January | February | March | April | May | June | July | August | September | October | November | December |
| Company | Product | Description | Indication | Phase II status | Date | Therapeutic area |
| Akeso Inc., of Hong Kong | Ivonescimab (AK-112) | PD-1/VEGF bispecific antibody | Locally advanced or metastatic nonsquamous non-small-cell lung cancer | First patient dosed | 1/31/22 | Cancer |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-40 | Fatty acid synthase inhibitor | Recurrent glioblastoma | First patient dosed in combination with bevacizumab | 1/23/22 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab (BGB-A317) | Humanized IgG4 anti-PD-1 monoclonal antibody designed to minimize binding to Fc?R on macrophage | Locally advanced, unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma | Interim results from Rationale305 study in combination with chemotherapy met the primary endpoint of overall survival in patients with PD-L1 expression; no new safety signals | 1/24/22 | Cancer |
| Checkpoint Therapeutics Inc., of New York | Cosibelimab | PD-L1 inhibitor | Cutaneous squamous cell carcinoma | The 78-patient study met the primary endpoint with 47.4% objective response rate; median duration of response has not yet been reached; BLA on track for submission by end of year | 1/25/22 | Cancer |
| Cstone Pharmaceuticals Ltd., of Suzhou, China | Cejemly (sugemalimab) | Anti-PD-L1 monoclonal antibody | Unresectable locally advanced, recurrent, or metastatic esophageal squamous cell carcinoma | Completed patient enrollment in GEMSTONE-304 study in combination with standard of care chemotherapy | 1/18/22 | Cancer |
| Cstone Pharmaceuticals Ltd., of Suzhou, China, and Eqrx Inc., of Cambridge, Mass. | Cejemly (sugemalimab) | Anti-PD-L1 monoclonal antibody | Metastatic (stage IV) non-small-cell lung cancer | Results from Gemstone-302 study met overall survival (OS) endpoint; sugemalimab in combination with chemotherapy showed statistically significant and clinically meaningful OS improvement in patients | 1/18/22 | Cancer |
| Deciphera Pharmaceuticals LLC, of Waltham, Mass. | Qinlock (ripretinib) | Switch-control tyrosine kinase inhibitor | Advanced gastrointestinal stromal tumor | Presented results of Intrigue study evaluating the efficacy and safety of ripretinib vs sunitinib; in patients with a KIT exon 11 primary mutation, ripretinib produced a median progression-free survival of 8.3 months compared to 7.0 months for the sunitinib (p=0.36); objective response rate was 23.9% for the 163 patients treated with ripretinib compared to 14.6% for the 164 patients treated with sunitinib (p=0.03) | 1/24/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Locally advanced or metastatic clear cell or non-clear cell (papillary or unclassified only) renal cell carcinoma | Completed enrollment in Contact-03 study in combination with atezolizumab vs. cabozantinib alone in patients | 1/5/22 | Cancer |
| ITM Isotope Technologies Munich SE, of Garching, Germany | ITM-11/n.c.a. 177Lu-edotreotide | High-quality radioisotope, no-carrier-added lutetium-177 chelated to the edotreotide | Somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors | First patient treated in the 202-patient Compose study comparing ITM-11 to investigator's choice of chemotherapy (CAPTEM or FOLFOX) or everolimus; primary endpoint is progression-free survival; secondary endpoint includes overall survival up to 2 years after disease progression | 1/25/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Stage Ib-IIIa non-small-cell lung cancer | Interim results from KEYNOTE-091 study met 1 of its dual primary endpoints of disease-free survival (DFS) at 200 mg intravenously every 3 weeks; resulted in statistically significant and clinically meaningful improvement in DFS compared with placebo; consistent safety profile | 1/10/22 | 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 endometrial cancer | Results from Study 309/Keynote-775 published in the New England Journal of Medicine demonstrated statistically significant improvements in the dual primary endpoints of overall survival and progression-free survival compared to chemotherapy | 1/20/22 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Product composed of functionalized hafnium oxide nanoparticles | Head and neck cancer | First patient randomized in Nanoray-312 study | 1/5/22 | Cancer |
| On Target Laboratories Inc., of West Lafayette, Ind. | Cytalux (pafolacianine) | Imaging agent targeting folate receptors | Thoracic surgery for confirmed or suspected cancer in the lung | The Elucidate study met its primary endpoint; data will be shared in the future | 1/13/22 | Cancer |
| Oncopeptides AB, of Stockholm | Pepaxto (melphalan flufenamide) aka melflufen | DNA alkylating agent; peptide-drug conjugate that targets aminopeptidases | Lenalidomide refractory patients with relapsed refractory multiple myeloma after 2-4 lines of prior therapy | Data from the 495-patient Ocean study published in Lancet Hematology showed melflufen plus dexamethasone produced a median progression-free survival of 6.8 months, compared to 4.9 months for pomalidomide plus dexamethasone (HR=0.79, p=0.03); overall response rate was 33% for melflufen and 27% for pomalidomide | 1/13/22 | Cancer |
| Zai Lab Ltd., of Shanghai | Tumor Treating Fields | Electric fields that penetrate the cancer cell membrane | Pancreatic cancer | First patient treated in greater China in PANOVA-3 study | 1/12/22 | Cancer |
| Chiesi USA Inc., of Cary, N.C., affiliate of Chiesi Farmaceutici SpA, of Parma, Italy | Kengreal (cangrelor) | P2Y12 platelet inhibitor | Percutaneous coronary intervention | Published results from the Champion Phoenix trial in Circulation: Cardiovascular Interventions that showed Engreal significantly decreased the primary composite endpoint of death, myocardial infarction, ischemia-driven revascularization or stent thrombosis by 25% compared with clopidogrel (p=0.002) | 1/25/22 | Cardiovascular |
| George Medicines Pty Ltd., of London | GMRx2 | Single pill, triple component combination of telmisartan, amlodipine and indapamide | Hypertension | Around 500 patients expected to enroll in U.K. | 1/17/22 | Cardiovascular |
| Lianbio Co. Ltd., of Shanghai | Mavacamten | Oral, allosteric modulator of cardiac myosin | Symptomatic obstructive hypertrophic cardiomyopathy | First patient dosed | 1/10/22 | Cardiovascular |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Axial spondyloarthritis | Top-line interim data from Mobile 1 study met the primary and all ranked secondary endpoints; statistically significant and clinically meaningful improvement over placebo in the proportion of patients who achieved the ASAS40 response at week 16; achieved significant improvements over placebo at week 16 in the signs and symptoms of disease as measured by BASDAI | 1/18/22 | Cardiovascular |
| Cara Therapeutics Inc., of Stamford, Conn., and Maruishi Pharmaceutical Co. Ltd., of Japan | Difelikefalin (Korsuva) | Kappa opioid receptor agonist | Pruritus | Study achieved primary and secondary endpoint; significantly improved from baseline compared to the placebo group; well-tolerated | 1/10/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | JAK Inhibitor | Alopecia areata | Completed patient enrollment in THRIVE-AA2, part 2 of phase III study; top-line data expected in third quarter of 2022 | 1/4/22 | Dermatologic |
| Inflarx NV, of Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Hidradenitis suppurativa | Initiated phase III study in patients | 1/5/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Pyrilutamide (KX-826) | Androgen receptor antagonist | Androgenetic alopecia | First patient dosed | 1/2/22 | Dermatologic |
| Lipidor AB, of Stockholm | AKP-02 | Cutaneous spray formulation of calcipotriol and betamethasone dipropionate (0.005%/0.05%) | Mild to moderate psoriasis | First patient enrolled; results expected in 2022 Q2 | 1/26/22 | 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 data from Prime Study met primary and key secondary endpoints at week 24; clinically meaningful reduction in itch from baseline (p<0.0001), at 60% vs. 18% for placebo; achieved clear or almost skin, a secondary endpoint (p="0.0004)," 48% greater improvements in measures of overall health-related quality life, skin pain, and symptoms anxiety depression; treatment-emergent adverse events were 71% dupixent 63% placebo< td> | 1/19/22 | Dermatologic |
| Amryt Pharma plc., of Dublin, Ireland | Mycapssa (octreotide) | Somatostatin derivative | Acromegaly | Results from 9-month randomized control treatment Mpowered trial published in Lancet Diabetes & Endocrinology showed 91% of patients on Mycapssa maintained response compared to 100% on iSRLs; mean IGF-1 remained same at the start and end of trial in normal limits for both arms; 15% of patients in the oral octreotide reported breakthrough symptoms of acromegaly compared with 31% in the iSRL group; significant reductions were shown in the proportions of participants with swelling of extremities (p=0.01) and fatigue (p=0.03); comparable safety profile to iSRLs | 1/4/22 | Endocrine/Metabolic |
| Amryt Pharma plc., of Dublin, Ireland | Mycapssa (octreotide) | Somatostatin derivative | Acromegaly | Long-term data from Mpowered trial showed 93%, 91% and 92% of patients completed year 1, 2 and 3, respectively; IGF-1 levels stably maintained within the normal limits; 47% reported excellent or very good symptomatic control; 58% had at least three active acromegaly symptoms on long-acting injectable somatostatin receptor ligands at the end of the RTC, decreasing to 37% at the end of the OLE after switching to Mycapssa;median treatment duration of 2.2 years and a maximal exposure of 3.5 years; median compliance rate of 99% over this period of time; statistically significant improvement in treatment satisfaction and convenience; no new safety signals with long-term exposure | 1/11/22 | Endocrine/Metabolic |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-APOC3 | RNAi candidate targeting apolipoprotein C-III (APOC3) | Familial chylomicronemia syndrome | First patient dosed in Palisade study | 1/12/22 | Endocrine/Metabolic |
| Daewoong Pharmaceutical Co. Ltd., of Seoul, South Korea | Enavogliflozin | SGLT-2 inhibitor | Type II diabetes | The 160-patient study met the primary endpoint of baseline change of glycated hemoglobin (HbA1c) with a difference of 0.99 percentage points between the enavogliflozin group and the placebo group (p<0.001); plans to submit new drug approval and launch enavogliflozin as monotherpay metformin fixed-dose-combination by 2023< td> | 1/25/22 | Endocrine/Metabolic |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Obesity due to biallelic or heterozygous POMC, PCSK1 or LEPR genetic variants or a clinical diagnosis of Bardet-Biedl syndrome with genetic confirmation | Treated first patient in the switching study comparing the weekly formulation of setmelanotide to the once-daily setmelanotide in 30 patients who were previously enrolled in Rhythm’s long-term, open-label daily setmelanotide extension trial; primary endpoint is the proportion of patients with weight gain of 5% or less from baseline to week 13 | 1/13/22 | Endocrine/Metabolic |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor | Heterozygous familial hypercholesterolemia and/or established atherosclerotic cardiovascular disease | First patient dosed; planning to complete enrollment in 2022 and data in first quarter of 2024 | 1/4/22 | Endocrine/Metabolic/Cardiovascular |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor (THR)-beta selective agonist | Nonalcoholic fatty liver disease | Top-line data from Maestro-NAFLD-1 study (n=972) achieved primary and key secondary endpoints; resmetirom was safe and well-tolerated at 80 mg and 100 mg; significant reduction in liver fat as measured by MRI-PDFF (p<0.0001) and reduced atherogenic lipids, including ldlc (p<0.0001), apolipoprotein b (p<0.0001) triglycerides< td> | 1/31/22 | Gastrointestinal |
| Obseva SA, of Geneva | Linzagolix | GnRH antagonist | Moderate to severe endometriosis-associated pain | Top-line results of Edelweiss study at 200 mg once-daily met co-primary efficacy objectives; reductions in dysmenorrhea and non-menstrual pelvic pain (NMPP) at 3 months; statistically significant and clinically meaningful improvements in the first 5 ranked secondary endpoints at 6 months; 75-mg dose without add-back therapy (ABT) showed statistically significant reduction vs. placebo in dysmenorrhea at 3 months; improvement in NMPP at 3 months but did not reach statistical significance vs. placebo; improvements in first 5 ranked secondary endpoints in combination with ABT dose; well-tolerated with minimal BMD decrease and few adverse events | 1/6/22 | Genitourinary/Sexual Function |
| Belief Biomed Inc., of Shanghai | BBM-H901 | AAVvector expressing factor IX gene | Hemophilia B | First patient dosed | 1/3/22 | Hematologic |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Oral factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | First patient enrolled in Redeem-1 pivotal trial | 1/7/22 | Hematologic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | Adeno-associated virus vector encoding human coagulation factor VIII | Hemophilia A | Results from ongoing global phase III GENEr8-1 study showed annualized bleeding rate was significantly reduced by 4.1 treated bleeds per year (p<0.0001); 133 significantly reduced the mean annualized factor viii infusion rate in rollover population by infusions per year (p<0.0001); well-tolerated; no new safety signals emerged; treatment-related serious adverse events < td> | 1/9/22 | Hematologic |
| Sierra Oncology Inc., of San Mateo, Calif. | Momelotinib | JAK1/JAK2 inhibitor; ACVR1/ALK2 inhibitor | Myelofibrosis | In the 195-patient Momentum study, 25% of patients taking momelotinib had a Total Symptom Score of at least 50% compared to 9% of patients taking danazol (p=0.0095), 31% of patients taking momelotinib were transfusion independent compared to 20% of patients taking danazol (p=0.0064); 23% of patients taking momelotinib had a splenic response rate of at least 35 compared to 3% of patients taking danazol (p=0.0006); plans to submit NDA in second quarter of 2022 | 1/25/22 | Hematologic |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Olumiant (baricitinib) | Oral JAK inhibitor | Systemic lupus erythematosus | Discontinued clinical program based on Brave-I and II studies; Brave-I study met the primary endpoint at 4-mg oral dose; statistically significant reduction in disease activity as measured by the proportion of adults with active lupus who achieved an SRI-4 response at week 52 vs. placebo; Brave-II study did not meet the primary endpoint of SRI-4 response nor secondary endpoints | 1/28/22 | Immune |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Active psoriatic arthritis | Top-line phase III Be complete study met its primary endpoint; significantly more patients treated with bimekizumab achieved 50% or greater improvement in signs and symptoms of disease from baseline vs. placebo at week 16; met all ranked secondary endpoints; significant improvements over placebo at week 16 in physical function | 1/21/22 | Immune |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland | ceftobiprole | Cephalosporin antibiotics | Staphylococcus aureus bacteremia | Completed patient enrollment; 390 patients enrolled in Eradicate study | 1/11/22 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | COVID-19 | Results from LIVE-AIR study published in medRxiv showed patients with baseline CRP<150 mg l who received lenzilumab had a more than 2.5-fold higher likelihood to survive without invasive mechanical ventilation patients placebo (p<0.001)< td> | 1/3/22 | Infection |
| Infant Bacterial Therapeutics AB, of Stockholm | IBP-9414 | Oral drug containing Lactobacillus reuteri | Necrotizing enterocolitis | Data monitoring committtee completed pre-scheduled safety analysis without any concerns; futility analysis performed; continuing recruitment | 1/19/22 | 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 data from B7471026 study in 570 adults co-administered Pfizer-Biontech COVID-19 vaccine or placebo showed similar responses for Prevnar 20; responses to booster dose of COVID-19 Vaccine were also similar when given with Prevnar 20 or placebo (n=189); safety profile of co-administration with booster dose generally reflected that observed with Pfizer-Biontech COVID-19vVaccine booster dose | 1/12/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Pfizer-BioNTech COVID-19 vaccine | Omicron-based vaccine | COVID-19 prophylaxis | First participants enrolled in clinical trial to evaluate the safety, tolerability and immunogenicity of the omicron-based vaccine candidate in healthy adults 18 through 55 years of age; participants divided into 3 cohorts: those that received 2 doses of the original vaccine who will receive 1 or 2 doses of the new vaccine, those who received 3 doses of the original vaccine who will receive 1 dose of the new vaccine or 1 dose of the old vaccine and vaccine-naïve participants who will receive 3 doses of the new vaccine | 1/25/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory and antiviral drug | Mild to moderate COVID-19 | 700 subjects enrolled and will complete enrollment in first quarter of 2022 | 1/6/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory and antiviral drug | Mild to moderate COVID-19 | Total of 701 subjects dosed; study to evaluate the safety and efficacy of oral use | 1/18/22 | Infection |
| Seres Therapeutics Inc., of Cambridge, Mass. | SER-109 | Oral microbiome therapeutic candidate consisting of a consortium of highly purified Firmicutes spores | Recurrent C. difficile infection | Results published in the New England Journal of Medicine from Ecospor phase III study achieved statistically significant and clinically meaningful improvements in key primary and secondary efficacy measures compared with placebo; 88% of SER-109 patients achieved a sustained clinical response compared to 60% on placebo; 27% absolute reduction of recurrence of CDI compared to placebo at eight weeks post-treatment; well-tolerated; no serious drug-related adverse events | 1/20/22 | Infection |
| Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine | Chikungunya virus infection | Initiated phase III study; 750 adolescents aged 12 to 17 years will be randomized at a 2:1 ratio to receive either VLA-1553 or placebo; to evaluate safety and immunogenicity following a single vaccination; subjects will be evaluated after 28 days and followed up to 12 months | 1/31/22 | Infection |
| Zealand Pharma A/S, of Copenhagen | Glepaglutide | Long-acting GLP2 analogue | Short bowel syndrome | Completed patient enrollment; results expected in third quarter of 2022; trial sample size reduced to 108 patients from the original sample size of 129 patients | 1/20/22 | Infection |
| Puretech Health plc, of Boston | LYT-100 (deupirfenidone) | Deuterated form of pirfenidone | Inflammation and fibrosis | Results from randomized, double-blind crossover study in healthy older adults showed clinically meaningful 50% reduction in GI-related adverse events compared to pirfenidone (17.4% vs. 34%); no serious adverse events; 1 AE-related discontinuation in each arm | 1/6/22 | Inflammatory |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Implantable matrix based upon Lyra’s Xtreo platform designed to elute mometasone furoate | Chronic rhinosinusitis | Initiated phase III Enlighten study for enrollment; planning to enroll 180 patients | 1/24/22 | Inflammatory |
| 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 | Long-term Restore study showed best-corrected visual acuity (BCVA) after 4 years; 71% of subjects achieved clinically relevant recovery (CRR) against nadir 4 years after treatment; 80.7% had on-chart vision (BCVA ? 1.6 LogMAR) in 1 or both eyes; well-tolerated; no serious adverse events; no discontinuations occurred due to ocular events | 1/24/22 | Neurology/Psychiatric |
| Idorsia Ltd., of Allschwil, Switzerland | Daridorexant | Dual orexin receptor antagonist | Insomnia | Results published in The Lancet Neurology showed improved sleep outcomes at 25-mg and 50-mg doses; improved daytime functioning; favorable safety profile; statistically significant improvements in the primary endpoints of sleep onset and maintenance as well as the secondary endpoints of total sleep time and daytime sleepiness; overall incidence of adverse events was comparable between treatment groups | 1/20/22 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | Xywav (calcium, magnesium, potassium and sodium oxybates) oral solution | GABA B receptor agonist; formulation of gamma-hydroxybutyrate containing a mixture of calcium, magnesium, potassium and sodium salts | Idiopathic hypersomnia | Results published in The Lancet Neurology showed clinically meaningful and statistically significant differences with Xywav compared to placebo in primary endpoint of change in the Epworth Sleepiness Scale (ESS) score (p<0.0001) and key secondary endpoints; ihss scores decreased statistically significant (p<0.0001); clinically meaningful differences compared to placebo on the patient global impression of change functional outcomes sleep questionnaire no deaths during trial< td> | 1/5/22 | Neurology/Psychiatric |
| Mesoblast Ltd., of New York | Rexlemestrocel-L (MPC-06-ID) | Allogeneic mesenchymal precursor cells | Chronic low back pain associated with degenerative disc disease | Results from 36-month follow-up from the 404-patient trial showed durable reduction in pain; 28% who received rexlemestrocel-L + hyaluronic acid were not taking an opioid compared with 8% of saline-treated controls (p= 0.0075) | 1/11/22 | Neurology/Psychiatric |
| Orchard Therapeutics Inc., of Boston and London | Libmeldy (atidarsagene autotemcel, OTL-200) | Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene | Early-onset metachromatic leukodystrophy | Results published in The Lancet showed Libmeldy was well-tolerated; no evidence of abnormal clonal proliferation or replication-competent lentivirus over the follow-up period; no treatment-related mortality or serious adverse events; total gross motor function measure scores significantly improved compared to the natural history cohort in both late-infantile (p<0.0001) 2 3 and early juvenile patients (p="0.036);" normal cognitive development; prevention or delay of central peripheral demyelination brain atrophy throughout follow-up; reconstituted arsa activity in blood mononuclear cells within above range from months post-treatment onward with levels significantly increased baseline at years post-treatment; 90% alive median follow-up 3.2 years< td> | 1/21/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-2191 | Intravitreal methotrexate 0.8% | Proliferative vitreoretinopathy | Completed enrollment in part 1 of Guard trial | 1/4/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Night vision disturbances | Completed enrollment of 140 subjects in LYNX-1 study; top-line data in early 2022 | 1/5/22 | Ocular |
| Meiragtx Holdings plc, of London, and Johnson & Johnson, of New Brunswick, N.J. | Botaretigene sparoparvovec | Gene therapy | X-linked retinitis pigmentosa | Initiated patient dosing in phase III Lumeos study | 1/27/22 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | NAV AAV8 vector encoding antibody fragment designed to inhibit VEGF | Wet age-related macular degeneration | Initiated second 2 phase III trials; Ascent trial will evaluate the efficacy and safety of subretinal delivery in patients | 1/10/22 | Ocular |
| Verona Pharma plc, of London | Ensifentrine | Bronchodilator with anti-inflammatory activities | Chronic obstructive pulmonary disease | Completed patient enrollment with more than 800 patients in Enhance-2 study; top-line data expected in third quarter of 2022 | 1/24/22 | Respiratory |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Smoking cessation | Last subject completed last study follow-up visit in ORCA-2 phase III trial ; top-line results expected in second quarter of 2022 | 1/4/22 | Toxicity and Intoxication |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Smoking cessation | Started screening for the Orca-3 registrational trial designed to test the efficacy and safety of 3 mg cytisinicline dosed 3 times daily for 6 weeks or for 12 weeks compared to placebo in 750 adult smokers; primary outcome is biochemically verified continuous abstinence during the last four weeks of treatment | 1/25/22 | Toxicity and Intoxication |
| Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate targeting HER2 | HER2-low unresectable and/or metastatic breast cancer | Results from Destiny-Breast04 phase III trial showed statistically significant and clinically meaningful improvement in both progression-free survival and overall survival in patients; consistent safety profile with previous clinical studies; no new safety concerns | 2/21/22 | Cancer |
| Bayer AG, of Leverkusen, Germany, and Orion Corp., of Espoo, Finland | Nubeqa (darolutamide) | Androgen receptor inhibitor | Metastatic hormone-sensitive prostate cancer | Results from Arasens study published in the New England Journal of Medicine in combination with plus androgen deprivation therapy and docetaxel met primary and secondary endpoints; statistically significant increase in overall survival (OS) with a reduction in the risk of death by 32.5% compared to ADT plus docetaxel (p<0.001); statistically significant benefits across multiple secondary endpoints compared to adt plus docetaxel, including delaying the time castration-resistant prostate cancer (crpc) (p<0.001), pain progression (p="0.01)," first symptomatic skeletal event and initiation of subsequent systemic antineoplastic therapy (p<0.001); treatment-emergent adverse events similar in both arms< td> | 2/17/22 | Cancer |
| Biofrontera Inc., of Woburn, Mass. | Ameluz | Aminolevulinic acid hydrochloride gel | Superficial basal cell carcinoma | Around 186 patients enrolled in combination study with photodynamic therapy; completion of patient recruitment anticipated by the end of 2022 | 2/1/22 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor | Oral exportin 1 (XPO1) inhibitor | Endometrial cancer | Top-line data showed SIENDO/ENGOT-EN5/GOG-3055 study met its primary endpoint of a statistically significant improvement in median PFS compared to placebo; median PFS of 5.7 months compared to 3.8 months for patients on placebo (p=0.0486); 30% reduction in the risk of disease progression or death; statistically significant improvement in median PFS (HR=0.38, p=0.0006) in patients with wild-type p53, 62% reduction in the risk of disease progression or death; well-tolerated with no new safety signals; low discontinuation rate of 10.5% due to adverse events | 2/8/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1 inhibitor | Triple-negative breast cancer | Keynote-522 study results published in The New England Journal of Medicine showed that neoadjuvant Keytruda in combination with chemotherapy followed by adjuvant Keytruda as monotherapy prolonged event-free survival by 37% compared to chemotherapy plus placebo; after 39 months of follow up, 15.7% of 784 patients who received Keytruda had an event compared to 23.8% of 390 patients who received placebo | 2/9/22 | Cancer |
| Merck KgaA, of Darmstadt, Germany | Bavencio (avelumab) | Human anti- PD-L1 antibody | Locally advanced or metastatic urothelial carcinoma | Exploratory analysis from Javelin Bladder 100 study plus best supportive care at 38 months median follow-up showed consistent overall survival (OS) benefit over patients on BSC alone; median OS was 23.8 months vs 15 months ; 43.7% of patients were alive at 30 months vs 33.5% of patients who received BSC alone; median OS was 30.9 months vs 18.5 months in population of patients with PD-L1+ tumors | 2/18/22 | Cancer |
| Renovorx Inc., of San Jose, Calif. | RenovoTAMP | Renovorx trans-arterial micro-perfusion | Locally advanced pancreatic cancer | First patient enrolled in TIGeR-PaC study | 2/22/22 | Cancer |
| Simcere Pharmaceutical Group, of Hong kong | Trilaciclib | Myeloprotective effect | Extensive-stage-small-cell-lung cancer | Study met its primary endpoint, demonstrating a significant decrease in duration of severe neutropenia of patients receiving chemotherapy in Cycle 1 | 2/24/22 | 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 | Initiated phase III Envision study; planning to enroll 220 patients | 2/3/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Mavacamten | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Top-line results of phase III study met primary endpoint at week 16; consistent safety profile with previous studies | 2/16/22 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Hypertrophic cardiomyopathy | Top-line data from cohort 3 Redwood-HCM study showed substantial reductions in the average resting left ventricular outflow tract gradient (LVOT-G below 50%) as well as the post-Valsalva LVOT-G; clinically relevant decrease in pressure gradients were achieved with modest decreases in average left ventricular ejection fraction; similar pharmacokinetic data in cohort 1 and 2; no treatment interruptions and no serious adverse events | 2/1/22 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Small-molecule cardiac myosin activator | Heart failure with reduced ejection fraction | In the Meteoric-HF trial, omecamtiv mecarbil showed no effect on change in peak oxygen uptake on cardiopulmonary exercise testing after 20 weeks of treatment compared to placebo | 2/15/22 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Aficamten | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Sequoia-HCM study open to enrollment | 2/23/22 | Cardiovascular |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream (ARQ-151) | Once-daily topical formulation of PDE4 inhibitor | Seborrheic dermatitis | Enrollment of last subject in Stratum study; total of 457 subjects enrolled; top-line data expected in mid-year 2022 | 2/1/22 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Monoclonal antibody targeting IL-4 and IL-13 | Urticaria | Pre-specified interim analysis of CUPID study B will be stopped due to futility; not meet statistical significance conducted by an independent review committee | 2/18/22 | Dermatologic |
| Innovent Biologics Inc., of San Francisco | IBI-306 | Anti-PCSK9 antibody | Hypercholesterolemia | Study met primary endpoint; low-density lipoprotein cholesterol (LDL-C) levels were significantly reduced compared with the placebo arm in Credit-1 study (p<0.0001 48 at 600-mg dose) week and also in credit-4 study (p<0.0001 450-mg 12; good safety profile both studies< td> | 2/16/22 | 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 | Third patient treated; enrollment of 4th patient is ongoing | 2/7/22 | Endocrine/Metabolic |
| Sanofi SA, of Paris | Olipudase alfa | Enzyme replacement that breaks down sphingomyelin | Acid sphingomyelinase deficiency (ASMD) | In the long-term extension Ascend study, olipudase alfa produced a mean increase improvement of 28.5% in the percent predicted diffusing capacity of the lung for carbon monoxide (Dlco) after 2 years compared to 22.2% after year 1; spleen volume decreased by a mean of 47% after 2 years compared to 39.5% after 1 year; liver volume decreased by a mean of 33.4% after 2 years compared to 27.8% after 1 year; for patients who crossed over from placebo after 1 year, at the end of 1 year on treatment, percent predicted DLco mean increase was 28%, spleen volume mean decrease was 36% and liver volume mean decrease was 30.7% | 2/9/22 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | Dasiglucagon | Glucagon analogue | Congenital hyperinsulinism | Completed patient enrollment in second phase III trial, 17103, in neonates up to 12 months old; results expected in second quarter of 2022 | 2/15/22 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Moderate to severe Crohn's disease | Top-line data showed study achieved both primary endpoints of clinical remission p<0.0001 12 45 and endoscopic response p<0.0001 vs. placebo at week mg; significantly higher proportion of upadacitinib-treated patients achieved steroid-free clinical remission placebo; no new safety risks observed< td> | 2/24/22 | Gastrointestinal |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Moderately to severely active ulcerative colitis | Interim results True North open-label extension study achieved clinical remission (45%), clinical response (86%), endoscopic improvement and corticosteroid-free remission was maintained through week 142; no new safety signals | 2/17/22 | 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 | Results from Lucent-1 study of mirikizumab achieved statistically superior rates of clinical remission at 12 weeks compared to placebo (p=0.00006); achieved statistically significant improvements across key secondary endpoints including clinical response (p<0.00001), symptomatic (p<0.001) compared to placebo; rapid improvement in their symptoms (p<0.001); statistically significant reduction on an 11-point bowel urgency severity scale; average of 2.59 points, 1.63 (1.18 2.09) points for patients placebo (p<0.00001)< td> | 2/18/22 | Gastrointestinal |
| Statera Biopharma Inc., of Fort Collins, Colo. | STAT-201 | Naltrexone (low-dose oral formulation) | Pediatric Crohn’s disease | Received central institutional review board approval for study, to enroll patients ages 12-17 | 2/15/22 | Gastrointestinal |
| Vanda Pharmaceuticals Inc., of Washington, D.C. | Tradipitant | Neurokinin-1 receptor antagonist | Gastroparesis | Study did not meet its prespecified primary endpoint; both treatment arms showed significant improvements from baseline on nausea as well as the other core symptoms; safe and well-tolerated | 2/4/22 | Gastrointestinal |
| Centessa Pharmaceuticals plc, of Boston | Lixivaptan | Vasopressin V2 antagonist | Autosomal dominant polycystic kidney disease | First subject dosed; expected to enroll 1,350 subjects | 2/24/22 | Genitourinary/Sexual Function |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | Adeno-associated virus vector encoding human coagulation factor VIII | Hemophilia A | Results from 2-year analysis of phase III GENEr8-1 study showed mean annualized factor VIII infusion rate was reduced by 133 infusions per year (p<0.0001) 0 1 or 98% from baseline; 95% of participants remain off factor viii prophylactic therapy; percentage with treated bleeds increased 32% on prophylaxis at baseline to 82% during year and 84% 2< td> | 2/4/22 | Hematologic |
| CSL Behring, of King of Prussia, Pa. | Etranacogene dezaparvovec | AAV5-based gene therapy | Hemophilia B | Long-term results of phase III Hope-B study showed single infusion had stable and durable increase in mean Factor IX (FIX) activity and hemostatic protection at 18 months; mean FIX activity of 39 IU/dL at 6 months and 36.9 IU/dL at 18 months post infusion; adjusted annualized bleeding rate for all bleeds was reduced by 64% (p=0.0002) and all FIX-treated bleeds was reduced by 77 %(p<0.0001) 7 97 98 over months to 18; % of subjects treated with a full dose discontinued use prophylaxis an overall reduction in mean unadjusted annualized fix consumption 257338.8 iu yr participant 8486.6 participant< td> | 2/4/22 | Hematologic |
| Orpha Labs AG, of Baar, Switzerland | ORL-101 | pharmaceutical-grade L-fucose | Leukocyte adhesion deficiency type II | Initiated phase III study | 2/23/22 | Hematologic |
| TG Therapeutics Inc., of New York | Ublituximab | Glycoengineered anti-CD20 monoclonal antibody | Relapsing forms of multiple sclerosis | Pooled post-hoc analysis of Ultimate I and II studies showed significant reductions in both the volume and number of new T1 hypointense lesions compared to teriflunomide at 96 weeks P<0.0001; 96 neutralizing antibodies and antidrug was 2.4% 17.8% at baseline 6.4% 86.5% any time postbaseline, respectively this reduced to 1.1% 16.5% week 96; transient no observable impact on b-cell depletion; decrease from in the mean number of cd19+ b cells (96.2% reduction) remained consistent through (97.6% reduction)< td> | 2/25/22 | Immune |
| UCB SA, of Brussels | Zilucoplan | Self-administered, subcutaneous peptide inhibitor of complement component 5 | Generalized myasthenia gravis | Top-line data showed study met primary and secondary endpoint; clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at week 12 was observed for the zilucoplan treatment group vs placebo; statistically significant improvements from baseline in Quantitative Myasthenia Gravis score, Myasthenia Gravis Composite score and MG-QoL15r score at week 12 for the zilucoplan treatment group vs placebo; well-tolerated and no major unexpected safety findings | 2/4/22 | Immune |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody designed to inhibit TLR4 | COVID-19 | Data showed more than 25% of the subjects have been randomized to date under the phase III design approved by Health Canada | 2/17/22 | Infection |
| Glaxosmithkline plc, of London | RSV MAT vaccine | Recombinant protein vaccine | Respiratory syncytial virus infection | Grace study voluntarily paused enrolment and vaccination and also in pregnant women | 2/18/22 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide | Nonsteroidal antiandrogen | COVID-19 outpatients | First patient dosed | 2/11/22 | Infection |
