Biologics Inc., of Vancouver, British Columbia | Bamlanivimab (LY-CoV555) | Monoclonal antibody targeting SARS-CoV-2 | COVID-19 prophylaxis | In the Blaze-2 study, treatment with bamlanivimab reduced the risk of developing symptomatic COVID-19 compared to placebo (p=0.00026) | 1/21/21 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Monoclonal antibody targeting granulocyte macrophage-colony stimulating factor | COVID-19 | Completed enrollment in the study; top-line data expected in March 2021 | 1/29/21 | Infection |
| Insmed Inc., of Bridgewater, N.J. | Arikayce (amikacin) | 30S ribosomal protein inhibitor | Nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex | Treated first patient in the front-line clinical program, consisting of the Arise study to validate cross-sectional and longitudinal characteristics of patient-reported outcome tools and the Encore pivotal study in newly diagnosed patients | 1/4/21 | Infection |
| Johnson & Johnson, of New Brunswick, N.J. | Janssen COVID-19 vaccine candidate | Adenovirus-based vaccine | COVID-19 prophylaxis | In the Ensemble study, protection against moderate to severe COVID-19 infection was 72% in the U.S., 66% in Latin America and 57% in South Africa, 28 days post-vaccination; vaccine was 85% effective in preventing severe disease across all regions studied | 1/29/21 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Oteseconazole | Antifungal | Recurrent vulvovaginal candidiasis | Top-line results showed Ultraviolet trial met all primary and secondary endpoints; recurrence rate was 5.1% with Oteseconazole vs. 42.2% for fluconazole to placebo recurrence rate (p<0.001); 1 drug was noninferior to fluconazole in the resolution of signs and symptoms at day 14; recurrence rate 3.8% vs. 41.1% for placebo (p<0.001); prevented a recurring episode 95% women about year< td> | 1/6/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | CoV-2373 | Recombinant nanoparticle-based vaccine with saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | In the U.K. study, the vaccine had 89.3% efficacy at preventing mild, moderate or severe COVID-19; post hoc analysis showed 95.6% efficacy against the original COVID-19 strain and 85.6% efficacy against the U.K. variant strain | 1/28/21 | Infection |
| Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Oral small-molecule potassium-competitive acid blocker | H. pylori infection | Completed patient enrollment in Phalcon-HP pivotal study testing combination with amoxicillin and combination with amoxicillin and clarithromycin; top-line data expected in second quarter of 2021 | 1/19/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGEN-COV (casirivimab/imdevimab) | Monoclonal antibodies targeting SARS-CoV-2 | COVID-19 prophylaxis | In patients with household exposure to a COVID-19 patient; 8 of 223 participants who received placebo developed symptomatic infection vs. 0 of 186 participants treated with REGEN-COV; infection rates (symptomatic and asymptomatic) were 23/223 for placebo vs. 10/186 for REGEN-COV | 1/26/21 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Interferon beta-1a | Hospitalized COVID-19 | Treated first of 610 patients who require supplemental oxygen in the SG018 study | 1/13/21 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Pa. | VP-102 | Drug-device combination containing GMP-controlled formulation of cantharidin | Molluscum contagiosum | Data from post-hoc pooled analysis of pivotal Camp trials showed treated participants with lesions in the upper extremities, head/neck, back/buttocks and chest/abdomen at baseline showed statistically significantly higher rates of complete clearance in those regions vs. vehicle beginning after the first treatment (visit 2; day 21) through the end-of-treatment visit; VP-102-treated participants with lesions in the lower extremities at baseline showed statistically significantly higher complete clearance rates compared to vehicle-treated participants beginning after 2 treatments (visit 3; day 42) through the EOS visit (day 84) | 1/19/21 | Infection |
| Organogenesis Holdings Inc., of Canton, Mass. | Renu | Cryopreserved amniotic suspension allograft | Knee osteoarthritis | First of 474 participants with moderate to severe symptomatic disease enrolled in pivotal trial; primary efficacy endpoint is difference in pain from baseline to 6 months vs. placebo as assessed by WOMAC index | 1/14/21 | Inflammatory |
| Pfizer Inc., of New York | PF-06939926 | DMD gene stimulator | Duchenne muscular dystrophy | First of 99 ambulatory boys, ages 4 to 7, dosed in Ciffreo study; primary endpoint is change from baseline in North Star Ambulatory Assessment at 1 year | 1/7/21 | Musculoskeletal |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Troriluzole | Glutamate modulating agent | Obsessive-compulsive disorder | First patient enrolled in study testing the drug in approximately 600 patients not responding to standard of care treatment | 1/4/21 | Neurology/Psychiatric |
| Gensight Biologics SA, of Paris | Lumevoq (GS-010) | MT-ND4 gene stimulator | ND4 Leber hereditary optic neuropathy | Data from the Rescue study published in Ophthalmology showed the average change against the worst recorded best-corrected visual acuity was -0.53 LogMAR (+26 ETDRS letters equivalent) in Lumevoq-treated eyes and -0.46 LogMAR (+23 ETDRS letters equivalent) in sham-treated eyes (p<0.0001)< td> | 1/13/21 | Neurology/Psychiatric |
| Intra-Cellular Therapies Inc., of New York | Caplyta (lumateperone) | Serotonin 5-HT2A receptor/dopamine D2 receptor antagonist | Schizophrenia | Data from 6-week switching study in people with stable disease that showed drug was well-tolerated with stable symptom control published online in Schizophrenia Research | 1/21/21 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ganaxolone | GABA A receptor modulator | Refractory status epilepticus | Enrolled first of approximately 125 patients in the Raise study; co-primary endpoints are the proportion of patients with SE cessation within 30 minutes of treatment initiation without other medications for the treatment of SE and the proportion of patients with no progression to I.V. anesthesia for 36 hours following treatment initiation | 1/26/21 | Neurology/Psychiatric |
| Prilenia Therapeutics BV, of Naarden, the Netherlands | Pridopidine | Sigma 1-receptor agonist | Huntington’s disease | Enrolled first European patient in the ongoing Proof-HD study | 1/28/21 | Neurology/Psychiatric |
| Tris Pharma Inc., of Monmouth Junction, N.J. | Amphetamine extended-release tablet | Stimulant | Attention deficit hyperactivity disorder | Treatment with amphetamine extended-release tablet improved the average Permanent Product Measure of Performance total score over all post dose time points by 302.8 compared to 279.6 points for those treated with placebo (p=0.0043) | 1/20/21 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | RASP inhibitor | Dry eye disease | In run-in cohort (n=23) of Tranquility trial, study, drug was statistically superior to vehicle for visual analogue scale ocular dryness score (p=0.001) and ocular discomfort score (p<0.0001) and showed statistically significant improvement vs. vehicle (p="0.03)" in ocular redness< td> | 1/7/21 | Ocular |
| Bausch + Lomb, of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany | NOV-03 | Perfluorohexyloctane ophthalmic solution | Dry eye disease associated with Meibomian glad dysfunction | First of 2 studies has been completely enrolled, with total of 599 participants | 1/19/21 | Ocular |
| Genentech, unit of Roche Holding AG, of Basel, Switzerland | Faricimab | Bispecific antibody targeting angiopoietin-2 and VEGF-A | Wet age-related macular degeneration | Identically designed Tenaya and Lucerne studies that collectively enrolled 1,329 participants met primary endpoint, with study drug, dosed at intervals of up to every 16 weeks, producing noninferior BCVA gains vs. aflibercept (Eylea, Regeneron Pharmaceuticals Inc./Bayer AG) dosed every 8 weeks | 1/25/21 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Formulation of phentolamine mesylate | Night vision disturbances | Started patient recruitment and screening for the 160-patient Lynx-1 study; primary endpoint is the percentage of patients with at least 3 lines of improvement in mesopic, low-contrast, best-corrected distance visual acuity after 7 days; secondary endpoints include pupil diameter reductions, other visual acuity measures (distance and near) and safety and tolerability | 1/5/21 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, stable eye drop formulation of phentolamine mesylate | Reverse pharmacologically induced mydriasis | Completed enrollment earlier than expected in Mira-2 registration study | 1/6/21 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | Gene therapy | Wet age-related macular degeneration | First of 2 pivotal studies, Atmosphere, is active; patient screening is ongoing in study to enroll about 300 across 2 dose arms vs. ranibizumab; primary endpoint is noninferiority to ranibizumab based on change in baseline in Best Corrected Visual Acuity at 1 year | 1/6/21 | Ocular |
| Actinium Pharmaceuticals Inc., of New York | Iomab-B (apamistamab -131I) | Anti-CD45 radiolabeled with iodine-131 | Relapsed or refractory acute myeloid leukemia | In the Sierra study, all patients taking therapeutic dose of Iomab-B had bone marrow transplant and engraftment compared to 18% of patients receiving physician's choice of salvage therapy | 2/11/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | Recurrent or metastatic head and neck cancer | The Kestrel trial did not meet the primary endpoint of improving overall survival (OS) vs. the EXTREME treatment regimen (chemotherapy plus cetuximab), a standard of care; also, the combination did not indicate an OS benefit in all-comer patients, a secondary endpoint | 2/5/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | Poly ADP-ribose polymerase inhibitor | Germline BRCA-mutated, high-risk HER2-negative early-stage breast cancer | At the interim analysis, the independent data monitoring committee for the Olympia study determined that the trial crossed the superiority boundary for the primary endpoint of invasive disease-free survival | 2/17/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Anti-PD-1 antibody | Urothelial carcinoma | Results from Checkmate-274 study showed treatment significant improved disease-free survival (21 months vs. 10.9 months; p<0.001) as adjuvant across all randomized patients with surgically resected, high-risk muscle-invasive disease and in subgroup of whose tumors express pd-l1 ?1% (disease-free survival not reached for opdivo vs. 10.8 months placebo; p<0.001), meeting both primary endpoints< td> | 2/9/21 | Cancer |
| Centre for Probe Development and Commercialization, of Hamilton, Ontario | [18F]PSMA-1007 | Positron-emitting tomography imaging agent targeting prostate-specific membrane antigen | Prostate cancer | First patient dosed in the study that will enroll 100 men with suspected persistent or recurrent prostate cancer based on detectable levels of prostate serum antigen and a negative or equivocal detection of disease by conventional imaging | 2/24/21 | Cancer |
| Erytech Pharma SA, of Lyon, France | Eryaspase | L-asparaginase encapsulated inside donor-derived red blood cells | Second-line pancreatic cancer | Following a planned interim superiority analysis by the independent data monitoring committee, the TRYbeCA-1 study will continue without modification | 2/8/21 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Multityrosine kinase inhibitor | Renal cell carcinoma | In retrospective analysis of medical records from patients with metastatic RCC with brain metastases, an intracranial response rate of 61%, including a complete response rate of 13%, was seen for patients with progressing intracranial metastases at baseline treated with Cabometyx; patients without progressing intracranial metastases had an intracranial response rate of 57% | 2/9/21 | Cancer |
| Innovent Biologics Inc., of San Francisco | IBI-310 | Anti-CTLA4 antibody | Advanced hepatocellular carcinoma | Dosed first patient in study testing combination with Tyvyt (sintilimab) as first-line treatment | 2/8/21 | Cancer |
| Ipsen SA, of Paris | Cabometyx (cabozantinib) | Multityrosine kinase inhibitor | Advanced renal cell carcinoma | New analyses from Checkmate-9ER trial testing combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) vs. sunitinib as first-line treatment showed, at median follow-up of 23.5 months, combination continued to show superior progression-free survival (17 months vs. 8.3 months), objective response rate (54.8% vs. 28.4%) and overall survival (34% reduction in death) vs. sunitinib, with a low rate of treatment-related adverse events leading to discontinuation | 2/9/21 | Cancer |
| Janssen Pharmaceutica NV, of Beerse, Belgium, part of Johnson & Johnson | Apalutamide | Selective androgen receptor inhibitor | Advanced hormone-sensitive prostate cancer | Results from final analysis of Titan study, with nearly 4 years of median follow-up, showed combination with androgen deprivation therapy (ADT) provided statistically significant improvement in overall survival, with 35% reduction in risk of death vs. ADT alone (p<0.0001); improvement in os increased to a 48% reduction risk of death after adjusting for patients who crossed over (p<0.0001)< td> | 2/9/21 | Cancer |
| Janssen Pharmaceutica NV, of Beerse, Belgium, part of Johnson & Johnson | Apalutamide and abiraterone acetate | Selective androgen receptor inhibitor and cytochrome P450 17 inhibitor | Prostate cancer | Acis study met primary endpoint of radiographic progression-free survival, with 31% reduction in risk of radiographic progression or death in patients with chemotherapy-naïve metastatic castration-resistant prostate cancer receiving androgen deprivation therapy; median rPFS was 22.6 months vs. 16.6 months for the control arm (p<0.0001)< td> | 2/9/21 | Cancer |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Libtayo (cemiplimab) | Monoclonal antibody targeting PD-1 | Locally advanced or metastatic non-small-cell lung cancer | Data published in The Lancet showed Libtayo reduced the risk of death by 32% in all patients and by 43% in those with confirmed PD-L1 expression of 50% or higher | 2/12/21 | Cancer |
| Sumitomo Dainippon Pharma Oncology Inc., of Cambridge, Mass. | Napabucasin | Beta-catenin modulator; homeobox protein NANOG modulator; STAT-3 inhibitor | Metastatic colorectal cancer | Canstem303C study showed combination with FOLFIRI, with or without bevacizumab, in previously treated patients failed to reach primary endpoint of overall survival; combination failed to show significant OS improvement in general study population and in patients whose tumors were positive for pSTAT3 | 2/9/21 | Cancer |
| TG Therapeutics Inc., of New York | Ublituximab | Glycoengineered anti-CD20 monoclonal antibody | Chronic lymphocytic leukemia | Final results from Genuine combination trial with ibrutinib (Imbruvica, Johnson & Johnson/Abbvie Inc.) in 126 people with relapsed/refractory high-risk disease, published in The Lancet Haematology, showed overall response rate 90% (53 of 59) for combination vs. 69% (40 of 58) for ibrutinib alone (p=0.0060), with complete response/complete response with incomplete hematologic recovery rate of 20% (12 of 59) and 5% (3 of 58), respectively (p=0.024) | 2/23/21 | Cancer |
| VBL Therapeutics Ltd., of Tel Aviv, Israel | VB-111 | Anticancer gene therapy agent | Recurrent ovarian cancer | Independent data safety monitoring board found no safety issues in its preplanned review of the ongoing Oval study and recommended its continuation as planned | 2/22/21 | Cancer |
| Astellas Pharma Inc., of Tokyo | Fezolinetant | Neurokinin-3 receptor antagonist | Moderate to severe vasomotor symptoms | Both the Skylight 1 and Skylight 2 studies met all 4 co-primary endpoints for the 30-mg and 45-mg doses, showing statistically significant reduction from baseline in the frequency and severity of moderate to severe vasomotor symptoms to week 4 and week 12 compared to placebo | 2/19/21 | Cardiovascular |
| Almirall SA, of Exton, Pa., and Athenex Inc., of Buffalo, N.Y. | Klisyri (tirbanibulin) | Microtubule inhibitor | Actinic keratosis | Data published in the The New England Journal of Medicine showed complete clearance in 44% and 54% of patients treated with Klisyri compared to 5% and 13% of patients treated with vehicle in the KX01-AK-003 and KX01-AK-004 studies, respectively (p<0.0001 for both)< td> | 2/11/21 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream (ARQ-151) | Phosphodiesterase type 4 inhibitor | Plaque psoriasis | In the DERMIS-1 study, roflumilast cream 0.3% produced an Investigator Global Assessment score of clear or almost clear with at least a 2-grade improvement from baseline in 42.4% of patients compared to a vehicle rate of 6.1% (p<0.0001); in dermis-2 an iga success rate was 37.5% for roflumilast compared to a vehicle of 6.9% (p<0.0001)< td> | 2/1/21 | Dermatologic |
| Bristol Myers Squibb Co., of New York | Deucravacitinib | Oral, selective TYK2 inhibitor | Moderate to severe plaque psoriasis | Results showed Poetyk PSO-2 trial met both co-primary endpoints vs. placebo, with significantly more patients achieving Psoriasis Area and Severity Index and a static Physician's Global Assessment score of clear or almost clear after 16 weeks of treatment; secondary endpoints also met, including superiority to Otezla (apremilast) | 2/2/21 | Dermatologic |
| Dermavant Sciences Inc., of Long Beach, Calif., subsidiary of Roivant Sciences Ltd., of Basel, Switzerland | Tapinarof | Aryl hydrocarbon receptor modulator | Plaque psoriasis | Interim analysis showed 57.3% (298/520) who entered long-term open-label safety study, Psoaring 3, with PGA score ? 2 achieved PGA score of 0 or 1, suggesting increased therapeutic effect beyond 12-week treatment periods in pivotal Psoaring 1 and 2 studies; 39.2% (299/763) included in interim analysis achieved complete disease clearance (PGA score = 0); discontinuation rate at interim analysis due to adverse events was 5.8%, consistent with other pivotal trials | 2/18/21 | Dermatologic |
| Amicus Therapeutics Inc., of Philadelphia | AT-GAA (cipaglucosidase alfa and miglustat) | Enzyme replacement therapy + alpha-glucosidase stimulator | Late-onset Pompe disease | In the Propel study, average 6-minute walk distance improved by 20.8 meters from baseline to week 52 for the 85 patients taking AT-GAA (n=85) compared to an improvement of 7.2 meters for the 37 patients treated with alglucosidase alfa (p=0.072); mean change in percent-predicted FVC at 52 weeks was -0.9 for AT-GAA and -4.0 for alglucosidase alfa (p=0.023) | 2/11/21 | Endocrine/Metabolic |
| Diurnal Group plc, of Cardiff, U.K. | Chronocort (hydrocortisone modified-release hard capsules) | Corticosteroid agonist | Congenital adrenal hyperplasia | Results published in the Journal of Clinical Endocrinology and Metabolism found that although the standard-deviation-score-focused primary endpoint was missed, Chronocort improved morning and early afternoon biochemical control over standard glucocorticoid therapy; in the safety extension study, biochemical control was sustained for 18 months on median hydrocortisone doses in the range recommended for cortisol replacement therapy and lower than glucocorticoid doses normally used in treatment of CAH | 2/2/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | Dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist | Type 2 diabetes | At 52 weeks in the Surpass-3 study, treatment with 5 mg, 10 mg and 15 mg of tirzepatide reduced A1C by 1.93%, 2.20% and 2.37%, respectively, compared to a reduction of 1.34% for insulin degludec; weight loss for tirzepatide was also statistically significant compared to insulin degludec; at 40 weeks in the Surpass-5 study, treatment with 5 mg, 10 mg and 15 mg of tirzepatide reduced A1C by 2.23%, 2.59% and 2.59%, respectively, compared to a reduction of 0.93% for placebo; weight loss for tirzepatide was also statistically significant in the study | 2/17/21 | Endocrine/Metabolic |
| I-Mab, of Shanghai | Eftansomatropin alfa (TJ-101) | Long-acting recombinant human growth hormone | Pediatric growth hormone deficiency | Treated first of 165 patients in the noninferiority Taller study comparing eftansomatropin alfa to Norditropin (somatropin, Novo Nordisk A/S) | 2/25/21 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel | Pegunigalsidase alfa (PRX–102) | Alpha-galactosidase stimulator | Fabry disease | Final results from Bridge trial showed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR), in both male and female patients who were switched from agalsidase alfa to pegunigalsidase alfa; mean annualized eGFR slope of participants improved from -5.90 mL/min/1.73m2/year while on agalsidase alfa to -1.19 mL/min/1.73m2/year on pegunigalsidase alfa in all patients | 2/10/21 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, unit of Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa (PRX-102) | Alpha-galactosidase stimulator | Fabry disease | Top-line data from phase III Bright switch-over trial showed 2 mg/kg of study drug every 4 weeks maintained stable clinical presentation in adults and no new participants developed treatment-induced anti-drug antibodies following switch | 2/23/21 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Upadacitinib | Oral JAK inhibitor | Ulcerative colitis | U-Accomplish induction study met primary endpoint of clinical remission, per Adapted Mayo Score and all ranked secondary endpoint; 33% of patients on upadacitinib achieved clinical remission at week 8 vs. 4% of patients on placebo (p<0.001)< td> | 2/22/21 | Gastrointestinal |
| Arena Pharmaceuticals Inc., of San Diego | Etrasimod | Once-daily, oral S1P modulator | Moderately to severely active ulcerative colitis | Completed enrollment in Elevate UC 52 study, with 433 participants; top-line data expected in the first quarter of 2022 | 2/2/21 | Gastrointestinal |
| Infant Bacterial Therapeutics AB, of Stockholm | IBP-9414 | Contains active substance Lactobacillus reuteri | Prevention of necrotizing enterocolitis and improvement of feeding tolerance in premature infants | Reached milestone after recruiting 300 premature infants to ongoing study | 2/10/21 | Gastrointestinal |
| Vectivbio Holding AG, of Basel, Switzerland | Apraglutide | Glucagon-like peptide 2 receptor agonist | Short bowel syndrome | First participant dosed in pivotal Stars trial in people with intestinal failure | 2/3/21 | Gastrointestinal |
| Calliditas Therapeutics AB, of Stockholm | Nefecon | Oral formulation of budesonide | IgA nephropathy | First patient dosed in global open-label extension study, designed to offer 9-month treatment to all qualifying patients who completed Nefigard study | 2/4/21 | Genitourinary/Sexual Function |
| Travere Therapeutics Inc., of San Diego | Sparsentan | Dual-acting antagonist of endothelin type A and angiotensin II type 1 receptors | Focal segmental glomerulosclerosis | Achieved prespecified interim FSGS partial remission of proteinuria endpoint after 36 weeks of treatment; drug showed statistically significant response on FPRE compared to the active control, irbesartan (p=0.009); based on data, company intends to pursue submissions for accelerated approval | 2/1/21 | Genitourinary/Sexual Function |
| Gamida Cell Ltd., of Boston | Omidubicel | Cell therapy | Patients with high-risk hematologic malignancies undergoing bone marrow transplant | Results showed faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital vs. standard umbilical cord transplant; median time to neutrophil engraftment was 12 days for patients randomized to omidubicel vs. 22 days for the comparator group (p<0.001), meeting primary endpoint< td> | 2/9/21 | Hematologic |
| Octapharma AG, OF Lachen, Switzerland | Nuwiq (simoctocog alfa) | Factor VIII agonist | Hemophilia A | Final results from NuProtect study on agent's immunogenicity in 105 previously untreated people with severe disease, published in Thrombosis and Haemostasis, showed 16.2% (n=17) developed high-titer inhibitors; 10.5% (n=11) developed low-titer inhibitors, including 5 that were transient; and 26.7% (n=28) developed any inhibitor | 2/22/21 | Hematologic |
| Aimmune Therapeutics, a unit of Société des Produits Nestlé SA, of Vevey, Switzerland | Palforzia | Peanut (arachis hypogaea) allergen powder-dnfp | Peanut allergy | Pooled data from the Palisade, Ramses and Artemis and 3 open-label extension studies showed 3 out of 4 patients were able to achieve the 300-mg maintenance dose; over an approximately 3.5-year treatment period, 14 participants (1.2%) experienced a treatment-related severe systemic allergic reaction | 2/25/21 | Immune |
| Evolve Biologics Inc., of Mississauga, Ontario | Plasmacap | Intravenous immunoglobulin | Primary immune deficiency disease | Completed its adult and pediatric study, showing no acute serious bacterial infections, meeting primary endpoint of <1 acute serious bacterial infection per patient-year; bla expected to be submitted fda and health canada< td> | 2/9/21 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | AZD-1222 | COVID-19 spike glycoprotein modulator | COVID-19 infection | Primary analysis of trials, published as preprint in The Lancet, showed efficacy of 76% (CI: 59% to 86%) after first dose with protection maintained to second dose; with inter-dose interval of 12 weeks or more, efficacy increased to 82% (CI: 63%, 92%); positive PCR readings were reduced by 67% (CI: 49%, 78%) after single dose and 50% (CI: 38% to 59%) after 2-dose regimen | 2/3/21 | Infection |
| Corvus Pharmaceuticals Inc., of Burlingame, Calif. | CPI-006 | Humanized monoclonal antibody designed to bind to and activate B cells | COVID-19 | Initiated trial in hospitalized patients; study expected to enroll about 1,000 patients at sites in North America, Europe, South Africa and Latin America | 2/4/21 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Vemlidy (tenofovir alafenamide 25 mg) | Nucleoside reverse transcriptase inhibitor | Chronic hepatitis B virus | Findings from 2 subanalyses demonstrated continued efficacy and improved safety vs. tenofovir disoproxil fumarate (TDF) in Asian patients; first study showed, over 240 weeks, patients in Vemlidy group had lower median declines in kidney function, and increases in bone mineral density were observed after switching from TDF to Vemlidy; results from second substudy showed that in virologically suppressed patients of Asian ethnicity with CHB on long-term TDF, switching to Vemlidy demonstrated maintenance of a high rate of viral suppression in 94% (n=376), along with continued high rates of normal ALT and improved markers of renal and bone health | 2/4/21 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide | Androgen receptor antagonist | COVID-19 | Completed recruitment of 588 hospitalized patients in Brazil; data expected to be available in March 2021 | 2/22/21 | Infection |
| Novan Inc., of Morrisville, N.C. | SB-206 | Antiviral gel | Molluscum contagiosum | Completed enrollment in the B-Simple4 study; top-line results expected before the end of the second quarter of 2021 | 2/1/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Recombinant protein-based vaccine | COVID-19 | Completed enrollment in Prevent-19 pivotal study in U.S. and Mexico | 2/22/21 | Infection |
| Polypid Ltd., of Petah, Tikva, Israel | D-PLEX100 | Prolonged-release broad-spectrum antibiotic doxycycline | Post-abdominal surgery incisional infections | Enrolled 100th patient in the Shield I study; adaptive study is expected to enroll between 616 and 900 patients; top-line data expected by the end of 2021 | 2/16/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Regen-Cov (casirivimab/imdevimab) | Monoclonal antibodies targeting SARS-CoV-2 | Non-hospitalized COVID-19 | Independent data monitoring committee recommended stopping enrollment into the placebo group due to clear clinical efficacy on reducing the rate of hospitalization and death with both the 1,200-mg and 2,400-mg doses; study will continue to enroll patients in both dose groups | 2/25/21 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Xanthine oxidase inhibitor | Mild to moderate COVID-19 | Plans to expand study from 14 clinical sites to up to 50 sites | 2/26/21 | Infection |
| Sinovac Biotech Ltd., of Beijing | Coronavac | SARS-CoV-2 vaccine | COVID-19 prophylaxis | Of the 12,396 health care workers in Brazil and Turkey in the study, efficacy rate against diseases caused by COVID-19 starting 14 days after the second dose was 50.65% for all cases, 83.7% for cases requiring medical treatment and 100% for hospitalized, severe and fatal cases; combined results of health care workers and the general population in Turkey showed efficacy rate for COVID-19 prevention was 91.25% after 14 days following the 2-dose vaccination | 2/5/21 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-620 (maribavir) | Anti-cytomegalovirus compound | Transplant recipients with refractory, with or without resistance, cytomegalovirus infection/disease | In the Solstice study, 55.7% of patients taking maribavir had cytomegalovirus viremia clearance at week 8 compared to 23.9% of those given standard of care (1 or a combination of ganciclovir, valganciclovir, foscarnet or cidofovir) (p<0.001)< td> | 2/12/21 | Infection |
| The Russian Direct Investment Fund, of Moscow | Sputnik V | Vaccine | COVID-19 | Data published in The Lancet showed efficacy of 91.6%; among cases analyzed, more than 98% of volunteers developed humoral immune response and 100% had cellular immune response; level of neutralizing antibodies was 1.3-1.5 times higher than in patients who recovered from COVID-19 | 2/2/21 | Infection |
| Tiziana Life Sciences plc, of New York and London | Foralumab | Anti-CD3 monoclonal antibody | COVID-19 | Data from exploratory study in Brazil testing drug alone or in combination with dexamethasone showed 80% lung CT scan improvement in foralumab-only group and 75% in combination group vs. 43% for control; cytokine IL-6 reduction was 69%, 41% and 37%, respectively, while C-reactive protein reduction as 85%, 55% and 40%, respectively | 2/2/21 | Infection |
| University of Oxford, of Oxford, U.K. | Almitrine | Respiratory stimulant | COVID-19 | Started study to test whether oral drug, administered over a 7-day period, is effective in reducing the need for other forms of ventilatory support | 2/10/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1553 | Live-attenuated, single-dose vaccine | Chikungunya | Initiated clinical lot-to-lot consistency trial to run in parallel to ongoing pivotal phase III study | 2/22/21 | Infection |
| Italfarmaco Group, of Milan, Italy | Givinostat | HDAC inhibitor | Duchenne muscular dystrophy | Ongoing, long-term phase II extension study continue to show delay in disease progression in boys at 7-year follow-up period; yearly rate of change of respiratory parameters such as Forced Vital Capacity % Predicted and Peak Expiratory Flow % predicted is -1.7% and 0% in contrast to the 4 to 6% yearly rate of decline in those parameters as demonstrated in natural history studies | 2/22/21 | Musculoskeletal |
| Mesoblast Ltd., of New York | Rexlemestrocel-L | Allogeneic mesenchymal precursor cell therapy | Chronic low back pain due to degenerative disc disease refractory to conventional treatments | Rexlemestrocel-L plus hyaluronic acid reduced pain on the VAS scale by -27.6 at 12 months and -26 at 24 months from a baseline mean VAS of 60.4 (p=0.014 and p=0.036, respectively, compared with saline-treated controls) | 2/11/21 | Musculoskeletal |
| PTC Therapeutics Inc., of South Plainfield, N.J. | Translarna (ataluren) | Regulator of nonsense transcripts modulator | Duchenne muscular dystrophy | Study 045 fell short of statistical significance on primary endpoint of change in dystrophin expression (6.56% increase as measured by electrochemiluminescence in the intent-to-treat population; p=0.24) | 2/5/21 | Musculoskeletal |
| Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Tokyo | Lecanemab (BAN-2401) | Beta amyloid antagonist | Alzheimer's disease | Target enrollment number increased by about 200 in pivotal Clarity AD trial in people with early disease to ensure robust dataset and mitigate potential impact of missed doses due to COVID-19 pandemic; data readout still expected by September 2022 | 2/3/21 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores normal shape and function of altered filamin A | Mild to moderate Alzheimer’s disease | Following an end-of-phase-II meeting with the FDA, company plans to start its phase III program in the second half of 2021; 1 study will evaluate disease-modifying effects over 18 months; the other study will evaluate symptomatic improvement over 6 months | 2/8/21 | Neurology/Psychiatric |
| Otsuka Pharmaceutical Development & Commercialization Inc., of Princeton, N.J., and Click Therapeutics Inc., of New York | CT-152 | Digital therapeutics | Major depressive disorder | Started the fully-remote study of up to 540 patients; 10-week study will measure the change from baseline in the Montgomery–Åsberg Depression Rating Scale total score | 2/24/21 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Fixed-dose combination of baclofen, naltrexone and sorbitol formulated as oral solution | Charcot-Marie-Tooth type 1A | On track to begin first patient/first dose in pivotal Premier study before March 31, 2021 | 2/2/21 | Neurology/Psychiatric |
| Zosano Pharma Corp., of Fremont, Calif. | Qtrypta | Zolmitriptan-coated titanium microneedles | Acute migraine | Post-hoc retrospective analyses of the Zotrip study showed 74% of patients treated with Qtrypta 3.8 mg who had pain relief at 30 minutes were pain free at 2 hours and 100% of patients who were pain free at 30 minutes were pain free at 2 hours; for placebo 35% of those with pain relief at 30 minutes were pain free at 2 hours and 50% of those who were pain free at 30 minutes were pain free at 2 hours | 2/1/21 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution | Small-molecule immune-modulating covalent inhibitor of RASP | Dry eye disease | Finalized design of Tranquility trial; ocular redness over 90 minutes in dry eye chamber will be primary endpoint; study to enroll about 150 patients per arm | 2/4/21 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Soluble form of vascular endothelial growth factor receptor 3 | Neovascular (wet) age-related macular degeneration | Finalized protocols for the Shore and Coast studies; 990-patient Shore study will compare OPT-302 and Lucentis (ranibizumab, Roche Holding AG) every 4 weeks to OPT-302 every 8 weeks (after loading doses) and Lucentis every 4 weeks to the control group of Lucentis every 4 weeks; 990-patient Coast study will compare OPT-302 every 4 weeks plus Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) every 8 weeks (after loading doses) to OPT-302 and Eylea every 8 weeks (after loading doses) to the control of Eylea every 8 weeks (after loading doses); primary endpoint for both trials is the mean change in Best Corrected Visual Acuity from baseline to week 52 | 2/24/21 | Ocular |
| Outlook Therapeutics Inc., of Monmouth Junction, N.J. | ONS-5010/Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Last patient completed the final visit in the Norse Three open-label safety study; data expected in the second quarter of 2021 | 2/11/21 | Ocular |
| Regentree LLC, a joint venture of Regenerx Biopharmaceuticals Inc., of Rockville, Md., and Gtreebnt Co. Ltd., of Seongnam, South Korea | RGN-259 | Thymosin beta 4 ligand | Dry eye syndrome | Database locked for the Arise-3 study | 2/24/21 | Ocular |
| Roche Holding AG, of Basel, Switzerland | Faricimab | Bispecific antibody targeting angiopoietin-2 and VEGF-A | Diabetic macular edema | Average vision gains from baseline was +11.6 and +10.8 eye chart letters for patients receiving faricimab at personalized treatment intervals (PTI), +10.7 and +11.8 for patients taking faricimab every 2 months and +10.9 and +10.3 letters for those taking aflibercept in the Yosemite and Rhine studies, respectively; of faricimab PTI patients, 52.8% in Yosemite and 51% in Rhine achieved 4-month dosing at 1 year | 2/12/21 | Ocular |
| Roche Holding AG, of Basel, Switzerland | Faricimab | Bispecific antibody targeting angiopoietin-2 and VEGF-A | Age-related macular degeneration | Average vision gains from baseline in the faricimab arms were +5.8 and +6.6 letters compared to +5.1 and +6.6 letters in the aflibercept arms in the Tenaya and Lucerne studies, respectively; 45.7% of patients in Tenaya and 44.9% in Lucerne were able to be treated every 4 months in the first year | 2/12/21 | Ocular |
| Otonomy Inc., of San Diego | Otividex | Glucocorticoid receptor agonist | Ménière’s disease | Trial did not achieve primary endpoint, defined as count of definitive vertigo days in month 3 vs. placebo for intent-to-treat population (p=0.312) using Negative Binomial Model; analysis did achieve statistical significance for per-protocol population (p=0.031) | 2/22/21 | Other/Miscellaneous |
| Amgen Inc., of Thousand Oaks, Calif., and Astrazeneca plc, of Cambridge, U.K. | Tezepelumab | Monoclonal antibody targeting thymic stromal lymphopoietin | Severe asthma | In the Navigator study, tezepelumab plus standard of care (SOC) produced a 56% reduction in the annualized asthma exacerbation rate (AAER) compared to SOC alone (p<0.001); 150 300 in patients with baseline eosinophil counts less than cells ?l, aaer was reduced by 41% (p<0.001); 39%; of ?l or greater, 70%< td> | 2/26/21 | Respiratory |
| Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif. | Ziritaxestat | Autotaxin inhibitor | Idiopathic pulmonary fibrosis | Companies decided to halt Isabela studies based on recommendation of independent data monitoring committee, which concluded after regular review of unblinded data that risk-benefit profile no longer supported continuing these studies | 2/10/21 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 (repurposed ondansetron) | 5-HT 3 receptor antagonist | Alcohol use disorder | Pivotal Onward trial reached 50% enrollment, with 86% retention rate | 2/25/21 | Toxicity and Intoxication |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor | Metastatic castrate-resistant prostate cancer | Results from study AB12003 are expected in April 2021 | 3/31/21 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Blincyto (blinatumomab) | CD19-directed CD3 T-cell bispecific T-cell engager | High-risk first-relapse B-cell precursor acute lymphoblastic leukemia | Data published in The Journal of the American Medical Association showed 69% of patients treated with Blincyto were alive and event-free compared with 43% of patients treated with chemotherapy; 93% of patients treated with Blincyto who had minimal residual disease (MRD) at baseline achieved MRD-negative remission compared with 24% of patients treated with chemotherapy; 36-month overall survival estimate was 81.1% for Blincyto compared to 55.8% in the chemotherapy group; median OS has not been met | 3/2/21 | Cancer |
| Astellas Pharma Inc., of Tokyo | Xospata (gilteritinib) | FLT3 inhibitor | Relapsed or refractory FLT3 mutation-positive acute myeloid leukemia | The Commodore study met its primary endpoint of overall survival for Xospata compared to chemotherapy at the planned interim analysis; enrollment of the study has stopped; data to be submitted to a peer-reviewed journal and/or scientific congress | 3/30/21 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | Rubraca (rucaparib) | Inhibitor of PARP1, PARP2 and PARP3 | Advanced, relapsed ovarian cancer and a deleterious BRCA mutation after 2 or more lines of chemotherapy | In the Ariel 4 study, median progression-free survival was 7.4 months for Rubraca compared to 5.7 months for chemotherapy (HR-0.64, p=0.001) | 3/19/21 | Cancer |
| Eirgenix Inc., of Taipei, Taiwan | EG-12014 (trastuzumab biosimilar) | Erbb2 tyrosine kinase receptor inhibitor | Breast cancer | Trial met primary endpoint, showing equivalent efficacy to Herceptin (Roche Holding AG/Genentech Inc.) in pathologic complete response; preparations to begin for submissions of BLA to FDA, MAA to EMA and NDA to TFDA | 3/24/21 | Cancer |
| Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK 4/6 dual inhibitor | Breast cancer | Patient-reported outcomes in trial combining use with standard adjuvant endocrine therapy in patients with HR+, HER2-negative high-risk early stage disease showed, in 1 analysis, data indicated that most patients (70%-75%) in both arms reported being bothered "a little bit" or "not at all" by treatment-related side effects | 3/17/21 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Previously untreated advanced intermediate- or poor-risk renal cell carcinoma | Completed enrollment in the Cosmic-313 study comparing Cabometyx plus Opdivo (nivolumab, Bristol Myers Squibb Co) and Yervoy (ipilimumab, BMS) to Opdivo plus Yervoy; primary endpoint of the trial is progression-free survival; additional endpoints include overall survival and objective response rate | 3/30/21 | Cancer |
| Gamida Cell Ltd., of Boston | Omidubicel | Cell therapy | High-risk hematologic malignancies undergoing a bone marrow transplant | Median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for standard umbilical cord blood transplant (p < 0.001); all 3 secondary endpoints demonstrated a statistically significant improvement for omidubicel compared to umbilical cord blood transplant | 3/15/21 | Cancer |
| Genentech, unit of Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | PD-L1 inhibitor | Non-small-cell lung cancer | At interim analysis of IMpower010 study, Tecentriq met primary endpoint of disease-free survival vs. best supportive care, with statistically significant improvement as adjuvant therapy following surgery and chemotherapy in stage II-IIIA disease; data on intent-to-treat population did not cross threshold and overall survival data remained immature at time of analysis | 3/22/21 | Cancer |
| Guerbet SA, of Villepinte, France | Gadopiclenol | MRI imaging agent | Cancer | 2 studies enrolling combined 560 participants met primary endpoints, showing diagnostic benefit at 0.05 mmol/kg during MRI based on superiority of exam vs. exam with no contrast agent and noninferiority to gadobutrol at 0.1 mmol/kg to visualize and detect lesions in CNS and other body areas | 3/24/21 | Cancer |
| Idera Pharmaceuticals Inc., of Exton, Pa. | Tilsotolimod | Toll-like receptor 9 agonist | Anti-PD-1 refractory advanced melanoma | In the Illuminate-301 study, tilsotolimod plus Yervoy (ipilimumab, Bristol Myers Squibb Co.) produced an objective response rate of 8.8% compared to 8.6% for Yervoy alone; disease control rate was 34.5% for the combination and 27.2% for Yervoy alone | 3/18/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J., and Eisai Co., Ltd., of Tokyo | Lenvima (lenvatinib) and Keytruda (pembrolizumab) | Receptor tyrosine kinase inhibitor and monoclonal antibody targeting PD-1 | Advanced, metastatic or recurrent endometrial cancer following 1 prior platinum-based regimen | In Study 309/Keynote-775, the combination reduced the risk of disease progression or death by 44% (p<0.0001), with a median progression-free survival of 7.2 months compared to 3.8 for chemotherapy; overall the combination was 18.3 11.4 chemotherapy (p<0.0001)< td> | 3/19/21 | Cancer |
| Natera Inc., of Austin, Texas, and Genentech, of South San Francisco, a unit of Roche Holding AG | Signatura | Companion diagnostic | Muscle-invasive urothelial carcinoma | First patient screened in trial to use personalized molecule residual disease test to identify patients eligible for treatment with Tecentriq (atezolizumab) | 3/10/21 | Cancer |
| Novartis AG, of Basel, Switzerland | Canakinumab (ACZ-885) | IL-1beta inhibitor | Non-small-cell lung cancer | Canopy-2 combination study with docetaxel in 237 adults with locally advanced or metastatic disease following platinum-based chemotherapy and PD-(L)1 inhibitor missed primary endpoint of overall survival; NSCLC trials continue in first-line and adjuvant settings | 3/9/21 | Cancer |
| Novartis AG, of Basel, Switzerland | 177Lu-PSMA-617 | Radioligand therapy targeting PSMA | Progressive PSMA-positive metastatic castration-resistant prostate cancer | The Vision study met both primary endpoints of overall survival and radiographic progression-free survival; data to be presented at an upcoming medical meeting | 3/23/21 | Cancer |
| Orbus Therapeutics Inc., of Palo Alto, Calif. | Eflornithine (repurposed) | Ornithine decarboxylase inhibitor | Anaplastic astrocytoma | Independent data monitoring committee recommended Stellar study continue without modification following preplanned interim futility analysis; full enrollment expected in second half of 2021, with preplanned interim superiority analysis expected in 2022 | 3/2/21 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | CPI-613 (devimistat) | Targets mitochondrial tricarboxylic acid cycle | Acute myeloid leukemia | Pivotal Armada 2000 combination trial with high-dose cytarabine + mitoxantrone vs. cytarabine + mitoxantrone alone in older adults with relapsed/refractory disease met enrollment milestone of 150 participants, toward expected enrollment of 500 | 3/30/21 | Cancer |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Libtayo (cemiplimab) | Monoclonal antibody targeting PD-1 | Recurrent or metastatic cervical cancer | Study is being stopped early based on the recommendation of the independent data monitoring committee; compared to chemotherapy, Libtayo reduced the risk of death by 31% in the total population (p<0.001), by 27% in patients with squamous cell carcinoma (p="0.003)" and 44% adenocarcinoma (p<0.005)< td> | 3/15/21 | Cancer |
| Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel | Ofranergene obadenovec (VB-111) | CD95 modulator; TNF receptor modulator | Recurrent platinum-resistant ovarian cancer | Based on data from the first 60 patients in the Oval study, authors of an article published in Gynecologic Oncology calculated the CA-125 GCIG response rate was 58% or higher in evaluable patients treated with the drug | 3/4/21 | Cancer |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Baricitinib | JAK1/JAK2 inhibitor | Alopecia areata | In Brave-AA2 study, once-daily 2-mg and 4-mg doses in adults with severe disease met primary efficacy endpoint at week 36, showing statistically significant improvement in scalp hair regrowth vs. placebo | 3/3/21 | Dermatologic |
| Krystal Biotech Inc., of Pittsburgh | Beremagene geperpavec (B-VEC) | COL7A1 gene stimulator | Epidermolysis bullosa dystrophica | Trial fully enrolled 31 participants; top-line results expected in fourth quarter of 2021 | 3/30/21 | Dermatologic |
| Sun Pharmaceutical Industries Inc. USA (Sun Pharma), of Princeton, N.J. | Ilumya (tildrakizumab-asmn) | IL-23 antagonist | Psoriasis | 5-year results based on a pooled analysis of Resurface 1 and 2 extension studies, published in British Journal of Dermatology, showed study drug maintained consistent, high level of skin clearance with no new safety signals in people with moderate to severe disease | 3/11/21 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | RNAi targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | Data from the Illuminate-A study published in The New England Journal of Medicine showed lumasiran reduced 24-hour urinary oxalate excretion from baseline to month 6 by 53.5% relative to placebo; 84% of patients taking lumasiran achieved normal or near-normal levels of urinary oxalate compared to 0% for placebo | 3/31/21 | Endocrine/Metabolic |
| Bluebird Bio Inc., of Cambridge, Mass. | Elivaldogene autotemcel (Lenti-D) | ABCD1 gene stimulator | Adrenoleukodystrophy | With 19 participants evaluable for safety in ALD-104 study, 17 achieved neutrophil engraftment and 15 had platelet engraftment, with no events of acute or chronic GVHD and no reports of graft failure or rejection, insertional oncogenesis or replication competent lentivirus | 3/15/21 | Endocrine/Metabolic |
| Diamyd Medical AB, of Stockholm | Diamyd | Diabetes vaccine | Newly diagnosed type 1 diabetes with HLA DR3-DQ2 haplotype | Plans to run a study in 2021 with the co-primary endpoints of change in endogenous insulin production (measured as stimulated C-peptide) and change in HbA1c, both measured at 24 months from baseline | 3/4/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | Dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist | Type 2 diabetes | In the Surpass-2 study, 5-mg and 15-mg doses of tirzepatide reduced A1C by 2.09% and 2.46%, respectively, compared to a reduction of 1.86% for semaglutide; 5-mg and 15-mg doses of tirzepatide reduced body weight by 7.8 kg (17.2 lb., 8.5%) and 12.4 kg (27.3 lb., 13.1%), respectively, compared to 6.2 kg (13.7 lb., 6.7%) for semaglutide; 51% of patients taking the 15-mg dose achieved an A1C of less than 5.7%, compared to 20% of patients taking semaglutide | 3/4/21 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | ORA-D-013-1 study enrolled and randomized 25% of planned 675 participants; full enrollment expected by year-end 2021 | 3/16/21 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | Screened first patient for the ORA-D-013-2 study; primary endpoint is A1c over a 26-week treatment period; secondary endpoint is maintaining A1c over 52 weeks; enrollment in the concurrent ORA-D-013-1 study has surpassed 25% | 3/23/21 | Endocrine/Metabolic |
| Recordati SpA, of Milan, Italy | Isturisa (osilodrostat) | 11beta-hydroxylase inhibitor | Cushing’s disease | In the Linc 4 study, after 12 weeks of treatment, 77% of patients treated with Isturisa achieved normal mean urinary free cortisol (mUFC) compared to 8% of patients taking placebo (p<0.0001); at week 36, 81% of patients treated with isturisa achieved normal mufc< td> | 3/23/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Mirikizumab | Monoclonal antibody targeting p19 subunit of interleukin 23 | Moderate to severe ulcerative colitis | The Lucent-1 study met the primary endpoint of clinical remission at week 12 compared to placebo (p<0.0001); secondary endpoints, including reduced bowel urgency, clinical response, endoscopic remission, symptomatic remission and improvement in histologic inflammation, were also statistically significant; full data to be disclosed at a future congress or publication< td> | 3/16/21 | Gastrointestinal |
| Chinook Therapeutics Inc., of Vancouver, British Columbia | Atrasentan | Endothelin A receptor inhibitor | IgA nephropathy | Enrolled first of approximately 320 patients in the Align study; primary endpoint is urine protein-to-creatinine ratio from baseline to 24 weeks; secondary endpoints include eGFR, safety and tolerability, as well as quality of life; top-line data expected in 2023 | 3/16/21 | Genitourinary/Sexual Function |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | VGX-3100 | Dual HPV E6/HPV E7 protein modulator | Cervical dysplasia | Pivotal Reveal 1 trial met primary endpoint of histopathological regression of high-grade squamous intraepithelial lesions + virologic clearance of HPV-16 and/or HPV-18 at week 36 in mITT population (n=193); 23.7% (31/131) responded in treatment group vs. 11.3% (7/62) for placebo (p=0.022) | 3/1/21 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Dublin | Terlipressin | Vasopressin analogue selective for V1 receptors | Hepatorenal syndrome (HRS) type 1 | Data from the Confirm study published in The New England Journal of Medicine showed 32% of patients who received terlipressin had verified reversal of HRS, compared to 17% of the placebo group (p=0.006); HRS reversal occurred in 39% of the terlipressin group and 18% of the placebo group (p<0.001); 30 hrs reversal without renal replacement therapy by day occurred in 34% of the terlipressin group and 17% placebo (p="0.001);" among patients with systemic inflammatory response syndrome 37% 6% (p<0.001)< td> | 3/4/21 | Genitourinary/Sexual Function |
| Mayne Pharma Group Ltd., of Adelaide, Australia | Nextstellis (drospirenone and estetrol) | Estrogen receptor modulator | Contraception | Data showed treatment resulted in limited changes in endocrine markers, including lower increases in hormone binding globulins, compared with combined oral contraceptives based on ethinyl-estradiol | 3/8/21 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland, and Pfizer Inc., of New York | Relugolix + estradiol + norethindrone acetate | GNRH receptor antagonist + estradiol agonist + progesterone receptor agonist | Uterine fibroids | Liberty randomized withdrawal study assessing treatment for up to 2 years met primary endpoint, with 78.4% of women who continued on combination therapy achieving sustained responder rate of menstrual blood loss < 80 mL through week 76 vs. 15.1% who discontinued treatment and initiated placebo at week 52 (p<0.0001); 3 all key secondary endpoints also met< td> | 3/24/21 | Genitourinary/Sexual Function |
| Swedish Orphan Biovitrum AB, of Stockholm | Avatrombopag | Thrombopoietin receptor agonist | Immune thrombocytopenia | Dosed first patient in pediatric trial | 3/12/21 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm, and Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Targeted C3 therapy | Paroxysmal nocturnal hemoglobinuria | Results from Pegasus study published in The New England Journal of Medicine demonstrated superiority in improving hemoglobin levels vs. eculizumab in adults at 16 weeks who had persistent anemia following treatment with eculizumab; superiority in significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.001); 16 85% of pegcetacoplan-treated patients were transfusion-free over weeks vs. 15% eculizumab-treated patients< td> | 3/18/21 | Hematologic |
| Uniqure NV, of Amsterdam | Etranacogene dezaparvovec (AMT-061) | F9 gene stimulator; factor IX modulator | Hemophilia B | Independent investigation of hepatocellular carcinoma in participant in pivotal Hope-B trial showed no evidence that AAV vector delivered in study played pathogenic role; participant had abnormalities on chromosomes 1 and 8, commonly associated with HCC, and mutations in other potentially oncogenic genes along with multiple risk factors for HCC; data shared with FDA with respect to status of clinical hold | 3/29/21 | Hematologic |
| Corbus Pharmaceuticals Inc., of Norwood, Mass. | Lenabasum | Cannabinoid receptor type 2 agonist | Diffuse cutaneous systemic sclerosis | Sponsor terminated open-label extension of Resolve-1 trial | 3/29/21 | Immune |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting p19 subunit of interleukin 23 | Psoriatic arthritis | At week 100 in the Discover-2a study, 59% of patients taking Tremfya every 4 weeks and 53% of patients taking the drug every 8 weeks had PASI 100; 76% and 74% of patients taking the drug every 4 and 8 weeks, respectively, achieved ACR 20 | 3/16/21 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | AZD-1222 | COVID-19 spike glycoprotein modulator | COVID-19 infection | U.S. vaccine trial showed statistically significant efficacy of 79% at preventing symptomatic infection and 100% at preventing severe disease and hospitalization, based on interim analysis of 32,449 participants who accrued 141 symptomatic cases; primary analysis will be submitted to FDA for EUA | 3/22/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | AZD-1222 | COVID-19 spike glycoprotein modulator | COVID-19 infection | Primary analysis showed 76% efficacy against symptomatic infection, 100% efficacy against severe or critical disease and hospitalization, and 85% efficacy against symptomatic infection in people 65 and older | 3/25/21 | Infection |
| Bharat Biotech International Ltd., of Hyderabad, India, and Ocugen Inc., of Malvern, Pa. | Covaxin | Virion inactivated COVID-19 vaccine | COVID-19 prophylaxis | At the first interim analysis, there were 36 cases of COVID-19 in the placebo group and 7 cases in the group who received Covaxin, resulting in a point estimate of vaccine efficacy of 80.6% | 3/3/21 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Vyrologix (leronlimab, PRO-140) | CCR5 antagonist | Severe to critically ill COVID-19 | In the critically ill population of the CD12 study, leronlimab plus standard of care decreased mortality at day 14 by 82% (p=0.0233) and improved the 7-point ordinal scale at day 14 by 400% (p=0.021) | 3/30/21 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab | CCR5 antagonist | COVID-19 infection | In people with severe to critical infection, trial showed 24% reduction vs. placebo in primary endpoint of all-cause mortality; average hospital length of stay was reduced by 6 days for study drug + standard of care vs. SOC only (p=0.005) | 3/8/21 | Infection |
| Eli Lilly and Co., of Indianapolis | Bamlanivimab (LY-CoV555) and etesevimab (LY-CoV-16) | Therapeutic antibody; COVID-19 spike glycoprotein modulator; COVID-19 spike glycoprotein inhibitor | COVID-19 | New data from Blaze-1 study showed combination significantly reduced COVID-19-related hospitalizations and death in high-risk patients recently diagnosed; there were 4 events in patients taking bamlanivimab with etesevimab and 15 events in patients taking placebo, representing an 87% risk reduction (p<0.0001)< td> | 3/10/21 | Infection |
| Genentech, unit of Roche Holding AG, of Basel, Switzerland, and Gilead Sciences Inc., of Foster City, Calif. | Actemra (tocilizumab) + Veklury (remdesivir) | IL-6 receptor antagonist + COVID-19 nonstructural protein 8 modulator/replicase polyprotein 1ab inhibitor | COVID-19 infection | In Remdacta trial, combination missed primary endpoint of improved time to hospital discharge for severe COVID-19 pneumonia and key secondary endpoints vs. Veklury alone | 3/10/21 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg) | HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor | HIV-1 | Long-term data from open-label extensions of Study 1489 and Study 1490 showed sustained efficacy and safety profile and no treatment-emergent resistance in treatment-naïve adults; in both studies, >98% of participants who remained on study achieved and maintained an undetectable viral load (HIV-1 RNA <50 4 144 copies ml) through years of follow-up; in a subgroup analysis participants with transmitted-drug resistance (tdr, n="248)" based on retrospective sequencing baseline samples, biktarvy achieved comparably high levels durable viral suppression weeks among and without tdr (98% vs. 97%; as treated analysis)< td> | 3/6/21 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Monoclonal antibody targeting granulocyte macrophage-colony stimulating factor | Hospitalized COVID-19 | Lenzilumab plus standard of care improved the likelihood of survival without need for invasive mechanical ventilation by 54% compared to standard of care alone (p=0.0365); mortality was 9.6% for lenzilumab and 13.9% for placebo (p=0.2287) | 3/29/21 | Infection |
| Incyte Corp., of Wilmington, Del. | Jakafi (ruxolitinib) | JAK1/JAK2 inhibitor | COVID-19-associated acute respiratory distress syndrome | In the Devent study, overall survival through day 29 was 55.2% for Jakafi compared to 74.3% for placebo (p=0.0280) in the 5-mg arm and 51.8% for Jakafi compared to 69.6% for placebo (p=0.0292) in the 15-mg arm | 3/18/21 | Infection |
| Incyte Corp., of Wilmington, Del. | Jakafi (ruxolitinib) | JAK1/2 inhibitor | COVID-19-associated acute respiratory distress syndrome | Terminated Ruxcovid-Devent study in patients who require mechanical ventilation | 3/15/21 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide | Androgen receptor antagonist | COVID-19 infection | Investigator-initiated trial in Brazil met primary endpoint at day 14 with reduction of 4.01 in WHO COVID-19 ordinal scale, from baseline of 5.663 to 1.653 for study drug, vs. reduction of 0.25 from baseline of 5.618 to 5.368 for standard of care (p<0.0001); 5 9 14 trial showed 92% reduction in mortality risk (3.7% vs 47.6%, respectively) and shortened median hospital length stay by days (median of vs. days, respectively)< td> | 3/11/21 | Infection |
| Marinomed Biotech AG, of Korneuburg, Austria | Inhaleen (Carragelose-based inhalation solution) | Exo-alpha sialidase inhibitor | COVID-19 infection | First of 204 participants hospitalized with infection dosed; primary endpoint is improvement in clinical status on day 8 vs. placebo | 3/24/21 | Infection |
| Medicago Inc., of Quebec City, and Glaxosmithkline plc, of London | CoVLP (SARS-CoV-2 vaccine) | Virus like particle-based vaccine | COVID-19 infection | Plant-derived vaccine candidate in combination with GSK's pandemic adjuvant advanced into 2-way crossover phase III portion of ongoing phase II/III study, expected to enroll up to 30,000 participants, initially including healthy adults ages 18 to 65 followed by adults >65 years | 3/16/21 | Infection |
| Molecular Partners AG, of Zurich-Schlieren, Switzerland, and Novartis AG, of Basel | Ensovibep (MP-0420) | ACE-2 inhibitor; COVID-19 spike glycoprotein inhibitor | COVID-19 infection | Study drug to be included in NIH-sponsored Activ-3 trial, with ensovibep arm initially to enroll 300 adults hospitalized with mild to moderate infection; primary efficacy endpoint is time from randomization to sustained recovery for 14 days following discharge | 3/15/21 | Infection |