| Laurent Pharmaceuticals Inc., of Montreal | LAU-7b | COVID-19 oral antiviral | COVID-19 | Dosed first patient in phase III expansion of Resolution phase II/III trial in hospitalized moderate to severe patients at high risk of developing lung complications | 2/15/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1345 | Vaccine consisting of mRNA encoding for a stabilized RSV prefusion F glycoprotein, encapsulated in lipid nanoparticles; single-dose vaccine | Respiratory syncytial virus | Data and safety monitoring board endorsed the start of the phase III portion of ConquerRSV study acceptable safety profile in older adults at the selected dose | 2/22/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373/Nuvaxovid | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Study achieved its primary efficacy endpoint in pediatric patients; overall protective efficacy of 79.5% against COVID-19; 82% against delta variant; immune responses were about 2-to-3-fold higher in adolescents than in adults against all variants; well-tolerated with no safety signals identified | 2/10/22 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | Synthetic form of vasoactive intestinal polypeptide | COVID-19 | ACTIV-3b study showed no new safety concerns in review of more than 448 patients; study cleared by the independent data safety monitoring board to continue enrollment to 640 patients; trial to commence enrollment in Brazil, EU, U.K. and Scandinavia in the coming months | 2/15/22 | Infection |
| Pop Biotechnologies Inc., of Buffalo, N.Y., and Eubiologics Co. Ltd., of Seoul, South Korea | Eucorvac-19 | Protein-based vaccine | COVID-19 prophylaxis | Approval of phase III plan from Ministry of Food and Drug Safety in South Korea | 2/6/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory and antiviral drug | Mild to moderate COVID-19 | Ethics committee of Istinye University in Turkey approved study to test the safety and efficacy of bucillamine; 13 clinical sites selected in Turkey for the study | 2/14/22 | Infection |
| Sanotize Research and Development Corp., of Vancouver, British Columbia, and Glenmark Pharmaceuticals Ltd., of Maharashtra, India | Nitric oxide nasal spray (Fabispray) | Nasal spray formulation | COVID-19 | Study results from 306 patients met the key endpoints in India; reduction of viral load of 94% in 24 hours and 99% in 48 hours; safe and well-tolerated; median time to virological cure was 4 days in the NONS group and 8 days in the placebo group (p<0.05); significantly higher proportion of patients demonstrated a 2-point improvement on the who progression scale (validated clinical endpoint) in nons group as compared to placebo (p<0.05); no severe events or deaths study< td> | 2/9/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Vulvovaginal candidiasis | In the 260-patient Candle study, 65.4% of patients taking ibrexafungerp had clinical success, defined as no recurrence at all, either culture-proven, presumed or suspected, through week 24 compared to 53.1% of patients taking placebo (p=0.02); difference was sustained over the 3-month follow-up period (p=0.034); plans to submit NDA in first half of 2022 | 2/10/22 | Infection |
| Theratechnologies Inc., of Montreal | Intravenous Trogarzo (ibalizumab) | CD4-directed post-attachment HIV-1 inhibitor | HIV infection | Results from phase III study met the primary and secondary endpoints; showed no difference in pharmacokinetics between intravenous (I.V.) Push and I.V. infusion; no serious adverse events; no difference in viral load and no antidrug antibodies detected | 2/16/22 | Infection |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Bioresorbable polymeric matrix | Chronic rhinosinusitis | Dosed the study's first patient | 2/28/22 | Inflammatory |
| Alkermes plc, of Dublin | Lybalvi (olanzapine and samidorphan) | Atypical antipsychotic | Schizophrenia and bipolar I disorder | Study met its prespecified primary endpoint; statistically significantly less weight gain than patients treated with olanzapine at week 12 (mean percent change from baseline body weight: 6.77% for olanzapine vs. 4.91% for Lybalvi, p=0.012); proportion of patients who gained 10% or more of their baseline body weight at three months was 30.4% for olanzapine vs. 21.9% for Lybalvi (p=0.075); mean change from baseline in waist circumference at 3 months was 3.90 cm for patients treated with olanzapine vs. 2.99 cm for patients treated with Lybalvi; improvements in symptoms of schizophrenia and bipolar I disorder over three months, as measured by the Clinical Global Impression of Severity (CGI-S) scale; safety profile was consistent with previous studies | 2/8/22 | Neurology/Psychiatric |
| Anavex Life Sciences Corp., of New York | ANAVEX 2-73 (blarcamesine) | Oral sigma-1 receptor activator | Rett Syndrome | Top-line data from Avatar study met primary and all secondary endpoints; consistent improvements in RSBQ AUC (p = 0.037), ADAMS (p=0.010) and CGI-I (p=0.037) response; significantly higher proportion of ANAVEX 2-73 treated adult patients with Rett syndrome (52.9%) than placebo-treated patients (8.3%) showed improvement (p=0.010), which corresponded to a Cohen’s d effect size of 0.609 (large); more patients achieved clinically meaningful CGI-I response over the treatment duration in the ANAVEX2-73-treated group (72.2%) than in the placebo group (38.5%) (p = 0.037) with a Cohen’s d effect size of 1.91 (very large); safe; clinically meaningful improvements in multiple common areas of impairment | 2/1/22 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. and Pfizer Inc., of New York | Nurtec ODT (rimegepant) | Oral CGRP receptor antagonist | Episodic migraine | The 1,431-patient study study run in China and South Korea met its co-primary endpoints of freedom from pain (p<0.0001) and freedom from most bothersome migraine?associated symptom including nausea, phonophobia or photophobia (p<0.0001) at 2-hours following a single 75mg dose of rimegepant< td> | 2/14/22 | Neurology/Psychiatric |
| Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab-cdon) | Anti-CD19 B-cell-depleting humanized monoclonal antibody | Neuromyelitis optica spectrum disorder | Results showed levels of the sGFAP biomarker were significantly higher during major attacks vs. minor attacks overall (p=0.023) and trended higher for optic neuritis specific attacks (n=20, p=0.06); concentration levels of the biomarker increased significantly from baseline at the time of attacks in those receiving placebo but not in those treated with Uplizna (p=0.31); SNfL levels were higher for major versus minor attacks overall (p=0.032), though the levels did not correlate with the severity of ON attacks | 2/16/22 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ganaxolone | Positive allosteric modulator of GABAA receptors | Status epilepticus | Company temporarily paused the RAISE trial due to the impact of the COVID-19 Omicron variant on hospital resources and an unexpected interruption of clinical supply material associated; study expected to be completed in the second half of 2023 | 2/22/22 | Neurology/Psychiatric |
| Minerva Neurosciences Inc., of Waltham, Mass. | Roluperidone | 5-HT 2a receptor antagonist; Opioid receptor sigma antagonist 2 | Schizophrenia | Study found that Negative Symptom Factor Score scores improved (were lower) for patients receiving roluperidone 64 mg vs. placebo; intent-to-treat analysis data set (p ?0.064) missed statistical significance, but reached nominal significance (p ?0.044) for the modified-ITT (m-ITT) data set; changes in Personal and Social Performance scale total score were statistically significantly better on roluperidone 64 mg compared to placebo for both ITT and m-ITT (p ?0.021 and p ?0.017, respectively). | 2/28/22 | Neurology/Psychiatric |
| Pharmazz Inc., of Willowbrook, Ill. | Sovateltide (PMZ-1620) | Highly selective endothelin B receptor agonist | Acute ischemic stroke | Study enrolled 158 patients in India | 2/17/22 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of New York | REL-1017 | NMDA receptor channel blocker | Major depressive disorder | Top-line studies showed a substantial (30+ points); statistically equivalent to placebo p-values <0.05; statistically significant difference in abuse potential vs. ketamine (p-values <0.05)< td> | 2/23/22 | Neurology/Psychiatric |
| Reviva Pharmaceuticals Holdings Inc., of Cupertino, Calif. | Brilaroxazine | Serotonin and dopamine receptor modulator | Schizophrenia | First patient dosed | 2/1/22 | 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 | Results from Coral study co-initiated with an antidepressant met its primary and secondary endpoints; rapid and statistically significant reduction in depressive symptoms at day 3 (HAMD-17 score p=0.0004) and over the 2-week treatment period (HAMD-17 total score, p=0.0012, 0.0381 and 0.2477 at day 8, 12 and 15, respectively); well-tolerated; no new safety signals; no evidence of increased suicidal ideation/behavior | 2/16/22 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Marlborough, Mass., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan, and Otsuka Pharmaceuticals Co. Ltd., of Tokyo | Non-racemic amisulpride (SEP-4199) | 5-HT7 receptor antagonist | Major depressive episodes associated with bipolar I disorder | First patient dosed | 2/9/22 | Neurology/Psychiatric |
| Vasopharm GmbH, of Wurzburg, Germany | Ronopterin (VAS-203) | Analogue of tetrahydrobiopterin | Traumatic brain injury | Combined post-hoc analysis of Nostra and Nostra III study showed positive and clinically meaningful impact on signs of efficacy and safety in patients aged 18-39 years; improved neurologic outcome at 6 months; continuous neurologic improvement over time; good recovery at 6 months vs. placebo: 50% vs. 26%; 12 months: 80% vs. 20%, p=0.02; significant increase in proportion of low therapy intensity levels 71% vs. 62%, p=0.01; decreased aggressiveness of treatment measures required to reduce intracranial pressure, 81% vs. 64%, p=0.0002 during second week; clinically relevant reduction in renal function (AKIN level 2 and 3) | 2/8/22 | Neurology/Psychiatric |
| Dompé Farmaceutici SpA, of Milan, Italy | Oxervate (cenegermin-bkbj) | Recombinant human nerve growth factor | Severe Sjögren's-related dry eye disease | First patient enrolled in the phase III program that consists of a 100-patient study measuring Schirmer's test and the Symptom Assessment Questionnaire in Dry Eye questionnaire as co-primary endpoints and a 48-patient study measuring Schirmer's test in patients already being treated with cyclosporine A | 2/9/22 | Ocular |
| Genentech, unit of Roche Holding AG, of Basel, Switzerland | Vabysmo (faricimab) and Susvimo (ranibizumab injection) 100 mg/mL; previously called Port Delivery System with ranibizumab | Bispecific antibody for the eye targeting Ang-2 and VEGF-A; refillable implant delivers ranibizumab continuously, surgically inserted into the eye during a one-time, | Diabetic macular edema | Long term results from Yosemite and Rhine stuides achieved and maintained vision gains seen with previous standard of care injections; 60% of Vabysmo treat and extend patients in Yosemite and 64.5% in Rhine achieved 4-month dosing at 2 years; 80% of Vabysmo patients could extend treatment to every 3 months or longer in both studies; 95% of Susvimo patients maintained a 6-month treatment schedule at 2 years in Archway study | 2/11/22 | 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 | Post-hoc analysis from Gather1 study consistent with the primary analysis results in the intent to treat population; slow the progression of disease and preserve foveal anatomy while sparing the foveal center in patients | 2/11/22 | Ocular |
| Kodiak Sciences Inc., of Palo Alto, Calif. | KSI-301 | Anti-VEGF antibody biopolymer conjugate | Diabetic macular edema | Completed enrollment in Gleam and Glimmer study; around 900 patients enrolled | 2/3/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Reverse pharmacologically-induced mydriasis | Completed enrollment in MIRA-3 study; top-line data expected at the end of first quarter of 2022 | 2/8/22 | Ocular |
| Tearclear Corp., of Boston | TC-002 | Latanoprost ophthalmic solution, 0.005% | Glaucoma | Initiated phase III prospective, double-masked, randomized, active-controlled, parallel-group, 3-month study to evaluate safety and ocular hypotensive efficacy | 2/7/22 | Ocular |
| Xbrane Biopharma AB, of Stockholm | Xlucane | Lucentis (ranibizumab) biosimilar; anti-VEGF antibody | Wet age-related macular degeneration | Top-line results from 12-month equivalence Xplore trial reveal no clinically meaningful differences regarding efficacy, safety, pharmacokinetics and immunogenicity between Xlucane and Lucentis; data support ongoing registration process | 2/15/22 | Ocular |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif. | TP-03 (lotilaner ophthalmic solution 0.25%) | Non-competitive antagonist of insect and GABA-Cl channels | Demodex blepharitis | Completed enrollment of Saturn-2 study; 408 patients enrolled; top-line data expected in April 2022 | 2/2/22 | Ocular |
| Aimmune Therapeutics UK Ltd., of London | Palforzia (defatted powder of Arachis hypogaea L., semen [peanuts]) | Oral immunotherapy | Peanut allergy | New clinical data from pooled analysis published in the Journal of Allergy and Clinical Immunology showed consistent and manageable safety profile; clinically meaningful desensitization to peanut after treatment | 2/16/22 | Other/Miscallaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Pro-opiomelanocortin-derived peptide that is an agonist of melanocortin-4 receptor | Bardet-Biedl syndrome | Interim data from long-term extension study showed mean percent reduction in body mass index from baseline was -14.3% (n=19); mean percent reduction in body weight from baseline among patients 18 or older was -14.9%; mean reduction in BMI Z score from baseline among patients younger than 18 was -0.72; well-tolerated; no new safety signals | 2/16/22 | Other/Miscallaneous |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Human monoclonal antibody that binds to the PD-L1 protein | Locally advanced cervical cancer | Calla study in combination with chemoradiotherapy (CRT) did not achieve statistical significance for the primary endpoint of improving progression-free survival (PFS) vs. CRT alone; safety and tolerability were consistent between the 2 arms; no new unexpected safety findings | 3/24/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | BRCA-mutatedHER2-negative high-risk early breast cancer | Results from phase III Olympia trial demonstrated a statistically significant improvement in overall survival vs. placebo; risk of death by 32% vs. placebo p=0.009; survival rate was 92.8% and 89.8% at 3 and 4 year, respectively | 3/17/22 | Cancer |
| Bristol Myers Squibb Co., of New York, and Nektar Therapeutics Inc., of San Francisco | Bempegaldesleukin (NKTR-214) | Immunostimulatory IL-2 cytokine prodrug | Untreated unresectable or metastatic melanoma | Data monitoring committee reported Pivot IO-001 study did not meet the primary endpoints of progression-free survival (PFS) and objective response rate (ORR) in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.); did not meet statistical significance at the first interim analysis; no additional clinical benefit in the doublet therapy arm compared to monotherapy arm | 3/14/22 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | Rubraca (rucaparib) | PARP inhibitor | Advanced ovarian cancer | Top-line data from monotherapy arm achieved the primary endpoint of significantly improved investigator-assessed progression-free survival (PFS) compared with placebo; median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months vs 11.3 months among those who received placebo (p=0.0004); median PFS for all patients enrolled in ATHENA-MONO was 20.2 months vs 9.2 months among those who received placebo (p<0.0001) in the itt population; median pfs for these patients treated with rucaparib was 12.1 months vs. 9.1 those who received placebo (n="49)" (p="0.0284)" hrd negative patients< td> | 3/31/22 | Cancer |
| Coherus Biosciences Inc., of Redwood City, Calif., and Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Advanced esophageal squamous cell carcinoma | Results published in Cancer Cell showed Jupiter-06 study achieved the co-primary endpoints of progression free survival (PFS) and overall survival(OS) for patients treated with toripalimab and chemotherapy vs. chemotherapy alone; median OS in the toripalimab and placebo arms were 17 months vs. 11 months; One-year OS rates were 66% and 43.7% for toripalimab and placebo arms, respectively P=0.0004; Median PFS in the toripalimab arm and placebo arm were 5.7 months vs 5.5 months, respectively, P<0.0001< td> | 3/4/22 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China | CS-1003 (nofazinlimab) | Humanized recombinant IgG4 monoclonal antibody targeting PD-1 | Advanced hepatocellular carcinoma | Trial met prespecified patient enrollment target ahead of schedule | 3/18/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Untreated advanced hepatocellular carcinoma | Final analysis of Cosmic-312 study in combination with atezolizumab vs. sorafenib showed neither improvement nor detriment on overall survival | 3/14/22 | Cancer |
| Genentech, a unit of Basel, Switzerland-based Roche Holding AG | Tiragolumab | Monoclonal antibody targeting TIGIT | Extensive-stage small-cell lung cancer | Data from Skyscraper-02 study in combination with Tecentriq (atezolizumab) did not meet co-primary endpoint of progression-free survival; co-primary endpoint of overall survival was not met ; well-tolerated and no new safety signals in combination therapy | 3/29/22 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Trodelvy (sacituzumab govitecan-hziy) | Antibody and topoisomerase inhibitor conjugate directed to Trop-2 receptor | HR+/HER2- metastatic breast cancer | Tropics-2 study met its primary endpoint with a statistically significant improvement in progression-free survival vs. physician’s choice of chemotherapy; 30% reduction in the risk of disease progression or death; trend in improvement for overall survival; no new safety concerns | 3/7/22 | Cancer |
| Greenwich Lifesciences Inc., of Stafford, Texas | GLSI-100 | Immunotherapy | Breast cancer recurrences in patients who have previously undergone surgery | Flamingo-01 study hasn’t started recruiting, but the study is registered on clinicaltrials.gov; primary endpoint is invasive breast cancer-free survival | 3/3/22 | 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 | Results from Soraya trial met primary endpoint with confirmed objective response rate of 32.4%; 5 complete responses; median duration of responses of 6.9 months; median progression-free survival was 4.3 months; well-tolerated, consistent with the known safety profile | 3/19/22 | Cancer |
| Junshi Biosciences Co. Ltd., of Shanghai, and Coherus Biosciences Inc., of Redwood City, Calif. | Toripalimab | Anti-PD-1 monoclonal antibody | Advanced squamous or non-squamous non-small-cell lung cancer | Results from Choice-01 study plus chemotherapy met both primary endpoint of progression free survival (PFS) and prespecified secondary endpoint of overall survival compared to chemotherapy alone; 1-year PFS rates for the toripalimab and placebo arms were 36.7% and 17.2%, respectively; statistically significant improvement in overall survival for the toripalimab arm over the placebo arm (median OS not reached vs. 17.1 months, HR = 0.69); manageable safety profile with no new safety signals observed |
3/15/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Resected stage IIB and IIC melanoma | Results from Keynote-716 study met key secondary endpoint of distant metastasis-free survival (DMFS) at a prespecified interim analysis; demonstrated a statistically significant improvement in DMFS compared to placebo; no new safety signals; improvement in recurrence-free survival (RFS) (p=0.00658); significant improvements in RFS and DMFS compared to placebo (p<0.001 for both)< td> | 3/7/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 monoclonal antibody | Metastatic castration-resistant prostate cancer | Results from Keylynk-010 study in combination with Lynparza (olaparib) planned to discontinue the study; independent data monitoring committee reviewed data from a planned interim analysis; did not demonstrate a benefit in overall survival | 3/15/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Stage Ib-IIIa non-small-cell lung cancer | Study showed Keytruda significantly improved disease-free survival (DFS); reduced the risk of disease recurrence or death by 24% compared to placebo ( p=0.0014); median DFS was 53.6 months vs. 42 months for placebo | 3/17/22 | Cancer |
| Prestige Biopharma Ltd., of Singapore | HD-201/Tuznue | Herceptin biosimilar | Cancer | Study results published in JAMA Oncology; met primary endpoint; showed equivalent efficacy and comparable safety profile to referent transtuzumab; tpCR rates were 45% and 48.7% for HD-201 and referent trastuzumab, respectively | 3/11/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agents | Clear cell renal cell carcinoma | Completed target enrollment; 252 patients dosed | 3/8/22 | Cancer |
| VBL Therapeutics Ltd., of Tel Aviv, Israel | Ofra-vec (VB-111) | Anti-cancer, gene-therapy agent | Recurrent platinum-resistant ovarian cancer | Enrolled 409 patients globally; top-line data expected in second half of 2022; independent safety committee recommended the trial to continue as planned | 3/8/22 | Cancer |
| Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Advanced ovarian cancer | Data from Prime study showed statistically significant and clinically meaningful improvement in progression-free survival (PFS) with a tolerable safety profile in Chinese patients; median PFS was significantly longer for patients treated with niraparib compared to placebo: 24.8 months vs. 8.3 months, p<0.001; gbrcamut patients: mpfs, not reached vs. 10.8 months; non-gbrcamut 19.3 months 8.3 overall survival data are still immature; tolerable; safety profile consistent with previous trials< td> | 3/20/22 | Cancer |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Unique form of eicosapentaenoic acid | Coronary artery disease | Post-hoc analysis from Reduce-IT study published in the Journal of the American Heart Association showed Vascepa significantly reduced the risk of cardiovascular death, strokes, heart attacks, coronary revascularization and unstable angina by 34% in patients with a history of percutaneous coronary intervention (p<0.001); 12 robust absolute risk reductions of 8.5% and 5.4% numbers needed to treat 19, respectively, for the primary key secondary composite endpoints; 40% reduction repeat coronary revascularization vs. placebo (p<0.001)< td> | 3/10/22 | Cardiovascular |
| Boehringer-Ingelheim GmbH, of Ingelheim, Germany | Jardiance (empagliflozin) | Sodium-glucose co-transporter-2 inhibitor | Acute heart failure | Clinical benefit reflected a composite primary endpoint that included all-cause mortality, frequency of heart failure events, time to first heart failure event and symptoms as measured by the Kansas City Cardiomyopathy Questionnaire total symptom score | 3/2/22 | Cardiovascular |
| Newamsterdam Pharma B.V., of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor | Atherosclerotic cardiovascular disease | Dosed first patient in Prevail RCT | 3/1/22 | Cardiovascular |
| Abeona Therapeutics Inc., of New York and Cleveland | EB-101 | Autologous, gene-corrected cell therapy | Recessive dystrophic epidermolysis bullosa | Target enrollment achieved in phase III Viital study | 3/14/22 | Dermatologic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | Moderate-to-severe plaque psoriasis | Top-line data from Comfort study (n=400) expected in the second quarter of 2022 | 3/8/22 | Dermatologic |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Moderate to severe plaque psoriasis | Initiated phase III Visible study | 3/22/22 | Dermatologic |
| Journey Medical Corp., of Scottsdale, Ariz., and Dr. Reddy's Laboratories Ltd., of Hyderabad, India | DFD-29 | Minocycline modified release capsules 40-mg | Papulopustular rosacea | First patient dosed | 3/17/22 | Dermatologic |
| Kalvista Pharmaceuticals Inc., of Cambridge, Mass. | KVD-900 | Small-molecule protease inhibitor | Hereditary angioedema | Initiated phase III Konfident study to evaluate safety and efficacy; trial to enroll a minimum of 84 HAE adolescent and adult patients | 3/7/22 | Dermatologic |
| Aeterna Zentaris Inc., of Charleston, S.C. | Macimorelin | Ghrelin agonist | Diagnosis of childhood-onset growth hormone deficiency | Clinical trial sites planned in Ukraine and Russia are being halted due to the conflict; enrollment expected to continue in 2023 | 3/21/22 | Endocrine/Metabolic |
| Ascendis Pharma Inc., of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Hypoparathyroidism | Top-line data showed study met primary and key secondary endpoints with statistically significant improvement; 78.7% of Transcon PTH-treated patients achieved serum calcium levels compared to 4.8% for patients in control group (p=<0.0001); statistically significant decrease in patient-reported, disease-specific physical and cognitive symptoms compared to patients control group on hypoparathyroidism patient experience scales symptom-physical domain scores (p="0.0038)" hpes symptom-cognitive reduction patient-reported disease impact impact-physical functioning impact-daily life improvements survey subscale well-tolerated with no discontinuations related study drug< td> | 3/13/22 | 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 | Final results of Bright study achieved key objectives for safety, efficacy and pharmacokinetics; well-tolerated; no patients developed treatment-induced anti-drug antibodies to PRX-102; plasma lyso?Gb3 concentrations and mean absolute eGFR remained stable; after completion of the study all patients enrolled in an extension study | 3/18/22 | Endocrine/Metabolic |
| Elgan Pharma, of Nazareth, Israel | ELGN-GI | Enteral insulin formulation | Intestinal malabsorption | Unprecedented improvement in gastrointestinal (GI) function and a reduction in related complications; results published in JAMA Pediatrics | 3/2/22 | Gastrointestinal |
| Pfizer Inc., of New York | Etrasimod | Selective S1P receptor modulator | Moderate to severe Crohn's disease | Study achieved statistically significant improvements in the primary endpoint of clinical remission at week 12 as compared with placebo; statistically significant improvements were achieved in all key secondary endpoints; consistent safety profile | 3/23/22 | Gastrointestinal |
| Astellas Pharma Inc., of Tokyo | Fezolinetant | Neurokinin-3 receptor antagonist | Moderate to severe vasomotor symptoms associated with menopause | Top-line data from Moonlight study (n=302) did not meet the pre-defined endpoints for efficacy; results did not meet statistical significance | 3/15/22 | Genitourinary/Sexual Function |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | Sodium-glucose co-transporter-2 inhibitor | Chronic kidney disease | Independent data monitoring committee recommended that the EMPA-KIDNEY trial be stopped early following a formal interim assessment | 3/16/22 | Genitourinary/Sexual Function |
| Clarus Therapeutics Holdings Inc., of Northbrook, Ill. | Jatenzo | Testosterone undecanoate capsules; testosterone replacement therapy | Adult hypogonadal men with chronic kidney disease | Initiated screening for the first patient in an investigator-initiated trial; results expected in the first half of 2023 | 3/16/22 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Linzagolix | GnRH antagonist | Moderate-to-severe endometriosis-associated pain | Aditional efficacy data from Edelweiss study showed average reduction of dysmenorrhea and non-menstrual pelvic pain at each month up to 6 months; rapid reductions compared to placebo with continued reduction up to 6 months of treatment and with higher reductions with linzagoli (200 mg) + add-back therapy vs. linzagoli (75 mg); reductions in endometriosis pain were associated with improvements in quality of life; reduction in physician and patient intentions to undergo surgery for endometriosis | 3/22/22 | Genitourinary/Sexual Function |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Hydrochloric acid binder | Metabolic acidosis in patients with chronic kidney disease | Top-line data from the 179-patient VALOR-CKD trial are expected in the fourth quarter of 2022, rather than the third quarter of 2022 as previously guided because of the war in Ukraine where 15% of the randomized patients are enrolled | 3/2/22 | 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 published in Therapeutics and Clinical Risk Management showed significant improvements compared with placebo in the efficacy outcomes of micturition frequency, urge urinary incontinence episodes, urgency episodes and volume voided; improvements in patient-reported outcomes; safe and well-tolerated | 3/22/22 | Genitourinary/Sexual Function |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | Adeno-associated virus vector encoding human coagulation factor VIII | Severe Hemophilia A | Results for one year or more of follow-up data from Gener8-1 study published in New England Journal of Medicine experienced substantially reduced annualized bleeding rates, reduced factor VIII utilization, and increased factor VIII activity; mean annualized bleeding rate reduced by 84% (p-value <0.001); mean annualized factor viii utilization rate reduced by 99% (p-value <0.001); no new safety signals; treatment-related serious adverse event< td> | 3/17/22 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm | Efanesoctocog alfa (BIVV-001) | Recombinant factor VIII therapy | Severe hemophilia A | Study met primary and secondary endpoint at once-weekly dosing; clinically meaningful prevention of bleeding episodes; statistically significant and clinically meaningful reduction in annualized bleeding rate compared to prior factor VIII prophylaxis therapy; well-tolerated | 3/9/22 | Hematologic |
| Allovir Inc., of Waltham, Mass. | Posoleucel | Allogeneic, off-the-shelf, multivirus specific T-cell therapy | Allogeneic hematologic cell transplantation | Initiated phase III study; planning to enroll 300 adult and pediatric patients; to evaluate the number of clinically significant infections or episodes of end-organ disease through the primary endpoint of the 14-week dosing interval | 3/22/22 | Immune |
| Argenx SE., of Breda, the Netherlands | Efgartigimod alfa; subcutaneous formulation | FcRn blocker | Generalized myasthenia gravis | Top-line data showed study achieved the primary endpoint of total IgG reduction (66.4%) from baseline at day 29 statistical noninferiority to I.V. form,Vyvgart (efgartigimod alfa) (p< 0.0001); secondary endpoints were met; well-tolerated; consistent safety profile | 3/22/22 | Immune |
| Equillium Inc., of La Jolla, Calif. | Itolizumab | Anti-CD6 monoclonal antibody | Acute graft-vs.-host disease | Initiation of Equator study; primary objective is to achieve early disease response; key secondary objectives to evaluate durability of response, corticosteroid use, survival outcomes, and chronic GVHD incidence | 3/4/22 | Immune |
| Novartis AG, of Basel, Switzerland | Kesimpta (ofatumumab) | Anti-CD20 mAb | Relapsing forms of multiple sclerosis | New data from the Alithios open-label extension study published in Multiple Sclerosis Journal showed that with up to 3.5 years of treatment with Kesimpta, no incidences of opportunistic infections were reported, and observed COVID-19 infections showed no evidence of an increase in incidence or severe outcomes in adults with relapsing forms of multiple sclerosis | 3/2/22 | Immune |
| Cansino Biologics Inc., of Tianjin, China | Convidecia | Adenovirus type 5-based vaccine; inhalation type | COVID-19 infection | Study results from 904 participants who had booster vaccination via intramuscular or inhaled version showed greater neutralizing antibody responses than those induced by the homologous inactivated vaccine booster or heterologous recombinant protein vaccine booster; significantly increased cellular immune protection against the transmission and infection with the omicron variant | 3/14/22 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon Lambda | Type III interferon | COVID-19-related hospitalizations | Results from Together study showed risk of COVID-19-related hospitalizations or emergency room visits greater than six hours reduced by 50% vs. placebo; 42% risk reduction when treated ?7 days of symptom onset; 60% risk reduction when treated ?3 days of symptom onset | 3/17/22 | Infection |
| Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. | Olumiant (baricitinib) | Oral JAK inhibitor | COVID-19 | Study showed baricitinib lowered the risk of death among hospitalized Covid patients by 13% in seriously ill patients in U.K. recovery trial | 3/4/22 | Infection |
| Evofem Biosciences Inc., of San Diego | EVO-100 (Phexxi) | Formulation comprising lactic acid, citric acid and potassium bitartrate | Urogenital chlamydia infectionand gonorrhea in women | Completed enrollment in Evoguard study; enrolled 1,903 participants; top-line data expected in second half of 2022 | 3/4/22 | Infection |
| Faron Pharmaceuticals Oy, of Turku, Finland | Traumakine (intravenous interferon beta-1a) | CD73 agonist; interferon-beta ligand | COVID-19 infection | Post-hoc analysis showed substantial reduction in mortality compared to patients without the gene mutation | 3/14/22 | Infection |
| Inflarx NV, Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Severe COVID-19 | Top-line data from mechanically ventilated COVID-19 patients showed a relative reduction in 28-day all-cause mortality of 23.9% (vilobelimab 31.7% vs. placebo 41.6%, p=0.094); pre-specified sensitivity analysis of the primary endpoint results in p-values of <0.05 in three out of four planned analyses; 43% relative reduction 28-day all-cause mortality detected pre-specified subgroup analysis western european patients comparing vilobelimab to placebo treatment (p-value="0.014);" safe; well-tolerated< td> | 3/31/22 | Infection |
| Inovio Inc., of Plymouth Meeting, Pa. | INO-4800 | DNA vaccine | COVID-19 | Paused enrollment to seek regulatory approval of endpoint amendment | 3/2/22 | Infection |
| Junshi Biosciences Co. Ltd., of Shanghai | VV-116 | Oral nucleoside analogue | Moderate to severe COVID-19 | First patient dosed in China | 3/16/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373/Nuvaxovid | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Extended analysis from its pivotal phase III trial conducted in the U.K. showed that a high level of efficacy for the vaccine was maintained over a 6-month period of surveillance; the analysis showed vaccine efficacy of 82.5% in protection against all COVID-19 infection, both symptomatic and asymptomatic, as measured by PCR+ or anti-N seroconversion | 3/1/22 | 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 participant enrollment in observer-blinded phase III study in the U.A.E | 3/25/22 | Infection |
| Pfizer Inc., of New York |
PF-06425090 | Vaccine | Clostridioides difficile infection (CDI) | In the phase III CLOVER trial the pre-specified secondary endpoint showed 0-11 vaccine to placebo case split for medically attended CDI, corresponding to 100% vaccine efficacy; the trial did not meet its pre-specified primary endpoint of prevention of primary CDI, however, for all CDI cases recorded at 14 days post dose 3, vaccine efficacy was 49%, 47% and 31% up to 12 months, 24 months and at final analysis, respectively; the vaccine was well tolerated and showed a favorable safety profile | 3/1/22 | Infection |
| Polypid Ltd., of Petach Tikva, Israel | D-Plex | Prolonged and constant release of broad-spectrum antibiotic doxycycline | Abdominal soft tissue surgical site infections | Unblinded interim analysis of ongoing trial Shield I will be conducted | 3/2/22 | Infection |
| SAB Biotherapeutics Inc., of Sioux Falls, S.D. | SAB-185 | Polyclonal human IgG antibody, purified from the plasma of immunized Tc bovines | COVID-19 | Reported NIH is closing enrollment in its ACTIV-2 trial due to low omicron-related COVID-19 hospitalization and death rates, making current phase III study design statistically unworkable; company is evaluating future clinical plans for SAB-185 including potential targeted applications such as prophylaxis and treatment in high-risk patient groups | 3/2/22 | Infection |
| Sanofi SA, of Paris | Nirsevimab | Monoclonal antibody targeting respiratory syncytial virus (RSV) | Infants at high risk of RSV infection | Data from the Melody study, published in The New England Journal of Medicine, showed nirsevimab produced a 74.5% reduction in lower respiratory tract infections caused by RSV requiring medical care compared to placebo (p<0.001); rsv-associated hospitalizations in the study were reduced patients who received nirsevimab compared to saline, but reduction wasn’t statistically significant (p="0.07);" prespecified pooled analysis of phase iii and iib studies showed lowered by 77.3% (p<0.001)< td> | 3/3/22 | Infection |
| Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine | Chikungunya virus infection | Completed phase III six-month follow-up; all endpoints met; seroprotection in 98.9% of participants after 1 month and 96.3% after 6 months ; good safety and tolerability profile; no safety concerns.; mild or moderate adverse events | 3/8/22 | Infection |
| Vaxxinity Inc., of Dallas | UB-612 | Multitope protein/synthetic peptide-based vaccine | COVID-19 prophylaxis | First subject dosed | 3/28/22 | Infection |
| Venatorx Pharmaceuticals Inc., of Malvern, Pa. | Cefepime-taniborbactam | Cephalosporin derivative; beta-lactamase inhibitor | Complicated urinary tract infections (cUTI), including acute pyelonephritis | Results from 661 adult patients showed study met the primary efficacy endpoint of statistical noninferiority to meropenem in the microITT population at test of cure with composite microbiologic and clinical success occurring in 70% of cefepime-taniborbactam-treated patients and 58% of meropenem treated patients; treatment-emergent adverse event rate was 35.5% vs. 29% for meropenem; well-tolerated with similar safety profile to meropenem | 3/10/22 | Infection |
| Ampio Pharmaceuticals Inc., of Englewood, Colo. | Ampion | Biologic drug containing a blood-derived cyclized peptide | Osteoarthritis of the knee | In the modified intent-to-treat population of the AP-013 study, Ampion reduced pain compared to saline control (p=0.042); in the per protocol population, Ampion reduced pain (p=0.020) and showed improved function (p=0.027) vs. saline control | 3/2/22 | Inflammatory |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Top-line results from Reopen1 study met both of its co-primary endpoints; statistically significant improvement on symptoms and CT scans in patients | 3/7/22 | Inflammatory |
| Paradigm Biopharmaceuticals Ltd., of Melbourne, Australia | Pentosan polysulfate sodium | Semi-synthetic drug ; sulfated to produce a negatively charged product that mimics glycosaminoglycan | Osteoarthritis of knee | Launched clinical program with an estimated 56 planned sites for phase III study | 3/17/22 | Inflammatory |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | Selective p38?/? mitogen activated protein kinase inhibitor | Facioscapulohumeral muscular dystrophy | The 230-patient Reach trial is scheduled to start in second quarter of 2022; primary endpoint is the absolute change from baseline in reachable workspace; secondary endpoints include muscle fat infiltration, patient global impression of change and quality of life in neurological disorders of the upper extremity | 3/3/22 | Musculoskeletal |
| Neurana Pharmaceuticals Inc., of San Diego | Tolperisone | Non-opioid, centrally acting muscle relaxant | Muscle spasm | Study not met primary and key secondary endpoints; safe and well-tolerated; no serious adverse events | 3/22/22 | Musculoskeletal |
| Biogen Inc., of Cambridge, Mass. | Spinraza (nusinersen) | Antisense oligonucleotide targeting SMN2 expression | Spinal muscular atrophy | First patient treated in the Ascend study; study planning to enroll 135 children, teens and adults previously treated with Evrysdi (risdiplam, Roche Holding AG/PTC Therapeutics Inc.) | 3/14/22 | Musculoskeletal |
| Biogen Inc., of Cambridge, Mass. | Spinraza (nusinersen) | Antisense oligonucleotide targeting SMN2 expression | Spinal muscular atrophy | Results from Respond study (n=9) from infants and toddlers who previously received the gene therapy showed suboptimal clinical status in 2 or more domains at baseline; no new safety findings | 3/14/22 | Musculoskeletal |
| Biogen Inc., of Cambridge, Mass. | Spinraza (nusinersen) | Antisense oligonucleotide targeting SMN2 expression | Spinal muscular atrophy | Results from Nurture study demonstrated early and sustained treatment for up to 5.7 years (median 4.9 years); maintained and progressive gains in motor function; most children achieved motor milestones within age-appropriate timelines; no major motor milestones lost | 3/14/22 | Musculoskeletal |
| Novartis AG, of Basel | Zolgensma (onasemnogene abeparvovec) | Gene therapy expressing SMN | Spinal muscular atrophy | Post-hoc analysis of START, STR1VE-EU and STR1VE-US study achieved or maintained important measures of bulbar function; 100% of patients met the primary endpoint of standing unassisted for ?3 seconds by 24 months of age in Sprint study; 93% of patients walked independently; 100% patients were independent of nutritional and respiratory support for the duration of the study; 95% met the communication endpoint in study; 92% had at least 1 occurrence of a normal swallow test and did not report any event indicating the inability to maintain airway protection; 80% achieved the composite endpoint of having the ability to speak, being able to swallow normally and maintaining airway protection | 3/14/22 | Musculoskeletal |
| Abbvie Inc., of North Chicago | Qulipta (atogepant) | CGRP antagonist | Chronic migraine | Study met its primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo both the 60-mg once-daily and 30-mg twice-daily doses; statistically significant improvements in all secondary endpoints after adjustment for multiple comparison (p?0.0009 and p?0.0024); consistent safety profile; decrease of 6.88 and 7.46 monthly migraine days at 60-mg and 30-mg, respectively, compared to patients in the placebo arm who experienced a decrease of 5.05 monthly migraine days (60 mg QD vs. placebo, p=0.0009; 30 mg BID vs. placebo, p<0.0001, 30 adjusted for multiple comparisons); decrease of 6.75 and 7.33 monthly migraine days, respectively, compared to patients in the placebo arm who experienced a 5.09 days (60 mg qd vs. placebo, p="0.0024;" bid comparisons)< td> | 3/10/22 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Aduhelm (aducanumab) | Monoclonal antibody directed against amyloid beta | Alzheimer’s disease | Long-term extension study showed Aduhelm significantly reduced amyloid beta plaque levels out to week 132; decreased plasma p-tau181 levels at 128 weeks; patients with effective amyloid beta clearance (SUVR lower than 1.1 by 78 weeks) also had greater decreases in p-tau181 at week 128 | 3/16/22 | Neurology/Psychiatric |
| Horizon Therapeutics plc, of Dublin | Uplizna (inebilizuma) | Anti-CD19 B-cell-depleting humanized monoclonal antibody | Neuromyelitis optica spectrum disorder | Post-hoc analysis of phase III trial showed safe and effective; 4.2% of those who were treated with Uplizna experienced an attack vs. 23.1% of those who were treated with placebo in 37 study participants who had experienced only one attack; 12.4% of those who were treated with Uplizna experienced an attack vs. 48.7% of those who were treated with placebo in 176 study participants who had experienced more than one attack | 3/31/22 | Neurology/Psychiatric |
| Innocoll Inc., of Athlone, Ireland | Xaracoll | Collagen drug-device implant | Pain in patients undergoing abdominoplasty | Statistically significant treatment effect as measured by time-weighted sum of pain intensity from time of implantation through 24 hours | 3/2/22 | 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 | Results from final study (n=401) showed primary endpoint of change in average daily pain in the affected leg over 4 weeks following the initial injection demonstrated LS Mean group difference of -1.08 (0.17) compared to placebo with a p-value <0.001; 4 28% improvement at weeks on secondary endpoint p-value < 0.001; no identified safety risks; serious adverse events< td> | 3/18/22 | Neurology/Psychiatric |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of muscle relaxant cyclobenzaprine hydrochloride | Fibromyalgia | Study did not achieve statistical significance on primary endpoint of reducing fibromyalgia daily pain at week 14 compared to placebo (p=0.115) at 5.6 mg; strong activity on sleep disturbance (p=0.004) and on the patient global impression of change (PGIC; p=0.038); well-tolerated; no new safety signals | 3/21/22 | Neurology/Psychiatric |
| Vallon Pharmaceuticals Inc., of Philadelphia | Adair | Abuse-deterrent formulation of immediate-release dextroamphetamine | Attention deficit hyperactivity disorder | Top-line data from SEAL study did not meet primary endpoint of significant reduction in Emax drug liking for Adair vs. reference dextroamphetamine although a trend in favor of Adair was observed (p=0.16); pharmacodynamic secondary endpoints of the study were met with Adair scoring significantly lower than reference dextroamphetamine on overall drug liking assessed at 12 hours (p<0.0001) 12 24 and hours post-dosing (p="0.024);" willingness to take drug again was also assessed at (p<0.0001) post dosing (p<0.0001); well-tolerated; no serious events< td> | 3/21/22 | Neurology/Psychiatric |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Intravitreal pegcetacoplan | Targeted C3 therapy | Geographic atrophy | Long-term study showed pegcetacoplan reduced GA lesion growth with both monthly (22%; p<0.0001 and 13%; p="0.0254" in oaks derby study, respectively) every-other-month treatment (16%; 12%; derby, respectively); clinically meaningful effects a favorable safety profile; no events of retinal vasculitis or vein occlusion were observed< td> | 3/16/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Reverse pharmacologically-induced mydriasis | Completed enrollment in MIRA-4 trial; to evaluate safety and efficacy in 23 pediatric subjects; top-line data expected in end of first quarter 2022 | 3/8/22 | Ocular |
| Orasis Pharmaceuticals Ltd., of Herzliya, Israel | CSF-1 | Undisclosed repurposed drug; Eye drops | Presbyopia | Completed near-1 and near-2 study | 3/15/22 | Ocular |
| Visus Therapeutics Inc., of Seattle | Brimochol | Preservative-free, fixed-dose combination of carbachol (a cholinergic miotic agent) and brimonidine tartrate (an alpha-2 agonist) | Presbyopia | Launched first part of 2 pivotal phase III trials; first patient dosed | 3/22/22 | Ocular |
| Nicox SA, of Sophia Antipolis, France, and Ocumension Therapeutics Ltd., of Shanghai | Zerviate (cetirizine) | Ocular formulation of the histamine-1 receptor antagonist | Allergic conjunctivitis | Positive results from a Chinese phase III trial showed that compared to emedastine difumarate ophthalmic solution, 0.05% (Emadine, Kanebo KK), Zerviate was non-inferior in the primary efficacy endpoint of change from baseline in the itching score in the 24 hours prior to the day 14 visit; Zerviate was safe and well-tolerated with no difference in the proportion of patients with adverse events | 3/1/22 | Respiratory |
| Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Leukemia | Data from Sierra trial showed 100% of patients able to proceed to a bone marrow transplant; achieved engraftment compared to only 18% of patients in control arm; non-relapse transplant-related mortality (TRM) 100 days post BMT was 9% in the Iomab-B arm compared to 14% in the control arm; statistically significantly rate of sepsis lower in the Iomab-B arm (p=0.002); 5% of patients experiencing grade 3 or greater sepsis compared to 24% of patients in the control arm; rates of febrile neutropenia were 34% lower in patients on Iomab-B arm compared to control; 78% of patients on Iomab-B evaluable for the dCR primary endpoint compared to 16% after taking into account rates of 100-day TRM | 4/25/22 | Cancer |
| Antengene Corp. Ltd., of Shanghai | Xpovio (selinexor) | Selective Inhibitor of Nuclear Export compound | Relapsed and/or refractory multiple myeloma | Results from 82 Chinese patients published in BMC Medicine showed an overall response rate (ORR) of 29.3% for all treated patients (ORR of 25% in triple-class refractory disease, 50% in those who received prior CAR-T and 25.5% in high-risk cytogenetic abnormalities; median duration of response of 4.7 months; median progression-free survival and overall survival were 3.7 months and 13.2 months, respectively; no human pharmacokinetic ethnicity difference was identified between Chinese and Western patients | 4/6/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Innate Pharma SA, of Marseille, France | Imfinzi (durvalumab) + oleclumab + monalizumab | PD-L1-targeting antibody + anti-CD73 monoclonal antibody and anti-NKG2A monoclonal antibody | Non-small-cell lung cancer | First patient dosed | 4/29/22 | Cancer |
| Beigene Co. Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma | In the Alpine trial, Brukinsa produced an overall response rate of 80.4% compared to 72.9% for ibrutinib (p=0.0264) | 4/11/22 | Cancer |
| Beigene Ltd., of Beijing, and Novartis AG, of Basel, Switzerland | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Recurrent or metastatic nasopharyngeal cancer | Updated data of Rationale-309 trial in combination with chemotherapy showed clinically significant progression-free surviva (PFS) at a median follow-up of 15.5 month compared to chemotherapy alone; manageable safety profile; median PFS of 9.6 months; median overall survival not reached | 4/19/22 | Cancer |
| Beigene Ltd., of Beijing, and Novartis AG, of Basel, Switzerland | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Advanced or metastatic esophageal squamous cell carcinoma | Interim analysis in combination with chemotherapy met primary endpoint of overall survival; consistent safety and tolerability profile; no new safety signals; statistically significant and clinically meaningful improvement in overall survival achieved for tislelizumab and chemotherapy compared to placebo and chemotherapy | 4/27/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Results from Checkmate-816 study in combination with chemotherapy significantly improved event-free survival (EFS) compared to chemotherapy alone in patients; minimum follow-up of 21 months, reduced the risk of disease recurrence, progression or death by 37% (p=0.0052) in combination with chemotherapy; median EFS was 31.6 months vs. 20.8 months for patients treated with chemotherapy alone; analysis did not reach statistical significance on additional endpoints; favorable early overall survival results | 4/11/22 | Cancer |
| Bristol Myers Squibb Co., of New York, and Nektar Therapeutics Inc., of San Francisco | Bempegaldesleukin (NKTR-214) | Immunostimulatory IL-2 cytokine prodrug | Renal cell carcinoma and bladder cancer | Study did not meet the prespecified boundary for statistical significance in comparison to the tyrosine kinase inhibitor (TKI) control arm; overall survival also did not meet the prespecified boundary for statistical significance in either of these populations; no clinical benefit in the doublet therapy arm compared to the TKI arm | 4/14/22 | Cancer |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Ontak (denileukin diftitox) | Cancer immunotherapy; engineered interleukin-2 diphtheria toxin fusion protein | Persistent or recurrent cutaneous T-cell lymphoma | Top-line data from phase III study achieved an overall response rate of 42.3%; significant reduction in tumor size; achieved a partial or complete response | 4/6/22 | Cancer |
| Isofol Medical AB, of Gothenburg, Sweden | Arfolitixorin | Folate receptor antagonist | Advanced, metastatic colorectal cancer | Initiated phase III agent study in combination with 5-FU, oxaliplatin and bevacizumab | 4/22/22 | Cancer |
| ITM Isotope Technologies München AG, of Garching, Germany | ITM-11 (n.c.a. 177Lu-edotreotide) | Radioisotope, no-carrier-added lutetium-177 chelated to the edotreotide | Somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors | Completed recruitment of the 300-patient Compete study | 4/11/22 | Cancer |
| Novartis AG, of Basel, Switzerland | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Locally advanced, recurrent or metastatic esophageal squamous cell carcinoma | Top-line interim data in combination with chemotherapy significantly improved overall survival compared to chemotherapy alone | 4/27/22 | Cancer |
| Orca Bio Inc., of Menlo Park, Calif. | Orca-T | Allogeneic cellular therapy | Hematologic malignancies | Pooled data (n=137) showed improved overall survival and reduced chronic graft-vs.-host disease compared to standard of care; well-tolerated and resulted in engraftment with regression of marrow fibrosis; relapse-free survival was 71% at 1 year for Orca-T patients compared to 21% in the CIBMTR-based control arm; overall survival for patients who received Orca-T was 90% at 1 year compared to 68% in the CIBMTR-based control arm | 4/25/22 | Cancer |
| Point Biopharma Inc., of Indianapolis | 177Lu-PNT2002 | PSMA-targeted radioligand | Metastatic castration-resistant prostate cancer | First patient dosed in the European Union in Splash trial | 4/26/22 | Cancer |
| Servier Pharmaceuticals LLC, of Paris and Boston | Tibsovo (ivosidenib) | Oral L-prolinamide-derived inhibitor of isocitrate dehydrogenase 1 | Previously untreated IDH1-mutated acute myeloid leukemia | Data published in the New England Journal of Medicine showed combination of Tibsovo with azacitidine met study's primary and all key secondary endpoints; improved event-free survival (EFS) and overall survival (OS) vs. azacitidine plus placebo; statistically significant improvement in EFS (p=0.0011); statistically significant improvement in OS (p=0.0005); median OS of 24 months vs. 7.9 months in the placebo plus azacitidine arm; complete remission rate was 47.2% in combination with azacitidine vs. 14.9% for placebo plus azacitidine (p<0.0001); objective response rate was 62.5% in combination with azacitidine vs. 18.9% for placebo plus (p<0.0001)< td> | 4/21/22 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Repatha (evolocumab) | Human monoclonal antibody that inhibits PCSK9 | Atherosclerotic cardiovascular disease | Top-line data from open-label extension of Fourier study showed significant and sustained reduction in low-density lipoprotein cholesterol (LDL-C) levels; 85% of patients achieved an LDL-C level of <40 2 140 420 mg dl; no new safety findings; safe and well-tolerated at every weeks or monthly< td> | 4/27/22 | Cardiovascular |
| Bristol Myers Squibb Co., of New York | Mavacamten | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | In the Valor-HCM study, after 16 weeks of treatment with mavacamten, 82% of patients had not proceeded with septal reduction therapy (SRT), compared to 32% of patients taking placebo; 17.9% of patients taking mavacamten met the composite endpoint of patients who proceeded with SRT plus those who remained eligible for SRT compared to 76.8% for placebo (p<0.0001)< td> | 4/2/22 | Cardiovascular |
| Bristol Myers Squibb Co., of New York | Mavacamten | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | In the Explorer-LTE cohort of the Mava-LTE study, mavacamten produced a -35.6-mmHg change in left ventricular outflow tract gradient at week 48; resting left ventricular ejection fraction decreased from baseline by -7% | 4/3/22 | Cardiovascular |
| Idorsia Pharmaceuticals Ltd., of Allschwil, Switzerland | Pivlaz (clazosentan sodium) | Selective endothelin A receptor antagonist | Cerebral vasospasm, vasospasm-related cerebral infarction and cerebral ischemic symptoms after aneurysmal subarachnoid hemorrhage | Data from 2 studies published in the Journal of Neurosurgery showed clazosentan reduced the occurrence of cerebral vasospasm-related morbidity and all-cause mortality events by >50% within 6 weeks after an aneurysmal subarachnoid hemorrhage (p<0.01 for both studies); treatment with clazosentan decreased the event rates individual components of primary composite endpoints: vasospasm-related delayed ischemic neurologic deficit was reduced by 60% (p="0.0004)" and new cerebral infarcts were 55% (p<0.0001) in patients taking clazosentan< td> | 4/4/22 | Cardiovascular |
| Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson | Xarelto (rivaroxaban) | Oral factor Xa inhibitor | Peripheral artery disease | New analyses from Voyager PAD trial showed the vascular dose (2.5 mg twice daily plus aspirin 100 mg once daily) significantly reduced hospitalizations due to thrombotic events in patients who underwent lower-extremity revascularization vs. patients who took aspirin alone (Kaplan-Meier estimate at 3 years: 8.7% vs. 12.1%); results of subgroup analysis of patients with and without chronic kidney disease, show consistent 4.7% absolute risk reduction with Xarelto/aspirin vs. aspirin alone (Kaplan-Meier estimate at 3 years: 7.9% vs. 12.6%) | 4/1/22 | Cardiovascular |
| Lexaria Bioscience Corp., of Kelowna, British Columbia | DehydraTECH-CBD | CBD formulation | Hypertension | Data showed tendency (p=0.1) during 15 minutes of simulated low levels of oxygen (hypoxia) for reduced pulmonary artery systolic pressure vs. placebo; pulmonary artery systolic pressure significantly attenuated by about 5 mmHg or 41% overall (p=0.045) ; well-tolerated; no serious side effects | 4/14/22 | Cardiovascular |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Sotagliflozin | Oral dual SGLT1/2 inhibitor | Heart failure and diabetes | In the 10,584-patient Scored trial, sotagliflozin reduced the relative risk of major adverse cardiovascular events of cardiovascular death, non-fatal myocardial infarction and non-fatal stroke by 21% for patients with cardiovascular disease (CVD) and by 26% for patients without CVD compared to placebo | 4/2/22 | Cardiovascular |
| Milestone Pharmaceuticals Inc., of Montreal | Etripamil | Intranasal spray formulation of short-acting calcium channel antagonist | Paroxysmal supraventricular tachycardia | Top-line data from open-label extension study Node-302 showed PSVT conversion rate at 30 minutes following etripamil administration was 60.2%, with median time to conversion of 15.5 minutes | 4/30/22 | Cardiovascular |
| Theravance Biopharma Inc., of Dublin | Ampreloxetine | Long-acting, oral, once-daily norepinephrine reuptake inhibitor | Symptomatic neurogenic orthostatic hypotension | Results from second phase III study, Study 0170, did not meet primary endpoint, defined as worsening of both Orthostatic Hypotension Symptom Assessment Scale question 1 and Patient Global Impression of Severity scores by 1 point, for overall population (p=0.196); odds ratio suggested patients receiving ampreloxetine had 40% reduction in odds of treatment failure compared to placebo; subgroup analysis suggested benefit was largely driven by multiple system atrophy (MSA) patients, which indicated a 72% reduction in odds of treatment failure vs. placebo; company plans to pursue path for treatment in MSA patients | 4/5/22 | Cardiovascular |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso DPI (inhaled treprostinil) | PGI2 agonist | Pulmonary arterial hypertension | Results from long-term safety Breeze study published in Pulmonary Circulation showed well-tolerated; transition from Tyvaso to Tyvaso DPI demonstrated safety and tolerability with significant improvements in 6-minute walk distance, device preference and satisfaction, and patient-reported outcomes; no study drug-related serious adverse events | 4/6/22 | Cardiovascular |
| Almirall SA, of Barcelona, and Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody that binds to IL-13 | Atopic dermatitis | Data from week 16 met all primary and secondary endpoints in combination with topical corticosteroids (TCS); 41% achieved clear or almost clear skin (IGA) compared to 22% receiving placebo with TCS; 70% of patients receiving lebrikizumab plus TCS achieved an EASI-75 response compared to 42 % in placebo plus TCS group; lebrikizumab plus TCS patients achieved statistically significant improvements across key secondary endpoints including skin clearance and itching, interference of itch on sleep, and quality of life measures, compared to placebo with TCS; consistent safety profiles | 4/11/22 | Dermatologic |
| Amgen Inc., of Thousand Oaks, Calif. | ABP-654 | Biosimilar candidate Stelara ustekinumab | Moderate to severe plaque psoriasis | Preliminary data showed study met primary efficacy endpoint; no clinically meaningful differences between ABP-654 and Stelara; percentage improvement of 0.14 | 4/18/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream | Topical cream formulation of PDE4 inhibitor | Scalp and body psoriasis | Enrollment of the last subject in the Arrector study; top-line data expected in late third quarter or early fourth quarter of 2022 | 4/6/22 | Dermatologic |
| Krystal Biotech Inc., of Pittsburgh | B-VEC | Gene therapy expressing COL7 protein | Dystrophic epidermolysis bullosa | Patients of Gem-3 open-label extension study offered option to be dosed at home by health-care professionals | 4/11/22 | Dermatologic |
| Leo Pharma Inc., of Madison, N.J. | Adbry (tralokinumab-ldrm) | Human monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Interim post-hoc efficacy analysis of the 5-year Ecztend extension study showed 83% of patients treated with Adbry for 3 years achieved at least 75% improvement in EASI score; 59% achieved EASI-90; 48% achieved an Investigator Global Assessment score of 0/1 | 4/11/22 | Dermatologic |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Takhzyro (lanadelumab) | Fully human monoclonal antibody that binds with plasma kallikrein | Hereditary angioedema | Study met its objectives in patients 2 to <12 years of age; no serious adverse events; dropouts due to events< td> | 4/12/22 | Dermatologic |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Pyrukynd/mitapivat | Pyruvate kinase activator | Pyruvate kinase (PK) deficiency | Data from Activate study published in the New England Journal of Medicine met its primary endpoint; 40% patient achieved a hemoglobin response; increase in hemoglobin concentration from baseline sustained at 2 or more scheduled assessments at weeks 16, 20 and 24 during the fixed-dose period, compared to 0 patients randomized to placebo (2-sided p<0.001)< td> | 4/14/22 | Endocrine/Metabolic |
| Amryt Pharma plc, of Dublin | Mycapssa (octreotide) | Somatostatin derivative | Acromegaly | Data from open-label extension study from second year had 100% of evaluable patients; maintained IGF-1 within normal limits; maintained long-term biochemical response at the end of the study; growth hormone levels also improved; median treatment duration of 2.1 years and a maximal exposure of 3.2 years; median compliance rate of 98% over period of time; no new safety signals with long-term exposure | 4/13/22 | Endocrine/Metabolic |
| Innovent Biologics Inc., of San Francisco | IBI-306 | Anti-PCSK9 antibody | Heterozygous familial hypercholesterolemia | In the Credit-2 study, the 150-mg Q2W or 450-mg Q4W doses reduced LDL-cholesterol (LDL-C) levels by 57.4% and 61.9% compared to placebo, respectively (p<0·0001 12 for both); 58.5% and 73.4% of patients taking the 150-mg q2w or 450-mg q4w doses, respectively, achieved ?50% ldl-c reductions at week as compared with corresponding placebo groups (p<0·0001 both)< td> | 4/4/22 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsværd, Denmark | Insulin icodec | Once-weekly insulin | Type 2 diabetes | Onwards 2 study achieved primary endpoint at week 26 with insulin icodec compared to insulin degludec; HbA1c of 8.13% in insulin icodec achieved a superior reduction in estimated HbA1c of 0.93% compared to 0.71% for insulin degludec; no statistical difference in estimated hypoglycaemia rates; no severe hypoglycaemia events | 4/28/22 | Endocrine/Metabolic |
| Pathalys Pharma Inc., of Research Triangle Park, N.C. | Upacicalcet | Calcimimetic | Secondary hyperparathyroidism | Planning to initiate 2 phase III studies | 4/7/22 | 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 | In the Balance trial, median estimated glomerular filtration rate slope was –2.514 mL/min/1.73 m2/year for PRX-102 and –2.155 mL/min/1.73 m2/year for agalsidase beta, which met the primary endpoint for noninferiority |
4/4/22 | Endocrine/Metabolic |
| Vifor Pharma Group, of St. Gallen, Switzerland | Veltassa (patiromer sorbitex calcium) | Potassium binder | Hyperkalemia | Diamond trial met its primary and all 5 secondary endpoints; treatment with Veltassa lowered the risk of hyperkalemia events by 37% | 4/4/22 | Endocrine/Metabolic |
| Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel | Aramchol | Stearoyl CoA desaturase-1 inhibitor | Liver fibrosis and Nonalcholic steatohepatitis | Results from aramchol 300 mg twice-daily dosing in open label part of the Armor study showed treatment effect was larger at 48 weeks compared to 24 weeks; AI analysis showed mean fibrosis composite severity reduction was (p=0.017) at wk24 and (p<0.0001) 48 at weeks and beyond< td> | 4/28/22 | Gastrointestinal |
| Ardelyx Inc. of Waltham, Mass. | Xphozah (tenapanor) | NHE3 inhibitor | Chronic kidney disease | Data from a responder analysis showed 77% of patients experienced phosphate lowering, with 41% of responders achieving a greater than 2-mg/dL decrease | 4/7/22 | Genitourinary/Sexual Function |
| Bayer AG, of Leverkusen, Germany | Kerendia (finerenone) | Nonsteroidal mineralocorticoid receptor antagonist | Chronic kidney disease in patients with type 2 diabetes | Subgroup analyses from pooled Fidelio-DKD and Figaro-DKD trials showed, over a median follow-up of 3 years, patients with atherosclerotic cardiovascular disease vs. those without had the following composite CV outcome of time to CV death, nonfatal myocardial infarction, nonfatal stroke and hospitalization for heart failure (HHF; incident rate [IR]/100 patient-years [PY] 6.9 vs. 3; hazard ratio [HR] 2.09; 95% confidence interval [CI] 1.89–2.30), composite outcome of time to CV death or HHF (IR/100 PY, 4.51 vs. 1.92; HR: 2.12; 95% CI 1.88–2.40) and composite kidney outcome (IR/100 PY 2.1 vs. 2.4; HR: 0.96; 95% CI 0.83–1.10) | 4/5/22 | Genitourinary/Sexual Function |
| Everest Medicines Ltd., of Shanghai | Nefecon | Oral, delayed-release formulation of budesonide | Primary IgA nephropathy | Top-line data from Chinese subpopulation showed reduction in proteinuria and stabilization of eGFR in NefIgard study | 4/5/22 | Genitourinary/Sexual Function |
| Prokidney LP, of Winston-Salem, N.C. | REGEN-006 and REGEN-016 | Renal autologous cell therapy | Chronic kidney disease | Expected to enroll up to 1,200 subjects in 2 phase III studies | 4/12/22 | Genitourinary/Sexual Function |
| Rockwell Medical Inc., of Wixom, Mich. | Triferic | Ferric pyrophosphate citrate | Hemodialysis | Final patient enrolled | 4/25/22 | 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 (OAB) | Post-hoc analysis data published in the International Journal of Clinical Practice showed significant reductions in urgency episodes and micturitions vs. placebo in both the OAB dry and wet populations | 4/19/22 | Genitourinary/Sexual Function |
| Argenx SE, of Breda, the Netherlands | Vyvgart (efgartigimod alfa-fcab) | FcRn blocker | Generalized myasthenia gravis | Interim results from ongoing, open-label ADAPT+ 3-year extension trial showed patients who continued on long-term treatment experienced consistent and clinically meaningful improvement on both the Myasthenia Gravis Activities of Daily Living and Quantitative Myasthenia Gravis scales; as of interim analysis, mean treatment duration was 363 days | 4/5/22 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Generalized myasthenia gravis | Follow-up results from Champion-Mg trial demonstrated long-term efficacy in adults, sustained through 60 weeks; statistically significant improvements from baseline MG-ADL total score (p<0.0001), quantitative myasthenia gravis total score (p<0.0001), improved quality of life (p<0.0001) and neurological fatigue subscale (p<0.0001); well-tolerated< td> | 4/6/22 | Immune |
| Gamida Cell Ltd., of Boston | Omidubicel | Advanced cell therapy | Allogeneic hematopoietic stem cell transplant | Data showed sustained clinical benefits in first year post-transplant; reduction in non-relapse mortality and no significant increase in relapse rates with omidubicel compared to UCBT (23% vs. 18%); rapid hematopoietic recovery; reduced rates of infections and no increase in GVHD rates compared with standard UCB; continued trend toward improved overall survival in favor of omidubicel arm over time (73% vs. 60%) | 4/27/22 | Immune |
| Maat Pharma SA, of Lyon, France | MaaT-013 | Off-the-shelf, standardized, pooled-donor, Microbiome Ecosystem Therapy | acute gastrointestinal graft-versus-host disease | Launched phase III trial; inclusion of first patient | 4/21/22 | Immune |
| Novartis AG, of Basel, Switzerland | Kesimpta (ofatumumab) | Anti-CD20 monoclonal antibody | Relapsing multiple sclerosis | Long-term data from Asclepios I/II trials and Alithios open-label extension showed drug maintained similar safety profile as seen in pivotal studies up to 4 years of treatment; participants who switched from teriflunomide to Kesimpta in extension phase demonstrated pronounced reductions in relapses and MRI lesions; in those receiving Kesimpta for up to 4 years, immunoglobulin G levels remained stable and mean immunoglobulin M levels decreased yet remained above the lower limit of normal, and no association between Ig levels and serious infection was observed | 4/5/22 | Immune |
| Arcturus Therapeutics Holdings Inc., of San Diego | ARCT-154 | mRNA vaccines targeting SARS-CoV-2 variants | COVID-19 prophylaxis | Top-line data showed study met its primary endpoint; 55% vaccine efficacy for protection against COVID-19 at 2 dose of 5-mcg; vaccine efficacy of 95% against severe (including fatal) COVID-19 disease; no reported cases of myocarditis or pericarditis | 4/20/22 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | Evusheld (tixagevimab/cilgavimab) | Combination of 2 long-acting monoclonal antibodies | COVID-19 | Results published in New England Journal of Medicine from Provent phase III study pre-exposure prophylaxis reduced the risk of developing symptomatic COVID-19 by 77% in the primary analysis (p<0.001) 83 at median follow-up of days; 83% relative risk reduction vs. placebo in the six-month analysis; no cases severe disease or covid-19 related deaths < td> | 4/20/22 | Infection |
| Bavarian Nordic A/S, of Copenhagen, Denmark | MVA-BN RSV | Recombinant vector vaccines | Respiratory syncytial virus infection | First participant dosed; planning to enroll 20,000 volunteers in U.S. and Germany; topline results expected mid-2023 | 4/20/22 | Infection |
| Entasis Therapeutics Inc., of Waltham, Mass., and Zai Lab Ltd., of Shanghai | Sulbactam-durlobactam (Sul-Dur) | Beta-lactam antibiotic; beta-lactamase inhibitor | Infections caused by Acinetobacter baumannii | In part A of Attack trial, primary efficacy endpoint of 28-day all-cause mortality in carbapenem- resistant Acinetobacter baumannii-calcoaceticus m-MITT cohort (n=125) was 19% (12/63) and 32.3% (20/62) for Sul-Dur vs. colistin, respectively; clinical cure rates at test-of-cure were 61.9% (39/63) and 40.3% (25/62), respectively; statistically significant reduction in nephrotoxicity observed vs. colistin (13.2% vs. 37.6%, p=0.0002) | 4/27/22 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Nucleotide derivative | COVID-19 | Post-hoc analysis of Pinetree phase III study showed drug significantly reduced the risk of hospitalization; statistically significant 87% reduction in risk for the composite primary endpoint of COVID-19-related hospitalization or all-cause death by day 28 (0.7% compared with placebo (5.3%) p=0.008); no deaths | 4/24/22 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Proxalutamide | Nonsteroidal antiandrogen | COVID-19 outpatients | Top-line data showed proxalutamide reduced the risk of hospitalization or death by 50% compared to the control group in 1 day of treatment; reduced the risk of hospitalization or death by 71% compared to the control group in more than 1 day of treatment; reduced the risk of hospitalization or death by 100% compared to the control group (p<0.02) 3 7 28 50 60 in more than days of treatment; significantly reduced the risk hospitalization death by100% (p<0.02), by 100% (p<0.02) patients or older; (p<0.02); no deaths older with without underlying medical conditions; and continuously sars-cov-2 viral load from day to compared control group (p<0.01 on 28, respectively); improved covid-19-related symptoms; well-tolerated< td> | 4/5/22 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J., and Ridgeback Biotherapeutics Inc., of Miami | Lagevrio (molnupiravir) | Oral antiviral | COVID-19 | Final analyses of virologic outcomes throughout and following 5-day course as part of phase III Move-Out trial showed, in participants with infectious virus isolated at baseline and for whom post-baseline infectivity data were available, treatment was associated with more rapid elimination of infectious virus than placebo; at day 3 of treatment, infectious SARS-CoV-2 was detected in 0.0% (n=0/92) vs. 21.8% (n=20/96) on placebo; at day 5, infectious virus was detected in 0.0% (n=0/91) vs. 2.2% (n=2/89) on placebo; at day 10, no infectious virus was detected in either arm for patients with infectious virus at baseline; molnupiravir was also associated with greater mean reductions from baseline in SARS-CoV-2 RNA than placebo from days 3 through 10, though both were associated with comparable rates of viral RNA clearance through day 29 | 4/1/22 | Infection |