| Nabriva Therapeutics plc, of Dublin | Xenleta (lefamulin) | Semisynthetic pleuromutilin antibiotic | Community-acquired bacterial pneumonia | Journal of Emergency Medicine published ad hoc analysis from Leap 2 trial showing success rates at early clinical response/test of cure (TOC) for outpatients treated with oral lefamulin were similar to those treated with moxifloxacin (91% vs. 89%/90%, respectively), including those with PORT risk class III/IV (89%/91% vs. 88%/91%) and CURB-65 scores of 2 to 3 (87%/90% vs. 82%/88%); TOC success rate was maintained through 30±3 days for both lefamulin (91%) and moxifloxacin (90%) | 3/16/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | COVID-19 spike glycoprotein modulator | COVID-19 | Vaccine showed final efficacy of 96.4% against mild, moderate and severe disease caused by original COVID-19 strain U.K. trial; demonstrated 100% protection against severe disease, including all hospitalization and death | 3/11/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Comirnaty (BNT-162b2, tozinameran) | COVID-19 spike glycoprotein modulator | COVID-19 infection | The New England Journal of Medicine published updated report on neutralizing activity of BNT-162b2-elicited serum from patient samples obtained from pivotal trial, showing effectiveness against SARS-CoV-2 variants originating in U.K. (B.1.1.7), South Africa (B1.351) and Brazil (P.1) | 3/8/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | COVID-19 spike glycoprotein modulator | COVID-19 | Data from trial in adolescents, ages 12-15, with or without prior evidence of SARS-CoV-2 infection, demonstrated 100% efficacy and robust antibody responses, exceeding those recorded earlier in vaccinated participants ages 16-25; vaccine was well-tolerated | 3/31/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Regen-Cov (casirivimab + imdevimab) | Monoclonal antibody targeting SARS-CoV-2 | High-risk non-hospitalized COVID-19 | Drug reduced the risk of hospitalization or death by 70% and 71% at the 1,200-mg and 2,400-mg doses, respectively, compared to placebo(p=0.0024 and p<0.0001, 10 14 respectively); study also met all secondary endpoints, including median time to resolution, which was days for both doses, compared placebo control groups (p<0.0001 both)< td> | 3/23/21 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine (repurposed formulation) | Xanthine oxidase inhibitor | COVID-19 infection | Trial expanding to up to 50 U.S. sites and on track to meet planned enrollment goal of 1,000 participants in second quarter of 2021; no serious adverse events or safety concerns to date | 3/24/21 | Infection |
| Roche Holding AG, of Basel, Switzerland, and Cipla Ltd., of Mumbai | Tocilizumab | IL-6 inhibitor | COVID-19 | Analysis of study conducted in hospitals across India, and published in The Lancet Respiratory Medicine, revealed subset of patients with severe disease in whom tocilizumab might have a reduced risk for progression to death if in addition to standard care, though clinical parameters or biomarkers to reliably identify those patients and optimal timing of treatment during COVID-19 progression remain unknown | 3/8/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | 1,3 beta glucan synthase inhibitor | Fungal infection | Interim analyses of ongoing Furi study showed benefit against difficult-to-treat, severe, mucocutaneous and invasive infections in 30 of 33 participants and no disease progression; first interim analysis of Cares study showed activity in people hospitalized with Candida auris, including complete response in 8/10 with invasive candidiasis and candidemia | 3/2/21 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-620 (maribavir) | Anti-cytomegalovirus compound | Transplant recipients with refractory, with or without resistance, cytomegalovirus (CMV) infection/disease | In the Solstice study, 55.7% of 117 transplant recipients with refractory, with or without resistance, CMV infection/disease treated with maribavir achieved confirmed CMV viremia clearance compared to 23.9% of 117 treated with 1 or a combination of antivirals ganciclovir, valganciclovir, foscarnet or cidofovir (p<0.001); 8 69 121 62.8% of the transplant recipients with confirmed genotypic resistant cmv infection at baseline treated maribavir achieved viremia clearance week compared 20.3% patients antiviral(s)< td> | 3/15/21 | Infection |
| Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London | VIR-7831/GSK-4182136 | Monoclonal antibody targeting SARS-CoV-2 | Hospitalized COVID-19 | The VIR-7831/GSK-4182136 arm of the ACTIV study met initial prespecified criteria to continue to the next phase, but sensitivity analyses of the available data raised concerns about the magnitude of potential benefit; independent data and safety monitoring board recommended the arm be closed to enrollment while the data matures | 3/3/21 | Infection |
| Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London | VIR-7831 | COVID-19 spike glycoprotein modulator | COVID-19 infection | Independent data monitoring committee recommended early halt of Comet-Ice monotherapy trial for early treatment of infection in adults at high risk of hospitalization after interim analysis of data from 583 participants showed 85% (p=0.002) reduction in hospitalization or death vs. placebo | 3/10/21 | Infection |
| Formosa Inc., of Cambridge, Mass., and Sosei Group Corp., of Tokyo | APP-13007 | Nanoparticle steroid formulation | Inflammation and pain after cataract surgery | Formosa dosed first patient in 370-patient, randomized study | 3/11/21 | Inflammatory |
| Horizon Therapeutics plc, of Dublin | Krystexxa (pegloticase injection) | Uricase stimulator | Gout | First of up to 30 people with uncontrolled gout enrolled in Forward trial assessing monthly dosing of 16 mg of study drug + methotrexate; primary outcome for 48-week trial is proportion of responders during month 6 of treatment, measured as serum uric acid (sUA) <6 mg dl at least 80% of time, and time to duration sua normalization< td> | 3/25/21 | Inflammatory |
| Clene Inc., of Salt Lake City | CNM-Au8 | Bioenergetic nanocatalyst | Amyotrophic lateral sclerosis | >50% of participants enrolled in Healey ALS platform trial at sites of Northeast ALS consortium; in CNM-Au8 treatment regimen, 160 participants are randomized 3:1, with 120 across 2 active arms (30 mg and 60 mg) and 40 in placebo | 3/9/21 | Musculoskeletal |
| Fibrogen Inc., of San Francisco | Pamrevlumab | Monoclonal antibody targeting connective tissue growth factor | Ambulatory Duchenne muscular dystrophy | Started the Lelantos-2 study comparing pamrevlumab plus systemic corticosteroids to corticosteroids alone in 70 patients for up to 52 weeks; primary endpoint is the change in North Star Ambulatory Assessment | 3/16/21 | Musculoskeletal |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J | TNX-102 SL (cyclobenzaprine HCl sublingual tablets) | Non-opioid, centrally acting analgesic | Fibromyalgia | The Rally study has randomized 50% of the 670 expected participants; interim results expected in the third quarter of 2021; top-line data expected in the fourth quarter of 2021 | 3/15/21 | Musculoskeletal |
| Adamas Pharmaceuticals Inc., of Emeryville, Calif. | Gocovri (amantadine) | Reduces the amount of glutamate hyperactivity | Parkinson’s disease | Data published in Frontiers in Neurology showed Gocovri increased the "on" time without dyskinesia by 2.9 hours compared to placebo | 3/29/21 | Neurology/Psychiatric |
| Grunenthal GmbH, of Aachen, Germany, and subsidiary Averitas Pharma Inc. | Qutenza (capsaicin) 8% patch | TRPV1 agonist | Postsurgical neuropathic pain | Plans to run a phase III study of 400 to 500 patients starting in the third quarter of 2021 with the goal of reducing average pain intensity after 12 weeks and 42 weeks compared to baseline; trial completion expected in 2024 | 3/30/21 | Neurology/Psychiatric |
| Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Pitolisant | Histamine 3 receptor antagonist/inverse agonist | Narcolepsy | Post-hoc analysis from 2 phase III studies published in Sleep Medicine showed, in the patients with a high burden of excessive daytime sleepiness (EDS) defined by Epworth Sleepiness Scale (ESS), least-squares mean change from baseline on the ESS was -6.1 for pitolisant compared to -2.3 for placebo (p<0.001); in patients with a high burden of eds as defined by maintenance wakefulness test (mwt), increase mean sleep latency on the mwt was 6.9 minutes for pitolisant compared to 3.4 placebo (p="0.017);" cataplexy, least-squares change weekly rate cataplexy –14.5 –0.1> | 3/17/21 | Neurology/Psychiatric |
| Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | Edaravone | Neuroprotectant | Amyotrophic lateral sclerosis | Post-hoc analysis showed risk reduction observed for the exploratory composite estimate of time to death, tracheostomy, permanent assisted ventilation (PAV) and hospitalization; during open-label period, there were 2 deaths in edaravone-edaravone (EE) group and 4 in edaravone-placebo (EP) group; survival analysis of cumulative occurrence of milestone events showed 53% relative risk reduction in EE group | 3/18/21 | Neurology/Psychiatric |
| Novartis AG, of Basel | Zolgensma (onasemnogene abeparvovec) | Gene therapy expressing SMN | Spinal muscular atrophy | In the SPR1NT study of presymptomatic patients with 2 or 3 copies of SMN2, 11 of 14 patients with 2 copies were able to sit without support for at least 30 seconds; 8 of 15 patients with 3 copies could stand alone for at least 3 seconds; in the long-term follow-up studies, 91% of the 23 patients in the I.V. cohort and 50% of the 8 patients in the IT cohort were not receiving concomitant disease-modifying SMA therapy; in the Restore registry study, 22 of 23 patients with 2 or more CHOP INTEND assessments improved or maintained their score | 3/15/21 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | PMP22 gene inhibitor | Charcot-Marie-Tooth disease type 1A | First subject enrolled in Premier trial; primary efficacy endpoint will be the Overall Neuropathy Limitations Scale, which measures functional motor disability | 3/31/21 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Tominersen | Antisense drug targeting the huntingtin protein | Huntington’s disease | Discontinuing dosing in the Generation HD1 study on recommendation of the independent data monitoring committee based on the benefit/risk profile; no new safety signals were identified; patients will continue to be followed; dosing paused in the open-label Gen-Extend extension study | 3/22/21 | Neurology/Psychiatric |
| Sage Therapeutics Inc., of Cambridge, Mass. | Zuranolone | Neuroactive steroid GABAA receptor positive allosteric modulator | Major depressive disorder | In the Shoreline study, patients with a clinical response to the initial 14-day course of zuranolone 30 mg, defined as a decrease in 17-item Hamilton Rating Scale for Depression (HAM-D) baseline score of ?50%, required a mean of 2.2 treatments over the 12-month follow-up; in the 50 mg cohort, at day 15, mean HAM-D change from baseline was -16; 80.5% achieved a response and 43.2% achieved remission; 12-month follow-up for the 50 mg dose expected in late 2021 | 3/17/21 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 (dihydroergotamine nasal powder) | 5-HT 1d receptor agonist | Migraine | New trial, planned to start in mid-2021 with top-line results expected in second half of 2022, will take into account findings from pivotal Emerge trial that missed co-primary endpoints; dose strengths greater than 5.2 mg first will be explored in phase I trial in second quarter of 2021, evaluating pharmacokinetics, safety and tolerability to select phase III dose | 3/1/21 | Neurology/Psychiatric |
| Eyenovia Inc., of New York | Mydcombi | MAP fixed-combination of tropicamide and phenylephrine | Mydriasis | Pooled results from 2 phase III studies published in Therapeutic Delivery showed 93% of eyes treated with Mydcombi achieved 6 mm or greater dilation at 35 minutes post-dose as compared to 78% with tropicamide and 2% with phenylephrine; 98% of pupils treated with Mydcombi were fully non-responsive at 35 minutes compared with 76% and 5% of pupils treated with tropicamide and phenylephrine alone, respectively | 3/17/21 | Ocular |
| Harbour Biomed Holdings Ltd., of Suzhou, China | Tanfanercept (HBM-9036) | TNF receptor-1 fragment-based drug | Moderate to severe dry eye disease | Dosed first patient in China trial | 3/11/21 | Ocular |
| Novartis AG, of Basel, Switzerland | Brolucizumab | VEGF-A ligand inhibitor | Diabetic macular edema | Study testing drug vs. aflibercept in patients with visual impairment due to DME cancelled due to COVID-19 | 3/4/21 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol (phentolamine mesylate eye drops) | Alpha 1/alpha 2 adrenoceptor antagonist | Mydriasis | In the Mira-2 study, 49% of study eyes treated with Nyxol returned to ? 0.2 mm of their baseline pupil diameter at 90 minutes compared to 7% of study eyes treated with placebo (p<0.0001); 2021 multiple secondary efficacy endpoints also met statistical significance; full results to be presented at the ascrs annual meeting in july 2021< td> | 3/15/21 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | VEGF-C/D trap inhibitor | Treatment-naïve wet age-related macular degeneration | First patient treated in the pivotal program comparing OPT-302 in combination with either Lucentis (ranibizumab, Roche Holding AG) or Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) to Lucentis or Eylea alone; primary endpoint for both studies is Best Corrected Visual Acuity from baseline to week 52 | 3/15/21 | Ocular |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Eylea (aflibercept) | VEGF inhibitor | Moderate to severe nonproliferative diabetic retinopathy | Initial results from NIH-sponsored study in patients with NPDR without center-involved diabetic macular edema (CI-DME) showed, at 2 years, a 68% reduced risk of developing vision-threatening complications (either proliferative diabetic retinopathy or CI-DME with vision loss) in patients who received Eylea in an every-16-weeks dosing regimen; in comparison, patients receiving sham injections were almost 5 times more likely to experience disease progression requiring Eylea rescue therapy; results published in JAMA Ophthalmology | 3/31/21 | Ocular |
| Regenerx Biopharmaceuticals Inc., of Rockville, Md. | RGN-259 | Thymosin beta 4 ligand; eye drops | Dry eye syndrome | Arise-3 study did not meet primary outcome measure; showed improvement in 1 of prespecified secondary symptom endpoints, improvement of ocular grittiness | 3/18/21 | Ocular |
| Albireo Pharma Inc., of Boston | Odevixibat | Ileal bile acid transport inhibitor | Alagille syndrome | Enrolled first patient in the Assert study; primary endpoint is change in Albireo Observer-Reported Outcome scratching score from baseline to month 6; top-line data expected in 2022 | 3/25/21 | Other/Miscellaneous |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Semaglutide (subcutaneous) | GLP1 receptor agonist | Obesity | Journal of the American Medical Association published findings from phase IIIa Step trial showing people dosed once-weekly at 2.4 mg following 20-week run-in had statistically significant additional mean weight loss of 7.9% to week 68 vs. those switched to placebo following run-in, who regained 6.9% for estimated treatment difference of -14.8% (p<0.0001); those on semaglutide 2.4 mg throughout 68-week trial achieved weight loss of 17.4%< td> | 3/23/21 | Other/Miscellaneous |
| Adlai Nortye Biopharma Co. Ltd., of Hangzhou, China | Buparlisib (AN-2025) | Pan-PI3K inhibitor | Recurrent or metastatic head and neck squamous cell carcinoma | Treated first of approximately 500 patients in the Buran study testing buparlisib plus paclitaxel; primary endpoint is overall survival | 4/14/21 | Cancer |
| Aravive Inc., of Houston | AVB-500 | Fusion protein targeting GAS6 | Platinum-resistant ovarian cancer | Treated first of 300-400 patients in the study testing AVB-500 plus paclitaxel; primary endpoint is progression-free survival | 4/27/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. and Veracyte Inc., of South San Francisco | Calquence (acalabrutinib) | BTK inhibitor | Untreated diffuse large B-cell lymphoma | First of up to 600 patients enrolled in the study using Veracyte’s Lymphmark lymphoma subtyping test to identify patients who may benefit from Calquence plus R-CHOP | 4/14/21 | Cancer |
| Beigene Co. Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma | At the interim analysis of the Alpine study, Brukinsa produced superior objective response rate compared to Imbruvica (ibrutinib, Johnson & Johnson/Abbvie Inc.) by investigator assessment (p=0.0006); by independent review committee assessment, ORR was numerically higher but not statistically significant (p=0.0121); data to be presented at an upcoming major medical conference | 4/28/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) + Yervoy (ipilimumab) | PD-1 inhibitor + CTLA4 inhibitor | Esophageal squamous cell carcinoma | In CheckMate -648 trial in people with unresectable advanced/metastatic disease, combination met primary and secondary endpoints, showing statistically significant improvement in overall survival (OS) in tumors expressing PD-L1 and in randomized population but missed co-primary endpoint of progression-free survival (PFS) by blinded independent central review in tumors expressing PD-L1; Opdivo + chemotherapy also showed statistically significant benefit for primary and secondary endpoints of OS in tumors expressing PD-L1 and in randomized population at prespecified interim analysis and showed statistically significant improvement in PFS | 4/8/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Monoclonal antibody targeting PD-1 | Unresectable advanced or metastatic esophageal squamous cell carcinoma | In the Checkmate-648, Opdivo plus chemotherapy improved overall survival in patients with tumors expressing PD-L1 and in the all-randomized patients at the prespecified interim analysis; the treatment also improved progression-free survival in patients with tumors expressing PD-L1; Opdivo plus Yervoy (ipilimumab) improved OS in patients with tumors expressing PD-L1 and in the all-randomized population; Opdivo plus Yervoy did not meet its other primary endpoint of PFS in patients with tumors expressing PD-L1; data will be shared at an upcoming medical conference, as well as with health authorities | 4/8/21 | Cancer |
| Eisai Inc., of Woodcliff Lake, N.J. | Lenvima (lenvatinib) | Multiple receptor tyrosine kinase inhibitor | Thyroid cancer | European Journal of Cancer published post-hoc analysis of Select study assessing impact of lung metastases on overall survival in people with locally recurrent/metastatic, progressive radioiodine-refractory differentiated disease, originally presented at ESMO annual meeting in September 2019 | 4/29/21 | Cancer |
| G1 Therapeutics Inc., of Research Triangle Park, N.C. | Cosela (trilaciclib) | Myelopreservation agent | Locally advanced unresectable or metastatic triple-negative breast cancer | Started the Preserve 2 study in patients receiving first- or second-line gemcitabine and carboplatin chemotherapy, evaluated separately for overall survival; secondary endpoints include quality of life assessments | 4/28/21 | Cancer |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | Erleada (apalutamide) + Zytiga (abiraterone) | Androgen receptor antagonist + cytochrome P450 17 inhibitor | Metastatic castration-resistant prostate cancer | Company will not pursue regulatory submissions after Acis combination study with prednisone, which met primary endpoint of progression-free survival, showed no benefit over active control of Zytiga + prednisone in key secondary endpoints, including overall survival | 4/19/21 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | Selective inhibitor of nuclear export | Unresectable dedifferentiated liposarcoma following progression on at least 2 prior therapies | Data from the phase III portion of the Seal study published in Future Oncology showed pain scores worsened in the placebo arm compared to the Xpovio arm across all post-baseline visits, though some visits were not statistically significant; median time to next treatment was significantly longer in patients receiving Xpovio compared to those receiving placebo | 4/19/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1 inhibitor | Renal cell carcinoma | Pivotal Keynote-564 trial met primary endpoint of statistically significant disease-free survival vs. placebo as adjuvant treatment following nephrectomy with or without resection of metastatic lesions | 4/8/21 | Cancer |
| Morphosys AG, of Planegg, Germany, and Incyte Corp., of Wilmington, Del. | Tafasitamab | CD19-targeting monoclonal antibody | Follicular or marginal zone lymphoma | First of 600+ adults dosed in Inmind combination study with lenalidomide + rituximab in people with relapsed/refractory disease; primary endpoint is progression-free survival in follicular lymphoma | 4/19/21 | Cancer |
| Shanghai Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | PD-1 inhibitor | Esophageal squamous cell carcinoma | DMC for Jupiter-06 study determined that toripalimab + paclitaxel/cisplatin in first-line advanced disease reached pre-specified primary endpoints of progression-free and overall survival at interim analysis and showed improvement in both measures vs. paclitaxel/cisplatin alone | 4/22/21 | Cancer |
| Steba Biotech SA, of Luxembourg | Padeliporfin Impact | Vascular-targeted photodynamic therapy | Low grade upper tract urothelial cancer | First of 100 patients treated in the Enlighted study; primary efficacy endpoint is the absence of tumors in the entire ipsilateral calyces’ renal pelvis and ureter; data expected in 2022 | 4/20/21 | Cancer |
| TG Therapeutics Inc., of New York | Ukoniq (umbralisib) + ublituximab + Venclexta (venetoclax, Abbvie Inc./Genentech Inc.) | Dual PI3K-delta/CK1-epsilon inhibitor + anti-CD20 monoclonal antibody + Bcl-2 protein inhibitor | Chronic lymphocytic leukemia | Phase III portion of Ultra-V trial initiated, evaluating triple combination vs. doublet of Ukoniq + ublituximab in people with front line and relapsed/refractory disease; primary endpoint is progression-free survival | 4/21/21 | Cancer |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | Bromodomain and extra-terminal inhibitor | Chronic kidney disease, type-2 diabetes mellitus and recent acute coronary syndrome | Data from the Betonmace study published in the American Society of Nephrology showed apabetalone produced a 52% hazard reduction for MACE events, such as cardiovascular death and heart failure, compared to placebo (p=0.03); in patients with CKD, apabetalone reduced alkaline phosphatase after 24 weeks of treatment (p=0.004) | 4/27/21 | Cardiovascular |
| Akari Therapeutics plc, of London | Nomacopan | Complement C5 inhibitor; leukotriene BLT antagonist | Bullous pemphigoid | Pivotal trial initiated, with enrollment of people with moderate to severe disease expected to begin in mid-2021; primary endpoint is disease remission on minimal oral corticosteroids | 4/12/21 | Dermatologic |
| Amgen Inc., of Thousand Oaks, Calif. | Otezla (apremilast) | Phosphodiesterase 4 inhibitor | Mild to moderate plaque psoriasis | In the Advance study, 21.6% of patients taking Otezla 30 mg twice daily achieved Physician's Global Assessment response at week 16 compared to 4.1% of patients taking placebo (p<0.0001); 71.7% of patients taking otezla achieved a body surface area (bsa) 3% or less compared to 35.8% placebo; 29% had at least 75% improvement in bsa 6.1% placebo< td> | 4/23/21 | Dermatologic |
| Arcutis Biotherapeutics Inc., of West Lake Village, Calif. | Roflumilast cream (ARQ-151) | Phosphodiesterase type 4 inhibitor | Mild to moderate atopic dermatitis | Started the 650-patient Integument-Ped study in patients ages 2 to 5; primary endpoint is Investigator Global Assessment Success, defined as a Validated Investigator Global Assessment - Atopic Dermatitis score of ‘clear’ or ‘almost clear’ plus a 2-grade improvement from baseline at week 4; secondary endpoints include Worst Itch-Numerical Rating Scale and proportion of patients who attain at least a 75% reduction in the Eczema Area and Severity Index at week 4; data expected in the second half of 2022 | 4/12/21 | Dermatologic |
| Boehringer Ingelheim Pharmaceuticals Inc., unit of Boehringer Ingelheim GmbH, of Ingelheim, Germany | Cyltezo (adalimumab-adbm) | TNF inhibitor | Psorasis | Voltaire-X study showed that switching several times from Humira (adalimumab, Abbvie Inc.) to Cyltezo produced similar outcomes in pharmacokinetics, efficacy, immunogenicity and safety in people with moderate to severe chronic disease, meeting primary endpoint at 32 weeks and supporting interchangeability application | 4/23/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo. | Sofpironium bromide gel | Acetylcholine receptor antagonist | Hyperhidrosis | Argyle long-term safety study in 300 participants showed daily treatment with 5% or 15% doses was well tolerated over 48 weeks of treatment; efficacy assessments showed clinically meaningful and sustained improvement in sweat severity through 48 weeks of treatment | 4/23/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo. | Sofpironium bromide gel | Blocks acetylcholine | Primary axillary hyperhidrosis | Completed enrollment in the Cardigan I study; Cardigan II study is more than 50% enrolled; top-line results from both studies expected in the fourth quarter of 2021 | 4/27/21 | Dermatologic |
| Bristol Myers Squibb Co., of New York | Deucravacitinib | Oral, selective TYK2 inhibitor | Moderate to severe plaque psoriasis | Results from 2 pivotal phase III trials showed both met co-primary endpoints vs. placebo, with significantly more patients achieving Psoriasis Area and Severity Index 75 response and a static Physician's Global Assessment score of clear or almost clear after 16 weeks of treatment; it was well tolerated with a low rate of discontinuation due to adverse events | 4/23/21 | Dermatologic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | Adenosine A3 receptor agonist | Psoriasis | Comfort study assessing noninferiority vs. Otezla (apremilast, Amgen Inc.) achieved 75% enrollment; top-line data expected in fourth quarter of 2021 | 4/29/21 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Olumiant (baricitinib) | Dual Jak1/Jak2 inhibitor | Atopic dermatitis | Analysis of Breeze-AD5 data showed improvement from baseline in EASI of 25.3% for study drug vs. 7.2% for placebo at 1 week and 50.9% vs. 24.0%, respectively, at 4 weeks; improvement from baseline in Itch Numeric Rating Scale at 1 week was 13.5% vs. -0.2% and at 4 weeks was 29.0% vs. 12.5%, respectively | 4/23/21 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | Monoclonal antibody targeting interleukin 17A | Plaque psoriasis | In 1-year network meta-analysis based on AUC, Taltz showed numerically greater cumulative benefits on completely clear skin vs. 7 other biologics, measured by PASI 100, providing 159 cumulative days (95% credible interval, 147.4-170 days) or 23 weeks of PASI 100 | 4/23/21 | Dermatologic |
| Eli Lily and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Baricitinib | JAK inhibitor | Severe alopecia areata | In the Brave-AA1 study, 35% of patients taking baricitinib 4 mg/day and 22% of patients treated with?baricitinib?2 mg/day achieved 80% or more scalp hair coverage at week 36, compared to 5% of patients in the placebo group (p?0.001 for both doses compared to placebo); in the Brave-AA2 study, 33% of the 4-mg group, 17% of the 2-mg group and 3% of the placebo group met the goal (p?0.001 for both doses compared to placebo) | 4/20/21 | Dermatologic |
| Incyte Corp., of Wilmington, Del. | Ruxolitinib cream | JAK inhibitor | Atopic dermatitis (AD) | Findings from 3 pooled analyses of its randomized, double-blind phase III studies showed ruxolitinib cream 0.75% applied twice daily (BID) and ruxolitinib cream 1.5% BID both demonstrated greater improvement compared to vehicle in all analyzed efficacy endpoints; higher responses were observed in patients with more severe disease; a subsequent analysis found higher rates of clinical responses with ruxolitinib cream vs. vehicle in severe AD patients | 4/23/21 | Dermatologic |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting the p19 subunit of IL-23 | Moderate to severe plaque psoriasis | In the Voyage 2 study, at week 252, 55.5% of patients taking Tremfya achieved an Investigator's Global Assessment score of 0 and 53% achieved a Psoriasis Area Severity Index 100 skin clearance response | 4/23/21 | Dermatologic |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23 inhibitor | Plaque psoriasis; psoriatic arthritis | In Voyage 2 study, Tremfya showed durable, complete skin clearance rates in most adults with moderate to severe psoriasis through 5 years (252 weeks), with 55.5% achieving IGA score of 0 and 53% achieving PASI 100 response; Discover-1 and -2 trials showed improved disease activity and axial symptoms in adults with active psoriatic arthritis through 52 weeks | 4/23/21 | Dermatologic |
| Lipidor AB, of Stockholm | AKP-02 (calcipotriol + betamethasone, spray formulation) | Corticosteroid agonist | Psoriasis | Registration-based study in India expected to recruit 294 participants with mild to moderate disease on track to begin enrollment in mid-2021, with data expected in first half of 2022 | 4/22/21 | Dermatologic |
| Ortho Dermatologics, unit of Bausch Health Companies Inc., of Laval, Quebec | IDP-126 (1.2% clindamycin phosphate + 3.1% benzoyl peroxide + 0.15% adapalene, topical gel) | Retinoic acid receptor agonist + antibacterial agent + antibiotic topical agent | Acne vulgaris | Second pivotal trial in 193 participants confirmed findings of first phase III, meeting co-primary endpoints at week 12 with statistical significance, including absolute change from baseline in inflammatory lesion count (p<0.001), absolute change from baseline in non-inflammatory lesion count (p<0.001) and percentage of people who achieved treatment success (p="0.001) | 4/22/21 | Dermatologic |
| UCB SA, of Brussels | Bimekizumab | Dual IL-17A/IL-17F inhibitor | Plaque psoriasis | New England Journal of Medicine published new results from phase IIIb Be Radiant study in adults with moderate to severe disease; study met primary endpoint, with 61.7% treated with study drug achieving complete skin clearance, measured by PASI 100 at week 16, vs. 48.9 for Cosentyx (secukinumab, Novartis, AG; p<0.001)< td> | 4/23/21 | Dermatologic |
| Ardelyx Inc., of Fremont, Calif., and Kyowa Kirin Co. Ltd., of Tokyo | Tenapanor | Sodium hydrogen exchanger 3 inhibitor | Hyperphosphatemia | Started 4 studies in Japan: a placebo-controlled, parallel-group comparative study; a phosphate binder-combination parallel-group comparative study; an open-label, single-arm study in hyperphosphatemia patients on peritoneal dialysis; and a long-term study evaluating serum phosphorus in patients who switch from 1 or more phosphate binders to tenapanor | 4/14/21 | Endocrine/Metabolic |
| Obseva SA, of Geneva | Yselty (linzagolix) | GNRH receptor antagonist | Uterine fibroids | Started the Primrose 3 long-term follow-up study to the Primrose 1 and 2 studies to measure bone mineral density at 12, 18 and 24 months | 4/27/21 | Genitourinary/Sexual Function |
| Urovant Sciences Inc., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Gemtesa (vibegron) | Beta-3 adrenergic agonist | Overactive bladder | Data from the Empowur trial and its extension study published in the Journal of Urology showed the least squares mean change from baseline to week 52 in micturitions was ?2.4 for Gemtesa compared to ?2 for tolterodine; urge urinary incontinence episodes was ?2.2 for Gemtesa compared to ?1.7 for tolterodine (p <0.05); incontinence episodes was ?2.5 for gemtesa compared to ?1.9 tolterodine (p <0.05)< td> | 04/15/21 | Genitourinary/Sexual Function |
| Vascular Therapies LLC, of Cresskill, N.J. | Sirogen | Formulation of sirolimus for local, perivascular drug delivery | End stage renal disease; chronic kidney disease | Access study missed primary endpoint of fistula suitability for dialysis at 6 months; subgroup of those 65 and older showed improved overall and forearm fistula maturation and improved suitability for dialysis at 12 months | 4/29/21 | Genitourinary/Sexual Function |
| Vifor Fresenius Medical Care Renal Pharma Ltd., of St. Gallen, Switzerland | Velphoro (PA-21) | Non-calcium, iron-based, chewable phosphate binder | Chronic kidney disease on dialysis | The PA-CL-CHINA-01 study met its primary endpoint demonstrating noninferiority vs. sevelamer carbonate in the change from baseline in serum phosphorus levels at week 12; Velphoro lowered serum phosphorus levels faster than sevelamer carbonate with fewer average daily tablets (3.23 for Velphoro vs. 6.31 for sevelamer carbonate) | 4/30/21 | Genitourinary/Sexual Function |
| Akebia Therapeutics Inc., of Cambridge, Mass. | Vadadustat | HIF prolyl hydroxylase inhibitor | Anemia | The New England Journal of Medicine published separate articles on phase III Inno2vate program to treat anemia due to chronic kidney disease in adults on dialysis, which met noninferiority vs. darbepoetin alfa for cardiovascular safety and maintenance of hemoglobin concentrations, and Pro2tect study in adults not on dialysis, which met prespecified noninferiority for hematologic efficacy but not for cardiovascular safety | 4/28/21 | Hematologic |
| Geron Corp., of Foster City, Calif. | Imetelstat | Telomerase inhibitor | Myelofibrosis | First of about 320 participants with disease refractory to JAK inhibitor dosed in ImpactMF trial; primary endpoint is overall survival, with interim analysis at 70% of projected events expected in 2024 and final analysis in 2025 | 4/13/21 | Hematologic |
| Global Blood Therapeutics Inc., of South San Francisco | Oxbryta (voxelotor) | Hemoglobin alpha subunit modulator | Sickle cell disease | The Lancet Haematology published analysis of 72-week data from Hope study showing sustained improvement in hemoglobin levels, reduction in hemolysis and improved overall health status in treated participants; about 90% achieved hemoglobin improvement of >1 g/dL from baseline at 1 or more time points vs. placebo (25%) and about 59% achieved hemoglobin increase of >2 g/dL and 20% achieved >3 g/dL at 1 or more time points vs. about 3% and none for placebo, respectively | 4/8/21 | Hematologic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Active psoriatic arthritis after 1 or more non-biologic disease modifying anti-rheumatic drugs | Data published in The New England Journal of Medicine showed 54% and 58% of patients taking 15-mg and 30-mg doses, respectively, achieved Leeds Enthesitis Index of 0 at week 24, compared to 32% of patients taking placebo (p<0.001 for both doses) and 47% of patients taking humira (adalimumab)< td> | 4/1/21 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Pooled data on 532 participants in Aura-LV and Aurora 1 studies showed Lupkynis in combination with standard-of-care (SOC) mycophenolate mofetil and low-dose corticosteroids led to treatment benefits across biopsy class subgroups vs. SOC alone, with odds ratio of 4.26 for pure class III (p=0.0054), 2.59 for pure class IV (p=0.0005), 1.5 for pure class V (p=0.4090) and 2.68 for mixed class III/IV and V (p=0.0166) | 4/8/21 | Immune |
| Biogen Inc., of Cambridge, Mass. | Tysabri (natalizumab) | Monoclonal antibody targeting alpha4-beta1 integrin | Multiple sclerosis | Tysabri produced statistically significant improvements in 10 of 12 domains of Quality of Life in Neurological Disorders compared to 8 of 12 domains for Ocrevus (ocrelizumab, Roche Holding AG); Tysabri was better than Ocrevus for satisfaction with social roles and activities (p=0.02), participation in social roles and activities (p=0.0001) and emotional and behavioral dyscontrol (p=0.01); extended interval dosing produced comparable real-world effectiveness on quantitative magnetic resonance imaging (MRI) outcomes compared to standard interval dosing (p>0.05 for all MRI outcomes) | 4/16/21 | Immune |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting the p19 subunit of IL-23 | Psoriatic arthritis | In the Discover-1 and -2 studies, at 52 weeks, Tremfya improved composite disease activity scores and Bath Ankylosing Spondylitis Disease Activity Index | 4/23/21 | Immune |
| Kedrion Biopharma SpA, of Fort Lee, N.J. | 10% intravenous immunoglobulin | Antibodies | Primary immunodeficiency disease | Enrolled first patient in the Kidscares10 study; primary efficacy objective is to show the drug produces a rate of acute serious bacterial infections of less than 1 | 4/1/21 | Immune |
| Roche Holding AG, of Basel, Switzerland | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Multiple sclerosis | Interim analysis of the phase IIIb Ensemble study showed 85% of patients with relapsing-remitting MS achieved no evidence of disease activity after 48 weeks; Expanded Disability Status Scale score improved from 1.71 at baseline to 1.55 after 48 weeks (p=0.002); a post-hoc analysis of the phase III Oratorio study showed the atrophied T2-lesion volume was 319 mm3 for primary progressive MS patients treated with Ocrevus at 120 weeks compared to 366 mm3 for placebo (p<0.015)< td> | 4/16/21 | Immune |
| TG Therapeutics Inc., of New York | Ublituximab | Glycoengineered monoclonal antibody targeting CD20 | Multiple sclerosis | Annualized relapse rate was 0.076 for ublituximab compared to 0.188 for Aubagio (teriflunomide, Sanofi SA) in the Ultimate I study (p<0.0001) and 0.091 for ublituximab compared to 0.178 aubagio in ultimate ii (p="0.0022);" i, 44.6% of treated patients achieved no evidence disease activity (neda), a 198% improvement over (p<0.0001); ii, neda rate was 43% ublituximab, 277% (p<0.0001)< td> | 4/16/21 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Hospitalized COVID-19 with risk of developing serious complications | The Dare-19 didn’t meet the primary endpoint of prevention of organ dysfunction and all-cause mortality or the primary endpoint of change in clinical status from early recovery to death, at 30 days; results will be presented at the American College of Cardiology Scientific Sessions in May 2021 | 4/12/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K., and Sanofi SA, of Paris | Nirsevimab | Monoclonal antibody targeting respiratory syncytial virus | Lower respiratory tract infections | The Melody study met its primary endpoint of reducing the incidence of medically attended lower respiratory tract infections caused by respiratory syncytial virus compared to placebo; data to be presented at a forthcoming medical meeting | 4/26/21 | Infection |
| Atea Pharmaceuticals Inc., of Boston | AT-527 | RNA polymerase inhibitor | COVID-19 | First of about 1,400 non-hospitalized participants with mild to moderate infection dosed in Morningsky trial; primary efficacy endpoint is time to alleviation or improvement of COVID-19 symptoms vs. placebo | 4/29/21 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-196 + BRII-198 | COVID-19 Spike glycoprotein inhibitors | COVID-19 | NIH Activ-2 trial advanced to phase III following data safety monitoring board review of data from 220 participants in phase II portion; phase III part designed to assess whether drug combination prevents composite endpoint of hospitalization or death through day 28 | 4/29/21 | Infection |
| Covis Pharma BV, of Luxembourg | Alvesco (ciclesonide) | Glucocorticoid receptor agonist | Non-hospitalized COVID-19 | In the 400-patient study, 70.6% of patients taking Alvesco and 63.5% of patients taking placebo had an improved time to alleviation of COVID-19 related symptoms (p=0.5502); Alvesco produced a 70% reduction in subsequent emergency department visits or hospital admissions for reasons attributable to COVID-19 by day 30 compared to a 30% reduction for placebo (p=0.0301) | 4/15/21 | Infection |
| Daré Bioscience Inc., of San Diego | DARE-BV1 | Thermosetting vaginal gel | Bacterial vaginosis | In the DARE-BVFREE study, 70% of patients treated with DARE-BV1 were clinically cured compared to 36% of patients taking placebo | 4/26/21 | Infection |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Olumiant (baricitinib) | JAK1/JAK2 inhibitor | COVID-19 infection | COV-Barrier trial of study drug + standard of care (SOC) vs. placebo + SOC in 1,525 people hospitalized with infection missed statistical significance (p=0.18) on primary endpoint of difference in proportion who progressed to non-invasive or invasive mechanical ventilation or death by day 28; trial showed reduction (nominal p=0.0018) in death from any cause by day 28 | 4/8/21 | Infection |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | COVID-HIG | SARS-CoV-2 immune globulin intravenous | COVID-19 | Top-line data from ITAC (Insight-013) trial in hospitalized patients testing 4 immunoglobulin candidates plus standard of care (SOC) vs. placebo plus SOC demonstrated additional of anti-SARS-CoV-2 hyperimmunoglobulin to standard of care, inclusive of remdesivir, did not provide clinical benefit when compared to SOC plus placebo; no serious safety concerns identified | 4/2/21 | Infection |
| Kintor Pharmaceuticals Ltd., off Suzhou, China | Proxalutamide | Nonsteroidal antiandrogen | Mild or moderate COVID-19 | First patient treated in the U.S.; primary endpoint is the percentage of hospitalization events (including death) by day 28; secondary endpoints include proportion of mortality by day 28, percentage of patients achieving each clinical status on days 7, 14 and 28 using National Institute of Allergy and Infectious Diseases 8-point scoring scale | 4/25/21 | Infection |
| Mesoblast Ltd., of New York | Remestemcel-L | Culture-expanded mesenchymal stromal cells | Ventilator-dependent COVID-19 with moderate/severe acute respiratory distress syndrome | Remestemcel-L reduced mortality through day 60 by 46% in the prespecified group below age 65, but not in patients 65 or older; remestemcel-L plus dexamethasone reduced mortality by 75% and increased days alive off mechanical ventilation in patients under age 65 compared to dexamethasone alone | 4/29/21 | Infection |
| Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holding AG, of Geneva | Zyesami (aviptadil) | VIP receptor agonist | COVID-19 | Aviptadil plus Veklury (remdesivir, Gilead Sciences Inc.) will be tested in the 640-patient Tesico study run National Institute of Allergy and Infectious Diseases; primary endpoint is participant recovery from respiratory failure over 90 days | 4/6/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Protein-based vaccine | COVID-19 prophylaxis | Started crossover (placebo to vaccine or vaccine to placebo) for the phase III study in the U.K. | 4/5/21 | Infection |
| Ocugen Inc., of Malvern, Pa. | Covaxin | Virion inactivated COVID-19 vaccine | COVID-19 infection | Co-development partner Bharat Biotech International Ltd., of Hyderabad, India, reported data from second interim analysis showing point estimate of vaccine efficacy of 78% (95%CI: 61-88) against mild, moderate and severe infection, based on 127 symptomatic cases; efficacy against asymptomatic COVID-19 infection was 70% | 4/21/21 | Infection |
| Oxford University | Inhaled budesonide | Corticosteroid | COVID-19 patients over 50 | In the Principle platform study, estimated median time to self-reported recovery for inhaled budesonide was 1.134 days compared to 5.410 days for standard of care; 32% of those taking inhaled budesonide, compared to 22% of those receiving SOC recovered within the first 14 days; 8.5% of patients in the budesonide group were hospitalized with COVID-19 compared to 10.3% of the SOC group | 4/12/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | RNAi-based vaccine | COVID-19 prophylaxis | Updated results from the study showed the vaccine was 91.3% effective from 7 days to 6 months after the second dose; 100% effective against severe COVID-19 as defined by the CDC and 95.3% effective against severe COVID-19 as defined by the FDA; 100% effective against the South African variant | 4/1/21 | Infection |
| Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Potassium-competitive acid blocker | H. pylori infection | Vonoprazan plus amoxicillin and clarithromycin therapy produced an H. pylori eradication rate of 84.7% compared to 78.5% for vonoprazan plus amoxicillin and 78.8% for lansoprazole plus amoxicillin and clarithromycin (p<0.0001 and p="0.0037" for the vonoprazan triple double combinations, respectively, compared to lansoprazole combination noninferiority)< td> | 4/29/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and F. Hoffmann-La Roche Ltd., of Basel, Switzerland | Regen-Cov (casirivimab and imdevimab) | Monoclonal antibody targeting SARS-CoV-2 | Recently infected asymptomatic COVID-19 | In the 2069B study, treatment reduced the overall risk of progressing to symptomatic COVID-19 by 31% through day 29 (p=0.0380) and by 76% after day 3 (p=0.0007); no patients treated with the antibodies had COVID-19-related hospitalizations or emergency room visit, compared to 6% of patients taking placebo (p=0.029) | 4/12/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and F. Hoffmann-La Roche Ltd., of Basel, Switzerland | Regen-Cov (casirivimab and imdevimab) | Monoclonal antibody targeting SARS-CoV-2 | COVID-19 prophylaxis among household contacts of SARS-CoV-2-infected individuals | In the 2069A study, the treatment reduced the risk of symptomatic infections by 81% through day 29 (p=0.0001); patients who developed symptomatic infection resolved their symptoms on average within 1.2 weeks, compared to 3.2 weeks for placebo | 4/12/21 | Infection |
| Romark Laboratories LC, of Tampa, Fla. | NT-300 (nitazoxanide extended-release tablets) | Inhibits viral replication | Mild or moderate COVID-19 | In the overall population, median time to sustained response was approximately 13 days for both patients treated with NT-300 and those treated with placebo; in the predefined subgroup with mild disease, median time to sustained response was 10.3 days for NT-300 and 13.4 days for placebo; 0.5% of NT-300-treated patients vs. 3.6% of patients treated with placebo progressed to severe illness | 4/14/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Brexafemme (ibrexafungerp) | Glucan synthase inhibitor | Vulvovaginal candidiasis | A subanalysis of the VANISH-303 study of patients with non-albicans Candida infection showed Brexafemme produced a 42.1% clinical cure rate at day 10 and 52.6% symptom resolution rate at the day 25; in severe patients, clinical cure rate at day 10 was 63.3% for Brexafemme compared to 44% for placebo (p=0.007) | 4/30/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1553 | Live attenuated viral vaccine | Chikungunya virus infection | Enrollment of 4,131 adults completed in pivotal trial, with top-line data expected in mid-2021; antibody persistence trial initiated to track immunogenicity subset for 5 years | 4/12/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 infection | Pivotal trial initiated in ~4,000 participants; primary endpoint is superiority vs. Vaxzevria (AZD-1222, Astrazeneca plc) | 4/21/21 | Infection |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | ION-363 | FUS gene inhibitor | Amyotrophic lateral sclerosis | Trial initiated in up to 54 participants with ALS with mutations in FUS gene | 4/5/21 | Musculoskeletal |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR gene inhibitor | Transthyretin-mediated amyloidosis | Previously reported findings from Helios-A, which met primary and secondary endpoints, presented at AAN annual meeting | 4/19/21 | Neurology/Psychiatric |
| Baudax Bio Inc., of Malvern, Pa. | Anjeso (meloxicam) | COX-2 inhibitor | Pain from unilateral total knee arthroplasty | Data published in Pain Medicine showed patients used 31.7% less opioids compared to placebo during the first 24 hours (p<0.0001); summed pain intensity score on the first postsurgical day was 22% lower for anjeso compared to placebo (p?0.0001) < td> | 4/26/21 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | Xywav (calcium, magnesium, potassium, and sodium oxybates) | GABA B receptor agonist | Idiopathic hypersomnia | After the open-label optimization of up to 14 weeks and the 2-week stable-dose period, Epworth Sleepiness Scale (ESS) score improved from a mean of 15.7 to 6.1; Idiopathic Hypersomnia Severity Scale (IHSS) score improved from a mean of 31.6 to 15.3; in the randomized withdraw period, ESS scores LS mean difference for those who continued Xywav treatment compared to those who were switched to placebo was ?6.51 (p<0.0001) and the difference in ihss scores was ?12 (p<0.0001), both favor of xywav< td> | 4/20/21 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Zolgensma (onasemnogene abeparvovec) | SMN1 gene stimulator | Spinal muscular atrophy | Phase IIIb Smart study will assess gene therapy in 24 young children with SMA who weigh ? 8.5 kg and ? 21 kg following one-time intravenous infusion; efficacy endpoints include change from baseline in motor milestones | 4/23/21 | Neurology/Psychiatric |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Brexpiprazole | Partial serotonin 5-HT1A/dopamine D2 agonist; serotonin 5-HT2A antagonist | Agitation | Enrollment of participants with Alzheimer's-type dementia to continue, based on interim analysis supporting progression to full enrollment of 330 people | 4/13/21 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 (baclofen + naltrexone hydrochloride + sorbitol) | PMP22 gene inhibitor | Charcot-Marie-Tooth disease type 1A | Interim analysis of ongoing open-label Pleo-CMT-FU extension study showed sustained benefits, measured by Overall Neuropathy Limitation Scale, at 54 months of total trial time; improvement seen across all cohorts | 4/28/21 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of New York | REL-1017 | NMDA receptor channel blocker | Major depressive disorder | Started the 364-patient Reliance II study; primary endpoint is the change from baseline on the Montgomery and Asberg Depression Rating Scale (MADRS) score at day 28; MADRS at day 7 is a secondary endpoint; data expected in the first half of 2022 | 4/1/21 | Neurology/Psychiatric |
| Revance Therapeutics Inc., of Nashville, Tenn. | DaxibotulinumtoxinA | Acetylcholine receptor antagonist; botulinum toxin A stimulator | Cervical dystonia | In the Aspen-1 study, mean improvement from baseline in Toronto Western Spasmodic Torticollis Rating Scale total score was 12.7 for 125U dose, 10.9 for 250U dose and 4.3 for placebo (p<0.0001 and p="0.0006" for 125u 250u doses, respectively)< td> | 4/1/21 | Neurology/Psychiatric |
| Revance Therapeutics Inc., of Nashville, Tenn., and Shanghai Fosun Pharmaceutical Group Co. Ltd., of Shanghai | DaxibotulinumtoxinA | Botulinum toxin | Glabellar lines and cervical dystonia | Started studies testing the drug in both indications | 4/27/21 | Neurology/Psychiatric |
| SK Life Science Inc., of Paramus, N.J. | Cenobamate | Inhibits voltage-gated sodium currents | Uncontrolled partial-onset seizures | In the C021 study, 33.9% of the 177 patients who remained on cenobamate had sustained seizure freedom for at least 12 months as of their last clinic visit; 46.9% of patients were seizure-free for at least 1 year at any time during the follow-up; 25% of patients discontinued all of their concomitant anti-seizure medications | 4/15/21 | Neurology/Psychiatric |
| Vallon Pharmaceuticals Inc., of Philadelphia | Adair (dexamfetamine immediate-release abuse deterrent) | Synaptic vesicular amine transporter stimulator; trace amine associated receptor 1 agonist | Attention deficit hyperactivity disorder; narcolepsy | Ongoing pivotal 4-way crossover Seal study assessing pharmacodynamics, pharmacokinetics, safety and tolerability on target to report data in second half of 2021, with potential NDA filing in second quarter of 2022 | 4/13/21 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (fenfluramine) | 5-HT 1d/5-HT 2a/5-HT 2c receptor modulator; opioid receptor sigma modulator | Lennox-Gastaut syndrome | Data in 137 evaluable participants ages 6 to 18 years showed improvement in executive function for study drug (n=92) vs. placebo (n=45), with statistical significance in cognition (27% vs. 13%, respectively, p=0.046) and Global Executive Composite score (25% vs. 11%, p=0.034) | 4/22/21 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Inhibitor of reactive aldehyde species | Allergic conjunctivitis | Compared to placebo, reproxalap produced a reduction in ocular itching score over all 11 prespecified primary endpoint comparisons from 110 to 210 minutes in the allergen chamber (p<0.0001 for each comparison); drug improved investigator-assessed ocular redness over the duration of allergen chamber (p<0.0001), change from baseline in patient-reported tearing score on a 0?3 point scale (p<0.0001) and total severity (p<0.0001)< td> | 4/27/21 | Ocular |
| Bausch + Lomb, unit of Bausch Health Cos. Inc., of Laval, Quebec | NOV-03 (perfluorohexyloctane) | Ophthalmic liquid formulation | Dry eye disease | Gobi trial met co-primary endpoints of statistically significant change from baseline in total corneal fluorescein staining at day 15 (p=0.001, secondary endpoint), with continued results through day 57 vs. control (p< 0.001, primary endpoint) and statistically significant change from baseline in dryness score at day 15 (p=0.009, secondary endpoint), with continued results through day 57 vs. control (p<0.001, primary endpoint)< td> | 4/13/21 | Ocular |
| Novaliq GmbH, of Heidelberg, Germany | Cyclasol | Topical anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine A | Dry eye disease | More than 50% of the 834 targeted patients have been randomized in the Essence-2 study; top-line results expected in the second half of 2021 | 4/28/21 | Ocular |
| Endo International plc, of Dublin | Qwo (collagenase clostridium histolyticum-aaes) | Combination of bacterial collagenases | Moderate to severe cellulite in the buttocks | Data from the Release-1 and -2 studies published in Dermatologic Surgery showed the drug met the primary endpoint of a composite 2-level response on a 5-point cellulite severity scale; over half of the women treated with Qwo in both studies also met the secondary endpoint, a 1-level improvement in the patient-reported assessment | 4/13/21 | Other/Miscellaneous |
| Innocoll Biotherapeutics plc, of Athlone, Ireland | Xaracoll (bupivacaine hydrochloride) | Collagen drug-device | Abdominoplasty and open soft-tissue surgeries | Started the INN-CB-024 placebo-controlled study in patients undergoing abdominoplasty and the INN-CB-025 open-label study in patients undergoing open ventral hernia repair, abdominoplasty, open abdominal hysterectomy, laparoscopic-assisted colectomy or reduction mammoplasty | 4/14/21 | Other/Miscellaneous |
| Mimedx Group Inc., of Marietta, Ga. | Amniofix | Micronized dehydrated human amnion chorion membrane | Plantar fasciitis and achilles tendonitis | Last patients completed their last clinical visits in both phase III studies; data from both studies expected in the third quarter of 2021 | 4/19/21 | Other/Miscellaneous |
| Novo Nordisk A/S, of Bagsværd, Denmark | Semaglutide | GLP-1 receptor agonist | Obesity | Pivotal phase IIIa program expected to begin in second half of 2021 and enroll ~1,000 people with obesity or overweight with comorbidities in 68-week trial assessing oral semaglutide dosed weekly at 50 mg | 4/21/21 | Other/Miscellaneous |
| Zambon SpA, of Milan | Liposomal cyclosporine A for inhalation | Calcineurin inhibitor | Bronchiolitis obliterans syndrome | Poster described patient safety and data integrity adaptations during Boston program to address COVID-19 pandemic, including remote visits and inclusion of remote spirometry assessments with home portable spirometers | 4/29/21 | Respiratory |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor targeting mast cells and macrophages | First-line metastatic castrate refractory prostate cancer | In Study AB12003, masitinib plus docetaxel produced a statistically significant increase in progression-free survival compared to docetaxel alone (p=0.0272); PFS rate at 12 months, 18 months and 24 months was 32%, 27.6% and 23.1%, respectively, for masitinib plus docetaxel, compared with 19.6%, 14.6% and 12%, respectively, for docetaxel alone (p=0.0035, p=0.0011 and p=0.0028, respectively) | 5/26/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) and tremelimumab | Monoclonal antibody targeting PD-L1 and anti-CTLA4 antibody | First-line stage IV non-small-cell lung cancer | A phase III trial of Imfinzi and tremelimumab plus chemotherapy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit vs. chemotherapy alone, but the OS trend observed did not achieve statistical significance; each combination demonstrated an acceptable safety profile, and no new safety signals were identified | 5/7/21 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | Monoclonal antibody targeting PD-1 | First-line recurrent or metastatic nasopharyngeal cancer | In the Rationale 309 study, tislelizumab plus chemotherapy improved progression-free survival at the interim analysis compared to chemotherapy alone; data to be presented at an upcoming medical conference | 5/21/21 | Cancer |
| Biolinerx Ltd., of Tel Aviv | Motixafortide | CXCR4 inhibitor | Hematopoietic stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma | In the Genesis study, 70% of patients taking motixafortide plus G-CSF mobilized ? 6 million CD34+ cells/kg in up to 2 apheresis sessions compared to 14.3% of patients taking G-CSF alone; 67.5% of patients taking motixafortide plus G-CSF mobilized ? 6 million CD34+ cells/kg in just 1 apheresis session compared to 4.8% of patients taking G-CSF alone | 5/4/21 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China, and Eqrx Inc., of Cambridge, Mass. | Sugemalimab | Anti-PD-L1 monoclonal antibody | Non-small-cell lung cancer | Gemstone-301 study in patients with stage III disease met primary endpoint at planned interim analysis reviewed by independent data monitoring committee; findings showed drug as consolidation therapy brought statistically significant and clinically meaningful improvement in Blinded Independent Central Review (BICR) assessing progression-free survival PFS in patients with locally advanced/unresectable NSCLC without disease progression after concurrent or sequential chemoradiotherapy; subgroup analyses showed drug associated with clinical benefit regardless of whether patients received concurrent or sequential chemoradiotherapy prior to sugemalimab | 5/28/21 | Cancer |
| Idera Pharmaceuticals Inc., of Exton, Pa. | Tilsotolimod | TLR-9 agonist | Melanoma | Illuminate-301 combination trial with Yervoy (ipilimumab, Bristol Myers Squibb Co.) vs. ipilimumab alone in people with anti-PD-1-refractory advanced disease halted; company reported in March 2021 that trial missed primary endpoint of objective response rate | 5/18/21 | Cancer |
| Innovent Biologics Inc., of San Francisco, and Incyte Corp., of Wilmington, Del. | Pemigatinib (IBI-375) | Fibroblast growth factor receptor 1/2/3 inhibitor | Unresectable or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 rearrangement | First patient in China treated in the global phase III study | 5/24/21 | Cancer |
| Isofol Medical AB, of Gothenburg, Sweden | Arfolitixorin | Folate receptor antagonist | Colorectal cancer | Agent study in people with first-line metastatic disease completed recruitment of 56 Japanese participants to meet PMDA regulatory requirements; top-line results for full study expected in first half of 2022 | 5/6/21 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Erleada (apalutamide) | Androgen receptor inhibitor | Metastatic castration-sensitive prostate cancer | No significant differences in quality of life, using the Brief Pain Inventory-Short Form and Functional Assessment of Cancer Therapy-Prostate questionnaires, between patients who received Erleada plus androgen deprivation therapy (ADT) and patients who received ADT alone | 5/26/21 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor | XPO1 inhibitor | Endometrial cancer after combination chemotherapy | Started recruiting patients for the 248-patient KCP-330-024/ENGOT-EN5/SIENDO study; primary endpoint is progression-free survival | 5/26/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1 inhibitor | Triple-negative breast cancer | Pivotal Keynote-522 combination trial with chemotherapy preoperatively (neoadjuvant) and as single agent (adjuvant) after surgery met co-primary endpoint of event-free survival vs. neoadjuvant chemotherapy alone in people with high-risk early stage disease after previously meeting endpoint of pathological complete response | 5/13/21 | Cancer |
| Morphosys AG, of Planegg, Germany, and Incyte Corp., of Wilmington, Del. | Tafasitamab | CD19-targeting monoclonal antibody | Diffuse large B-cell lymphoma | First of about 880 participants dosed in Frontmind study assessing tafasitamab + lenalidomide in addition to rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) vs. R-CHOP alone as front-line treatment; primary endpoint is investigator-assessed progression-free survival | 5/11/21 | Cancer |