| Mundipharma International Ltd., of Cambridge, U.K., and Cidara Therapeutics Inc., of San Diego | Rezafungin | Next-generation once-weekly echinocandin derivative | Candidemia and/or invasive candidiasis | Results from Restore study met primary endpoints; 23.7% all-cause mortality on day 30 for rezafungin compared to 21.3% for caspofungin; negative blood culture at 24 hours was 53.7% for rezafungin vs. 46.2% for caspofungin and 74.2% vs. 64.1% at 48 hours; median time to negative blood culture was 23.9 hours for rezafungin vs. 27 hours for caspofungin (p=0.175) | 4/25/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2 | COVID-19 | Initiated administration of the first booster doses in the pediatric expansion of the Prevent -19 study; to evaluate the safety and immunogenicity of a third dose in trial participants aged 12 through 17 | 4/22/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Pfizer-Biontech COVID-19 vaccine | mRNA vaccine | COVID-19 | Results published in The Lancet Respiratory Medicine showed booster dose provided strong protection against delta and omicron variants; vaccine effectiveness against omicron was 41% against hospital admission and 31% against emergency department visits at 9 months after 2 months; effectiveness against omicron-related hospitalization was 85% at less than 3 months but fell to 55% at 3 months or longer after 3 doses | 4/22/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative that contains 2 donatable thiol groups; anti-inflammatory agents | COVID-19 | Data safety monitoring board scheduled to evaluate current clinical and safety data; recommendations on the study due to positive efficacy based on other clinical outcomes evaluated such as the rate of sustained clinical resolution of symptoms of COVID-19 | 4/11/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative that contains 2 donatable thiol groups; anti-inflammatory agents | COVID-19 | Interim data published in Modern Rheumatology in Japanese patients with rheumatic diseases showed that antibody levels were significantly lower in the groups treated with TNF inhibitor compared with those treated with sulfasalazine and/or bucillamine or CNI (p<0.01) < td> | 4/18/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Severe fungal infections | Data from Furi and Cares study showed complete or partial responses or clinical improvement in 58.4% and 77.8% of particpants, respectively; 23.9% of participants achieved stable disease in Furi, while 11.1% hit that benchmark in Cares | 4/22/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Candida auris infections | Interim data of Cares study (n=18) showed 78% of patients showed complete or partial response; 11% had stable disease; 1 patient died of other causes and 1 outcome was indeterminate; mean duration of 18 days | 4/27/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Invasive candidiasis and candidemia | Interim analysis of combined data (n=49) patients showed 68% had complete or partial response; 14% had stable disease; 6% showed disease progression; 8% were indeterminate; and 4% died from progression of an underlying disease | 4/27/22 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Livtencity (maribavir) | Antiviral agent that inhibits the pUL97 protein kinase | Hematopoietic stem cell transplant recipients with cytomegalovirus infection/disease | Data from an exploratory analysis showed 31.9% of patients treated with Livtencity had at least one hospitalization vs. 36.8% of patients treated with conventional therapies, representing a 34.8% time-on-treatment-adjusted reduction vs. conventional therapy group (p=0.021); length of hospital stay during the treatment phase was 13.3 days/person/year in maribavir-treated patients vs. 28.7 for those who received conventional treatment, a 53.8% decrease (p=0.029) | 4/22/22 | Infection |
| Veru Inc., of Miami | Sabizabulin (VERU-111) | Cytoskeleton disruptor | COVID-19 patients at high risk for acute respiratory distress syndrome | Independent data safety monitoring committee unanimously recommended the study be halted early due to efficacy; treatment produced a 55% reduction in deaths compared to placebo (p=0.0029) | 4/11/22 | Infection |
| Timber Pharmaceuticals Inc., of Basking ridge, N.J. | TMB-002 | Topical rapamycin cream | Facial angiofibromas associated with tuberous sclerosis complex | Earlier in April, Nobelpharma America LLC's sirolimus topical gel was approved by the FDA for the indication; Timber said it will not proceed with a pivotal phase III as TMB-002 is intended for the same indication | 4/25/22 | Musculoskeletal |
| Avadel Pharmaceuticals plc, of Dublin | FT-218 (sodium oxybate controlled-release) | GABA B receptor agonist | Narcolepsy | Data from Rest-On study published in CNS Drugs showed clinically meaningful improvement in assessments of disrupted nighttime sleep compared to placebo in adults with narcolepsy; statistically significant decrease in the number of transitions from stages N1, N2, N3 and rapid eye movement (REM) sleep to wake and from N2, N3 and REM sleep to N1 (p<0.001 6 9 at all doses) and number of nocturnal arousals (p<0.05 g; p<0.001 7.5 g) compared to placebo; significantly improved sleep quality refreshing nature placebo (p<0.001)< td> | 4/6/22 | Neurology/Psychiatric |
| Harmony Biosciences Holdings Inc., of Plymouth meeting, Pa. | Pitolisant | Selective histamine 3 receptor antagonist/inverse agonist | Idiopathic hypersomnia | Initiated phase III study | 4/27/22 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ztalmy (ganaxolone) | Neuroactive steroid; positive allosteric modulator of the GABAA receptor | Seizures associated with CDKL5 deficiency disorder | Data published in The Lancet Neurology showed study met primary endpoint; showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for placebo (p=0.0036); well-tolerated; consistent safety profile | 4/14/22 | Neurology/Psychiatric |
| Taurx Pharmaceuticals Ltd., of Singapore | TRx-0237 | Agent targeting the tau pathology | Alzheimer’s disease | Last patient completed treatment in the blinded phase | 4/14/22 | Neurology/Psychiatric |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of muscle relaxant cyclobenzaprine hydrochloride | Fibromyalgia | First participant enrolled in the Resilient study; interim results expected in first quarter 2023 | 4/7/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution | Small-molecule modulator of RASP | Dry eye disease | Completed enrollment in Tranquility-2 trial; top-line results expected in second quarter 2022 | 4/5/22 | Ocular |
| Allergan, a unit of North Chicago-based Abbvie Inc. | Vuity (pilocarpine HCl ophthalmic solution, 1.25%) | Muscarinic cholinergic agonist | Presbyopia | Virgo trial met primary endpoint, improving near vision without compromising distance vision at hour 9 (3 hours after second drop) on day 14; data will serve as basis for supplemental NDA for optional twice-daily administration to U.S. FDA in second quarter 2022 | 4/5/22 | Ocular |
| Novaliq GmbH, of Heidelberg, Germany | Cyclasol | Topical anti-inflammatory and immunomodulating ophthalmic solution containing 0.1% cyclosporine A | Dry eye disease | Data from Essence-2 study showed consistent and superior therapeutic effects on the ocular surface in patients; 71.6% of patients showed a clinically meaningful improvement in total corneal fluorescein staining (tCFS) at 4 weeks (p values = 0.0022 and 0.0278 at day 15 and 29, respectively); excellent safety and tolerability profile; proportion of patients showing a clinically meaningful improvement in tear production (?10 mm increase) was statistically significantly higher compared to the vehicle at day 29; 71.6% of treated patients had a ?3 grade improvement in tCFS at week 4; proportion of responders was significantly higher compared to vehicle-treated patients (p = 0.0002); statistically significant improvements in a variety of symptoms including but not limited to dryness (p=0.0074) compared to non-responders at day 29 |
4/26/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Reverse pharmacologically-induced mydriasis | Results from MIRA-4 pediatric safety trial achieved primary endpoints; favorable safety and tolerability profile; no adverse events; no change in vital signs; 64% of Nyxol-treated participants had returned to baseline pupil diameter (PD) vs. 25% on placebo at 90 minutes post-dose; 82% of Nyxol treated subjects had returned to baseline PD vs. 33% on placebo at 3 hours post-dose; 91% of Nyxol treated subjects had returned to baseline PD vs. 51% on placebo at 24 hours post-dose; effective for all 3 dilating agents used and across light and dark irides | 4/28/22 | Ocular |
| Orasis Pharmaceuticals Ltd., of Herzliya, Israel | CSF-1 | Eye drops; pilocarpine hydrochloride 0.4% | Presbyopia | Study met its primary and key secondary endpoints on day 8; achieved statistically significant 3-line or more gain in distance-corrected near visual acuity; no loss of 1-line or more in distance visual acuity; 40% and 50% of participants demonstrated these gains 1-hour post-dose 1 and 1-hour post-dose 2 respectively (p<0.0001); 1 8 20 achieved statistically significant 3-line improvement at all measured time points on days and 15; or more in dcnva as early minutes up to hours post-dose day excellent tolerability safety profile; mild adverse events< td> | 4/21/22 | Ocular |
| Aeglea Biotherapeutics Inc., of Austin, Texas | Pegzilarginase | Recombinant human arginase 1 enzyme | Arginase 1 deficiency | Results from phase III patient-level outcomes analysis showed 65% of patients treated with pegzilarginase reached or exceeded prespecified response criteria for at least 1 mobility assessment compared to four patients (44%) receiving placebo; 47% met or exceeded prespecified clinical response criteria for at least two of the mobility outcomes compared to no patients receiving placebo; 6 of the patients met or exceede the clinical response threshold for at least 2 mobility outcomes and showed no worsening on any other mobility endpoint; post-hoc analysis showed improved GMFM-D score of patients from baseline by 2.25 units compared to placebo (p=0.0896); statistically significant biochemical improvements in measures of ornithine and guanidino compounds compared to placebo (p<0.0001,argininic acid)p="0.0003" (guanidinoacetic acid); p<0.0001 ( ?-keto-?-guinidinovaleric acid ) and p<0.0001 (?-n-acetylarginine)< td> | 4/11/22 | Other/Miscallaneous |
| Eli Lilly and Co., of Indianapolis | Tirzepatide | GIP and GLP-1 receptor agonist | Obesity | Top-line data showed tirzepatide met both co-primary endpoints of superior mean percent change in body weight from baseline and greater percentage of participants achieving body weight reductions of at least 5% compared to placebo for both estimands; achieved all key secondary endpoints at 72 weeks; achieved average weight reductions of 16%, 21.4% and 22.5% at 5 mg, 10 mg and 15 mg, respectively compared to placebo; 89% (5 mg) and 96% (10 mg and 15-mg) tirzepatide achieved at least 5% body weight reductions compared to 28% of placebo; 55% (10 mg) and 63% (15 mg) of tirzepatide achieved at least 20% body weight reductions compared to 1.3% of placebo; consistent overall safety and tolerability profile | 4/28/22 | Other/Miscallaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Pro-opiomelanocortin-derived peptide that is an agonist of melanocortin-4 receptor | Obesity | First patient enrolled in Emanate study; modifications include 4 independent substudies evaluating setmelanotide in patients with obesity due to a heterozygous variant of the POMC/PCSK1 genes, the LEPR gene, the SRC1 gene and the SH2B1 gene | 4/6/22 | Other/Miscallaneous |
| Fibrogen Inc., of San Francisco | Pamrevlumab | Connective tissue growth factor inhibitor | Idiopathic pulmonary fibrosis | Completed patient enrollment in first of 2 phase III study; 356 patients enrolled; top-line data expected in mid-2023 | 4/20/22 | Respiratory |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Smoking cessation | Top-line results from ORCA-2 trial at 3 mg showed clinically robust and statistically significant results in primary and secondary endpoints for both 6- and 12-week cytisinicline treatment compared to placebo; 6-8x increased odds of smoking abstinence with cytisinicline compared to placebo (p<0.0001); 3 9 24 abstinence rate during weeks 3-6 was 25.3% for cytisinicline compared to 4.4% placebo; well-tolerated with single-digit rates of adverse events observed; continuous from week 21.1% the 12-week arm 4.8% placebo, an odds ratio 5.3 (p<0.0001); 8.9% 6-week 2.6% 3.7 (p="0.0016) | 4/27/22 | Toxicity and Intoxication |
| Astex Pharmaceuticals Inc., of Pleasanton, Calif. | ASTX-727 | Oral decitabine and cedazuridine fixed-dose combination | Acute myeloid leukemia | Ascertain study met its primary endpoint | 5/12/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Untreated unresectable or metastatic urothelial carcinoma | Results from Checkmate-901 trial did not meet the primary endpoint of overall survival in combination with Yervoy (ipilimumab); no new safety signals; independent data monitoring committee recommended that the trial continue to assess other primary and secondary endpoints | 5/16/22 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China | Sugemalimab | Anti-PD-L1 monoclonal antibody | Non-small-cell lung cancer | Results of phase III Gemstone-301 study showed sugemalimab maintained a statistically significant and clinically meaningful improvement in PFS as assessed by blinded independent central review; clinical benefits in patients; well-tolerated and no new safety signals | 5/17/22 | Cancer |
| Curium Pharma UK Ltd., of London and Paris | Lu-177-PSMA-I&T | Intravenous radioligand therapy | Metastatic castration-resistant prostate cancer | Enrolled and dosed patients in the Eclipse study | 5/3/22 | Cancer |
| Genentech, a unit of Basel, Switzerland-based Roche Holding AG | Tiragolumab | Monoclonal antibody targeting TIGIT | PD-L1-high locally advanced or metastatic non-small-cell lung cancer | Skyscraper01 study did not meet its co-primary endpoint of progression-free survival in combination with Tecentriq (atezolizumab); co-primary endpoint of overall survival was immature; well-tolerated and no new safety signals | 5/10/22 | Cancer |
| IO Biotech ApS, of Copenhagen | IO102-IO103 | Cancer immunotherapy targets indoleamine 2,3-dehydrogenase (IDO) and PD-L1. | Melanoma | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 5/18/22 | 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 | Data from Emerald study met its primary endpoint of PFS in overall population and in ESR1 mutated patients; PFS rate at 12 months with elacestrant was 22.32% vs. 9.42% with SOC in the overall population, and 26.76% vs. 8.19% in the ESR1 mutation population; elacestrant significantly reduced risk of disease progression or death by 30% in all patients and by 45% in patients with ESR1 mutation; statistically significant PFS and reduced the risk of progression or death by 32% in the overall population and 50% in the ESR1 mutation population vs. fulvestrant; PFS was prolonged in all patients (p=0.0018) and those with ESR1 mutation (p=0.0005) | 5/20/22 | Cancer |
| Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | HR+/HER2-negative advanced or metastatic breast cancer | Follow-up of Monaleesa-3 study in combination with fulvestrant achieved a median overall survival of more than 5.5 years (67.6 months) compared to 51.8 months with fulvestrant alone; estimated survival rate at 5 years was 56.5% compared to 42.1%; no new adverse events | 5/4/22 | Cancer |
| Pierre Fabre Group, of Castres, France | Encorafenib (Braftovi) and binimetinib (Mektovi) | BRAF and MEK inhibitors | Resected stage II BRAF-mutant melanoma | First patient screened in Columbus-AD study; approximately 815 patients will be enrolled | 5/24/22 | Cancer |
| Sanofi SA, of Paris | Sarclisa (isatuximab-irfc) | Monoclonal antibody targeting an epitope on the CD38 receptor on multiple myeloma cells | Relapsed or refractory multiple myeloma | Data in combination with carfilzomib and dexamethasone (kd) showed median progression-free survival (mPFS) of 35.7 months compared to 19.2 months in patients treated with Kd alone; mPFS increased from 19.2 months to 35.7 months; serious treatment-related adverse events were higher in the Sarclisa combination therapy arm vs. Kd alone (70.1% vs. 59.8%) | 5/15/22 | Cancer |
| Sorrento Therapeutics Inc., of San Diego, and China Oncology Focus Ltd., an affiliate of Lee’s Pharmaceutical Holdings Ltd., of Hong Kong | Socazolimab/ZKAB-001 | Anti-PD-1 antibody | Extensive-stage small-cell-lung cancer | Completed enrollment with 498 patients; interim data expected in April 2023 | 5/17/22 | Cancer |
| Springworks Therapeutics Inc., of Stamford, Conn. | Nirogacestat | Oral gamma secretase inhibitor | Desmoid tumors | Top-line data of Defi trial met its primary and all secondary endpoints, with statistically significant improvement for nirogacestat over placebo; 71% reduction in the risk of disease progression (p<0.001); well-tolerated with a manageable safety profile< td> | 5/24/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Heart failure in patients with preserved ejection fraction | The Deliver study met its primary endpoint with Farxiga improving the primary composite endpoint of cardiovascular (CV) death or worsening heart failure; data expected to be submitted for presentation at a forthcoming medical meeting | 5/5/22 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Hypertrophic cardiomyopathy | Data from Redwood-HCM OLE study showed aficamten was associated with substantial reductions in the average resting LVOT-G (p<0.0001 1 2 12 24 at weeks) and (p="0.0003" weeks); no patients had a worsening of nyha class; 72% improved by class 7% classes weeks; 61% 17% week 24; significant improvements in cardiac biomarkers including ntpro-bnp (reduction 70% from baseline, p<0.001) troponin (20% reduction, p="0.002);" well-tolerated with temporary discontinuation due to lvef <50% down-titration< td> | 5/24/22 | Cardiovascular |
| Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Small-molecule cardiac myosin activator | Heart failure with reduced ejection fraction | Data showed an absolute risk reduction of 9.8 events per 100 patient-years interaction (p=0.051); improvements in blood pressure over time; incidence of treatment-emergent serious adverse events in patients with low blood pressure who received omecamtiv mecarbil (p<0.001) and adjudicated first stroke (p="0.009)" was lower compared to placebo< td> | 5/24/22 | Cardiovascular |
| Idorsia Ltd., of Allschwil, Switzerland | Aprocitentan | Dual endothelin receptor antagonist | Resistant hypertension | Data showed study met primary and secondary endpoints; statistically significant and clinically meaningful reduction in the primary endpoint measure of systolic blood pressure in 12.5-mg (p<0.005) and 25-mg (p<0.005) aprocitentan groups compared to placebo; well-tolerated; no major safety concerns< td> | 5/23/22 | Cardiovascular |
| Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson | Xarelto (rivaroxaban) | Oral factor Xa inhibitor | Peripheral/coronary artery disease | Results from Compass long-term open-label extension study in combination with aspirin (Xatoa) showed vascular protection in both patients; major bleeding rate had an incidence rate of 1.01 and randomized phase showed major bleeding of 1.67; results published in European Heart Journal, Cardiovascular Pharmacotherapy | 5/23/22 | Cardiovascular |
| Alvotech hf, of Reykjavik, Iceland | AVT-04 | Monoclonal antibody; biosimilar to Stelara (ustekinumab) | Moderate to severe chronic plaque-type psoriasis | Data showed study met its primary endpoint; no clinically meaningful differences in safety were observed through week 28 | 5/24/22 | Dermatologic |
| Bristol Myers Squibb Co., of New York | Deucravacitinib | Tyrosine kinase 2 inhibitor | Moderate to severe plaque psoriasis | Long-term extension study showed durable efficacy and a consistent safety profile in adult patients; clinical efficacy as maintained through up to 2 years of deucravacitinib treatment with response rates at week 60 of 77.7% and 58.7% for PASI 75 and sPGA 0/1, respectively | 5/12/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | Oral JAK1 and JAK2 inhibitor | Moderate to severe alopecia areata | Study met its primary and secondary endpoints with statistical significance in both the 8-mg twice-daily and 12-mg twice-daily dose groups relative to placebo; well-tolerated; greater scalp regrowth compared to placebo; SALT score of 20 or less was 41.5% in the 12-mg twice-daily dose group and 29.6% in the 8-mg twice-daily dose group compared to 0.8% of patients in the placebo group (p<0.0001)< td> | 5/23/22 | Dermatologic |
| Lipidor AB of Stockholm | AKP-02 | Cutaneous spray combination of calcipotriol and betamethasone dipropionate | Mild to moderate psoriasis | Half of target enrollment complete; last patient expected to begin treatment in June 2022; top-line data expected in Q3 2022 | 5/18/22 | Dermatologic |
| Polarityte Inc., of Salt Lake City | Skinte | Human cellular and tissue-based product derived from a patient’s own skin | Wagner grade 2 diabetic foot ulcers | First subject enrolled; planning to enroll 100 subjects at 20 clinical sites | 5/3/22 | Dermatologic |
| Inflarx NV, Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Hidradenitis suppurativa and ANCA-associated vasculitis | Halted phase III study for time-being | 5/12/22 | Dermatologic/Cardiovascular |
| Adocia SA, of Lyon, France, and Tonghua Dongbao Pharmaceutical Co. Ltd., of Tonghua, China | Biochaperone Lispro | Ultra-rapid insulin | Type 1 and type 2 diabetes | First patient dosed | 5/9/22 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran | RNAi therapeutic | Transthyretin-mediated amyloidosis | Data from 18-month analysis of exploratory Helios-A study showed improvement in NT-proBNP levels with an adjusted geometric fold change of 0.95 compared to 1.93 in the external placebo group (p=0.0004); improvement in echocardiographic parameters at month 18 relative to external placebo, including cardiac output (p=0.0426), LV end-diastolic volume (p=0.0607), global longitudinal strain (p=0.0781) and mean LV wall thickness (p=0.5397) | 5/23/22 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Oxlumo (lumasiran) | RNAi therapeutic targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | Exploratory study resulted in improvements in cardiac measures, nephrocalcinosis and kidney stone events; improvements in most burdensome symptoms; 2 patients remained stable; 3 improved (2 unilateral and 1 bilateral improvement) and none worsened following 6 months of treatment with lumasiran | 5/24/22 | Endocrine/Metabolic |
| Ascendis Pharma Inc., of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Pediatric growth hormone deficiency | Results from Visen's study achieved primary endpoint; greater annualized height velocity at 52 weeks (p=0.0010) compared to patients treated with daily growth hormone | 5/23/22 | Endocrine/Metabolic |
| Hua Medicine Co. Ltd., of Shanghai | Dorzagliatin | Glucokinase activator | Diabetes type 2 | Results from drug-naive patients (Seed study) and add-on therapy from Dawn study with metformin published in the Nature Medicine showed tha both studies effectively reduce HbA1c in patients; 1.07% and 1.02% HbA1c reduction from baseline in 24 weeks compared to placebo (p<0.001) 24 52 in seed and dawn study, respectively; standard-reaching rate of hba1c was 42.5% 44.4% weeks compared to placebo (p<0.001); 3.28 2.43 increase homa2-? with placebo; continued remain steady both studies; no drug-related saes< td> | 5/12/22 | Endocrine/Metabolic |
| Kamada Ltd., of Rehovot, Israel | Inhaled Alpha-1 Antitrypsin (AAT) therapy | Inhaled formulation of AAT administered using PARI GmbH eFlow electronic nebulizer | Alpha-1 antitrypsin deficiency | Independent data safety monitoring board recommended trial continue without modification; no patients discontinued; no drug-related serious adverse events; 9 patients at the Leiden site already completed the full two-year treatment period and are now under follow up; company expanding the study to new European sites | 5/17/22 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin capsule | Type 2 diabetes | Completed patient enrollment in ORA-D-013-1 study; enrolled 710 patients; top-line data expected in January 2023 | 5/3/22 | Endocrine/metabolic |
| Recordati SpA, of Milan, Italy | Isturisa (osilodrostat) | 11beta-hydroxylase inhibitor | Cushing’s disease | Data showed long-term mean urinary free cortisol (mUFC) control and clinical improvement; decrease in patient weight and the severity of physical features, maintained through to week 72; decrease in waist circumference 4.2% at week 48 and 5.8% at week 72; improvement in body mass of the patients from 30.3 kg/m2 at baseline to 28.4 kg/m2 (-4.6%) at week 48 and 27.9 kg/m2 (-5.8%) at week 72 | 5/13/22 | Endocrine/Metabolic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Evkeeza (evinacumab-dgnb) | Monoclonal antibody targeting angiopoietin-like 3 | Homozygous familial hypercholesterolemia | Study met its primary endpoint; reduced low-density lipoprotein-cholesterol (LDL-C) by 48% at week 24; 79% of patients reduced their LDL-C by at least half; absolute 132 mg/dL reduction in LDL-C from baseline; well-tolerated | 5/21/22 | Endocrine/Metabolic |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Hydrochloric acid binder | Metabolic acidosis in patients with chronic kidney disease | Stopped Valor-CKD renal outcomes trial early for administrative reasons pursuant to the existing study protocol to allow for 6 months of financial runway following the reporting of top-line results; expected in the fourth quarter of 2022 | 5/19/22 | Endocrine/Metabolic |
| Visen Pharmaceuticals Co. Ltd., of Shanghai | Lonapegsomatropin (Transcon hGH) | Once-weekly long-acting drug | Growth hormone deficiency | Top-line results of trial met its primary objective; lonapegsomatropin was observed to be noninferior and additionally superior to daily growth hormone on the primary endpoint of annualized height velocity (AHV) at 52 weeks; AHV for lonapegsomatropin was significantly greater than the daily hGH (p=0.0010); well-tolerated and with comparable safety to daily growth hormone | 5/23/22 | Endocrine/Metabolic |
| Xeris Biopharma Holdings Inc., of Chicago | Recorlev (levoketonconazole) | Cortisol synthesis inhibitor | Cushing’s syndrome | Results from randomized-withdrawal (RW) study met primary endpoint of significantly more patients on placebo having loss of mean urinary-free cortisol (mUFC) response than those who continued on levoketoconazole at end of RW phase; reversed loss of cortisol control in most patients; mean total and LDL cholesterol levels were significantly increased with placebo vs. levoketoconazole; increases were reversed during the restoration phase; no new safety signals | 5/12/22 | Endocrine/Metabolic |
| Xeris Biopharma Holdings Inc., of Chicago | Recorlev (levoketonconazole) | Cortisol synthesis inhibitor | Cushing’s syndrome | Result from patient-reported burden of illness study showed mean VAS score was 3.6 out of 10 indicates low pain; reported moderate/severe fatigue; moderate sleep and anxiety burden; moderate burden and health-related quality of life in patients | 5/12/22 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | Dasiglucagon | Glucagon analogue | Congenital hyperinsulinism | Top-line results in pediatric patients met the primary endpoint with statistical significance; reduced requirement for intravenous glucose by 55% compared to placebo in this pediatric patient population compared to placebo p=0.0037; well-tolerated | 5/19/22 | Endocrine/Metabolic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Crohn's disease | Results achieved the co-primary endpoints of endoscopic response and clinical remission, as well as the secondary endpoint of endoscopic remission at 1 year compared to placebo; 15-mg and 30-mg doses achieved clinical remission per the CDAI at week 52; clinical remission per CDAI of 37% and 48%, respectively vs. 15% in the placebo group (p<0.0001); 52 36% and 46% of patients who received upadacitinib 15-mg 30-mg doses, respectively, achieved clinical remission at week per sf ap compared to 14% in the placebo group (p<0.0001); 28% 40% endoscopic response 7% (p<0.0001) 52; no new safety risks< td> | 5/11/22 | Gastrointestinal |
| Eli Lilly and Co., of Indianapolis | Mirikizumab | Humanized IgG4 monoclonal antibody that binds to the p19 subunit of interleukin 23 | Ulcerative colitis | Results from Lucent-2 study achieved and maintained statistically superior and clinically meaningful improvements at 1 year compared to placebo across the primary endpoint of clinical remission and all key secondary endpoints at week 12; 49.9% of patient achieved clinical remission at 1 year compared to one-fourth of patients on placebo (p<0.001); 1 4 achieved resolution or near of bowel urgency severity at year compared to in on placebo (p<0.001); clinical response the 12-week induction study; statistically significant average reduction 3.80 2.74 for p<0.001< td> | 5/24/22 | Gastrointestinal |
| Pfizer Inc., of New York | Etrasimod | Selective S1P receptor modulator | Ulcerative colitis | Study achieved all primary and key secondary endpoints; clinical remission was 27% for patients receiving etrasimod compared to 7.4% for patients receiving placebo at week 12 (p=?0.001) and was 32.1% compared to 6.7% at week 52 (p=?0.001) | 5/24/22 | Gastrointestinal |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis in patients with systemic lupus erythematosus | Results from Aurora2 continuation study showed drug safe and well-tolerated; no new safety signals; overall rates of serious adverse events were similar in both the control (28%) and voclosporin (26%) arms; no increase in infectious events; significant and meaningful reductions in proteinuria achieved in Aurora 1 were maintained; significant difference in eGFR slope in favor of voclosporin (-0.2 mL/min/1.73 m2) compared to control arm (-5.4 mL/min/1.73 m2); mean urine protein creatinine ratio was lower in the voclosporin-treated groups at all time points during the 3 years; 4 deaths in active control group vs. none in voclosporin | 5/20/22 | 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 | Subgroup analysis of Empowur extension study showed safe and well-tolerated in adults 65 and older; sustained reductions from baseline in average daily micturition, urge urinary incontinence, episodes and urgency episodes; sustained and patient-perceived meaningful improvements at week 52 in both the OAB questionnaire and PGI scores | 5/16/22 | Genitourinary/Sexual Function |
| Anthos Therapeutics Inc., of Cambridge, Mass. | Abelacimab | Fully human monoclonal antibody with dual activity against factor XI and factor Xia | Venous thromboembolism (VTE) recurrence and bleeding in patients with cancer associated VTE | First patient enrolled in Aster study | 5/5/22 | Hematologic |
| Argenx SE, of Breda, the Netherlands | Vyvgart (efgartigimod alfa-fcab) | FcRn blocker | Generalized myasthenia gravis | The Advance trial met its primary endpoint with 21.8% of patients who received Vyvgart having a sustained platelet response compared to 5% of patients who received placebo (p=0.0316); top-line data for the subcutaneous version expected in first quarter 2023 | 5/5/22 | Immune |
| Astrazeneca plc, of Cambridge, U.K. | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Neuromyelitis optica spectrum disorder (NMOSD) | In the 58 patients followed for a median of 73 weeks in the Champion-NMOSD trial, no relapse and zero adjudicated relapses were observed among patients taking Ultomiris | 5/5/22 | Immune |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Lok | Antibiotic lock solution | Catheter-related bloodstream infections | Expanded additional sites oustide the U.S. | 5/6/22 | Infection |
| Evofem Biosciences Inc., of San Diego | Phexxi | Contraceptive vaginal gel comprising lactic acid, citric acid, and potassium bitartrate | Urinary tract infections (UTI) | New data of Ampower study showed significantly fewer UTI in the women compared to the general population | 5/6/22 | Infection |
| Inovio Inc., of Plymouth Meeting, Pa. | INO-4800 | DNA vaccine | COVID-19 | Discontinued Innovate study due to lower incidence of severe COVID-19 cases | 5/10/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Pfizer-Biontech COVID-19 vaccine | mRNA vaccine | COVID-19 | Top-line phase II/III data (n=1678) from third 3-µg dose in children 6 months to under 5 years of age met all immunobridging criteria required for emergency use authorization; well-tolerated; vaccine efficacy of 80.3% | 5/23/22 | Infection |
| Polypid Ltd., of Petach Tikva, Israel | D-Plex | Prolonged and constant release of broad-spectrum antibiotic doxycycline | Abdominal soft tissue surgical site infections | Data safety monitoring board recommended to conclude the study upon enrollment of 950th patient in Shield 1 study expected within days; top-line data expected in third quarter of 2022 | 5/23/22 | Infection |
| Sanofi SA, of Paris | Nirsevimab | Monoclonal antibody targeting respiratory syncytial virus (RSV) | Infants at high risk of RSV infection | Results from a prespecified pooled analysis of the pivotal phase III Melody and phase IIb study demonstrated an efficacy of 79.5% against medically attended lower respiratory tract infections, including hospitalizations caused by RSV (p<0.0001)< td> | 5/11/22 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Formulation for inhalation containing the broad-spectrum antiviral protein interferon-beta | COVID-19 | Post-hoc data from Sprinter trial showed well-tolerated; significantly reduced the risk of progression to severe disease and death compared to placebo by 70% (p=0.046) | 5/16/22 | Infection |
| Valneva SA, of Saint-Herblain, France | VLA-2001 | Inactivated COVID-19 vaccine | COVID-19 prophylaxis | Started a 150-patient heterologous booster trial testing VLA-2001 at least 6 months after vaccination with a licensed mRNA COVID-19 vaccine or following natural COVID-19 infection; top-line data expected in the third quarter of 2022 | 5/4/22 | Infection |
| Alvotech hf, of Reykjavik, Iceland | AVT-04 | Monoclonal antibody; biosimilar to Stelara (ustekinumab) | Inflammatory | Top-line data showed bioequivalence between AVT-04 and the reference products in healthy volunteers | 5/16/22 | Inflammatory |
| Paradigm Biopharmaceuticals Ltd., of Australia | Zilosul | Pentosan polysulfate sodium | Pain associated with osteoarthritis of the knee | First patient randomized and dosed in the PARA_OA_002 study; primary endpoint is change from baseline at day 56 in WOMAC pain; secondary outcomes include change from baseline at multiple time points out to day 168 in WOMAC pain and function, Patient Global Impression of Change and Quality of Life | 5/4/22 | Inflammatory |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Axial spondyloarthritis | Results from Be Mobile 1 and Be Mobile 2 studies met their primary and all ranked secondary endpoints at week 16 with statistical significance compared to placebo; 47.7% and 44.8% of bimekizumab-treated patients achieved ASAS40 vs. 21.4 % and 22.5% with placebo (p<0.001 1 16 for both) at week in be mobile and 2, respectively; 25.8 % 24% of bimekizumab-treated patients achieved asas-pr vs. 7.1% 7.2% with placebo (p<0.001) both studies; mean change from baseline basdai, basfi asqol studies< td> | 5/23/22 | Musculoskeletal |
| Abbvie Inc., of North Chicago | Vraylar (cariprazine) | Oral, once-daily atypical antipsychotic | Major depressive disorder | Study met its primary endpoint of statistically significant improvement using the MADRS total score in patients compared with placebo; statistically significant change from baseline to week 6 in the MADRS total score for patients treated with cariprazine at 1.5 mg/day compared with placebo (p= 0.0050); improvement in MADRS total score at week 6 compared to placebo, but did not meet statistical significance (p=0.0727) at 3 mg/day; consistent safety profile | 5/23/22 | Neurology/Psychiatric |
| Athira Pharma Inc., of Bothell, Wash. | Fosgonimeton (ATH-1017) | Small molecule designed to enhance the activity of hepatocyte growth factor and its receptor | Mild to moderate Alzheimer's disease | Reported extending the length of the current open-label extension study of Lift-AD and Act-AD study; study duration increased from 6 months to 18 months of open label treatment; top-line data from Lift-AD are targeted in the first half of 2023 and from ACT-AD by the end of the second quarter of 2022 | 5/9/22 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Troriluzole (BHV-4157) | Prodrug form of a glutamate modulating agent | Spinocerebellar ataxia | Study did not reach statistical significance in the overall patient population; troriluzole and placebo groups had mean baseline scores of 4.9 on the f-SARA; both groups showed minimal change at the 48-week endpoint with f-SARA scores of 5.1 and 5.2, respectively (p=0.76); greater numerical treatment benefit on the change in f-SARA score from baseline to week 48 compared to placebo (p=0.031); 58% reduction in the relative risk of falls in troriluzole-treated patients vs. placebo; favorable safety and tolerability profile | 5/23/22 | Neurology/Psychiatric |
| Bioxcel Therapeutic Inc., of New Haven, Conn. | BXCL-501 | Orally dissolving thin film formulation of dexmedetomidine | Agitation in patients with Alzheimer’s disease | First patient dosed; top-line data expected in fourth quarter 2022 or early first quarter 2023 | 5/3/22 | Neurology/Psychiatric |
| Karuna Therapeutics Inc., of Boston | Karxt | Combination of xanomeline and trospium chloride; muscarinic agonist and antagonist | Schizophrenia | Completed enrollment in the Emegent-2 study; data from Emergent-2 and Emergent-3 expected in the third quarter of 2022 and in the first quarter of 2023, respectively | 5/5/22 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ganaxolone | Positive allosteric modulator of GABAA receptors | Refractory status epilepticus | The Raise trial resumed screening and recruitment using new batches of the drug after particulates of aluminum phosphate were previously found in the drug solution; top-line results expected in the second half of 2023 | 5/5/22 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor | Tardive dyskinesia | Results showed substantial clinician-rated and self-rated global improvements of symptoms; stability of psychiatric symptoms was maintained | 5/23/22 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Fixed-dose combination of baclofen, naltrexone and sorbitol; target PMP22 expression | Charcot-Marie-Tooth disease type 1A | Data showed good tolerability and safety profile over the course; best efficacy signal was observed in the cohort of patients treated with PXT-3003 during 5 years; sustained treatment benefit | 5/16/22 | Neurology/Psychiatric |
| Scilex Holdings Inc., of Mountain View, Calif., and Sorrento Therapeutics Inc., of San Diego | Semdexa (dexamethasone sodium phosphate) | Epidural steroid injectable viscous gel forumlation | Lumbosacral radicular pain or sciatica | Study met its primary endpoint with a highly statistically significant reduction in average daily leg pain in patients vs. placebo (p<0.001); 4 57 99 median days to repeat injection in placebo group was vs. the sp-102 according kaplan-meier estimation (p<0.001); no identified safety risks; serious adverse events; 28% improvement quaity of life at weeks compared baseline, with minimal clinically meaningful 8%-12% (p< 0.001)< td> | 5/5/22 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Austedo (deutetrabenazine) | Synaptic vesicular amine transporter inhibitor | Postmenopausal women with tardive dyskinesia | Study showed mean change in total motor Abnormal Involuntary Movement Scale score; 61% and 68% of patients showed improvement at week 15 and week 145, respectively, in patient global impression of change; 63% and 82% of patients achieved much improved or very improved ratings at week 15 and week 145, respectively, in Clinical Global Impression of Change; no new safety signals | 5/23/22 | Neurology/Psychiatric |
| UCB SA, of Brussels, Belgium | Fintepla (fenfluramine) | Serotonin-releasing agent | Seizures associated with Lennox-Gastaut syndrome | Study published in JAMA Neurology met its primary endpoint; mean difference in the reduction of drop seizure frequency by 19.9% from placebo (p=0.001) at 0.7 mg/kg/day; median percent reduction in the frequency of drop seizures in the 0.7-mg/kg/day group was 26.5% compared with 14.2% in the 0.2-mg/kg/day group vs. 7.6% in patients taking placebo (p=.09); 50% or greater reduction in drop seizure frequency compared with patients in the placebo group; reduction in the frequency of generalized tonic-clonic seizures in 50% of patients; decrease in frequency in maintenance and titration period at 0.7-mg/kg/day group (p=0.001 and p<0.001, respectively); reduction in tonic or atonic seizure frequency was 46.7% compared with 6.8% the placebo group (p="0.046);" well-tolerated; improvement cgi-i scale investigators 26% vs. 20% 6% and caregivers 34% 27% 5% for 0.7 mg kg 0.2 placebo, respectively< td> | 5/3/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel (cannabidiol) | Transdermal permeation-enhanced gel formulation of cannabidiol | Fragile X syndrome | Ongoing long-term safety and efficacy study of Zygel in children and adolescents found drug well-tolerated; long-term administration with up to 38 months of exposure | 5/2/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon,Pa. | Zygel | Cannabidiol transdermal gel | Fragile X syndrome | Long-term data showed well-tolerated; median duration of exposure of 16 months (range 21 days to 38 months); achieved and maintained clinically meaningful change in ABC-CFXS social avoidance; demonstrated similar improvements in ABC-CFXS Irritability subscale scores | 5/11/22 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham | AR-15512 | TRPM8 agonist | Dry eye disease | First patient dosed; expected to enroll approximately 460 patients; top-line results of Comet study expected in the second half of 2023 | 5/24/22 | Ocular |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution | Small-molecule modulator of RASP | Dry eye disease | Post-hoc data from Tranquility trial demonstrated statistical significance (p=0.020) in favor of reproxalap over vehicle for the primary endpoint of reduction of ocular redness; top-line results from Tranquility-2 expected in Q2 2022 | 5/18/22 | Ocular |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Intravitreal pegcetacoplan | Targeted C3 therapy | Geographic atrophy secondary to age-related macular degeneration | Long-term data of Derby and Oaks study showed continuous and clinically meaningful reduction in the growth of both extrafoveal and foveal lesions at month 18 (all p-values nominal); reduced extrafoveal GA lesion growth in Oaks by 33% (p<0.0001) and 17% (p="0.0422)" with both monthly every-other-month treatment, respectively; derby by 23% reduced foveal ga lesion growth in oaks 18% 19% 9% 4% pegcetacoplan extrafoveal 26% (p<0.0001) 21% 13% respectively< td> | 5/2/22 | 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 associated with Meibomian gland dysfunction | Study met primary sign and symptom efficacy endpoints; well-tolerated; change from baseline in total Corneal Fluorescein Staining (tCFS) was statistically significant compared to the control saline group (p<0.001) at day 57; vas eye dryness score was statistically significantly improved compared to control group (p<0.001); tcfs and were also significant 15< td> | 5/3/22 | Ocular |
| Formosa Inc., of Cambridge, Mass. | APP-13007 | ophthalmic nanosuspension formulation of a clobetasol propionate(0.05%) | Inflammation and pain after cataract surgery | Top-line data met two primary endpoints by producing rapid and sustained clearance of ocular inflammation and cure of ocular pain which were statistically and clinically superior to placebo; 26.5% of subjects who showed sustained anterior chamber cell from post-operative day 8 through POD15 in the APP-13007 arm as compared to 5.1% in the placebo arm (p<0.001); 58.6% of subjects had acc count="0" on pod15 following app-13007 treatment as compared to 15.7% placebo (p<0.001); 68% showed sustained ocular pain grade="0" (pain free) early pod4 through in the arm 23% 77.3% were free at 43.7% well-tolerated< td> | 5/12/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Night vision disturbances | Top-line data showed U.S. FDA-agreed primary endpoint was met; statistically significant greater percentage of Nyxol-treated subjects having gained 15 or more letters of mesopic low contrast distance visual acuity (mLCVA) at day 8 compared to placebo (13% vs 3%; p<0.05); 8 10 15 secondary efficacy endpoints were also met with statistical significance; effect of nyxol increased at day 21% subjects gaining or more letters mlcva compared to 3% placebo (p<0.01); significantly the percentage both 41% vs. 22% (p<0.05) and 44% 23% (p<0.05); favorable safety tolerability profile; no serious adverse events< td> | 5/19/22 | Ocular |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif. | TP-03 | Topical ophthalmic solution of lotilaner 0.25% | Demodex blepharitis | Primary endpoint of complete collarette cure achieved in 56% of patients treated with TP-03 vs. 13% on vehicle (p<0.0001); secondary endpoints of mite eradication, complete lid erythema cure and composite all met; company plans to submit nda u.s. fda in q2 2022< td> | 5/2/22 | Ocular |
| Astrazeneca plc, of Cambridge, U.K., and Avillion LLP, of London | PT-027 (albuterol/budesonide) | Glucocorticoid receptor agonist; beta 2 adrenoceptor agonist | Asthma | Results from Mandala trial published in The New England Journal of Medicine demonstrated a statistically significant reduction in the risk of severe exacerbation vs. albuterol rescue in patients; reduced the risk of a severe exacerbation by 27% (p<0.001) in adults and adolescents; 33% reduction mean annualized total systemic corticosteroid exposure (p="0.002);" 24% severe exacerbation rate improvement symptom control quality of life< td> | 5/16/22 | Respiratory |
| Atyr Pharma Inc., of San Diego | Efzofitimod (ATYR-1923) | Immunomodulator; selective modulation of neuropilin-2 | Pulmonary sarcoidosis | Planning to initiate EFZO-FIT study in the third quarter of 2022 | 5/16/22 | Respiratory |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 | Resectable non-small-cell-lung cancer | Interim Aegean phase III study in combination with neoadjuvant chemotherapy showed statistically significant and meaningful improvement in pathologic complete response compared to neoadjuvant chemotherapy alone; statistically significant improvement in major pathologic response | 6/30/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | Data published in the New England Journal of Medicine showed Lynparza in combination with abiraterone significantly improved radiographic progression-free survival vs. abiraterone alone; reduced the risk of disease progression or death by 34% vs. abiraterone alone (p<0.0001)< td> | 6/21/22 | Cancer |
| Beigene Ltd., of Beijing, and Novartis AG., of Basel | Tislelizumab | Anti-PD-1 antibody | Advanced or metastatic esophageal squamous cell carcinoma | Data from Rationale 306 study in combination with chemotherapy showed statistically significant and clinically meaningful improvement in OS with a median OS of 17.2 months vs. 10.6 months; reduced the risk of death by 34%, p<0.0001 10 compared to chemotherapy plus placebo; median os for patients with pd-l1 score ?10% was 16.6 months in the tislelizumab group vs. receiving placebo, p="0.0020;" pfs 7.3 5.6 p<0.0001; orr 63.5% vs 42.4%; duration of response 7.1 5.7 months< td> | 6/30/22 | Cancer |
| Canariabio Inc., of Seoul, South Korea | Oregovomab | Murine IgG against CA 125 | Front-line advanced ovarian cancer | Reached 50% of target enrollment of 602 patients in combination with standard of care chemotherapy (carboplatin and paclitaxel) | 6/29/22 | Cancer |
| Corcept Therapeutics Inc., of Menlo Park, Calif. | Relacorilant | Nonsteroidal, selective modulator of the glucocorticoid receptor | Recurrent platinum-resistant ovarian cancer | Plans to start the 360-patient Rosella registrational study of relacorilant plus nab-paclitaxel at the end of June 2022; primary endpoint will be progression-free survival; overall survival will be a key secondary endpoint | 6/6/22 | Cancer |
| Corcept Therapeutics Inc., of Menlo Park, Calif. | Relacorilant | Nonsteroidal, selective modulator of the glucocorticoid receptor | Platinum-resistant ovarian cancer | Initiated Rosella study in combination with nab-paclitaxel; planning to enroll 360 participants | 6/29/22 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Datopotamab deruxtecan (Dato-DXd) | TROP2-directed DXd antibody-drug conjugate | Untreated locally recurrent inoperable or metastatic triple-negative breast cancer | First patient dosed | 6/13/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | XL-092 | Tyrosine kinase inhibitor | Metastatic colorectal cancer | Initiated Stellar-303 study; enroll approximately 600 patients | 6/21/22 | Cancer |
| G1 Therapeutics Inc., of Research Triangle Park, N.C. | Trilaciclib | I.V.-administered transient CDK4/6 inhibitor | Metastatic colorectal cancer | Last patient randomized; completed enrollment at 326 patients; initial data expected in early 2023 | 6/13/22 | Cancer |
| Medexus Pharmaceuticals Inc., of Toronto | Treosulfan | Bifunctional alkylating agent | Patients with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation | Data published in the American Journal of Hematology showed treosulfan was superior to reduced-intensity conditioning busulfan regimen for survival without disease recurrence, graft failure or death from any cause; overall survival for treosulfan was superior to busulfan; non-relapse mortality for treosulfan was lower than for busulfan | 6/6/22 | Cancer |
| Novocure Ltd., of St. Helier, Jersey, and Merck and Co. Inc., of New York | Tumor Treating Fields | Electric fields that penetrate cancer cell membranes | Locally advanced or metastatic intrathoracic non-small cell lung cancer | First patient enrolled to evaluate the safety and effectiveness in combination with Keytruda (pembrolizumab) | 6/21/22 | Cancer |
| Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Obstructive outflow disease/hypertrophic cardiomyopathy | New data from cohorts 1 and 2 in Redwood-HCM study in patients with severe left ventricular outflow tract (LVOT) obstruction showed 88% had associated systolic anterior motion (SAM) of the mitral valve, and 90% had mitral regurgitation; improved ventricular relaxation and filling (p<0.01); increase in lateral e’ (p<0.05); improved mitral valve dynamics; reduction the proportion of patients with sam (placebo: 92.3% at baseline to 75% week 10; aficamten: 85.7% 35.7% p="0.038" for comparison placebo); those eccentric regurgitation 25% 33.3% 42.9% 7.1% placebo) 10< td> | 6/13/22 | Cardiovascular |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast foam | Foam formulation of PDE4 inhibitor | Moderate to severe seborrheic dermatitis | Stratum trial met the primary endpoint, with 80.1% of patients treated with drug achieving Investigator Global Assessment Success at week 8 compared to 59.2% of patients treated with vehicle (p<0.0001); nda submission anticipated in the first half of 2023< td> | 6/6/22 | Dermatologic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | Moderate to severe plaque psoriasis | Top-line data showed clinically equivalent efficacy responses at 2 mg and 3 mg twice daily; statistically significant improvement when compared with placebo; PASI75 responses for 3-mg: 9.7% vs. placebo: 2.6% (P< 0.04); PASI75 (17% vs. 26.2%, respectively) and PASI50 (34.1% vs. 49.5%, respectively); piclidenoson was superior to Otezla (apremilast, Amgen Inc.) in the Psoriasis Disability Index (PDI) (20.5% vs. 10.3%, respectively, p<0.05)< td> | 6/29/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | JAK inhibitor | Moderate to severe alopecia areata | Patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 met primary efficacy endpoint with statistically significant differences (p <0.001) 24 relative to placebo in the percentage of patients achieving a ? 50% change from baseline at weeks using severity alopecia tool score; data published journal american academy dermatology< td> | 6/7/22 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Lebrikizumab | IL-13 inhibitor | Moderate to severe atopic dermatitis | Advocate 1 and 2 trials showed 80% of patients who acheived EASI-75 with lebrixizumab monotherapy at 16 weeks maintained skin clearance at 1 year of treatment with once every two weeks or four weeks regimen | 6/7/22 | Dermatologic |
| Galderma SA, of Lausanne, Switzerland | Nemolizumab | Humanized monoclonal antibody directed against IL-31 receptor | Prurigo nodularis | Data showed clinically and statistically significant improvement in both primary endpoints compared to placebo after 16 weeks of treatment; 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions compared to 11% in the placebo group (p<0.0001); 56% of nemolizumab-treated patients achieved an at least 4-point reduction in itch, as measured by the peak-pruritus numerical rating scale (pp-nrs) score, compared to 21% placebo group (p<0.0001); trial also met all key secondary endpoints; consistent safety profile< td> | 6/22/22 | Dermatologic |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Takhzyro (lanadelumab) | Fully human monoclonal antibody that binds with plasma kallikrein | Hereditary angioedema | Spring study showed Takhzyro reduced the rate of HAE attacks in children by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during treatment; majority of patients (76.2%) were attack-free during the 52-week treatment period, with an average of 99.5% attack-free days; no deaths or serious adverse events; no clinically meaningful safety findings | 6/30/22 | Dermatologic |
| Timber Pharmaceuticals Inc., of Basking Ridge, N.J. | TMB-001 | Topical isotretinoin formulated using IPEG delivery system | Congenital ichthyosis | First 4 patients enrolled in the Ascend trial that’s expected to enroll more than 140 patients; study will test once-a-day treatment for 3 weeks followed by 9 weeks of treatment twice-a-day compared to a matched vehicle; after 12 weeks, all patients will be switched to drug once- or twice-a-day for an additional 12 weeks; primary endpoint is change in investigator global assessment score at 12 weeks | 6/23/22 | Dermatologic |
| Diurnal Group plc, of Cardiff, U.K. | DNL-0200 | Hydrocortisone modified-release hard capsules | Congenital adrenal hyperplasia | First patient dosed in Connect study | 6/1/22 | Endocrine/Metabolic |
| Genexine Inc., of Seoul, South Korea | Eftansomatropin alfa (GX-H9/TJ-101) | Long-acting growth hormone | Pediatric and adult growth hormone deficiency | Completed patient enrollment | 6/14/22 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsværd, Denmark | Insulin icodec | Once-weekly insulin | Type 2 diabetes | Results from Onwards 1 (52 weeks) and Onwards 6 (26 weeks) achieved primary endpoint of demonstrating non-inferiority in reducing HbA1c at week 52/26 with insulin icodec vs. insulin glargine; Onwards 1 showed overall baseline HbA1c of 8.5% once-weekly insulin icodec achieved superior reduction in estimated HbA1c of -1.55% compared to -1.35% for insulin glargine; no statistically significant difference in estimated rates of severe or clinically significant hypoglycemia with 0.30 events per patient year exposed to once-weekly insulin icodec and 0.16 events per patient-year exposed to insulin glargine; Onwards 6 showed an overall baseline HbA1c of 7.6%, icodec achieved a reduction in estimated HbA1c of -0.47% compared to -0.51% for insulin degludec; statistically significant higher estimated rate of severe or clinically significant hypoglycaemia with 19.93 events per patient year exposed to once-weekly insulin icodec and 10.37 events per patient-year exposed to insulin degludec; safe and well-tolerated profile in both studies | 6/3/22 | Endocrine/Metabolic |
| Recordati SpA, of Milan, Italy | Isturisa (osilodrostat) | 11beta-hydroxylase inhibitor | Cushing’s disease | Linc4 study showed normal mean urinary free cortisol (mUFC) was 68.5% at week 48, 61.5% at week 72 and 72.4% at the end-of-treatment extension (EOT) visit; median mUFC decreased from 2.5x the upper limit of normal (ULN) (core baseline) to 0.5xULN at weeks 48 and 72, to 0.4xULN; Lin3 study showed an initial increase during the core phase; mean testosterone levels decreased toward baseline levels in females and stabilized in males during long-term treatment; hirsutism score improved or did not change from baseline in 63/76 patients at week 48 and 55/64 patients at week 72; few patients discontinued because of adrenal hormone precursor accumulation-related adverse events | 6/15/22 | Endocrine/Metabolic |
| 9 Meters Biopharma Inc., of Raleigh, N.C. | Larazotide | Tight junction regulator | Celiac disease | Prespecified interim analysis determined that the additional number of patients needed to determine a significant clinical outcome between placebo and larazotide is too large to support trial continuation; 0.5-mg significantly reduced symptoms of celiac disease | 6/21/22 | Gastrointestinal |
| Albireo Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | Pooled analysis of the Pedfic 1 and 2 trials showed patients whose pruritus improved on Bylvay had improvement in sleep and quality of life as measured by mean PEDsQL total scores (p=0.048) and PEDsQL FI total scores (p=0.007); serum bile acids and hepatic parameters also improved | 6/23/22 | Gastrointestinal |
| Astrazeneca plc, of Cambridge, U.K., | ALXN-1840 | Copper binder | Wilson disease | Focus trial met the primary endpoint by demonstrating 3x greater copper mobilization from tissues compared to the standard-of-care arm with a least square mean difference of 2.18 µmol/L (p< 0.0001); copper mobilization was seen at 4 weeks and was sustained through 48 weeks | 6/23/22 | Gastrointestinal |
| Gilead Sciences Inc., of Foster City, Calif. | Hepcludex (bulevirtide) | Viral entry inhibitor | Chronic hepatitis delta virus infection in adults with compensated liver disease | 48 weeks of 2 mg or 10 mg of Hepcludex dosed once daily produced a combined virological and biochemical response in 45% and 48% of patients, respectively, compared to 2% of patients who had not received antiviral treatment | 6/23/22 | Gastrointestinal |
| Intercept Pharmaceuticals Inc., of New York | Ocaliva (obeticholic acid) | Farnesoid X receptor agonist | Primary biliary cholangitis | Cobalt study did not demonstrate a statistically significant difference between Ocaliva and placebo on the primary endpoint; 71 subjects in the Ocaliva arm progressed to clinical events vs. 80 in the placebo arm (p=0.30); consistent safety and tolerability profile | 6/3/22 | Gastrointestinal |
| Palisade Bio Inc., of Carlsbad, Calif. | LB-1148 | Oral formulation of broad-spectrum serine protease inhibitor | Return of bowel function in adult patients undergoing gastrointestinal surgery | Initiated phase III study; U.S. clinical site is open; primary endpoint is time to recovery of the upper and lower gastrointestinal tract following surgery | 6/29/22 | Gastrointestinal |
| Apellis Pharmaceuticals Inc., of Waltham, Mass., and Swedish Orphan Biovitrum AB, of Stockholm | Pegcetacoplan | Complement C3 inhibitor | Primary immune-complex membranoproliferative glomerulonephritis and C3 glomerulopathy | First patient dosed in Valiant trial | 6/7/22 | Genitourinary/Sexual Function |
| Centessa Pharmaceuticals plc, of Boston | Lixivaptan | Vasopressin V2 antagonist | Autosomal dominant polycystic kidney disease | Company made the strategic decision to discontinue development; decision based on reassessment of commercial potential of lixivaptan following recent observation of ALT/AST elevations in ALERT Study | 6/2/22 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland, and Pfizer Inc., of New York | Myfembree (relugolix + estradiol + norethindrone acetate) | Once-daily tablet fixed-dose combination of a gonadotropin-releasing hormone receptor antagonist plus hormones | Moderate-to-severe pain associated with endometriosis | Results from phase III Spirit 1 and Spirit 2 study met co-primary endpoints; achieved a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at week 24, respectively (both p < 0.0001); clinically meaningful reduction in 59% and 66% of women compared with 40% and 43% of women in the placebo groups (p < 0.0001) in non-menstrual pelvic pain; achieved statistical significance, including reductions in dyspareunia and opioid use; well-tolerated safety profile; bone mineral density loss of < 1% over 24 weeks; published in The Lancet | 6/17/22 | Genitourinary/Sexual Function |
| Pharmaessentia Corp., of Burlington, Mass., and AOP Orphan Pharmaceuticals GmbH, of Vienna | Besremi (ropeginterferon alfa-2b-njft) | Monopegylated, long-acting interferon | Polycythemia vera | Final results from continuation-PV study showed reduction of the disease-causing mutated JAK2 allele burden below 1% in a sizeable proportion of patients (>20%); event-free survival over the entire 7.5-year treatment period was significant compared to control (p=0.04); minority of patients experienced disease-related symptoms | 6/20/22 | Hematologic |
| Rigel Pharmaceuticals Inc., of South San Francisco | Fostamatinib | Oral spleen tyrosine kinase inhibitor | Warm autoimmune hemolytic anemia | Top-line data showed trial did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population (p=0.398); no new safety issues; post-hoc data showed favorable durable hemoglobin response compared to placebo in U.S., Canadian, Australian and Western European trial sites | 6/8/22 | hematologic |
| Biogen Inc., of Cambridge, Mass., and Biothera Solutions Ltd., of China | BIIB-800 (BAT-1806) | Tocilizumab biosimilar | Moderate to severe rheumatoid arthritis | Results from 621 patients showed ACR20 response rates in BIIB-800 and tocilizumab group as about 69% vs. 64.8% at week 12 and 69.9% vs. 67.9% at week 24, respectively; comparable pharmacokinetics, safety and immunogenicity | 6/3/22 | Immune |
| Celltrion Healthcare Inc., of Incheon, South Korea | Remsima (infliximab biosimilar, subcutaneous) | TNF-alpha ligand inhibitor; TNF binding agent | Rheumatoid arthritis | Post-hoc analysis showed statistically significant greater improvements in clinical outcomes compared to the intravenous form at week 30, including low disease activity rates and remission rate; switching from intravenous to subcutaneous infliximab was associated with improvements in clinical response | 6/1/22 | Immune |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Active psoriatic arthritis | New data from Discover-2 study achieved consistent, long-term efficacy across domains of active PsA (joint, skin, enthesitis, dactylitis, spinal pain and disease severity endpoints), irrespective of baseline characteristics; 40% of patients treated every 8 weeks had achieved MDA, 59% achieved a Disease Activity Index for Psoriatic Arthritis (DAPSA)g score of ?14 and 24% achieved DAPSA ?4, indicating remission at week 100; maintained resolution of dactylitis and enthesitis through 2 years; low rates of radiographic progression through 2 years; no new safety concerns; consistent and durable improvements in pain with greater improvements relative to placebo; sustained improvements in self-reported HRQoLj and work productivity (WP) through 2 years; robust improvements in WP and daily activity at week 24 were maintained and increased through this time period | 6/1/22 | Immune |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Active psoriatic arthritis | Data from Cosmos study showed complete resolution of dactylitis was achieved in ?80% of patients at week 48 | 6/1/22 | Immune |
| Roivant Sciences Ltd., of Basel, Switzerland , and Pfizer Inc., of New York | Brepocitinib | Dual, selective inhibitor of TYK2 and JAK1 | Dermatomyositis and Lupus | Initiated phase III study | 6/28/22 | Immune |
| Sanofi SA, of Paris | Tolebrutinib | Brain-penetrant Bruton’s tyrosine kinase inhibitor | Multiple sclerosis and myasthenia gravis | U.S. FDA placed on partial hold; enrollment paused; participants who have completed at least 60-days in the trial can continue treatment in the U.S. | 6/30/22 | Immune |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy product | Immunosuppression | Enrolled 22 donor-recipient pairs in the Freedom-1 study; 7 patients dosed; achieved and maintained T-cell chimerism levels >50% at each of the 3-, 6- and 12-month timepoints post-transplant; 3 of the patients dosed more than 12 months prior to the data cutoff date have been successfully weaned off all chronic anti-rejection drugs | 6/30/22 | Immune |
| Xenikos BV, of Nijmegen, the Netherlands | T-Guard | Toxin-conjugated monoclonal antibodies that target CD3 and CD7 | Grade III or IV Steroid-refractory acute graft-vs.-host disease | First patient dosed following allogeneic hematopoietic stem cell transplant | 6/28/22 | Immune |
| Agenus Inc., of Lexington, Mass. | QS-21 Stimulon adjuvant | Saponin extracted from the bark of the Quillaja saponaria | Shingles | Interim data showed primary endpoint was exceeded with no unexpected safety concerns; statistically significant and clinically meaningful efficacy in adults aged 60 years and older | 6/10/22 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | Evusheld (tixagevimab/cilgavimab) | Combination of 2 long-acting monoclonal antibodies | COVID-19 | Data published in The Lancet Respiratory Medicine showed clinically and statistically significant protection against progression compared to placebo; reduced the relative risk of progressing to severe COVID-19 or death by 50% (p=0.010) through day 29 compared to placebo in non-hospitalized patients; reduced the risk of respiratory failure by 72% (p=0·036) vs. 11 placebo participants; well-tolerated | 6/8/22 | Infection |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland | Ceftobiprole medocaril | Prodrug of the active moiety ceftobiprole; cephalosporin antibiotic | Bacterial bloodstream infections caused by Staphylococcus aureus | Study (n=390) met primary and secondary efficacy endpoints; overall success rate was 69.8% with ceftobiprole compared to 68.7% with daptomycin, with or without aztreonam; no significant differences between the 2 treatment groups; well-tolerated | 6/28/22 | Infection |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | Capsid virus-like particle vaccine | COVID-19 prophylaxis | Trial redesigned to include Comirnaty (Pfizer Inc./Biontech SE) in the comparator arm of the study; planning to initiate trial in August 2022; to enroll 4,000 adult subjects; initial data in the end of 2022 | 6/16/22 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-196 and BRII-198/amubarvimab/romlusevimab | Human monoclonal antibody targeting the spike glycoprotein of SARS-CoV-2 | COVID-19 | Data showed safe and well-tolerated; no drug-related serious adverse events; RNA conversion rate (93% in treatment arm vs. 64% in placebo arm); median time to symptom resolution (8 days in treatment arm vs. 10 days in placebo arm); proportion of progression to severe cases (0% in treatment arm vs. 5% in placebo arm) | 6/9/22 | Infection |
| Bristol Myers Squibb Co., of New York | Abatacept (Orencia) | Selective modulator of a co-stimulatory signal required for full T-cell activation | COVID-19 | Results showed strong but not statistically significant improvement in the primary endpoint of time to recovery as measured by day of hospital discharge vs. placebo; reduced patient’s risk of death and improved their clinical status at 28 days; 11% vs. 15% for risk of death; 34.2% better odds of clinical improvement than in placebo; no new safety signals | 6/2/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 (CpG 1018/Alum) | Protein recombinant S-Trimer vaccine | COVID-19 | First subject dosed | 6/13/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 (CpG 1018/Alum) | Protein recombinant S-Trimer vaccine | COVID-19 | Data from individuals vaccinated with a third dose showed significant 19-fold increase in neutralizing antibody levels against the omicron BA.2 variant compared to pre-booster levels among baseline seronegative participants; 12-fold increase in neutralizing antibodies against the omicron BA.1 variant compared to pre-booster levels; favorable safety and tolerability profile | 6/27/22 | Infection |
| Merck & Co. Inc., of Rahway, N.J., and Ridgeback Biotherapeutics Inc., of Miami | Lagevrio (molnupiravir) | RNA polymerase inhibitor | COVID-19 | Analyses of prespecified exploratory endpoints in Move-Out trial indicated 7.2% of participants who received Lagevrio reported an acute care visit through Day 29, versus 10.6% of placebo participants, [CI, 4.4% to 51.7%]; 6.6% of participants who received Lagevrio reported a COVID-19-related acute care visit, versus 10% of placebo participants [CI, 5.6% to 53.6%] | 6/7/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1010 | Seasonal influenza vaccine | Influenza | First participants dosed in study expected to enroll about 6,000 adults in Southern hemisphere countries | 6/7/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.214 containing mRNA-1273 (Spikevax) | mRNA vaccine | COVID-19 | Data at 50-µg booster dose met all prespecified endpoints; well-tolerated; GMR with the lower bound of the confidence interval >1 was met; primary endpoints of noninferiority against ancestral SARS-CoV-2 were also met; booster dose of mRNA-1273.214 increased neutralizing geometric mean titers against omicron approximately 8-fold above baseline levels; binding antibody titers were also significantly higher against all other variants of concern (alpha, beta, gamma, delta, omicron) for mRNA-1273.214 when compared to mRNA-1273 | 6/8/22 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Vivjoa (oteseconazole) | lanosterol-14 demethylase inhibitor | Recurrent vulvovaginal candidiasis | Top-line data showed 85% completed 96 weeks without a recurrent VVC episode; average time without recurrence was 92 weeks; long-term reduction in the incidence of disease recurrence for women with RVVC | 6/22/22 | Infection |
| Relief Therapeutics Holding SA, of Geneva | Aviptadil (RLF-100) | Adrenergic receptor antagonist; VIP receptor agonist | COVID-19 | Discontinued trial for futility; company to continue development of aviptadil in other indications | 6/1/22 | Infection |
| Sanofi SA, of Paris and GSK plc, of London | Sanofi-GSK next-generation vaccine candidate | COVID-19 vaccine | COVID-19 | Data from VAT-02 cohort 2 study showed vaccine induced significant boost in antibody titers above baseline against multiple variants of concern (15-fold increase against D614 parent virus, 30-fold increase against beta strain) in adults previously primed with mRNA COVID-19 vaccines; 40-fold increase against BA.1; generated double the number of neutralizing antibodies against omicron BA.1 and BA.2 compared to the D614-based (original parent virus) booster; well-tolerated; favorable safety profile | 6/13/22 | Infection |
| Sanofi SA, of Paris and GSK plc, of London | Sanofi-GSK next-generation vaccine candidate | COVID-19 vaccine | COVID-19 | Data from COVIBOOST study following primary vaccination with 2 doses of Comirnaty generated a higher immune response; 76.1% for the Sanofi-GSK next-generation booster vs. 63.2% of Comirnaty vs. 55.3% for the Sanofi-GSK D614 (first-generation parent booster candidate); well-tolerated; favorable safety profile | 6/13/22 | Infection |
| Sanofi SA, of Paris and GSK plc, of London | SP-0253 | COVID-19 vaccine | COVID-19 | Data from Vat-08 trial showed efficacy of 64.7% against symptomatic COVID-19; 72% efficacy against omicron-confirmed symptomatic cases; favorable safety and tolerability profile | 6/24/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Brexafemme (ibrexafungerp) | 1,3-beta-glucan synthase inhibitor | Vulvovaginal candidiasis | First patient enrolled in 150-patient Vanquish study in patients who have failed treatment with fluconazole; study will test treatment regimens of 1, 3 or 7 days; primary endpoint is clinical cure at 14 days post baseline | 6/23/22 | Infection |
| Seres Therapeutics Inc., of Cambridge, Mass. | SER-109 | Oral microbiome therapy | Recurrent C. difficile infection | Overall safety profile observed in open-label ECOSPOR IV through 24 weeks indicated that SER-109 was well tolerated, consistent with the safety profile observed in the prior placebo-controlled ECOSPOR III study; Rolling BLA submission initiated and on track for mid-2022 completion | 6/7/22 | Infection |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-003 | Dengue vaccine | Dengue prophylaxis | Lomg-term exploratory analysis showed 84.1% vaccine efficacy against hospitalized dengue; 85.9% vaccine efficacy in seropositive individuals and 79.3% vaccine efficacy in seronegative individuals; well-tolerated; no reported safety risks | 6/9/22 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | 3-antigen prophylactic hepatitis B vaccine | Genetically engineered protein vaccine containing pre-S1, pre-S2 and S antigens | Hepatitis B virus infection | Follow-up study in participants after 2.5 years showed seroprotection rate of 88.1% for participants who recived VBI’s 3-antigen HBV vaccine (3A-HBV) vs. 72.4% for those who received Engerix-B (1A-HBV) in adult 18+ cohort; seroprotection rate of 90.3% 3A-HBV vs. 75 % 1A-HBV for adults 45-64 years; 81.8% 3A-HBV vs. 65.2% 1A-HBV for adults ? 65 years; 86.3% 3A-HBV vs. 69.6% 1A-HBV for obesity (BMI > 30); peak antibody titers at day 196 were 2.1x higher for 3A-HBV vs. 1A-HBV in the study; 2.5-year follow-up study, mean peak antibody titers were 5.5x higher in participants who received 3A-HBV vs. 1A-HBV | 6/28/22 | Infection |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Top-line data from Reopen2 study showed significant improvement in both symptoms and inflammation inside the sinuses; consistent safety and tolerability profile as per currently labeled safety profile | 6/13/22 | Inflammatory |
| Selecta Biosciences Inc., of Watertown, Mass. | SEL-212 | Combination medicine designed to sustain SUA levels | Chronic refractory gout | On track to enroll 140 patients by the end of second quarter 2022; top-line data from Dissolve I and II expected in first quarter of 2023 | 6/13/22 | Inflammatory |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Axial spondyloarthritis | 2 studies from phase III SELECT-AXIS 2 met the primary endpoint of assessment of spondyloarthritis international society 40% response criteria (ASAS40) at week 14 vs. placebo; SELECT-AXIS 2 non-radiographic axial spondyloarthritis study showed significantly more nr-axSpA patients achieved the primary endpoint of ASAS40 at week 14 with upadacitinib vs. placebo (45% vs. 23%; p<0.0001); 2 12 14 statistical significance was also achieved in the first of multiplicity-controlled secondary endpoints compared to placebo at week 14; select-axis ankylosing spondylitis (bdmard-ir) study, significantly more patients primary endpoint asas40 with upadacitinib vs. (45% 18%; p<0.0001); all no new safety risks< td> | 6/1/22 | Musculoskeletal |
| Acer Therapeutics Inc., of Newton, Mass. | Edsivo (celiprolol) | Beta-adrenoceptor antagonist | COL3A1-positive vascular Ehlers-Danlos syndrome | Initiated patient screening | 6/27/22 | Musculoskeletal |