| Oncopeptides AB, of Stockholm | Pepaxto (melphalan flufenamide) | Peptide-drug conjugate | Relapsed refractory multiple myeloma | In the Ocean study, Pepaxto was noninferior to pomalidomide for progression-free survival (PFS); hazard ratio for PFS favoring Pepaxto was 0.817 (p=0.0640); median PFS for the Pepaxto was more than 40% higher than for the pomalidomide; overall response rate was 32.1% for Pepaxto vs. 26.5% for pomalidomide | 5/25/21 | Cancer |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Libtayo (cemiplimab) | PD-1 inhibitor | Cervical cancer | In overall population with recurrent/metastatic disease, study drug (n=304) vs. chemotherapy (n=304) resulted in 31% reduction in risk of death (1-sided p=0.00011), 25% reduction in disease progression (1-sided p=0.00048); overall response rate was 16% (1-sided p=0.00004) vs. 6% with chemotherapy (n=19); Libtayo also showed improved overall survival in squamous cell carcinoma and adenocarcinoma subgroups | 5/12/21 | Cancer |
| Tavanta Therapeutics, of Radnor, Pa. | TAVT-45 (abiraterone acetate) | Granule formulation of the antiandrogen antineoplastic agent abiraterone acetate | Metastatic castrate sensitive prostate cancer and metastatic castrate-resistant prostate cancer | First patient treated in the study comparing TAVT-45 to Zytiga (abiraterone acetate, Johnson & Johnson), both in combination with prednisone, primary objective of the trial is to establish therapeutic equivalence between the 2 drugs; secondary objective is to characterize the multiple-dose pharmacokinetic profile of TAVT-45; the 108-patient study is expected to be fully enrolled within the second quarter of 2022 | 5/25/21 | Cancer |
| Telix Pharmaceuticals Inc., of Melbourne, Australia | TLX-591 (177Lu-DOTA-rosopatamab) | Antibody-based radioimmunoconjugate targeting prostate-specific membrane antigen | Advanced metastatic castrate-resistant prostate cancer | Initiated Australian sites in the 390-patient phase III Prostact study measuring progression-free survival as the primary endpoint; secondary endpoints include overall survival and quality-of-life assessment; plans to open additional global sites during the second half of 2021, subject to regulatory approvals | 5/9/21 | Cancer |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Fish oil | Elevated triglycerides | In the Reduce-It study, prespecified and post-hoc analyses showed Vascepa reduced first and total primary and key secondary endpoint events by approximately 30% to 40% in patients with dyslipidemia, defined as triglyceride levels of TG ?200 and high-density lipoprotein cholesterol levels ?35 mg/dL; post-hoc analysis showed heart failure and new heart failure requiring hospitalization was reduced in patients who achieve serum eicosapentaenoic acid levels higher than approximately 150 µg/mL | 5/17/21 | Cardiovascular |
| Bristol Myers Squibb Co., of New York | Mavacamten | Myosin inhibitor | Obstructive hypertrophic cardiomyopathy | Interim results from ongoing 5-year Mava-LTE extension study of Explorer-HCM trial showed durable improvement in left ventricular outflow tract gradients, diastolic function N-terminal-pro hormone B-type natriuretic peptide and symptoms | 5/3/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Myosin stimulator | Heart failure | Secondary analysis of Galactic-HF trial, published simultaneously in Journal of American College of Cardiology, showed effect of study drug on primary composite endpoint of heart failure events or cardiovascular death vs. placebo was consistent across most prespecified subgroups, with progressively larger treatment effect with decreasing ejection fraction (interaction p=0.004) | 5/17/21 | Cardiovascular |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Atopic dermatitis | Data from the Measure Up 1 and 2 studies published in The Lancet showed placebo-adjusted EASI-75 at week 16 was 53.3% and 46.9% for the 15-mg dose and 63.4% and 59.6% for the 30-mg dose for the 2 studies, respectively; placebo-adjusted proportion of patients who achieved a vIGA-AD response at week 16 was 39.8% and 34% for the 15-mg dose and 53.6%and 47.4% for the 30-mg dose for the 2 studies, respectively (all p<0.0001); 15 16 30 in the ad up study, published separately lancet, at week 16, proportion of patients who had achieved easi-75 was 65% for upadacitinib mg plus topical corticosteroid and 77% corticosteroids compared to 26% alone (p<0.0001 both doses); a viga-ad response 40% 59% 11% doses)< td> | 5/24/21 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | ARQ-151 (roflumilast topical cream) | PDE4 inhibitor | Plaque psoriasis | Presented data from pivotal DERMIS-1 and -2 trials, which met primary efficacy endpoint of IGA success at 8 weeks for 0.3% dose vs. vehicle (DERMIS-1: 42.4% vs. 6.1%; DERMIS-2: 37.5% vs. 6.9%, respectively, p<0.001 for both)< td> | 5/6/21 | Dermatologic |
| Concert Pharmaceuticals Inc. | CTP-543 | Janus kinase inhibitor | Moderate to severe alopecia areata | Started the 440-patient Thrive-AA2 study; primary endpoint is percent of patients achieving a SALT score ? 20 at week 24; top-line results expected in the second half of 2022 | 5/27/21 | Dermatologic |
| Incyte Inc., of Wilmington, Del. | Ruxolitinib cream | JAK inhibitor | Vitiligo | Top-line results from pivotal True-V1 and True-V2 trial in adolescents and adults showed both trials met primary endpoint (p<0.0001 for both), demonstrating significantly more patients treated with ruxolitinib cream 1.5% twice daily achieved a ?75% improvement from baseline in facial vitiligo area scoring index (f-vasi75) vs. those given vehicle control at week 24; trials also met secondary endpoints, including patient-reported outcomes< td> | 5/17/21 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib cream | Pan-JAK inhibitor | Moderate to severe chronic hand eczema | First patient enrolled in the first of 2 pivotal studies; primary endpoint for both studies is the Investigator’s Global Assessment for chronic hand eczema treatment success at week 16; secondary endpoints at week 16 include reduction of itch and pain scores of ?4 points measured by the Hand Eczema Symptom Diary as well as Hand Eczema Severity Index improvements of at least 75% and at least 90% | 5/18/21 | Dermatologic |
| Meiji Seika Pharma Co. Ltd., of Tokyo | DMB-3115 | Biosimilar to Stelara (ustekinumab, Johnson & Johnson) | Plaque psoriasis | Started the 590-patient study comparing the efficacy, safety, pharmacokinetics and immunogenicity of DMB-3115 and reference products | 5/21/21 | Dermatologic |
| Reistone Biopharma Co. Ltd., of Shanghai | SHR-0302 | JAK1 inhibitor | Atopic dermatitis | First of 330 participants 12 and older with moderate to severe disease dosed in trial evaluating 4 mg or 8 mg vs. placebo for up to 52 weeks | 5/13/21 | Dermatologic |
| Aeglea Biotherapeutics Inc., of Austin, Texas | Pegzilarginase | Arginase-I stimulator | Arginase 1 deficiency | Randomization for pivotal Peace trial completed, with top-line data expected in fourth quarter of 2021 | 5/3/21 | Endocrine/Metabolic |
| Aeterna Zentaris Inc., of Charleston, S.C. | Macimorelin | Ghrelin receptor agonist | Growth hormone deficiency | Pivotal Detect-Trial initiated in at least 80 children with suspected GHD to test agent's ability to diagnose childhood-onset disease | 5/13/21 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Oxlumo (lumasiran) | Hydroxyacid oxidase 1 modulator | Primary hyperoxaluria type 1 | 12-month analysis of Illuminate-A study showed, among those with valid renal ultrasounds at baseline (n=24), 46% (11/24) improved in nephrocalcinosis grade relative to baseline, 17% (4/24) remained stable and 13% (3/24) worsened; of 14 with baseline nephrocalcinosis and available ultrasounds, 79% (11/14) improved relative to baseline and 73% (8/11) of these improved in both kidneys | 5/3/21 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR gene inhibitor | Transthyretin-mediated amyloidosis | Started a clinical study of a biannual dosing regimen, being conducted during a randomized treatment extension period in the HELIOS-A phase III study | 5/11/21 | Endocrine/Metabolic |
| Chiasma Inc., of Needham, Mass. | Mycapssa (oral octreotide capsules) | Somatostatin receptor agonist | Acromegaly | Patient-reported outcomes from Mpowered trial showed significantly improved quality of life and work productivity after transitioning from long-acting injectable somatostatin analogues; significant improvements in index scores of EuroQol-5 Dimensions-5 Levels measuring quality of life over 5 domains (mobility, ability to self-care, ability to undertake usual activities, pain and discomfort, and anxiety and depression) as well as in comparing baseline of run-in to end of run-in on the Work Productivity and Activity Impairment Questionnaire: Specific Health Problem -impairment at work/reduced on-the-job effectiveness (p=0.024), work productivity loss (p=0.022) and activity impairment (p=0.022) | 5/27/21 | Endocrine/Metabolic |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex Faspro (daratumumab and hyaluronidase-fihj) | Subcutaneous monoclonal antibody targeting CD38 | Newly diagnosed light chain amyloidosis | In the Andromeda study, after a median follow-up of 20.3 months, 59% of patient taking Darzalex Faspro plus Velcade (bortezomib, Takeda Pharmaceutical Co. Ltd.), cyclophosphamide and dexamethasone (D-Vcd) had an overall complete hematologic response compared to 19% of patients taking Vcd; 79% of patients taking D-Vcd achieved a very good partial response or better compared to 50% of patients taking Vcd | 5/26/21 | Endocrine/Metabolic |
| Poxel SA, of Lyon, France | Imeglimin | Tetrahydrotriazine compound | Type 2 diabetes | After 52 weeks of treatment of Japanese patients with imeglimin added to other oral antidiabetics, HbA1c improvements from baseline ranged from -0.56 ± 0.08 to -0.92 ± 0.11 | 5/20/21 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen, Denmark | Dasiglucagon | Glucagon analogue | Severe hypoglycemia in diabetes | Data published in Diabetes Care showed administration resulted in reversal of hypoglycemia (with a median recovery time of 10 minutes) with 99% of trial participants reaching recovery within 15 minutes | 5/3/21 | Endocrine/Metabolic |
| Shield Therapeutics plc, of London | Feraccru/Accrufer (ferric maltol) | Oral iron replacement therapy | Non-severe active inflammatory bowel disease and iron-deficiency anemia | Data published in Inflammatory Bowel Diseases showed ferric maltol did not meet the prespecified noninferiority margin for hemoglobin responder rate compared to intravenous (I.V.) iron over 12 weeks; over 52 weeks, ferric maltol produced efficacy comparable to I.V. iron in maintaining hemoglobin improvements and increasing ferritin | 5/18/21 | Gastrointestinal |
| Bayer AG, of Leverkusen, Germany | Finerenone | Nonsteroidal, selective mineralocorticoid receptor antagonist | Chronic kidney disease and type 2 diabetes | The Figaro-DKD study met its primary endpoint, reducing the composite risk of time to first occurrence of cardiovascular (CV) death or nonfatal CV events (myocardial infarction, stroke or hospitalization for heart failure); data to be presented at an upcoming scientific meeting | 5/10/21 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Yselty | GnRH receptor antagonist | Moderate to severe endometriosis-associated pain | Completed enrollment of 486 women in the Edelweiss 3 study; data from the primary endpoint expected in the fourth quarter of 2021 | 5/4/21 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva, Switzerland | Yselty (linzagolix) | GnRH receptor antagonist | Heavy menstrual bleeding due to uterine fibroids | In the Primrose 1 study, 76-week data were consistent with the data from the Primrose 2 study; the 100-mg dose and the 200-mg dose plus hormonal add-back therapy produced evidence of bone mineral density recovery | 5/20/21 | Genitourinary/Sexual Function |
| Catalyst Biosciences Inc., of South San Francisco | Marzeptacog alfa (MarzAA) | Subcutaneous next-generation engineered recombinant coagulation factor VIIa | Congenital hemophilia A or B with inhibitors | Dosed first patient in pivotal Crimson 1 study in adults and adolescents; trial will assess effectiveness vs. standard of care, using up to 3 doses to treat a bleeding episode; submission of first report to data and safety monitoring board expected in 2021 | 5/5/21 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm | Pegcetacoplan | Complement C3 inhibitor | Treatment-naïve paroxysmal nocturnal hemoglobinuria | In the Prince study, 86% of patient taking pegcetacoplan achieved hemoglobin stabilization, defined as an avoidance of a >1 g/dL decrease in hemoglobin levels in the absence of transfusions, compared to 0% of patients on standard of care (p<0.0001); mean decrease in lactate dehydrogenase was 90% for pegcetacoplan and 14% standard of care (p<0.0001)< td> | 5/25/21 | Hematologic |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Data from the Aurora 1 study published in The Lancet showed 41% of patients treated with Lupkynis plus mycophenolate mofetil (MMF) and low-dose corticosteroids had a complete renal response compared to 23% of patients taking only MMF and low-dose corticosteroids (p<0.0001); drug also met all prespecified hierarchical secondary endpoints< td> | 5/10/21 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Interim analysis of the Aurora 2 study showed patients taking Lupkynis had a -3/1-mg/mg change in urine protein creatinine ratio after 103 weeks compared to -2.1 mg/mg for placebo (p=0.0004); mean estimated glomerular filtration rate remained stable over 104 weeks for Lupkynis and placebo | 5/20/21 | Immune |
| Medeor Therapeutics Inc., of South San Francisco | MDR-101 | Allogeneic HLA-matched living donor hematopoietic stem cell therapy | Kidney transplant rejection | Enrollment and kidney transplantation completed in pivotal trial assessing establishment of immune tolerance that allows for complete withdrawal of immunosuppressive drugs | 5/12/21 | Immune |
| Constant Therapeutics LLC, of Boston | TXA-127 | Angiotensin II receptor modulator | COVID-19 | Agent added to NIH-funded Activ-4d trial testing multiple compounds, each against placebo; TXA-127 segment expected to enroll 300 participants | 5/12/21 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon lambda | Interferon receptor modulator | COVID-19 | Additional arm of up to 800 high-risk people will be added to ongoing Together platform study to assess agent in outpatients with infection; primary endpoint is reduction in ER visits and hospitalizations vs. placebo | 5/3/21 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | GM-CSF ligand inhibitor | COVID-19 | Data from Live-Air trial, published on preprint server medRxiv, showed survival without need for ventilator increased by 54% to 90% over treatment with remdesivir and/or corticosteroids in people newly hospitalized with COVID-19 pneumonia | 5/5/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | V-114 | 15-valent pneumococcal conjugate vaccine | Pneumococcal disease prophylaxis | In the Pneu-Direction study, infants given a mixed dose schedule of the currently available 13-valent pneumococcal conjugate vaccine (PCV13) followed by V-114 were generally comparable for the 13 serotypes targeted by both vaccines; in the Pneu-Plan study, immune responses were generally comparable to PCV13 for the 13 shared serotypes when V-114 was used as a catch-up regimen in children 7 months to 17 years of age who were either pneumococcal vaccine-naïve or who previously received a partial or full regimen of a lower valency pediatric pneumococcal conjugate vaccine; serotypes 22F and 33F immunogenicity in Pneu-Plan were higher for V-114 than PCV13 | 5/20/21 | Infection |
| Novan Inc., of Durham, N.C. | SB-206 (berdazimer sodium) | Topical antiviral gel | Molluscum contagiosum | Final participant completed last week-12 visit in pivotal B-Simple4 study; top-line results targeted by end of second quarter of 2021 | 5/3/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | COVID-19 spike glycoprotein modulator | COVID-19 | Pediatric expansion initiated in ongoing pivotal Prevent-19 trial and set to enroll up to 3,000 people age 12 to 17 | 5/3/21 | Infection |
| Omeros Corp., of Seattle | Narsoplimab | MASP-2 inhibitor | COVID-19 | Preliminary results from second cohort of critically ill patients treated in Bergamo, Italy, showed that 80% of patients recovered, survived and were discharged from hospital; there 2 deaths; detailed data will be published in peer-reviewed journal | 5/28/21 | Infection |
| Pfizer Inc., of New York, Biontech SE, of Mainz, Germany | 20vPnC; Pfizer-Biontech COVID-19 Vaccine booster | 20-valent pneumococcal conjugate vaccine; mRNA based vaccine against SARS-CoV-2 | Pneumococcal disease and COVID-19 prophylaxis | Enrolled first of 600 participants in the study of adults ages 65 and older testing the coadministration of the vaccines; primary endpoint is safety; secondary endpoints include immune responses produced by each of the vaccines | 5/24/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGEN-COV (casirivimab with imdevimab) | Monoclonal antibody targeting SARS-CoV-2 | COVID-19 | Detailed results showed significantly reduced risk of hospitalization or death, shortened symptom duration and reduced viral load in non-hospitalized patients; results showed those treated with 1,200 mg or 1,400 mg showed 70% (p=0.0024) and 71% (p<0.0001) 7 10 14 29 reduced risk of hospitalization or death through day vs. placebo, as well 4-day shorter time to symptom resolution, with a median days both regen-cov groups compared placebo; viral load by 0.71 log10 copies ml and 0.86 in placebo (p<0.0001 for both)< td> | 5/17/21 | Infection |
| Russian Direct Investment Fund, of Moscow | Sputnik V | Adenoviral-based vaccine | COVID-19 prophylaxis | In a study run by Argentina's Institute of Virology of the National University of Cordoba and the Government of Cordoba, 99.65% of participants developed IgG antibodies to COVID-19 on 42nd day after receiving the second dose; 85.5% of participants had IgG antibodies to COVID-19 on 14th day after receiving the first dose | 5/24/21 | Infection |
| Sanofi SA, of Paris, and Glaxosmithkline plc, of London | COVID-19 vaccine candidate | Adjuvanted recombinant protein-based vaccine | COVID-19 prophylaxis | Started enrollment of more than 35,000 volunteers into the study testing a vaccine formulation targeting the original D.614 virus (Wuhan), followed by a second stage that will evaluate a second formulation targeting the B.1.351 (South African) variant; primary endpoint of the study is the prevention of symptomatic COVID-19; secondary endpoints are the prevention of severe COVID-19 disease and prevention of asymptomatic infection | 5/27/21 | Infection |
| Seqirus USA Inc., of Summit, N.J. | Flucelvax Quadrivalent | Cell-based quadrivalent seasonal influenza vaccine | Seasonal influenza | Vaccine was as safe and immunogenic as standard quadrivalent seasonal influenza vaccine in children 6 months through <4 20 2019 years of age during u.s. influenza season< td> | 5/3/21 | Infection |
| Sinovant Sciences Ltd., of Beijing, and Nabriva Therapeutics Ireland DAC, of Dublin | Lefamulin | Pleuromutilin antibiotic | Community-acquired bacterial pneumonia | Lefamulin was noninferior to moxifloxacin, with 76.8% of lefamulin-treated patients meeting the efficacy endpoint of Investigator Assessment of Clinical Response at Test of Cure, compared to 71.4% of patients treated with moxifloxacin | 5/25/21 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-003 | Dengue vaccine | Dengue prophylaxis | In the TIDES (DEN-301) study, 3 years after the second dose, overall vaccine efficacy (VE) was 62% against virologically-confirmed dengue, with 65% VE in seropositive individuals and 54.3% VE in seronegative individuals; VE against hospitalized dengue was 83.6%, with 86% VE in seropositive individuals and 77.1% VE in seronegative individuals | 5/22/21 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | Sci-B-Vac | Hepatitis B large envelope protein modulator | Hepatitis B virus infection | The Lancet Infectious Diseases published results from pivotal Protect study that showed 10 µg of VBI’s 3-antigen vaccine candidate achieved superior seroprotection rate in adults vs. 20 µg of single-antigen HBV vaccine Engerix-B (Glaxosmithkline plc) | 5/12/21 | Infection |
| Veru Inc., of Miami | Sabizabulin (VERU-111) | Tubulin alpha/beta inhibitor | COVID-19 | First of 300 participants hospitalized with infection and at high risk of acute respiratory distress syndrome enrolled; primary efficacy endpoint is proportion who die on study up to day 60 | 5/19/21 | Infection |
| Xenothera SAS, of Nantes, France | XAV-19 | Anti-SARS-CoV-2 polyclonal antibody | COVID-19 | Started Eurovax study to recruit 722 patients with moderate COVID-19, hospitalized or monitored remotely; trial will take place in Greece, Bulgaria, Romania, Spain and Turkey | 5/3/21 | Infection |
| Amo Pharma Ltd., of London | AMO-02 (tideglusib) | Disrupts pathogenic RNA repeat in CDM1 and inhibits excess levels of kinase GSK3? | Congenital myotonic dystrophy | Activated 4 additional trial sites in Canada and the U.S. for pivotal Reach-CDM study |
5/17/21 | Musculoskeletal |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 (sodium phenylbutyrate-taurursodiol) | Histone deacetylase inhibitor | Amyotrophic lateral sclerosis | Phoenix study expected to begin in the third quarter of 2021; primary efficacy outcome will be a joint assessment of ALSFRS-R total score progression over 48 weeks and survival; secondary efficacy outcomes will include change in slow vital capacity, serial assessments of patient-reported outcomes and ventilation-free survival rates | 5/12/21 | Musculoskeletal |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Chaperonin stimulator; Hsp70 stimulator | Amyotrophic lateral sclerosis | The ORARIALS-01 pivotal trial did not meet its primary and secondary endpoints evaluating impact on function and survival; no important safety signals were reported in the trial | 5/7/21 | Musculoskeletal |
| Acelrx Pharmaceuticals Inc., of Hayward, Calif. | Dsuvia (sufentanil) | Opioid analgesic | Pain associated with hip or knee replacement | Plans to run an investigator-initiated study comparing Dsuvia to standard of care in 100 patients undergoing same-day hip or knee joint replacement; primary endpoint is length of stay in the hospital; secondary endpoints include average total morphine milligram equivalents dosed per patient, ability to complete physical therapy prior to discharge from the postanesthesia care unit and overall cost of the stay | 5/6/21 | Neurology/Psychiatric |
| Acelrx Pharmaceuticals Inc., of Hayward, Calif. | Dsuvia (sufentanil) | Opioid receptor mu agonist | Pain | Investigator-initiated study at Tampa General Hospital will evaluate drug in 100 people with sickle cell disease who present to emergency department (ED) with vaso-occlusive crisis for management of moderate to severe acute pain until I.V. line started for opioid and other parenteral medications; endpoints include time from ED arrival to first analgesic medication, ED length of stay, hospital admission rates, patient and clinician satisfaction and adverse events | 5/20/21 | Neurology/Psychiatric |
| Baudax Bio Inc., of Malvern, Pa. | Anjeso (meloxicam) | Non-opioid, once daily, intravenous nonsteroidal anti-inflammatory agent | Postoperative pain | Data from phase IIIb study testing preoperative administration prior to colorectal surgery published in Pain Management showed drug was well-tolerated, with 92%+ of patients reporting satisfaction with postoperative pain medication; treated patients also experienced statistically significant reductions in opioid consumption, time to first bowel sounds, time to first bowel movement and time to hospital discharge, all compared to placebo | 5/17/21 | Neurology/Psychiatric |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | BI-425809 | Glycine transporter-1 inhibitor | Cognitive impairment associated with schizophrenia | Plans to run 3 phase III studies (CONNEX-1, -2 and -3); primary endpoint of all 3 studies is the change in overall composite T-score of the Measurement and Treatment Research to Improve Cognition in Schizophrenia Consensus Cognitive Battery after 26 weeks of treatment | 5/24/21 | Neurology/Psychiatric |
| Minerva Neurosciences Inc., of Waltham, Mass. | Roluperidone | 5-HT 2a receptor antagonist; opioid receptor sigma antagonist 2 | Schizophrenia | 40-week open-label extension showed continuous improvement in negative symptoms, measured by Positive and Negative Syndrome Scale Marder Negative Symptom Factor Score, over 1 year in those who received 64 mg (n=167, mean improvement of 7.5 points) and 32 mg (n=166, mean improvement of 6.8 points) doses and continuous improvement over 1 year in Personal and Social Performance total score (mean of 14.5 and 12.3 points, respectively) | 5/11/21 | Neurology/Psychiatric |
| Multidisciplinary Association for Psychedelic Studies (MAPS), of San Jose, Calif. | MDMA-assisted therapy | 3,4-methylenedioxymethamphetamine-assisted therapy | Severe post-traumatic stress disorder | Results from demonstrated 88% of participants who received 3 controlled and supervised MDMA-assisted therapy sessions experienced a clinically significant reduction in symptoms, with 67% no longer qualifying for PTSD diagnosis in comparison to 32% of participants randomized to placebo; the study was conducted by MAPS Public Benefit Corp., a wholly owned subsidiary of MAPS, and results were published in Nature Medicine | 5/11/21 | Neurology/Psychiatric |
| Pacira Biosciences Inc., of Parsippany, N.J. | Exparel (bupivacaine) | Bupivacaine liposome injectable suspension | Pain after lower extremity surgery | Exparel didn’t demonstrate statistical significance for reduction in cumulative pain scores from 0 to 96 hours as measured by the area under the curve vs. bupivacaine HCl; Exparel did reduce cumulative pain scores from 24 to 96 hours post-surgery (p<0.001), 12 24 96 total opioid consumption from to hours post-surgery (p<0.01) and area under the curve cumulative pain scores (p<0.02)< td> | 5/26/21 | Neurology/Psychiatric |
| Prilenia Therapeutics B.V., of Naarden, the Netherlands | Pridopidine | S1R agonist | Huntington's disease | Proof-HD trial enrolled 120 participants on schedule, reaching 25% of goal, and remains on target to complete enrollment by fourth quarter of 2021 | 5/13/21 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Synthetic neurosteroid nasal spray | Social anxiety disorder | Started the 200-patient Palisade-1 study testing the ability of PH-94B to reduce anxiety in a laboratory-simulated public speaking challenge; top-line results expected in mid-2022 | 5/26/21 | Neurology/Psychiatric |
| Zosano Pharma Corp., of Fremont, Calif. | Qtrypta (zolmitriptan) | Transdermal microneedle delivery of the serotonin receptor agonist zolmitriptan | Migraine | Data published in The Journal of Headache and Pain showed 81% of migraine attacks achieved pain relief at 2 hours post-dose, pain freedom in 44% of attacks and most bothersome symptom freedom in 62% of attacks | 5/26/21 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel (cannabidiol transdermal gel) | Cannabinoid CB2 receptor modulator | Fragile X syndrome | Following FDA guidance, confirmatory pivotal Reconnect trial will enroll about 200 children and adolescents, including about 160 with complete methylation and about 40 with partial methylation of FMR1 gene; primary endpoint will be change in Aberrant Behavior Checklist-Community FXS Specific Social Avoidance subscale in those with complete methylation of FMR1 gene; trial expected to begin in third quarter of 2021 | 5/5/21 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Marlborough, Mass. | Latuda (lurasidone) | 5-HT/dopamine D2 antagonist | Bipolar depression | Post-hoc analysis of pooled data from 2 placebo-controlled studies showed treatment improved psychic anxiety from baseline to week 6 both as monotherapy (p<0.001 6 for 20-60 mg d; p<0.01 80-120 d) and as adjunctive therapy with lithium valproate (p<0.01); treatment also associated improvement in somatic anxiety from baseline to week numeric observed monotherapy (p="0.09" p="NS" d)< td> | 5/3/21 | Neurology/Psychiatric |
| Adverum Biotechnologies Inc., of Redwood City, Calif. | ADVM-022 (AAV.7m8 vector encoding aflibercept) | VEGF gene inhibitor | Wet age-related macular degeneration | Long-term data from Optic trial showed 60% of participants (9/15) were injection-free beyond 1 year following 2 x 10^11 vg/eye dose and 87% (13/15) were injection free following 6 x 10^11 vg/eye dose | 5/1/21 | Ocular |
| Eyenovia Inc., of New York | Microline | Formulation of the cholinergic agonist pilocarpine | Presbyopia | In the Vision-1 study, Microline improved the proportion of patients who had a 3-line or more improvement in distance corrected near visual acuity in low light conditions at 2 hours post-treatment compared to placebo; plans to run a second phase III study, Vision-2 | 5/25/21 | Ocular |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab-dbll) | VEGF-A ligand inhibitor | Diabetic macular edema | 1-year results from Kestrel and Kite studies showed Beovu, dosed at 6 mg, met primary endpoints of noninferiority in change in BCVA from baseline vs. aflibercept 2 mg; 3-mg arm, tested only in Kestrel, did not meet primary endpoint | 5/1/21 | Ocular |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | After 1 year in the Merlin study testing 4-week dosing regimens, Beovu was noninferior to Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) for best corrected visual acuity from baseline and superior to Eylea on select anatomical secondary endpoints; idiopathic orbital inflammation was 9.3% for Beovu and 4.5% for Eylea; rate of vision loss of 15 letters or more was 4.8% for Beovu and 1.7% for Eylea | 5/28/21 | Ocular |
| Regentree LLC, a joint venture of Regenerx Biopharmaceuticals Inc., of Rockville, Md., and Gtreebnt Co. Ltd., of Seongnam, South Korea | RGN-259 | Thymosin beta 4 ligand; eye drops | Dry eye syndrome | Results of additional analysis of the phase III Arise-3 study showed improvement of ocular grittiness, a secondary endpoint, and statistically significant differences were seen with improvement in ocular discomfort during the first week of treatment; in addition, statistically significant differences were seen in the central corneal fluorescein staining score for sign efficacy at 2 weeks after treatment in a subpopulation of Arise-3 and in the pooled population of 3 trials of Arise-1, -2, and -3 comprising corneal sum fluorescein staining score at the baseline (Arise-3, p = 0.0352; pooled data, p = 0.0074) and in a subpopulation of Arise-2 and in the pooled population comprising inferior corneal fluorescein staining score and Schirmer's test score at the baseline (Arise-2, p = 0.0057; pooled data, p = 0.0196) | 5/14/21 | Ocular |
| Sandoz, unit of Novartis AG, of Basel, Switzerland | Aflibercept biosimilar | Dual VEGF-A/VEGF-B ligand inhibitor | Wet age-related macular degeneration | First participant expected to enroll shortly in confirmatory Mylight trial vs. Eylea (aflibercept, Bayer AG/Regeneron Pharmaceuticals Inc.); primary endpoint is mean change in BCVA score from baseline to week 8 | 5/3/21 | Ocular |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif. | TP-03 | Non-competitive antagonist of insect and arachnid GABA-Cl channels | Demodex blepharitis | Commenced enrollment in Saturn-2, a pivotal phase III trial evaluating safety and efficacy | 5/7/21 | Ocular |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Semaglutide (2.4 mg once-weekly subcutaneous) | GLP-1 agonist | Obesity | 43.8% of participants in phase IIIa Step program achieved clinically meaningful improvement in weight-related quality of life score at week 68 and 51.2% vs. 32.9% for placebo had increased weight-related physical function score indicating improvements in daily physical activities | 5/13/21 | Other/Miscellaneous |
| Astrazeneca plc, of Cambridge, U.K. | Fasenra (benralizumab) | IL-5 receptor antagonist | Asthma | Meltemi open-label extension trial showed drug was well-tolerated for up to 5 years, with long-term safety profile consistent with previous phase III trials in adults with severe disease | 5/19/21 | Respiratory |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Monoclonal antibody targeting interleukin-4 and interleukin-13 pathways | Moderate to severe asthma | Data from Voyage trial showed drug significantly reduced severe asthma attacks, and within 2 weeks rapidly improved lung function in children ages 6 to 11 with uncontrolled moderate to severe asthma, with evidence of type 2 inflammation; reduced rate of severe asthma attacks, with 65% (p<0.0001) 1 and 59% (p<0.0001) average reduction over year vs. placebo< td> | 5/17/21 | Respiratory |
| Actinium Pharmaceuticals Inc., of New York | Iomab-B | Antibody-radiation conjugate targeting CD45 | Active, relapsed or refractory acute myeloid leukemia | In the Sierra study, all 49 patients who received a therapeutic dose of Iomab-B had a successful bone marrow transplant compared to 18% of the 57 patients who received conventional care; all 30 of the patients who crossed over from conventional care to Iomab-B had a successful transplant | 6/15/21 | Cancer |
| Asieris Pharmaceuticals Co. Ltd., of Shanghai, and Photocure ASA, of Oslo, Norway | APL-1702 (Cevira) | Hexaminolevulinate; photodynamic therapy | High-grade squamous intraepithelial lesions | First European patient dosed | 6/22/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Calquence (acalabrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Final results of Elevate-RR trial showed lower incidence of all-grade atrial fibrillation in patients treated with calquence vs.ibrutinib (9.4% [n=25/266] versus 16.0% [n=42/263]); more tolerable option with reduced cardiovascular toxicity and overall fewer discontinuations due to adverse events | 6/7/21 | Cancer |
| Beigene Ltd., of Cambridge, Mass., and Beijing | Ociperlimab (BGB-A1217) | Anti-TIGIT antibody | Non-small-cell lung cancer | First patient dosed in AdvanTIG-302 trial testing combination with tislelizumab in first-line treatment of patients with locally advanced, unresectable or metastatic disease whose tumors exhibit high PD-L1 expression and do not harbor EGFR-sensitizing mutations or ALK translocations | 6/17/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Breyanzi (lisocabtagene maraleucel) | CAR T therapy; anti-CD19 | Second-line relapsed or refractory large B-cell lymphoma | Positive top-line results from Transform trial evaluating Breyanzi vs. salvage therapy followed by high-dose chemotherapy and hematopoietic stem cell transplant; met its primary endpoint as significant improvement in event-free survival; secondary endpoints of complete response rate and progression-free survival compared to standard of care; consistent safety results and no new safety concerns | 6/10/21 | Cancer |
| Byondis BV, of Nijmegen, the Netherlands | [vic-]trastuzumab duocarmazine (SYD-985) | Antibody-drug conjugate targeting HER2 | Pretreated HER2-positive unresectable locally advanced or metastatic breast cancer | The Tulip study met its primary endpoint with [vic-]trastuzumab duocarmazine improving progression-free survival compared to physician’s choice; study demonstrated preliminary support for improvements in overall survival; data to be presented at future scientific conferences | 6/7/21 | Cancer |
| Cel-Sci Corp., of Vienna, Va. | Multikine | CD4 agonist; IL-2 receptor agonist; unspecified cytokine receptor agonist | Advanced primary squamous cell carcinoma of the head and neck | Patients treated with the Multikine treatment regimen plus standard of care (SOC) had overall survival benefit of 14.1% at 5 years compared to standard of care alone (p=0.0236, HR=0.68); OS of Multikine plus CIZ (cyclophosphamide, indomethacin, zinc-multivitamins) plus SOC was 72.4% at 3 years, 62.7% at 5 years, Multikine (no CIZ) plus SOC was 78.8% at 3 years, 55.5% at 5 years and SOC alone was 67.5% at 3 years, 48.6% at 5 years | 6/28/21 | Cancer |
| Corcept Therapeutics Inc., of Menlo Park, Calif. | Relacorilant | Nonsteroidal, selective modulator of the glucocorticoid receptor | Metastatic pancreatic cancer | Interim results of phase III Reliant study (n=43) in combination with nab-paclitaxel, at cutoff date of April 15, 2021, with 31 efficacy-evaluable patients showed 6% of patients had partial response; 48% of patients showed stable disease, and 6% of patients had stable disease greater than 18 weeks; consistent with known safety profile; combination therapy was well-tolerated; expected to initiate phase III pivotal trial in the first quarter of 2022 | 6/22/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Enhertu (trastuzumab deruxtecan) | HER2-directed antibody-drug conjugate | HER2-positive metastatic breast cancer | First patient treated in the Destiny-Breast09 study comparing Enhertu to Enhertu plus Perjeta (pertuzumab, Roche Holding AG) and to a taxane plus Perjeta and Herceptin (trastuzumab, Roche); primary endpoint is progression-free survival; secondary endpoints include overall survival, objective response rate, duration of response and time to second progression or death | 6/14/21 | Cancer |
| Exelixis Inc., of Alameda, Calif., and Ipsen SA, of Paris | Cabometyx (cabozantinib) | Kinase inhibitor | First-line advanced hepatocellular carcinoma | Interim analysis of the 740-patient Cosmic-312 trial showed Cabometyx plus Tecentriq (atezolizumab, Roche Holding AG) reduced the risk of disease progression or death by 37% compared with Nexavar (sorafenib, Bayer AG) (HR=0.63, p=0.0012); overall survival favored Cabometyx plus Tecentriq but wasn't statistically significant and has a low probability of reaching statistical significance at the time of the final analysis; final results are anticipated in early 2022 | 6/28/21 | Cancer |
| Fibrogen Inc., of San Francisco | Pamrevlumab | Connective tissue growth factor inhibitor | Metastatic pancreatic cancer | Agent + standard of care included as first- or second-line therapy in Pancreatic Cancer Action Network's Precision Promise adaptive trial platform | 6/16/21 | Cancer |
| Innovent Biologics Inc., of San Francisco | Tyvyt (sintilimab) | Immunoglobulin G4 monoclonal antibody targeting PD-1 | Locally advanced recurrent or metastatic esophageal squamous cell carcinoma | In the interim analysis of the Orient-15 study, sintilimab plus chemotherapy (cisplatin plus paclitaxel or cisplatin plus 5-fluorouracil) improved overall survival compared to chemotherapy alone regardless of PD-L1 expression status; results will be presented at an upcoming medical meeting | 6/22/21 | Cancer |
| Kite, of Santa Monica, Calif., a unit of Foster City, Calif.-based Gilead Sciences Inc. | Yescarta (axicabtagene ciloleucel) | CAR T-cell therapy targeting CD19 | Second-line relapsed or refractory indolent follicular lymphoma | Interim analysis of the 359-patient Zuma-7 study showed Yescarta improved event-free survival compared to standard of care (SOC) (HR=0.398, p<0.0001); drug also had a better objective response rate than soc; overall survival trend favored yescarta over data will be submitted for presentation at future medical congress; further analyses are planned the future< td> | 6/28/21 | Cancer |
| Kyowa Kirin Inc., of Bedminster, N.J., an affiliate of Kyowa Kirin Co. Ltd. | Poteligeo (mogamulizumab) | Humanized monoclonal antibody targeting CCR4 | Cutaneous T-cell lymphoma | Post-hoc analysis from Mavoric study of mogamulizumab in patients with B1 and B2 levels of blood tumor burden reported more benefit in progression-free survival as a relative reduction in the risk of disease progression of 47%; median time-to-next-treatment, improvement in mSWAT vs. vorinostat; good tolerability with manageable safety profile | 6/10/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 humanized monoclonal antibody | First-line treatment of adult patients with persistent, recurrent or metastatic cervical cancer | Keynote-826 study (n=617) in combination with platinum-based chemotherapy (paclitaxel plus cisplatin or paclitaxel plus carboplatin) with or without bevacizumab met its dual primary endpoints; demonstrated statistically significant and clinically meaningful improvements in overall survival and progression-free survival compared to same platinum-based chemotherapy regimens with or without bevacizumab alone | 6/22/21 | Cancer |
| Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | Inhibitor of cyclin-dependent kinases 4 and 6 | HR+, HER2-negative metastatic breast cancer | Exploratory analysis of the Monalessa-3 study testing Kisqali plus fulvestrant showed median overall survival was 53.7 months for the combination compared to 41.5 months for fulvestrant alone (HR=0.73); in first-line patients, median OS was not reached for the combination compared to 51.8 months for fulvestrant alone (HR=0.64); in second-line patients, median OS was 39.7 months for the combination compared to 33.7 months for fulvestrant alone (HR=0.78) | 6/2/21 | Cancer |
| Novartis AG, of Basel, Switzerland | 177Lu-PSMA-617 | PSMA-targeted radioligand therapy | PSMA-positive metastatic castration-resistant prostate cancer | Data published in The New England Journal of Medicine show combination with standard of care (SOC) significantly improved both overall survival (p<0.001; median 15.3 vs. 11.3 months) and imaging-based progression-free survival (p<0.001; median, 8.7 3.4 soc alone; overall response rate in patients with measurable or non-measurable disease at baseline was 29.8% partial complete combo arm 1.7% soc-only (two-sided p<0.001)< td> | 6/23/21 | Cancer |
| Pfizer Inc., of New York | Talazoparib | PARP inhibitor | Metastatic castration-sensitive prostate cancer | First of 550 patients treated in the Talapro-3 trial; primary endpoint is radiographic progression-free survival; secondary endpoint is overall survival; anticipated primary completion date is late-2024 | 6/23/21 | Cancer |
| Point Biopharma Inc., of Indianapolis | PNT-2002 | 177Lu-PSMA targeted radioligand | PSMA expressing metastatic castration-resistant prostate cancer not eligible for chemotherapy | First of approximately 450 patients treated in the Splash study comparing PNT-2002 to Zytiga (abiraterone, Johnson & Johnson) or Xtandi (enzalutamide, Astellas Pharma Inc.); primary endpoint is radiographic progression-free survival; secondary endpoints include overall response rate, overall survival and pharmacokinetics | 6/4/21 | Cancer |
| Polyphor AG, of Allschwil, Switzerland | Balixafortide | CXCR4 chemokine antagonist | HER2-negative, locally recurrent or metastatic breast cancer | Fortress combination study with eribulin missed co-primary endpoint, showing no improvement in objective response rate vs. eribulin alone (13% vs. 13.7%; p=1) in third-line and later population (n=330 patients) followed for at least 6 months | 6/28/21 | Cancer |
| Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | HER2-positive metastatic breast cancer | After 29.6 months of follow-up in the HER2CLIMB study, median overall survival was 24.7 months for Tukysa plus capecitabine and trastuzumab and 19.2 months for capecitabine and trastuzumab alone (p=0.004); progression-free survival was 7.6 months for Tukysa combination compared to 4.9 months for the control arm (p<0.00001)< td> | 6/3/21 | Cancer |
| Servier Pharmaceuticals, of Boston | Vorasidenib | Oral, brain-penetrant dual inhibitor of mutant IDH1 and IDH2 | Low-grade glioma | Started enrolling patients with IDH-mutant grade 2 residual or recurrent low-grade glioma in phase III Indigo trial | 6/16/21 | Cancer |
| Sierra Oncology Inc., of San Mateo, Calif. | Momelotinib | JAK1/JAK2 inhibitor; ACVR1/ALK2 inhibitor | Myelofibrosis | Momentum trial fully enrolled 195 participants, exceeding target of 180 people; top-line data expected in first quarter of 2022 | 6/21/21 | Cancer |
| Soligenix Inc., of Princeton, N.J., | Hybryte (SGX-301 and Hypericin ointment 0.25%) | Photodynamic therapy, photosensitizers | Cutaneous T-cell lymphoma | Positive pivotal results from phase III Flash trial (n=169) showed 50% reduction in lesions in 16% of patients compared to 4% of patients in placebo group at 8 weeks (p=0.04) during cycle 1; in cycle 2 (n=155) for 12-week treatment group, response rate of 40% (p<0.0001 2 3 vs. the placebo treatment rate in cycle 1); statistically significant improvement (p<0.0001) between groups; additional analyses of hybryte showed reduction plaque (response 42%, p<0.0001 relative to 1) and patch 37%, p="0.0009" 3, 49% patients demonstrated a response (p<0.0001 receiving was well-tolerated safe cycles< td> | 6/22/21 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Subcutaneously administered single-domain antibody against PD-L1 | Sarcoma | Independent data monitoring committee for the Envasarc pivotal trial recommended that it proceed as planned following the review of safety data from more than 20 patients enrolled into the trial to date | 6/1/21 | Cancer |
| Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel | Ofranergene obadenovec (VB-111) | CD95 modulator; TNF receptor modulator | Platinum-resistant ovarian cancer | Added a second primary endpoint of progression-free survival to the Oval study; PFS readout expected in 2022, which could be the basis of a BLA submission; readout of the other primary endpoint, overall survival, expected in 2023 | 6/3/21 | Cancer |
| Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel | VB-111 (ofranergene obadenovec) | CD95 modulator; TNF receptor modulator | Platinum-resistant ovarian cancer | Recruitment of new patients into the U.S.-based clinical trial sites of the Oval study were paused due to temporary shortage of drug supply; release of new batches of the drug are pending clearance by the FDA, which is evaluating the comparability of VB-111 manufacturing between different source sites; study has enrolled approximately 75% of the expected 400 patients | 6/15/21 | Cancer |
| Veru Inc., of Miami | Sabizabulin (VERU-111) | Small molecule targets alpha and beta subunits of tubulin; tubulin polymerization inhibitor; androgen receptor transport disruptor | Metastatic castration-resistant prostate cancer | First patient enrolled in its phase III Veracity study; expected to enroll 245 patients; primary endpoint is median radiographic progression-free survival; secondary endpoints are overall response rate, duration of objective response, overall survival, time to chemotherapy and pain progression | 6/25/21 | Cancer |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Patisiran | RNAi targeting transthyretin | Cardiomyopathy in patients with transthyretin-mediated amyloidosis | Completed enrollment of more than 300 patients in the Apollo-B study; top-line data expected in mid-2022 | 6/1/21 | Cardiovascular |
| Biocardia Inc., of San Carlos, Calif. | Cardiamp | Stem cell therapy consisting of autologous bone marrow cells derived from iliac crest | Heart failure | Data from high doses of CD34+ cells delivered to patients in pivotal trial supported clinical efficacy | 6/22/21 | Cardiovascular |
| Biocardia Inc., of San Carlos, Calif. | Cardiamp | Autologous bone marrow-derived stem cell therapy | Heart failure | After review of data from 97 patients, the independent data safety monitoring board recommended continuing the study as designed | 6/23/21 | Cardiovascular |
| Chemocentryx Inc., of San Carlos | Avacopan | Complement C5a Receptor Inhibitor | ANCA-associated vasculitis with renal disease | In the Advocate study, avacopan improved estimated glomerular filtration rate more than prednisone; avacopan produced a faster improvement in the urine albumin-to-creatinine ratio compared to prednisone | 6/7/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Selective cardiac myosin activator | Heart failure with reduced ejection fraction | Completed enrollment in the 270-patient METEORIC-HF study; data expected in early 2022 | 6/15/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Selective cardiac myosin activator | Heart failure | 27% had atrial fibrillation or flutter (AFF) at baseline; primary composite endpoint of heart failure events or cardiovascular death was greater in patients without baseline AFF compared to those patients with AFF at baseline (interaction p=0.012); fewer serious adverse events of atrial fibrillation in patients without AFF at baseline in patients vs. placebo (p=0.046); in patients without AFF, primary composite endpoint was increased in patients with a baseline NT-proBNP above the median (hazard ratio, 0.81; 95% confidence interval 0.73-0.90) compared to patients with baseline NT-proBNP equal to or below the median (HR, 0.94; 95% CI 0.80-1.09; interaction p=0.095); well-tolerated and with no significant changes in blood pressure, renal function and potassium compared to placebo | 6/30/21 | Cardiovascular |
| Idorsia Pharmaceuticals Ltd., of Allschwil, Switzerland | Selatogrel | P2Y12 receptor antagonist | Acute myocardial infarction | Started the 14,000-patient SOS-AMI study to evaluate the efficacy and safety of self-administered subcutaneous selatogrel; primary efficacy endpoint is the occurrence of death from any cause or non-fatal AMI after any study treatment self-administration | 6/28/21 | Cardiovascular |
| Incarda Therapeutics Inc., of San Francisco | Inrhythm (flecainide, inhaled formulation) | Sodium channel protein type 5 subunit alpha blocker | Atrial fibrillation | Company plans to initiate pivotal phase III Restore1 trial in the third quarter of 2021; data readout expected in the second half of 2022 | 6/23/21 | Cardiovascular |
| Pharmazz Inc., of Willowbrook, Ill. | Lyfaquin (centhaquin) | Alpha 2B adrenoceptor agonist | Hemorrhagic shock | Drugs published manuscript describing study of drug as resuscitative agent in hypovolemic shock that showed survival advantage, with 8.8% absolute reduction in mortality | 6/1/21 | Cardiovascular |
| Alvotech hf, of Reykjavik, Iceland | AVT-02 | Biosimilar of Humira (adalimumab) | Moderate to severe chronic plaque psoriasis | Study reached the primary completion date; top-line results are expected later this year | 6/15/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo. | Sofpironium bromide gel 15% | Anticholinergic | Primary axillary hyperhidrosis | Completed enrollment in both phase III Cardigan I and Cardigan II studies | 6/29/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., of Tokyo | Sofpironium bromide (BBI-4000) | Anticholinergic | Primary axillary hyperhidrosis | At week 52, in a long-term extension study published in the Journal of Dermatology, 57.4% of patients in the group who switched from placebo to sofpironium and 58.2% of patients in the group who remained on sofpironium had a hyperhidrosis disease severity score of 1 or 2 and a 50% or more reduction in total gravimetric weight of sweat | 6/1/21 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Dual IL-13/IL-4 receptor antagonist | Atopic dermatitis | Open-label extension trial showed long-term safety of 300-mg weekly dose in adults with moderate to severe disease observed up to 3 years was generally consistent with that in controlled pivotal phase III trials | 6/28/21 | Dermatologic |
| Anji Pharmaceuticals Inc., of Cambridge, Mass. | ANJ-900 | Gut-targeted delayed-release metformin | Type 2 diabetes with chronic kidney disease | Initiated a pivotal trial in patients; assessing for HbA1c levels throughout the treatment period | 6/10/21 | Endocrine/Metabolic |
| Crinetics Pharmaceuticals Inc., of San Diego | Paltusotine | Nonpeptide SST2 receptor agonist | Acromegaly | First of 52 patients in the Pathfnder-1 study dosed; primary endpoint is proportion of patients who maintain biochemical response in IGF-1 (?1× the upper limit of normal at the end of the randomized (EOR) control phase; secondary endpoints are change from baseline in IGF-1 to EOR, proportion of subjects with GH <1 34 ng ml at week to those who had gh <1 screening and change from baseline in total acromegaly symptoms diary score eor; top-line data expected the end of 2023< td> | 6/28/21 | Endocrine/Metabolic |
| Cyclo Therapeutics Inc., of Gainesville, Fla. | Trappsol Cyclo | Formulation of hydroxypropyl beta cyclodextrin | Niemann-Pick disease type C1 | Started pivotal TransportNPC study to enroll at least 93 pediatric (age 3 to less than 18 years) and adult patients; top-line results from interim analysis expected in first half of 2023 | 6/17/21 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | Enrolled and randomized over 50% of the 675 patients planned for the ORA-D-013-1 study; top-line results expected in 2022 | 6/7/21 | Endocrine/Metabolic |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Hsp70 inducer | Niemann-Pick disease type C | In the 41-patient open-label extension trial, mean change in the 5-domain NPC Clinical Severity Scale was 3.5 points after 36 months for patients who received arimoclomol from the start of the double-blind phase; disease progression for routine clinical care for 36 months was estimated to be a mean increase of 5.2 points | 6/28/21 | Endocrine/Metabolic |
| Oxthera AB, of Stockholm | Oxabact | Lyophilized live Oxalobacter formigenes cells isolated from human gut | Primary hyperoxaluria | Top-line results from pivotal ePHex study (n=25) showed drug was stable and maintained kidney function; mean plasma oxalate concentration, the primary endpoint, separated after week 24 in favor of Oxabact; estimated of mean difference between treatment arms reached – 3.8 (2) ?mol/L at week 52 (p=0.06), where Oxabact group was essentially stable and placebo increased; trend in favor of Oxabact observed for overall comparison, and was consistent across most predefined subgroups; supportive slope model demonstrated similar magnitude of change of -6.1 (3) µmol/L (p=0.04) | 6/11/21 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa (PRX-102) | Galactosidase enzyme replacement therapy | Fabry disease | In the Balance study, the lower boundary of the confidence interval for the mean difference between PRX–102 and Fabrazyme (agalsidase beta) for the primary endpoint of mean annualized changes of the eGFR after completion of at least 12 months was below the noninferiority margin prespecified for this interim analysis in the intent-to-treat analysis and above the limit in the per protocol analysis | 6/2/21 | Endocrine/Metabolic |
| Sanofi SA, of Paris | Soliqua 100/33 (insulin glargine and lixisenatide injection) | Insulin and GLP-1 receptor agonist | Type 2 diabetes | Met its 2 primary endpoints, demonstrating noninferiority of blood sugar (HbA1c) reduction and superiority on body weight change from baseline compared to premixed insulin, and all key secondary endpoints, achieving a greater proportion of people reaching a HbA1c target of <7% without weight gain, a greater proportion of people reaching hba1c target <7% gain and hypoglycemia, superiority in reduction compared with those using premixed insulin< td> | 6/29/21 | Endocrine/Metabolic |
| Vifor Pharma Group, of St. Gallen, Switzerland | Veltassa (patiromer sorbitex calcium) | Potassium binder | Hyperkalemia | Phase IIIb Diamond study amended with new endpoints, due to recommendation from independent study executive committee and following the significant impact of COVID-19 on recruitment; new endpoint will investigate role of Veltassa in controlling serum potassium and potentially preventing hyperkalemia in heart failure patients treated with renin-angiotensin aldosterone system inhibitors; read-out and remaining data collection expected to be completed in 2021 | 6/24/21 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | IL-23 inhibitor | Crohn's disease | Fortify maintenance study of risankizumab, dosed at 360 mg (subcutaneous) every 8 weeks achieved co-primary endpoints of endoscopic response and clinical remission at 1 year in adults with moderate to severe disease | 6/2/21 | Gastrointestinal |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | Oral, selective and reversible JAK inhibitor | Ulcerative colitis | Met the primary endpoint of clinical remission and all secondary endpoints at 1 year in the phase III maintenance study; significantly more upadacitinib-treated patients achieved clinical remission at week 52 compared to placebo (15 mg: 42% and 30 mg: 52% vs. placebo: 12%; p<0.001); all secondary endpoints were met, including the achievement of endoscopic improvement, histologic-endoscopic mucosal improvement and corticosteroid-free clinical remission at week 52< td> | 6/29/21 | Gastrointestinal |
| Albireo Pharma Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | In the Pedfic 1 and 2 studies, mean height Z scores for patients group treated with Bylvay for 48 weeks increased from –1.6 to –0.5 (p=0.02); bilirubin levels decreased by 20 – 25 µmol/L | 6/3/21 | Gastrointestinal |
| Albireo Pharma Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | Pooled analysis of 77 participants from Pedfic 1 and 2 studies showed, 4 weeks after starting therapy, serum bile acids (sBAs) were reduced by 88 ?mol/L and pruritus score by 0.7; at end of analysis period, sBAs decreased from baseline by 213 ?mol/L in people with available data and pruritus score dropped by 1.4; height Z scores increased from –1.9 at baseline to –0.8 at week?48, with similar improvements for weight Z scores | 6/21/21 | Gastrointestinal |
| Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif. | Filgotinib | JAK1 inhibitor | Moderately to severely active ulcerative colitis | Post-hoc analysis from the Selection study published in The Lancet showed filgotinib 200 mg improved sustained clinical remission, 6-month corticosteroid-free remission, Mayo Clinic Score (MCS), endoscopic and histologic remission, MCS response and endoscopic improvement compared to placebo; 32.7% of patients achieved mucosal healing after 58 weeks of treatment with filgotinib compared to 10.2% of patients taking placebo | 6/4/21 | Gastrointestinal |
| Gilead Sciences Inc., of Foster City, Calif. | Hepcludex (bulevirtide) | Entry inhibitor | Chronic hepatitis delta virus | Interim results from MYR301 study indicate that, after 24 weeks, proportion of people achieving combined virological and biochemical response was 36.7% with bulevirtide 2 mg, 28% in participants receiving bulevirtide 10 mg and 0% in participants under observation who have not received antiviral treatment at this stage of study; treatment for 24 weeks with 2 mg or 10 mg had a superior response (p<0.001) 2 10 to the no treatment group, with bulevirtide mg having a numerically higher response rate vs. mg; rapid alt reduction and normalization observed in>50% of patients in 2-mg group vs. 10-mg or no treatment groups | 6/24/21 | Gastrointestinal |
| Ironwood Pharmaceuticals Inc., of Boston | Linzess (linaclotide) | Guanylate cyclase-C agonist | Irritable bowel syndrome with constipation | American Journal of Gastroenterology published data from phase IIIb trial in adults, reported in June 2019, showing statistically significant improvement in overall change in abdominal score vs. placebo (p<0.0001)< td> | 6/16/21 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor beta agonist | Nonalcoholic steatohepatitis | Data from 115 patients in the open-label portion of the MAESTRO-NAFLD-1 study showed change in MRI-PDFF from baseline to week 52 of -53.4% (p<0.0001); treatment also changed fibroscan cap by -45.2 db m (p<0.0001), vcte -2.8 kpa (p="0.0006)" and mre -0.43> | 6/25/21 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor (THR)-? selective agonist | Nonalcoholic steatohepatitis | Achieved the planned target enrollment; top-line 52-week data expected in the third quarter of 2022 | 6/30/21 | Gastrointestinal |
| Orphalan SA., of Paris | Cuprior (form of trientine tetrahydrochloride) | Chelating agent | Wilson’s disease | Top-line data from Chelate trial met its primary endpoint; trientine tetrahydrochloride was well-tolerated and noninferior to d-Penicillamine as measured by copper speciation evaluation of non-ceruloplasmin copper (NCC); prespecified composite endpoint of NCC and 24-hour urinary copper excretion was achieved in 50% of patients vs .d-Penicillamine (24% of patients); no serious adverse events (SAE) in 26 patients vs. d-Penicillamine reported 5 SAE in 27 patients | 6/25/21 | Gastrointestinal |
| Travere Therapeutics Inc., of San Diego | Sparsentan | Dual-acting antagonist of endothelin type A and angiotensin II type 1 receptors | IgA nephropathy | Completed enrollment in Protect study; top-line efficacy data from interim 36-week proteinuria endpoint analysis expected in August 2021 | 6/2/21 | Genitourinary/Sexual Function |
| Beyondspring Inc., of New York | Plinabulin | Selective immunomodulating microtubule-binding agent | Chemotherapy-induced neutropenia | In the Protective-2 study, plinabulin plus pegfilgrastim decreased production of immature neutrophil band (p=0.0012) and decreased promyelocytes and myelocyte production (p=0.0488) compared to pegfilgrastim alone | 6/8/21 | Hematologic |
| Beyondspring Inc., of New York | Plinabulin | Selective immunomodulating microtubule-binding agent | Prevention of chemotherapy-induced neutropenia | Positive results from the Protective-1 phase III study (n=105) vs. pegfilgrastim; plinabulin as single agent infused on the same day as chemotherapy showed week 1 early onset of action; met its primary endpoint of days of neutropenia cycle with noninferiority; secondary endpoints are improved clinical outcomes such as reduction of febrile neutropenia, hospitalization and bone pain, chemotherapy dose reduction and delay | 6/10/21 | Hematologic |
| Argenx BV, of Breda, the Netherlands | Efgartigimod | FcRn antagonist | Generalized myasthenia gravis | Results from pivotal Adapt trial, published in The Lancet Neurology, showed significantly more acetylcholine receptor-antibody positive (AChR-Ab+) gMG patients were responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score vs. placebo (67.7% vs. 29.7%; p<0.0001); 0 2 40% of patients treated with efgartigimod achieved minimal symptom expression defined as mg-adl scores (symptom free) or 1, compared to 11.1% who received placebo; among achr-ab+ responders, 84.1% showed clinically meaningful improvement on the score within first weeks treatment; safety profile was comparable placebo< td> | 6/16/21 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | Anifrolumab | Interferon type I receptor antagonist | Systemic lupus erythematosus | Pooled data from Tulip trials in people with moderate to severe disease showed study drug was associated with improved response vs. placebo at 52 weeks in skin rash (13.5% difference in SLEDAI, 15.5% in BILAG and 15.6% in modified CLASI) and in arthritis (8.2% in SLEDAI, 11.8% in BILAG and 12.6% in joint response) | 6/2/21 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Results of the phase III Aurora 1 study presented at the European Renal Association – European Dialysis and Transplant Association 2021 Congress, showed(n=179) patients treated with voclosporin in addition to mycophenolate mofetil and low-dose steroids achieved statistically significant increased renal response rates; stringent urine protein creatinine ratio levels ?0.3 mg/mg target; tolerated with no unexpected safety signals | 6/7/21 | Immune |
| Biogen Inc., of Cambridge, Mass. | BIIB-059 | Humanized IgG1 monoclonal antibody targeting blood dendritic cell antigen 2 | Systemic lupus erythematosus | First patient dosed in Topaz-1 study to enroll 540 patients with active SLE; 52-week study aims to demonstrate disease activity as measured by proportion of participants who achieve an SLE Responder Index-4 response as primary endpoint | 6/17/21 | Immune |
| Biogen Inc., of Cambridge, Mass., and Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-1806 (tocilizumab biosimilar) | IL-6 receptor modulator | Rheumatoid arthritis | Study met primary endpoints, showing equivalence to reference medicine in people with moderate to severe disease inadequately controlled by methotrexate therapy | 6/1/21 | Immune |
| Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-2506 | Biosimilar of Simponi (golimumab, Johnson & Johnson) | Psoriatic arthritis | Started dosing in the 480-patient study | 6/8/21 | Immune |
| Celltrion Healthcare Inc., of Incheon, South Korea | Yuflyma (adalimumab biosimilar, CT-P17) | TNF-alpha monoclonal antibody | Rheumatoid arthritis | 1-year data, published in supplement of Annals of Rheumatic Diseases, showed equivalent efficacy and comparable safety to reference drug in people with moderately to severely active disease; sustained and comparable efficacy measured by ACR20/50/70 response rates seen in those who received either drug as maintenance therapy and in those switched at week 26 from reference to study drug | 6/1/21 | Immune |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Olumiant (baricitinib) | JAK1/JAK2 inhibitor | Rheumatoid arthritis | Post-hoc analyses of Ra-Beam study in people with moderate to severe disease suggested Olumiant reduced pain and duration of morning joint stiffness and improved overall physical function at 12 weeks vs. Humira (adalimumab, Abbvie Inc.) and placebo | 6/1/21 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Cleaves IgG-antibodies | Kidney transplant | The study will enroll 64 highly sensitized kidney patients with a cPRA score of ?99.9%; study will compare the surrogate endpoint of eGFR at 12 months after randomization for imlifidase to placebo; first patient expected to be included in second half of 2021 with enrollment expected to be completed in the second half of 2022; results expected in the second half of 2021, setting up a potential BLA submission in the first half of 2024 | 6/23/21 | Immune |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Psoriatic arthritis | Phase IIIb Cosmos data showed 57.7% of treated participants achieved ?20% improvement in ACR20 and 53.4% achieved PASI 100 at 1 year | 6/2/21 | Immune |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Monoclonal antibody targeting interleukin-17A | Juvenile psoriatic arthritis and enthesitis-related arthritis | In the Junipera study, Cosentyx delayed time to flare vs. placebo (p<0.001); 2021 data to be presented at eular annual european congress of rheumatology< td> | 6/2/21 | Immune |
| Partner Therapeutics Inc., of Lexington, Mass. | Leukine (sargramostim) | Yeast-derived rhuGM-CSF | Hospitalized COVID-19 | In the 122-patient iLeukPulm trial, inhaled Leukine treatment in combination with standard of care (SOC) produced a P(A-a)O2 of 100 mm Hg (31%) compared to 35 mm Hg (5%) for SOC alone (p=0.033); 84% of patients treated with Leukine had an improvement in oxygenation, compared to 64% of patient in the control arm (p=0.023) | 6/28/21 | Immune |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-620 (maribavir) | Serine/threonine protein kinase UL97 (viral) inhibitor | Transplant recipients with refractory, with or without resistance, cytomegalovirus (CMV) infection/disease | Results from subgroup analysis of Solstice study across solid organ transplant (SOT) type patients presented at the American Transplant Congress 2021, achieved confirmed CMV viremia clearance, consistent efficacy in heart, lung and kidney transplants; lower incidence of treatment-related toxicities | 6/7/21 | Immune |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor targeting mast cells and macrophages | Mastocytosis, amyotrophic lateral sclerosis and COVID-19 | Suspending inclusions and treatment initiations for the phase III AB15003 study in mastocytosis, the phase III AB19001 study in amyotrophic lateral sclerosis and the phase II AB20001 study in COVID-19 due to a potential risk of ischemic heart disease; continuing investigation of the risk | 6/1/21 | Infection |
| Celltrion Group, of Incheon, of South Korea | Regdanvimab (CT-P59) | Monoclonal antibody targeting SARS-CoV-2 virus | Mild to moderate COVID-19 | Compared to placebo, CT-P59 reduced the risk of hospitalization or death by 72% for patients at high-risk of progressing to severe COVID-19 up to day 28 (p< 0.0001); CT-P59 reduced the risk of hospitalization or death by 70% in all patients (p< 0.0001); patients at high-risk of progressing to severe COVID-19 treated with CT-P59 recovered in a median of 9.3 days compared to at least 12 days for placebo (p< 0.0001); for all patients, recovery was 8.4 days for CT-P59 and 13.3 days for placebo (p< 0.0001) | 6/14/21 | Infection |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Lok | Antibiotic lock solution | Catheter-related blood stream infections | Next planned interim analysis of the data for safety, superiority and futility is scheduled for the end of June 2021 | 6/7/21 | Infection |
| Glaxosmithkline plc, of London, and Vir Biotechnology Inc., of San Francisco | Sotrovimab | COVID-19 spike glycoprotein modulator | COVID-19 | Confirmatory results showed Comet-Ice trial met primary endpoint; primary efficacy analysis of all participants (n=1,057) showed 79% reduction (p<0.001) in hospitalization for>24 hours or death due to any cause by day 29 vs. placebo | 6/21/21 | Infection |
| Glenmark Pharmaceuticals Ltd., of Mumbai, India | Favipiravir | RNA polymerase inhibitor | Mild to moderate COVID-19 | Interim analysis of 503 patients in the post-marketing surveillance study showed no new safety signals or concerns; time to fever resolution was seen on day 3; 2/3 of the patients achieved clinical cure on day 7 | 6/7/21 | Infection |
| Medigen Vaccine Biologics Corp., of Taiwan | EV71 | Vaccine | Hand, foot and mouth disease | Vaccine that showed efficacy of 100% against a virus that causes hand, foot and mouth disease | 6/24/21 | Infection |
| Molecular Partners, AG of Zurich-Schlieren, Switzerland | Ensovibep | Binds to the SARS-CoV-2 spike protein at 3 distinct locations | Mild to moderate COVID-19 | First patient dosed in the 1,000-patient substudy of the ACTIV-3 platform study; primary endpoint is time to sustained recovery for 14 days after release from the hospital; interim analysis for futility after the first 300 patients have been randomized and recruited | 6/13/21 | Infection |
| Novabiotics Ltd., of Aberdeen, U.K. | Cysteamine bitartrate (NM-002) | Immunomodulator antimicrobial | Community-acquired pneumonia | Intravenous candidate included in trial funded and conducted through global adaptive Remap-CAP trial consortium | 6/21/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Recombinant nanoparticle protein-based COVID-19 vaccine | COVID-19 prophylaxis | In the Prevent-19 study, the vaccine produced an overall efficacy of 90.4%; vaccine produced 100% protection against moderate and severe COVID-19 | 6/14/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Nanoparticle protein-based vaccine | COVID-19 prophylaxis | In a substudy of the U.K.-based phase III study of NVX-CoV2373, 431 participants were given an approved seasonal influenza vaccine from Seqirus UK Ltd. co-administered with NVX-CoV2373; vaccine efficacy against COVID-19 in the substudy was 87.5% compared to 89.8% in the main study | 6/14/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Final analysis of pivotal phase III study published in The New England Journal of Medicine showed overall efficacy of 89.7%, with over 60% against B.1.1.7 (Alpha) variant, 96.4% efficacy against non-B.1.1.7 (non-Alpha) variants and 90% protection against all strains at that time; mild and transient side effects | 6/30/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGEN-COV (casirivimab + imdevimab) | COVID-19 spike glycoprotein inhibitor | COVID-19 | U.K. Recovery trial met primary endpoint, showing that addition of agent to usual care reduced risk of death by 20% in people without immune response against SARS-CoV-2 (p=0.001) | 6/16/21 | Infection |
| Russian Direct Investment Fund, of Moscow | Sputnik V | Adenoviral-based vaccine | COVID-19 prophylaxis | Demonstrated 97.8% efficacy against COVID-19 cases and 100% efficacy against severe cases of COVID-19, according to data from UAE's Ministry of Health | 6/29/21 | Infection |
| Shenzhen Kangtai Biological Products Co. Ltd., of Shenzhen, China | Inactivated COVID-19 Vaccine | COVID-19 vaccine | COVID-19 prophylaxis | First subjects vaccinated in the study | 6/22/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Completed enrollment of more than 4,000 participants in the Cov-Compare (VLA2001-301) study; top-line data expected by September 2021 | 6/3/21 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Avigan/Reeqonus (favipiravir) | Broad-spectrum antiviral in tablet form | Mild to moderate COVID-19 | Added clinical research sites in Mexico and Brazil to test potential oral therapy; top-line data expected in the 3Q of 2021 | 6/17/21 | Infection |
| Novan Inc., of Durham, N.C. | SB-206 (berdazimer sodium) | Nitric oxide-releasing antiviral product | Molluscum contagiosum | Positive top-line results from pivotal B-Simple4 study (n=891), up to 12 weeks with a follow-up visit at week 24, showed SB-206 was well-tolerated and achieved its primary endpoint; 32% of patients had total clearance at week 12 and 43% of patients with total clearance or 1 remaining lesion at week 12 vs. placebo; no serious adverse events | 6/11/21 | Infection |
| Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | IL-17 inhibitor | Axial spondyloarthritis | Coast-Y study showed long-term improvements in signs and symptoms, functionality and quality of life; 56.7% of participants treated continuously through 2 years achieved Assessment of Spondyloarthritis International Society 40% response | 6/1/21 | Musculoskeletal |
| Neurana Pharmaceuticals Inc., of San Diego | Tolperisone | Non-opioid, centrally acting muscle relaxant | Symptoms associated with acute and painful muscles spasms of the back | Enrolled 50% patient (500/1000) in Resume-1 study; top-line data expected in 4Q021; | 6/7/21 | Musculoskeletal |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of the muscle relaxant cyclobenzaprine hydrochloride | Fibromyalgia | In the Relief study, TNX-102 SL reduced daily pain compared to placebo (p=0.01); drug produced a higher rate of patients with ?30% pain improvement compared to placebo (p=0.006) | 6/3/21 | Musculoskeletal |
| Acadia Pharmaceuticals Inc., of San Diego | Nuplazid (pimavanserin) | Serotonin inverse agonist and antagonist preferentially targeting the 5-HT2A receptor | Parkinson’s disease psychosis | In the open-label extension (OLE) published in Parkinsonism and Related Disorders patients who remained on Nuplazid 34 mg had a change in Scale for the Assessment of Positive Symptoms-PD of -0.8 from the end of the initial study to week 4 of the OLE compared to -2.9 for patients who switched from placebo to Nuplazid 34 mg | 6/3/21 | Neurology/Psychiatric |
| Adamas Pharmaceuticals Inc., of Emeryville, Calif. | Gocovri (amantadine) | Extended-release of amantadine capsules | Motor complications in Parkinson's disease | In new post-hoc analysis, at week 12, patients treated with Gocovri reported statistically significant improvements in motor control for daily activities compared to placebo (least-squares mean changes from baseline of –3.4 points for Gocovri and –1.4 for placebo) | 6/10/21 | Neurology/Psychiatric |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Onpattro (patisiran) | Small interfering RNA targeting transthyretin (TTR) | TTR-related amyloid polyneuropathy | Positive results from Helios-A study presented in the 2021 Peripheral Nerve Society’s Annual Meeting, achieved rapid and sustained reduction in serum TTR levels in patients following orthotopic liver transplant; improvement in the Modified Neuropathy Impairment Score (mNIS+7) and Norfolk Quality of Life-Diabetic Neuropathy score (Norfolk QOL-DN) from vutrisiran treatment consistently observed across all pre-specified patient subgroups; safety and tolerability profile after 12 months, diarrhea as common adverse event; | 6/7/21 | Neurology/Psychiatric |
| Alzheon Inc., of Framingham, Mass. | ALZ-801 | Anti-amyloid drug | Early Alzheimer’s disease APOE4/4 homozygotes | Treated first patient in the APOLLOE4 study; primary endpoint is change of score on the Alzheimer's Disease Assessment Scale – cognitive subscale; secondary endpoints include assessments of function, ability to perform daily activities and neuropsychiatric symptoms | 6/4/21 | Neurology/Psychiatric |
| Avadel Pharmaceuticals plc, of Dublin | FT-218 (sodium oxybate controlled-release) | GABA B receptor agonist | Narcolepsy | Post hoc analyses from pivotal Rest-On trial showed statistically significant improvement vs. placebo in excessive daytime sleepiness (EDS) at evaluated doses in narcolepsy subtypes with/without cataplexy and greater improvement in mean sleep latency on maintenance of wakefulness test and in Clinical Global Impression-Improvement; study drug also showed statistically significant EDS improvement at tested doses with/without stimulant use and greater decrease in weight and BMI vs. placebo | 6/9/21 | Neurology/Psychiatric |
| Gensight Biologics SA, of Paris | Lumevoq (GS-010) | MT-ND4 gene stimulator | Lebers hereditary optic neuropathy (LHON) | Frontiers in Neurology published results of indirect comparison of evolution of visual outcomes in people treated with Lumevoq vs. spontaneous evolution in natural history (NH) studies of people with LHON carrying m.11778G>A ND4 mutation, showing statistically and clinically relevant difference in BCVA in favor of study drug vs. NH from months 12 to 52 after vision loss | 6/1/21 | Neurology/Psychiatric |
| H. Lundbeck A/S, of Valby, Denmark | Vyepti (eptinezumab-jjmr) | CGRP receptor antagonist | Migraine | Journal of the American Medical Association published results from Relief study that met co-primary endpoints of time to headache pain freedom and time to absence of most bothersome symptoms vs. placebo (both p<0.001)< td> | 6/15/21 | Neurology/Psychiatric |
| Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Narcolepsy | New analysis from Harmony 1 and CTP trials showed the magnitude of its clinical effectiveness for excessive daytime sleepiness and reduction in the weekly rate of cataplexy in adults with narcolepsy; NNT was 5 in HARMONY 1 and 3 in HARMONY CTP; effect size was 0.61 in HARMONY 1 and 0.86 in HARMONY CTP; results for reduction in the WRC from HARMONY CTP showed NNT was 3 for a 50% reduction in the WRC and 4 for a 75% reduction in the WRC; effect size was 0.86 | 6/10/21 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Zolgensma (onasemnogene abeparvovec) | SMN1 gene stimulator | Spinal muscular atrophy | In late-breaker data from 2-copy cohort of Spr1nt trial, all participants (14/14) achieved primary endpoint of sitting independently for at least 30 seconds, including 11 who achieved milestone within WHO window of normal development; all participants also met secondary endpoint of survival without ventilatory support at 14 months of age vs. 26% in pediatric neuromuscular clinical research natural history cohort; in Str1ve-EU trial, 27/33 achieved developmental motor milestones not seen in natural history of SMA type 1.5; 14/32 in intent-to-treat population achieved primary endpoint of sitting independently for ?10 seconds, observed at median age of 15.9 months (7.7–18.6) | 6/18/21 | Neurology/Psychiatric |
| Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. | Zuranolone (SAGE-217/BIIB-125) | Oral neuroactive steroid GABA-A receptor positive allosteric modulator | Major depressive disorder | In the 543-patient Waterfall study, zuranolone produced a least squares mean change from baseline in 17-item Hamilton Rating Scale for Depression total score of -14.1 at day 15 compared to -12.4 for placebo (p=0.0141); placebo-adjusted change from baseline in the Clinical Global Impression-Severity of Illness at day 15 was -0.2 (p=0.1193) | 6/15/21 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | CGRP receptor antagonist | Migraine | Pooled analysis of Halo episodic/chronic migraine studies and Focus study showed changes in heart rate and blood pressure were not affected by action of any CGRP inhibitor at recommended dose | 6/21/21 | Neurology/Psychiatric |
| Vasopharm GmbH, of Wurzburg, Germany | Ronopterin (VAS-203) | Analogue of tetrahydrobiopterin | Traumatic brain injury | Nostra study didn’t meet its primary endpoint of improvement in extended Glasgow Outcome Scale (eGOS) at 6 months after trauma; post-hoc analysis showed patients with moderate and severe TBI treated within 12 hours after trauma had significant and clinically meaningful increases in eGOS | 6/3/21 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Netarsudil ophthalmic solution | Rho associated protein kinase inhibitor | Open-angle glaucoma or ocular hypertension | Completed patient enrollment for trial in Japan, comparing drug administered once per day to ripasudil hydrochloride hydrate ophthalmic solution administered twice per day; trial expected to complete by end of 2021 with top-line data shortly thereafter | 6/17/21 | Ocular |
| Biogen Inc., of Cambridge, Mass. | Timrepigene emparvovec (BIIB-111/AAV2-REP1) | AAV2 vector based gene therapy expressing choroideremia | Choroideremia | Star study didn’t meet the primary endpoint of the proportion of patients with a ?15 letter improvement from baseline in best corrected visual acuity at month 12 for timrepigene emparvovec compared to the non-interventional control group; key secondary endpoints also weren’t met | 6/14/21 | Ocular |
| Eyenovia Inc., of New York | Microline | Formulation of the cholinergic agonist pilocarpine | Presbyopia | In the Vision-1 study, a greater proportion of patients treated with Microline had a 3-line or greater improvement in near vision compared to placebo (p<0.05); proportion of patients with a 2-line or greater improvement also favored microline (p<0.05)< td> | 6/15/21 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq (GS-010) | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 (ND4) gene | Leber hereditary optic neuropathy | Top-line results from Reflect phase III study showed statistically significantly mean best-corrected visual acuity (BCVA) in treated eyes better than baseline vs. placebo after 1.5 years injection; contralateral effect reduced and the trial did not meet the predefined primary endpoint; difference of the change from baseline in BCVA between the second affected Lumevoq and placebo-treated eyes was -0.05 LogMAR (+3 ETDRS letters equivalent; p=0.6080); mean BCVA at 1.5 years for bilaterally and unilaterally treated subjects reached 1.35 and 1.45 LogMAR, respectively, with an absolute difference between arms of +5 letters in favor of bilaterally treated subjects; at 1.5 years, improvement by at least 3 lines from nadir was demonstrated by 69% and 64% of bilaterally and unilaterally treated subjects, respectively; favorable safety profile | 6/30/21 | Ocular |
| Iveric Bio Inc., of New York | Zimura (avacincaptad pegol) | Pegylated aptamer targeting complement C5 | Geographic atrophy secondary to age-related macular degeneration | Post-hoc analyses of Gather1 study showed 19.6% reduction in rate of progression from drusen to iRORA/cRORA as compared to sham at 18 months, for relative risk reduction of 72%; 21.8% reduction in rate of progression from iRORA to cRORA as compared to sham at 18 months, for relative risk reduction of 52%; company expects to complete enrollment in Gather2 study in late July of 2021 and top-line data are expected in the second half of 2022 | 6/18/21 | Ocular |
| Laboratorios Salvat SA, of Barcelona | Clobetasol propionate | Corticosteroid nanoemulsion | Ocular inflammation and pain after cataract surgery | Completed studies; data expected in the second half of 2021 | 6/3/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010 (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Completed dosing in the Norse Two study; top-line data expected in the third quarter of 2021 | 6/7/21 | Ocular |
| Xbrane Biopharma AB, of Solna, Sweden | Xlucane (ranibizumab biosimilar) | VEGF-1 receptor antagonist | Wet age-related macular degeneration; diabetic macular edema | Top-line results from 6-month interim read-out in pivotal equivalence trial showed study drug met primary endpoint, demonstrating equivalent efficacy in BCVA change at week 8 of treatment vs. Lucentis | 6/28/21 | Ocular |
| Galera Therapeutics Inc., of Malvern, Pa. | Avasopasem manganese (GC-4419) | Selective small-molecule dismutase mimetic | Severe oral mucositis | Completed enrollment in the 445-patient Roman trial to evaluate the ability of avasopasem to reduce radiation-induced SOM in patients with locally advanced head and neck cancer; top-line data expected in the second half of 202 | 6/28/21 | Other/Miscellaneous |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso (treprostinil) | PGI2 agonist | Idiopathic pulmonary fibrosis | Enrolled first of approximately 396 patients in the 52-week study; primary endpoint is the change in forced vital capacity (FVC); secondary endpoints include time to clinical worsening, time to first acute exacerbation of IPF, overall survival at week 52, change in percent predicted FVC from baseline to week 52 and change in the King's Brief Interstitial Lung Disease questionnaire | 6/3/21 | Respiratory |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso (treprostinil) | PGI2 agonist | Idiopathic pulmonary fibrosis | Post-hoc analysis showed primary efficacy endpoint was the change in 6-minute walk distance (6MWD) measured at peak exposure from baseline to week 16; well-tolerated and improved 6MWD by 21 meters vs. placebo (p=0.0043) at week 16; increased 6MWD by 31 meters relative to placebo after 16 weeks of treatment (p<0.001); 12 15 16 secondary endpoints include change in plasma concentration of the cardiac biomarker n-terminal pro-brain natriuretic peptide (nt-probnp) from baseline to week 16; 42% reduction nt-probnp with tyvaso vs. placebo at (p<0.001); 39% risk a clinical worsening event (p="0.04);" 22.7% patients treated experienced 33.1% patients; significantly fewer treprostinil group than had exacerbations underlying lung disease (26.4% 38.7%, p="0.02);" significant improvements peak 6mwd compared (31.29 m, p<0.001) and through (21.99, results published lancet respiratory medicine < td> | 6/30/21 | Respiratory |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid that binds to the nicotinic acetylcholine receptor | Nicotine addiction | ORCA-2 trial reached its enrollment target of 750 adult smokers | 6/29/21 | Toxicity and Intoxication |
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Treatment-naïve chronic lymphocytic leukemia or small lymphocytic lymphoma | Top-line interim analysis showed trial met its primary endpoint of progression-free survival with median follow-up of 25.8 months compared to chemoimmunotherapy; safe and well- tolerated | 7/29/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | Human monoclonal IgG4 antibody against PD-1 and human antibody targeting the CTLA4 receptor | Recurrent or metastatic squamous cell carcinoma of the head and neck | Trend toward overall survival in patients whose tumors express PD-L1 with a combined positive score (CPS) ? 20, though study did not met its primary endpoint; results did not reach statistical significance; consistent safety profile | 7/16/21 | Cancer |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | A3 adenosine receptor agonist | Hepatocellular carcinoma | Completed preparatory work and expected to initiate patient enrollment for pivotal phase III; interim analysis planned for after 50% enrolled patients treated | 7/8/21 | Cancer |
| Da Volterra SAS, of Paris | DAV-132 | Charcoal-based adsorbent; microbiome protector | Prevention of chemotherapy-induced intestinal microbiome dysbiosis in patients with hematological malignancies | First patient randomized in phase III Microcare study | 7/26/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate comprised of a humanized anti-HER2 antibody attached to a topoisomerase I inhibitor payload via a tetrapeptide linker | HER2-positive advanced gastric cancer | Dosed first patient | 7/8/21 | Cancer |
| GNT Biotech & Medicals Co. Ltd., of Taipei, Taiwan | Chidamide (Kepida) | Histone deacetylase inhibitor |
Hormone receptor-positive and HER2-negative advanced breast cancer | Placebo-controlled phase III study in China (n=365) and Taiwan (n=55) in combination with exemestane showed median progression-free survival was 7.4 months for chidamide and 3.7 months for placebo; median progression-free survival was 8.6 months for chidamide and 3.7 months for placebo in Taiwan patients in separate data | 7/23/21 | Cancer |
| Kyowa Kirin Co. Ltd., of Tokyo | Poteligeo (mogamulizumab-kpkc) | Humanized IgG1kappa monoclonal antibody against human CC chemokine receptor type 4 | Mycosis fungoides; Sézary syndrome | In 12 cycles of treatment, 43.5% had a ?50% improvement in skin response vs. vorinostat (22%); complete or partial skin responses in blood involvement (45.2% and 56% at B1 and B2, respectively) vs. B0 (25%); 17.6% mogamulizumab-treated patients with B2 level blood involvement had 100% compared to vorinostat; progression-free survival was significantly longer for patients compared to vorinostat (7.7 months and 3.1 months, respectively (p<0.0001)); overall response rate higher compared to vorinostat at 28% vs 4.8%, respectively; p< 0.0001) and significantly with the highest level of blood involvement (b2: 37.4% vs. 3.2%; p<0.0001); time-to-next-treatment was longer for patients treated mogamulizumab b1 (12.63 3.07 months, p="0.0018)" b2 (13.07 3.53 results published in european academy dermatology venereology< td> | 7/22/21 | Cancer |
| Lee’s Pharmaceutical Holdings Ltd., of Hong Kong, its affiliate, China Oncology Focus Ltd., and Sorrento Therapeutics Inc., of San Diego | Socazolimab (ZKAB-001) | Anti-PD-L1 antibody | Extensive-stage small-cell lung cancer | First patient enrolled | 7/23/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 antibody | Triple-negative breast cancer | Median follow-up of 39 months showed Keytruda regimen showed statistically significant and clinically meaningful event-free survival by 37% (HR=0.63 [95% CI, 0.48-0.82]; p=0.00031) vs. chemotherapy-placebo regimen; 28% reduction in the risk of death with Keytruda vs. placebo (HR=0.72 [95% CI, 0.51-1.02]; p=0.03214); consistent safety profile and no new safety signals | 7/15/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 | Metastatic triple-negative breast cancer | Keynote-355 trial met its primary endpoint in combination with chemotherapy; demonstrated a statistically significant and clinically meaningful improvement in overall survival compared with chemotherapy alone in patients with tumors expressing PD-L1; no new safety signals | 7/27/21 | Cancer |
| Oncopeptides AB, of Stockholm | Pepaxto (melphalan flufenamide) | DNA alkylating agent; peptide-drug conjugate that targets aminopeptidases | Relapsed refractory multiple myeloma | FDA requested partial clinical hold based on OS in pre-specified subgroups; OS HR was 1.104 (0.846-1.441) in favor of pomalidomide; melflufen met the primary endpoint of superior PFS compared to pomalidomide with a hazard ratio (HR) of 0.792 (95% CI 0.640-0.979, p-value 0.0311) as determined by the independent review committee in 29 patients in Ocean study | 7/8/21 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J | CPI-613 (devimistat) | Small molecule that targets the mitochondrial tricarboxylic acid | Relapsed or refractory acute myeloid leukemia | Preplanned interim futility analysis of its pivotal phase III Armada trial demonstrated that the response rate achieved the predefined threshold and independent data monitoring committee recommended to continue the trial | 7/1/21 | Cancer |
| Rain Therapeutics Inc., of Newark, Calif., | RAIN-32 (milademetan) | Oral mouse double minute 2 inhibitor | De-differentiated liposarcoma | First patient randomized in phase III Mantra study | 7/20/21 | Cancer |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | SGLT2 inhibitor | Heart failure | Top-line data of Emperor-preserved phase III trial met its primary endpoint; demonstrated significant risk reduction for the composite of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction; consistent safety profile | 7/6/21 | Cardiovascular |
| George Medicines Pty Ltd., of London | Single-pill triple combination | Fixed-dose combination of telmisartan, amlodipine and indapamide | Hypertension | Initiated 2 phase III studies | 7/1/21 | Cardiovascular |
| Abeona Therapeutics Inc., of New York | EB-101 | Cell-based gene therapy consisting of autologous epithelial keratinocyte sheets engineered to express human alpha-1 chain of type VII collagen (Col7A1) | Recessive dystrophic epidermolysis bullosa | Activated second clinical trial site in pivotal phase III Vital study | 7/23/21 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast foam (ARQ-154) | PDE4 inhibitor | Seborrheic dermatitis | First of about 450 participants 9 years and older with moderate to severe disease enrolled in single pivotal Stratum trial; primary endpoint is proportion who achieve IGA score of “clear” or “almost clear” plus 2-point improvement at 8 weeks; top-line data expected by third quarter of 2022 | 7/12/21 | Dermatologic |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Orladeyo (berotralstat) | Kallikrein inhibitor | Hereditary angioedema | In 96-week data from Apex-2 trial, study drug reduced HAE attack rate by 80% from baseline; median attack rates also decreased from 2.7 attacks/month at baseline to 0 attacks per month in 16/17 months through same period | 7/10/21 | Dermatologic |
| Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-2206 | Biosimilar of Stelara (ustekinumab); human monoclonal antibody targeting the p40 subunit of IL-12 and IL-23 | Psoriasis | First patient dosed | 7/15/21 | Dermatologic |
| Jupiter Wellness Inc., of Jupiter, Fla. | JW-100 | Topical lotion containing cannabidiol and aspertame | Mild to moderate eczema | Initiated phase III study | 7/13/21 | Dermatologic |
| Mediwound Ltd., of Yavne, Israel | Nexobrid | Concentrate of proteolytic enzymes comprising bromelain | Thermal burns | Study met its 3 primary endpoints with a high degree of statistical significance; safe and well-tolerated; significantly shorter time to achieve complete eschar removal compared with patients treated with SOC (median time to complete eschar removal - Nexobrid: 0.99 days vs. SOC: 5.99 days, p=0.00081); significant reduction in surgical need for excisional eschar removal; substantially lower percent of wound area surgically excised for eschar removal compared with patients treated with SOC (Nexobrid: 1.5% vs. SOC: 48.1%, p<0.0001); demonstrated a statistically significant lower incidence of surgical excision for eschar removal compared with patients treated soc (nexobrid: 8.33% (6 72) vs. soc: 64.38% (47 73), p<0.0001); blood loss during the procedure (mean volume – nexobrid: 32.36 ml 202.55 ml, p="0.134);" reduction autograft performed in dpt wounds (on target level analysis) 25.93% (21 81) 37.63% (26 69), numerically percent area wound autografted 15.9% 22.8%,> | 7/20/21 | Dermatologic |
| Polarityte Inc., of Salt Lake City | Skinte | Human cellular and tissue-based product derived from a patient’s own skin | Diabetic foot ulcers | Study met its primary and secondary endpoints; 70% of participants receiving Skinte plus standard of care (SOC) had wound closure at 12 weeks vs. 34% of participants receiving SOC alone (p=0.00032); percent area reduction assessed at 4, 6, 8, 10 and 12 weeks was significantly greater for the Skinte plus SOC treatment group vs. SOC alone (p=0.009); increased the odds of wound closure by 5.37 times vs. SOC (p=0.001); wound size for the Skinte plus SOC treatment group was 3.5 cm2 vs. 3.2 cm2 for the SOC treatment group (p=0.46); top-line data from the venous leg ulcers trial expected in the second half of 2021 | 7/28/21 | Dermatologic |
| Samsung Bioepis Co. Ltd., of Incheon, South Korea | SB-17 (ustekinumab biosimilar) | Human monoclonal antibody targeting the p40 subunit of IL-12 and IL-23 | Psoriasis | Initiated phase III study | 7/19/21 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Human monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13 proteins | Moderate to severe chronic spontaneous urticaria | Study (n=138) met primary and all key secondary endpoints at 24 weeks; 63% reduction in itch severity with Dupixent vs. 35% with placebo, as measured by 21-point itch severity scale (10.24-point reduction with Dupixent vs. 6.01-point reduction with placebo, p<0.001); 65% reduction in urticaria activity (itch and hives) severity with dupixent vs. 37% placebo (20.53-point 12-point placebo, p<0.001)< td> | 7/29/21 | Dermatologic |
| Stratatech Corp., of Madison, Wis. | Stratagraft | Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen | Deep partial-thickness burns | Results published in Burns showed the study met its co-primary efficacy endpoints of autograft sparing and durable wound closure by 3 months; significantly smaller area of burn wounds treated with Stratagraft required autografting by 3 months compared to the area of burn wounds treated exclusively with autograft (p<0.0001); 3 12 14 96% of the burn sites treated with stratagraft did not require autografting; 83.1% patients achieved durable wound closure treatment site without autografting (95% ci: 74.4, 91.8) in months similar to autograft control (86% [95% 77.8, 94.0%]); secondary endpoint data showed cosmesis at and was clinically months; significantly lower mean donor pain intensity observed through day < td> | 7/7/21 | Dermatologic |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Takhzyro (lanadelumab) | Kallikrein inhibitor | Hereditary angioedema | Open-label extension of Help study showed mean reduction in attack rate compared to baseline in study population (n=212) of 87.4% and median reduction of 97.7% for those treated for mean duration of 29.6 months | 7/10/21 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Oxlumo (lumasiran) | Subcutaneous small interfering RNA therapeutic targeting glycolate oxidase | Primary hyperoxaluria type 1 | Top-line results of 6 months treatment showed substantial reduction (n=21) in plasma oxalate from baseline in both dialysis-independent and -dependent patients; demonstrated positive results across key secondary endpoints, including measures of urinary oxalate and additional measures of plasma oxalate; no deaths and serious events; company plans to submit supplemental NDA to FDA and EMA in the late 2021 | 7/29/21 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon PTH (palopegteriparatide) | Parathyroid hormone ligand/receptor agonist | Hypoparathyroidism | Target enrollment achieved in Pathway trial; top-line data now anticipated in first quarter of 2022, with potential NDA submission in mid-2022; Japan's PMDA accepted clinical trial notification for Pathway Japan trial to enroll at least 12 Japanese participants, who will start with 18-µg dose and be followed over 26 weeks while titrating to optimal dose | 7/6/21 | Endocrine/Metabolic |
| Savara Inc., of Austin, Texas | Molgradex (molgramostim nebulizer solution) | Inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor | Autoimmune pulmonary alveolar proteinosis | First patient dosed; expected to enroll 160 patients | 7/1/21 | Endocrine/Metabolic |
| Galapagos NV, of Mechelen, Belgium | Filgotinib | JAK inhibitor | Ulcerative colitis | Post-hoc analyses from Selection program showed 18.8% of biologic-naive participants dosed at 200 mg vs. 9.5% for placebo achieved composite score of RB=0 and SF?1 as early as day 9 in induction study A (p<0.05); 7 200 10.7% of biologic-experienced participants dosed at mg vs. 4.2% for placebo achieved same results as early day in induction study b (p<0.05)< td> | 7/10/21 | Gastrointestinal |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | Stelara (ustekinumab) | Dual IL-12/IL-23 receptor antagonist | Ulcerative colitis | In long-term extension of Unifi study, 55.2% of adults with moderately to severely active disease who initially responded to treatment at 90 mg every 8 or 12 weeks (n=348) sustained symptomatic remission rates at week 152 and 96.4% of these were corticosteroid-free | 7/9/21 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor (THR)-? selective agonist | Nonalcoholic steatohepatitis | First patient dosed | 7/13/21 | Gastrointestinal |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | Adeno-associated virus vector encoding human coagulation factor VIII | Hemophilia A | Over 90 % (n=134) in the GENEr8-1 study had an annualized bleed rate of 0 or a lower bleed rate than baseline after week 4; in prespecified group of non-interventional baseline observational study (rollover population; n=112) decreased from baseline on factor VIII prophylaxis by 99% from 3,961.2 (median 3,754.4) to 56.9 (median 0) IU/kg/year after week 4 (p<0.001); 1 significant increase in mean endogenous factor viii expression level from an imputed baseline of iu dl to 42.9 (median 23.9) (p<0.001)< td> | 7/19/21 | Hematologic |
| Glaxosmithkline plc, of London | Daprodustat | HIF-PH inhibitor | Anemia in chronic kidney disease | Ascend study (n=8,000) met its primary endpoint; improvement in hemoglobin (Hgb) levels in untreated patients and maintaining Hgb levels in patients treated with an erythropoietin stimulating agent (ESA); noninferior to cardiovascular outcomes compared to ESA and well-tolerated in both non-dialysis and dialysis patients | 7/16/21 | Hematologic |
| Global Blood Therapeutics Inc., of South San Francisco | Inclacumab | P-selectin inhibitor | Vaso-occlusive crises associated with sickle cell disease | Initiated 2 phase III studies in patients ages 12 and older | 7/22/21 | Hematologic |
| Roche Holding AG, of Basel, Switzerland | Hemlibra (emicizumab) | Bispecific factor IXa- and factor X-directed antibody | Hemophilia A | Final results showed Hemlibra was effective, consistent annualized bleed rate with observations reported from the pivotal Haven studies; no new safety signals; pooled data showed Hemlibra was effective, well tolerated and no impact in body weight among obese and non-obese adults with hemophilia A | 7/2/21 | Hematologic |
| Roche Holding AG, of Basel, Switzerland | Hemlibra (emicizumab) | Bispecific factor IXa- and factor X-directed antibody | Hemophilia A | Hemlibra continued to demonstrate effective bleed control in the Stasey study with 82.6% of participants experiencing no bleeding episodes that required treatment; no new safety signals in longer treatment in adults and adolescents (once-weekly for up to 2 years); associated with a low incidence of anti-drug antibody (ADA) development, 5.2% tested positive for ADA; 2.6% were classified as having ADAs that were neutralizing in vitro | 7/19/21 | Hematologic |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Recombinant von Willebrand factor | Recombinant proteins | von Willebrand's disease | Study met its primary endpoint; reduced spontaneous, treated annualized bleeding rates (sABRs) and maintained level of hemostatic control in patients who switched from prophylaxis with plasma-derived von Willebrand factor; sABR was reduced by 91.5% compared with patients previously taking on-demand VWF historical ratio (95% CI): 0.09 (0.02, 0.35) in prior OD arm; 0.55 (0.09, 3.52) in switch arm; no new risks and serious adverse events | 7/20/21 | Hematologic |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Generalized myasthenia gravis | Top-line results showed study met its primary endpoint; high statistical significance change from baseline in the MG-ADL total score in patient-reported assessment at week 26; well-tolerated and consistent safety profile | 7/15/21 | Immune |
| Equillium Inc., of La Jolla, Calif. | Itolizumab | Monoclonal antibody targeting CD6 | First-line acute graft-vs.-host-disease | Company plans to intitate single pivotal phase III study in the fourth quarter of 2021 | 7/12/21 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Immunoglobulin G-degrading enzyme from Streptococcus pyogenes | Kidney transplant rejection | 84% of crossmatch positive patients who received study drug prior to transplant had functioning allograft after 3 years; 3 allograft losses occurred during first 6 months with 2 more between 2 and 3 years; after 3 years, survival rate was 90% and mean estimated glomuleral filtration rate was 55 mL/min/1.73m2; data accepted for publication in American Journal of Transplantation | 7/9/21 | Immune |
| Incyte Corp., of Wilmington, Del. | Jakafi (ruxolitinib) | JAK1/JAK2 inhibitor | Graft-vs.-host disease | Reach-3 Study showed significant improvements in overall response rate (ORR) at week 24 (49.7% vs. 25.6%; p<0.001) and best orr (76.4% vs. 60.4%) available therapy (bat) arm; statistically significant clinically meaningful improvements in key secondary endpoints; ruxolitinib achieved longer failure-free survival than bat arm (median ffs not yet reached 5.7 months; hazard ratio, 0.37; 95% ci, 0.27 to 0.51; p<0.0001); also had greater self-reported symptoms compared (modified lee symptom scale responder rate: 24.2% 11%; or, 2.62; p="0.001);" new subgroup analysis demonstrated higher for patients treated with at baseline; no safety signals; mortality rates similar (18.8% 16.5% arm); results published the england journal of medicine < td> | 7/14/21 | Immune |
| Kedrion Biopharma Inc., of Fort Lee, N.J. | KIG-10 | 10% intravenous immunoglobulin | Primary immunodeficiency disease | First patient dosed; expected to enroll 30 patients to assess the efficacy, safety and pharmacokinetics in pediatric patients ages 2 to 17 | 7/13/21 | Immune |
| Aicuris Anti-infective Cures GmbH, of Wuppertal, Germany | Pritelivir | Helicase primase inhibitor | Acyclovir-resistant mucocutaneous herpes simplex virus | Initiated pivotal phase III study; plans to enroll 128 patients | 7/8/21 | Infection |
| AM-Pharma BV, of Utrecht, the Netherlands | Ilofotase alfa | Recombinant human alkaline phosphatase (AP) constructed from 2 naturally occurring human isoforms of the AP enzyme | Sepsis-associated acute kidney injury | First patient enrolled | 7/1/21 | Infection |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Lok | Antibiotic lock solution | Catheter-related bloodstream infections | Independent data monitoring committee recommended to continue the trial with protocol defined sample size to achieve primary endpoint; no safety concerns | 7/1/21 | Infection |
| Corvus Pharmaceuticals Inc., of Burlingame, Calif. | Mupadolimab | Anti-CD73 | COVID-19 | Discontinued phase III study due to positive trends of COVID-19 vaccines, lowering serious infection and hospitalizations; discontinuation not related to safety or efficacy; company continues to focus mupadolimab in phase I/Ib cancer studies | 7/16/21 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon lambda (Lambda) | Pegylated recombinant human interferon lambda 1; interferon lambda receptor 1 agonist | COVID-19 | First patient dosed in phase III together study | 7/6/21 | Infection |
| Entasis Therapeutics Inc., of Waltham, Mass., and Zai Lab Ltd., of Shanghai | Sulbactam-durlobactam | Beta-lactam antibiotic; beta-lactamase inhibitor | Carbapenem-resistant Acinetobacter infections | Completed patient enrollment; top-line data expected early in the fourth quarter of 2021 | 7/27/21 | Infection |
| Frontier Biotechnologies Inc., of Nanjing, China | Aikening (albuvirtide) | Long-acting HIV-1 fusion inhibitor | HIV infection | Top-line results of phase III Talent study using albuvirtide and boosted lopinavir (LPV) combination was noninferior to LPV-based 3-drug arm (75.7% vs. 77.3%); % of HIV RNA <400 48 copies ml subjects treated at weeks was 88.1% and 85.4%, respectively; viral load decreased by an average of 2.2 2.1 log10 (p>0.05); CD4 increased by an average of 139.1 and 142.3 cells/µL (p>0.05); rapid and durable viral suppression for 48 weeks compared to standard second-line 3-drug combination therapy; good clinical adherence (>97%) of long-acting weekly injections and demonstrated together with good overall safety and tolerability | 7/19/21 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg) | HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor | HIV infection | Pooled analysis of a 48-week open-label extension of 2 phase III studies showed 99% of participants achieved and maintained an undetectable viral load (HIV-1 RNA <50 copies ml) in treatment-naïve adults; well-tolerated< td> | 7/19/21 | Infection |
| Johnson & Johnson, of New Brunswick | Single-shot COVID-19 vaccine | Single-shot COVID-19 vaccine | COVID-19 prophylaxis | Results published in Biorxiv showed vaccine generated neutralizing antibody activity against the Delta variant at an even higher level and other highly prevalent SARS-CoV-2 viral variants; 85% effective against severe/critical disease and demonstrated protection against hospitalization and death; durability of the immune response lasted through at 8 months in blood samples obtained from a subset of participants (n=8) from phase III Ensemble study | 7/1/21 | Infection |
| Mesoblast Ltd., of New York | Remestemcel-L | Mesenchymal stem cells obtained from the bone marrow of healthy adult donors | Ventilator-dependent COVID-19 with moderate/severe acute respiratory distress syndrome | Improved respiratory function in prespecified analyses by resolution or improvement using the Berlin Criteria at each of days 7, 14, 21 and 30 post-randomization with odds ratio (OR) at day 30 relative to controls of 2.2, 95% CI (1.0, 4.7) in patients under 65 years old; improved respiratory function at day 7 relative to controls and also prolonged or higher dosing in patients older than 65; enhanced respiratory function to an even greater extent in patients under 65 who received dexamethasone at days 7, 14, 21 and 30 post-randomization with OR at day 30 relative to controls on dexamethasone alone of 3.6, 95% CI (1.2, 10.7) in patients under 65 | 7/15/21 | Infection |
| Mesoblast Ltd., of New York | Remestemcel-L | Mesenchymal stem cells obtained from the bone marrow of healthy adult donors | Ventilator-dependent COVID-19 with moderate/severe acute respiratory distress syndrome | 90-day survival outcome study showed significantly reduced mortality by 48% compared to controls in a prespecified analysis of 123 treated patients under 65 years old (26% vs. 44%, hazard ratio 0.52, 95% CI [0.277, 0.964], p=0.038); 46% mortality reduction reported at 60 days (p=0.048) and with durable treatment benefit | 7/19/21 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Oteseconazole (VT-1161) | Lanosterol 14-alphademethylase inhibitor | Recurrent vulvovaginal candidiasis | Study met primary and secondary endpoints; protected more than 90% of participants from having a recurrence during the 48-week maintenance phase compared to approximately 40% in the control group; safe and well-tolerated; no drug-related severe adverse events; | 7/29/21 | Infection |
| Novan Inc., of Durham, N.C. | SB-206 (berdazimer sodium) | Topical nitric oxide-releasing antiviral product | Molluscum contagiosum | Last patient completed week 24 follow-up visit in the pivotal B-Simple4 study | 7/28/21 | Infection |
| Ocugen Inc., of Malvern, Pa., and Bharat Biotech International Ltd., of Hyderabad, India | Covaxin | COVID-19 spike glycoprotein modulator | COVID-19 | Study showed efficacy of 77.8% across mild, moderate and severe infection and 93.4% protection against severe symptomatic infection; vaccine candidate provided 65.2% protection against B.1.617.2 Delta variant | 7/2/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp (SCY-078, Brexafemme) | 1,3-beta-glucan synthase inhibitor | Refractory fungal disease | Interim analysis of Furi study (n=33) showed 70% achieved clinical improvement; 21% maintained stable disease; 9% indeterminate; well-tolerated with common treatment-related adverse event; interim analysis of Cares study (n=10) showed 80% complete response and 10% indeterminate; 10% died of other causes | 7/12/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp (Brexafemme) | 1,3-beta-glucan synthase inhibitor | Vulvovaginal candidiasis | Pooled results (n=376) from phase III Vanish-303 and Vanish-306 studies showed oral treatment reported 56.9% efficacy in the treatment arm vs. 35.7% in the placebo arm (p=0.001); well-tolerated and common treatment-related adverse events; efficacy was 57% (n=374) in patients <65 years, 52.3% (n="107)" in black patients, 54.7% hispanic or latino patients and 46.2% with bmi>35; consistent overall clinical cure rate | 7/29/21 | Infection |
| Viiv Healthcare Ltd., of London | Dovato (dolutegravir/lamivudine) | 2-drug antiviral regimen | HIV infection | 48-week data from Salsa study met primary endpoint; demonstrated noninferior efficacy compared to continuation of a current antiretroviral regimen (CAR) of at least 3 drugs in the intention-to-treat exposed analysis (snapshot virologic failure: 0.4% of participants in the Dovato arm vs. 1.2% in the CAR arm; adjusted difference: -0.8% [95% CI: -2.4%, 0.8%]); demonstrated a noninferior rate of virologic suppression at week 48, with 94.3% (232/246) of participants achieving HIV-1 RNA <50 c ml vs. 92.7% (229 247) of participants on a car (adjusted difference: 1.6% [95% ci: -2.8%, 5.9%]); overall adverse events were similar between the dovato and arms (73% [180 246] 70% [172 247], respectively; mild no serious events; changes in fasting lipids small comparable study arms; bone proximal tubular renal biomarkers inflammatory both directions observed across evidence immune activation or inflammation treatment arms< td> | 7/19/21 | Infection |
| Viiv Healthcare Ltd., of London, and Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Cabenuva | Extended release injectable suspensions of cabotegravir and rilpivirine hydrochloride | HIV infection | Customize trial with 115 patients and 24 health care providers evaluated both qualitative and quantitative measures across a range of clinic types; findings showed that 100% of participants with available viral load results maintained viral suppression (<50 copies ml, n="102)" and there were no virologic failures; injection site reactions the most common overall adverse event, reported in 72% (78 109) of participants who received ?1 through month< td> | 7/19/21 | Infection |
| Zydus Cadila Group, of Ahmedabad, India | ZyCoV-D | Plasmid DNA vaccine; 3-dose intradermal vaccine | COVID-19 prophylaxis | Interim results from phase III study demonstrated vaccine safe and well-tolerated; showed efficacy of 66.6% for symptomatic disease and 100% efficacy for moderate disease | 7/2/21 | Infection |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Completed recruitment in the first of 2 pivotal clinical trials to evaluate the safety and efficacy; top-line results expected in first quarter 2022 | 7/30/21 | Inflammatory |
| Strongbridge Biopharma plc, of Dublin | Keveyis (dichlorphenamide) | Carbonic anhydrase inhibitor | Primary periodic paralysis | Post-hoc analysis results published in Muscle and Nerve showed long-term treatment was safe and effective; efficacy was maintained over entire 61-week study with no warning | 7/13/21 | Musculoskeletal |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of the muscle relaxant cyclobenzaprine hydrochloride | Fibromyalgia | Interim analysis (n=337) by independent data monitoring committee showed Rally study did not reached statistically significant improvement in the primary endpoint of overall change from baseline in daily diary pain severity scores at 5.6 mg; no new safety signals; stopped new patient enrollment; company plans to continue study with enrolled patients and top-line data expected in the fourth quarter of 2021 | 7/23/21 | Musculoskeletal |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor targeting mast cells and macrophages | Mild to moderate Alzheimer’s disease | Results published in the Alzheimer’s and Dementia showed significant treatment effect relative to placebo in the primary endpoint of change from baseline in ADAS-Cog; 4.5 mg/kg/day (n=182) showed significant benefit relative to placebo (n=176), with a respective change in ADAS-cog from baseline of -1.46 (representing an overall improvement in cognition) vs. +0.69 (representing increased cognitive deterioration); a corresponding ADAS-cog between-group difference of -2.15 (97.5%CI [-3.48, -0.81]), p=0.0003; change in ADCS-ADL from baseline of +1.01 (representing an overall functional improvement) vs. -0.81 for placebo (representing increased functional deterioration); a corresponding ADCS-ADL between-group difference of +1.82 (97.5%CI [(-0.15, 3.79]), p=0.038; incidence of severe adverse event for masitinib 4.5 mg/kg/day was 26.5% vs. 19.3% in the placebo-control group, corresponding to an incidence rate ratio of 1.4 | 7/29/21 | Neurology/Psychiatric |
| Acadia Pharmaceuticals Inc., of San Diego | Pimavanserin | Inverse agonist and antagonist activity 5-HT2A | Dementia-related psychosis | Harmony study (n=392) met its primary endpoint by significantly reducing the risk of relapse of psychosis by 2.8-fold compared to placebo in the double-blind period (hazard ratio (HR)=0.35, 2-sided p=0.005; 1-sided p=0.0023); study also met its secondary endpoint by significantly reducing the time to trial discontinuation for any reason (HR=0.45, 2-sided p=0.005; 1-sided p=0.0024); sustained reduction of psychotic symptoms in 12-week open-label period; 3-fold reduction of risk of psychosis relapse compared to patients on placebo, low rates of adverse event and serious adverse events in 26-week double-blind period; well-tolerated over 9 months; study results published in The New England Journal of Medicine | 7/21/21 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Inc., of Woodcliff Lake, N.J. | Aduhelm (aducanumab) | Human monoclonal antibody directed against amyloid beta | Alzheimer’s disease | Emerge phase III trial showed high-dose aducanumab reduced clinical decline evidenced by a statistically significant treatment effect on prespecified primary and secondary clinical efficacy endpoints compared to placebo; showed reduction in the behavioral and psychiatric symptoms measured by the Neuropsychiatric Inventory-10, tertiary efficacy endpoint; dose-dependent reduction in brain amyloid beta plaques; greater reduction in cerebrospinal fluid markers of tau and neurodegeneration as well as less decline on clinical endpoints; 76% of aducanumab-treated participants with amyloid-related imaging abnormalities showed no symptoms | 7/27/21 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | Zynrelef | Extended-release fixed-dose combination of bupivacaine and meloxicam | Pain | Results published in the Pain Management showed Zynrelef was well-tolerated with similar safety profiles to bupivacaine; demonstrated reduction in both pain and the need for opioids in 58% of bunionectomy and 87% of hernia repair patients ages 65 and older | 7/21/21 | Neurology/Psychiatric |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen (AKCEA-TTR-LRx) | Antisense oligonucleotide that targets human transthyretin | Hereditary transthyretin-mediated amyloid polyneuropathy | Completed and exceeded the enrollment target in phase III study; interim results expected in mid-2022 | 7/29/21 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 (baclofen + naltrexone hydrochloride + sorbitol) | PMP22 gene inhibitor | Charcot-Marie-Tooth disease type 1A | First participant in Europe enrolled in pivotal Premier trial, which is on track to complete enrollment in second quarter of 2022 | 7/12/21 | Neurology/Psychiatric |
| Prilenia Therapeutics B.V., of Naarden, the Netherlands | Pridopidine | Selective S1R agonist | Huntington's disease | Achieved 50% of patient enrollment in pivotal phase III trial | 7/8/21 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 (dihydroergotamine nasal powder) | Targets 5-HT 1d receptor | Migraine | Randomization of the first subject in Summit phase III study; top-line data expected in the second half of 2022 | 7/28/21 | Neurology/Psychiatric |
| Scilex Holdings Inc., of Mountain View, Calif., and Sorrento Therapeutics Inc., of San Diego | SP-102, Semdexa | Dexamethasone sodium phosphate viscous gel | Lumbosacral radicular pain or sciatica | Completed enrollment (n=400) in the phase III Clear trial | 7/20/21 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa., and Jiangsu Nhwa Pharmaceutical Co., of Shenzhen, China | Olinvyk (oliceridine) | G protein-selective mu-opioid receptor agonist | Acute pain | Enrolled first patient | 7/8/21 | Neurology/Psychiatric |
| Abbvie Inc., of North Chicago | AGN-190584 (pilocarpine 1.25%) | Pilocarpine (ophthalmic solution) | Presbyopia | Met both its primary and key secondary efficacy endpoints with patients achieving near and intermediate vision gains; AGN-190584 patients gained 3 lines or more in mesopic (in low light), high contrast, binocular Distance Corrected Near Visual Acuity (DCNVA) at day 30, hour 3 (22.5%, p < 0.0001) and hour 6 (9.7%, p = 0.0114) vs. vehicle; rapid onset of 15 minutes and duration of up to 6 hours in mesopic DCNVA without loss of distance vision after administration at day 30; 75% of participants treated with AGN-190584 achieved a ?2-line improvement in mesopic DCNVA and 93% of participants achieved ?20/40 vision in photopic (daylight) DCNVA | 7/25/21 | Ocular |
| Bausch + Lomb, unit of Bausch Health Cos. Inc., of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany | NOV-03 (perfluorohexyloctane) | Ophthalmic liquid formulation | Dry eye disease | Completed enrollment of 622 participants with meibomian gland dysfunction in Mojave study, designed to replicate findings of previous phase III Gobi trial | 7/7/21 | Ocular |
| Iveric Bio Inc., of New York | Zimura (avacincaptad pegol) | Pegylated aptamer targeting the C5 component of the complement cascade | Geographic atrophy secondary to age-related macular degeneration | Early completion of patient enrollment; top-line data in the second half of 2022 | 7/27/21 | Ocular |
| Radius Health Inc., of Boston | RAD-011 | Synthetic cannabidiol oral solution | Prader-Willi syndrome | Company plans to initiate pivotal phase II/III study by end of this year or first quarter of 2022; expected top-line results in the second half of 2024 | 7/23/21 | Other/Miscellaneous |