| Biogen Inc., of Cambridge, Mass. | Tofersen (BIIB-067) | Antisense oligonucleotide binds to SOD1 mRNA | Amyotrophic lateral sclerosis | Results from 12-month data showed that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength, and quality of life; 33% and 21% reduction in SOD1 protein; 51% and 41% reduction in plasma neurofilament | 6/3/22 | Musculoskeletal |
| Cytokinetics Inc., of South San Francisco | Reldesemtiv | Next-generation fast skeletal muscle troponin activator | Amyotrophic lateral sclerosis | Initiated Courage-ALS OLE study to assess long-term safety and tolerability | 6/16/22 | Musculoskeletal |
| Fibrogen Inc., of San Francisco | Pamrevlumab | Inhibits the activity of connective tissue growth factor | Duchenne muscular dystrophy | Completed patient enrollment; 73 patients enrolled; top-line data expected in the second half of 2023 | 6/8/22 | Musculoskeletal |
| Italfarmaco Group, of Milan, Italy | Givinostat | HDAC inhibitor | Duchenne muscular dystrophy | Study met primary and secondary endpoint; slower decline to perform this functional task in the Givinostat-treated group p=0.0345; delayed fat infiltration by 30% p=0.035; mild to moderate in severity adverse events | 6/26/22 | Musculoskeletal |
| Jazz Pharmaceuticals plc., of Dublin | JZP-378/Nabiximols oromucosal spray | Complex botanical mixture formulated from extracts of the cannabis sativa plant and contains the cannabinoids delta-9-tetrahydrocannabinol and cannabidiol | Spasticity in individuals with multiple sclerosis | Top-line data of Release MSS1 trial did not meet the primary endpoint of change in Lower Limb Muscle Tone-6 (LLMT-6) between baseline and day 21 | 6/28/22 | Musculoskeletal |
| Kubota Vision Inc., of Seattle, a subsidiary of Kubota Pharmaceutical Holdings Co. Ltd. | Emixustat hydrochloride | Inhibits RPE65 | Macular atrophy secondary to Stargardt disease | Completed phase III study; last patient completed the study; database of this study will be locked in the third quarter 2022 | 6/24/22 | Musculoskeletal |
| Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | Radicava (edaravone) | Oral formulation of neuroprotectant | Amyotrophic lateral sclerosis | Results from 48-week study showed favorable safety profile; treatment-emergent adverse events (TEAEs) reported by ?10% of patients; no serious events; 8.6% discontinued the study due to TEAEs; average change from baseline in ALSFRS-R score was -11.3; at beginning of the study patients had an average ALSFRS-R score of 40 | 6/1/22 | Musculoskeletal |
| Alpha Cognition Inc., of Vancouver, British Columbia | ALPHA-1062 | Prodrug of galantamine | Mild to moderate Alzheimer’s disease | Top-line data confirmed in fed and fasted bioequivalence studies that ALPHA-1062 achieved bioequivalent area-under-the-curve and peak exposures relative to galantamine hydrobromide IR; no adverse events reported | 6/21/22 | Neurology/Psychiatric |
| Astrazeneca plc, of Cambridge, U.K., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen | Ligand-conjugated antisense medicine | Transthyretin-mediated amyloid polyneuropathy | Study met its co-primary endpoints in a planned interim analysis at 35 weeks; reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin (TTR) concentration; reduced TTR protein production; met its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy; significantly improved patient-reported quality of life vs. external placebo group; favourable safety and tolerability profile | 6/21/22 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-05 (dextromethorphan-bupropion) | Oral NMDA receptor antagonist with multimodal activity | Major depressive disorder | Gemini trial showed rapid, substantial and statistically significant improvement in symptoms of anhedonia compared with placebo; change from baseline to week 6 on the MADRS anhedonia subscale was significantly greater with AXS-05 than with placebo (-9.7 points vs. -7.22 points; p=0.001); improvement was rapid with the change on the MADRS anhedonia subscale from baseline to week 1, the first timepoint assessed, being significantly greater with AXS-05 than with placebo (-4.44 points vs. -2.69 points; p<0.001); at week 6, response on the anhedonia subscale was achieved by 54% of patients treated with axs-05 compared to 36% placebo (p="0.002) | 6/2/22 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Nurtec ODT (rimegepant) | CGRP receptor antagonist | Migraine | Data showed superior to placebo for both co-primary endpoints; pain freedom at 2 hours post-dose (19.8% vs. 10.7%, p<0.0001) and freedom from most bothersome symptom (50.5% vs. 35.8%, p<0.0001)< td> | 6/13/22 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Zavegepant | CGRP receptor antagonist | Migraine | Data showed superior to placebo for the co-primary endpoints: freedom from pain 2 hours post-dose (23.6% vs. 14.9%, p<0.0001) 2 15 30 48 and freedom from most bothersome symptom hours post-dose (39.6% vs. 31.1%, p="0.0012);" pain relief at minutes (15.9% 8%, p<0.0001) (58.7% 49.7%, return to normal function (10.5% 6.1%, (35.8% 25.6%, sustained (36.1% 29.6%, post-dose< td> | 6/13/22 | Neurology/Psychiatric |
| Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Excessive daytime sleepiness and cataplexy | Post-hoc analysis of pooled data from the Harmony 1 and Harmony CTP studies showed a Cohen's d effect size values for pitolisant vs. placebo were 0.80 for Epworth Sleepiness Scale, 0.31 for Maintenance of Wakefulness Test and 1.31 for weekly rate of cataplexy | 6/6/22 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ztalmy (ganaxolone) | Neuroactive steroid; positive allosteric modulator of the GABAA receptor | Refractory status epilepticus | Expanded enrollment criteria in Raise trial to allow for enrollment of patients previously treated with I.V. anesthesia, as well as patients transferred from other hospitals or treated in the emergency room | 6/8/22 | Neurology/Psychiatric |
| Neurelis Inc., of San Diego | Valtoco | Diazepam nasal spray | Seizure clusters in epilepsy | Post-hoc analysis showed mean SEIVAL clinically and statistically increased for day 271-360 compared with days 1-90 (p<0.001); 5 valtoco within the first minutes of seizure onset reduced time to cessation, total duration< td> | 6/7/22 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ongentys (opicapone) | Catechol-O-methyltransferase inhibitor | Sleep disturbances and "off" time in Parkinson's disease | Post-hoc analysis of data from 2 phase III studies showed 34.4% of the 964 participants experienced an "off" episode before going to sleep for a mean duration of 1.8 hours; 89.4% of participants experienced an "off" episode upon waking in the morning; mean duration of the time to morning "on" episode was 1.5 hours | 6/6/22 | Neurology/Psychiatric |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Rexulti (brexpiprazole) | Partial agonist of serotonin 5-HT1A and dopamine D2 receptors | Agitation in Alzheimer’s dementia | Results showed statistically significantly greater reduction in agitation compared to placebo; statistically significant difference (p=0.0026) in the mean change from baseline to Week 12 in the Cohen-Mansfield Agitation Inventory (CMAI) total score between brexpiprazole and placebo; statistically superior improvement on the key secondary endpoint of CGI-S, as related to agitation (p=0.0055); safe and well-tolerated; no new safety signals | 6/27/22 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland | Crenezumab | Monoclonal antibody designed to neutralize neurotoxic oligomers, a form of beta-amyloid | Alzheimer’s disease | Trial did not demonstrate a statistically significant clinical benefit in either of its co-primary endpoints; did not slow or prevent cognitive decline in people with a specific genetic mutation; no new safety signals | 6/16/22 | Neurology/Psychiatric |
| Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. | Zuranolone | GABAA receptor modulator | Postpartum depression | Study met its primary endpoint, showing statistically significant and clinically meaningful improvement in depressive symptoms at day 15 (p=0.0007); met secondary endpoints, showing improvement at days 3, 28 and 45 (p=0.0008, p=0.0203 and p=0.0067, respectively); well-tolerated; consistent safety profile | 6/1/22 | Neurology/Psychiatric |
| SK Life Science Inc., of Paramus, N.J. | Xcopri (cenobamate) | Positive allosteric modulator of the ?-aminobutyric acid (GABAA) ion channel | Uncontrolled focal seizures | Long-term study results published in Neurology showed median treatment duration was 54 months; 13% to 16% of patients achieved seizure freedom; 100% seizure reduction; median duration of seizure freedom during each 12-month interval was 48 months, 47.2 months and 45.1 months, respectively; median percent seizure frequency reduction over baseline that increased with each 6-month interval, up to 76% (IQR: 45%), during months 43-48 of the OLE; no new safety issues | 6/22/22 | Neurology/Psychiatric |
| Taurx Pharmaceuticals Ltd., of Singapore | Hydromethylthionine mesylate | Hydromethylthionine mesylate | Alzheimer's disease | Lucidity study (n=598) showed favorable safety profile; participants moved forward to an additional 1-year open-label phase | 6/1/22 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Pherine nasal spray | Social anxiety disorder | Last patient completed the study in Palisade phase III study | 6/22/22 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Anxiolytic | Social anxiety disorder | Last patient completed treatment in the Palisade-2 trial; top-line results expected in mid-2022 | 6/22/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol transdermal gel | Fragile X syndrome | Ongoing long-term study showed improvement in social avoidance in the full population; greatest improvement in patients with complete methylation of their FMR1 gene; well-tolerated | 6/1/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution | Small-molecule modulator of RASP | Dry eye disease | Trial achieved primary endpoint; statistically superior to vehicle for each of the 2 prespecified primary endpoints, Schirmer test (p=0.0001) and ?10 mm Schirmer test responder proportions (p<0.0001), after a single day of dosing< td> | 6/8/22 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating prostaglandin analogue | Open-angle glaucoma or ocular hypertension | Closed screening for additional patients in its Mont Blanc clinical tiral; top-line results in Nov.2022 | 6/3/22 | Ocular |
| Oculis SA, of Lausanne, Switzerland | OCS-01 | High concentration, preservative-free, topical optireach formulation of dexamethasone | Inflammation and pain after cataract surgery | First patient enrolled | 6/29/22 | Ocular |
| Regenerx Biopharmaceuticals Inc., of Rockville, Md. | RGN-259 | Sterile, preservative-free, eye drop | Neurotrophic keratopathy and dry eye disease | Planning to initiate 2 phase III trials | 6/22/22 | Ocular |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut | Epicutaneous patch | Peanut allergy | Study met its primary endpoint; statistically significant treatment effect (p<0.001), 12 with 67% of subjects in the viaskin peanut arm meeting treatment responder criteria after months compared to 33.5% placebo arm; consistent safety profile; trial completion rate was 84.8%; 3.3% patients discontinued due adverse events< td> | 6/8/22 | Other/Miscallaneous |
| Soleno Therapeutics Inc., of Redwood City, Calif. | Diazoxide choline extended-release | Once-daily crystalline salt of diazoxide | Prader-Willi syndrome | Data showed patients experienced improvements in body composition; statistically significant changes in lean body mass (p<0.0001); ratio of lean body mass to fat (p="0.0005)" based on dxa scanning; reduced levels leptin (p<0.0001), fasting insulin and an improvement in sensitivity (homa ir p="0.0033);" statistically significant increase adiponectin (p<0.0001)< td> | 6/13/22 | Other/Miscallaneous |
| AB Science SA, of Paris | Masitinib | Protein kinase inhibitor | Severe asthma | Data published in the Journal of Asthma and Allergy showed significant reduction in severe asthma exacerbations of 35% (p=0.0103); statistically significant reduction in rate of severe asthma exacerbations of 38% (p=0.0156) in prespecified subgroup of severe asthma patients with high eosinophil counts (? 150 cells/?L) | 6/7/22 | Respiratory |
| Verona Pharma plc., of London | Ensifentrine/RPL-554 | Dual inhibitor of the enzymes phosphodiesterase 3 and 4; CFTR activator | Chronic obstructive pulmonary disease | Mmore than 800 subjects have been randomized in Enhance-1 trial | 6/9/22 | Respiratory |
| ADC Therapeutics SA, of Lausanne, Switzerland | Zynlonta (loncastuximab tesirine) | CD19-targeted antibody-drug conjugate | Diffuse large B-cell lymphoma | First patient dosed in China in combination with rituximab | 7/11/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 | Unresectable liver cancer | Exploratory subanalysis from the Himalayas phase III study of Stride regimen with tremelimumab showed improved survival; liver function was stable over time for patients; improved quality of life | 7/1/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 | Advanced biliary tract cancer | Exploratory subanalysis from the Topaz-1 study in combination with standard-of-care chemotherapy showed consistent overall survival benefit compared to chemotherapy alone across all primary tumor locations | 7/1/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Anti-PD-1 antibody | Localized renal cell carcinoma | Results from checkmate-914 study in combination with Yervoy (ipilimumab) did not meet the primary endpoint of disease-free survival as assessed by blinded independent central review; consistent safety profile | 7/29/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabozantinib (Cabometyx) | Inhibitor of tyrosine kinases | Kidney cancer | Cosmic-313 study met its primary endpoint in combination of Opdivo (nivolumab, Bristol Myers Squibb Co.) and Yervoy (ipilimumab, BMS); improvement in progression-free survival; significantly reduced the risk of disease progression or death compared with the combination of nivolumab and ipilimumab (p=0.01); no new safety signals | 7/11/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Unresected locally advanced head and neck squamous cell carcinoma | Study did not meet its primary endpoint of event-free survival (EFS); improvement in EFS for patients at final analysis did not meet statistical significance per the prespecified statistical plan; consistent safety profile | 7/20/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J., and Astrazeneca plc, of Cambridge, U.K. | Lynparza (olaparib) | PARP inhibitor | Unresectable or metastatic colorectal cancer | Prespecified interim analysis as monotherapy and in combination with bevacizumab relative to control met the criteria for futility; both experimental arms will be discontinued; no new safety signals; consistent safety profile | 7/18/22 | Cancer |
| Philogen SpA, of Siena, Italy | Nidlegy | Immunocytokines L19IL2 and L19TNF | Melanoma | Enrolled 214 patients; recurrence-free survival as primary endpoint; overall survival, local recurrence-free survival and distant metastasis-free survival and safety as secondary endpoints | 7/25/22 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Perjeta (pertuzumab) | HER2-targeting antibody | HER2-positive early breast cancer | Results at 8.4 years median follow-up (101 months) showed 28% reduction in the risk of recurrence or death corresponding to an absolute benefit at 8 years of 4.9%; more patients disease-free than those treated with Herceptin, chemotherapy and placebo (88.4% and 85.8%, respectively, showing an absolute benefit of 2.6%) | 7/14/22 | Cancer |
| Sesen Bio Inc., of Cambridge, Mass. | Vicineum (oportuzumab monatox) | EpCAM-directed fusion protein engineered to deliver a cytotoxic payload of Pseudomonas exotoxin A | Non-muscle invasive bladder cancer | Voluntarily paused further development of Vicineum; includes the incremental development timeline and associated costs for an additional phase III study; company intends to seek a partner for the further development | 7/18/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX-250-CDx (89Zr-DFO-girentuximab) | Monoclonal antibody targeting carbonic anhydrase IX | Renal cancer | Completed recruitment; dosed 300 patients; exceeded target enrollment of 252 patients | 7/10/22 | Cancer |
| VBL Therapeutics Ltd., of Tel Aviv, Israel | VB-111 (ofranergene obadenovec) | Gene therapy | Platinum-resistant ovarian cancer | Top-line data showed Oval study did not meet the primary endpoints of achieving a statistically significant improvement in progression-free survival (PFS) or overall survival (OS); median PFS of 5.29 months vs. 5.36 months for the paclitaxel control arm (HR=1.03); median OS of 13.37 months vs. 13.14 months in the control arm (HR= 0.97); study discontinued | 7/19/22 | Cancer |
| Incarda Therapeutics Inc., of San Francisco | Inrhythm (flecainide, inhaled formulation) | Sodium channel protein type 5 subunit alpha blocker | Atrial fibrillation or recurrent paroxysmal atrial fibrillation | First patient enrolled; top-line study results expected in late 2023 | 7/26/22 | Cardiovascular |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Pelacarsen | Antisense medicine designed to inhibit the production of apolipoprotein(a) | Cardiovascular events in patients with cardiovascular disease | Completed enrollment of 8,325 study participants in Lp(a) Horizon study; top-line data expected in 2025 | 7/20/22 | Cardiovascular |
| Mesoblast Ltd., of New York | Rexlemestrocel-L (MPC-06-ID) | Allogeneic mesenchymal precursor cells | Chronic heart failure with reduced ejection fraction | Dream-HF study showed greater improvements in the prespecified analysis of left ventricular ejection fraction (LVEF) at 12 months relative to control (p=0.021); 65% reduction in 2-point MACE compared to controls (p=0.001); 33% reduction in 3-point MACE compared to controls (p=0.021); 68% reduction in the rate of recurrent hospitalizations from non-fatal heart attacks (p=0.0002); prespecified subgroup showed 86% greater increase in LVEF from baseline to 12 months compared to control (p=0.005); 79% and 45% reduction in 2-point and 3-point MACE compared to controls (p=0.004 and 0.012, respectively); reduced life-threatening mucosal bleeding events compared with controls 17% vs. 33% (p=0.02) | 7/19/22 | Cardiovascular |
| Amryt Pharma plc, of Dublin | Filsuvez (Oleogel-S10) | Herbals, dry birch bark extract combined with sunflower oil | Dystrophic epidermolysis bullosa | Data showed accelerated wound healing in patients; proportion of patients with target wound closure within 45 days was 44.3% on Oleogel-S10 vs. 29.6% on control gel (p=0.017); reduced pain associated with dressing changes (32% with Oleogel-S10 vs. 50% with control gel at day 90); 53% reduction in body surface area percentage with 15 months of Oleogel-S10 treatment in patients; EBDASI skin activity score reduced with continued Oleogel-S10 use from 19.9 at baseline to 14.8 after 15 months of treatment; no new safety signals | 7/7/22 | Dermatologic |
| Leo Pharma Inc., of Madison, N.J. | Adbry (tralokinumab) | Human monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Post-hoc analysis of 32-week results of Ecztra3 study published in American Journal of Clinical Dermatology with topical corticosteroids showed improvements in extent and severity ; 70.2% achieving improvement of 75% or more in the EASI-75 and 50.4% achieving EASI-90; 70.8% improvement in weekly average eczema-related sleep interference numeric rating scale compared to baseline; 66.8% improvement in dermatology life quality index compared to baseline | 7/20/22 | Dermatologic |
| Novan Inc., of Durham, N.C. | Berdazimer (SB-206) | Topical nitric oxide-releasing agent (10.3% gel) | Acne | Data from phase III B-simple4 study published in JAMA Dermatology showed favorable efficacy and safety; 32.4% of patients in the berdazimer group achieved complete clearance of lesions as compared to 19.7% in the vehicle group at week 12; low adverse events; 43% of patients had ?90% reduction from baseline in the number of molluscum lesions at week 12 compared with 23.9% of vehicle patients | 7/13/22 | Dermatologic |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor; oral, low-dose | Heterozygous familial hypercholesterolemia | Initiated phase III Brooklyn study in adult patients; first patient dosed; initial data in 2024 | 7/28/22 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsværd, Denmark | Insulin icodec | Once-weekly insulin | Type 2 diabetes | Results from Onwards 3 and Onwards 4 achieved primary endpoint of demonstrating noninferiority in reducing HbA1c at week 26 with insulin icodec vs. insulin glargine; Onwards 3 showed overall baseline HbA1c of 8.5%, achieved a superior reduction in estimated HbA1c of -1.57% compared with -1.36% for insulin degludec; Onwards 4 showed overall baseline HbA1c of 8.3%, reduction in estimated HbA1c of -1.16% compared with -1.18% for insulin glargine; no statistically significant difference in estimated rates of severe or clinically significant hypoglycemia (blood glucose below 3 mmol/L) with 5.64 events per patient-year exposed to once-weekly insulin icodec and 5.62 events per patient-year exposed to insulin glargine; safe and well-tolerated profile | 7/29/22 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin capsule | Type 2 diabetes | Enrolled and randomized more than 50% of planned 450 patients; primary endpoint of trial is to compare the efficacy of ORMD-0801 to placebo in improving glycemic control as assessed by A1c over a 26-week treatment period; secondary endpoint will compare ORMD-0801 to placebo in maintaining glycemic control over a 52-week treatment period | 7/26/22 | Endocrine/Metabolic |
| Valbiotis SA, of La Rochelle, France | Totum-63 | Combination of 5 plant extracts | Prediabetes to untreated type 2 diabetes | Completed recruitment of 600 volunteers in Reverese-IT study; to evaluate reduction of fasting blood glucose levels after 24 weeks as primary endpoint | 7/28/22 | Endocrine/Metabolic |
| Intercept Pharmaceuticals Inc., of Morristown, N.J. | Obeticholic acid | Semisynthetic bile acid derivative | Liver fibrosis due to nonalcoholic steatohepatitis | Top-line results from interim analysis of Regenerate study met the primary endpoint of achieving at least 1 stage of fibrosis improvement with no worsening of NASH at month 18 on liver biopsy compared with 9.6% of subjects on placebo (p<0.0001); demonstrated double the response rate in reduction of liver fibrosis without worsening nash vs. placebo< td> | 7/7/22 | Gastrointestinal |
| Palisade Bio Inc., of Carlsbad, Calif. | LB-1148 | Oral formulation of broad-spectrum serine protease inhibitor | Accelerate return of bowel function in patients undergoing gastrointestinal surgery | First patient screened; enrollment expected to complete within 18-24 months | 7/26/22 | Gastrointestinal |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Inhibits interleukin-4 and interleukin-13 | Eosinophilic esophagitis | Study met its primary endpoint of histological disease remission at 16 weeks with both higher and lower dose weight-tiered regimens in children ages 1 to 11; 86% reduction in peak esophageal intraepithelial eosinophil count from baseline compared to 21% increase for placebo (p<0.0001); 0.88 and 0.84 reduction from baseline in disease severity extent, respectively, as measured at the microscopic level biopsy specimens compared to a 0.02 0.05 increase for placebo (both p<0.0001); 3.5-point abnormal endoscopic findings 0.3-point (p<0.0001)< td> | 7/14/22 | Gastrointestinal |
| Albireo Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | Study met both primary endpoints, with statistically significant improvements in pruritus severity scores and reductions in serum bile acid levels; 55% for the all-Bylvay group (58% and 52% in the Bylvay 40- and 120-?g/kg/day groups, respectively) compared to 30% in the placebo group (p=0.0038); ?70% reduction from baseline in fasting serum bile acids or serum bile acids ?70 ?mol/L at week 24; 33% in the all-Bylvay group (including 43% and 21% of patients in the Bylvay 40- and 120-?g/kg/day groups, respectively), compared to no patients in the placebo group (p=0.0030); meaningful improvements in secondary endpoints related to pruritus and serum bile acids (p=0.0022); improved patient sleep; well-tolerated; no drug-related serious adverse events | 7/6/22 | Gastrointestinal |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | IONIS-FB-LRx | Ligand-conjugated antisense medicine designed to reduce the production of complement factor B | Immunoglobulin A nephropathy | Advanced to phase III study; achieved its primary endpoint of change in 24-hour urinary protein at 29 weeks compared to baseline in phase II study; favorable safety and tolerability profile | 7/11/22 | Genitourinary/Sexual Function |
| Genentech, of South San Francisco, a member of the Roche Group | Hemlibra (emicizumab) | Bispecific factor IXa- and factor X-directed antibody | Moderate or mild hemophilia A without factor VIII inhibitors | Data showed low treated bleed rates across the study period; 66.7% of participants showed no bleeds that required treatment; 81.9% experienced no spontaneous bleeds that required treatment; 88.9% experienced no joint bleeds that required treatment; consistent safety profile; no new safety signals | 7/11/22 | Hematologic |
| Novo Nordisk A/S of Bagsværd, Denmark | Concizumab | Anti-tissue factor pathway inhibitor antibody | Hemophilia A or B | Results from explorer7 study showed an 86% reduction in treated spontaneous and traumatic bleeds; estimated mean annualized bleed rate (ABR) of 1.7 compared to 11.8 with no prophylaxis; overall median ABR of concizumab was 0 compared to 9.8 for no prophylaxis; 63.6% patients experienced no treated bleeds, compared to 10.5% on prophylaxis; no thromboembolic events | 7/11/22 | Hematologic |
| Sanofi SA, of Paris | Fitusiran | Small interference RNA therapy | Hemophilia A or B | Data from Atlas-PPx study showed overall median annualized bleeding rate (ABR) was 0 for fitusiran prophylaxis compared to a median ABR of 4.4 with prior prophylaxis; statistically significant reduction in estimated ABR of 61.1% (p=0.0008) vs. factor or BPA prophylaxis; 63.1% of adults and adolescents treated with fitusiran experienced 0 treated bleeds compared to 16.9% (n=11) with prior factor or BPA prophylaxis | 7/11/22 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm, and Sanofi SA, of Paris | Efanesoctocog alfa (BIVV-001) | Recombinant factor VIII therapy | Severe hemophilia A | Results from Xtend-1 study in previously treated adults and adolescents ?12 years achieved primary and secondary endpoint; superior bleed protection (p<0.0001) 52 over prior factor viii prophylaxis with an estimated annualized bleeding rate (abr) reduction of 77% and a mean abr 0.69 compared to 2.96 on prophylaxis; statistically significant clinically meaningful improvements in physical health (p="0.0001)," pain intensity joint when comparing week baseline measurements; 96.7% bleeds were resolved single 50-iu kg dose; well-tolerated< td> | 7/11/22 | Hematologic |
| Akari Therapeutics plc, of New York | Nomacopan | Bispecific recombinant inhibitor of complement C5 and leukotriene B4 | Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy | Patient completed the course in part A study | 7/7/22 | Immune |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-109 | Polymer matrix-based epinephrine prodrug; oral drug | Anaphylaxis, severe allergy | Top-line data from the final 2 arms of part 3 of Epiphast study showed that administration of film sublingually 2 minutes after consuming a peanut butter sandwich had no statistical impact on the resulting pharmacokinetics when compared to previous data; Tmax of 12 minutes; high level of gastrointestinal absorption; Cmax of 286 pg/mL | 7/11/22 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes | Desensitization of highly sensitized adult patients prior to kidney transplant | First patient treated | 7/11/22 | Immune |
| Contrafect Corp., of Yonkers, N.Y. | Exebacase | Antistaphylococcal lysin agent | Staphylococcus aureus bacteremia | Data safety monitoring board recommended that the trial be stopped; conditional power of the study was below the prespecified threshold for futility | 7/13/22 | Infection |
| Gilead Sciences Inc., of Foster, Calif. | Biktarvy | Bictegravir 50-mg/emtricitabine 200-mg/tenofovir alafenamide 25-mg tablets | HIV/hepatitis B virus infection | Interim data from Alliance trial showed superior HBV DNA suppression vs. dolutegravir+emtricitabine/tenofovir disoproxil fumarate (63% vs. 43%, p=0.0023); hepatitis B e-antigen (HBeAg) seroconversion (23% vs. 11%, p=0.031) at week 48; high rates of HIV suppression at week 48 (95% vs. 91%; p=0.21) with mean CD4 cell count increases; higher hepatitis B surface antigen (HBsAg) loss (13% vs. 6%, p=0.059), HBeAg loss (26% vs. 14%, p=0.055) and alanine aminotransferase normalization (73% vs. 55%, p=0.066); long-term use of Biktarvy showed no significant changes to metabolic, bone and renal markers | 7/28/22 | Infection |
| Glenmark Pharmaceuticals Ltd., of Maharashtra, India, and Sanotize Research and Development Corp., of Vancouver, British Columbia | Nitric oxide nasal spray (Fabispray) | Nasal spray formulation | COVID-19 | Results published in The Lancet Regional Health Southeast Asia showed study met key endpoints in India; significant reduction in viral load within 24 hours; sustained over 7 days of treatment; 93.7% reduction in 24 hours and for 99% within 48 hours of treatment; safe and well-tolerated | 7/13/22 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | COVID-19 | Data published in Thorax showed improved clinical outcomes in hospitalized non-mechanically ventilated hypoxic COVID-19 patients; reduced CRP level below 150 mg/L; likelihood of survival without mechanical ventilation was achieved in 90% of LIVE-AIR patients treated with lenzilumab plus standard of care compared to 79% treated with placebo plus standard of care (p=0.0009); 62% relative reduction in the risk of progression to invasive mechanical ventilation or death (OR=0.38; p=0.0053) | 7/6/22 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Vivjoa (oteseconazole) | lanosterol-14 demethylase inhibitor | Recurrent vulvovaginal candidiasis | Results published in the NEJM Evidence showed 2 global Violet phase III studies met primary and key secondary endpoints; 93.3% and 96.1% of women with RVVC who received Vivjoa did not have a recurrence, compared to 57.2% and 60.6% of patients who received placebo (p<0.001), 1 48 respectively; significantly lower averaged percentage of occurrence or more positive candida cultures through week compared with the placebo groups (29.4% vs. 84.2% in violet trial 1, p<0.001; and 27.6% 84.0% 2, p<0.001)< td> | 7/26/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory drug | Mild to moderate COVID-19 | Unblinded the predose selection data to support the amended study protocol with the new primary efficacy endpoints; rate of sustained clinical resolution of symptoms of COVID-19 as new primary endpoint | 7/25/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Glucan synthase inhibitor | Recurrent vulvovaginal candidiasis | Data from Candle open-label study (n=24) showed 17/24 patients achieved a significant reduction or elimination of signs and symptoms after treatment with ibrexafungerp; favorable clinical response in subset study in 80% of patients | 7/19/22 | Infection |
| Veru Inc., of Miami | Sabizabulin (oral 9 mg) | Cytoskeleton disruptor; antiviral and anti-inflammatory agent | Moderate to severe hospitalized COVID-19 | Results published in The New England Journal of Medicine Evidence showed study met primary and secondary endpoints; study halted early due to clear efficacy benefit; clinically and statistically meaningful 55.2% relative reduction in deaths (p=0.0042) in the intent-to-treat population; placebo group had a 45.1% mortality rate compared to the sabizabulin-treated group, which had a 20.2% mortality rate; mortality through day 29 with a placebo mortality rate of 35.3% compared to sabizabulin treatment mortality rate of 17%; absolute reduction of 18.3% points and a relative reduction in deaths of 51.8%; 43% relative reduction in days in ICU (p=0.0013); 49% relative reduction in days on mechanical ventilation (p=0.0013); 26% relative reduction in days in hospital (p=0.0277) compared to placebo group; lower adverse and serious adverse events; well-tolerated | 7/6/22 | Infection |
| Viiv Healthcare Ltd., of London | Rukobia (fostemsavir tromethamine) | HIV gp120 protein inhibitor | HIV-1 infection | Results from Brighte study showed durable virologic responses and continued clinically meaningful improvements in CD4+ cell count and CD4+/CD8+ ratio at week 240; consistent safety profile | 7/29/22 | Infection |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Preplanned exploratory secondary analysis of Reopen2 study showed larger improvements than patients receiving placebo comparator at the 4-week-time point on each of the 4 individual defining symptoms; greater improvement on the sinonasal outcomes test; greater reduction in sinus opacification in this subgroup (-6.31% vs -1.55%, nominal p-value of 0.004); improvement in quality of life | 7/8/22 | Inflammatory |
| Optinose Inc., of Yardley, Pa. | Xhance | Fluticasone propionate nasal spray | Chronic sinusitis | Preplanned analysis of pooled data from Reopen study showed statistically significant reduction of 66% in the incidence of exacerbations for patients receiving 2 sprays twice daily of Xhance compared to placebo comparator | 7/13/22 | Inflammatory |
| Aytu Biopharma Inc., of Englewood, Co. | AR-101 (enzastaurin) | PKC-beta inhibitor | Vascular Ehlers-Danlos syndrome | Initiate phase III Prevent study; initiated patient identification and study site contracting | 7/20/22 | Musculoskeletal |
| Capricor Therapeutics Inc., of San Diego | Cap-1002 | Allogeneic cardiosphere-derived cells | Late-stage Duchenne muscular dystrophy | First patient dosed; planning to enroll 70 patients in the U.S. | 7/19/22 | Musculoskeletal |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | Small molecule; p38 inhibition | Facioscapulohumeral muscular dystrophy | First patient dosed in Reach study | 7/5/22 | Musculoskeletal |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD | Allogeneic mesenchymal-like cells | Muscle injury following arthroplasty for hip fracture | Top-line data of PLX-PAD showed an effective accelerator of muscle strength and regeneration; significant increase in Hip Abduction Strength (HAS) at week 26 and week 52 in injured leg of the patients (p=0.047, p=0.0022) and uninjured leg (p=0.073, p=0.0046) compared to placebo; study did not meet the primary endpoint, short physical performance battery (SPPB) test at week 26; well-tolerated; patients able to walk 296 meters vs. 266 meters in placebo | 7/13/22 | Musculoskeletal |
| Tris Pharma Inc., of Monmouth Junction, N.J. | Dyanavel XR (amphetamine) | Extended-release once-daily tablets | Attention deficit hyperactivity disorder | Results published in the Journal of Clinical Psychiatry showed improvement in symptoms over patients who took placebo; statistically significant vs. placebo in primary endpoint (p=0.0043); increase in PERMP-T scores 0.5 hours (p=0.01), 1 hour (p< 0.001), 2 hours (p= 0.0003), 4 hours (p=0.031), 8 hours (p=0.026), and 13 hours (p=0.006) post dose compared with placebo; well-tolerated | 7/22/22 | Musculoskeletal |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Taldefgrobep alfa (BMS-986089) | Modified adnectin designed to specifically bind to myostatin (GDF-8); fully human anti-myostatin recombinant protein | Spinal muscular atrophy | Enrolled first patient | 7/7/22 | Neurology/Psychiatric |
| Kowa Pharmaceuticals America Inc., of Montgomery, Ala. | Seglentis | Co-crystal combination of celecoxib 56 mg and tramadol hydrochloride 44 mg | Moderate to severe acute pain | Study met primary efficacy endpoint; significantly greater effect on summed pain intensity difference from 0 to 48 hours than tramadol hydrochloride or celecoxib individually or placebo (p<0.001); no serious events; similar safety profile of tramadol< td> | 7/19/22 | Neurology/Psychiatric |
| Mindbloom Inc., of New York | Ketamine | NMDA receptor antagonist | Moderate to severe anxiety and depression | Data from 1,247 participants through telehealth platforms showed 89% reported improvement in depression or anxiety symptoms; 63% of participants experienced a greater than 50% reduction; 30% achieved remission; 62% of participants who reported suicidal ideation at baseline no longer reported any suicidal ideation after 4 sessions; less than 5% of participants reported side effects from treatment; results published in the Journal of Affective Disorders | 7/18/22 | Neurology/Psychiatric |
| Neurelis Inc., of San Diego | Valtoco | Diazepam nasal spray | Seizure clusters in epilepsy | Results published in Epilepsy & Behavior showed stable or increased quality of life, clinical important improvement during the 12-month study, reduced seizure worry and improved social functioning | 7/20/22 | Neurology/Psychiatric |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Hyqvia (human normal immunoglobulin 10% and recombinant human hyaluronidase) | Subcutaneous immunoglobulin replacement therapy | Chronic inflammatory demyelinating polyradiculoneuropathy | Top-line data showed study met its primary endpoint; reduced relapse of neuromuscular disability and impairment compared to placebo (9.7% vs. 31.4%, respectively; p= 0.0045); favorable safety profile; mild to moderate adverse events | 7/21/22 | Neurology/Psychiatric |