| Beyondspring Inc., of New York | Plinabulin | Selective immunomodulating microtubule-binding agent | Non-small-cell lung cancer with EGFR wild-type | Top-line results of Dublin phase III study (n =559) in combination with docetaxel (DP) met its primary and secondary endpoints; statistically significant improvements in overall survival (OS) compared to docetaxel (D) alone (mean OS: p=0.03; OS log rank: p<0.04); 1 4 8 improvement in objective response rate (p<0.03) and progression-free survival(p<0.01); significant reduction the incidence of grade neutropenia cycle day (dp: 5.3% vs. d: 27.8%; p<0.0001); 24-month os 22.1% 12.5%; p <0.01) 36 Month OS rate (DP: 11.7% vs. D: 5.3%; p = 0.04) 48 Month OS rate (DP: 10.6% vs. D: 0%; p value cannot be calculated); no unexpected adverse events concerns were identified |
8/4/21 | Cancer |
| Coherus Biosciences Inc., of Redwood City, Calif., and Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Advanced squamous or non-squamous non-small-cell lung cancer (NSCLC) | Interim analysis from the pivotal Choice-01 (n=465) study revealed it met the primary endpoint; demonstrated a statistically significant and clinically meaningful improvement in progression free survival (PFS) per RECIST v1.1 compared to chemotherapy alone; 218 PFS events were observed with a median follow-up of 7.1 and 7 months in the toripalimab and placebo, respectively; significant improvement in PFS was detected for toripalimab over placebo [hazard ratio (HR)=0.58,95% confidence interval (CI): 0.44-0.77, P=0.0001] with median PFS of 8.3 vs. 5.6 months; 1-year PFS rates for toripalimab and placebo arms were 32.6% and 13.1%, respectively; improvement in PFS was observed in both squamous [HR = 0.55 (95% CI: 0.38-0.83)] and non-squamous [HR=0.59 (95% CI: 0.40-0.87)] NSCLC and regardless of PD-L1 expression; Toripalimab in combination with chemotherapy, as compared with chemotherapy alone, resulted in better objective response rate (squamous NSCLC: 68.7% vs. 58.9%; non-squamous NSCLC: 58.6% vs. 26.5%) and median duration of response (squamous NSCLC: 6.9 months vs. 4.2 months; non-squamous NSCLC: 8.6 months vs. 5.1 months); overall survival data were not yet mature as of 3-7-21 | 8/18/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate comprising a humanized anti-HER2 antibody attached to a topoisomerase I inhibitor payload via a tetrapeptide linker | HER2-positive metastatic breast cancer | Independent data monitoring committee concluded Destiny-Breast03 study met primary endpoint in interim analysis; showed highly statistically significant and clinically meaningful improvement of progression-free survival; strong trend toward improved overall survival compared to trastuzumab emtansine (Astrazeneca plc); no new safety issues | 8/9/21 | Cancer |
| Everest Medicines Ltd., of Shanghai | Sacituzumab govitecan-hziy | Humanized anti-Trop2 monoclonal antibody-drug conjugate | Metastatic urothelial cancer | First patient dosed | 8/26/21 | Cancer |
| Geron Corp., of Foster City, Calif. | Imetelstat | Telomerase inhibitor | Lower-risk myelodysplastic syndromes | Achieved 91% of the planned enrollment in IMerge phase III study; expects the trial to be fully enrolled in the fourth quarter of 2021; top-line results expected in the first quarter of 2023 | 8/16/21 | Cancer |
| Innovent Biologics Inc., of San Francisco, and Eli Lilly and Co., of Indianapolis | Tyvyt (sintilimab) | PD-1-directed human monoclonal antibody | Nonsquamous non-small-cell lung cancer | Results published in the Journal of Thoracic Oncology showed Orient-11 study demonstrated, with median follow-up of 22.9 months, sustained overall survival (OS) benefit of sintilimab in combination with pemetrexed and platinum chemotherapy (HR=0.60, 95% CI: 0.45-0.79; p=0.0003) and placebo combination was 16.8 months; high or medium immune cell infiltration was strongly associated with improved progression-free survival (PFS) in the sintilimab combination group; high MHC class-II presentation pathway expression was significantly correlated with prolonged PFS (HR=0.32, 95% CI: 0.19-0.54; p<0.0001) and os (hr="0.36," 95% ci: 0.20-0.64; p="0.0005)" in the sintilimab combination group< td> | 8/5/21 | Cancer |
| Innovent Biologics Inc., of San Francisco, and Eli Lilly and Co., of Indianapolis | Tyvyt (sintilimab) | PD-1-directed human monoclonal antibody | Unresectable, locally advanced, recurrent or metastatic gastric or gastroesophageal junction adenocarcinoma | Phase III Orient-16 study met its predefined primary endpoint of overall survival (OS) in combination with chemotherapy; demonstrated a statistically significant improvement in OS in both the intention-to-treat group and PD-L1-positive group; consistent safety profile and no additional safety signals | 8/15/21 | Cancer |
| Mei Pharma Inc., of San Diego, and Kyowa Kirin Co. Ltd., of Tokyo | Zandelisib (ME-401) | Selective phosphatidylinositol 3-kinase delta inhibitor | Relapsed or refractory follicular or marginal zone lymphomas | First patient dosed in Coastal phase III study in combination with rituximab | 8/17/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 | Stage II resected high-risk melanoma | Keynote-16 phase III study met its primary endpoint of recurrence-free survival (RFS); interim analysis as a single agent showed a statistically significant and clinically meaningful improvement in RFS compared with placebo; no new safety signals | 8/5/21 | Cancer |
| Novartis AG, of Basel, Switzerland | Kymriah (tisagenlecleucel) | CAR T therapy | Aggressive B-cell non-Hodgkin lymphoma | Phase III Belinda study did not meet primary endpoint of event-free survival in patients; consistent safety profile | 8/24/21 | Cancer |
| Point Biopharma Global Inc., of Indianapolis | PNT-2002 | Next generation PSMA radioligand | Metastatic castration-resistant prostate cancer | Completed enrollment and initial dosing of 25-patient safety and dosimetry lead-in of Splash study | 8/10/21 | Cancer |
| Polyphor AG, of Allschwil, Switzerland | Balixafortide (POL-6326) | Selective antagonist of chemokine receptor CXCR4 | HER2-negative, locally recurrent or metastatic breast cancer | Updated Fortress data showed combination with eribulin did not meet primary endpoint; analysis conducted following decision to initiate closure of study showed median progression-free survival in overall population was 3.5 months for combo therapy vs. 4 months for patients only on eribulin (p=0.445); in patients with at least 2 prior lines of chemotherapy, median PFS was 3.5 months vs. 4 months (p=0.6158); prespecified analysis of overall survival showed no statistically significant differences in overall population (p=0.5879) or in second-line population (p=0.6126) | 8/3/21 | Cancer |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Libtayo (cemiplimab) | Monoclonal antibody targeting PD-1 | Advanced non-small-cell lung cancer | Top-line initial analysis (n=466) showed combination with chemotherapy reduced the risk of death by 29% compared to chemotherapy alone (hazard ratio: 0.71; 95% confidence interval [CI]: 0.53-0.93; p=0.014); median overall survival was 22 months (95% CI: 16 months to not evaluable) for Libtayo and chemotherapy, and 13 months (95% CI: 12 to 16 months) for chemotherapy alone; no new safety signals | 8/5/21 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Polivy (polatuzumab vedotin) | Anti-CD79b antibody-drug conjugate | Diffuse large B-cell lymphoma | Study met its primary endpoint by demonstrating significantly improved and clinically meaningful progression-free survival in patients; consistent safety outcomes; 40% of patients relapse after initial therapy achieved meaningful treatment effects | 8/8/21 | Cancer |
| Roche Holding AG, of Basel, Switzerland, and Translational Research in Oncology, of Edmonton, Alberta | Giredestrant (GDC-9545) | Oral selective estrogen receptor degrader | Breast cancer | First patient enrolled | 8/31/21 | Cancer |
| Servier Pharmaceuticals, of Boston, part of Servier Group | Tibsovo (ivosidenib) | Oral L-prolinamide-derived inhibitor of isocitrate dehydrogenase 1 | IDH1-mutated acute myeloid leukemia | Agile study in combination with azacitidine met its primary and secondary endpoints; demonstrated a statistically significant improvement in event-free survival compared to placebo; met complete remission rate, overall survival, complete remission with partial hematologic recovery rate and objective response rate; study halted further enrollment based on the recommendation of the independent data monitoring committee | 8/2/21 | Cancer |
| Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel | VB-111 (ofranergene obadenovec) | CD95 modulator; TNF receptor modulator | Platinum-resistant ovarian cancer | Resumed enrollment of new patients after CMC clearance from the FDA | 8/30/21 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Repatha (evolocumab) | Anti-PCSK9 antibody | Coronary artery disease | Huygen study met its primary endpoint; improved plaque stability, increased fibrous cap thickness by 42.7 um in comparison with an increase of 21.5 um (75% increase vs. 39%) on optimized statin therapy alone (p=0.01), as measured by optical coherence tomography (OCT); improved all of the study's secondary endpoints, including decreasing the maximum lipid arc by -57.5° vs. -31.4° (p=0.01); reduced LDL-C from 140 to 28 mg/dL (-80%) vs. reductions from 142 to 87 mg/dL (-39%) with statin optimization alone; no new safety risks | 8/27/21 | Cardiovascular |
| Bayer AG, of Leverkusen, Germany | Elinzanetant | Small-molecule dual antagonist of both the neurokinin-1 and 3 receptors | Vasomotor symptoms (hot flashes) | Initiated phase III study | 8/31/21 | Cardiovascular |
| Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim GmbH, of Ingelheim, Germany | Jardiance (empagliflozin) | SGLT2 inhibitor | Heart failure with reduced ejection fraction | Results from the landmark Emperor-Preserved phase III trial (n=5988) published in The New England Journal of Medicine showed drug significantly reduced the relative risk of the composite endpoint of cardiovascular death or hospitalization for heart failure by 25% compared with placebo | 8/27/21 | Cardiovascular |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Uptravi (selexipag) | Prostacyclin receptor agonist | Pulmonary arterial hypertension | Post-hoc pooled data analysis in patients treated with selexipag within 6 months of diagnosis in the phase III Griphon and phase IIIb Triton trials showed that early initiation of selexipag reduced the risk of disease progression by 52% compared with the control group (hazard ratio [HR] 0.48; 95% confidence interval [CI] 0.35, 0.66; n=649); selexipag on top of double therapy (triple therapy group) showed risk of disease progression was reduced by 48% compared with the double therapy control group (n = 285; HR: 0.52 [95 percent CI 0.30, 0.92] | 8/30/21 | Cardiovascular |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa. | Bentracimab | Recombinant human monoclonal antibody antigen-binding fragment designed to reverse the antiplatelet activity of ticagrelor | Uncontrolled major or life-threatening bleeding or patients who require urgent surgery or invasive procedure | Enrolled 143 patients; top-line results from interim analysis expected later this year | 8/12/21 | Cardiovascular |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR (mutant) expression inhibitor; siRNA agent | Cardiomyopathy in patients with transthyretin-mediated (ATTR) amyloidosis | Achieved full patient enrollment in phase III Helios-B study; enrolled 600 adult patients | 8/9/21 | Cardiovascular |
| Daiichi-Sankyo Co. Ltd., of Tokyo | Lixiana (edoxaban) | Selective inhibitor of factor Xa | Atrial fibrillation | Results from ETNA-AF-Europe Registry (n=9084) showed patients with worsening renal function (WRF) had higher mortality than those without (all-cause mortality: 3.78% vs. 1.9%; cardiovascular death: 2.06% vs. 0.92%, respectively); numerically higher major bleeding and stroke rates vs. those without WRF; intracranial hemorrhage rates remained low irrespective of WRF (0.17% in those with WRF vs. 0.19% in those without) | 8/28/21 | Cardiovascular |
| Daiichi-Sankyo Co. Ltd., of Tokyo | Lixiana (edoxaban) | Selective inhibitor of factor Xa | Atrial fibrillation | Results from ENVISAGE-TAVI AF (n=1426) published in The New England Journal of Medicine showed study met its primary endpoint of edoxaban being noninferior to vitamin K antagonists; rate of the composite primary efficacy outcome was 17.3 per 100 person-years in the edoxaban group and 16.5 per 100 person-years in the vitamin K antagonist group (hazard ratio, 1.05; 95% confidence interval [CI], 0.85 to 1.31; p=0.01 for noninferiority); lower rates of all-cause mortality and ischemic stroke; study did not meet its primary safety endpoint of ISTH-defined major bleeding | 8/28/21 | Cardiovascular |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif., and Novartis AG, of Basel, Switzerland | Pelacarsen (AKCEA-APO(a)-LRx) | Ligand-conjugated antisense (LICA) therapy targeting apolipoprotein A | Cardiovascular disease | Reached 50% enrollment with a target goal of 7,680 trial participants in pivotal phase III study | 8/2/21 | Cardiovascular |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Sotagliflozin | Dual sodium glucose transporter-1/transporter-2 inhibitor | Heart failure | Results from phase III Soloist and Scored trial showed rapid benefit of sotagliflozin; 33% of absolute risk reduction in the composite cardiovascular endpoint in type 2 diabetes patients with acute decompensated heart failure; consistent results in patients with preserved ejection fraction and reduced ejection fraction, with benefit evident within 1 month; 26% reduction in the composite cardiovascular endpoint in patients with type 2 diabetes and chronic kidney disease, with benefit evident within 3 months; reductions in heart failure-related events were more pronounced at the target once-daily dose of sotagliflozin 400 mg; reductions in both myocardial infarction and stroke were greater than reported in studies of selective SGLT2 inhibitors; overall tolerability was comparable to placebo across both trials | 8/23/21 | Cardiovascular |
| Quantum Genomics SA, of Paris | Firibastat | Brain aminopeptidase A inhibitor; prodrug | Myocardial infarction | In phase IIb Quorum study (n=295), after 12 weeks of treatment, the left ventricular ejection fraction evaluated by cardiac MRI increased from 53% to 59% in the firibastat 100-mg twice-daily group, from 51% to 58% in the firibastat 500-mg twice-daily group and 50% to 57% in the ramipril 5-mg twice-daily control group; difference between the 3 groups was not statistically significant; in subgroup of severe patients with an ejection fraction less than 50%, the ejection fraction increased by 5.32% ± 1.67% under firibastat 500 mg, and by 3.51% ± 1.64% under ramipril; safe and well-tolerated | 8/27/21 | Cardiovascular |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK1 inhibitor | Moderate to severe atopic dermatitis | Phase IIIb Heads Up study 24-week results published in JAMA Dermatology; demonstrated statistically significant greater efficacy across all ranked secondary endpoints compared to Dupixent (dupilumab, Sanofi SA/Regeneron Pharmaceuticals Inc.) through week 16, including early reduction in itch and rates of skin clearance improvement; after 1 week, treatment showed 31% reduction in itch (as measured by Worst Pruritus Numerical Rating Scale [NRS]) compared to 9% in the dupilumab (p<0.001) 2 16 24 75 100 at 30-mg; after weeks of treatment, 44% receiving upadacitinib achieved easi vs. 18% dupilumab (p<0.001); weeks, 28% people treated with clear skin (easi 100; p<0.001) and 61% almost 90; p<0.001), compared to 8% 39%, respectively, those dupilumab; additional endpoints week included 75, 90, improvement from baseline worst pruritus nrs (weekly average)< td> | 8/5/21 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast foam (ARQ-154) | PDE4 inhibitor | Scalp and body psoriasis | Enrolled first patient; readout expected in 2022 | 8/25/21 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo. | Sofpironium bromide gel 15% | Anticholinergic | Primary axillary hyperhidrosis | Final patient completed the phase III pivotal Cardigan II study; top-line results of phase III Cardigan I and Cardigan II studies expected in the fourth quarter of 2021 | 8/16/21 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Top-line results of phase III Advocate 1 and 2 study of monotherapy treatment met primary and all key secondary endpoints; skin clearance and itch improvement were met at week 16; consistent safety profile with that of the previous phase II study | 8/16/21 | Dermatologic |
| Pfizer Inc., of New York | Abrocitinib | JAK inhibitor | Moderate to severe atopic dermatitis | Study met its co-primary and key secondary efficacy endpoints; proportion of patients achieving at least a 4-point improvement in the severity of Peak Pruritus Numerical Rating Scale from baseline at week 2 and the proportion of patients achieving EASI90 (?90% improvement from baseline) at week 4; proportion of patients achieving EASI90 at week 16 | 8/30/21 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Human monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13 proteins | Moderate to severe atopic dermatitis | Study met its primary and all secondary endpoints with Dupixent every 4 weeks (200 mg or 300 mg); 28% achieved clear or almost-clear skin compared to 4% with placebo (p=<0.0001); 53% achieved 75% or greater overall disease improvement from baseline compared to 11% with placebo (p="<0.0001);" 70% average in easi 20% 49% itch 2% (p<0.0001); significantly improved measures of observed patient outcomes < td> | 8/30/21 | Dermatologic |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Takhzyro (lanadelumab) | Human monoclonal IgG1 antibody targeting plasma kallikrein | Hereditary angioedema | Open-label extension of Help phase III study showed markedly reduced the frequency of HAE attacks in patients 12 and older who received treatment for a mean duration of almost 2.5 years (29.6 months; 8.2 standard deviation); secondary endpoints of the study showed the mean HAE attack rate observed in the study population (n=209) was reduced by 87.4% (-100; 852.8) overall vs. baseline, and attacks requiring acute treatment (n=106) were reduced by 93.4% (-100; -52.8); reduced the HAE disease burden by being attack-free for a mean (SD) of 97.7% at 300-mg every 2 weeks and the average duration of the attack-free period was 14.8 months; 68.9% experienced an attack-free period of more than 12 months; 54.7% of patients had treatment-emergent adverse events; no reports of serious events | 8/5/21 | Dermatologic |
| UCB SA, of Brussels | Bimekizumab | Humanized IgG1-kappa monoclonal antibody targeting interleukin 17A | Moderate to severe plaque psoriasis | Interim data from Be Bright phase III (n=989) showed 87.5% achieved IGA 0/1, 74.9% achieved BSA ?1% and 62.7% achieved PASI 100 at week 16; 9 out of 10 patients maintained IGA 0/1 to week 48 (94.4% and 96.2% with continuous Q4W and Q8W maintenance dosing, respectively); week 16 BSA ?1% responders, over 9 out of 10 patients maintained BSA ?1% to week 48 in the OLE trial (90.7% and 92.5% with continuous Q4W and Q8W maintenance dosing, respectively); 8 out of 10 patients who achieved complete skin clearance (PASI 100) at week 16 maintained response to week 48 (80.7% and 86.1% with continuous Q4W and Q8W maintenance dosing, respectively) | 8/7/21 | Dermatologic |
| Innovent Biologics Inc., of Suzhou, China | IBI-306 | Recombinant fully human anti-PCSK9 monoclonal antibody | Heterozygous familial hypercholesterolemia | Phase III Credit -2 study (n=148) met primary endpoint; low-density lipoprotein cholesterol levels were significantly reduced from baseline compared with the placebo arm after 12 weeks of treatment; favorable safety profile | 8/11/21 | Endocrine/Metabolic |
| Lysogene SA, of Paris | LYS-GM101 | Adeno-associated virus vector-based gene therapy consisting of a recombinant AAVrh.10 vector encoding feline beta-galactosidase (beta-gal) | GM1 gangliosidosis | First patient dosed; expected to enroll 16 patients | 8/30/21 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | Enrolled and randomized more than 25% of the planned 450 patients for its phase III study | 8/24/21 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | ALXN-1840 | Orally administered small molecule; targeted de-coppering therapy | Wilson disease | Focus phase III (n=214) trial met its primary endpoint with a statistically significant improvement in daily mean copper mobilization from tissues; demonstrated superiority compared with standard-of-care treatments; well-tolerated and mild to moderate adverse events; no neurological worsening upon initiation of treatment was observed | 8/26/21 | Gastrointestinal |
| Zealand Pharma A/S, of Copenhagen | Glepaglutide | Polypeptides; GLP-2 receptor agonist | Short bowel syndrome | First subject dosed in EASE-SBS 1 pivotal phase III trial; results expected in 2022 | 8/19/21 | Gastrointestinal |
| Bayer AG, of Leverkusen, Germany | Finerenone (Kerendia) | Nonsteroidal, selective mineralocorticoid receptor antagonist | Chronic kidney disease and type 2 diabetes | Results from FIGARO-DKD phase III study and Fidelity prespecified meta-analysis published in The New England Journal of Medicine; finerenone significantly reduced the risk of composite primary endpoint of time to first occurrence of cardiovascular death or nonfatal CV events by 13% (relative risk reduction, HR 0.87 [95% CI, 0.76-0.98; p=0.0264]) over a median duration of follow-up of 3.4 years when added to maximum tolerated labeled dose of angiotensin-converting enzyme inhibitor or angiotensin receptor blocker; sustained decrease of eGFR ?40% from baseline over a period of at least 4 weeks, or renal death was lower with finerenone than with placebo, affecting 9.5% and 10.8% patients, respectively, though difference was not statistically significant (HR 0.87 [95% CI, 0.76-1.01; p=0.0689]) over a median duration of follow-up of 3.4 years; reduced the incidence of the primary composite endpoint of a sustained decline in eGFR of ?40%, kidney failure or renal death (HR 0.82 [95% CI, 0.73-0.93; p=0.001]); prespecified exploratory meta-analysis showed finerenone reduced the risk of the composite CV outcome of time to CV death, nonfatal myocardial infarction, nonfatal stroke or hospitalization for heart failure by 14% compared with placebo; sustained ?57% decrease in eGFR from baseline over ?4 weeks, or renal death occurred in 360 (5.5%) patients receiving finerenone and 465 (7.1%) receiving placebo (HR 0.77 [95% CI, 0.67–0.88]); sustained decrease in eGFR by 57% or more from baseline, or renal death occurred in 252 (8.9%) patients and 326 (11.5%) patients in the finerenone and placebo groups, respectively (HR 0.76 [95% CI, 0.65–0.90] | 8/28/21 | Genitourinary/Sexual Function |
| Travere Therapeutics Inc., of San Diego | Sparsentan | Dual-acting antagonist of endothelin type A and angiotensin II type 1 receptors | IgA nephropathy | Top-line interim results of ongoing pivotal phase III Protect study (n=404) met its prespecified primary efficacy endpoint with statistical significance; greater than 3-fold reduction of mean proteinuria (49.8%) from baseline after 36 weeks of treatment compared to active control irbesartan (15.1%) (p<0.0001); well-tolerated and consistent with the observed safety profile; top-line results from confirmatory endpoint analysis expected in second half of 2023< td> | 8/16/21 | Genitourinary/Sexual Function |
| X4 Pharmaceuticals Inc., of Boston | Mavorixafor | CXCR4 receptor antagonist | Waldenström’s macroglobulinemia patients with both MYD88 and CXCR4 mutations | Achieved enrollment milestone in phase III 4WHIM study; 23 patients enrolled and top-line data expected in the fourth quarter of 2022 | 8/3/21 | Hematologic |
| Aimmune Therapeutics, a unit of Société des Produits Nestlé SA, of Vevey, Switzerland | Palforzia | Peanut-derived oral immunotherapy | Peanut allergy | Results from Palisade-ARC004 trial published in the Allergy showed after 2 years of treatment, 80% of patients tolerated a daily maintenance dose of 2,000 mg; demonstrated immunomodulation during first 2 years treatment; IgE levels decreased from the time of Palisade entry to ARC004 exit and IgG4 levels increased; mean peanut SPT wheal diameters decreased from screening to ARC004 exit and reduction after updosing; children and teenagers demonstrated clinically meaningful improvement in the Food Allergy Quality of Life Questionnaire total and domain scores (?0.5) generally rose with the duration of treatment; mild to moderate adverse events and low rates of serious events | 8/4/21 | Immune |
| Biogen Inc., of Cambridge, Mass. | Tysabri (natalizumab | Humanized monoclonal IgG4 kappa antibody against human alpha 4 integrin | Relapsing-remitting multiple sclerosis | Results from phase IIIb Nova study testing every 6-week (Q6W and Q4W) 300-mg natalizumab intravenous administration showed no statistically significant or clinically meaningful differences in primary and secondary endpoints; primary endpoint showed a numerical difference between the mean number of new or newly enlarging T2 hyperintense lesions at week 72 of 0.05 (Q4W) and 0.20 (Q6W) (p=0.0755); proportion of patients that developed new or newly enlarging T2 lesions in each arm was 4.1 % (Q4W) and 4.3 % (Q6W); annualized relapse rates were low at 0.00010 (Q4W) and 0.00013 (Q6W), with 97.9 % patients in the Q4W arm remaining relapse-free compared to 97.2 % of patients in the Q6W arm; proportion of patients that developed T1 hypointense lesions in each arm was 1.1 % (Q4W) and 1.4 % (Q6W); both arms demonstrated 0.5% of participants with gadolinium-enhancing T1 lesions; 1 patient developed lesions 3 months after treatment discontinuation; second patient developed asymptomatic progressive multifocal leukoencephalopathy (PML); safety analysis from Touch prescribing program showed an average Q6W dosing regimen is associated with an 88% reduction (hazard ratio 0.118, p<0.0001) in the probability of pml comparison to approved q4w dose< td> | 8/2/21 | Immune |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Unique form of eicosapentaenoic acid | COVID-19 | Top-line results from Prepare-IT-1 study (n=2,000 )did not meet the primary and/or other endpoints | 8/31/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K., | AZD-7442 | Combination of 2 long-acting antibodies (tixagevimab and cilgavimab) derived from B cells donated by convalescent patients after SARS-CoV-2 virus | COVID-19 | Provent phase III trials (n=5197) met primary endpoint; achieved a statistically significant reduction in the incidence of symptomatic COVID-19; 77% reduction (95% confidence interval: 46, 90), compared to placebo; well-tolerated | 8/20/21 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-196 and BRII-198 | Human monoclonal antibody targeting the spike glycoprotein of SARS-CoV-2 | COVID-19 | Completed enrollment of 846 outpatients at high risk of clinical progression who presented with symptomatic COVID-19 both early (?5 days) and late (5-10 days) following symptom onset; to evaluate combined primary endpoint of hospitalizations and death, relative to placebo, in the 28 days following treatment | 8/5/21 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-196 and BRII-198 | Human monoclonal antibody targeting the spike glycoprotein of SARS-CoV-2 | COVID-19 | Combination of antibodies demonstrated a statistically significant reduction of 78% in the combined endpoint of hospitalization and death vs. placebo in 837 non-hospitalized COVID-19 patients at high risk of clinical progression; reduction in both hospitalizations (12 active vs. 45 placebo) and deaths (1 active vs. 9 placebo) was observed | 8/24/21 | Infection |
| Curevac NV, of Tübingen, Germany | CVnCoV | Non-chemically modified mRNA, encoding the prefusion stabilized full-length spike protein of the SARS-CoV-2 virus formulated within lipid nanoparticles | COVID-19 | Results from Herald study published in The Lancet showed overall vaccine efficacy of 48% against COVID-19 disease of any severity, including single non-respiratory mild symptoms; significant protection was demonstrated among participants in the age group of 18 to 60, with an efficacy of 53% against disease of any severity and across all 15 identified strains; protection against moderate to severe disease for this age group was calculated to be 77%; CVnCoV provided 100% protection against hospitalization or death | 8/31/21 | Infection |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Olumiant (baricitinib) | JAK1/JAK2 inhibitor | Hospitalized patients with COVID-19 requiring oxygen | Substudy in patients with mechanical ventilation or extracorporeal membrane oxygenation who received baricitinib plus standard of care (SOC) were 46% less likely to die by day 28 compared to patients who received placebo plus SOC (nominal p=0.0296; hazard ratio [HR] [95% CI] = 0.54 [0.31, 0.96]; cumulative proportion of patients who died by day 28 was 39.2 % (n/N: 20/51) in the baricitinib arm vs. 58 % in the placebo arm (n/N: 29/50); mortality benefit was observed by day 60 (HR [96% CI] = 0.56 [0.33, 0.97]) with a cumulative proportion of death of 45.1% (n/N: 23/51) for baricitinib compared to 62% for placebo (n/N: 31/50); frequency of adverse events, serious adverse events and serious infections were similar in the baricitinib group (88%, 50% and 44%, respectively) compared to placebo (95.9%, 71.4% and 53.1%, respectively) in day 28; venous thromboembolic events were reported in 6% of patients treated with baricitinib and 6.1% of patients treated with placebo; no new safety signals were identified | 8/3/21 | Infection |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | COVID-HIG | SARS-CoV-2 immune globulin intravenous; plasma-derived therapy | COVID-19 | Initiated phase III trial to evaluate the efficacy and safety in adult patients at risk of progression to severe disease (55 and older and those 18 and older who are immunocompromised) | 8/25/21 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | COVID-19 | Live-Air phase III study in hospitalized Black and African American patients having a CRP<150 mg l showed 9-fold increase in likelihood of survival without ventilation (swov) [n="51," p="0.0412];" overall population with crp<150 2.5-fold increased swov [mitt, n="351," < td> | 8/4/21 | Infection |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Mavrilimumab | Fully-human monoclonal antibody that blocks activity of GM-CSF | COVID-19-related acute respiratory distress syndrome and in giant cell arteritis | Study continues to enroll non-mechanically ventilated patients; discontinued mechanically ventilated patients in the phase III study; results expected in the first quarter of 2022 | 8/3/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Vaxneuvance | Pneumococcal 15-valent conjugate vaccine | Prevention of invasive disease caused by Streptococcus pneumoniae serotypes | Top-line results from PNEU-PED study met key immunogenicity and safety endpoints in healthy infants enrolled between 42-90 days of age; Vaxneuvance had a safety profile comparable to PCV13 at any vaccine dose; non-inferior to PCV13 for all 13 shared serotypes based on serotype-specific response rates | 8/25/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Initiated third COVID-19 vaccine dose in participants with impaired immune systems in OCTAVE-DUO study | 8/25/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | mRNA vaccine | COVID-19 prophylaxis | Initial data from ongoing phase I/II/III booster trial of a third dose given 6 months after the second dose showed consistent tolerability profile; showed high neutralization titers in both younger and older adults compared to those observed after 2 primary doses; immune sera showed neutralizing titers against the original SARS-CoV-2 wild-type strain that are more than 5- to 8-fold, and more than 15- to 21-fold against the B.1.351 lineage (Beta variant) than after 2 primary doses; neutralizing titers against the Delta variant that are more than 5- to 11-fold than after 2 doses | 8/9/21 | Infection |
| SK Bioscience Co. Ltd., a vaccine business subsidiary of Seoul-based SK Group, and Glaxosmithkline plc, of London | GBP-510 | Self-assembled nanoparticle vaccine candidate targeting the receptor binding domain of the SARS-CoV-2 spike protein combined with pandemic adjuvant | COVID-19 | Initiated phase III study; expected to enroll 4,000 participants; results expected in the first half of 2022 | 8/31/21 | Infection |
| Summit Therapeutics Inc., of Cambridge, Mass. | Ridinilazole | Small-molecule antibacterial agent | Initial and recurrent Clostridioides difficile infection | Company plans to combine its 2 blinded pivotal Ri-Codify trials testing ridinilazole vs. vancomycin into a single study; current enrollment is 753 patients, with each trial having enrolled just over 50% of targeted goal | 8/11/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1553 | Single-shot chikungunya vaccine; monovalent single-dose live-attenuated vaccine | Chikungunya virus infection | Top-line results of phase III pivotal study (n=4115) met its primary endpoint, inducing protective CHIKV neutralizing antibody titers in 98.5% of participants 28 days after receiving a single shot (264 of 268 subjects from the per-protocol subgroup tested for immunogenicity, 95% CI: 96.2-99.6); mild to moderate adverse events and resolved within 3 days; well-tolerated in all groups; final results expected within next 6 months | 8/5/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Initiated further phase III (VLA2001-304) study | 8/11/21 | Infection |
| Vanda Pharmaceuticals Inc., of Washington | Tradipitant | Neurokinin-1 receptor antagonist | COVID-19 pneumonia | Enrollment closed because study met predefined futility criteria; independent data and safety monitoring board determined the study showed a significant difference between treatment arms at the prespecified primary endpoint and recommended termination of the study for futility | 8/3/21 | Infection |
| Cidara Therapeutics Inc., of San Diego | Rezafungin | Echinocandin antifungal | Candidemia and invasive candidiasis | Completed enrollment of 184 patients in phase III Restore trial; top-line data expected by the end of 2021 | 8/17/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | Synthetic form of the vasoactive intestinal polypeptide | Critical COVID-19 | Data safety monitoring board found no new safety concerns in the trial and recommended to continue the enrollment | 8/18/21 | Infection |
| Bone Therapeutics SA, of Gosselies, Belgium | JTA-004 | Viscosupplement | Knee osteoarthritis | Top-line results of phase III study did not meet the primary and consequently the key secondary endpoints; no statistically significant difference in knee pain reduction among JTA-004, placebo and active comparator; favorable safety profile | 8/30/21 | Inflammatory |
| Cytokinetics Inc., of South San Francisco | Reldesemtiv | Oral skeletal troponin activator | Amyotrophic lateral sclerosis | Initiated patient enrollment in phase III Courage-ALS trial | 8/2/21 | Musculoskeletal |
| Amneal Pharmaceuticals Inc., of Bridgewater Township, N.J. | IPX-203 | Oral formulation of CD/LD extended-release capsules | Parkinson’s disease | Top-line results of phase III study met its primary endpoint; demonstrated statistically significant improvement in efficacy for IPX-203 compared to immediate-release CD/LD; IPX-203 treatment resulted in 0.53 more hours of good on time than immediate-release CD/LD (p=0.0194), when comparing change from baseline (week 7) in both study arms; secondary endpoint for change from baseline in off time showed IPX-203 resulted in significantly less off time compared with immediate-release CD/LD (-0.48 hr, p=0.0252) | 8/25/21 | Neurology/Psychiatric |
| Antibe Therapeutics Inc., of Toronto | Otenaproxesul | Hydrogen sulfide-releasing derivative of naproxen | Pain and inflammation | Study paused because of pre-specified safety threshold was exceeded | 8/3/21 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-05 | Oral NMDA receptor antagonist; 45-mg dextromethorphan and 105-mg bupropion | Resistant depression | Merit study met primary and key secondary endpoints; substantially and statistically significantly delayed the time to relapse of depressive symptoms as compared to placebo (p=0.002), with no relapses observed with AXS-05 over at least 6 months of double-blind treatment; relapse prevention, based on the rates of relapse during the double-blind treatment period (0% of AXS-05 patients, 36.4% of patients switched from AXS-05 to placebo, p=0.004); well-tolerated; no treatment-emergent adverse events reported in >1 patient in the AXS-05; 1 patient experienced 2 serious adverse events not related to study | 8/9/21 | Neurology/Psychiatric |
| Biovie Inc., of Santa Monica, Calif. | NE-3107 | Orally bioavailable small-molecule adrenal steroid hormone and insulin sensitizer; NFKB activation inhibitor; selective inhibitor of inflammatory ERK signaling | Alzheimer’s disease | First patient enrolled; data readout expected in the end of 2022 | 8/5/21 | Neurology/Psychiatric |
| Cortexyme Inc., of South San Francisco | Atuzaginstat (COR-388) | Lysine gingipain inhibitor; brain penetrant oral small molecule targeting P. gingivalis infection | Mild to moderate Alzheimer’s disease | Ongoing phase II/III study in patients analyzed at baseline in the Gain trial were positive for anti-P. gingivalis antibodies (IgG) in their cerebrospinal fluid; less than 2% of patients analyzed had a leaky blood brain barrier, defined by an albumin index greater than 9; anti-P. gingivalis IgG in the CSF was only very weakly correlated to the albumin index (r=0.22); top-line data expected by mid-November 2021 | 8/25/21 | Neurology/Psychiatric |
| Gensight Biologics SA, of Paris | Lumevoq | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 (ND4) gene | Leber hereditary optic atrophy | Published long-term results of Restore study in the Journal of Neuro-Ophthalmology showed sustained improvement in best-corrected visual acuity (BCVA) and quality of life scores 3 years after treatment; improvement in both eyes; mean BCVA steadily improved to 1.26 LogMAR at 48 months after onset (3 years post injection), remaining on?chart (i.e., better than 1.6 LogMAR) throughout the follow-up period | 8/31/21 | Neurology/Psychiatric |
| Grunenthal GmbH, of Aachen, Germany, and subsidiary Averitas Pharma Inc. | Qutenza (capsaicin) 8% patch | TRPV1 agonist | Postsurgical neuropathic pain | Enrolled first patient in the randomized, double-blind AV-001 trial | 8/10/21 | Neurology/Psychiatric |
| Impel Neuropharma Inc., of Seattle | INP-104 (Trudhesa) | Dihydroergotamine mesylate | Acute migraine | Trial (n=360) exhibited well-tolerated safety profile throughout the initial 24-week treatment period and the full 52-week treatment period; no serious adverse events; 36.7% patient-reported adverse events; 6.8% discontinued treatment over 24 weeks; 74% of patients completed 24-week period; 98% of patients with pain freedom had sustained pain freedom through 24 hours and 95% through 48 hours; 38% of patients who self-reported 2-hour pain freedom for their first treated attack; 7.1% and 14.3% of patients self-reported recurrence of migraine at 24 and 48 hours post-administration, respectively; faster and more consistent onset of effect than other treatment; results published in Headache | 8/9/21 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Valbenazine | VMAT2 inhibitor | Chorea in Huntington disease | Completed patient enrollment in phase III Kinect-HD study; top-line results expected by the end of 2021; initiated expanded patient enrollment in open-label extension study Kinect-HD2 study | 8/5/21 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Olinvyk (oliceridine) | G protein-selective mu-opioid receptor agonist | Pain | Results of an exploratory analysis showed oliceridine-treated patients are 50% less likely to experience an adverse event compared to morphine patients at equivalent levels of analgesia; odds ratio (OR) for a comparison of the effect of treatment with oliceridine vs. morphine was 0.507 for the pooled hard- and soft-tissue studies data (p = 0.009); OR <1 indicate a lower likelihood of achieving the safety composite endpoint associated with treatment vs. morphine; or was 0.499 (p="0.042)" and 0.542 for hard-tissue soft-tissue studies, respectively< td> | 8/10/21 | Neurology/Psychiatric |
| Novaliq GmbH, of Heidelberg, Germany | Cyclasol | Topical anti-inflammatory and immunomodulating ophthalmic solution containing 0.1% cyclosporine A | Dry eye disease | Completed enrollment of 834 participants | 8/10/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010, Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Top-line data from Norse two study met its primary and key secondary endpoint for efficacy; gained at least 15 letters BCVA was met and highly statistically significant and clinically relevant; 41% of patients gained at least 15 letters with bevacizumab-vikg (p = 0.0052) vs. 23% for ranibizumab; primary endpoint was also statistically significant and clinically relevant in the secondary per-protocol (PP) dataset (p = 0.04), 41% with bevacizumab-vikg vs. ranibizumab 23% in intent-to-treat (ITT) dataset; BCVA score change from baseline to month 11 in the primary ITT dataset was also highly statistically significant and clinically relevant (p = 0.0043); mean change in BCVA was observed with ranibizumab of 5.8 letters and the mean change with bevacizumab-vikg was 11.2 letters; statistically significant in the secondary PP dataset (p = 0.05) with a mean change in letters with ranibizumab of 7 letters and with bevacizumab-vikg 11.1 letters; safe and well tolerated | 8/3/21 | Ocular |
| Zhaoke Ophthalmology Pharmaceutical Ltd., of Hong Kong | Cyclosporine A | Ophthalmic gel; natural cyclic polypeptide immunosuppressant; calcineurin inhibitor | Dry eye disease | Study met its primary endpoint in inferior fluorescein corneal staining score; demonstrated statistically significant (P<0.0001) and clinically meaningful improvements compared to the patient group receiving comparator treatment< td> | 8/19/21 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Extracellular domains of human VEGFR-3 antibodies fused to the Fc-domain | Wet age-related macular degeneration | Initiated recruitment in Canada for 2 phase III pivotal studies | 8/9/21 | Ocular |
| Vertex Pharmaceuticals Inc., of Boston | Trikafta | Combination of elexacaftor/tezacaftor/ivacaftor and ivacaftor | Cystic fibrosis | Results from phase III study published in The New England Journal of Medicine showed Trikafta improved the percent predicted forced expiratory volume in 1 second (ppFEV1) by 3.7% points (95% CI, 2.8 to 4.6; p<0.001) from baseline and by 3.5 percentage points (95% ci, 2.2 to 4.7; p<0.001) vs. active control; improved sweat chloride concentration ?22.3 mmol liter ?24.5 ?20.2; ?23.1 ?26.1 ?20.1; change in the cfq-r respiratory domain score was +10.3 8.0 12.7) +8.7 control 5.3 12.1)< td> | 8/26/21 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 | 5-HT3 receptor antagonist | Alcohol use disorder | Successfully enrolled 302 patients | 8/31/21 | Toxicity and Intoxication |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor targeting mast cells and macrophages | Metastatic castrate refractory prostate cancer | Results of AB12003 phase III study published in the Journal of Urology showed masitinib plus docetaxel had significant progression-free survival (PFS) benefit in mCRPC patients with ALP ? 250 IU/ml; hazard ratio of 0.79 [0.64;0.97] (p=0.0087), corresponding to a 21% reduction in risk of progression relative to control; progressively greater masitinib treatment effect was observed for lower baseline ALP levels with a significant 47% reduced risk of progression in patients with ALP?100 IU/mL (hazard ratio=0.53, p=0.002); no PFS benefit was observed for the overall population; consistent safety profile with no new safety signals observed | 9/13/21 | Cancer |
| Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Active, relapsed or refractory acute myeloid leukemia | Completed enrollment of pivotal phase III study; data expected in the fourth quarter of 2021; top-line data in mid-2022 | 9/15/21 | Cancer |
| Astex Pharmaceuticals Inc., of Pleasanton, Calif., and Otsuka Pharmaceutical Co. Ltd., of Tokyo | Inqovi (decitabine and cedazuridine) | Tablet formulation of hypomethylating agent and cytidine deaminase inhibitor | Myelodysplastic syndromes and chronic myelomonocytic leukemia | Results of Ascertain phase III study achieved median overall survival of 31.7 months; efficacy data demonstrated an overall response rate of 62%, with 22% of patients achieving a complete response | 9/23/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) and tremelimumab | PD-L1-targeting antibody and CTLA4-targeting antibody | Non-small-cell lung cancer | In the Poseidon trial, Imfinzi plus tremelimumab and chemotherapy produced improvements in progression-free survival (HR=0.72, p=0.00031) and overall survival (HR=0.77, p=0.00304) compared to chemotherapy alone | 9/9/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Hutchmed Ltd., of Hong Kong | Orpathys (savolitinib) and Tagrisso (osimertinib) | Inhibitors of c-Met and EGFR | Non-small-cell lung cancer | Initiated phase III Sanovo study in China | 9/8/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | High-level results from the Propel phase III trial in combination with abiraterone demonstrated a statistically significant and clinically meaningful improvement in radiographic progression-free survival vs. standard of care; improved overall survival; consistent safety and tolerability profile | 9/24/21 | Cancer |
| Bayer AG, of Leverkusen, Germany | Nubeqa (darolutamide) | Androgen receptor inhibitor | Non-metastatic castration-resistant prostate cancer | Results showed darolutamide and androgen deprivation therapy (ADT) prolonged time to first prostate cancer-related invasive procedures, an exploratory endpoint (HR=0.416; 95% CI, 0.279-0.620), and was associated with a reduction in locally invasive procedures vs. ADT alone (4.7% vs. 9.6%); post-hoc analysis of the trial delayed the time to deterioration in quality of life in 2 of the 6 subscales of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, including urinary symptoms (25.8 vs. 14.8 months; HR=0.64; 95% CI, 0.54-0.76) and bowel symptoms (18.4 vs. 11.5 months; HR=0.78; 95% CI, 0.66-0.92) vs. ADT alone | 9/14/21 | Cancer |
| Candel Therapeutics Inc., of Needham, Mass. | CAN-2409 | Replication-deficient adenovirus expressing herpes simplex virus thymidine kinase | Intermediate-high-risk localized prostate cancer | Completed patient enrollment in combination study with valacyclovir | 9/7/21 | Cancer |
| Coherus Biosciences Inc., of Redwood City, Calif., and Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Recurrent or metastatic nasopharyngeal carcinoma | Results of Jupiter-02 pivotal phase III study using toripalimab plus chemotherapy published in Nature Medicine showed superior progression-free survival (PFS) compared to gemcitabine-cisplatin (GP) alone (median PFS of 11.7 vs. 8 months, hazard ratio [HR] = 0.52 [95% confidence interval]: 0.36–0.74; p = 0.0003) with a manageable safety profile; 40% reduction in risk of death was observed with toripalimab compared to placebo (HR = 0.603 [95% CI: 0.364–0.997]); incidence of grade ?3 treatment emergent adverse events (TEAEs) (89% vs. 89.5%); discontinuation of toripalimab/placebo (7.5% vs. 4.9%), and fatal TEAEs (2.7% vs. 2.8%) was similar between both arms; Immune-related AEs (39.7% vs. 18.9%) ; grade ?3 irAEs (7.5% vs. 0.7%) were more frequent with toripalimab | 9/15/21 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China, and Eqrx Inc., of Cambridge, Mass. | Sugemalimab | Anti-PD-L1 monoclonal antibody | Non-small-cell lung cancer | GEMSTONE-302 study with sugemalimab plus chemotherapy showed investigator-assessed progression-free survival was 9 months vs. 4.9 months, HR=0.48; median overall survival (OS) was 22.8 months vs 17.7 months, HR=0.67; OS events have not yet met the predefined interim analysis plan; 12-month PFS rate was 36.4% vs. 14.8%, and 24-month OS rate was 47.1% vs. 38.1%; objective response rate (ORR) was 63.4% vs. 40.3%, duration of response (DoR) was 9.8 months vs. 4.4 months; PFS benefits observed in all PD-L1 expression levels; PD-L1<1% showed median pfs was 7.4 months vs. 4.9 months, hr="0.55;" pd-l1 1-49% 8.8 4.8 pd-l1?50% 12.9 5.1 sugemalimab plus chemotherapy well-tolerated safety profile and no new signals were observed< td> | 9/14/21 | Cancer |
| Hutchmed Ltd., of Hong Kong | Surufatinib | Oral angio-immuno kinase inhibitor | Advanced neuroendocrine tumors | Started Japan registration-enabling bridging study; dosed first patient Sept. 15, 2021 | 9/20/21 | Cancer |
| Hutchmed Ltd., of Hong Kong, Shanghai and Florham Park, N.J. | Surufatinib (Sulanda in China) | Oral angio-immuno kinase inhibitor targeting FGFR1, CSF-1R and all 3 subtypes of VEGF receptors | Advanced neuroendocrine carcinoma | Initiated phase III study in combination with toripalimab (Coherus Biosciences Inc. and Junshi Biosciences Co. Ltd.) | 9/20/21 | Cancer |
| Huyabio International LLC, of San Diego | HBI-8000 | Epigenetic immunomodulator; oral histone deacetylase inhibitor | Unresectable or metastatic melanoma | First patient treated in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) | 9/21/21 | Cancer |
| Immunocore Ltd., of Oxfordshire, U.K. | Tebentafusp (IMCgp100) | Bispecific protein targeting gp100 | Metastatic uveal melanoma | Results from phase III study published in The New England Journal of Medicine demonstrated a statistically significant and clinically meaningful improvement in overall survival HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, vs. investigator’s choice (82% pembrolizumab; 12% ipilimumab; 6% dacarbazine); treatment-related adverse events were manageable and consistent with the proposed mechanism | 9/22/21 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha (IMP-321) | Soluble LAG-3Ig fusion protein | Non-small-cell lung cancer and head and neck squamous cell carcinoma | Completed enrollment and safely dosed in stage 2 of part B of its phase II TACTI-002 (Keynote-798) trial in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 9/1/21 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Erleada (apalutamide) | Oral androgen receptor antagonist | Metastatic and non-metastatic castration-resistant prostate cancer | Post-hoc analysis showed rapid, deep and durable prostate-specific antigen declines; statistically significant improvement in overall survival with a consistent safety profile and maintaining health-related quality of life | 9/11/21 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Margenza (margetuximab-cmkb) | HER2-targeting monoclonal antibody | Metastatic breast cancer | Final overall survival (OS) analysis did not demonstrate statistically significant advantage for Margenza plus chemotherapy over trastuzumab plus chemotherapy (hazard ratio [HR]=0.95; 95% confidence interval [CI]: 0.77-1.17; p=0.62); median survival was 21.6 months for Margenza plus chemotherapy (N=266) compared to 21.9 months for trastuzumab plus chemotherapy (N=270); median OS was prolonged by 2.5 months for Margenza arm vs. trastuzumab arm (23.3 months vs. 20.8 months; HR=0.86; 95% CI: 0.69-1.08; nominal p=0.19) in CD16A 158F allele patients; numerical OS advantage was observed in the subgroup of CD16A patients who were homozygous for F-allele at position 158 in favor of Margenza plus chemotherapy (HR=0.72; 95% CI: 0.52-1.00; nominal p=0.05); median OS was 23.6 months for Margenza plus chemotherapy (102 of 266 patients) compared to 19.2 months for trastuzumab plus chemotherapy (90 of 270 patients); in subgroup of CD16A F/V patients, the median OS was 21.3 months for Margenza plus chemotherapy compared to 22 months for trastuzumab plus chemotherapy (HR=0.96; 95% CI: 0.71-1.30; nominal p=0.78); small subgroup of CD16A V/V patients, OS was greater for trastuzumab plus chemotherapy than Margenza plus chemotherapy (HR=1.77; 95% CI: 1.01-3.12; nominal p=0.04); subgroup, the median survival was 22 months in patients treated with Margenza plus chemotherapy compared to 31.1 months for trastuzumab plus chemotherapy | 9/7/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 humanized monoclonal antibody | Advanced hepatocellular carcinoma | Phase III keynote-394 study met its primary endpoint of overall survival compared with placebo plus best supportive care; met its key secondary endpoints of progression-free survival and objective response rate; no new safety signals were observed | 9/27/21 | Cancer |
| Oncopeptides AB, of Stockholm | Pepaxto (melphalan flufenamide) | DNA alkylating agent; peptide-drug conjugate that targets aminopeptidases | Relapsed refractory multiple myeloma | Melflufen plus dexamethasone in Ocean study met the primary endpoint of superior progression-free survival (PFS); median PFS of 6.8 months compared to 4.9 months for pomalidomide (HR of 0.79, p=0.03); overall survival favored pomalidomide with a HR of 1.10 and overall response rate (ORR) where melflufen had a numerically higher ORR of 33% compared to 27% for pomalidomide; melflufen plus dexamethasone treatment resulted in substantially more grade 3/4 hematologic adverse events compared to pomalidomide | 9/11/21 | Cancer |
| Point Biopharma Global Inc., of Indianapolis | PNT-2002 | Next generation PSMA radioligand | Metastatic castration-resistant prostate cancer | Dosimetry and safety run-in of Splash phase III study (n=27) met all pre-specified criteria allowing for initiation of randomization phase without changes to the study design; enrollment for the randomization phase commenced in Canada | 9/23/21 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Pevonedistat | NEDD8-activating enzyme inhibitor | Higher-risk myelodysplastic syndromes | Panther phase III study did not achieve predefined statistical significance for the primary endpoint of event-free survival; consistent safety profile | 9/2/21 | Cancer |
| TG Therapeutics Inc., of New York | Ukoniq (umbralisib) and ublituximab | PI3K delta inhibitor and anti-CD20 antibody | Chronic lymphocytic leukemia | Data from Unity-CLL trial showed, in comparison to obinutuzumab plus chlorambucil (O+Chl) in patients with treatment-naïve and relapsed/refractory disease, median follow-up of 36.7 months showed ublituximab/umbralisib (U2) significantly prolonged independent review committee-assessed progression-free survival vs. O+Chl (median 31.9 months vs. 17.9 months; p<0.0001); overall response rate was significantly higher with u2 compared to o+chl (83.3% vs. 68.7%; p<0.001)< td> | 9/20/21 | Cancer |
| VBL Therapeutics Ltd., of Tel Aviv, Israel | VB-111 (ofranergene obadenovec) | Gene therapy | Ovarian cancer | Independent data safety monitoring committee conducted fifth preplanned review of ongoing Oval study and provided clearance to proceed, with no changes to protocol | 9/17/21 | Cancer |
| Scpharmaceuticals Inc., of Burlington, Mass. | Furoscix (furosemide injection) | Furosemide solution | Heart failure (HF) with mild to moderate volume overload | In the Freedom-HF trial, excluding the cost of Furoscix, the mean 30-day HF-related costs were $17,753 lower for patients treated with Furoscix versus the comparator group (p<0.0001)< td> | 9/10/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Hypertrophic cardiomyopathy | REDWOOD-HCM study resulted in statistically significant reductions from baseline compared to placebo in the average resting left ventricular (LV) outflow tract pressure gradient (LVOT-G) (p=0.0003, p=0.0004, cohort 1 and cohort 2, respectively) and the average post-Valsalva LVOT-G (p=0.001, p<0.0001, 1 2 10 cohort and 2, respectively) for 10-weeks; patients (78.6% in 92.9% 2) achieved the target goal of treatment, defined as resting gradient <30 mmhg post-valsalva <50 at week compared to placebo (7.7%); both also experienced statistically significant reductions nt-probnp (p="0.003);" improvement heart failure functional class measured by new york association class; least was 31% group, 43%>0.1) and 64% of patients in cohort 2 (p=0.08) | 9/12/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Selective cardiac myosin activator | Heart failure | GALACTIC-HF study resulted in a trend toward reduction in the primary endpoint by 18% (HR=0.82, 95% CI 0.64-1.04), corresponding to a reduction in the primary event rate of 7.7/100 patient-years with a number-needed-to-treat of 13 patients; reduction in hospitalizations (HR=0.80) and HF events (HR=0.82), with no effect on cardiovascular mortality (HR=1.03); no significant differences in adverse events in Black patients between the groups treated with omecamtiv mecarbil and placebo; statistically similar overall benefit from treatment with omecamtiv mecarbil (HR=0.83 vs. HR=0.88), with a numerically greater risk reduction in hospitalization (HR=0.81 vs. HR=0.90) in black patients | 9/12/21 | Cardiovascular |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Xarelto (rivaroxaban) | Oral factor Xa inhibitor | Congenital heart disease | Phase III study published in the Journal of the American Heart Association showed comparable and low prevalence of bleeding events compared to aspirin; 1 major non-fatal bleeding event; slightly lower prevalence of non-major clinically relevant bleeding and trivial bleeding; prevalence and pattern of adverse events were comparable between 2 groups; study not powered for efficacy outcomes (post-hoc log-rank test p=0.095) | 9/27/21 | Cardiovascular |
| Theravance Biopharma Inc., of Dublin | Ampreloxetine (TD-9855) | Norepinephrine reuptake inhibitor | Symptomatic neurogenic orthostatic hypertension | Top-line results showed study did not meet its primary endpoint; majority of treatment-related adverse events were mild or moderate in severity; serious adverse events occurred in 2 patients on placebo and 4 on ampreloxetine and none were considered related to the study drug; no deaths were reported; no signal for supine hypertension | 9/15/21 | Cardiovascular |
| Alvotech hf, of Reykjavik, Iceland | AVT-02 | Biosimilar of Humira (adalimumab) | Moderate to severe chronic plaque psoriasis | Topline results demonstrated bioequivalence of repeated switches between Humira and AVT-02 to administration of Humira without switching; no significant differences observed in clinical efficacy, safety or immunogenicity between the switching cohort and the reference product cohort | 9/9/21 | Dermatologic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | Adenosine A3 receptor agonist | Moderate to severe plaque psoriasis | Completed enrollment of patients (>400); top-line results expected in first quarter 2022 | 9/2/21 | Dermatologic |
| Dermavant Sciences Ltd. of Long Beach, Calif., and Basel, Switzerland | Tapinarof | Aryl hydrocarbon receptor modulating agent | Atopic dermatitis | First patient dosed in ADORING study | 9/9/21 | Dermatologic |
| Revance Therapeutics Inc., of Newark, Calif. | Daxibotulinumtoxin A | Botulinum toxins | Static glabellar lines | Post-hoc analysis of the phase III Sakura study (n=568) published in Dermatologic Surgery showed a substantial reduction in static glabellar lines; improvements in static glabellar lines were achieved within 2 to 4 weeks; remained greater than baseline over 24 weeks of follow-up after treatment cycles 1 and 2; proportion with no static glabellar lines had increased to more than 70% at 4 weeks after their third treatment cycle; complete elimination of those lines was achieved in 54.4% subjects 4 weeks after the first treatment; increased to 66.5% and 69.7% 4 weeks after treatment cycle 2 and 3, respectively | 9/1/21 | Dermatologic |
| Sanofi SA, of Paris | Rilzabrutinib | Oral BTK inhibitor | Pemphigus | Pegasus trial did not meet primary or key secondary endpoints; no new safety signals identified | 9/9/21 | Dermatologic |
| UCB SA, of Brussels | Bimekizumab | Humanized IgG1-kappa monoclonal antibody targeting interleukin 17A | Moderate to severe plaque psoriasis | Interim phase III results achieved sustained levels of skin clearance (PASI 90 and PASI 100) through to 2 years with continuous maintenance dosing; well-tolerated, with no new safety signals; sustained increase in PASI 90 and PASI 100 responder rates up to 2 years; sustained increase in PASI 100 responder rates up to week 100; PASI 90 response rates were 91.2% at both weeks 16 and 104; PASI 100 response rates were 61.6% at week 16 and 72.3% at week 104; 73.6% of bimekizumab-treated patients had achieved PASI 100 | 9/29/21 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR (mutant) expression inhibitor; siRNA agent | Cardiomyopathy in patients with transthyretin-mediated (ATTR) amyloidosis | 9-month data showed HELIOS-A phase III study met the primary and all secondary endpoints, with statistically significant improvements in neuropathy impairment, QoL and gait speed, relative to external placebo; improvement in the exploratory endpoint of the cardiac biomarker NT-proBNP was observed in the vutrisiran arm in both the prespecified cardiac subpopulation [p=0.0016] and the modified intent-to-treat (mITT) population [p=9.2×10-7], relative to external placebo | 9/7/21 | Endocrine/Metabolic |
| Ardelyx Inc., of Fremont, Calif. | Tenapanor | Sodium hydrogen exchanger 3 inhibitor | Hyperphosphatemia in patients with chronic kidney disease on dialysis | Long-term 52-week phase III Phreedom study results published in the Journal of the American Society of Nephrology showed a clinically meaningful decrease in mean serum phosphorus in tenapanor-treated patients from 7.7 mg/dL at baseline to 5.1 mg/dL at the end of the 26-week treatment period in the efficacy analysis set (n=131); clinically meaningful decrease in mean serum phosphorus in the tenapanor-treated patients from 7.4 mg/dL at baseline to 5.9 mg/dL at week 26 in the intent-to treat analysis set (n=248); difference in estimated mean change in serum phosphorus level between tenapanor and placebo from the beginning to the end of the randomized withdrawal period was -1.4 mg/dl (p<0.0001); median relative reductions from baseline of 23% for ifgf23 and 14% cfgf23 at the end randomized 26-week treatment period participants to tenapanor; diarrhea as drug related adverse event reported more than 5% patients resulted in discontinuation 16% patients< td> | 9/3/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | Once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist | Type 2 diabetes | Results from Surpass-3 MRI substudy (n=296) showed greater absolute reduction from baseline in liver fat content (LFC) for pooled 10-mg and 15-mg arms (-8.09% from 15.67% at baseline) compared to insulin degludec (-3.38% from 16.58% at baseline); greater relative reduction from baseline in LFC (29.78%-47.11% across 3 doses) compared to 11.17% for insulin degludec; 30% reduction in LFC from baseline (66.9%-81.4% across the 3 doses) compared to a third of those taking insulin degludec (32.12%); -1.65-liter reduction from baseline of 6.81 liter in VAT (15 mg) and -2.25-liter reduction from baseline of 10.21 liter in ASAT (10 mg) compared to an increase with insulin degludec (+0.38 liter from 6.34 liter baseline and +0.63 liter from 10.04 liter baseline respectively) | 9/30/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | Once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist | Type 2 diabetes | Results showed Surpass-3 CGM data achieved its primary and secondary endpoints; spent between 84.9% and 91.2% of the 24-hour period in the target time in range (71-180 mg/dL), compared to 75% for those taking insulin degludec; spent less time in hypoglycemia across all 3 doses compared to insulin degludec; time spent ?70 mg/dL was between 0.6% and 1% for tirzepatide and 2.4% for insulin degludec; time spent <54 mg dl was between 0.11% and 0.14% for tirzepatide 0.39% insulin degludec; significant reduction in the coefficient of variation (cv) during a 24-hour period (between 0.9%-3.4%) compared to an increase cv those taking degludec< td> | 9/30/21 | Endocrine/Metabolic |
| Hua Medicine Ltd., of Shanghai | Dorzagliatin | Glucokinase stimulator | Type 2 diabetes | Dream phase III study (n=69) showed the subjects had a 52-week glucose remission rate of 65.2% (95% CI, 53.4%, 77%); reached normal blood glucose level; maintained their blood glucose level and beta cell function after discontinuation of medication | 9/26/21 | Endocrine/Metabolic |
| Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Monoclonal antibody against FGF23 | X-linked hypophosphatemia | Long-term treatment showed statistically significant improvements in patient-reported outcomes and measures of mobility up to 96 weeks compared to baseline; statistically significant improvements in ambulatory function measured by the 6-minute walk test | 9/24/21 | Endocrine/Metabolic |
| Infant Bacterial Therapeutics AB, of Stockholm | IBP-9414 | Oral drug containing Lactobacillus reuteri | Necrotizing enterocolitis | 600 premature infants recruited | 9/30/21 | Gastrointestinal |
| Inventiva SA, of Daix, France | Lanifibranor | PPAR agonist | Nonalcoholic steatohepatitis | Initiated Nativ3 trial to test long-term efficacy and safety in adults with biopsy-proven non-cirrhotic NASH and F2/F3 stage of liver fibrosis | 9/8/21 | Gastrointestinal |
| Bayer AG, of Whippany, N.J. | Kerendia (finerenone) | Nonsteroidal mineralocorticoid receptor antagonist | Chronic kidney disease in patients with nondiabetic CKD | Started Find-CKD study to test drug in addition to guideline-directed therapy; primary outcome measure is mean rate of change in kidney function over time from baseline to month 32 | 9/20/21 | Genitourinary/Sexual Function |
| Urovant Sciences Inc., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Gemtesa (vibegron) | Beta-3 adrenergic agonist | Overactive bladder | Post-hoc subgroup analyses of phase III Empowur trial showed once-daily treatment (75 mg) with Gemtesa was associated with significantly reduced daily urgency episodes and micturitions in patients vs. placebo; Gemtesa was not associated with statistically significant or clinically meaningful effects on blood pressure or heart rate | 9/13/21 | Genitourinary/Sexual Function |
| Harbour Biomed Ltd., of Suzhou, China | Batoclimab (HBM-9161) | Anti-FcRn monoclonal antibody | Generalized myasthenia gravis | First patient dosed | 9/27/21 | Immune |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Avigan/Reeqonus (favipiravir) | Oral antiviral | COVID-19 | Completed enrollment in viral shedding substudy portion of Preseco study designed to identify COVID-19 variants in patients and evaluate direct antiviral effect | 9/17/21 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Avigan/Reeqonus (favipiravir) | Oral antiviral | COVID-19 | Completed enrollment; enrolled 1231 patients; top-line results expected in November 2021 | 9/23/21 | Infection |
| Corvus Pharmaceuticals Inc., of Burlingame, Calif. | Mupadolimab (CPI-006) | Humanized monoclonal antibody directed against CD73 | COVID-19 | Results of phase III study published in Medrxiv showed improvement in primary and key secondary endpoints in patients treated with single doses of mupadolimab at 2mg/kg and 1mg/kg compared to placebo; no drug related adverse event; 93.3% of patients were alive and free from respiratory failure at 2 mg/kg, compared to 85.7% in 1 mg/kg cohort and 81.1% in the placebo; 2 mg/kg cohort demonstrated higher cross-reactivity with the beta, gamma and delta variants compared to 1mg/kg and placebo | 9/22/21 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | CCR5 antagonist | COVID-19 | First of 612 patients treated; interim analysis to be conducted 28 days following enrollment of 245 patients | 9/10/21 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon Lambda | Type III interferon | COVID-19 | Data safety monitoring board recommended investigators continue enrollment of Peginterferon Lambda arm in Together platform study, following interim analysis based on sample size of 453 newly diagnosed, high-risk, non-hospitalized patients; the Peginterferon Lambda arm targets enrollment of up to 800 patients | 9/20/21 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Nucleotide analogue targeting viral RNA polymerase | COVID-19 | Results of phase III study (n=562) in non-hospitalized patients at high risk for disease progression showed statistically significant 87% reduction in risk for the composite primary endpoint COVID-19 related hospitalization or all-cause death by day 28 compared with placebo p=0.008; 81% reduction in risk for the composite secondary endpoint of medical visits due to COVID-19 or all-cause death by day 28 for participants treated with Veklury compared with placebo p=0.002;n o deaths were observed in either arm by day 28 | 9/22/21 | Infection |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | RSV vaccine | RSV vaccine | Respiratory syncytial virus infection | Initiated phase III study | 9/29/21 | Infection |
| Janssen Vaccines & Prevention BV, a unit of New Brunswick, N.J.-based Johnson & Johnson | Mvabea (MVA-BN filo) and Zabdeno (Ad26.ZEBOV) | Modified vaccinia virus ankara- and adenovirus serotype 26-based vaccines | Ebola virus infection | Results from phase III EBOVAC-Salone study published in The Lancet Infectious Diseases using vaccine regimens of Ad26.ZEBOV, administered intramuscularly as the first dose vaccination followed by MVA-BN-Filo as the second dose vaccination after 56 days, generated robust humoral (antibody) immune responses in adults and children (ages 1-17) with the immune responses persisting in adults for at least 2 years; booster vaccination with Ad26.ZEBOV, administered to adults 2 years after the initial vaccination, induced a strong immune response within 7 days; well-tolerated and no safety signals of concern | 9/13/21 | Infection |
| Johnson & Johnson, of New Brunswick, N.J. | Single-shot COVID-19 vaccine | Single-shot COVID-19 vaccine | COVID-19 prophylaxis | Booster dose provided 94% protection 2 months after the first shot; antibody levels raised to 4 to 6 times higher than observed after the single shot; 6 months after the single shot, antibody levels increased 9-fold 1 week after the booster and continued to climb to 12-fold higher 4 weeks after the booster | 9/21/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics LP, of Miami | Molnupiravir | Nucleoside analogue; RNA-directed RNA polymerase inhibitor | COVID-19 | Initiated phase III study | 9/1/21 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | COVID-19 vaccine | COVID-19 prophylaxis | Third dose administered for people living with an organ (liver or kidney) transplant; trial will dose patients with an extra shot and monitor their immune antibody response 28 days from the third shot | 9/21/21 | Infection |
| Novan Inc., of Durham, N.C. | SB-206 (berdazimer sodium) | Topical nitric oxide-releasing antiviral product | Molluscum contagiosum | Data from phase III B-Simple4 study showed favorable and consistent safety profile; no treatment-related serious adverse events were reported at or before week-24; lower occurrence of scarring at week-24 visit when compared to vehicle (4% in vehicle vs. 2.7% in SB-206) | 9/23/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | Nanoflu | Recombinant quadrivalent seasonal influenza vaccine candidate with Matrix-M adjuvant | Influenza virus infection | Results from a pivotal phase III study published in The Lancet Infectious Diseases showed vaccine well-tolerated and produced significantly enhanced humoral and cellular immune responses vs. comparator vaccine; potent induction of polyfunctional antigen-specific CD4+ T cells against A/H3N2 and B/Victoria strains, with a 126%-189% increase in various post-vaccination cell-mediated immunity markers as compared to Fluzone Quadrivalent; comparable safety profile | 9/23/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | Synthetic form of the vasoactive intestinal polypeptide | COVID-19 | Independent data safety monitoring board found no new safety concerns; reviewed 231 patients and recommended continued enrollment; cleared to continue enrollment of more 600 patients | 9/29/21 | Infection |
| Pfizer Inc., of New York | PF-07321332 | SARS-CoV2-3CL protease inhibitor | COVID-19 | First participant dosed in a pivotal phase II/III study | 9/1/21 | Infection |
| Pfizer Inc., of New York | RSVpreF | Vaccine comprising recombinant prefusion glycoprotein (F) | Respiratory syncytial virus | Initiated phase III study to evaluate efficacy, immunogenicity and safety of a single-dose vaccine in adults ages 60 or older | 9/2/21 | Infection |
| Pfizer Inc., of New York | Prevnar 20 | 20-valent pneumococcal vaccine comprising polysaccharides of Streptococcus pneumoniae serotypes conjugated to diphtheria CRM19 | Influenza virus infection | Top-line results from phase III study showed vaccine noninferior in combination with seasonal influenza vaccine (Fluad Quadrivalent ) when administered 1 month apart | 9/29/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGEN-COV (casirivimab and imdevimab) | 2 fully human antibodies targeting distinct regions of the receptor binding domain region of the spike protein of human COVID-19 coronavirus | COVID-19 | Phase III results published in The New England Journal of Medicine showed study met its primary and all secondary endpoints; significantly reduced the risk of hospitalization or death by 70% in high-risk non-hospitalized patients infected with COVID-19; consistent safety profile | 9/29/21 | Infection |
| Rigel Pharmaceuticals Inc., of South San Francisco | Fostamatinib | SYK inhibitor | COVID-19 | Results of phase II study (n=59), published in Clinical Infectious Diseases, study met its primary endpoint; drug did not increase the incidence of serious adverse events (SAE) compared with placebo; overall incidence of SAEs by day 29 was 5% less in the fostamatinib group (10.5%) compared with the placebo group (22%) (p=0.2); well-tolerated and associated with clinically meaningful improvement in clinical outcomes in hospitalized COVID-19 patients who required supplemental oxygen in combination with standard-of-care therapy; favorable prespecified secondary endpoints including mortality, time to sustained recovery, change in ordinal scale assessment, number of days on oxygen and number of days in the ICU; consistently greater reduction in NETosis and other inflammatory biomarkers (CRP, ferritin, D-dimer, fibrinogen) in the fostamatinib group compared to the placebo group | 9/1/21 | Infection |
| Swedish Orphan Biovitrum AB, of Stockholm | Kineret (anakinra) | Recombinant interleukin-1 receptor antagonist | COVID-19 pneumonia | Phase III results published in Nature Medicine demonstrated that patients treated with anakinra were significantly more improved than those treated with placebo (p<0.0001) ; reduces risk of death, icu admission and increases likelihood full recovery in hospitalized covid-19 patients with poor prognosis due to severe respiratory failure< td> | 9/7/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Completed recruitment of the initial cohort of elderly participants in phase III study; top-line data from this cohort will read out in early 2022 | 9/14/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Initiated recruitment of adolescents aged 12 to 17 years in phase III study in U.K.; top-line data expected in fourth quarter of 2021; initiated booster dose to volunteers in phase I/II study | 9/23/21 | Infection |
| Vicore Pharma Holding AB, of Gothenburg, Sweden | C-21 | AT2 receptor agonist | COVID-19 | Dosed first patients in Attract-3 trial; study to enroll hospitalized patients, with top-line data expected during first half of 2022 | 9/17/21 | Infection |
| Seres Therapeutics Inc., of Cambridge, Mass., and Nestlé Health Science, of Lausanne, Switzerland | SER-109 | Oral microbiome therapeutics | Recurrent Clostridioides difficile infection | Achieved enrollment of 300 subjects | 9/15/21 | Infection |
| Ampio Pharmaceuticals Inc., of Englewood, Colo. | Ampion | Biologic drug containing a blood-derived cyclized peptide | Osteoarthritis of the knee | Top-line results of AP-013 study demonstrated a statistically significant reduction in pain (p=0.0260) and improvement in function (p=0.0073) with p-value of 0.05 vs. saline at 12 weeks in severe patients; strong safety profile with no treatment-related serious events | 9/15/21 | Inflammatory |
| Amo Pharma Ltd., of London | AMO-02 (tideglusib) | GSK-3beta inhibitor | Congenital myotonic dystrophy type 1 | First patient enrolled in 52-week open-label study to evaluate the long-term safety and efficacy | 9/30/21 | Musculoskeletal |
| Axsome Therapeutics Inc., of New York | AXS-12 | Selective norepinephrine reuptake inhibitor | Narcolepsy | Enrolled first patient in Symphony study; top-line data expected in first half of 2023 | 9/16/21 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass. | Spinraza (nusinersen) | Antisense oligonucleotide targeting SMN2 expression | Spinal muscular atrophy | Initiated phase IIIb study with a higher dose | 9/15/21 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Verdiperstat | Myeloperoxidase inhibitor | Multiple system atrophy | Results of phase III study did not statistically differentiate from placebo on the prespecified primary efficacy measure or on key secondary efficacy measures; consistent safety data | 9/27/21 | Neurology/Psychiatric |
| Braxia Scientific Corp., of Toronto | Ketamine | NMDA receptor antagonist | Depression | Monotherapy group achieved response and remission rates of 39.1% and 17.4%, respectively; ketamine treatment adjunct to antidepressants, saw corresponding rates of 21.9% and 6.7% | 9/30/21 | Neurology/Psychiatric |
| Intra-Cellular Therapies Inc., of New York | Lumateperone | 5-HT2a receptor antagonist | Bipolar disorder | Results of phase III monotherapy study published in The American Journal of Psychiatry showed treatment with lumateperone resulted in a substantial reduction in depressive symptoms; lumateperone 42 mg was associated with a statically significant greater reduction in MADRS score from baseline to day 43 (drug placebo difference -4.6 (p<0.0001; effect size="0.56);" significantly improved madrs total score compared with placebo as early week 1; met the key secondary endpoint of statistically significant improvement on cgi-bp-s (p<0.0001; and cgi component that specifically assesses depression (cgi-bp-s score; p<0.001; favorable safety tolerability profile < td> | 9/27/21 | Neurology/Psychiatric |
| Ixaka Ltd., of London | REX-001 | Autologous multi-cell therapy | Chronic limb-threatening ischemia | Interim results showed REX-001 resulted in complete ulcer healing in over 70% of patients; 30% and 50% of patients enrolled have reached the 12-month follow-up visit; independent data monitoring committee recommended continuation of the trial unchanged | 9/8/21 | Neurology/Psychiatric |
| Newron Pharmaceuticals SpA, of Milan | Evenamide | Targets voltage-gated sodium channels | Schizophrenia | Initiated phase III study | 9/6/21 | Neurology/Psychiatric |
| Pharma Two B Ltd., of Rehovot, Israel | P2B-001 | Fixed-dose combination of rasagiline mesilate and pramipexole hydrochloride | Early Parkinson's disease | Last patient completed study; top-line results expected in the fourth quarter of 2021 | 9/13/21 | Neurology/Psychiatric |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | Small-molecule selective BET inhibitor | Cognitive disorder | Study results published in the Journal of Alzheimer's Disease showed significantly improved cognition and reduced cognitive decline among high-risk patients with cardiovascular disease and diabetes mellitus; in patients with baseline MoCA score ? 21, apabetalone treatment resulted in a significant 2.1-unit increase in MoCA score (p=0.02), a clinically meaningful improvement when compared with the placebo group; alkaline phosphatase reduced in the apabetalone-treated group relative to placebo (p=0.03) | 9/13/21 | Neurology/Psychiatric |
| Servier Pharmaceuticals, of Boston, part of Servier Group, and Neurochlore SAS, of France | Bumetanide | NKCC2 and NKCC1 inhibitors | Autism spectrum disorders | Study showed no sign of effectiveness in both primary and secondary endpoints in children and adolescents; companies by mutual agreement decided an early termination of the 2 clinical studies in progress; studies not revealed any unexpected safety issue bumetanide |
9/7/21 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Marlborough, Mass. | Kynmobi (apomorphine hydrochloride) | Formulation of the dopamine agonist apomorphine | Treatment of OFF episodes associated with Parkinson’s disease | In CTH-301 safety study, 88% of patients successfully titrated to an effective and tolerable dose without concomitant antiemetics; in the pivotal study, 57% of cases of nausea were mild and lasted an average of 46 minutes | 9/10/21 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | Anti-CGRP subcutaneous injection | Episodic migraine | Post-hoc analysis in combination with atogepant (Abbvie Inc.)and rimegepant (Biohaven Pharmaceutical Holding Co. Ltd.) showed increases from baseline in consecutive migraine-free days (MFD) during 12 weeks were significantly higher for fremanezumab (least-squares mean change from baseline during 12 weeks: quarterly, 8.3 [0.82]; monthly, 9.6 [0.81]) vs. placebo (4.0 [0.81]; both p<0.0001); both quarterly and monthly dosing of fremanezumab resulted in favorable migraine frequency category reversions to a greater extent than placebo< td> | 9/8/21 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Vomeropherin compound acting on chemosensory receptors; pherine nasal spray | Social anxiety disorder | Initiated phase III Palisade-2 study | 9/13/21 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (fenfluramine) | 5-HT releasing agents; 5-HT1D, 2A, and 2C receptor agonists; positive modulator of sigma1R | Seizures associated with Lennox-Gastaut syndrome | Interim data (n=247) of an ongoing 12-month phase III open-label extension study led to median reduction in drop seizure frequency of 39.4% at 3 months (n=227; p<0.0001) 10 12 and 51.8% for those patients assessed at months to (n="170;" p<0.0001); well-tolerated with no observed valvular heart disease or pulmonary hypertension in long-term study; treatment-emergent adverse events were decreased< td> | 9/30/21 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol formulated in a transdermal gel | Fragile X syndrome | Initiated a pivotal phase III Reconnect study to evaluate the efficacy and safety; expected to enroll 200 children and adolescents, ages 3-17; top-line results expected in the second half of 2023 | 9/13/21 | Neurology/Psychiatric |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Intravitreal pegcetacoplan | Targeted C3 therapy | Geographic atrophy (GA) | Oaks study met primary endpoint for both monthly and every-other-month treatment with pegcetacoplan, demonstrating a significant reduction in GA lesion growth of 22% (p=0.0003) and 16% (p=0.0052), respectively, compared to pooled sham at 12 months; Derby study didn’t meet primary endpoint with a reduction of 12% (p=0.0528) and 11% (p=0.0750), respectively; plans to submit NDA in the first half of 2022 | 9/9/21 | Ocular |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Intravitreal pegcetacoplan | Targeted C3 therapy | Geographic atrophy | Oaks study (n=637) met the primary endpoint; significantly reduced GA lesion growth compared to pooled sham at 12 months with monthly treatment (p=0.0003) and every-other-month treatment (p=0.0052); Derby study (n=621) missed the primary endpoint; reduction in GA lesion growth with monthly (p=0.0528) and every-other-month treatment (p=0.0750) compared to pooled sham at 12 months; favorable safety profile in both studies; pooled rate of new-onset exudations was 6.0%, 4.1% and 2.4% in the pegcetacoplan monthly, every-other-month and sham groups, respectively | 9/30/21 | Ocular |
| Bausch + Lomb, unit of Bausch Health Cos. Inc., of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany | NOV-03 (perfluorohexyloctane) | Ophthalmic liquid formulation | Dry eye disease | Top-line data showed phase III Mojave trial met both primary and secondary endpoints; statistical superiority over placebo: change from baseline in tCFS at day 15 [p = 0.001]; change from baseline in dryness score at day 15 [p = 0.001]; change from baseline in VAS burning/stinging at day 57 [p = 0.001]; and change from baseline in central Corneal Fluorescein Staining (cCFS) at day 57 [p < 0.001]; well-tolerated and no treatment emergent adverse events | 9/30/21 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 (ND4) gene | Leber hereditary optic atrophy | Study analyzing visual parameters of ND4-LHON subjects before Lumevoq treatment in phase III trials found that in the first 6 months post-vision loss each month after onset was associated with a mean visual acuity loss of +0.24 LogMAR (-12 ETDRS letters equivalent); time from onset was not associated with substantial worsening of visual acuity for patients between 6 and 12 months post-vision loss; findings reinforce the medical consensus on the natural history of LHON over the first year post-vision loss, in which an acute phase of visual decline in the first months after onset is followed by a relative stabilization of visual acuity in the dynamic phase of the disease | 9/9/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010, Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Norse three (n=197) open-label study met its goal; showed positive safety profile in patients receiving 3 monthly intravitreal doses with no reports of ocular inflammation | 9/13/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010, Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Neovascular age-related macular degeneration | 12-month safety data from pivotal phase III Norse 2 trial showed strong safety profile consistent with previous trials; clinically relevant and highly statistically significant results with robust safety profile; well-tolerated and no unanticipated safety signals | 9/28/21 | Ocular |
| Tarsier Pharma Ltd., of Tel Aviv, Israel | TRS-01 | Fast-acting immunomodulator eye drops | Active non-infectious anterior uveitis and uveitic glaucoma | Initiated phase III study and randomized first patient; planned to evaluate 162 patients | 9/22/21 | Ocular |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide (Imcivree) | Melanocortin receptor agonist | Obesity associated with Bardet-Biedl Syndrome | Subgroup analysis of data from the 52-week phase III study showed setmelanotide achieved statistically significant weight loss and hunger reduction compared to minimal effect observed with placebo during a 14-week in double-blind treatment period; in patients 12 years old or older treated showed greater weight loss of 3.8 kg, or 3% of their baseline body weight (p<0.05); 12 greater reduction in most hunger score of 20.4% from baseline (p<0.05); patients younger than years (n="16)" achieved an average bmi 1.5 kg m2, or 3.8%, treated with placebo (p<0.05)< td> | 9/22/21 | Other/Miscellaneous |
| Soleno Therapeutics Inc., of Redwood City, Calif. | DCCR | K(ATP) channel activator; extended release of diazoxide choline once daily | Prader-Willi syndrome | Top-line results of C602 study showed improvement in hyperphagia after receiving DCCR for 13, 26 and 39 weeks (all p<0.0001); 26 39 52 decrease in the hq-ct total score, of -9.9 (0.77) was highly significant (p<0.0001) after receiving dccr for weeks; statistically improvements all behavioral domains (all p<0.0001) 13, and weeks p<0.0004); no change body fat mass, lean mass ratio to (p="0.0005);" leptin (p<0.0001), adiponectin fasting insulin improvement sensitivity measured using homeostatic model assessment resistance also observed; consistent safety profile< td> | 9/8/21 | Other/Miscellaneous |
| PTC Therapeutics Inc, of South Plainfield, N.J. | PTC-AADC | Gene therapy | Aromatic L-amino acid decarboxylase | 5-year results showed sustained motor function improvements; cognitive and language skills reported to improve significantly from baseline; rate of respiratory infection declined from an average of 2.4 episodes per year at 12 months to 0.6 episodes per year at 2 years and 0.3 episodes per year at 5 years | 9/29/21 | Other/Miscellanous |
| Astrazeneca plc, of Cambridge, U.K., and Amgen Inc., of Thousand Oaks, Calif. | Tezepelumab | Human monoclonal antibody against human thymic stromal lymphopoietin | Asthma | Tezepelumab achieved an 86% reduction in the annualized asthma exacerbation rate in NP+ patients (95% CI: 70, 93) and 52% (95% CI: 42, 61) in NP- patients over 52 weeks, compared to placebo when added to standard of care; improved lung function at week 52 in both groups of patients with an increase in pre-bronchodilator forced expiratory volume in 1 second (FEV1) of 0.20 L (95% CI: 0.02, 0.37) and 0.13 L (95% CI: 0.08, 0.18) vs. placebo in NP+ and NP- patients, respectively; clinically relevant improvement in nasal polyp symptoms at week 52, as measured by the Sinonasal Outcome Test (SNOT-22), reducing the SNOT-22 score in NP+ patients by 9.6 points (95% CI: 0.9, 18.2) vs. placebo; adjusted mean score reductions from baseline for tezepelumab and placebo were 20.1 points (SE: 3.07) and 10.55 points (SE: 2.94). Baseline mean (sd) SNOT-22 score was 49.4 (21.5) and 47.8 (19.0) for tezepelumab and placebo | 9/5/21 | Respiratory |
| Avillion LLP, of London, and Astrazeneca plc, of Cambridge, U.K. | PT-027(albuterol/budesonide) | Glucocorticoid receptor agonist; beta 2 adrenoceptor agonist | Asthma | Results from Mandala and Denali trials showed both doses of 180/160 mcg and 180/80 met all primary endpoints, demonstrating statistically significant benefits vs. individual components; Mandala met primary endpoint of statistically significant and clinically meaningful reductions in risk of severe exacerbations vs. albuterol, when used as a rescue medicine in response to symptoms; Denali met dual primary endpoints, showing a statistically significant improvement in lung function measured by forced expiratory volume in 1 second vs. individual components and compared to placebo | 9/9/21 | Respiratory |
| Zambon SpA, of Milan | CMS I-neb | Adaptive aerosol delivery system of colistimethate sodium | Non-cystic fibrosis bronchiectasis | Phase III PROMIS-I study met its primary endpoint; annual rate of exacerbations was significantly lower in patients receiving CMS I-neb vs. placebo (0.58 per patient per year vs. 0.95, rate ratio (RR) 0.61 95% CI 0.46-0.82, p=0.00101); well-tolerated and improved quality of life; treatment effect was even larger in adherent subjects (43.5% reduction in exacerbations, p= 0.00080); frequency of severe exacerbations also reduced (RR 0.41 95% CI 0.23-0.74, p=0.00300); after 28 days treatment, P. aeruginosa density was significantly reduced in the treatment arm (p < 0.00001) | 9/8/21 | Respiratory |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Movantik (naloxegol) | Opioid antagonist | Opioid-induced constipation | Data showed rapid and sustained improvement of both spontaneous and complete spontaneous bowel movements vs. placebo, as evaluated across high and low opioid dosages | 9/7/21 | Toxicity and Intoxication |
| Alkermes plc, of Dublin | Nemvaleukin alfa | Engineered fusion protein comprising modified interleukin-2 (IL-2) and IL-2 alpha receptor chain | Platinum-resistant ovarian cancer | Initiated Artistry-7 phase III study in combination with Keytruda (pembrolizumab, Merck & Co Inc.) | 10/26/21 | Cancer |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-046 | PD-L1/CTLA4 bispecific antibody | Advanced unresectable or metastatic squamous non-small-cell-lung cancer | Enrolled 482 patients in 61 clinical research centers nationwide; interim data expected in first quarter of 2022 | 10/18/21 | Cancer |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-046 | PD-L1/CTLA4 bispecific antibody | PD-(L)1-refractory advanced non-small-cell lung cancer | First patient dosed | 10/29/21 | Cancer |
| Apexigen Inc., of San Carlos, Calif. | BD-0801 | Monoclonal antibody targeting vascular endothelial growth factor | Recurrent, platinum-resistant ovarian cancer | First patient dosed in study comparing the efficacy and safety of BD-0801 plus chemotherapy to chemotherapy alone; primary endpoint is PFS | 10/6/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) and tremelimumab | PD-L1-targeting antibody and CTLA4-targeting antibody | Unresectable hepatocellular carcinoma | Himalaya trial met primary endpoint; demonstrated a statistically significant and clinically meaningful overall survival benefit vs. sorafenib | 10/15/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | PD-L1-targeting antibody | Advanced biliary tract cancer | Interim results from Topaz-1 phase III trial in combination with standard-of-care chemotherapy met primary and secondary endpoints; statistically significant and clinically meaningful overall survival benefit vs. chemotherapy; improvement in progression-free survival and overall response rate | 10/25/21 | Cancer |
| Avelas Biosciences Inc., of San Diego | Pegloprastide (AVB-620) | Fluorescent imaging agent | Agent for visualizing breast cancer margins | Planning to initiate phase III in first half of 2022 | 10/18/21 | Cancer |
| Bayer AG, of Leverkusen, Germany | Vitrakvi (larotrectinib) | Oral tropomyosin kinase inhibitor | Solid tumors harboring an NTRK gene fusion | Updated thyroid subanalysis of adult and pediatric TRK fusion cancer patients showed overall response rate of 71% in all types of thyroid cancers, 86% in differentiated thyroid cancer and 29% in anaplastic thyroid cancer; median time to response was 1.9 months; 24-month duration of response was 81%; 2 patients had grade ?3 treatment-related adverse events; none discontinued treatment | 10/4/21 | Cancer |
| Candel Therapeutics Inc., of Needham, Mass. | CAN-2409 | Oncolytic viral immunotherapy | Localized prostate cancer | Completed enrollment; 100% of patients reported overall feeling positive about?their involvement in the study in tolerability assessment of intraprostatic injections | 10/28/21 | Cancer |
| Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK4/6 inhibitor; non-chemotherapy oral tablet | Breast cancer | Results published in Annals of Oncology included median follow-up of 27 months in combination with endocrine therapy; absolute improvement rates in invasive disease-free survival (IDFS) and distant relapse-free survival were 5.4% and 4.2%, respectively; consistent benefit in reducing risk of recurrence regardless of having a low (<20%) or high (?20%) ki-67 score among patients; consistent safety profile< td> | 10/14/21 | Cancer |
| Erytech Pharma SA, of Lyon, France | Eryaspase | L-asparaginase encapsulated inside donor-derived red blood cells | Advanced pancreatic cancer | Top-line results from phase III Trybeca-1 study did not meet primary endpoint of overall survival (OS); demonstrated an improvement in OS compared to chemotherapy alone with a hazard ratio of 0.92 (95% (CI), 0.76-1.11) in the intent-to-treat population; difference was not statistically significant (p=0.375); median OS for patients treated with eryaspase plus chemotherapy was 7.5 months (95% CI, 6.5-8.3), compared to 6.7 months (95% CI, 5.4-7.5) for chemotherapy alone; benefit in progression-free survival, disease control rate and objective response rate; consistent safety profile | 10/25/21 | Cancer |
| Geron Corp., of Foster City, Calif. | Imetelstat | Telomerase inhibitor | Lower-risk myelodysplastic syndrome | Completed patient enrollment in Imerge study; top-line results expected in January 2023 | 10/18/21 | Cancer |
| Hutchmed Ltd., of Hong Kong | HMPL-523 | Syk inhibitor | Primary immune thrombocytopenia | First patient dosed in trial | 10/27/21 | Cancer |
| I-Mab Biopharma Co. Ltd., of Shanghai | TJ-202 (felzartamab) | Human IgG1 antibody targeting CD38 | Multiple myeloma | Completed patient enrollment in combination with lenalidomide | 10/13/21 | Cancer |
| Immunitybio Inc., of Culver City, Calif. | Anktiva (N-803) | Protein complex consisting of a superagonist human interleukin 15 variant N72D and a dimeric IL-15 receptor alpha sushi domain-IgG1 Fc fusion protein | Non-small-cell-lung cancer | Initiated lung-Map trial in combination with pembrolizumab (Keytruda, Merck & Co. Inc.); enrolled 478 patient | 10/4/21 | Cancer |
| Innovent Biologics Inc., of San Francisco | Tyvyt (sintilimab) | PD-1-directed human monoclonal antibody | Nonsquamous non-small-cell-lung cancer | Interim analysis of Orient-31 study met its prespecified primary endpoint of progression-free survival (PFS); in combination with Byvasda (bevacizumab biosimilar) and chemotherapy, it showed statistically significant and clinically meaningful improvement in PFS compared with chemotherapy alone; prespecified PFS futility analysis did not cross futility stopping boundary; consistent safety profile with no additional safety signals | 10/17/21 | Cancer |
| Menarini Group, of Florence, Italy, and Radius Health Inc., of Waltham, Mass. | Elacestrant | Selective estrogen receptor degrader | ER+/HER2- advanced or metastatic breast cancer | Top-line Emerald study met both primary endpoints; statistically significant progression-free survival in the overall population and ESR1 mutation subgroup; consistent safety profile | 10/20/21 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda | Monoclonal antibody targeting PD-1 | Advanced melanoma | In the Keynote-006 study, overall survival was 32.7 months for Keytruda and 15.9 months for ipilimumab (HR=0.70); 7-year OS rates were 37.8% for Keytruda and 25.3% for ipilimumab; at the second interim analysis of the Keynote-716 study, 14.8% of patients who received Keytruda had recurrence or died compared to 23.5% of patients on placebo; recurrence rate was 12.3% for Keytruda and 6.4% for placebo | 10/31/21 | Cancer |
| Novartis Pharmaceuticals Canada Inc., unit of Novartis AG, of Basel, Switzerland | Ilaris (canakinumab, ACZ-885) | Human monoclonal antibody that binds with high affinity and selectivity to human interleukin-1beta | Lung cancer | Top-line results of Canopy-1 trial did not meet its primary endpoints of overall survival (OS) and progression-free survival (PFS); did not demonstrate the statistically significant primary endpoints in combination with pembrolizumab (Keytruda, Merck & Co. Inc.) plus platinum-based doublet chemotherapy compared to placebo; clinically meaningful improvements in both PFS and OS were observed among prespecified subgroups of patients with inflammatory biomarkers; no unexpected safety signals | 10/25/21 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J | CPI-613 (devimistat) | Small molecule that targets the mitochondrial tricarboxylic acid | Metastatic adenocarcinoma of the pancreas | Study did not meet its primary endpoint of overall survival in combination with modified Folfirinox (mFFX); did not significantly improve overall survival (HR=0.95, p=0.66); median overall survival in devimistat and mFFX arm was 11.1 months, compared to 11.7 months in the mFFX arm | 10/28/21 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J | CPI-613 (devimistat) | Small molecule that targets the mitochondrial tricarboxylic acid | Acute myeloid leukemia | Independent data monitoring committee recommended that the trial be stopped due to lack of efficacy from prespecified interim analysis | 10/28/21 | Cancer |
| Servier SA, of Paris | Lonsurf (trifluridine/tipiracil) | Thymidine-based nucleoside analogue and thymidine phosphorylase inhibitor | Unresectable metastatic colorectal cancer | Solstice trial did not meet primary endpoint of progression-free survival; trial will continue as planned given that no deleterious effects and no new safety issues | 10/22/21 | Cancer |
| Veru Inc., of Miami | Enobosarm | Selective androgen receptor modulator | Metastatic breast cancer | Enrolled first patient | 10/13/21 | Cancer |
| Biocardia Inc., of San Carlos, Calif. | Cardiamp | Autologous bone marrow-derived stem cell therapy | Chronic myocardial ischemia with refractory angina | First patient treated | 10/21/21 | Cardiovascular |
| Brickell Biotech Inc., of Boulder, Colo. | Sofpironium bromide gel | Anticholinergic | Primary axillary hyperhidrosis | In the Cardigan I and Cardigan II studies, the proportion of patients who achieved at least a 2-point improvement in the HDSM-Ax1 score from baseline to end of therapy was 49.3% and 63.9% for the drug and 29.4% and 47% for placebo, respectively for the 2 studies (p<0.001 and p="0.003," respectively); change in gsp3 from baseline to end of therapy was -129.5mg -145.9 mg for the drug -99.3mg -131.7mg placebo, respectively (p="0.002" respectively)< td> | 10/7/21 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | JAK Inhibitor | Alopecia areata | Completed patient enrollment; top-line data expected in second quarter of 2022 | 10/25/21 | Dermatologic |
| Helixmith Co. Ltd., of Seoul, South Korea | Engensis (VM-202) | DNA vector expressing 2 isoforms of hepatocyte growth factor | Diabetic foot ulcers | Interim data showed positive trend toward wound closure from month 3 to month 7; prominent ulcer closure effect higher at months 3, 4 and 5 (p = 0.0391, 0.0391 and 0.0361, respectively); improved hemodynamic features; increase in ABI (p=0.0776); acceptable safety profile | 10/22/21 | Dermatologic |
| Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib) | JAK inhibitor | Nonsegmental vitiligo | 24-week results from pivotal phase III True-V studies showed drug achieved ?75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75); ?50% improvement from baseline in F-VASI (F-VASI50), and more than 15% of patients applying ruxolitinib cream achieved ?90% improvement from baseline in F-VASI (F-VASI90); consistent safety profile; no serious treatment-related adverse events | 10/2/21 | Dermatologic |
| Krystal Biotech Inc., of Pittsburgh | Beremagene geperpavec | Non-integrating viral vector expressing the collagen VII gene; topical therapy | Dystrophic epidermolysis bullosa | Last participant completed 26-week dosing period and 30-day safety follow up visit in the GEM-3 study; top-line data expected in fourth quarter of 2021 | 10/26/21 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Adtralza (tralokinumab) | Monoclonal antibody targeting interleukin-13 | Moderate to severe atopic dermatitis | 16-week Ecztra trial met primary and secondary endpoints; well-tolerated with an overall frequency and severity of adverse events comparable with placebo; 21.4% (p<0.001) 150 of patients on tralokinumab 150-mg dose and 17.5% (p="0.002)" 300-mg achieved clear or almost-clear skin compared to 4.3% with placebo as measured by iga; 28.6% (p<0.001) mg- 27.8% for group 75% greater disease improvement from baseline 6.4% easi; 4-point in adolescent weekly average worst daily pruritus nrs score, health-related quality life related dermatological conditions< td> | 10/22/21 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Human monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13 proteins | Uncontrolled prurigo nodularis | Top-line results (n=78 in Dupixent arm) from trial met primary and all key secondary endpoints; 37% of Dupixent patients experienced a clinically meaningful reduction in itch from baseline compared to 22% of placebo patients (p=0.0216) at week 12; 58% of Dupixent patients compared to 20% of placebo patients (p<0.0001) 24 at week achieved clear or almost skin (p<0.0001); experienced significantly greater improvements in measures of health-related quality life, pain and symptoms anxiety depression< td> | 10/22/21 | Dermatologic |
| Vyne Therapeutics Inc., of Bridgewater, N.J., and Cutia Therapeutics Co. Ltd., of Shanghai | Amzeeq (minocycline) topical foam, 4% | Topical antibacterial | Moderate to severe acne | First patient dosed in study in China | 10/5/21 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | TTR (mutant) expression inhibitor; siRNA agent | Hereditary transthyretin-mediated amyloidosis in patients with polyneuropathy | Top-line results showed study met all 18-month secondary endpoints; statistically significant improvements in progression of neuropathy, quality of life, gait speed, nutritional status and overall disability relative to external placebo; improvements in exploratory endpoints; improved safety and tolerability profile | 10/27/21 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | Once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and GLP-1 receptor agonist | Type 2 diabetes | Results from Surpass-4 trial published in the Lancet with 5 mg, 10 mg and 15 mg in 2002 adults with increased CV risk achieved each of its primary and key secondary endpoints; statistically significant and superior A1C and body weight reductions compared to insulin glargine for both estimands at 52 weeks; highest dose of tirzepatide led to an A1C reduction of 2.58 % and reduced body weight by 11.7 kg (-25.8 lb., -13%) vs. results for those treated with insulin glargine (A1C reduction of 1.44 % and weight gain of 1.9 kg [+4.2 lb., +2.2 %]) | 10/19/21 | Endocrine/Metabolic |
| Fortress Biotech Inc., of New York, and Cyprium Therapeutics Inc., of New York | CUTX-101 (copper histidinate) | Subcutaneous injectable formulation; copper complexes | Menkes disease | Pivotal studies (n=129) met both prespecified primary and secondary efficacy endpoints; significantly greater median overall survival (OS) compared to untreated historical control patients; 79% reduction in risk of death was observed in Cuhis-ET patients compared with HC-ET patients and median OS was 177.1 and 16.1 months, respectively, with a hazard ratio of (95% CI) = 0.208 (0.094, 0.463) p<0.0001; 75% reduction in the risk of death was also observed cuhis-lt patients compared with hc-lt subjects and median os 62.4 17.6 months, respectively, hr (95% ci)="0.253" (0.119, 0.537); p<0.0001; well-tolerated< td> | 10/8/21 | Endocrine/Metabolic |
| Gan & Lee Pharmaceuticals Co. Ltd., of Beijing | GL-GLA | Biosimilar insulin glargine | Type 1 and type 2 diabetes | Completed 2 studies comparing drug to reference biologic, which concluded equivalent treatment-induced immunogenicity; efficacy estimates between groups were within prespecified similarity margins and concluded to be equivalent; safety endpoints were comparable between biosimilar and reference biologic | 10/14/21 | Endocrine/Metabolic |
| Idorsia Ltd., of Allschwil, Switzerland | Lucerastat | Glucosylceramide synthase inhibitor | Fabry disease | Phase III trial, Modify, missed primary endpoint; awaiting results of interim analysis of open-label study before making decision on program's future | 10/11/21 | Endocrine/Metabolic |
| Mannkind Corp., of Westlake Village, Calif. | Afrezza | Technosphere Insulin | Type 1 or type 2 diabetes | First pediatric patient enrolled; planned to enroll 260 patients | 10/4/21 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, a business unit of Parma, Italy-based Chiesi Farmaceutici SpA | Pegunigalsidase alfa (PRX-102) | Modified stabilized version of the recombinant human alpha-galactosidase A protein | Fabry disease | Completed final dosing of last patient | 10/15/21 | Endocrine/Metabolic |
| Galapagos NV, of Mechelen, Belgium | Filgotinib | JAK1 inhibitor | Crohn’s disease | Randomized last patient; enrolled around 1,374 patients; top-line data expected in first half of 2023 | 10/4/21 | Gastrointestinal |
| Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Oral small-molecule potassium-competitive acid blocker | Erosive esophagitis | Top-line results of Phalcon-EE study met primary and key secondary superiority endpoints; met the healing phase with a healing rate of 93% compared to 85% for lansoprazole (p<0.0001); 2 3 8 10 15 20 24 30 significantly faster healing than lansoprazole (70% for vonoprazan mg and 53% mg) (p="0.0004);" was also compared to in a superiority test onset of sustained resolution heartburn by day but did not achieve statistical significance rates were numerically greater at week moderate severe patients (p<0.0001); met the primary all secondary endpoints maintenance phase; vs. (79% mg, 81% 72% (p<0.0001 both noninferiority comparisons; p="0.0218" comparison; comparison); percentage with disease who maintained ee through (75% 77% 61% consistent safety profile< td> | 10/18/21 | Gastrointestinal |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Human monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13 proteins | Eosinophilic esophagitis | Trial met its co-primary endpoints in patients at 300-mg weekly dose; significantly improved the ability to swallow and reduced eosinophils in the esophagus compared to placebo; 64% reduction in disease symptoms from baseline compared to 41% for placebo (p=0.0008); 23.78-point improvement on the 0-84 DSQ scale compared to a 13.86-point improvement for placebo (p<0.0001); 36 38 84 89 baseline dsq scores were approximately and points, respectively; 59% of patients achieved histological disease remission compared to 6% placebo (p<0.0001); proportion who a peak esophageal intraepithelial eosinophil count ?6 eos hpf; mean levels hpf, overall rates adverse events 84% for dupixent 71% placebo< td> | 10/25/21 | Gastrointestinal |
| Seres Therapeutics Inc., of Cambridge, Mass. | SER-109 | Oral microbiome therapeutics | Recurrent Clostridioides difficile infection (CDI) | Exploratory data from Ecospor phase III study showed significantly reduced recurrence rates compared to placebo; well-tolerated in the study with no treatment-related serious adverse events observed in the active arm | 10/26/21 | Gastrointestinal |
| Galapagos NV, of Mechelen, Belgium | Filgotinib | Oral, once-daily JAK1 inhibitor | Moderately to severely active ulcerative colitis | Results of 2 post-hoc analyses from Selection and Selection LTE studies showed benefits of continued dosing at 200 mg; in Selection, continuing filgotinib 200 mg among nonresponders resulted in 65.7% biologic-naïve and 62.2% biologic-experienced patients achieving partial Mayo Clinic Score (pMCS) response by week 12, with 17.1% biologic-naïve and 16.7% biologic-experienced patients in pMCS remission | 10/5/21 | Gastrointestinal |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Movantik (naloxegol) | Opioid antagonist | Opioid-induced constipation | Movantik at 12.5-mg and 25-mg doses provided rapid onset with early symptom relief and consistent, predictable and sustained efficacy in improving multiple symptoms despite high baseline symptom burden; favorable safety profile and tolerability | 10/25/21 | Gastrointestinal |
| Galera Therapeutics Inc., of Malvern, Pa. | Avasopasem manganese (GC-4419) | Selective small molecule dismutase mimetic | Oral mucositis | Roman trial missed primary endpoint of reduction in the incidence of severe oral mucositis (SOM) and relative reduction in all key SOM endpoints; 16% relative reduction in the incidence of SOM in the avasopasem (54%) vs. placebo group (64%) (p=0.113); 56% relative reduction in the number of days of SOM in the avasopasem (8 days) vs. placebo group (18 days) (p=0.011) ; 27% relative reduction in the severity (incidence of Grade 4 OM) of SOM in the avasopasem (24%) vs. placebo group (33%) (p=0.167); well-tolerated with similar rates of adverse events in the active and placebo arms | 10/19/21 | Gastrointestinal/Cancer |
| Endo International plc, of Dublin | Xiaflex | Collagenase clostridium histolyticum | Peyronie's disease | Post-hoc analysis showed incremental benefits were obtained from each of the 4 treatment cycles; achieved <20% 2 reduction in penile curvature after cycles of injections< td> | 10/22/21 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Linzagolix | Oral GnRH receptor antagonist | Uterine fibroids | Data from Primrose 1 and 2 showed 200-mg linzagolix without ABT after 24 weeks reduced uterine volume by 39%; 49% fibroid volume reduction; median serum E2 was suppressed to 9 pg/mL; 21% reduction from baseline and median serum E2 increased to 43 pg/mL at 52 weeks | 10/20/21 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Linzagolix | Oral GnRH receptor antagonist | Severe adenomyosis | Data from pilot study at 200 mg reduced uterine volume by 55% from baseline at 12 weeks and 32% from baseline at 24 weeks (p<0.006) 4 12 24 after continued treatment with 100-mg dose; pelvic pain reduced initial signs at week and markedly (p="0.0035)" weeks (p<0.0001); significant improvements in quality of life (p<0.05); no serious adverse events; bone mineral density consistent estradiol suppression, decrease the lumbar spine was 2.4 ± 3.6% mean z-score -0.65 (range -1.6, 0.9)< td> | 10/20/21 | Genitourinary/Sexual Function |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Non-radiographic axial spondyloarthritis | In the Select-Axis 2 study, 45% of patients taking Rinvoq had a Assessment in SpondyloArthritis International Society (ASAS) 40 response compared to 23% of patients who got placebo (p<0.0001); percent of patients achieving asdas low disease activity was 42% for rinvoq and 18% placebo (p<0.0001); mean change from baseline in magnetic resonance imaging spondyloarthritis research consortium canada score -2.49 0.57 (p<0.0001)< td> | 10/7/21 | Immune |
| Angion Biomedica Corp., of Uniondale, N.Y., and Vifor Pharma., of St. Gallen, Switzerland | ANG-3777 | Small molecule designed to mimic the biological activity of hepatocyte growth factor | Kidney transplant patients at risk for delayed graft function | Top-line results showed trial did not demonstrate a statistically significant difference from placebo on the primary endpoint of estimated glomerular filtration rate (eGFR) at 12 months in patients (p=0.33); inconsistent benefit on key secondary endpoints; consistent overall safety profile | 10/27/21 | Immune |
| Argenx BV, of Breda, the Netherlands | Efgartigimod | FcRn antagonist | Generalized myasthenia gravis | Additional phase III ADAPT trial demonstrated consistent depth of response across first 2 treatment cycles as measured by minimal symptom expression; consistent disease score improvements by patient subgroup based on affected muscle domain or concomitant medication | 10/8/21 | Immune |
| Biogen Inc., of Cambridge, Mass. | Tysabri (natalizumab | Humanized monoclonal IgG4 kappa antibody against human alpha 4 integrin | Multiple sclerosis | Exploratory outcomes and additional secondary endpoints from Nova study showed time to first relapse was similar between the 2 dosing schedules with the proportion who were relapse-free at 72 weeks at 96.9% for Q6W and 97.6% for Q4W; proportion of patients free of disability worsening was 90% in Q6W arm and 92% in Q4W arm; disease activity rates were similar between both groups; no evidence of disease activity was 70% for Q6W and 67.4% for Q4W; known safety profile | 10/13/21 | Immune |
| Biogen Inc., of Cambridge, Mass. | Vumerity (diroximel fumarate) | Monomethyl fumarate prodrug | Relapsing-remitting multiple sclerosis | EVOLVE-MS-2 study demonstrated an improved GI tolerability profile compared to Tecfidera; weekly incidence of GI adverse events remained consistent and low throughout the 5-week treatment period for Vumerity but increased in number and severity for Tecfidera after titration to the full dose, peaking in weeks 3 and 4; 1.6%-4.8% vs. 4.4%-13.9% | 10/13/21 | Immune |
| Genentech, of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing-remitting multiple sclerosis | Long-term results from phase III Opera I and II demonstrated sustained reduction in disability progression and suppression of disease activity; resulted in a 35% reduction in the risk of patients with RMS needing a walking aid over 7.5 years compared with patients who switched from interferon beta-1a to Ocrevus after the 96-week double-blind period (5.2% vs. 7%, respectively; 95% CI: 0.65 [0.44–0.97]; p=0.034); EDSS?6 was sustained for at least 48 weeks in a post-hoc analysis; rapid and robust reduction in annualized relapse rate (ARR); maintained through the 5.5-year period; maintained a low ARR of 0.03 after 7.5 years of Ocrevus treatment | 10/13/21 | Immune |
| Genentech, of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing-remitting multiple sclerosis | Long-term results from Oratorio study showed Ocrevus resulted in a 29% reduction in 48-week confirmed disability progression (CDP) in patients with PPMS over 8 years compared with patients who switched to Ocrevus from placebo after the double-blind period of at least 120 weeks (95% CI: 0.71 [0.57–0.87]; p=0.001); 24% (95% CI: 0.76 [0.62–0.92]; p=0.005) reduced risk of recurrent 48-week CDP (re-baselining EDSS after onset of CDP event) was seen in patients who were continuously treated with Ocrevus compared with those who switched from placebo; upper limb disability progression also reduced in patients (95% CI: 0.66 [0.50–0.86] respectively; p=0.002) | 10/13/21 | Immune |
| Merck KGaA, of Darmstadt, Germany, and its EMD Serono unit | Evobrutinib | Bruton’s tyrosine kinase inhibitor | Relapsing multiple sclerosis | Completed patient enrollment | 10/4/21 | Immune |
| Mesoblast Ltd., of New York | Remestemcel-L | Mesenchymal stem cells obtained from the bone marrow of healthy adult donors | Steroid-refractory acute graft-vs.-host disease | Results published in Bone Marrow Transplantation showed treatment resulted overall response, with 67% day 28 and 64% day 180 overall survival compared with 10% day 28 and 10% day 180 OS in the MAGIC cohort (both p=0.01) when treated with various biologics in children | 10/18/21 | Immune |
| TG Therapeutics Inc., of New York | Ublituximab | Anti-CD20 antibody | Relapsing forms of multiple sclerosis | Results from Ultimate 1 and 2 trials, resulted in an annualized relapse rate (ARR) of 0.076 compared to 0.188 for teriflunomide, representing a relative reduction of approximately 60% (p<0.0001); 1 arr of 0.091 compared to 0.178 for teriflunomide, representing a relative reduction approximately 50% (p="0.0022);" total number t1 gadolinium (gd) enhancing lesions were reduced as result ublituximab treatment by 97% and 96% with teriflunomide in ultimate & 2, respectively (p<0.0001); new or enlarging t2 92% 90% 44.6% ublituximab-treated patients achieved neda 198% improvement over (p<0.0001), 43% 277% improvements disability observed both studies, significantly more achieving 12-week 24-week cdi vs. teriflunomide; well-tolerated no unexpected safety signals< td> | 10/15/21 | Immune |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Relapsing forms of multiple sclerosis | Daybreak extension study showed consistent safety profile and no new safety signals; low annualized relapse rate of 0.103; at months 36 and 48, 75% and 71% of participants were relapse-free and 3- and 6-month confirmed disability progression was observed in 13.9% and 11.4% of participants in the trial, respectively | 10/13/21 | Immune |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Ponvory (ponesimod) | S1P1 modulator | Relapsing forms of multiple sclerosis | Optimum study subgroup results from treatment-naïve patients showed ponesimod significantly reduced annualized relapse rate (RR = 0.714; 99% CLs: 0.486, 1.049; p=0.0241) and MS-fatigue was significantly lower in the ponesimod group compared with the teriflunomide group at week 108, MD = -5.30 (95%CLs: -8.25, -2.35; p=0.0004); combined unique active lesions (CUALs) were consistent with the overall population; patients significantly benefited from ponesimod compared with teriflunomide in both the EDSS ?3 (RR = 0.424; 95% CLs: 0.329, 0.546; p<0.0001) and treatment-naïve (rr="0.411;" 95% cls: 0.310, 0.545; p<0.0001) < td> | 10/13/21 | Immune |
| Arcturus Therapeutics Inc., of San Diego | ARCT-154 | Next generation STARR mRNA Vaccine | COVID-19 | Vietnam Ministry of Health approved phase IIIb study; initiated study and expected to enroll 20,000 subjects | 10/12/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | AZD-7442 | Combination of 2 long-acting monoclonal antibodies tixagevimab and cilgavimab | COVID-19 | Tackle trial met the primary endpoint, with a dose of 600 mg of AZD-7442 given by intramuscular injection reducing the risk of developing severe COVID-19 or death from any cause by 50% vs. placebo in outpatients who had been symptomatic for 7 days or less; company seeking EUA from FDA | 10/11/21 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-196 + BRII-198 | Human monoclonal antibody targeting spike glycoprotein of SARS-CoV-2 | COVID-19 | Interim results showed treatment reduced the risk of hospitalization and death over placebo by 78% in 837 outpatients at high risk of clinical progression; at 28-day primary endpoint, 0 (n=418) deaths were observed on BRII-196/BRII-198 vs. 8 (n=419) deaths on the placebo arm; at 5 days of symptom onset, 2% progressed to hospitalization or death compared with 11% in the placebo arm; no drug-related severe adverse events | 10/3/21 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | CCR5 antagonist | COVID-19 | First patient treated in CD-16 trial | 10/25/21 | Infection |
| Entasis Therapeutics Inc., of Waltham, Mass., and Zai Lab Ltd., of Shanghai | Sulbactam-durlobactam | Beta-lactam antibiotic; beta-lactamase inhibitor | Infections caused by Acinetobacter baumannii | Top-line results of Attack trial (n =207) met the primary endpoint of 28-day all-cause mortality in patients with carbapenem-resistant Acinetobacter infections; mortality was 19% vs. 32.3% in colistin; statistically significant difference in clinical cure at Test of Cure was observed with 61.9% in sulbactam-durlobactam (SUL-DUR) arm vs. 40.3% colistin arm (95% CI: 2.9, 40.3); favorable safety profile with statistically significant reduction in nephrotoxicity; SUL-DUR nephrotoxicity was 13.2% vs. 37.6% in the colistin arm (p = 0. 0002); overall adverse events in the safety population were comparable between treatment groups with 87.9% in the SUL-DUR vs. 4.2% colistin | 10/18/21 | Infection |