| Vertex Pharmaceuticals Inc., of Boston | VX-548 | NaV1.8 inhibitor | Acute and neuropathic pain | Advanced to phase III study in the fourth quarter 2022 | 7/22/22 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Anxiolytic | Social anxiety disorder | Top-line results showed Palisade-1 study did not achieve primary endpoint; favorable and consistent tolerability profile; no serious adverse events | 7/22/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap ophthalmic solution | Small-molecule modulator of RASP | Dry eye disease | Study achieved primary and secondary endpoints; statistically superior to vehicle for each of the 2 prespecified primary endpoints, ocular redness in a dry eye chamber (p=0.0004) and Schirmer test (p=0.0005); statistically lower than vehicle for the secondary endpoints of ocular dryness (p=0.0068), discomfort (p<0.0001), 1 2 grittiness (p="0.0001)," stinging burning (p<0.0001) and itching symptoms; well-tolerated; no treatment-emergent moderate or serious adverse events; safety signals; mild transient instillation site discomfort; patients discontinued due to events of vehicle reproxalap< td> | 7/12/22 | Ocular |
| Arctic Vision Biotechnology Co. Ltd., of Shanghai | ARVN-003 | Pilocarpine formulation based on microdosing platform Optejet | Presbyopia | First patient enrolled | 7/4/22 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq (GS-010) | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 gene | Leber hereditary optic atrophy | Data from 5 years of follow-up in Restore study showed patients continued to experience significantly improved vision with 1-time injection of the gene therapy treatment; improved best-corrected visual acuity (BCVA) for a substantial proportion of the study participants; 71% of Restore subjects achieved clinically relevant recovery against nadir 5 years after treatment; 80.7% of them had on-chart vision (BCVA ? 1.6 LogMAR) in 1 or both eyes; well-tolerated; no serious adverse events; no discontinuations occurred due to ocular events; no systemic serious adverse events or discontinuations related to study treatment | 7/20/22 | Ocular |
| Iveric Bio Inc., of Parsippany, N.J. | Zimura (avacincaptad pegol) | Targets and inhibits the cleavage of complement protein C5 | Geographic atrophy | Post-hoc analysis of Gather1 study showed 84% of patients had lesions within 500 microns of the foveal center point at baseline; 28% of patients had lesions within 100 microns of the foveal center point at baseline; reduction of geographic atrophy lesion growth compared to sham; no drug-related adverse events | 7/16/22 | Ocular |
| Roche Holding AG, of Basel, Switzerland, and its Genentech subsidiary | Vabysmo (faricimab) | Bispecific antibody for the eye targeting Ang-2 and VEGF-A | Neovascular age-related macular degeneration | 2-year data from Tenaya and Lucerne studies showed Vabysmo achieved comparable vision gains vs. aflibercept given every 2 months; reductions in central subfield thickness; no new safety signals; well-tolerated; favorable benefit-risk profile | 7/14/22 | Ocular |
| Vasopharm GmbH, of Würzburg, Germany | Ronopterin (formerly VAS-203) | iNOS inhibitor | Traumatic brain injury | Ronopterin significantly decreased brain microdialysate lactate levels with a lower proportion of lactate levels ? 2.5 mmol/L (70% vs. 76%, p=0.001) after early infusion ? 12 hours; significant reduction was predominant during the actual 2-day infusion phase (63% vs. 76%, p<0.0001) and on the first day after infusion phase (65% vs. 84%, p<0.0001); reduced glutamate-induced lactate production (p<0.0001)< td> | 7/12/22 | Ocular |
| Innocoll Pharmaceuticals Ltd., of Athlone, Ireland | Posimir | Bupivacaine solution for infiltration use | Arthroscopic subacromial decompression | Data showed Posimir at 5 ml reduced mean postoperative pain on movement over 72 hours by 1.3 points on a 0-10 scale compared with vehicle control's relative reduction of 20% (p=0.012); simultaneous improvements in postoperative opioid use during the same 72-hour period, and observed pain reduction was clinically meaningful; median time to first request for opioid rescue medication was 12.4 hours among patients treated with Posimir compared with 1.2 hours in patients who received vehicle control (p=0.014); median total opioid consumption in intravenous morphine milligram equivalents over 72 hours was 4 mg in the Posimir group vs. 12 mg in the vehicle control group (p=0.010); proportion of patients who were opioid-free at 72 hours was 40% in the Posimir group vs. 16% in the vehicle control group (p=0.027) | 7/6/22 | Other/Miscallaneous |
| Sound Pharmaceuticals Inc., of Seattle | SPI-1005 | Small-molecule ebselen; selenorganic compound that mimics and induces glutathione peroxidase activity | Meniere's disease | First patient enrolled | 7/28/22 | Other/Miscallaneous |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 | 5-HT3 receptor antagonist | Alcohol use disorder | Study achieved a statistically significant reduction from baseline at month 6 in heavy drinking days for the prespecified patient group of heavy drinkers (avg. less than 10 drinks per drinking day at baseline; p=0.03); mean reduction of 79% in heavy drinking compared with baseline; overall significant difference in the severity of AUD diagnosis (p=0.04); no serious events; 2 cardiac events in placebo group and none in the AD-04 group | 7/20/22 | Toxicity and Intoxication |
| 2seventy Bio Inc., of Cambridge, Mass., and Bristol Myers Squibb Co., of New York | Abecma (idecabtagene vicleucel) | CAR T targeting B-cell maturation antigen | Relapsed and refractory multiple myeloma | Interim analysis showed study met its primary endpoint; statistically significant improvement in progression-free survival; improvement in the key secondary endpoint of overall response rate compared to standard regimens; no new safety signals | 8/10/22 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Lumakras (sotorasib) | KRAS-G12C inhibitor; fully human monoclonal antibody that binds to EGFR | KRAS G12C-mutated non-small-cell lung cancer | Study met its primary endpoint of progression-free survival; statistical significance and superiority over standard-of-care chemotherapy | 8/30/22 | Cancer |
| Anthos Therapeutics Inc., of Cambridge, Mass. | Abelacimab | Dual-acting fully human monoclonal antibody targeting both factor XI and factor XIa | Gastrointestinal/genitourinary cancer | Initiated recruitment for its second phase III study; targeting to enroll approximately 2,700 patients | 8/25/22 | Cancer |
| Astrazeneca plc and Daiichi Sankyo Co. Ltd. | Enhertu (fam-trastuzumab deruxtecan) | Antibody-drug conjugate targeting HER2 | HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab emtansine | The Destiny-Breast02 study met its primary endpoint of progression-free survival and its secondary endpoint of overall survival compared to physician’s choice of treatment (trastuzumab/capecitabine or lapatinib/capecitabine); data to be presented at an upcoming medical meeting | 8/15/22 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Unresectable hepatocellular carcinoma | Study met its primary endpoint of noninferior overall survival vs. sorafenib in adult patients; no new safety signals | 8/9/22 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Patritumab deruxtecan (HER3-DXd) | HER3-directed antibody-drug conjugate | EGFR-mutated locally advanced or metastatic nonsquamous non-small-cell lung cancer | First patient dosed | 8/8/22 | Cancer |
| Dizal Pharmaceutical Co. Ltd., of Shanghai | Sunvozertinib (DZD-9008) | Oral EGFR Ex20ins inhibitor | Non-small-cell lung cancer | Results showed best overall response rate (ORR) of 52.4% according to RECIST guidelines at 300 mg in platinum-pretreated patients; patients with baseline brain metastasis showed 44% of ORR at 300 mg; clinically manageable and grade 1/2 adverse events | 8/5/22 | Cancer |
| Eqrx Inc., of Cambridge, Mass., and Cstone Pharmaceuticals Ltd., of Suzhou, China | Sugemalimab | Anti-PD-L1 monoclonal antibody | Non-small-cell lung cancer | Gemstone-301 study demonstrated a statistically significant improvement in progression-free survival (PFS) vs. placebo in patients; median PFS was 10.5 months for sugemalimab and 6.2 months for placebo (p=0.0012); overall survival not reached and immature at the time of the analysis | 8/7/22 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Trodelvy (sacituzumab govitecan) | Antibody-drug conjugate targeting Trop-2 | HR+/HER2- metastatic breast cancer who received prior endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy | The second interim analysis of the Tropics-02 study showed the drug produced a significant improvement in overall survival compared to physicians’ choice of chemotherapy (eribulin, capecitabine, gemcitabine, or vinorelbine); data to be presented at an upcoming medical meeting | 8/15/22 | Cancer |
| Greenwich Lifesciences Inc., of Stafford, Texas | GLSI-100 | Immunotherapy | Breast cancer | Flamingo-01 study started; begun the screening and enrolling process | 8/11/22 | Cancer |
| Halozyme Therapeutics Inc., of San Diego, and Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) with Enhanze technology | Monoclonal antibody targeting PD-L1 | Locally advanced or metastatic non-small-cell lung cancer | Results from Mscin001 study with Enhanze technology met its co-primary endpoints; study showed noninferior levels of Tecentriq in the blood via subcutaneous administration compared to intravenous infusion | 8/2/22 | Cancer |
| Innate Pharma SA, of Marseille, France, and Astrazeneca plc, of Cambridge, U.K. | Monalizumab | Immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor-infiltrating cytotoxic CD8+ T cells and NK cells | Recurrent or metastatic squamous cell carcinoma of the head and neck | Interim analysis of Interlink-1 study in combination with cetuximab did not meet a predefined threshold for efficacy; study will be discontinued; no new safety findings | 8/1/22 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Metastatic castration-resistant prostate cancer | KEYNOTE-921 study in combination with docetaxel did not meet its dual primary endpoints of overall survival and radiographic progression-free survival; did not meet statistical significance per the prespecified statistical plan; consistent safety profile | 8/3/22 | 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 | Unresectable hepatocellular carcinoma | Leap-002 study did not meet its dual primary endpoints of overall survival and progression-free survival; results did not meet statistical significance per the prespecified statistical plan; consistent safety profile | 8/3/22 | Cancer |
| Novartis AG, of Basel, Switzerland | Canakinumab | Antibody targeting interleukin-1beta | Stages II-IIIA and IIIB completely resected non-small-cell lung cancer | The Canopy-A study didn’t meet its primary endpoint of disease-free survival compared to placebo; data to be presented at an upcoming medical meeting | 8/15/22 | Cancer |
| Orca Bio Inc., of Menlo Park, Calif. | Orca-T | Allogeneic cellular therapy | Hematologic malignancies | Initiated phase III study; planning to enroll 174 patients | 8/2/22 | Cancer |
| Panbela Therapeutics Inc., of Minneapolis | SBP-101 | Polyamine analogue | Metastatic pancreatic ductal adenocarcinoma | First patient enrolled in Aspire trial in combination with gemcitabine and nab-paclitaxel; interim data expected by early 2024 | 8/11/22 | Cancer |
| Photocure ASA, of Oslo, Norway, and Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1702 | Photodynamic drug-device combination product | Cervical high-grade squamous intraepithelial lesions | Completed enrollment of 402 patients | 8/9/22 | Cancer |
| Rain Therapeutics Inc., of Newark, Calif. | Milademetan | Oral mouse double minute 2 (MDM2) inhibitor | Liposarcoma | Completed enrollment of 175 patients; progression-free survival as the primary endpoint; top-line data in the first half of 2023 | 8/4/22 | Cancer |
| Sorrento Therapeutics Inc., of San Diego | Fujovee (abivertinib) | EGFR/BTK inhibitor | Advanced non-small-cell-lung cancer | Top-line results from long-term follow up data showed significant treatment benefits in 209 response evaluable, heavily treated patients with ORR of 56.5%; 5.3% complete response rate; median overall survival of 28.2 months | 8/23/22 | Cancer |
| Tavanta Therapeutics Inc., of Radnor, Pa. | TAVT-45 (abiraterone acetate) | Granules for oral suspension | Metastatic castrate-sensitive prostate cancer and metastatic castrate-resistant prostate cancer | Completed patient enrollment; primary objective of the study is to establish therapeutic equivalence between TAVT-45 and Zytiga tablets; top-line data expected by the end of 2022 | 8/1/22 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab (KN-035) | Single-domain antibody against PD-L1 | Solid tumors | Independent data monitoring committee recommended that the trial proceed as planned following the review of 3-week safety data from more than 20 patients enrolled | 8/10/22 | Cancer |
| Biocardia Inc., of Sunnyvale, Calif. | Cardiamp | Cell therapy | Heart failure | Independent data safety monitoring committee completed its prespecified data review; no significant safety concerns; recommended study continue as designed | 8/31/22 | Cardiovascular |
| Lianbio Co. Ltd., of Shanghai | Mavacamten | Oral, allosteric modulator of cardiac myosin | Symptomatic obstructive hypertrophic cardiomyopathy | Completed enrollment in Chinese patients; top-line results in mid-2023 | 8/10/22 | Cardiovascular |
| Pfizer Inc., of New York | PF-07265803 (formerly ARRY-371797) | Oral, small-molecule inhibitor of p38? mitogen activated protein kinase pathway | Symptomatic dilated cardiomyopathy | Study not achieved primary endpoint; discontinued development; decision is not based on safety concerns | 8/3/22 | Cardiovascular |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone (RVX-208) | BD2 selective BET inhibitor | Acute coronary syndrome, type 2 diabetes, nonalcoholic fatty liver disease | Post-hoc analysis of Betonmace data published in the American Journal of Preventive Cardiology showed apabetalone treatment reduced the hazard of heart failure failure (HR=0.53, p=0.01), as well as a composite MACE and HHF (HR=0.76, p=0.03) in participants with elevated Angulo fibrosis score; significantly smaller increase in liver fibrosis scores | 8/22/22 | Cardiovascular |
| Amgen Inc., of Thousand Oaks, Calif., and Kyowa Kirin Co. Ltd., of Tokyo | KHK-4083 (AMG-45, rocatinlimab) | Immunomodulating anti-OX40 monoclonal antibody | Atopic dermatitis | Enrollment paused to amend studies | 8/4/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) | Topical PDE4 inhibitor | Atopic dermatitis | Completed enrollment of the last subject in Integument-2 study; top-line study from trials expected by the end of 2022 | 8/23/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) | Topical PDE4 inhibitor | Atopic dermatitis | Enrollment completed; total of 654 subject enrolled in the Integument-1 study; top-line results expected by the end of 2022 | 8/4/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | JAK inhibitor | Moderate to severe alopecia areata | Top-line data from Thrive-AA2 study achieved primary and secondary endpoints; met statistical significance in both 8-mg twice-daily and 12-mg twice-daily dose groups relative to placebo; proportion of patients achieving a SALT score of 20 or less was 38.3% in 12-mg and 33% in 8-mg group compared to 0.8% in placebo (p<0.0001); well-tolerated< td> | 8/1/22 | Dermatologic |
| CSL Behring, a unit of Melbourne, Australia-based CSL Ltd. | CSL-312 (garadacimab) | Monoclonal antibody targeting factor XIIa | Hereditary angioedema | Top-line data of study met its primary and secondary efficacy endpoints; favorable safety and tolerability | 8/17/22 | Dermatologic |
| Journey Medical Corp., of Scottsdale, Ariz. | DFD-29 | Minocycline modified release capsules | Papulopustular rosacea | Reached 50% enrollment in the study; data expected in the first half of 2023 | 8/30/22 | Dermatologic |
| Kalvista Pharmaceuticals Inc., of Cambridge, Mass. | Sebetralstat | Oral plasma kallikrein inhibitor | Hereditary angioedema | Initiated Konfident-S open label extension study; results expected in the second half of 2023 | 8/23/22 | Dermatologic |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Pyrukynd (mitapivat) | Pyruvate kinase activator | Pyruvate kinase deficiency | Results from Activate-T study published in The Lancet Haematology showed trial met primary endpoint, with 37% of patients treated with Pyrukynd achieving a transfusion reduction response (p=0.0002), defined as a ?33% reduction in transfusion burden in the 24-week fixed dose period compared with individual historical transfusion burden standardized to 24 weeks; 9 of 10 responders achieved a ?50% reduction; 22% reached transfusion-free status during the fixed-dose period, and 11% achieved hemoglobin concentrations in the normal range at least once; no serious adverse events | 8/18/22 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Onpattro (patisiran) | RNAi therapeutic | Transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy | Study met the primary endpoint of change from baseline in the 6MWT at 12 months compared to placebo (p=0.0162); achieved secondary endpoint of change from baseline in quality of life compared to placebo as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ) (p=0.0397); favorable safety and tolerability profile | 8/3/22 | Endocrine/Metabolic |
| Lannett Co. Inc., of Philadelphia | Biosimilar insulin glargine | Biosimilar insulin glargine | Diabetes | Completed dosing; no serious adverse events; top-line data expected later in 2022 | 8/31/22 | Endocrine/Metabolic |
| Recordati SpA, of Milan, Italy | Isturisa (osilodrostat) | 11beta-hydroxylase inhibitor | Cushing’s disease | Long-term outcomes from the open-label extension period of the LINC-3 study published in the European Journal of Endocrinology showed well-tolerated oral therapy; improvements in most cardiovascular- and metabolic-related parameters; improvement in quality of life; estradiol and testosterone levels tended to return to baseline levels with longer follow-up in female patients; well-tolerated; no unexpected adverse events | 8/31/22 | Endocrine/Metabolic |
| Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Completed enrollment for Response phase III study; targeted enrollment of 180 patients | 8/1/22 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor-beta selective agonist | Nonalcoholic steatohepatitis | Initiated Maestro-NASH outcome study; first patient screened; top-line data in the fourth quarter of 2022 | 8/31/22 | Gastrointestinal |
| Palisade Bio Inc., of Carlsbad, Calif. | LB-1148 | Oral formulation of broad-spectrum serine protease inhibitor | Accelerate return of bowel function in patients undergoing gastrointestinal surgery | First patient dosed; planning to enroll 600 patients; trial will assess safety and efficacy | 8/17/22 | Gastrointestinal |
| Futura Medical plc, of Guilford, U.K. | MED-3000 | Topical gel formulation | Erectile dysfunction | Study achieved all primary and secondary endpoints; highly statistically significant improvement (p<0.001) 24 against baseline at weeks in erectile function; showed 10-minute onset of action; highly favorable side-effect profile; no serious adverse effects< td> | 8/31/22 | Genitourinary/Sexual Function |
| Vascular Therapies Inc., of Cresskill, N.J. | Sirogen | Sirolimus formulation for local, perivascular drug delivery | End-stage renal disease | First patient enrolled in the 120-patient Access 2 study evaluating the ability of Sirogen to improve hemodialysis AV fistula outcomes | 8/29/22 | Genitourinary/Sexual Function |
| Amgen Inc., of Thousands Oaks, Calif. | ABP-959 | Eculizumab Biosimilar | Paroxysmal nocturnal hemoglobinuria | Top-line data from Dahlia study met its primary endpoints; similar efficacy, safety and immunogenicity profile | 8/23/22 | Hematologic |
| TG Therapeutics Inc., of New York | Ublituximab | Anti-CD20 monoclonal antibody | Relapsing forms of multiple sclerosis | Results from Ultimate I and II study published in The New England Journal of Medicine showed significant reductions in risk of relapses; reduction of active or new brain lesions; resulted in annualized relapse rate (p<0.001 and p="0.002);" mean total number of gadolinium enhancing lesions per t1-weighted mri scan (p<0.001 for both); new or enlarging hyperintense t2-weighted no evidence disease activity was observed in 44.6% 43% ublituximab-treated patients 15% 11.4% the teriflunomide-treated patients< td> | 8/25/22 | Immune |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 | Recombinant trimeric Spike-protein subunit vaccine | COVID-19 | Elicited 12 to 61-fold increase in neutralizing antibodies against BA.5 among participants receiving SCB-2019 (CpG 1018/Alum) as a homologous third dose or with a history of prior SARS-CoV-2 infection; BA.5 neutralizing antibody levels were about 2 to 4-fold higher than BA.1 neutralizing antibody levels | 8/29/22 | Infection |
| Evofem Biosciences Inc., of San Diego | Phexxi | Lactic acid, citric acid, potassium bitartrate | Chlamydia and gonorrhea infection | Last subject completed last visit in Evoguard study; top-line data expected in October 2022 | 8/1/22 | Infection |
| Jiangsu Recbio Technology Co. Ltd., of Taizhou, China | REC-603 | Recombinant HPV 9-valent vaccine | HPV infection | Completed subject enrollment; first dose vaccination in respect of immuno-bridging study | 8/10/22 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Vivjoa (oteseconazole) | lanosterol-14 demethylase inhibitor | Recurrent vulvovaginal candidiasis | Results of Ultraviolet study published in the American Journal of Obstetrics and Gynecology showed drug statistically superior to placebo; 94.9% of patients did not have a VVC episode compared to 57.8% of patients treated with fluconazole in the induction phase followed by placebo; in intent-to-treat population, 93.2% of patients treated with Vivjoa had a resolved VVC episode by day 14 compared to 95.8% of patients treated with fluconazole | 8/25/22 | Infection |
| Pfizer Inc., of New York | 20vPnC | 20-valent pneumococcal polysaccharide conjugate vaccine | Invasive pneumococcal disease | Top-line data showed study met the co-primary objectives in four-dose series; all serotypes met noninferiority for the key secondary objective of IgG GMCs after dose 3; robust functional responses and increase in antibody responses after dose 4; favorable safety and tolerability profile similar to Prevnar 13 | 8/12/22 | Infection |
| Pfizer Inc., of New York | RSVpreF | Bivalent RSV prefusion F vaccine candidate | Respiratory syncytial virus | Top-line data showed vaccine efficacy of 85.7%; well-tolerated; no safety concerns | 8/25/22 | Infection |
| Pfizer Inc., of New York, and Valneva SE, of Saint Herblain, France | VLA-15 | Multivalent protein subunit vaccine targets the outer surface protein A of Borrelia burgdorferi | Lyme disease | Initiated phase III study; to investigate the efficacy, safety and immunogenicity; to enroll 6000 participants 5 years of age and older | 8/8/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Oral anti-inflammatory drug | Mild to moderate COVID-19 | Company amended the study protocol with the proposed new primary efficacy endpoints and submitted to the FDA for further discussion and agreement | 8/16/22 | Infection |
| Sanofi, SA, of Paris | Nirsevimab | Antibody targeting respiratory syncytial virus (RSV) | RSV prophylaxis | Started the Harmonie study of more than 20,000 infants in conjunction with the National Institute for Health and Care Research; study will measure whether the antibody can prevent hospitalizations due to RSV | 8/12/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Brexafemme (ibrexafungerp) | 1,3-beta-glucan synthase inhibitor | Recurrent vulvovaginal candidiasis | Candle study achieved statistically significant superiority over placebo in both primary and key secondary endpoints (p=0.034 and 0.029, respectively); 65.4% of patients achieved clinical success with no recurrence compared to placebo 53.1% (p=0.02); no mycologically proven recurrence in 70.8% of patients compared to 58.5% in placebo (p=0.019); significant reduction or elimination of signs and symptoms; safe and well-tolerated; no serious drug-related adverse events; no patients discontinued therapy | 8/4/22 | Infection |
| Valneva SE, of Saint Herblain, France | VLA-2001 | Whole virus, inactivated, adjuvanted COVID-19 vaccine | COVID-19 | Subset analysis 6 months after primary vaccination showed VLA-2001 induced broad antigen-specific IFN-gamma producing T cells reactive against the S-protein, as well as the N- and M-proteins up to day 208; favorable and well-tolerated | 8/29/22 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Pa., and Torii Pharmaceutical Co. Ltd., of Tokyo | VP-102 | Drug-device combination product that contains a GMP-controlled formulation of cantharidin delivered via a single-use applicator | Molluscum contagiosum | First patient dosed | 8/1/22 | Infection |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | Kinase inhibitor | Aggressive systemic mastocytosis | Top-line results from Pioneer study achieved primary and all key secondary endpoints; highly significant difference in mean change in total symptom score (TSS) at 24 weeks (p=0.003); reduction of 15.6 points in mean TSS at 24 weeks; 20.2 points at 48 weeks; significant improvements across all measures of mast cell burden; ?50 percent reduction of serum tryptase, compared to no patients in the control arm (53.9% vs. 0%; p<0.0001); well-tolerated; favorable safety profile; no patients in the control arm discontinued due to treatment-related adverse events< td> | 8/17/22 | Musculoskeletal |
| Annovis Bio Inc., of Berwyn, Pa. | Buntanetap | Oral translational inhibitor of neurotoxic aggregating proteins | Parkinson's disease | First patient dosed; study to evaluate the efficacy, safety and tolerability | 8/24/22 | Neurology/Psychiatric |
| Grunenthal GmbH, of Aachen, Germany | Resiniferatoxin | TRPV1 agonist | Pain associated with knee osteoarthritis | Enrolled first patient | 8/18/22 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc., of Dublin | Epidiolex | Plant-derived cannabis-based medicine | Epilepsy with myoclonic-atonic seizures | Initiated phase III study | 8/18/22 | Neurology/Psychiatric |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Rexulti (brexpiprazole) | Partial agonist of serotonin 5-HT1A and dopamine D2 receptors | Agitation in Alzheimer’s dementia | Results showed statistically significantly greater reduction in agitation compared to placebo; improvements from baseline on the primary endpoint of Cohen-Mansfield Agitation Inventory (CMAI) in patients treated with brexpiprazole 2 mg/day or 3 mg/day were statistically greater than placebo (p=0.0026); statistically superior improvement on the key secondary endpoint of CGI-S p=0.0055 | 8/4/22 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 | Nasal powder formulation of dihydroergotamine mesylate | Acute migraine | Completed subject enrollment; more than 1,400 subjects randomized; top-line data expected in the fourth quarter of 2022 | 8/2/22 | Neurology/Psychiatric |
| Karuna Therapeutics Inc., of Boston, and Zai Lab Ltd., of Shanghai | Karxt | Combination of xanomeline and trospium chloride; muscarinic agonist and antagonist | Schizophrenia | Top-line results from Emergent study met its primary and key secondary endpoints; statistically significant 9.6-point reduction in PANSS total score compared to placebo at week 5 (p<0.0001); statistically significant reductions in positive and negative symptoms of schizophrenia; 1.8-point reduction the panss subscale with karxt compared to placebo (-3.4 vs. -1.6 placebo, p="0.0055);" 2.2-point marder factor (-4.2 -2 well-tolerated; discontinuation rates were similar between groups (25% 21%)< td> | 8/8/22 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham | AR-15512 | TRPM8 agonist | Dry eye disease | First subject dosed in COMET-3 study | 8/1/22 | Ocular |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Targeted C3 therapy | Geographic atrophy secondary to age-related macular degeneration | Top-line data showed robust reduction of GA lesion growth compared to sham in DERBY trial (36% monthly, p<0.0001; 24 29% every-other-month [eom], p="0.0002)" and in oaks trial (24% monthly, 25% eom, no clinically meaningful difference on key functional endpoints observed at months; favorable safety profile< td> | 8/24/22 | Ocular |
| Belite Bio Inc., of San Diego | LBS-008 (tinlarebant) | Orally available, small-molecule retinol binding protein 4 (RBP4) antagonist | Stargardt disease | Initiated enrollment; planning to enroll 60 patients | 8/22/22 | Ocular |
| Kodiak Sciences Inc., of Palo Alto, Calif. | Tarcocimab tedromer (KSI-301) | Anti-VEGF antibody biopolymer conjugate | Non-proliferative diabetic retinopathy | Completed enrollment in Glow study | 8/5/22 | Ocular |
| Palatin Technologies Inc., of Cranbury, N.J. | PL-9643 | Melanocortin agonist | Dry eye disease | Interim analysis completed for 120 patients; data monitoring committee recommended the study continue with a sample size target of up to 350 patients; no safety concerns; top-line results expected in the second quarter of 2023 | 8/16/22 | Ocular |
| Alume Biosciences Inc., of La Jolla, Calif. | ALM-488 | Fluorescent peptide-dye conjugate | Surgical nerve visualization | First patient dosed | 8/23/22 | Other/Miscallaneous |
| Verona Pharma plc, of London | Ensifentrine | Dual inhibitor of the enzymes phosphodiesterase 3 and 4; CFTR activator | Chronic obstructive pulmonary disease | Top-line data of Enhance-2 study met its primary and secondary endpoints; change from baseline in average FEV1 area under the curve 0-12 hours post dose at week 12 was 94 mL (p<0.0001) 12 24 49 146 for ensifentrine; statistically significant and clinically meaningful improvements; increase in peak fev1 of ml (p<0.0001) 0-4 hours post dose at week secondary endpoints; morning trough (p="0.0017)" 12; 42% reduction the rate moderate to severe copd exacerbations over weeks compared placebo significantly decreased risk a exacerbation with by well-tolerated< td> | 8/9/22 | Respiratory |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Rahway, N.J. | Lynparza (olaparib) | PARP inhibitor | Advanced ovarian cancer | Updated results from Paola-1 study in combination with bevacizumab increased median OS to 56.5 months vs. 51.6 months with bevacizumab alone; not statistically significant; improved median PFS to almost 4 years (46.8 months) vs. 17.6 months with bevacizumab plus placebo; 46.1% of patients treated with Lynparza plus bevacizumab remain progression free at 5 years vs. 19.2% of patients treated with bevacizumab alone; no new safety signals | 9/9/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Rahway, N.J. | Lynparza (olaparib) | PARP inhibitor | Advanced ovarian cancer | Updated results from Solo-1 study showed clinically meaningful improvement in overall survival vs. placebo; reduced the risk of death by 45% (p=0.0004), not statistically significant; median overall survival was still not reached with Lynparza vs. 75.2 months with placebo; no new safety signals | 9/9/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Anti-PD-1 antibody | Stage IIB/C melanoma | Prespecified interim analysis from Checkmate-76 study met its primary endpoint and demonstrated a statistically significant and clinically meaningful benefit in recurrence-free survival vs. placebo; no new safety signals | 9/15/22 | Cancer |
| CNS Pharmaceuticals Inc., of Houston | Berubicin | Anthracycline for crossing the blood-brain barrier | Recurrent glioblastoma multiforme | Activated its first clinical trial sites in Europe for the ongoing global study | 9/28/22 | Cancer |
| Genelux Corp., of Westlake Village, Calif. | Olvi-Vec | Oncolytic virus | Platinum-resistant/refractory ovarian cancer | Registrational trial initiated evaluating Olvi-Vec combined with platinum-doublet + bevacizumab compared to platinum-doublet + bevacizumab | 9/21/22 | Cancer |
| Isofol Medical AB, of Gothenburg, Sweden | Arfolitixorin | Folate receptor antagonist | Metastatic colorectal cancer | Agent study terminated; study met neither its primary endpoint nor its key secondary endpoints; progression-free survival was 12.8 months for the arfolitixorin arm and 11.6 months for the control arm (p=0.76); no difference between the study arms in safety data | 9/7/22 | Cancer |
| Lianbio Co. Ltd., of Shanghai, and Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide crystal nanoparticles | Head and neck cancer | Randomized first patient in Nanoray-312 study | 9/7/22 | Cancer |
| On Target Laboratories Inc., of West Lafayette, Ind. | Cytalux (pafolacianine) | Imaging agent targeting folate receptors | Imaging of folate receptor positive ovarian cancer | Study results published in the peer-reviewed Journal of Clinical Oncology showed cytalux identified additional lesions which would have been left behind (P < 0.001) | 9/13/22 | Cancer |
| Oncternal Therapeutics Inc., of San Diego | Zilovertamab (ZILO-301) | Monoclonal antibody targeting tyrosine kinase-like orphan receptor 1 | Relapsed/refractory mantle cell lymphoma | Treated first of 365 patients in the study testing zilovertamab in patients who have a partial response or stable disease after treatment with ibrutinib; objective response rate plus duration of response endpoints will be used for accelerated approval; primary endpoint of progression-free survival will be used for full approval; secondary endpoints include ORR, DOR, complete response rate, overall survival and the proportion of patients experiencing grade 3 or 4 neutrophil count decrease | 9/27/22 | Cancer |
| Shanghai Henlius Biotech Inc., of Shanghai | Hansizhuang (serplulimab) | Anti-PD-1 monoclonal antibody | Extensive-stage small-cell lung cancer | Median overall survival was significantly longer in the serplulimab group (15.4 months) than placebo (10.9 months; p <0.001)< td> | 9/28/22 | Cancer |
| Alnylam Therapeutics Inc., of Cambridge, Mass. | Patisiran | RNAi therapeutic | Transthyretin-mediated amyloidosis with cardiomyopath | 12-month findings across a comprehensive set of exploratory endpoints suggest that treatment with patisiran was associated with favorable impacts on key measures of cardiac stress and injury, NT-proBNP and Troponin I, respectively | 9/30/22 | Cardiovascular |
| Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | IL-23 inhibitor | Active psoriatic arthritis | Long-term data analysis of Keepsake 1 and 2 study showed improvement in skin and joint symptoms at week 100; well-tolerated; no new safety signals; 90% reduction in PASI90; 64% and 57% of patients, respectively, achieved ACR20 response in studies at week 100; 76% and 57% of patients, respectively, achieved resolution of dactylitis and enthesitis; mean scores of PGA-F and mNAPSI were maintained at week 100 compared with week 52 | 9/10/22 | Dermatologic |
| Amgen Inc., of Thousand Oaks, Calif. | Otezla (apremilast) | Oral small-molecule inhibitor of PDE4 specific for cyclic adenosine monophosphate | Moderate to severe plaque psoriasis | Sprout study met primary and secondary endpoints; 33.1% sPGA response for Otezla vs. 11.5% for placebo (p<0.0001); 75% or more reduction in the pasi75 with 45.4% for otezla vs. 16.1% placebo (p<0.0001)< td> | 9/8/22 | Dermatologic |
| Amgen Inc., of Thousand Oaks, Calif. | Otezla (apremilast) | Oral small-molecule inhibitor of PDE4 specific for cyclic adenosine monophosphate | Moderate to severe genital psoriasis | Discreet study achieved primary and secondary endpoints; 38.7% for Otezla vs. 19.1% for placebo (p=0.0003 in sPGA-G scale); 46% for Otezla vs. 19.6% for placebo in Genital Psoriasis Itch Numeric Rating Scale response (p< 0.0001); greater reduction in affected body surface area; 4.12% mean reduction with Otezla vs. 0.79% with placebo (p= 0.0005); dermatology life quality index burden score reduction of 5.3 for Otezla vs. 2.6 for placebo (p= 0.0008); genital psoriasis symptoms scale reduction in mean score of 20.5 for Otezla vs. 5.3 for placebo (p< 0.0001); improvement in sexual health impacted by psoriasis | 9/8/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) | Topical PDE4 inhibitor | Moderate to severe seborrheic dermatitis | Data from Stratum study met the primary endpoint with 80.1% of individuals treated with roflumilast foam achieving IGA success vs. 59.2% of patients treated with vehicle (p<0.0001) 4 8 at week 8; 63.6% of individuals with a worst itch-numerical rating score (wi-nrs) or higher baseline treated roflumilast foam achieved ?4-point reduction in itch vs. 42.3% vehicle (p="0.0002);"> 50% of individuals treated with roflumilast foam achieved an erythema score of 0 at week 8; > 50% of individuals treated with roflumilast foam achieved a scaling score of 0 at week 8; well-tolerated; mild-to-moderate severity adverse events; no treatment-related events | 9/9/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream 0.3% | Topical inhibitor of phosphodiesterase-4 | Scalp and body psoriasis | Arrector study met its co-primary endpoints and all secondary endpoints; 67.3% of individuals treated with roflumilast foam achieved S-IGA Success compared to 28.1% of individuals treated with a matching vehicle foam at week 8 (p<0.0001); 8 46.5% of individuals treated with roflumilast foam achieved b-iga success compared to 20.8% a matching vehicle at week (p<0.0001); statistically significant improvements on all secondary endpoints; well-tolerated; mild moderate severity adverse events< td> | 9/26/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream 0.3% | Topical inhibitor of phosphodiesterase-4 | Chronic plaque psoriasis | Treatment in the DERMIS-1 and DERMIS-2 studies resulted in significant improvements across multiple efficacy endpoints, including plaque clearance and itch at 8 weeks in adults and adolescents compared to vehicle | 9/20/22 | Dermatologic |