| Inflarx NV, of Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | COVID-19 | Study enrolled 369 patients; top-line results expected in first quarter of 2022 | 10/12/21 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide (GT-0918) | Nonsteroidal anti-androgen | COVID-19 | First patient dosed | 10/4/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Islatravir and doravirine | Fixed-dose combination | HIV-1 infection | Top-line results from SWITCH A and SWITCH B trials at week 48 met their primary efficacy endpoint of percentage of participants with HIV-1 RNA levels ?50 copies/mL | 10/25/21 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1647 | Combination of 6 mRNAs in 1 vaccine which encode for 2 proteins located on the surface of CMV; 5mRNA encoding subunits that form the membrane-bound pentamer complex and 1 mRNA encoding the full-length membrane-bound glycoprotein B | Cytomegalovirus | First participant dosed | 10/26/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Comirnaty (tozinameran) | mRNA vaccine | COVID-19 | Final results from 30-microgram booster dose showed relative vaccine efficacy of 95.6% when compared to those who did not receive a booster; consistent efficacy in multiple subgroup analyses; favorable safety profile | 10/21/21 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Free radical scavenger | Mild to moderate COVID-19 | Data safety monitoring board recommended continuation of its phase III trial; no serious adverse events and safety concerns reported to date; expected to complete enrollment in fourth quarter of 2021 | 10/26/21 | Infection |
| SAB Biotherapeutics Inc., of Sioux Falls, S.D. | SAB-185 | Fully human polyclonal IgG antibody targeting human SARS-CoV-2 | COVID-19 | First patient dosed | 10/4/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Vulvovaginal candidiasis | Post-hoc analysis results from Vanish-303 study published in Infectious Diseases Society of America's journal Clinical Infectious Diseases showed similar rates of clinical cure and clinical improvement at test-of-cure for African American patients (54.8% and 63.4%, respectively) and patients with a body mass index >35 (54.5% and 68.2%, respectively) compared with overall rates; well-tolerated; primarily gastrointestinal adverse events | 10/21/21 | Infection |
| Sifi SpA, of Catania, Italy | Polihexanide | Polymer targeting trophozoites and cysts of the protozoan Acanthamoeba | Acanthamoeba keratitis | Study met its primary endpoint of clinical resolution rate over a 12-month timeframe | 10/19/21 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Top-line results of VLA2001-301 Cov-compare trial (n=4012) met both co-primary endpoints; well-tolerated and statistically significant better tolerability profile compared to active comparator vaccine; demonstrated superiority over AZD-1222 (ChAdOx1-S), in terms of geometric mean titer for neutralization antibodies (GMT ratio=1.39, p<0.0001), 2 7 30 43 (vla-2001 gmt 803.5 (95% ci: 748.48, 862.59)), (azd-1222(chadox1-s) 576.6 ci 543.6, 611.7)), as well noninferiority in terms of seroconversion rates (scr above 95% both treatment groups) at weeks after the second vaccination day adults ages and older; induced broad antigen-specific ifn-gamma producing t cells reactive against s- (74.3%), n- (45.9%) m- (20.3%) protein; fewer adverse events up to days vaccination; injection-site reactions (73.2% vla-2001 vs. 91.1% azd-1222 (chadox1-s), p<0.0001) systemic (70.2% p<0.0001); no treatment-related serious events< td> | 10/18/21 | Infection |
| Emphycorp Inc., of Flemington, N.J. | N-115 | Sodium pyruvate (inhaled) | COVID-19, long COVID and pulmonary fibrosis | In active COVID-19-infected patients, treatment lowered viral numbers below 10,000 viral genome copies by day 6.4, vs 10.3 days as reported for untreated patients; in long COVID patients, drug clinically and significantly reduced coughing/sneezing, reduced headaches and body aches, increased SaO2 levels and improved breathing; in pulmonary fibrosis, there was statistically and clinically significant improvement in all lung functions, compared to baseline, including increase in FEV-1, SaO2, FVC, FEV-1/FVC ratios (from 52% to 86%) and a reduction in coughing and fatigue | 10/5/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics LP, of Miami | Molnupiravir (MK-4482, EIDD-2801) | Nucleoside analogue; RNA-directed RNA polymerase inhibitor | COVID-19 | Interim analysis showed molnupiravir reduced the risk of hospitalization or death by approximately 50%; 7.3% of patients who received molnupiravir were either hospitalized or died through day 29 compared with 14.1% of placebo-treated patients p=0.0012 | 10/1/21 | Infection |
| Russian Direct Investment Fund, of Moscow | Sputnik Light vaccine | COVID-19 vaccine | COVID-19 | Study from 28,000 participants demonstrated 70% efficacy against infection from the Delta variant during the first 3 months after vaccination; 75% efficacy among subjects under the age of 60 | 10/13/21 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Inhaled interferon beta | Mild to moderate COVID-19 | External data safety monitoring board recommended phase III Sprinter study in hospitalized COVID-19 patients | 10/20/21 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | Sci-B-Vac | Hepatitis B large envelope protein modulator | Hepatitis B virus infection | Study results published in The Journal of the American Medical Association Network Open demonstrated robust and consistent immune responses across all 3 lots after 2 and 3 doses; higher seroprotection rates (SPR) and antibody geometric mean concentration (GMC) compared to Engerix-B; no safety signals were observed; SPR after 2 doses, at day 168, was 90.4% for 3-antigen vaccine candidates compared to 51.6% for Engerix-B, increasing to 99.3% and 94.8%, respectively, after the third dose; mean GMC of anti-HBs titers was more than 7.5x higher after 2 doses, at day 168, and 3.5x higher after 3 doses, at day 196 (5,442.4 mIU/mL VBI vs. 1,567.2 mIU/mL Engerix-B); higher percentage of participants with anti-HBs titers ? 100 mIU/mL, a more stringent titer threshold, of 95.8% vs. 86.3% for Engerix-B at day 196 | 10/13/21 | Infection |
| Westvac Biopharma Co. Ltd., of Chengdu, China | Recombinant COVID-19 vaccine (Sf9 cells) | Recombinant COVID-19 vaccine (Sf9 cells) | COVID-19 | Philippines currently conducting trial, along with countries including Indonesia, Kenya and Mexico; 10,000+ subjects worldwide have received vaccine | 10/14/21 | Infection |
| Horizon Therapeutics plc, of Dublin | Krystexxa (pegloticase) | Pegylated uric acid specific enzyme | Gout | Mirror trial in combination with methotrexate met primary endpoint (p<0.001); 3 achieved complete and durable response to therapy; no new safety concerns; 42% of patients had completed serum uric acid (sua), maintaining sua <6 mg dl at least 80% the time in months 6< td> | 10/25/21 | Inflammatory |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Completed recruitment in the second of 2 phase IIIb pivotal studies; top-line results expected in second quarter of 2022 | 10/28/21 | Inflammatory |
| Biogen Inc., of Cambridge, Mass. | Tofersen (BIIB-067) | Antisense oligonucleotide binds to SOD1 mRNA | Amyotrophic lateral sclerosis | Top-line data from Valor study did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale; did not reach statistical significance as measured by a joint-rank analysis (difference of 1.2; p=0.97); signs of reduced disease progression across multiple secondary and exploratory endpoints; secondary endpoint of change from baseline in total CSF SOD1 protein observed between the tofersen and placebo groups of 38% and 26% in the faster- and slower-progressing populations, respectively; change from baseline in plasma neurofilament light chain, with differences observed between the tofersen and placebo groups of 67% and 48% in the faster- and slower-progressing populations, respectively; favored on measures of respiratory function and muscle strength; median time to event not estimated for survival analyses due to the low number of events over the 28-week period; procedural pain, headache, pain in extremity, fall and back pain as common adverse events; serious adverse events were reported in 18.1% of tofersen and 13.9% of placebo; 5.6% of participants discontinued treatment due to an event; serious neurologic events were reported in 4.8% of patients; 1 death reported and not related to tofersen | 10/17/21 | Musculoskeletal |
| Radius Health Inc., of Boston | Tymlos (abaloparatide) | 34 amino acid analogue of native human PTHrP | Osteoporosis | Top-line data of Atom study met the primary and secondary endpoint; change in lumbar spine bone mineral density (BMD) at 12 months (p<0.0001); 6 12 average increase in lumbar spine bone mineral density of 8.5% compared to 1.2% for patients receiving; percentage change placebo bmd at months, total hip and femoral neck months also met endpoints; consistent safety profile< td> | 10/18/21 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | SRP-9001 (delandistrogene moxeparvovec) | AAV vector-based gene therapy | Duchenne muscular dystrophy | Results from SRP-9001-101 study found that 4 SRP-9001-treated participants improved 8.6 points on the North Star Ambulatory Assessment (NSAA) vs. matched natural history cohort (MNHC) 3 years after treatment; 12 others treated in SRP-9001-102 study had +2.9-point difference on NSAA vs. MNHC 1 year after treatment; functional results from Study SRP-9001-103 cohort 1 found participants (11) improved 3 points on NSAA 6 months after treatment | 10/11/21 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass., and Roche Holding AG, of Basel, Switzerland | SRP-9001 | Gene therapy expressing microdystrophin | Duchenne muscular dystrophy | Initiated pivotal Embark study | 10/4/21 | Musculoskeletal |
| Abbvie Inc., of North Chicago | Vraylar (cariprazine) | Antipsychotic | Major depressive disorder | Study 3111-301-001 met its primary endpoint demonstrating statistically significant change from baseline to week 6 in the Montgomery-Åsberg Depression Rating Scale total score | 10/29/21 | Neurology/Psychiatric |
| Abbvie Inc., of North Chicago | Vraylar (cariprazine) | Antipsychotic | Major depressive disorder | Study 3111-302-001 demonstrated numerical improvement in depressive symptoms from baseline to week 6 in Montgomery-Åsberg Depression Rating Scale total score compared with placebo but did not achieve statistical significance | 10/29/21 | Neurology/Psychiatric |
| Abbvie Inc., of North Chicago, Ill. | ABBV-951 | Foslevodopa/foscarbidopa | Advanced Parkinson's disease | Study met its primary endpoint of increase from baseline in "on" time at week 12, which was 2.72 hours for ABBV-951 vs. 0.97 hours for oral levodopa/carbidopa (p= 0.0083); decreases in "off" time after 12 weeks were 2.75 hours for ABBV-951 vs. 0.96 hours for oral LD/CD (p=0.0054); consistent safety profile; non-serious adverse events, mild or moderate in severity | 10/28/21 | Neurology/Psychiatric |
| Avadel Pharmaceuticals plc, of Dublin | FT-218 (sodium oxybate controlled-release) | GABA B receptor agonist | Narcolepsy | Post-hoc analysis from Rest-On trial showed statistically significant mean sleep latency improvements and reductions in weekly cataplexy attacks compared to placebo ; significantly greater proportion of participants who received ON-SXB vs. placebo experienced increased mean sleep latency change from baseline ranging from ?5 minutes to 30 minutes | 10/19/21 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Small molecule targeting the altered form of filamin A | Alzheimer’s disease | Started a 750-patient study to evaluate safety and efficacy of simufilam over 52 weeks; second phase III efficacy study expected to begin by the end of 2021 | 10/6/21 | Neurology/Psychiatric |
| Corium International Inc., of Menlo Park, Calif. | Azstarys (serdexmethylphenidate and dexmethylphenidate) | Alpha 2 adrenoceptor agonist | Attention deficit hyperactivity disorder | Data published in the Journal of Child and Adolescent Psychopharmacology showed patients had a mean change from baseline in Swanson, Kotkin, Agler, M-Flynn, and Pelham Rating Scale – Combined scores of -4.87 for Azstarys and 0.54 for placebo (p<0.001); 13 30 post hoc analysis showed drug had an onset of minutes and a duration efficacy hours< td> | 10/29/21 | Neurology/Psychiatric |
| Genentech, of South San Francisco, a member of the Roche Group | Enspryng (satralizumab) | Humanized monoclonal IgG2kappa antibody against human IL-6R | Anti-aquaporin-4 antibody seropositive neuromyelitis optica spectrum disorder | 4-year data from open-label extension periods of Sakurastar and Sakurasky studies found that 73% and 71% of people treated with Enspryng remained relapse-free after 192 weeks (3.7 years), respectively, and 90% and 91% remained free from severe relapse; favorable safety and tolerability profile in overall treatment period of up to 7 years and no new safety signals observed | 10/14/21 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Fixed-dose synergistic combination of baclofen, naltrexone and sorbitol ; target PMP22 expression | Charcot-Marie-Tooth disease type 1A | Results from PLEO-CMT trial published in Orphanet Journal of Rare Diseases demonstrated a statistically significant improvement in the primary endpoint compared to placebo in Overall Neuropathy Limitations scale at high-dose (mean difference: -0.37 points; 97.5% CI: [-0.68 to -0.06]; p=0.008); extension study ongoing and 130 patients received high-dose:safe and well-tolerated in high and low doses | 10/19/21 | Neurology/Psychiatric |
| Prilenia Therapeutics B.V., of Naarden, the Netherlands | Pridopidine | Selective S1R agonist | Huntington disease | Completed patient enrollment in Proof-HD study | 10/20/21 | Neurology/Psychiatric |
| Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. | Zuranolone (SAGE-217/BIIB-125) | Oral neuroactive steroid GABA-A receptor-positive allosteric modulator | Major depressive disorder and postpartum depression | Data from Waterfall and Shoreline studies showed consistent safety profile with no evidence of withdrawal, weight gain, sexual dysfunction, euphoria or sleep disruption; rapid improvement in quality of life and overall health across all functioning and well-being domains at day 15 and across all domains at day 42 | 10/4/21 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | TV-46000/mdc-IRM | Risperidone extended-release injectable suspension for subcutaneous use | Schizophrenia | In the Rise study, time to impending relapse was prolonged by 3.5 times compared to placebo for the overall group (p<0.0001, 2 5 hr="0.283)," by times for the once-monthly treatment (p<0.0001, and 2.7 once every months> | 10/30/21 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Netarsudil ophthalmic solution | Rho associated protein kinase inhibitor | Open-angle glaucoma or ocular hypertension | Study (n=245) met primary endpoint; netarsudil 0.02% reduced mean diurnal IOP by 4.7 mmHg (22.6%) from baseline compared to 3 mmHg (14.3%) with ripasudil 0.4% (p<0.0001); safe and well-tolerated; mild adverse events< td> | 10/12/21 | Ocular |
| Clearside Biomedical Inc., of Alpharetta, Ga. | CLS-TA | Corticosteroid triamcinolone acetonide formulated for suprachoroidal administration | Uveitic macular edema | Post-hoc analyses of the Peachtree and Azalea studies showed that anatomic response may precede visual response in patients treated with CLS-TA; regardless of baseline visual acuity, patients experienced clinically significant improvement in vision at 24 weeks and macular thickness improved to approximately 300 microns | 10/4/21 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Extracellular domains of human VEGFR-3 antibodies fused to the Fc-domain | Wet age-related macular degeneration | Initiated patient enrollment for phase III Shore and Coast study in Europe | 10/4/21 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Extracellular domains of human VEGFR-3 antibodies fused to the Fc-domain | Wet age-related macular degeneration | Initiated patient enrollment for phase III Shore and Coast studies in Asia-Pacific region | 10/19/21 | Ocular |
| Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab-trbw) | Monoclonal antibody targeting insulin-like growth factor-1 receptor | Thyroid eye disease | Comprehensive results from the OPTIC-X open-label extension study published in Ophthalmology showed median duration of 12.9 months vs. 6.3 months in those treated in OPTIC study; 90.6% for proptosis; 91.7% for overall response; 95.2% for a Clinical Activity Score of 0 or 1; 85.7% for diplopia; no new safety concerns at week 48 | 10/21/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010, Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Norse three study showed no additional serious adverse events and no unanticipated safety signals | 10/12/21 | Ocular |
| Frequency Therapeutic Inc., of Lexington, Mass. | FX-322 | Combination of small-molecule drugs designed to activate inner-ear progenitor cells | Sensorineural hearing loss | First patient dosed | 10/21/21 | Other/Miscellaneous |
| X4 Pharmaceuticals Inc., of Boston | Mavorixafor | CXCR4 receptor antagonist | Warts, hypogammaglobulinemia, infections and myelokathexis | Completed enrollment of 31 patients; top-line data expected in the fourth quarter of 2022 | 10/4/21 | Other/Miscellaneous |
Hutchmed Ltd., of Hong Kong, and Astrazeneca plc, of Cambridge, U.K. |
Savolitinib (orpathys) + Imfinzi (durvalumab) | MET inhibitor + PD-L1 inhibitor | MET-driven advanced papillary renal cell carcinoma | First of 200 participants dosed; primary endpoint is median progression-free survival; secondary endpoints include median overall survival, objective response rate, duration of response, 6-months and 12-months disease control rate, time to second progression, safety, pharmacokinetics and quality of life | 11/1/21 | Cancer |
| Apollomics Inc., of Foster City, Calif. | APL-106/uproleselan injection | E-selectin antagonist | Relapsed/refractory acute myeloid leukemia | First patient dosed | 11/22/21 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Checkmate-816 trial met the primary endpoint of improved event-free survival (EFS) in patients; in combination with chemotherapy showed a statistically significant and clinically meaningful improvement in EFS compared to chemotherapy alone when given before surgery | 11/8/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Quizartinib | Oral FLT3 inhibitor | FLT3-ITD-positive acute myeloid leukemia | Top-line results from Quantum study met its primary endpoint in combination with standard induction and consolidation chemotherapy; statistically significant and clinically meaningful improvement in overall survival compared to standard; manageable and consistent with the known safety profile | 11/18/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Datopotamab deruxtecan | Antibody-drug conjugate targeting TROP2 | Metastatic breast cancer | First patient dosed | 11/18/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Enhertu (trastuzumab deruxtecan) | HER2-targeting antibody-drug conjugate | HER2-positive early stage breast cancer | First patient dosed | 11/30/21 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Qinlock (ripretinib) | Switch-control tyrosine kinase inhibitor engineered to broadly inhibit KIT and PDGFRA mutated kinases | Gastrointestinal stromal tumor | Top-line results from Intrigue study did not meet the primary endpoint of improved progression-free survival (PFS) compared to sunitinib; median PFS of 8.3 months compared to 7 months for sunitinib (Hazard Ratio [HR] 0.88, p=0.360) in patients with a KIT exon 11 primary mutation (n=327); mPFS of 8 months compared to 8.3 months for sunitinib (HR 1.05, nominal p=0.715) in all patient intent-to-treat population | 11/5/21 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Metastatic non-small-cell-lung cancer | Completed enrollment | 11/9/21 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Advanced liver cancer | Interim results from Cosmic-312 study showed at a median follow-up of 13.6 months data favored cabozantinib in combination with atezolizumab but did not reach statistical significance for overall survival ( P=0.438); median OS was 15.4 months for cabozantinib in combination with atezolizumab (n=432) vs. 15.5 months for sorafenib (n=217); cabozantinib monotherapy reduced the risk of disease progression or death in the ITT population by 29% vs. sorafenib (p=0.0107; prespecified critical p-value of 0.00451); median PFS was 5.8 months for cabozantinib (n=188) vs. 4.3 months for sorafenib (n=217); objective response rate was 11% for cabozantinib in combination with atezolizumab, 3.7% for sorafenib and 6.4% for cabozantinib monotherapy; disease control rates (complete response + partial response + stable disease) were 78%, 65% and 84%, respectively | 11/20/21 | Cancer |
| Glycomimetics Inc., of Rockville, Md. | Uproleselan | Antagonist of E-selectin | Relapsed/refractory acute myeloid leukemia | Completed enrollment; 388 patients enrolled; top-line data expected in year end of 2022 | 11/15/21 | Cancer |
| Hutchmed Ltd., of Hong Kong, and Astrazeneca plc, of Cambridge, U.K. | Savolitinib (orpathys) +Tagrisso (osimertinib) | MET tyrosine kinase inhibitor + irreversible EGFR inhibitor | Non-small-cell lung cancer | Initiated phase III SACHI study; first patient dosed | 11/24/21 | Cancer |
| Immunogen Inc., of Waltham, Mass. | Mirvetuximab soravtansine | Antibody-drug conjugate comprising FR?-binding antibody, cleavable linker and tubulin-targeting agent DM4 | Platinum-resistant ovarian cancer | Top-line data of Soraya trial (n=106) met primary endpoint with confirmed objective response rate of 32.4%; median duration of response 5.9 months; favorable tolerability profile; treatment-related adverse events led to dose reductions in 19% of patients, dose delays in 32% of patients and discontinuations in 7% of patients | 11/30/21 | Cancer |
| Innovent Biologics Inc., of San Francisco and Suzhou, China | Sintilimab | PD-1 inhibitor | EGFR-mutated nonsquamous non-small-cell lung cancer | Interim analysis of Orient-31 study in combination with Byvasda (bevacizumab biosimilar injection) plus chemotherapy group demonstrated statistically significant and clinically meaningful improvement in progression-free survival compared with chemotherapy group (p<0.0001); median progression-free survival was 6.9 months vs. 4.3 for chemotherapy; did not cross futility stopping boundary (hr="0.726);" objective response rate and duration of were improved compared to consistent safety profile no new unexpected signals< td> | 11/21/21 | Cancer |
| Jazz Pharmaceuticals plc, of Dublin | Zepzelca (lurbinectedin) | Alkylating drug that binds guanine residues within DNA | Extensive-stage small-cell lung cancer | First patient enrolled in trial in combination with Tecentriq (atezolizumab, Roche Holding AG) | 11/30/21 | Cancer |
| Urogen Pharma Ltd., of Princeton, N.J. | UGN-102 (mitomycin) | Intravesical solution of mitomycin; reverse-thermal hydrogel | Low-grade intermediate-risk non-muscle invasive bladder cancer | Planning to initiate phase III study in early 2022; expected to enroll 220 patients | 11/10/21 | Cancer |
| Xoma Corp., of Emeryville, Calif., and Novartis AG, of Basel, Switzerland | NIS-793 | Monoclonal antibody targeting TGF beta | First-line metastatic pancreatic ductal adenocarcinoma | Started the study comparing NIS-793 plus gemcitabine and nab-paclitaxel compared to gemcitabine and nab-paclitaxel alone; primary efficacy endpoint is overall survival; progression-free survival, overall response rate, disease control rate and time to response will be measured as secondary endpoints | 11/4/21 | Cancer |
| Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Advanced ovarian cancer | Prime study met its primary endpoint; statistically significant and clinically meaningful progression-free survival benefit; tolerable safety profile | 11/30/21 | Cancer |
| Zymeworks Inc., of Vancouver, British Columbia, and Beigene Ltd., of Cambridge, Mass., and Beijing | Zanidatamab | Bispecific antibody binding 2 non-overlapping epitopes of HER2 | HER2?positive gastroesophageal adenocarcinoma | Initiated HERIZON-GEA-01 study; enroll approximately 700 patients | 11/9/21 | Cancer |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Unique form of eicosapentaenoic acid | Peripheral artery disease | Post-hoc analysis showed significantly reduced total (first and subsequent) total ischemic events as primary endpoints by 32%; trended toward a 22% reduction in first events; substantial cardiovascular risk reduction in the high-risk REDUCE-IT population; consistent safety profile; atrial fibrillation/flutter occurred with icosapent ethyl vs. placebo in patients (5.2% vs. 2.6%, respectively; P=0.07) | 11/15/21 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Small molecule cardiac myosin activator | Heart failure with reduced ejection fraction | Additional results from GALACTIC-HF study showed greater reductions in stroke for patients by 77% p-value=0.001; atrial fibrillation by 51% p=0.001; reduction in new onset atrial fibrillation/flutter p=0.044; primary composite endpoint of cardiovascular death or heart failure events was similar in patients with a history of stroke (HR 0.86; p=0.18) and in patients without a history of stroke (HR 0.93; p=0.06); risk of first fatal or non-fatal stroke was reduced by 35% (HR 0.65; p=0.004), and the risk of fatal stroke was reduced by 44% (HR 0.5; p=0.048) | 11/15/21 | Cardiovascular |
| Merck & Co. Inc., of Kenilworth, N.J., and Bayer AG, of Leverkusen, Germany | Verquvo (vericiguat) | Soluble guanylate cyclase stimulator | Chronic heart failure and reduced ejection fraction | Initiated Victor phase III study; enrolling 6,000 patients; primary efficacy endpoint is time to first event of cardiovascular death or hospitalization for heart failure | 11/11/21 | Cardiovascular |
| Mesoblast Ltd., of New York | Rexlemestrocel-L | Immunomodulatory therapy | Chronic heart failure with reduced ejection fraction | Pre-specified outcomes showed single dose reduced the incidence of heart attacks or strokes by 65% across all 537 NYHA class II or class III treated patients compared with standard of care (SOC) alone, p=0.001; reduced the incidence of heart attacks or strokes by 79% compared with SOC alone, p<0.001 301 537 in patients high levels of inflammation (hs-crp ? 2mg l); reduced the incidence cardiovascular death by 80% compared with soc alone, p="0.005" death, heart attacks or strokes 33% across all nyha class ii iii patients, and 45% l),> | 11/15/21 | Cardiovascular |
| Milestone Pharmaceuticals Inc., of Montreal | Etripamil nasal spray | Short-acting calcium channel blocker | Paroxysmal supraventricular tachycardia (SVT) | Post-hoc data from NODE-301 trial showed etripamil significantly decreased heart rate (HR) (p<0.0001); 1 3 10 16 40 rapidly and significantly decreased hr prior to conversion of svt sinus rhthym starting within minutes (p<0.03) lasting up after treatment (p<0.004); non-statistically different baseline hrs in the etripamil (179 beats per minute) placebo (174 groups; greater (p<0.0001) group over hour with a maximum minute between groups at minutes; maximal change from was positively correlated patient-reported relief symptoms (p="0.0027);" effectiveness> | 11/15/21 | Cardiovascular |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa., and San Diego | Bentracimab | Recombinant, human monoclonal antibody antigen-binding fragment | Uncontrolled major or life-threatening bleeding | Interim results from Reverse-IT (n=150) demonstrated bentracimab achieved the primary endpoint, reversing the antiplatelet effects of ticagrelor; 135% reduction in platelet inhibition (P<0.001) 5 10 24 was observed within to minutes after initiation of bentracimab infusion and sustained through all timepoints over hour; 90% eligible patients achieved the co-primary endpoint trial (p<0.001); well-tolerated non-serious adverse events; pain associated with surgical procedures as main event< td> | 11/15/21 | Cardiovascular |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Donidalorsen | Antisense medicine designed to reduce the production of prekallikrein | Hereditary angioedema | Initiated OASIS-HAE study; planning to enroll 84 patients; randomized to receive monthly or bi-monthly subcutaneous donidalorsen for 25 weeks | 11/18/21 | Dermatologic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Donidalorsen | Antisense medicine designed to reduce the production of prekallikrein | Hereditary angioedema | Initiated OASIS-HAE study; planning to enroll 84 patients; randomized to receive monthly or bi-monthly subcutaneous donidalorsen for 25 weeks | 11/18/21 | Dermatologic |
| Krystal Biotech Inc., of Pittsburgh | Beremagene geperpavec (Vyjuvek) | Non-integrating viral vector expressing the collagen VII gene; topical therapy | Dystrophic epidermolysis bullosa | Top-line results from Gem-3 trial met primary and secondary endpoint; 67% achieved complete wound healing at the 6-month timepoints as compared to 22% of wounds treated with placebo (p<0.005); 71% complete wound healing at the 3-month timepoints as compared to 20% of wounds treated with placebo ( p<0.005); both 3- and 6-month (p<0.005) in ad-hoc analysis; well-tolerated; no drug-related serious adverse events or discontinuations; mild event; meaningful change anti-hsv-1 anti-col7 antibodies< td> | 11/29/21 | Dermatologic |
| Anji Pharmaceuticals Inc., of Cambridge, Mass. | ANJ-900 | Delayed-release formulation of metformin | Type 2 diabetes with chronic kidney disease | Full enrollment of Beijing study; top-line data expected in 2023 | 11/30/21 | Endocrine/Metabolic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Olezarsen (IONIS-APOCIII-LRx) | Antisense drug designed to inhibit production of apoC-III | Severe hypertriglyceridemia | Initiated Core study in patients with triglyceride levels ? 500 mg/dL on standard-of-care therapies for elevated triglycerides; primary endpoint is the percent change in fasting triglycerides from baseline at month 6 | 11/2/21 | Endocrine/Metabolic |
| Albireo Pharma Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | All types of progressive familial intrahepatic cholestasis | Long-term study showed sustained improvements in mean serum bile acids (sBA) and pruritus scores over time; overall median exposure from the first dose of Bylvay was 53 (3-128) weeks; 40% of patients met criteria for sBA response (?70% reduction in sBAs or sBAs ?70 ?mol/L); reduced autotaxin (-50%), pruritus (-1.4) and sBAs (-49%); improvement in hepatic health parameters in transaminases and total bilirubin levels, quality of sleep and growth, with greater improvement observed in responders compared with non-responders | 11/12/21 | Gastrointestinal |
| Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel | Aramchol | Stearoyl CoA desaturase-1 inhibitor | Liver fibrosis | Results showed first 20 patients had 60% fibrosis improvement by at least 1 stage as early as 24 weeks; statistically significant reductions in biomarkers associated with liver fibrosis including ALT (p<0.0001), ast(p<0.0001), fib-4 (p="0.0006)" and proc-3 (p<0.0001) at week 48< td> | 11/8/21 | Gastrointestinal |
| Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Ulcerative colitis | First patient enrolled in Conclude study | 11/24/21 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor (THR)-? selective agonist | Nonalcoholic steatohepatitis | Readout of 52-week open-label study (n=171) showed drug provided rapid and sustained reduction in liver fat and liver volume (p<0.0001) 100 as well fibrosis; reduced atherogenic lipids, including ldl-c and triglycerides, liver enzymes inflammatory biomarker; blood pressure ~2mmhg, (p="0.02);" safe well-tolerated at mg per day in patients< td> | 11/12/21 | Gastrointestinal |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Chronic kidney disease | Data from regional analysis showed relative risk reduction of the primary composite endpoint of sustained ?50% decline in estimated glomerular filtration rate (eGFR) 3.3-8 % for dapagliflozin compared to placebo; no significant heterogeneity between regions (p=0.77); prespecified analysis showed dapagliflozin significantly slowed yearly eGFR decline in patients living with CKD over a median duration of 2.3 years; favorable hemodynamic changes in glomerulus | 11/8/21 | Genitourinary/Sexual Function |
| Bayer AG, of Whippany, N.J. | Kerendia (finerenone) | Nonsteroidal mineralocorticoid receptor antagonist | Chronic kidney disease in patients with type 2 diabetes | In Fidelity prespecified pooled analysis showed sustained ?57% decrease in eGFR or renal death with finerenone compared to placebo, analyzed with and without SGLT2i use at baseline (with SGLT2i: HR=0.42, 95% CI 0.16-1.08; without SGLT2i: HR=0.80, 95% CI 0.69-0.92; p-interaction 0.29); sustained ?40% decrease in eGFR, or renal death with finerenone compared to placebo, analyzed with and without SGLT2i use at baseline (with SGLT2i: HR=0.70, 95% CI 0.41-1.21; without SGLT2i: HR=0.84, 95% CI 0.76-0.92; p-interaction 0.59); reduced the risk of the composite CV outcome of time to CV death, nonfatal myocardial infarction, nonfatal stroke or hospitalization for heart failure, analyzed with and without SGLT2i at baseline (with SGLT2i: HR=0.63, 95% CI 0.40-1; without SGLT2i: HR=0.87, 95% CI 0.79-0.96; p-interaction 0.41; hyperkalemia was common adverse event | 11/8/21 | Genitourinary/Sexual Function |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | SGLT2 inhibitor | Heart failure associated with chronic kidney disease | Prespecified subanalysis of the Emperor-Preserved trial reduced the combined relative risk of cardiovascular death and hospitalization for heart failure and slowed kidney function decline; consistently improved outcomes across the full range of kidney function down to an eGFR of 20 mL/min/1.73 m2; well-tolerated | 11/8/21 | Genitourinary/Sexual Function |
| Glaxosmithkline plc, of London | Daprodustat | HIF-PH inhibitor | Anemia in chronic kidney disease | Results of Ascend-ND and Ascend-D studies published in The New England Journal of Medicine showed studies met their primary efficacy and safety endpoints; improved or maintained patients within their target hemoglobin (Hb) range; hazard ratio reflecting time to first occurrence of MACE of 1.03; 95% CI, (0.89 to 1.19), noninferiority with the predefined margin of 1.25; hazard ratio reflecting time to first occurrence of MACE of 0.93; 95% CI, (0.81-1.07), achieving noninferiority with the predefined margin of 1.25 | 11/8/21 | Genitourinary/Sexual Function |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Immunoglobulin G-degrading enzyme from Streptococcus pyogenes | Goodpasture's disease | Initiated phase III study; expected to enroll approximately 50 patients | 11/15/21 | Genitourinary/Sexual Function |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Oteseconazole (SHR-8008) | Lanosterol-14 demethylase inhibitor | Acute vulvovaginal candidiasis | Completed study comparing drug to fluconazole | 11/3/21 | Genitourinary/Sexual Function |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy | Kidney transplant | First 2 patients treated for more than 12 months have been successfully weaned off all chronic immunosuppression without evidence of rejection and with stable kidney function; 3 patients treated for at least 3 months achieved and maintained >50% T-cell chimerism | 11/4/21 | Genitourinary/Sexual Function |
| Vascular Therapies LLC, of Cresskill, N.J. | Sirogen | Sirolimus formulation for intraoperative local drug delivery | End-stage renal disease; chronic kidney disease | Access1 study (n=243) showed improved overall arteriovenous fistula (AVF) maturation and forearm (radio-cephalic) AVF maturation; improved suitability for dialysis at 12 months and secondary patency | 11/4/21 | Genitourinary/Sexual Function |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Oral factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | First patient enrolled in Redeem-2 trial | 11/29/21 | Hematologic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | Oral, once-daily selective JAK inhibitor | Rheumatoid arthritis | Post-hoc analysis of Select-Beyond trial showed that 34% of patients with Rinvoq and background csDMARDs achieved Clinical Disease Activity Index (CDAI) remission (CDAI ?2.8) at first occurrence of response before week 60; 79% achieved CDAI low disease activity (LDA) (CDAI ?10); CDAI remission and CDAI LDA in 39% and 61% of patients, respectively, at 60 weeks | 11/9/21 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis in patients with systemic lupus erythematosus | From pre-treatment baseline in the Aurora 1 study to month 30 in the Aurora 2 study, mean urine protein/creatinine ratio was -3.32 mg/mg for Lupkynis and -2.55 mg/mg for the control group; estimated glomerular filtration rate remained stable through month 30 for patients taking Lupkynis | 11/1/21 | Immune |
| Immunic Inc., of New York | Vidofludimus calcium (IMU-838) | Selective oral DHODH inhibitor | Relapsing multiple sclerosis | First patient enrolled in Ensure study | 11/18/21 | Immune |
| Immunic Inc., of New York | Vidofludimus calcium (IMU-838) | Selective oral DHODH inhibitor | Relapsing multiple sclerosis | First patient enrolled in Ensure study | 11/18/21 | Immune |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Psoriatic arthritis | In the Discover 2 trial, approximately 91% of patients receiving treatment every 4 weeks (q4w) and 87% of patients receiving treatment every 8 weeks (q8w) who achieved 20% improvement in joint signs and symptoms and low levels of disease activity (ACR20) at week 52 maintained the response at week 100; 83% of q4w and 79% of q8w patients maintained an ACR50 response; 72% of q4w and 80% of q8w patients maintained an ACR70 response | 11/1/21 | Immune |
| Rigel Pharmaceuticals Inc., of South San Francisco | Fostamatinib | Syk tyrosine kinase inhibitor | Warm autoimmune hemolytic anemia | Completed enrollment of Forward trial; top-line data expected mid-2022 | 11/2/21 | Immune |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody that selectively and directly inhibits both interleukin 17A (IL-17A) and interleukin 17F (IL-17F) | Psoriatic arthritis | Top-line interim results from Be Optimal study met the primary endpoint and all ranked secondary endpoints ; achieved 50 % or greater improvement in signs and symptoms of disease from baseline compared with placebo at week 16; significant improvements at week 16 over placebo in physical function | 11/19/21 | Immune |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody that selectively and directly inhibits both interleukin 17A (IL-17A) and interleukin 17F (IL-17F) | Psoriatic arthritis | Top-line interim results from Be Optimal study met the primary endpoint and all ranked secondary endpoints; achieved 50% or greater improvement in signs and symptoms of disease from baseline compared with placebo at week 16; significant improvements at week 16 over placebo in physical function | 11/19/21 | Immune |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Avigan/Reeqonus (favipiravir) | Broad-spectrum antiviral in oral tablet form; selective inhibitor of viral RNA-dependent RNA polymerase | Mild to moderate COVID-19 | Study (n=1231) did not achieve statistical significance on the primary endpoint of time to sustained clinical recovery; additional analyses of the trial data are ongoing | 11/12/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | AZD-7442 | Combination of long-acting monoclonal antibodies tixagevimab and cilgavimab targeting the SARS-CoV-2 spike protein | COVID-19 | New data from 6-month follow-up of prevention trial showed 83% reduced risk of symptomatic COVID-19, with no severe disease or deaths at 300-mg; well-tolerated; no new safety issues | 11/18/21 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | AZD-7442 | Combination of long-acting monoclonal antibodies tixagevimab and cilgavimab targeting the SARS-CoV-2 spike protein | COVID-19 | Tackle trial resulted in 88% reduced risk of severe COVID-19 or death when treated within 3 days of symptom onset; well-tolerated | 11/18/21 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif | Lonafarnib (boosted w/ritonavir + peginterferon alfa) | Oral prenylation inhibitor | Chronic hepatitis D virus infection | Completed enrollment in the D-Livr study with over 400 patients; top-line data expected by the end of 2022 | 11/1/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics Inc., of Miami | Molnupiravir (MK-4482, EIDD-2801) | Oral antiviral | Mild to moderate COVID-19 | Results from Move-out study (n=1433) showed reduced risk of hospitalization or death from 9.7% in placebo group to 6.8% in molnupiravir group; absolute risk reduction of 3% (nominal p=0.0218); relative risk reduction of 30%; 9 deaths in placebo and 1 in molnupiravir group | 11/26/21 | Infection |
| Molecular Partners, AG of Zurich-Schlieren, Switzerland | Ensovibep | Binds to the SARS-CoV-2 spike protein at 3 distinct locations | COVID-19 | Independent data and safety monitoring board recommended that the recruitment of patients in ACTIV-3 not continue in hospitalized patients; 470 patients randomized in the ensovibep arm of the study; safe and well-tolerated with reported side effects consistent with standard of care | 11/16/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | VIP receptor agonist | COVID-19 | Safety update from Activ-3b Critical Care study sponsored by NIH indicated independent drug safety monitoring board found no new safety concerns after reviewing more than 300 patients and recommended continued enrollment | 11/2/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | VIP receptor agonist | COVID-19 | Subgroup analysis in patients previously treated with remdesivir in the COVID-AIV trial showed statistically significant (p=0.03) 2.5-fold increased odds of being alive and free of respiratory failure at 60 days as primary endpoint; statistically significant (p=0.006) 4-fold higher odds of being alive at day 60 among patients treated with aviptadil compared placebo | 11/29/21 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Comirnaty (tozinameran) | COVID-19 spike glycoprotein modulator | COVID-19 prophylaxis | Updated analysis of phase III study in individuals ages 12-15 (n=2228) showed strong protection against COVID-19; vaccine efficacy of 100% against COVID-19 in long-term analysis; no serious safety concerns identified | 11/22/21 | Infection |
| Polypid Ltd., of Petah Tikva, Israel | D-PLEX??? | Drug candidate designed to provide local prolonged and controlled anti-bacterial activity directly at the surgical site | Surgical site infections in abdominal surgery | 500th patient enrolled for Shield I study | 11/18/21 | Infection |
| Pulmonem Inc., of Dieppe, New Brunswick | PULM-001 | Repurposed and reformulated version of Dapsone | COVID-19 | Initiated recruitment of U.S.-based patients | 11/22/21 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Regen-Cov (casirivimab and imdevimab) | 2 fully human antibodies targeting distinct regions of the receptor binding domain region of the spike protein of human COVID-19 coronavirus | COVID-19 | New analysis showed 81.6% reduced risk of developing infection during the prespecified follow-up period, between months 2-8 (7 REGEN-COV, 38 placebo; 95% confidence interval [CI]: 59.8%, 91.6%; nominal p<0.0001); 0 6 8-month assessment period showed hospitalizations for covid-19 in regen-cov group and placebo group; long-lasting protective effects without any artificial mutations or sequences; no new safety signals < td> | 11/8/21 | Infection |
| Sinovac Biotech Ltd., of Beijing | Coronavac | Inactivated coronavirus vaccine | COVID-19 | Results from subgroup safety study (n=684) found to be safe for healthy pediatric and adolescent population ranging from 3 to 17 years of age; no suspicious and unexpected serious adverse effects occurred | 11/9/21 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Inhaled interferon beta | Hospitalized COVID-19 | Achieved enrollment target of 610 patients in Sprinter study; top-line results expected in early 2022 | 11/11/21 | Infection |
| Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London | Sotrovimab | Intramuscular administration; SARS-CoV-2 neutralizing monoclonal antibody | COVID-19 | Data showed Comet-Tail study achieved its primary endpoint; noninferior to intravenous (I.V.) therapy; IM administration showed 2.7% rate of progression to hospitalization for more than 24 hours or death through day 29 compared to 1.3% in the I.V. administration arm; low rates of serious adverse events; 79% reduction in hospitalization and death at day 29 vs. placebo | 11/12/21 | Infection |
| Amylyx Pharmaceuticals Inc. of Cambridge, Mass. | AMX-0035 | Sodium phenylbutyrate and taurursodiol | Amyotrophic lateral sclerosis | First of approximately 600 patients dosed in the Phoenix study; primary efficacy outcome of the 48-week study is a joint assessment of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised and survival; secondary endpoints include slow vital capacity, measured both at home using a self-administered spirometer to support virtual data collection and at clinic sites using standard spirometry, quality of life patient-reported outcome assessments, ventilation-free survival rates and other measures | 11/4/21 | Musculoskeletal |
| Astrazeneca plc, of Cambridge, U.K. | Saphnelo (anifrolumab) | Monoclonal antibody targeting subunit 1 of the type I interferon receptor | Systemic lupus erythematosus | Post-hoc analysis of pooled data from the Tulip-1 and -2 studies showed patients diagnosed within the last 2 years had a 14.4% improvement in the BILAG–based Composite Lupus Assessment (BICLA) over standard therapy alone at week 52 compared to a 17.1% improvement for patients with established disease; higher BICLA response rates were seen for Saphnelo compared to standard of care alone regardless of the type of baseline standard therapy use; biologic-naïve patients who received Saphnelo had similar benefits in comprehensive disease activity, OCS taper and flare rate compared to those previously treated with other biologics | 11/1/21 | Musculoskeletal |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD | Allogeneic mesenchymal-like cells | Muscle injury following arthroplasty for hip fracture | Completed enrollment; 240 patients enrolled; top-line data in third quarter of 2022 | 11/15/21 | Musculoskeletal |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of the muscle relaxant cyclobenzaprine hydrochloride | Fibromyalgia | Study met its pre-specified primary endpoint in Relief trial; significantly reduced daily pain compared to placebo (p=0.01); higher rate of responders to TNX-102 SL (47%) than to placebo (35%; p=0.006) showed activity in key secondary endpoints, improvements in sleep quality, mitigation of fatigue, and fibromyalgia-specific functional recovery at 5.6-mg | 11/8/21 | Musculoskeletal |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous MSC-NTF cells | Amyotrophic lateral sclerosis | Trial did not reach statistical significance on the primary and secondary endpoints; post-hoc analysis showed greater percentage of responders compared to placebo (33% vs. 14%) predicted by ENCALS model in patients (ALSFRS-R ? 25); greater percentage of responders compared to placebo (34.6% vs. 15.6%) in prespecified subgroup of participants with ALSFRS-R greater than or equal to 35; | 11/29/21 | Musculoskeletal |
| Neurana Pharmaceuticals Inc., of San Diego | Tolperisone | Non-opioid, centrally acting muscle relaxant | Muscle spasm | Completed patient enrollment; 1,000 patients enrolled in Resume-1 study; top-line data in first quarter of 2022 | 11/29/21 | Musculoskeletal |
| Amring Pharmaceuticals Inc., of Berwyn, Pa., and Amzell BV, of Hoofddorp, the Netherlands | AMZ-002 | Purified synthetic polypeptide | Infantile epileptic disease | Entered phase III study | 11/19/21 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass. | Aduhelm (aducanumab-avwa) | Monoclonal antibody directed against amyloid beta | Alzheimer’s disease | Results showed significant reduction in tau pathology compared to placebo; statistically significant correlation between change in plasma p-tau181 and lowering of amyloid beta plaque; lowered plasma p-tau181 in a dose- and time-dependent manner vs. placebo; p-tau decreased 13% from baseline (p<0.001), 13 while placebo group rose 8% in emerge high-dose group; p-tau decreased 16% from baseline (p<0.001), 9% engage cdr-sb(p="0.0166" and 0.0005); mmse (p < 0.0001 0.0002); adas-cog adcs-adl-mci 0.0010) studies, respectively< td> | 11/11/21 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Verdiperstat (BHV-3241) | Selective, brain-penetrant, and myeloperoxidase enzyme inhibitor | Amyotrophic lateral sclerosis | Completed enrollment; 160 patients enrolled; top-line data expected in mid-2022 | 11/15/21 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Small molecule drug that restores the normal shape and function of altered filamin A (FLNA) protein | Alzheimer’s disease | Initiated phase III study to evaluate safety and efficacy of simufilam over 78 weeks in 1,000 patients | 11/18/21 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | Vesicular monoamine transporter 2 inhibitor | Tardive dyskinesia | Pooled data from patients 65 and older in the Kinect trial program and long-term extension studies found that long-term use of Ingrezza produced substantial and clinically meaningful improvements | 11/1/21 | Neurology/Psychiatric |
| Sage Therapeutics Inc., of Cambridge, Mass. | Zuranolone | GABA A receptor modulator | Major depressive disorder | Updated endpoint of Coral study to measure HAMD-17 change from baseline at day 3 | 11/2/21 | Neurology/Psychiatric |
| Scholar Rock Holding Corp., of Cambridge, Mass. | Apitegromab | Selective inhibitor of the activation of latent myostatin | Type 2 and type 3 spinal muscular atrophy | Finalized Sapphire study design | 11/30/21 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | Anti-CGRP subcutaneous injection | Episodic migraine | Finesse interim data of the non-interventional study showed 48.7% of the patients with 6-month data achieved the primary endpoint, with a higher percentage in episodic (53.2%) than chronic patients (43%); mean number of migraine days per month (d/m) decreased from 12.7 (baseline) to 6.2 (month 6); acute migraine medication use decreased from 9.6 days/month at baseline to 4.4 d/m at month 6 | 11/8/21 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution; 0.25% | Small-molecule immune-modulating covalent inhibitor of RASP | Dry eye disease | Completed enrollment in Tranquility trial | 11/9/21 | Ocular |
| Arctic Vision Biotechnology Co. Ltd., of Shanghai | ARVN-001 (triamcinolone acetonide) | Suspension of the corticosteroid triamcinolone acetonide | Macular edema associated with uveitis | First set of patients dosed | 11/22/21 | Ocular |
| Eyenovia Inc., of New York | Microline | Formulation of pilocarpine | Presbyopia | First of approximately 140 patients enrolled in the Vision-2 study; primary endpoint is improvement in high contrast binocular distance corrected near visual acuity measured in low light conditions 2 hours after treatment; top-line results expected in the second quarter of 2022 | 11/4/21 | Ocular |
| Oculis SA, of Lausanne, Switzerland | OCS-01 (dexamethasone preservative-free intravitreal eye drops) | Glucocorticoid receptor agonist | Diabetic macular edema | First patient dosed in Diamond trial | 11/12/21 | Ocular |
| Outlook Therapeutics Inc., of Iselin, N.J. | ONS-5010, Lytenava (bevacizumab-vikg) | Monoclonal antibody targeting VEGF | Wet age-related macular degeneration | Data from the phase III NORSE TWO trial (n=288) met both primary and secondary endpoints with statistically significant and clinically relevant results; 41.7% (p = 0.0052) subjects gained ? 15 letters of vision; 56.5% (p = 0.0016) subjects gained ? 10 letters of vision; 68.5% (p = 0.0116) subjects gained ? 5 letters of vision; gained 11.2 letters (p = 0.0043) in BCVA; strong safety profile | 11/13/21 | Ocular |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Smoking cessation | Completed fifth and final data safety monitoring committee review; no concerns regarding phase III ORCA-2 study; safety and adverse event profile remains favorable; study medication was excellent; preliminary data expected in first half of 2022 | 11/22/21 | Other/Miscellaneous |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor | Alagille syndrome | Data from the Iconic study published in The Lancet showed the drug produced a significant response as measured by serum bile acid, xanthomas, improved quality of life and growth; inhibition of enterohepatic circulation, 380-µg/kg dose improved pruritus response in more than 80% of patients at week 48; responses were durable across the 204-week period analyzed | 11/1/21 | Other/Miscellaneous |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Wegovy (semaglutide, 2.4 mg injection) | GLP-1 receptor agonist | Obesity | Study achieved significant and sustained weight loss over the 2-year study period; reduced calorie meal plan and increased physical activity in 304 adults for 104 weeks; significantly reduced body weight from baseline to week 104 compared to placebo ( p<0.0001); lost 5% of body weight with wegovy vs. placebo (77.1% 34.4%; p<0.0001)< td> | 11/5/21 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin receptor agonist | Bardet-Biedl syndrome | After 1 year on setmelanotide, adults had a 9.1% reduction in BMI and children had a 9.5% reduction in BMI; 85% of patients reported clinically meaningful improvements in their health-related quality of life (HRQOL) status or preserved their non-impaired HRQOL status; Impact of Weight on Quality of Life Questionnaire-Lite scores increased by a mean of 12 points for adults; Pediatric Quality of Life Inventory improved by a mean of 11.2 points for pediatric patients | 11/1/21 | Other/Miscellaneous |
| Orexo AB, of Uppsala, Sweden | OX-124 | Intranasal formulation of naloxone | Opioid overdose | Study met its primary endpoints with naloxone exposure within the targeted interval in healthy volunteers; significantly faster and higher absorption of naloxone compared to intramuscular dosing ; well-tolerated; | 11/16/21 | Toxicity and Intoxication |
| Astrazeneca plc, of Cambridge, U.K. | Enhertu (trastuzumab deruxtecan) | HER2-targeting antibody-drug conjugate | HER2-positive early stage breast cancer | Results from Destiny-breast03 study demonstrated superior progression-free survival for Enhertu vs. trastuzumab emtansine (T-DM1) in patients | 12/3/21 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | HER2-targeting antibody-drug conjugate | HER2-positive unresectable and/or metastatic breast cancer | Destiny-Breast03 phase III study showed higher progression-free survival (PFS) and objective response rate (ORR) in pre-specified patient subgroups compared to trastuzumab emtansine (T-DM1) in patients; higher PFS compared to T-DM1 in patients with stable brain metastases at baseline; Enhertu improved PFS to a median of 15 months vs. 3 months for T-DM1 ; consistent known safety profile | 12/9/21 | Cancer |
| Bayer AG, of Leverkusen, Germany | Nubeqa (darolutamide) | Androgen receptor inhibitor | Metastatic castration-resistant prostate cancer | Arasens study met its primary endpoint in combination with docetaxel and androgen deprivation therapy (ADT); significantly increased overall survival compared to docetaxel and ADT | 12/3/21 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab (BGB-A317) | Humanized IgG4 anti-PD-1 monoclonal antibody | Recurrent or metastatic nasopharyngeal cancer | Results showed rationale 309 study in combination with chemotherapy achieved primary endpoint; statistically significant progression-free survival (PFS) improvement in combination with gemcitabine and cisplatin (arm A) vs. placebo and chemotherapy, median PFS was 9.2 months vs. 7.4 months (p < 0.0001); objective response rate and complete response were 69.5% and 16% compared to 55.3% and 6.8%, respectively; median duration of response was 8.5 months vs. 6.1 months; serious treatment related adverse events reported in 36 patients vs. 44 patients | 12/10/21 | Cancer |
| CG Oncology Inc., of Irvine, Calif. | CG-0070 | Adenovirus expressing GM-CSF | Non-muscle-invasive bladder cancer unresponsive to Bacillus Calmette-Guerin | First patient dosed in Japan; study to enroll 110 patients to evaluate safety and efficacy as monotherapy | 12/20/21 | Cancer |
| Checkpoint Therapeutics Inc., of New York | Cosibelimab | PD-L1 inhibitor | Non-squamous-non-small-cell-lung cancer | Initiated Conterno study in combination with pemetrexed and platinum chemotherapy | 12/8/21 | Cancer |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | I/Ontak | Engineered IL-1 diphtheria toxin fusion protein | Persistent or recurrent cutaneous T-cell lymphoma | Completed patient enrollment in pivotal study; top-line results expected in first half of 2022 | 12/6/21 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate targeting HER2 | ER2 mutant unresectable, locally advanced or metastatic nonsquamous non-small-cell lung cancer | First patient dosed in the Destiny-Lung04 study comparing Enhertu to platinum-pemetrexed doublet chemotherapy plus Keytruda (pembrolizumab, Merck & Co.); primary endpoint is progression-free survival as assessed by blinded independent central review (BICR); secondary endpoints include overall survival, investigator-assessed PFS, overall response rate and duration of response as assessed by BICR and investigator, pharmacokinetics, patient-reported tolerability, immunogenicity and safety | 12/23/21 | Cancer |
| Galera Therapeutics Inc., of Malvern, Pa. | Avasopasem manganese (GC-4419) | Selective small-molecule dismutase mimetic | Radiotherapy- induced severe oral mucositis | Corrected top-line results from Roman trial achieved primary endpoint; 16% relative reduction in the incidence of severe oral mucositis (SOM) vs. placebo (p=0.0451); 56% relative reduction in the number of days of SOM vs. placebo (p=0.0022); 27% relative reduction in the severity vs. placebo (p=0.052) | 12/14/21 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Trodelvy (sacituzumab govitecan-hziy) | Antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor | Triple-negative breast cancer | Subgroup analysis showed Trodelvy improved progression-free survival (PFS), with 56% reduction in the risk of disease worsening or death (p=0.008); median PFS of 5.4 months vs. 2.2 months with chemotherapy; extended median overall survival to 13.8 months vs. 8.5 months with physician’s choice of chemotherapy (p=0.159) | 12/10/21 | Cancer |
| Henlius Biotech Inc., of Shanghai | Serplulimab (HLX-10) | Monoclonal antibody targeting PD-1 | Extensive-stage-small-cell-lung cancer | Interim analysis met the primary endpoint of overall survival in combination with chemotherapy in previously untreated patients; good safety and no detection of a new safety signal | 12/7/21 | Cancer |
| Henlius Biotech Inc., of Shanghai | Serplulimab (HLX-10) | Monoclonal antibody targeting PD-1 | Extensive-stage small-cell lung cancer | Interim results of Astrum-005 study in combination with carboplatin-etoposide showed prolonged median overall survival (OS) in both the overall population and the Asian subgroup; median OS of 15.38 months vs. 11.1 months for placebo; reducing risk of death by 38% (41% in the Asian subgroup; p <0.001); 2 2-year os rate in treatment groups were 43.2% and 8%, respectively; manageable safety profile< td> | 12/20/21 | Cancer |
| Hutchmed Ltd., of Hong Kong and Shanghai | Fruquintinib | Oral inhibitor of VEGFR-1, -2 and -3 | Metastatic colorectal cancer | Completed patient enrollment in Fresco-2 registrational study; primary endpoint is overall survival; top-line results expected in second half of 2022 | 12/6/21 | Cancer |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | VGX-3100 | DNA-based Immunotherapy | HPV-associated cervical high-grade squamous intraepithelial lesions | Reveal2 study fully enrolled and top-line efficacy and safety data expected in second half of 2022; first patient dosed in China; trial expected to enroll up to 84 participants | 12/14/21 | Cancer |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | VGX-3100 | DNA-based Immunotherapy | High-grade cervical dysplasia caused by HPV-16 and/or HPV-18 | Completed 52-week safety follow-up in Reveal 1 study; well-tolerated; patients treated with VGX-3100 who met the primary endpoint at week 36 remained clear of HPV-16 and/or HPV-18 at week 88 ; no substantial change in the per-protocol assessment of primary efficacy | 12/14/21 | Cancer |
| Jazz Pharmaceuticals plc, of Dublin, and Pharmamar SA, of Madrid, Spain | Zepzelca (lurbinectedin) | Alkylating drug that binds guanine residues within DNA | Relapsed small-cell lung cancer | Initiated confirmatory phase III Lagoon study testing monotherapy and in combination with irinotecan compared with investigator's choice of topotecan or irinotecan | 12/13/21 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor (ATG-010) | XPO1 inhibitor | Advanced or recurrent endometrial cancer | Completed patient recruitment in Siendo study; to evaluate efficacy and safety for front-line maintenance therapy; top-line data expected by end of 2021 or early 2022 | 12/2/21 | Cancer |
| Kronos Bio Inc., of San Mateo, Calif. | Entospletinib | Selective inhibitor targeting SYK | Acute myeloid leukemia | First patient dosed in registrational Agility trial testing combination with standard-of-care anthracycline and cytarabine chemotherapy; primary endpoint is measurable residual disease | 12/6/21 | Cancer |
| Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | (HR+/HER2-) advanced or metastatic breast cancer | Broad ad hoc exploratory analysis in combination with endocrine therapy (ET) showed significant overall survival compared to ET alone (Luminal A and B= p=.021 and p=.018, respectively); significant improvement in median OS of 40.3 months compared to 29.4 months for ET alone in in HR+/HER2- breast cancer patients; basal-like subtype patients had had poorer OS in both the Kisqali combination and ET alone groups p=.148 | 12/8/21 | Cancer |
| Samsung Bioepis Co. Ltd., of Incheon, Korea | SB-3 (Ontruzant, trastuzumab-dttb) | Herceptin biosimilar | HER2-positive early or locally advanced breast cancer | 5-year follow-up data in larger group of patients (n=171 ) showed event-free survival (EFS) between SB-3 and Herceptin (trastuzumab, Roche Holding AG) group (p=0.335); overall survival rate p=0.073; post-hoc analysis (n=271) showed EFS rate of 79.8% and the Non-drifted Herceptin group had 82.5%p=0.391; overall survival was p=0.975 | 12/8/21 | Cancer |
| Sumitomo Dainippon Pharma Oncology Inc., of Cambridge, Mass. | DSP-7888 | Immunotherapeutic cancer vaccine targeting Wilms tumor 1 | Recurrent or progressive glioblastoma | Phase III Wizard 201-G study terminated after second interim analysis; study achieving low probability of meeting the primary endpoint of overall survival at the final analysis | 12/14/21 | Cancer |
| Urogen Pharma Ltd., of Princeton, N.J. | UGN-102 (mitomycin) | Intravesical solution of mitomycin; reverse-thermal hydrogel | Low-grade intermediate-risk non-muscle invasive bladder cancer | First patient dosed in home instillation study | 12/21/21 | Cancer |
| Zymeworks Inc., of Vancouver, British Columbia, and Beigene Ltd., of Cambridge, Mass. and Beijing | Zanidatamab | Bispecific antibody binding 2 non-overlapping epitopes of HER2 | HER2?positive gastroesophageal adenocarcinoma | First patient dosed in HERIZON-GEA-01 study | 12/9/21 | Cancer |
| Bridgebio Pharma Inc., of Palo Alto, Calif. | Acoramidis | Stabilizes tetrameric transthyretin | Transthyretin amyloid cardiomyopathy | In the Attribute-CM study, the mean decline in 6-minute walk distance after 12 months was 9 meters for acoramidis and 7 meters for placebo; study will continue to month 30 to measure all-cause mortality and cardiovascular hospitalizations | 12/27/21 | Cardiovascular |
| Mesoblast Ltd., of New York | Rexlemestrocel-L | Immunomodulatory therapy | Heart failure with reduced ejection fraction | New analyses from Dream-HF trial showed greatest benefit in patients with diabetes and/or ischemia, who are at high risk of cardiovascular mortality, heart attacks or strokes; compared to control patients, rexlemestrocel-L reduced the incidence of 3-point MACE by 37% overall in NYHA class II or III HFrEF patients with diabetes and/or myocardial ischemia (n=385, p=0.02) and by 54% in those with diabetes and/or myocardial ischemia who had evidence of systemic inflammation, as defined by elevated baseline levels of hs-CRP >2 mg/L (n=212, p=0.003) | 12/5/21 | Cardiovascular |
| Pfizer Inc., of New York | Vyndaqel (tafamidis meglumine); Vyndamax (tafamidis) | Oral transthyretin stabilizer | Transthyretin amyloid cardiomyopathy | Post-hoc results published in Circulation: Heart Failure showed Vyndaqel reduced by 30% mortality at 30 months compared to placebo; clinically significant 41% reduction in the risk of all-cause mortality among patients who received continuous treatment of Vyndaqel and Vyndamax (median follow-up 58.5 months) compared to patients who first received placebo in study before transitioning to Vyndaqel and Vyndamax (median follow up 57.1 months p<0.001); 67 median survival was months; preliminary 5-year rate 53.2% in the continuous treatment arm vs. 32.4% placebo; 39% reduction risk of all-cause mortality patients with wild-type attr-cm (p="0.006);" 43% hereditary 44% baseline nyha class i or ii 5% iii> | 12/20/21 | Cardiovascular |
| Vifor Pharma Group, of St. Gallen, Switzerland | Veltassa (patiromer sorbitex calcium) | Potassium binder | Hyperkalemia | Diamond trial demonstrated a statistically significant difference vs. placebo for the primary endpoint to serum potassium levels in a high-risk population; detailed data expected in first half of 2022 | 12/21/21 | Cardiovascular |
| Amgen Inc., of Thousand Oaks, Calif. | Otezla (apremilast) | Oral, small-molecule PDE4 inhibitor | Moderate to severe psoriasis | Top-line data from Discreet trial showed 30-mg twice daily achieved clinically meaningful and statistically significant improvement compared with placebo in the primary endpoint of the modified static Physician's Global Assessment of Genitalia response; secondary endpoints also met with meaningful and significant improvements in Genital Psoriasis Itch Numerical Rating Scale response; consistent safety and tolerability | 12/1/21 | Dermatologic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | Psoriasis | Last of about 400 patients enrolled in Comfort study expected to complete 16-week cycle of treatment at beginning of January 2022; top-line resulted expected during first quarter of 2022 | 12/7/21 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Study met all primary and secondary endpoints testing lebrikizumab in combination with topical corticosteroids by week 16 | 12/21/21 | Dermatologic |
| Novartis AG, of Basel, Switzerland | Ligelizumab | High-affinity anti-IgE antibody | Chronic spontaneous urticaria | Top-line data showed phase III Pearl 1 and Pearl 2 studies met their primary endpoints of superiority vs. placebo at week 12 | 12/20/21 | Dermatologic |
| Akeso Inc., of Hong Kong, and Dawnrays Biotechnology Capital Ltd., of Hong Kong | Ebronucimab | Monoclonal antibody targeting PCSK9 | Primary hypercholesterolemia and mixed hyperlipidemia | Completed enrollment in the study being run in China earlier than expected | 12/23/21 | Endocrine/Metabolic |
| Idorsia Ltd., of Allschwil, Switzerland | Lucerastat | Glucosylceramide synthase inhibitor | Fabry disease | Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment; significant (p<0.0001) 6 difference in the change from baseline to month plasma gb3 between lucerastat and placebo; significant (p="0.02)" percent lysogb3 higher increase egfr was observed group vs. average decline was -2.75 mL/min/1.73m2 per year on treatment overall; decrease of high left ventricular mass index; well-tolerated; no clinically meaningful change in vital signs |
12/13/21 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Upadacitinib | JAK inhibitor | Moderate to severe Crohn’s disease | Top-line results from U-Exceed induction study showed drug achieved both primary endpoints of clinical remission and endoscopic response at week 12; significantly greater proportion of patients treated with 12-week induction regimen of 45 mg daily achieved clinical remission per Crohn's Disease Activity Index vs. placebo (39% vs. 21%; p<0.0001); 2 more patients in upadacitinib 45-mg group also achieved endoscopic response vs. placebo (35% 4%; p<0.0001); study enrolled who had inadequate or were intolerant to biologic therapy, with over 60% having previously failed biologics< td> | 12/6/21 | Gastrointestinal |
| Albireo Pharma Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | Results from 72-weeks of treatment showed reductions in mean serum bile acids (sBA) and pruritus; change in sBAs from baseline to weeks 49-72 was significantly correlated with change in pruritus scores during that interval (r=0.58; p<0.001); no serious drug-related treatment-emergent adverse events< td> | 12/13/21 | Gastrointestinal |
| Allakos Inc., of Redwood City, Calif. | Lirentelimab (AK-002) | Anti-Siglec-8 antibody | Eosinophilic gastritis and/or eosinophilic duodenitis | Data from 24-week Enigma 2 study in patients showed study met histologic co-primary endpoint but did not achieve statistical significance on the patient reported symptomatic co-primary endpoints | 12/21/21 | Gastrointestinal |
| Bausch Health Companies Inc., of Laval, Quebec | Relistor (methylnaltrexone bromide) | Mu-opioid receptor antagonist | Opioid-induced constipation | Post-hoc analysis of 2 phase III studies and 1 phase IV study published in The Journal of Emergency Medicine showed rescue-free laxation (RFL) occurred in 61.4% of patients treated with Relistor compared to 16% of patients treated with placebo 4 hours after the initial dose; after 24 hours, RFL occurred in 72.1% of patients taking Relistor compared to 40.1% of patients taking placebo | 12/22/21 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor (THR)-? selective agonist | Nonalcoholic fatty liver disease, presumed nonalcoholic steatohepatitis | Provided update on Maestro-NAFLD-1 study, citing unexpected staffing issues at vendor that would delay analysis of initial MRI-PDFF and lipid data planned for release by year-end 2021; top-line 52-week results now expected in January 2022 | 12/30/21 | Gastrointestinal |
| Zealand Pharma A/S, of Copenhagen | Glepaglutide | Long-acting GLP2 analogue | Short bowel syndrome | Enrolled last patient for interim analysis of pivotal study | 12/16/21 | Gastrointestinal |
| Eli Lilly and Co., of Indianapolis | Mirikizumab | Humanized IgG4 monoclonal antibody that binds to the p19 subunit of interleukin 23 | Moderately to severely active ulcerative colitis | Study met the primary endpoint and all key secondary endpoints (p<0.001) 1 at year; statistically higher proportion met the primary endpoint of clinical remission year compared to patients who were re-randomized placebo (p<0.001); achieved endoscopic remission, corticosteroid-free resolution or near-resolution bowel urgency, improvement in histologic intestinal inflammation; maintenance and greater reduction from baseline urgency symptoms placebo< td> | 12/14/21 | Gastrointestinal |
| Intercept Pharmaceuticals Inc., of New York | Ocaliva (obeticholic acid) | Farnesoid X receptor agonist | Compensated cirrhosis due to nonalcoholic steatohepatitis | Top-line data from phase III Reverse trial delayed; trial remains ongoing; results expected in first quarter of 2022 | 12/20/21 | Gastrointestinal |
| Centessa Pharmaceuticals plc of Boston | Lixivaptan | Vasopressin V2 antagonist | Autosomal dominant polycystic kidney disease | Initiated active recruitment in Action study | 12/14/21 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Linzagolix | Once-daily, oral GnRH receptor antagonist | Uterine fibroids | 52-week data showed reduction in heavy menstrual bleeding and other symptoms compared to placebo (p?0.003); reduced mean pain scores of 2-4 compared to <1 24 in the placebo groups (p?0.002); substantially decreased uterine volume by 40% at weeks; sustained safety profile< td> | 12/10/21 | Genitourinary/Sexual Function |
| Urovant Sciences Inc., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Gemtesa (vibegron) | Beta-3 adrenergic agonist | Overactive bladder | Results of 12-week data from Empowur study published in Advances in Therapy showed reductions in symptoms at 75 mg compared to placebo; ?15% reduction in micturitions (56.3% vs. 44.6%, respectively); ?50 % reduction in urgency episodes (39.5% vs. 32.8%), ?75 % reduction in UUI episodes (49.3% vs. 32.8%), and ?90% reduction in UUI episodes (35.2% vs. 23.5%) at week 12 (p<0.05 each)< td> | 12/20/21 | Genitourinary/Sexual Function |
| Rigel Pharmaceuticals Inc., of South San Francisco, and Kissei Pharmaceutical Co. Ltd., of Tokyo | Fostamatinib disodium hexahydrate | SYK inhibitor | Chronic immune thrombocytopenia | Top-line data showed study met its primary endpoint; achieved a stable platelet response significantly higher than patients receiving a placebo control; no new or unusual safety issues observed | 12/21/21 | Hematologic |
| Uniqure NV, of Amsterdam and CSL Behring, of Lexington,Mass. | Etranacogene dezaparvovec | AAV5 based gene therapy | Severe to moderately severe hemophilia B | Study achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression at 18 months; achieved secondary endpoint with statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy; stable and durable FIX levels with mean FIX activity of 36.9 % of normal in full study population at 18-months compared to a mean of 39% of normal at 6 months | 12/9/21 | Hematologic |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis in patients with systemic lupus erythematosus | Top-line data from Aurora 2 study showed mean estimated glomerular filtration rate (eGFR) was stable over 36 month in 116 subjects; well-tolerated with no unexpected safety signals; serious adverse events(19% voclosporin vs. 24% control); 4 deaths in control group and none in voclosporin; mean urine protein creatinine ratio lower in voclosporin-treated groups at all time points; sustained meaningful reductions in proteinuria compared to mycophenolate mofetil and low-dose oral corticosteroids alone; stable renal function | 12/9/21 | Immune |
| Catalyst Pharmaceuticals Inc., of Coral Gables, Fla., and Dydo Pharma Inc., of Osaka, Japan | Firdapse (amifampridine) | Potassium channel inhibitor | Lambert-Eaton myasthenic syndrome | Dydo initiated registrational study in Japan | 12/6/21 | Immune |
| Navidea Biopharmaceuticals Inc., of Dublin, Ohio | Tc99m Tilmanocept | Imaging agent | Moderate to severe active rheumatoid arthritis | Launched study for early prediction of anti-TNF-alpha therapy response | 12/17/21 | Immune |
| UCB SA, of Brussels | Rozanolixizumab | Humanized monoclonal antibody that specifically binds to human neonatal Fc receptor | Generalized myasthenia gravis | Top-line data from Mycaring study met its primary and secondary endpoint; statistically significant and clinically meaningful change from baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at day 43; well-tolerated and no new safety signals | 12/10/21 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Imlifidase | Immunoglobulin G-degrading enzyme from Streptococcus pyogenes | Kidney transplant rejection | Enrolled first patient | 12/29/21 | Immune |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Psoriatic arthritis | Results of Cosmos trial published in Annals of the Rheumatic Diseases showed significantly higher proportions of patients had improvement in joint signs and symptoms as well as 53.4% complete skin clearance vs. placebo at week 24; 44.4% of patients treated with Tremfya vs. 19.8 % of patients who received placebo achieved at least 20% improvement in the American College of Rheumatology criteria (ACR20) at week 24; higher ACR20 and ACR50 response rates vs. placebo as early as weeks 4 and 8, respectively; resolved enthesitis (39.7% vs. 18.8%) or dactylitis (44.8% vs. 25%) at week 24; achieved clinically meaningful improvements in physical function(37.5% vs. 16.1%) | 12/3/21 | Immune |
| Cidara Therapeutics Inc., of San Diego | Rezafungin | Echinocandin antifungal | Candidemia and invasive candidiasis | Top-line data met FDA and EMA-pre-specified primary endpoints vs. daily standard of care; | 12/14/21 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon Lambda | Type III interferon (IFN) that stimulates immune responses | COVID-19 | Data safety monitoring board conducted a second interim futility analysis and recommended continuation of the study based on analysis of 1,003 patients | 12/15/21 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | Hospitalized COVID-19 | Results from Live-Air phase III study published in The Lancet Respiratory Medicine achieved its primary endpoint of survival without ventilation measured through day 28 following treatment (HR: 1.54; 95%CI: 1.02-2.32, p=0.040); statistically significant 54% relative improvement in the likelihood of survival without the need for invasive mechanical ventilation | 12/1/21 | Infection |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Mavrilimumab | Monoclonal antibody targeting granulocyte macrophage colony stimulating factor receptor alpha | COVID-19-related acute respiratory syndrome | In the phase III portion of the phase II/III study, neither dose level of mavrilimumab improved the proportion of patients alive and free of mechanical ventilation at day 29 compared to placebo | 12/28/21 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide | Nonsteroidal antiandrogen | Mild to moderate COVID-19 | Statistical criteria were not met at the interim analysis of 348 patients; company plans to amend the protocol and continue to enroll only higher risk COVID-19 patients with multiple co-morbidities and/or patients who haven’t been vaccinated for COVID-19 | 12/27/21 | Infection |
| Medicago Inc., of Quebec City, and London-based Glaxosmithkline plc | CoVLP and pandemic adjuvant | Plant-derived vaccine plus an adjuvant | COVID-19 prophylaxis | Study met primary and secondary endpoints; overall vaccine efficacy rate against all variants of SARS-CoV-2 was 71%; no previous exposure to COVID-19 was 75.6%; efficacy of 75.3% against dominant delta variant; 88.6% against gamma variant;no related serious adverse events; well-tolerated | 12/7/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Islatravir | Nucleoside reverse transcriptase translocation inhibitor; oral | HIV-1 infection | Paused enrollment for IMPOWER 22 and IMPOWER 24 trials | 12/6/21 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics Inc., of Miami | Molnupiravir | Oral antiviral | COVID-19 | Findings published in The New England Journal of Medicine from Move-Out trial in non-hospitalized, high-risk adults with mild to moderate disease showed early treatment significantly reduced risk of hospitalization or death in unvaccinated adults | 12/16/21 | Infection |
| Novabiotics Ltd., of Aberdeen, U.K. | Cysteamine bitartrate (NM-002) | Immunomodulator antimicrobial | Community-acquired pneumonia | First patient enrolled | 12/8/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | VIP receptor agonist | Acute respiratory failure due to COVID-19 | Data safety monitoring board found no new safety concerns in 348 patients; recommended to continue enrollment | 12/14/21 | Infection |
| Quantum Leap Healthcare Collaborative, of San Francisco, and Implicit Bioscience Inc., of Seattle | IC-14 | CD14 monoclonal antibody | COVID-19 | IC-14 arm of the I-Spy COVID trial was terminated, due to high probability that drug would not have a large impact on either shortening time to recovery or mortality in critically ill patients; testing discontinued at recommendation of data monitoring committee | 12/2/21 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Talicia | Fixed-dose oral capsule combination of 2 antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor | Helicobacter pylori infection | Post-hoc analysis data from 269 patients from the Eradicate Hp and Eradicate Hp2 studies showed Talicia maintained high efficacy across all BMI groups compared to the active comparator including in obese and severely obese patients (P<0.0001); bmi between 30-40 kg m2 and those with>40kg/m treated with Talicia achieved eradication rates of 90% (88.1% and 90.9% respectively) vs. active comparator rates of 62.9% and 31.8% respectively | 12/2/21 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative that contains 2 donatable thiol groups; anti-inflammatory and antiviral oral agent | COVID-19 | Expanded phase III study against Omicron variant (B.1.1.529) | 12/3/21 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory and antiviral drug | Mild to moderate COVID-19 | Study screened about 700 patients to date, and independent data safety monitoring board supported continuation; in light of recent studies and FDA approval of oral antiviral treatments, company decided to fill part of patient enrollment quote outside U.S., targeting areas in Eastern Europe, such as Turkey | 12/29/21 | Infection |
| Sanofi SA, of Paris, and London-based Glaxosmithkline plc | Recombinant adjuvanted COVID-19 vaccine | Recombinant adjuvanted COVID-19 vaccine | COVID-19 | Preliminary results from VAT0002 study showed neutralizing antibodies increased 9- to 43-fold regardless of the vaccine received; booster was well-tolerated; safety profile similar to currently approved COVID-19 vaccines; data safety monitoring board recommended the trial to continue in early 2022 | 12/15/21 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Antifungal | Invasive candidiasis and/or candidemia | Started global study to test oral drug as step-down therapy for patients following intravenous echinocandin therapy in the hospital vs. currently available outpatient therapies; study to enroll about 220 patients, with primary endpoint of all-cause mortality at 30 days after initiation of antifungal therapy | 12/6/21 | Infection |
| Summit Therapeutics Inc., of Cambridge, Mass. | Ridinilazole | Small-molecule antibacterial agent | Clostridioides difficile infection | Top-line data showed ridinilazole resulted in a higher observed sustained clinical response rate than vancomycin; did not meet the primary endpoint for superiority; substantially less recurrence of C. diff. infection as compared to vancomycin (nominal p-value = 0.0002) | 12/20/21 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-620 (maribavir) | Serine/threonine protein kinase UL97 (viral) inhibitor | Post-transplant refractory cytomegalovirus infection with or without resistance | Results published in Clinical Infectious Diseases showed study met primary and secondary endpoint; 55.7% achieved confirmed CMV DNA level below the lower limit of quantification at week 8 compared to 23.9% of patients on conventional antiviral therapies P<0.001; 8 16 achieved cmv dna level | 12/8/21 |
Infection |
|
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon Lambda | Type III interferon | Chronic hepatitis delta virus infection | First patient enrolled in LIMT-2 study testing 48-week treatment; target enrollment of 150 patients | 12/21/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2372 | Recombinant nanoparticle protein-based COVID-19 vaccine with Matrix-M adjuvant | COVID-19 | First booster doses administered in extension of Prevent-19 pivotal trial to evaluate safety and efficacy of heterologous or homologous third dose | 12/21/21 | Infection |
| Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine | Chikungunya | Top-line results from lot-to-lot trial showed study met primary endpoint, demonstrating that 3 consecutively manufactured vaccine lots elicited equivalent immune responses measured by neutralizing antibody titer GMT ratios on day 29 after vaccination | 12/21/21 | Infection |
| Kolon Tissuegene Inc., of Rockville, Md. | TG-C | Allogeneic cell and gene therapy | Knee osteoarthritis | Restarted dosing in the 2 studies | 12/28/21 | Inflammatory |
| Kolon Tissuegene Inc., of Rockville, Md. | TG-C | Allogeneic cell and gene therapy | Knee osteoarthritis | Resumed patient dosing | 12/29/21 | Inflammatory |
| Selecta Biosciences Inc., of Watertown, Mass., and Swedish Orphan Biovitrum AB of Stockholm, Sweden | SEL-212 | Consisting of pegadricase, pegylated uricase, co-administered with ImmTOR | Chronic refractory gout | Completed enrollment in Dissolve I study | 12/1/21 | Inflammatory |
| Amo Pharma Ltd., of London | AMO-02 (tideglusib) | Disrupts pathogenic RNA repeat in CDM1 and inhibits excess levels of the kinase GSK3beta | Congenital myotonic dystrophy | Added clinical trial sites in Australia and New Zealand as part of the Reach-CDM study that has enrolled more than 50% of its target enrollment | 12/22/21 | Musculoskeletal |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | Kinase inhibitor | Advanced systemic mastocytosis | Data published in Nature Medicine from Explorer and Pathfinder trials showed overall response rates of 75% for both, median duration of response of 38 months for the Explorer study and overall survival of 76% and 86% estimated for each study, respectively; across both trials there were statistically significant improvements in patient-reported symptoms, as measured by the Advanced SM Symptom Assessment Form Total Symptom Score, and drug showed activity across all advanced SM subtypes | 12/7/21 | Musculoskeletal |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous MSC-NTF cells | Amyotrophic lateral sclerosis | Post-hoc analysis published in in Muscle and Nerve produced clinical benefits in patients with less severe disease; well-tolerated; those at ALSFRS-R ? 35 at baseline (31% of trial participants) showed 34.6% on Nurown achieved response vs. 15.6% on placebo (p=0.29); for those at ALSFRS-R >25 at baseline (77% of participants) 34.7% on Nurown achieved response vs. 20.5% on placebo (p=.053); statistically significant improvements from baseline were observed in cerebrospinal fluid biomarkers related to neuroinflammation, neurodegeneration and neuroprotection; no clinically significant differences in safety assessments observed between treatment groups | 12/13/21 | Musculoskeletal |
| Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | Edaravone/MT-1186 | Neuroprotectant | Amyotrophic lateral sclerosis | 24-week results from study (n=185) showed favorable safety profile; 5.9% subjects discontinued the study due to treatment emergent adverse events and 2 events related to drug; patients had an average ALSFRS-R score of 40 at the beginning and the average change from baseline in ALSFRS-R score was ?5.6 at week 24 | 12/9/21 | Musculoskeletal |
| Radius Health Inc., of Waltham, Mass., | Abaloparatide transdermal | PTH type 1 receptor agonist |
Postmenopausal women with osteoporosis | Wearable study did not meet its primary endpoint; demonstrated an increase of lumbar spine BMD by 7.1% vs. baseline; abaloparatide subcutaneous injection (Tymlos) demonstrated an increase of 10.9% vs. baseline; total hip and femoral neck BMD increased by an avg. of 2% and 1.9%; Tymlos showed 3.7% and 3.4%; treatment-emergent adverse events were similar in both groups; well tolerated with no significant safety signals identified | 12/8/21 | Musculoskeletal |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Active ankylosing spondylitis | Top-line interim analysis showed Be Mobile 2 study met primary endpoint, as measured by proportion of patients who achieved Assessment of SpondyloArthritis International Society 40 (ASAS40) response at week 16, when compared to placebo; study also met all ranked secondary endpoints, including significant improvements over placebo at week 16 in patient-reported disease activity, as measured by the Bath Ankylosing Spondylitis Disease Activity Index; achievement of ASAS partial-remission and Ankylosing Spondylitis Disease Activity Score Major Improvement; and nocturnal spinal pain score | 12/16/21 | Musculoskeletal |
| Acadia Pharmaceuticals Inc., of San Diego | Trofinetide | Synthetic analogue of amino?terminal tripeptide of IGF-1 | Rett syndrome | Top-line results from Lavender study showed statistically significant improvement over placebo for both co-primary endpoints; on the Rett Syndrome Behaviour Questionnaire, change from baseline to week 12 was -5.1 vs. -1.7 (p=0.0175; effect size=0.37); Clinical Global Impression-Improvement score at week 12 was 3.5 vs. 3.8 (p=0.0030; effect size=0.47); on secondary endpoint of Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist-Social composite score, change from baseline to week 12 was -0.1 vs. -1.1 (p=0.0064; effect size=0.43) | 12/6/21 | Neurology/Psychiatric |
| Acasti Pharma Inc., of Laval, Quebec | GTX-104 | Nanoparticle formulation of nimodipine; intravenous infusion | Subarachnoid hemorrhage | Interim analysis meets all primary endpoints in 20 of 50 normal healthy subjects; achieved comparable bioavailability with oral nimodipine in the full study cohort of 50 subjects; no serious adverse events observed; only mild adverse events | 12/2/21 | Neurology/Psychiatric |
| Biogen Inc. and Sage Therapeutics Inc., both of Cambridge, Mass. | Zuranolone (SAGE-217/BIIB-125 | Oral neuroactive steroid; GABA-A receptor positive allosteric modulator | Major depressive disorder | Results from 12-month data for the cohort of patients (n=199) at 50-mg was well-tolerated and no new safety findings; rapid and sustained improvements in depressive symptoms; HAMD-17 mean change from baseline was -16.0 ± 6.04 (n=185) at day 15; 74.9% patients achieved response and 40.2% achieved remission | 12/1/21 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Intranasal zavegepant | Small-molecule CGRP receptor antagonist | Acute migraine | Top-line results from second pivotal trial showed drug achieved co-primary regulatory endpoints of pain freedom and freedom from most bothersome symptom at 2 hours and demonstrated statistically significant superiority to placebo across 15 prespecified primary and secondary outcome measures | 12/6/21 | Neurology/Psychiatric |
| Bioxcel Therapeutics Inc., of New Have, Conn. | BXCL-501 | Orally dissolving thin film formulation of dexmedetomidine | Agitation | Initiated phase III study to enroll a total of 300 patients | 12/15/21 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores the normal shape and function of altered filamin A protein | Mild to moderate Alzheimer’s disease | Launched a website to support the study that is being run at over 25 sites | 12/23/21 | Neurology/Psychiatric |
| Harmony Biosciences Holdings Inc., of Plymouth meeting, Pa. | Pitolisant | Selective histamine 3 receptor antagonist/inverse agonist | Excessive daytime sleepiness and cataplexy | Post-hoc analysis from 61 patients from Harmony 1 and 105 patients from Harmony CTP showed significantly greater mean change in weekly rate of cataplexy observed at week 2 for Harmony CTP and week 5 for Harmony 1; percentage of treatment responders was significantly greater with pitolisant vs. placebo beginning at week 3 for EDS and week 2 for cataplexy | 12/13/21 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor | Chorea associated with Huntington disease | Study met primary endpoint of reduction in severity of chorea; valbenazine resulted in placebo-adjusted mean reduction in the TMC score of 3.2 units (p < 0.0001); statistically significant in secondary endpoints; no suicidal behavior or worsening of suicidal ideation; consistent treatment emergent adverse events | 12/7/21 | Neurology/Psychiatric |
| Pharma Two B Ltd., of Rehovot, Israel | P2B-001 | Fixed-dose combination of rasagiline mesilate and pramipexole hydrochloride | Early Parkinson's disease | Study met its primary and key secondary endpoints; superior to the pramipexole component by 2.66 points (p=0.0018) and superior to the rasagiline component by 3.30 points (p=0.0001); comparable efficacy to a marketed pramipexole ER with significantly less daytime sleepiness (somnolence); reduction of 2.66 points (p<0.0001) as measured by epworth sleepiness scale< td> | 12/15/21 | Neurology/Psychiatric |
| Sage Therapeutics Inc., and Biogen Inc. both of Cambridge, Mass. | Zuranolone (SAGE-217/BIIB-125 | Oral neuroactive steroid; GABA-A receptor positive allosteric modulator | Major depressive disorder | Waterfall study at 50-mg demonstrated rapid improvements in depressive and anxiety symptoms; average improvements maintained through the end of the study (day 42); well-tolerated in a subgroup of patients aged 65 years and older in Shoreline study | 12/8/21 | Neurology/Psychiatric |
| Scilex Holdings Inc., of Mountain View, Calif., and Sorrento Therapeutics Inc., of San Diego | SP-102, Semdexa | Dexamethasone sodium phosphate viscous gel | Lumbosacral radicular pain or sciatica | Top-line data from Clear trial met primary and key secondary efficacy endpoints with highly statistical significance; average daily pain in the affected leg over 4 weeks following the initial injection had demonstrated lumbosacral spine (LS) compared to placebo with a p-value <0.001; 4 ls mean (se) group difference in odi compared to placebo at week p-value <0.001; significantly longer duration of initial treatment clean safety profile with no identified risks; adverse events< td> | 12/9/21 | Neurology/Psychiatric |
| SK Life Science Inc., of Paramus, N.J. | Xcopri (cenobamate) | Positive allosteric modulator of the ?-aminobutyric acid (GABAA) ion channel; inhibits voltage-gated sodium currents | Focal seizures | Post-hoc analysis (n=240) in patients with partial-onset seizures showed early onset responder rates and high rates of sustained seizure reductions across a variety of seizure types for as long as 43 months (median 30.2 months); seizure reductions of 50% occurred in 56% in focal aware motor (FAM), 51% in focal impaired awareness (FIA), and 70% of focal to bilateral tonic-clonic (FBTC) at 12-week phase; complete seizure reduction occurred in 48% of patients with FAM seizures, 54% of patients with FIA seizures and 91% of patients with FBTC seizures at 24-27 months | 12/3/21 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Small-molecule immune-modulating covalent inhibitor of RASP | Dry eye disease | Tranquility study did not meet primary endpoint; statistical significance (p=0.0001) achieved, sign of Schirmer test; statistical significance (p<0.0001) 300 400 was also achieved for the post-hoc assessment of schirmer test responders ?10 mm; target enrollment increased from to patients; top-line results expected in mid-2022< td> | 12/20/21 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 (ND4) gene | Leber hereditary optic atrophy | Sustained efficacy after 2 years of injection showed statistically significant visual acuity improvement from baseline and nadir in treated eyes; p=0.0006 (13 ETDRS letters) and p=0.01 (+9 ETDRS letters) for bilaterally injected in 2 affected eyes; p=0.02 (+8 ETDRS letters) and p=0.1 (+5 ETDRS letters) for unilaterally injected in 2 affected eyes; favorable safety profile; no study discontinuation related to systemic or ocular adverse events; no serious events | 12/14/21 | Ocular |
| Kodiak Sciences Inc., of Palo Alto, Calif. | KSI-301 | Anti-VEGF antibody biopolymer conjugate | Macular edema due to retinal vein occlusion | Completed enrollment in Beacon trial | 12/16/21 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating prostaglandin analogue | Glaucoma | First patient screened in China in ongoing Denali trial enrolling patients in China and U.S.; results expected by end of 2023 | 12/16/21 | Ocular |
| Novaliq GmbH, of Heidelberg, Germany, and Jiangsu Hengrui Pharmaceuticals Co. Ltd., of Shanghai | SHR-8058 | Eye drops; perfluorohexyloctane | Dry eye disease | Completed trial, enrolling 312 patients in China; met prespecified primary sign endpoint improvement of total corneal fluorescein staining and prespecified primary symptom endpoint improvement of eye dryness score of Visual Analogue Scale at 8 weeks with high statistical significance | 12/16/21 | Ocular |
| Palatin Technologies Inc., of Cranbury, N.J. | PL-9643 | Melanocortin agonist | Dry eye disease | Started the Melody-1 study of up to 400 patients; top-line results expected in the second half of 2022 | 12/28/21 | Ocular |
| Novaliq GmbH, of Heidelberg, Germany | Cyclasol | Topical anti-inflammatory and immunomodulating ophthalmic solution containing 0.1% cyclosporine A | Dry eye disease | Essence-2 study showed superior effects over vehicle on primary sign endpoint, improvement of total corneal fluorescein staining (tCFS) at day 29 (p= 0.0278); 71.6% of patients responded within 4 weeks with a clinically meaningful improvement of ?3 grades in total corneal staining; proportion of responders was significantly higher compared to vehicle-treated patients (p=0.0002); statistically significant improvements in a variety of symptoms compared to non-responders at day 29 | 12/21/21 | Ocular |
| Aeglea Biotherapeutics Inc., of Austin, Texas | Pegzilarginase | Recombinant human enzyme | Arginase 1 deficiency | Pivotal Peace study met primary endpoint with statistically significant reduction in plasma arginine from baseline after 24 weeks; sustained plasma arginine reduction accompanied by positive trend in Gross Motor Function Measure Part E, a key clinical assessment of a patient's mobility; Aeglea plans to submit BLA to FDA in first half of 2022 | 12/6/21 | Other/Miscellaneous |
| Albireo Pharma Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Alagille Syndrome | Study expected to enroll 63 patients to evaluate the safety and efficacy of Bylvay for 24 weeks in relieving pruritus in patients | 12/13/21 | Other/Miscellaneous |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Human monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13 proteins | Uncontrolled moderate-to-severe asthma | Results published in The New England Journal of Medicine showed signficant reduction in severe asthma attacks in children aged 6 to 11 years; rapidly improved lung function; consistent known safety profile | 12/8/21 | Respiratory |
| Beyondspring Inc., of New York | Plinabulin | Selective immunomodulating microtubule-binding agent; antigen presenting cell inducer | Chemotherapy-induced neutropenia | Results from protective-2 study in combination with pegfilgrastim showed patients experienced less bone pain vs. pegfilgrastim only (p=0.03); higher absolute neutrophil count (ANC) at the nadir; treatment-emergent adverse events of bone pain (p=0.03); statistically significantly correlated with bone pain scores (p=0.019); decreased toxicity and improved health-related quality of life; significant change in EQ-5D-5L utility values (p=0.024) | 12/10/21 | Cancer |
| Vertex Pharmaceuticals Inc., of Boston | VX-147 | Oral small molecule APOL1 inhibitor | APOL1-mediated focal segmental glomerulosclerosis | Study achieved a statistically significant, substantial and clinically meaningful mean reduction of 47.6% in urine protein to creatinine ratio (UPCR) at week 13 compared to baseline; well-tolerated; no treatment discontinuations due to adverse events; no adverse events | 12/1/21 | Genitourinary/Sexual Function |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor | Selective Inhibitor of Nuclear Export compound | Myelofibrosis | Dosed first patient in study testing oral drug as monotherapy vs. physician’s choice in patients previously treated with JAK1/2 inhibitor; primary endpoint is percentage of patients with spleen volume reduction of ?35% from baseline | 12/6/21 | Hematologic |
| Vifor Pharma AG, of St. Gallen, Switzerland | Vamifeport (VIT-2763) | Oral ferroportin inhibitor | Sickle cell disease | First patient enrolled; top-line data expected at the end of 2022 | 12/10/21 | Hematologic |
| Acelyrin Inc., of Los Angeles, Affibody AB, of Solna, Sweden, and Inmagene Biopharmaceuticals Co. Ltd., of Shanghai | Izokibep | IL-17A-inhibiting antibody mimetic | Psoriatic arthritis | Results from 16-week phase II study met its primary endpoint; no new safety issues | 12/14/21 | Immune |
| Lynk Pharmaceuticals Co Ltd., of China | LNK-01001 | Selective kinase inhibitor | Rheumatoid arthritis | First patient dosed | 12/1/21 | Immune |
| Navidea Biopharmaceuticals Inc., of Dublin, Ohio | Tc99m tilmanocept | Imaging agent | Rheumatoid arthritis | Achieved full enrollment in phase IIb trial to establish healthy subject database | 12/15/21 | Immune |
| 60 Degrees Pharmaceuticals LLC, of Washington | Arakoda (tafenoquine) | 8-aminoquinoline chemically derived from primaquine | Mild to moderate COVID-19 | Preliminary data showed decreased proportion of patients not recovered by 27% in the intent-to-treat population at day 14, and by 47% in the per protocol population; drug-related adverse events in 2.4% of placebo patients vs. 8.7% of tafenoquine patients; study was terminated early and, therefore, underpowered to show statistical significance on primary endpoint | 12/15/21 | Infection |
| Acurx Pharmaceuticals Inc., of Staten, Island, N.Y. | Ibezapolstat | Antibiotic | C. difficile infection | Enrolled first patient in phase IIb trial testing drug against standard-of-care vancomycin; 64-patient study expected to complete in mid-2022 | 12/6/21 | Infection |
| Akston Biosciences Inc., of Beverly, Mass. | AKS-452 | Vaccine containing the receptor binding domain of SARS-CoV2 | COVID-19 prophylaxis | Seroconversion response rate at day 56 was 100% for 2 doses of 45 ?g and 96% for 1 dose of 90 ?g; plans to start a phase II/III study in India | 12/22/21 | Infection |
| Antios Therapeutics Inc., of Mendham, N.J. | ATI-2173, AB-729 and Tenofovir disoproxil fumarate | Active site polymerase inhibitor; RNAi therapeutics; nucleotide reverse transcriptase inhibitor | Chronic hepatitis B virus infection | First patient dosed | 12/14/21 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | rNAPc2 (AB-201) | Small recombinant protein; selective inhibitor of tissue factor | Hospitalized severe COVID-19 | Completed enrollment of 160 patients; top-line data in the 1Q of 2022 | 12/2/21 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | COVID-19 | Initiated CATCO trial in 65 hospital in Canada | 12/1/21 | Infection |
| Axcella Inc., of Cambridge, Mass. | AXA-1125 | Multitargeted EMM composition consisting of 5 amino acids and an amino acid derivative | Long COVID | Patient screening underway in phase IIa trial; primary endpoint is change from baseline to day 28 in phosphocreatine recovery time as measured by 31-phosphorus magnetic resonance spectroscopy | 12/16/21 | Infection |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | Capsid-virus-like particle vaccine | COVID-19 | Top-line results from 103-patient trial in those previously vaccinated with mRNA or adenoviral COVID-19 vaccines who received single booster with ABNCoV2 showed, at 1 week post vaccination, a 2- to 34-fold increase in levels of neutralizing antibodies against the Wuhan variant, which peaked at 2 weeks with 2- to 40-fold increase depending on initial antibody levels; all subjects, irrespective very low or high neutralizing titers initially, were boosted to absolute antibody levels reported to be associated with >90% efficacy against SARS-CoV2 | 12/6/21 | Infection |
| Bonus Biogroup Ltd., Israel | Mesencure | Allogeneic adipose tissue-derived mesenchymal stromal cells | Severe COVID-19 | Initial analysis showed treatment reduced severe patients mortality by 70% compared to control group; shortened the hospitalization period of severe; reduction of 45% relative to the control group; average hospitalization period for severely ill patients treated with Mesencure measured 9.4 days compared to 17.2 days for the control group; 94% survival rate at the end of a 30-day follow-up period for the 50 hospitalized patients | 12/2/21 | Infection |
| Brii Biosciences Ltd., of Durham, N.C. | BRII-179 (VBI-2601) | Virus-like particle vaccine | Chronic hepatitis B virus infection | Dosed first patient in the study testing BRII-179 plus PEG-IFN-alpha and nucleos(t)ide reverse transcriptase inhibitor standard of care; primary endpoint of the 120-patient phase IIa portion is the percentage of patients with HBsAg loss at the completion of treatment; the primary endpoint of the 480-patient phase IIb portion is the percentage of patients achieving sustained HBsAg and HBV DNA loss | 12/27/21 | Infection |
| Capricor Therapeutics Inc., of Los Angeles | CAP-1002 | Allogeneic cardiac-derived cell therapy | Severe COVID-19 | Trial completed enrollment and randomization of 63 patients; top-line data expected in first quarter 2022 | 12/13/21 | Infection |
| Cyxone AB, of Malmo, Sweden | Rabeximod | B-220 analogue | COVID-19 | Top-line results from trial in moderate disease showed almost all patients alive and free of respiratory failure at day 28 in different treatment arms; no statistical difference between treatment arms could be observed at day 28, so endpoint assessment did not reveal if rabeximod has additional benefits over standard of care at day 28; secondary and exploratory endpoints will be analyzed | 12/6/21 | Infection |
| Fortress Biotech Inc. and Helocyte Inc., both of New York | Triplex | Cytomegalovirus vaccine | Cytomegalovirus | Initiated trial to evaluate safety and efficacy of vaccine in eliciting a CMV-specific immune response and reducing CMV replication in people living with HIV | 12/16/21 | Infection |
| Geovax Labs Inc., of Atlanta | COH-04S1 | Multi-antigenic SARS-CoV-2 investigational vaccine to target both the spike and nucleocapsid proteins | COVID-19 | Initiated vaccine dosing in phase II portion | 12/15/21 | Infection |
| Iliad Biotechnologies LLC, of Weston, Fla. | BPZE-1 | Live attenuated intranasal pertussis vaccine | Pertussis | First children enrolled; study to assess the immunological response and safety profile of a single dose with and without co-administration of Boostrix | 12/17/21 | Infection |
| Janssen Pharmaceutical Cos., of Johnson & Johnson, of Raritan, N.J. | Respiratory syncytial virus adult vaccine | Vaccine | Respiratory syncytial virus | Data from phase IIb Cypress study showed candidate highly effective in protecting against three clinical definitions of lower respiratory tract disease caused by RSV, demonstrating efficacy of 70%-80% in adults ages 65 and older | 12/7/21 | Infection |
| Johnson & Johnson, of New Brunswick, N.J. | COVID-19 vaccine (Ad26.COV2.S) | SARS-CoV-2 recombinant adenoviral vector vaccine | COVID-19 | Results from COV2008 study showed booster shot administered 6 months after 2-dose primary regimen of BNT-162b2 (Pfizer Inc./Biontech SE) increased both antibody and T-cell responses | 12/6/21 | Infection |
| Matinas Biopharma Holdings Inc., of Bedminster, N.J. | MAT-2203 | Amphotericin B | Cryptococcal meningitis | Independent data and safety monitoring board completed prespecified review of third cohort of Enact trial and unanimously recommended progression to fourth and final cohort; enrollment in next randomized cohort, with 40 active-treatment patients, expected to begin early first quarter 2022 | 12/16/21 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Anti-spike IgG titers after dose 3 increased 61.1-fold (prototype) and 73.5-fold (Omicron) after dose 3 boost; increase was 5.4-fold (prototype) to 9.3-fold (Omicron) from peak responses seen after 2-dose primary vaccination; ACE2-inhibition titers increased 54.4-fold (prototype), 24.4-fold (Delta) and 36.3-fold (Omicron); increase was 6-fold (prototype) to 19.9-fold (Omicron) compared to peak responses following 2-dose primary series | 12/22/21 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Brilife vaccine | Viral vector vaccine | COVID-19 | Independent data safety monitoring board concluded safety analysis; based on input received, Nrx is proceeding with plans to initiate phase IIb/III registration trial | 12/6/21 | Infection |
| Ocugen Inc., of Malvern, Pa., and Bharat Biotech International Ltd., of Hyderabad, India | Covaxin | Whole-virion inactivated COVID-19 vaccine manufactured using a vero cell platform | COVID-19 | Results from phase II/III trial in children, ages 2-18, posted on Medrxiv, showed 2-dose regimen administered 28 days apart resulted in antibody responses comparable to adult data from previous phase III study demonstrating greater than 93% reduction in severe disease | 12/30/21 | Infection |
| Pfizer Inc., of New York | Paxlovid (PF-07321332; ritonavir) | 3CL protease inhibitor specifically designed to combat SARS-CoV-2 | Mild-to-moderate COVID-19 | Final results from EPIC-HR study (n=2246) showed 89% reduction (3 days of symptoms onset) and 88% (within 5 days of symptom onset) in COVID-19-related hospitalization or death from any cause compared to placebo; statistical significance of these results was high (p<0.0001); no deaths compared to placebo< td> | 12/14/21 | Infection |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD | Allogeneic mesenchymal-like cells | Acute respiratory distress syndrome associated with COVID-19 | Data from 89 patients in 2 phase II studies showed the number of ventilator-free days through day 28 didn’t improve for PLX-PAD compared to placebo; in the U.S. study survival for the 300M PLX-PAD cell dose was 50% compared to 35% for placebo; in the EU study, survival was 60% for PLX-PAD compared to 50% for placebo | 12/27/21 | Infection |
| Pulmotect Inc., of Houston | PUL-042 | Synergistic combination of 2 Toll-like receptor agonists | Severity of COVID-19 | Study showed single dose was well-tolerated though 28 days; cleared virus by day 15 compared to none of the 3 placebo treated subjects; no statistically significant difference between treatment groups in the primary endpoint of change in ordinal scale for clinical improvement; significant difference in prospectively defined subgroup of males (p=0.04); statistically significant difference in time to improvement of respiratory symptoms (p=0.0227) | 12/1/21 | Infection |
| Revelation Biosciences Inc., of San Diego | REVTx-99 | Intranasal drop formulation; TLR4 agonist | H3N2 influenza (influenza A) infection | Site initiation for phase IIB study to evaluate the efficacy of intranasal in healthy humans; enrolled patients 18 to 55 years old | 12/1/21 | Infection |
| SAB Biotherapeutics Inc., of Sioux Falls, S.D. | SAB-176 | Multivalent neutralizing fully human polyclonal IgG antibody targeting type A and type B influenza viruses | Influenza virus infection | Top-line data from 60 healthy adults challenged with a pandemic influenza virus strain (pH1N1) showed study met its primary endpoint; safe and well-tolerated; statistically significant (p = 0.026) reductions in nasopharyngeal viral load, clinical signs and symptoms compared to placebo; achieved statistical significance in the secondary endpoint as reduction of clinical flu signs and symptoms at day 4 (p = 0.013, one sided) in symptomatic patients; no serious adverse events | 12/1/21 | Infection |
| Vir Biotechnology Inc., of San Francisco | VIR-2218 | HBV-targeting siRNA | Chronic hepatitis B virus infection | First patient dosed in combination with Selgantolimod (Gilead Sciences Inc.) and Opdivo (nivolumab, Bristol Myers Squibb Co.) | 12/9/21 | Infection |
| Eupraxia Pharmaceuticals Inc., of Victoria, British, Columbia | EP-104IAR | Encapsulated fluticasone propionate within a microns-thin polymer membrane | Osteoarthritis | Expanded clinical trial locations; top-line data in fourth quarter of 2022 | 12/2/21 | Inflammatory |
| 180 Life Sciences Corp., of Palo Alto, Calif. | Adalimumab | Anti-TNF biologic | Dupuytren’s disease | Top-line data met primary endpoint of nodule hardness and secondary end point of nodule size on ultrasound scan with statistically significant differences; no related severe adverse events | 12/1/21 | Musculoskeletal |
| Akeso Inc., of Hong Kong | Gumokimab (AK-111) | Monoclonal antibody targeting IL-17A | Active ankylosing spondylitis | Completed enrollment in the study | 12/27/21 | Musculoskeletal |
| Clene Inc., of Salt Lake City | CNM-Au8 | Catalytically active gold nanocrystal suspension | Amyotrophic lateral sclerosis | Data from Rescue-ALS phase II study in limb and bulbar onset participants showed consistent clinical benefit for the pre-specified endpoint; consistent clinical benefit in the proportion without a 6-point decline on the ALSFRS-R in post-hoc analysis; improved average CAFS summated score of +4.4 compared to -4.6 decline for the placebo group (p=0.22) | 12/1/21 | Musculoskeletal |
| Revalesio Inc., of Tacoma, Wash. | RNS-60 | Activates intracellular signaling pathways to increase mitochondrial biogenesis and function and reduce inflammation | Amyotrophic lateral sclerosis | Data from 147-patient study showed no impact on candidate biomarkers or ALSFRS-R, though there was statistically significant slowing of decline in lung function, as measured by FVC, in patients receiving RNS-60; well-tolerated, with overall safety profile similar to placebo | 12/7/21 | Musculoskeletal |
| Virios Therapeutics Inc., of Atlanta | IMC-1 (famciclovir + celecoxib) | Dual COX-2/COX-1 inhibitor | Fibromyalgia | More than 50% of the patients in in Fortress phase IIb study randomized | 12/1/21 | Musculoskeletal |
| ZZ Biotech Inc., of Houston | 3K3A-APC | Vitamin K dependent protein C stimulator | Amyotrophic lateral sclerosis | First patients dosed; study will enroll 16 patients into 2 dose cohorts; primary outcomes are safety and tolerability and reduction in pathological changes thought to cause ALS | 12/2/21 | Musculoskeletal |
| Clene Inc., of Salt Lake City | CNM-Au8 | Catalytically active gold nanocrystal suspension | Amyotrophic lateral sclerosis | Data from Rescue-ALS phase II study showed significant reduction in risk of disease progression (p=0.0125); decreased risk of 6-point ALSFRS-R decline (p=0.035); improvement in quality of life; primary endpoint of percent change in MUNIX score from baseline to week 36 was not met; 45% relative reduction in MUNIX score decline (p=0.0741); well-tolerated and safe; no drug-related serious adverse events or drug discontinuations | 12/10/21 | Musculoskeletal |
| Apnimed Inc., of Cambridge, Mass. | AD-109 | Combines selective norepinephrine reuptake inhibitor atomoxetine with new chemical entity aroxybutynin, a selective antimuscarinic | Obstructive sleep apnea | Dosed first patient in phase IIb Mariposa study | 12/16/21 | Neurology/Psychiatric |
| Aptinyx Inc., of Evanston, Ill. | NYX-783 | Oral, positive allosteric modulator of NMDA receptors | Post-traumatic stress disorder | Initiated phase IIb study in 300 patients; data anticipated in second half of 2023 | 12/16/21 | Neurology/Psychiatric |
| Atai Life Sciences AG, of New York | RL-007 | Targets the cholinergic, NMDA and GABA type B receptor systems | Cognitive impairment associated with schizophrenia | Top-line data showed dose-related improvements on exploratory cognitive endpoints; dose dependent response in qEEG; increase in amplitude in the alpha band (up to 17% increase in normalized; alpha-slow wave index (up to 21% increase); well-tolerated | 12/14/21 | Neurology/Psychiatric |
| Compass Pathways plc, of London | COMP-360 | Psilocybin therapy | Treatment-resistant depression | Post-hoc analysis of 19 sustained responders at 25-mg group found changes in quality of life, self-reported depression severity and functioning; clinically meaningful with mean scores and maintained to 12 weeks | 12/1/21 | Neurology/Psychiatric |
| Douglas Pharmaceuticals Ltd., of Auckland, New Zealand | R-107 | Ketamine 30, 60, 120 or 180 mg | Treatment-resistant depression | Top-line data showed rapid-acting antidepressant effect and sustained efficacy over 12 weeks vs. placebo; 73% of patients remitted (MADRS score ?12) within 1 week from open-label treatment of 120 mg daily during enrichment phase; clear dose-response pattern identified, with 180-mg dose group showing statistically significant mean reduction of 6.1 points in MADRS score at end of study vs. placebo (p=0.019) | 12/16/21 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | Suvecaltamide (JZP385) | Selective modulator of T-type calcium channels | Essential tremor | First patient enrolled | 12/15/21 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | JZP-150 | Small-molecule inhibitor of enzyme fatty acid amide hydrolase | Post-traumatic stress disorder | Enrolled first patient; study to enroll 270 adults, ages 18-70, and will measure changes from study start to end of treatment using Clinical-Administered PTSC Scale (CAPS-5) | 12/30/21 | Neurology/Psychiatric |
| Luye Pharma Group Ltd., of Yantai, China | LY-03005 | Serotonin-norepinephrine-dopamine reuptake inhibitor | Major depressive disorder | At all 4 dose levels tested the drug improved 17-item Hamilton Depression Rating Scale total score compared to placebo after 6 weeks of treatment; all 4 dose levels improved the Montgomery-Åsberg Depression Rating Scale score and the CGI-I score after the 6-week treatment compared to placebo; 40 mg, 80 mg and 160 mg improved the CGI-S score compared to placebo (p<0.05)< td> | 12/23/21 | Neurology/Psychiatric |
| Mind Medicine Inc., of New York | Lysergic acid diethylamide | Psychedelic | Attention deficit hyperactivity disorder | Initiated phase IIa proof-of-concept trial in adults | 12/17/21 | Neurology/Psychiatric |
| Novaremed AG, of Basel, Switzerland | NRD.E1 | Non-opioid development compound | Diabetic peripheral neuropathy | Imitated NIH-supported phase IIb trial as Serendipity-1; study to start in second quarter of 2022 | 12/21/21 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of New York | REL-1017 | NMDA receptor channel blocker | Major depressive disorder | Data from the 62-patient study published in the American Journal of Psychiatry showed Montgomery-Åsberg Depression Rating Scale score improved for both dose levels compared to placebo on day 4, which was sustained through the last dose on day 7 and day 14 (7 days after last dose) (p?0.0308, effect size 0.7 to 1) | 12/22/21 | Neurology/Psychiatric |
| Sorrento Therapeutics Inc., of San Diego | Resiniferatoxin | Target TRPV1 receptors | Knee pain from osteoarthritis | Began enrollment | 12/7/21 | Neurology/Psychiatric |
| Xenon Pharmaceuticals Inc., of Burnaby, British Columbia | XEN-1101 | Potassium channel modulator | Focal epilepsy | New subanalyses from phase IIb X-Tole trial showed 29.5% of patients in 25-mg group, 29.4% in 20-mg group, 8.7% in 10-mg group and 6.1% in placebo group reached at least 75% reduction in monthly focal seizure frequency from baseline; 6.3% in the 25-mg group, 7.8% in the 20-mg group, 2.2% in the 10-mg group and 1.8% in the placebo group showed 100% reduction in monthly focal seizure frequency from baseline | 12/6/21 | Neurology/Psychiatric |
| Ocular Therapeutics Inc., of Bedford, Mass. | OTX-DED (dexamethasone intracanalicular ophthalmic insert) | Glucocorticoid receptor agonist | Dry eye disease | Top-line results showed 166-subject trial achieved prespecified primary endpoint, demonstrating statistically significant change of bulbar conjunctival hyperemia from baseline to day 15 compared to vehicle hydrogel using a central reading photographic assessment in the modified intent-to-treat (ITT) population; change from baseline using the CCLRU Grading scale (0-4) was -0.51 for the OTX-DED 0.2-mg group (p=0.004), -0.43 for the OTX-DED 0.3-mg group (p=0.028) and -0.21 for vehicle hydrogel insert group; both formulations were observed to have favorable safety profile and were generally well-tolerated | 12/6/21 | Ocular |
| Ribomic Inc., of Tokyo | RBM-007 | Oligonucleotide-based aptamer targeting FGF2 | Wet age-related macular degeneration | In the Tofu study of 86 patients who were previously treated with anti-VEGF drugs, neither RBM-007 monotherapy nor RBM-007 plus Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) improved vision compared to Eylea monotherapy | 12/28/21 | Ocular |
| Proqr Therapeutics NV, of Leiden, the Netherlands | QR-421a | RNA therapy | USH2A mediated retinitis pigmentosa and Usher syndrome | First patients dosed in phase II/III Sirius and Celeste trials; Sirius will enroll 81 participants with advanced vision loss (baseline best corrected visual acuity (BCVA) of worse than 20/40), with primary endpoint of mean change from baseline in BCVA at 18 months; Celeste will enroll 120 participants with early to moderate vision loss (baseline BCVA of equal or better than 20/40), with primary endpoint of mean change from baseline in static perimetry at 12 months | 12/16/21 | Ocular/Other/miscellaneous |
| Applied Therapeutics Inc., of New York | AT-007 | CNS-penetrant aldose reductase inhibitor | Sorbitol dehydrogenase deficiency | Initiated registrational phase II/III study; primary biomarker efficacy endpoint will measure reduction in sorbitol at 3 months of treatment compared to baseline, while primary clinical outcome measure will assess changes in Charcot-Marie-Tooth Functional Outcome Measures lower limb function domain | 12/16/21 | Other/miscellaneous |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor | Alagille syndrome and progressive familial intrahepatic cholestasis with BSEP deficiency | Post-hoc analysis at week 48 from 20 patients met itch-reported outcome; improved health-related quality of life; multidimensional fatigue total scale score from baseline to week 48 (increase of 13.9 points) more than 2 times the minimal clinically important difference in responders; 6 sleep-related items demonstrated significantly larger changes from baseline to week 48 in responders | 12/13/21 | Other/miscellaneous |
| NFL Biosciences SA, of France | NFL-101 | Natural nicotine-free product, extracted from tobacco leaves | Smoking cessation | Launched CESTO phase II/III study | 12/14/21 | Other/Miscellaneous |
| Saniona AB, of Copenhagen, Denmark | Tesomet | Fixed-dose combination therapy of tesofensine and metoprolol | Prader-Willi syndrome | Initiated the clinical trial | 12/29/21 | Other/miscellaneous |
| Bellus Health Inc., of Laval, Quebec | BLU-5937 | Selective P2X3 antagonist | Refractory chronic cough | Top-line results from phase IIb soothe trial (n=249) achieved statistical significance on the primary endpoint with 34% placebo-adjusted reduction in 24-hour cough frequency observed (p ? 0.005) at day 28; well-tolerated; low rates of taste-related adverse events reported (?6.5%) at all doses; no treatment-emergent serious adverse events | 12/13/21 | Respiratory |
| Sterna Biologicals GmbH & Co. KG, of Marburg, Germany | SB-010 | Inhaled liquid formulation of a special type of catalytic antisense oligonucleotide | Moderate-to-severe type-2 driven asthma | First patient dosed | 12/14/21 | Respiratory |