| Bristol Myers Squibb Co., of Princeton, N.J. | Sotyktu (deucravacitinib) | Oral, selective, allosteric TYK2 inhibitor | Moderate to severe plaque psoriasis | Long-term extension study showed modified non-responder imputation (mNRI) response rates were 82.4% for PASI75, 55.2% for PASI 90 and 66.5% for static PGA; 171/262 patients achieved PASI 75 at week 16; efficacy was maintained for up to 112 weeks; tolerated | 9/10/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 | JAK inhibitor | Moderate to severe alopecia areata | New data from Thrive-AA1 study achieved more stringent criteria for hair regrowth than the primary endpoint of absolute Severity of Alopecia Tool (SALT) score of 20 or less (meaning 20% or less scalp hair loss) at week 24; achieved a SALT score of 10 or less at week 24 compared to 0% in placebo (p<0.0001); significant improvement of loss eyebrow or eyelash hair at baseline compared to placebo over the 24-week treatment period (p<0.001); well-tolerated< td> | 9/10/22 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Lebrikizumab | IL-13 inhibitor | Moderate to severe atopic dermatitis | Monotherapy studies showed robust and durable improvements in skin clearance and itch for patients who achieved a clinical response at week 16; EASI-75 of 79% at 250 mg Q4W and 250 mg Q2W, respectively ?4-point improvement of 80 % and 81% at respective doses |
9/8/22 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Adtralza (tralokinumab) | Monoclonal antibody targeting interleukin-13 | Moderate to severe atopic dermatitis | Interim results showed consistent safety from the parent trials for up to 3.5 years; mild adverse events; most serious events were reported as single events without clustering on type; most frequently reported treatment-emergent events (?5.0% of patients) were viral upper respiratory tract infection | 9/8/22 | Dermatologic |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Interleukin-17A inhibitor | Hidradenitis suppurativa | Results showed significantly higher proportion of patients achieved a Hidradenitis Suppurativa Clinical Response (HiSCR) at 300 mg dosed every 2 weeks compared with placebo at week 16 in Sunshine and Sunrise studies (45% vs. 33.7% [p=0.0070] and 42.3% vs. 31.2% [p=0.0149], respectively); superior to placebo for achieving HiSCR in the Sunrise study (46.1% vs. 31.2% [p=0.0022]); did not meet statistical significance in the Sunshine study 41.8% vs. 33.7% [p=0.0418]; no new safety signals | 9/10/22 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Inhibits interleukin-4 and interleukin-13 | Uncontrolled prurigo nodularis | Study met its primary and key secondary endpoint; clinically meaningful reduction in itch from baseline, compared to placebo patients (18%; p<0.0001); 3 times as many (48%) achieved clear or almost skin vs. placebo patients (18%; p="0.0004) | 9/8/22 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Inhibitor of interleukin-4 and interleukin-13 | Uncontrolled moderate-to-severe atopic dermatitis | Results published in The Lancet showed adding Dupixent to low-potency topical corticosteroids (TCS) significantly improved skin clearance and reduced overall disease severity and itch compared to TCS alone at 16 weeks for children aged 6 months to 5 year-; significant improvement in measures of sleep quality and skin pain; improvement in patient or caregiver reported outcomes and health-related quality of life were also observed | 9/15/22 | Dermatologic |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Moderate to severe plaque psoriasis | New 3-year results from the Be Bright open-label extension study in bimekizumab-randomized patients showed 89.3% patient achieved PASI 100 at year 1 and 82% at year 3; 96.5% achieved PASI ?2 at year 1 and 94.2% at year 3; 92% achieved the Dermatology Life Quality Index (DLQI) at year 1, and 88% at year 3; well-tolerated with no new safety signals identified | 9/7/22 | Dermatologic |
| Intercept Pharmaceuticals Inc., of Morristown, N.J. | Ocaliva (obeticholic acid) | Semi-synthetic bile acid analog | Non-alcoholic steatohepatitis | 25-mg dose met agreed primary endpoint of improvement in liver fibrosis without worsening of disease at 18 months | 9/30/22 | Endocrine/metabolic |
| Zealand Pharma A/S, of Copenhagen | Dasiglucagon | Glucagon analogue | Congenital hyperinsulinism | Data from children ages 7 days to 12 months showed dasiglucagon significantly reduced the mean intavenous (I.V.) glucose infusion rate (GIR) p=0.0037; reduced GIR over the entire 48-hour treatment period by 3.5 mg/kg/min compared to placebo (p=0.0107); resulted in a reduction of 31 g/day in total carbohydrate intake (I.V. and gastric) compared to placebo (107 g/day for dasiglucagon vs. 138 g/day for placebo; p = 0.024), a 22% reduction in carbohydrate calories; well-tolerated; results from 21-day open-label part 2 study showed dasiglucagon enabled reduction and either periodic or permanent discontinuation of I.V. glucose infusion in 10 out of 12 infants; no increase in hyperglycemia; no adverse events or serious adverse events | 9/19/22 | Endocrine/Metabolic |
| Intercept Pharmaceuticals Inc., of Morristown, N.J. | Obeticholic acid | Semisynthetic bile acid derivative | Primary biliary cholangitis | Data published in Gastroenterology showed patients treated in the Poise study had approximately 70% lower relative risk of death or liver transplant than historical patients who didn’t receive the drug; the drug also improved the composite of death, liver transplant and hepatic decompensation compared to historical controls | 9/20/22 | Gastrointestinal |
| Ironwood Pharmaceuticals Inc., of Boston | Linaclotide | Guanylate cyclase-C agonist | Constipation | Top-line data showed study met its primary and secondary endpoints at 72 mcg in pediatric patients aged 6-17; well-tolerated; improved frequency of spontaneous bowl movements (SBM) and stool consistency; greater than 2-fold least squares mean change from baseline in SBMs/week compared to placebo (p<0.0001)< td> | 9/6/22 | Gastrointestinal |
| Zealand Pharma A/S, of Copenhagen | Glepaglutide | GLP-2 analog | Short bowel syndrome | Met primary endpoint with twice-weekly dosing achieving a statistically significant reduction in weekly parenteral support volume by 5.13 liters/week from baseline at 24 weeks | 9/30/22 | Gastrointestinal |
| Allakos Inc., of Redwood City, Calif. | Lirentelimab (AK-002) | Anti-Siglec-8 antibody | Eosinophilic duodenitis | Top-line data showed study met its histologic co-primary endpoint; did not achieve statistical significance on the patient-reported symptomatic co-primary endpoint in both the intent to treat population and in a prespecified subpopulation; mild to moderate infusion-related reactions | 9/9/22 | Hematologic |
| Astrazeneca plc., of Cambridge, U.K. | Danicopan (ALXN-2040) | Oral factor D inhibitor | Paroxysmal nocturnal haemoglobinuria | Interim analysis met primary endpoint of change in hemoglobin from baseline at 12 weeks; statistically significant and clinically meaningful improvements in hemoglobin levels; well-tolerated; no clinically meaningful differences in safety results | 9/16/22 | Hematologic |
| Horizon Therapeutics plc, of Dublin | Dazodalibep (HZN-4920) | CD40 ligand antagonist | Sjögren's syndrome | Study met the primary endpoint; achieved a 6.3-point reduction in ESSDAI score vs. placebo achieved a 4.1-point reduction; statistically significant least squares mean difference of 2.2 points (p=0.017); well-tolerated | 9/12/22 | Immune |
| Remegen Ltd., of Yantai, China | RC-18 (telitacicept) | TACI-Fc fusion protein | Systemic lupus erythematosus | Preliminary results of showed significantly higher rate of SRI-4 (Systemic Lupus Erythematosus Responder Index 4) response for telitacicept-treated participants than those given placebo (82.6% vs. 38.1%, p<0.001); non-response also revealed a significantly higher rate of sri-4 response in the telitacicept- treated group compared to placebo (67.1% vs. 32.7%, p<0.001)< td> | 9/19/22 | Immune |
| Mapi Pharma Ltd., of Ness Ziona, Israel | GA Depot | Injectable glatiramer acetate | Relapsing multiple sclerosis | Top-line results showed 40-mg dosage met the primary endpoint, significantly reducing the annualized relapse rate by 30.1% compared to placebo | 9/21/22 | Immune |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | Capsid virus-like particle vaccine | COVID-19 prophylaxis | First participant dosed; initial results expected at the end of 2022 | 9/2/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 | Recombinant trimeric Spike-protein subunit vaccine | COVID-19 | Data showed superior levels of neutralizing antibodies against the Omicron BA.5 subvariant; 6.5-fold increase in antibody titers against omicron BA.5 relative to pre-booster levels; 5-fold higher as a heterologous third dose after two doses of inactivated vaccine compared to a third dose of inactivated vaccine | 9/20/22 | Infection |
| Inflarx NV, of Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Critically Ill COVID-19 | Results published in the Lancet Respiratory Medicine showed primary statistical analysis of the primary outcome of 28-day mortality prespecified in the statistical analysis plan (SAP) (p=0.0941) missed the significance level; original per-protocol planned method (p=0.0266); prespecified or post-hoc analyses p-values of <0.05 3 or <0.01; strong relative reduction in 28-day all-cause mortality for vilobelimab treatment over placebo of 43% was reported the prespecified western european region analysis (p="0.014);" subgroup analyses patients higher severity all showed a with compared to (p<0.05 3); renal replacement therapy as secondary endpoint lower vilobelimab-treated group (p<0.05); well-tolerated < td> | 9/8/22 | Infection |
| Pfizer Inc., of New York | Quadrivalent modRNA vaccine | Quadrivalent modRNA vaccine | Influenza | First participant dosed; to evaluate the efficacy, safety, tolerability and immunogenicity | 9/14/22 | Infection |
| Pfizer Inc., of New York | MenABCWY (PF-06886992) | Pentavalent meningococcal vaccine candidate | Meningococcal disease | Top-line results from healthy individuals 10 to 25 years of age met all primary and secondary endpoints, with the investigational vaccine demonstrating non-inferiority to licensed vaccines for the five meningococcal serogroups that cause the majority of invasive meningococcal disease: serogroups A, B, C, W and Y | 9/15/22 | Infection |
| Pfizer Inc., of New York | 20vPnC | 20-valent pneumococcal polysaccharide conjugate vaccine | Invasive pneumococcal disease, pneumonia, and acute otitis media | Top-line results showed robust functional antibody responses to the vaccine serotypes between doses 2 and 3; favorable safety profile; well-tolerated | 9/19/22 | Infection |
| Polypid Ltd., of Petach Tikva, Israel | D-Plex | Prolonged and constant release of broad-spectrum antibiotic doxycycline | Abdominal soft tissue surgical site infections | Top-line results showed Shield-I study did not achieve its primary endpoint of reduction in surgical site infections and mortality; local administration of D-PLEX100 and standard of care (SoC) resulted in decreased infection and mortality of 23% compared to SoC alone (p=0.1520); did not achieve statistical significance on the key secondary endpoint; statistically significant reduction of 54% on the primary endpoint, compared to SoC alone (p<0.0032) in prespecified subgroup itt analysis; decreased from 8.5% the soc treatment arm vs. 4.2% d-plex100 arm< td> | 9/2/22 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Brexafemme (ibrexafungerp) | 1,3-beta-glucan synthase inhibitor | Candida auris infection | Interim data from FURI study in refractory Candida-infected patients showed 56% of patients had a complete or partial response; 27% had stable response; 9% showed disease progression and 8% were indeterminate; clinical improvement in 71% of refractory vulvovaginal candidiasis cases; 7% had stable response, 14% showed disease progression and 7% were indeterminate | 9/8/22 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | Ensitrelvir fumaric acid | 3CL protease inhibitor | Mild/moderate COVID-19 | Ensitrelvir hit the primary endpoint in phase III part of the Asian phase II/III trial, with a significant reduction vs. placebo in time to first resolution of 5 typical omicron-related symptoms | 9/28/22 | Infection |
| Atyr Pharma Inc., of San Diego | Efzofitimod (ATYR-1923) | Immunomodulator; selective modulation of neuropilin-2 | Pulmonary sarcoidosis | Treated first of 264 patients in the Efzo-Fit study; primary endpoint is steroid-sparing effect during a forced steroid taper of 2 dose levels of efzofitimod compared to placebo; secondary endpoints include measures of lung function and sarcoidosis symptoms | 9/27/22 | Inflammatory |
| Horizon Therapeutics plc, of Dublin | Krystexxa (pegloticase) | Pegylated uric acid specific enzyme | Gout | Results published in Arthritis & Rheumatology showed study achieved primary endpoint; greater than 30 percentage-point increase in patient response rate during month 6 (p<0.0001); 1 20 24 marked reduction in infusion reactions; no new safety signals; percentage-point increase the complete resolution of at least tophus week (p="0.043) | 9/14/22 | Inflammatory |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Local, intranasal, anti-inflammatory therapy | Chronic rhinosinusitis | First patient enrolled | 9/8/22 | Inflammatory |
| Axsome Therapeutics Inc., of New York | AXS-07 (meloxicam + rizatriptan) | Oral fixed-dose combination of meloxicam and rizatriptan | Migraine | Enrolled first patient in the Emerge trial | 9/1/22 | Neurology/Psychiatric |
| Corium Inc., of Boston | Azstarys (serdexmethylphenidate and dexmethylphenidate) | Alpha 2 adrenoceptor agonist | Children with attention-deficit hyperactivity disorder | After 1 month of taking Azstarys, participants had statistically significant improvement in sleep as assessed by the Children's Sleep Habits Questionnaire; improvement was sustained through 12 months of treatment | 9/21/22 | Neurology/psychiatric |
| Eisai Co. Ltd., of Tokyo, and Biogen Inc., of Cambridge, Mass. | Lecanemab (BAN-2401) | Anti-amyloid beta protofibril antibody | Mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD with confirmed presence of amyloid pathology in the brain | Met primary endpoint of reduced clinical decline on global cognitive and functional scale (CDR-SB) by 27% at 18 months compared with placebo, representing a treatment difference in the score change of -0.45 (p=0.00005); as early as 6 months, treatment showed highly statistically significant changes in CDR-SB from baseline vs. placebo (all p-values less than 0.01); all key secondary endpoints also met with statistically significant results vs. placebo (p<0.01)< td> | 9/28/22 | Neurology/Psychiatric |
| GNT Pharma Inc., of Yongin, South Korea | Nelonemdaz | NR2B-selective N-methyl d-aspartate receptor antagonist and spin trapper | Acute ischemic stroke | Independent data monitoring committee completed a pre-specified interim analysis; trial to continue as planned without modification; achieved >33% of target enrollment in ENIS-3 trial; achieved >45% of target enrollment in Rodin study; well-tolerated; no appearance of the psychotic symptoms | 9/19/22 | Neurology/Psychiatric |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif., and Astrazeneca plc, of Cambridge, U.K. | Eplontersen | Ligand-conjugated antisense medicine | Transthyretin-mediated amyloid polyneuropathy | Results from planned 35-week interim analysis of eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to the external placebo group; favorable safety and tolerability profile; achieved 81.2% (p<0.0001) mean reduction in the co-primary endpoint of serum transthyretin concentration compared to baseline; reduced ttr protein production; significant treatment effect on modified neuropathy impairment score, statistically difference change from baseline vs. external placebo group (p<0.0001); achieved secondary norfolk quality life questionnaire-diabetic neuropathy; eplontersen significantly improved patient-reported (p<0.0001)< td> | 9/7/22 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution | Charcot-Marie-Tooth disease type 1A | First patient enrolled | 9/12/22 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor (NMDAR) channel blocker | Major depressive disorder | Completed patient treatment in Reliance III monotherapy study; top-line data expected in fourth quarter 2022 | 9/20/22 | Neurology/Psychiatric |
| Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. | Zuranolone | GABAA receptor modulator | Major depressive disorder and postpartum depression | Results from 30-mg cohort of the Shoreline Study showed mean reduction in the 17-item Hamilton Rating Scale for Depression (HAMD-17) total score from baseline to day 15; ? 50% reduction in HAMD-17 total score; well-tolerated; improvements in depressive symptoms with zuranolone at day 15 were sustained beyond the end of treatment in landscape program; statistically significant reduction in depressive symptoms in Waterfall study | 9/19/22 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 | Nasal powder formulation of dihydroergotamine mesylate | Acute migraine | Ascend study showed favorable safety and tolerability profile; mild (82.7%) and transient treatment related adverse events; freedom from pain by 2 hours post-treatment (2hPF response) was achieved in 34.2% of all treated attack; freedom from most-bothersome-symptom by 2 hours post-treatment (2hMBS response) was achieved in 53.4% of all treated attacks; improved clinical performance in post-hoc analyses | 9/20/22 | Neurology/Psychiatric |
| Spine Biopharma Inc., of New York | SB-01 | 7-amino acid peptide that binds to and antagonizes TGF?1 activity | Pain-related disability, associated with degenerative disc disease | First patient treated | 9/7/22 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | Anti-CGRP subcutaneous injection | Migraine with co-morbid depression | Results at 3 months showed that 32% of patients on quarterly fremanezumab and 36% of patients on monthly fremanezumab achieved a ? 50% reduction in monthly migraine days compared to 19% of placebo; reductions were observed in both monthly migraine days and monthly headache days with both quarterly and monthly fremanezumab compared with placebo | 9/8/22 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Anxiolytic | Social anxiety disorder | Independent biostatisticians recommended continuing PALISADE-2 study without any changes after conducting interim analysis of 140 completed subjects; top-line data expected in the first half of 2023 | 9/8/22 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Pherine nasal spray | Social anxiety disorder | Data from volunteers showed significantly increased the electrogram response recorded from the nasal mucosa; rapidly lowering heart rate, respiratory rate, and electrodermal activity consistent with decreased sympathetic nervous system activity; activated nasal chemosensory cells and rapidly reduced sympathetic tone; provided rapid self-reported calmness | 9/19/22 | Neurology/Psychiatric |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Targeted C3 therapy | Geographic atrophy | Positive trends with increasing effects over time, demonstrating both monthly and every-other-month treatment preserved visual function of retinal cells near the lesion border compared to sham | 9/30/22 | Ocular |
| Glaukos Corp., of San Clemente, Calif. | Idose TR | Sustained-release travoprost implant | Glaucoma | Top-line data showed Idose TR successfully achieved its prespecified primary efficacy endpoints through 3 months; excellent tolerability and a favorable safety profile through 12 months | 9/7/22 | Ocular |
| Iveric Bio Inc., of Parsippany, N.J. | Zimura (avacincaptad pegol) | Complement C5 inhibitor | Geographic atrophy | Top-line data showed study met its prespecified primary endpoint; 14.3% reduction (p-value = 0.0064) in mean rate of growth (Slope) in area over 12 months using square root transformation; 17.7% reduction (p-value = 0.0039) using observed area; statiscally significant result with favourable safety profile; post-hoc analysis showed 25.5% reduction (p-value = 0.0037) using square root transformation; 32% reduction (p-value=0.0033) using observed area; no events of endophthalmitis; no intraocular inflammation events, and no ischemic optic neuropathy events through month 12 | 9/6/22 | Ocular |
| Neurophth Therapeutics Inc., of Wuhan, China | NR-082 | Gene therapy expressing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 | Leber hereditary optic neuropathy with ND4 mutation | First patient treated in the study testing the safety, tolerability and efficacy of NR-082 | 9/27/22 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating prostaglandin analogue | Open-angle glaucoma or ocular hypertension | Last patients completed their final (3-month) visit in the Mont Blanc study; 691 patients enrolled. top-line results in early Nov. 2022 | 9/19/22 | Ocular |
| Tearclear Inc., of Chicago. | TC-002 (latanoprost) | Prodrug analog of prostaglandin F2 alpha | Glaucoma | Met all primary and secondary endpoints in top-line results | 9/30/22 | Ocular |
| Vyluma Inc., of Bridgewater, N.J. | NVK-002 | Preservative-free eye drop | Myopia progression in children | Last patient visit completed | 9/1/22 | Ocular |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Aflibercept | Inhibitor of vascular endothelial growth factor | Diabetic macular edema and wet age-related macular degeneration | Photon and Pulsar trial meets primary endpoints in 12- and 16-week dosing; 91% and 89% of diabetic macular edema (DME) patients were rapidly initiated and maintained on 12- and 16-week dosing intervals (without need for regimen modification) through week 48, respectively; 79% and 77% of wet age-related macular degeneration (wAMD) patients were rapidly initiated and maintained on 12- and 16-week dosing intervals (without need for regimen modification) through week 48, respectively; BCVA improvement (p<0.0001 and p="0.0031)" in photon study (p="0.0009" pulsar at 12-week 16-week, respectively < td> | 9/8/22 | Ocular |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut | Epicutaneous patch | Peanut allergy | Received feedback from the U.S. FDA in the form of a partial clinical hold on its phase III VITESSE study | 9/21/22 | Other/Miscallaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Melanocortin-4 receptor agonist | Obesity and control of hunger in Bardet-Biedl syndrome (BBS) | Results showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients ages 6 to 17 years old; mean reduction of -0.75 in BMI-Z score for patients with BBS; mean reduction of -0.55 in BMI-Z score for patients (n=4) with LEPR deficiency; mean reduction of -1.51 in BMI-Z score for patients with POMC deficiency | 9/19/22 | Other/Miscallaneous |
| Boehringer Ingelheim GmbH., of Germany | Nintedanib | Small molecule tyrosine kinase inhibitor | Interstitial lung disease | Results published in the European Respiratory Journal showed improved primary endpoints; acceptable safety and tolerability profile with no new safety signals | 9/5/22 | Respiratory |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Inhibits interleukin-4 and interleukin-13 | Moderate to severe asthma | Results from open-label extension trial in children showed low rate of severe asthma attacks with an average of 0.118-0.124 events per year compared to 2.16-2.56 events per year at baseline in the pivotal trial; sustained improvement in lung function at 52 weeks; improvement of 8.71% points in lung function at 2 weeks ; 61%-68% of adverse events in 52-week treatment period | 9/5/22 | Respiratory |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Smoking cessation and nicotine addiction | Completed enrollment of 750 patients in the Orca-3 study; top-line data expected in the first half of 2023 | 9/27/22 | Toxicity and Intoxication |
| Astrazeneca plc, of Cambridge, U.K. | Capivasertib | AKT inhibitor | HR-positive breast cancer | Study met primary endpoints; statistically significant and clinically meaningful improvement in progression-free survival vs. placebo; overall survival data immature | 10/26/22 | Cancer |
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Alpine study showed zanubrutinib achieved superior progression-free survival vs. ibrutinib; well-tolerated | 10/12/22 | Cancer |
| Bristol Myers Squibb Co., of New York, and Merck & Co. Inc., of Rahway, N.J. | Reblozyl (luspatercept-aamt) | Erythroid maturation agent | Anemia due to very low-, low- or intermediate-risk myelodysplastic syndrome | The Commands study met its primary endpoint during an interim analysis, showing Reblozyl improved red blood cell transfusion independence with concurrent hemoglobin increase compared to epoetin alfa; results to be presented at an upcoming medical meeting | 10/31/22 | Cancer |
| Cel-Sci Corp., of Vienna, Va. | Multikine | Leukocyte interleukin injection | Locally advanced primary squamous cell carcinoma of the head and neck | In the 928-patient It-Matters study, there were 45 early tumor responses, including 5 complete responses and 40 partial responses | 10/17/22 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | Rubraca (rucaparib) | Oral, small molecule inhibitor of PARP1, PARP2 and PARP3 | Chemotherapy-naïve metastatic castration-resistant prostate cancer | Achieved the primary endpoint of improved radiographic progression-free survival by independent radiology review | 10/3/22 | Cancer |
| Epicentrx Inc., of Torrey Pines, Calif. | RRx-001 | Tumor-activated small molecule that inhibits NLRP3 inflammasome and repolarizes tumor-associated macrophages | Small-cell lung cancer | First patient dosed | 10/6/22 | Cancer |
| G1 Therapeutics Inc., of Research Triangle Park, N.C. | Cosela (trilaciclib) | CDK4/6 inhibitor | Metastatic triple-negative breast cancer | Completed enrollment; last patient randomized; enrolled 187 patients | 10/10/22 | Cancer |
| Galera Therapeutics Inc., of Malvern, Pa. | Avasopasem manganese | Selective small-molecule dismutase mimetic | Severe oral mucositis (SOM) in patients with locally advanced head and neck cancer | After 1-year follow up data in patients treated in combination with intensity-modulated radiation therapy+cisplatin showed 10% incidence of chronic kidney disease compared to placebo 20% (p=0.0043); tumor outcomes and survival maintained in patients at 1 year | 10/26/22 | Cancer |
| Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Non-small-cell lung cancer | Results in combination with chemotherapy showed significant improvements in progression-free survival and overall survival compared with chemotherapy alone; median PFS was 8.4 vs. 5.6 months; 1-year PFS rates of 36.7% vs. 17.2%; median OS not reached vs. 17.1 months; OS rates at 2 years were 51.2% vs. 33.9%; no new safety signals | 10/12/22 | Cancer |
| Oncopeptides AB, of Stockholm | Melflufen | Lipophilic peptide conjugated alkylating drug | Relapsed refractory multiple myeloma | Lighthouse study in combination with daratumumab subcutaneous plus dexamethasone showed superior progression-free survival with a HR of 0.062(p=0.0005); superior overall response rate (p=0.0051) | 10/26/22 | Cancer |
| OSE Immunotherapeutics SA, of Nantes, France | Tedopi | Cancer vaccine | Second-line advanced or metastatic non-small-cell lung cancer after ICI failure | Preparing to run a confirmatory pivotal study; filed for a type C meeting with the FDA to validate the study protocol | 10/20/22 | Cancer |
| Pfizer Inc., of New York | Talzenna (talazoparib) | Poly ADP-ribose polymerase inhibitor | Metastatic castration-resistant prostate cancer, with or without mutations in homologous recombination repair genes | The Talapro-2 study met its primary endpoint of improved radiographic progression-free survival for Talzenna plus Xtandi (enzalutamide) compared to Xtandi alone (HR<0.696); overall survival showed a trend towards the combination therapy being longer but data aren’t mature< td> | 10/4/22 | Cancer |
| Y-Mabs Therapeutics Inc., of New York | Omblastys (131I-omburtamab) | Radiopharmaceutical | Central nervous system/leptomeningeal metastasis from neuroblastoma | 12-month overall survival 73.5%, with a median follow-up of 25 months | 10/3/22 | Cancer |
| Applied Therapeutics Inc., of New York | AT-001 | Aldose reductase inhibitor | Diabetic cardiomyopathy | Completed enrollment in the Arise-HF study; data expected in 2023 | 10/25/22 | Cardiovascular |
| Biocardia Inc., of Sunnyvale, Calif. | Cardiamp | Autologous cell therapy | Heart failure | Clinical improvement with 100% survival over two years. | 10/3/22 | Cardiovascular |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Sotagliflozin | Dual SGLT1 and SGLT2 inhibitor | Heart failure | Reduced first and subsequent heart failure-related events in patients admitted for worsening disease | 10/2/22 | Cardiovascular |
| Merck KGaA, of Darmstadt, Germany | Sotatercept | Activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein | Pulmonary arterial hypertension | Stellar study met primary endpoints; statistically significant and clinically meaningful improvement in 6-minute walk distance (6MWD); 8/9 secondary efficacy outcome measures achieved statistical significance; improvement in N-terminal pro-B-type natriuretic peptide (NT-proBNP) level; significant improvement in exercise capacity and key secondary outcome measures compared to placebo when added to background therapy; did not achieve statistical significance on Cognitive/Emotional Impacts domain score of PAH-SYMPACT | 10/10/22 | Cardiovascular |
| Milestone Pharmaceuticals Inc., of Montreal | Etripamil | Calcium channel blocker | Paroxysmal supraventricular tachycardia | In the 706-patient Rapid study, 64.3% of patients taking etripamil converted to sinus rhythm within 30 minutes compared to 31.2% for patients on placebo (HR=2.62, p<0.001); 3 median time to conversion for patients who took etripamil was times faster than placebo< td> | 10/17/22 | Cardiovascular |
| Tenax Therapeutics Inc., of Morrisville, N.C. | Levosimendan | Inhibits PDGF receptors and c-KIT | Pulmonary hypertension and heart failure with preserved ejection fraction | Substudy of patients who transitioned from intravenous to oral showed further numerical improvements in 6-minute walk distance, BNP/NT-ProBNP and all key KCCQ domains; no serious adverse events; well-tolerated | 10/10/22 | Cardiovascular |
| Abeona Therapeutics Inc., of New York | EB-101 | Autologous cell therapy expressing the COL7A1 gene | Recessive dystrophic epidermolysis bullosa | Completed patient follow-up in the Viital study; top-line data expected by early November 2022 | 10/4/22 | Dermatologic |
| Abeona Therapeutics Inc., of New York | EB-101 | Autologous, engineered cell therapy | Recessive dystrophic epidermolysis bullosa | Database locked on Oct. 18, 2022 for Viital study | 10/19/22 | Dermatologic |
| Connect Biopharma Holdings Ltd., of San Diego | CBP-201 | Antibody targeting interleukin-4 receptor alpha | Moderate-to-severe atopic dermatitis | Of the patients treated with CBP-201, 30.3% had an IGA of 0 or 1 and at least 2 grades of reduction at week 16 relative to baseline compared to 7.5% of patients receiving placebo (p <0.001); percent of patients achieving easi-50, easi-75, easi-90 were also greater for cbp-201 compared placebo (p <0.001)< td> | 10/4/22 | Dermatologic |
| Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib cream) | JAK1/JAK2 inhibitor | Nonsegmental vitiligo | Data from the True-V1 and -V2 studies published in The New England Journal of Medicine showed about 30% of patients treated with Opzelura achieved ?75% improvement from baseline on the Facial Vitiligo Area Scoring Index (F-VASI75) at week 24, compared to 13% of patients who received vehicle in 1 of the studies; at week 52, approximately 50% of patients taking the drug achieved F-VASI75 | 10/19/22 | Dermatologic |
| Novartis AG, of Basel, Switzerland | Iptacopan | Factor B inhibitor of the alternative complement pathway | Paroxysmal nocturnal hemoglobinuria | Study met its 2 primary endpoints (hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks and hemoglobin levels of 12 g/dL or more without the need for blood transfusions at 24 weeks) showing superiority over eculizumab or ravulizumab; data to be presented at an upcoming medical meeting | 10/24/22 | Dermatologic |
| Quoin Pharmaceuticals Ltd., of Ashburn, Va. | QRX-003 | Topical lotion; broad-spectrum serine protease inhibitor | Netherton syndrome | Planning to initiate second multicentr open label study in patients currently receiving treatment including systemic therapy | 10/18/22 | Dermatologic |
| Applied Therapeutics Inc., of New York | Gavorestat (AT-007) | CNS penetrant; aldose reductase inhibitor | Galactosemia | Data showed trend in clinical benefit vs. placebo; study did not yet reach statistical significance at 12 months of treatment; trial will continue to 18 months in blinded format; safe and well-tolerated | 10/6/22 | Endocrine/Metabolic |
| Crinetics Pharmaceuticals Inc., of San Diego | Paltusotine | Nonpeptide SST2 receptor agonist | Acromegaly | Completed enrollment in Pathfndr-1 study; top-line results expected in the 2023 Q3; Pathfndr-2 study’s enrollment is ongoing and top-line data expected in the 2023 Q4 | 10/13/22 | Endocrine/Metabolic |
| Saol Therapeutics Inc., of Roswell, Ga. | Sodium dichloroacetate (SL-1009) | Inhibitor of pyruvate dehydrogenase kinases | Pyruvate dehydrogenase complex deficiency | Completed enrollment in the study; top-line data expected in the second half of 2023 | 10/4/22 | Endocrine/Metabolic |
| Abivax SA, of Paris | ABX-464 (obefazimod) | Binds to the RNA cap binding protein (CBC 80/20) complex of RNA | Moderate to severe active ulcerative colitis | First patient enrolled; top-line data expected in the end of 2024 | 10/11/22 | Gastrointestinal |
| Albireo Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Progressive familial intrahepatic cholestasis | Pooled analysis from PEDFIC 1 and PEDFIC 2 trials showed Bylvay reduced serum bile acids and pruritus in children (31%); improvement in sleep, growth and quality of life; well-tolerated | 10/13/22 | Gastrointestinal |
| Astrazeneca SA, of Cambridge, U.K. | Fasenra (benralizumab) | Monoclonal antibody targeting IL-5 receptor alpha | Eosinophilic esophagitis | Fasenra produced a statistically significant improvement in histological disease remission, but not a change in dysphagia symptoms, compared to placebo; data to be presented at an upcoming medical meeting | 10/25/22 | Gastrointestinal |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Stelara (ustekinumab) | IL-12/IL-23 receptor antagonist | Moderately to severely active ulcerative colitis | Long-term extension study (UNIFI) showed 64.9% of patients were in symptomatic remission after 44 weeks of maintenance treatment; 67.2% were in symptomatic remission at week 200 compared to 71.8% of patients who were in symptomatic remission at week 44 in 174 biologic-naïve patients; 72.9% in clinical remission at week 44 were also in symptomatic remission at week 200; no new safety signals; rates of corticosteroid-free symptomatic remission at week 200 were generally similar between the q8w group (63.6%) and the q12w group (66.7%); 94.8% in the q8w group and 97.9% in the q12w group were corticosteroid-free at week 200 | 10/10/22 | Gastrointestinal |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor | Progressive familial intrahepatic cholestasis | Change from baseline in ItchRO severity was -1.7 for Livmarli and -0.6 for placebo (p=0.0098) for PFIC2 patients; change from baseline in serum bile acid was -176 for Livmarli and 11 for placebo (p=0.0013) | 10/24/22 | Gastrointestinal |
| Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Potassium-competitive acid blocker | Non-erosive gastroesophageal reflux disease | Completed enrollment in the Phalcon-Nerd Daily Dosing study; top-line data from the primary endpoint expected in the first quarter of 2023; full results from the study expected in late 2023 | 10/24/22 | Gastrointestinal |
| Sanofi SA, of Paris, and Regeneron Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Fully human monoclonal antibody that inhibits the signaling of the IL-4 and IL-13 pathways | Eosinophilic esophagitis | Data showed Dupixent led to significant improvements in the primary efficacy measure for higher and lower dose groups at 16 weeks; 68% of patients on higher dose and 58% of patients on lower dose achieved the primary endpoint of significant histological disease remission compared to 3% for placebo (both p<0.0001); significant improvements in abnormal endoscopic findings of esophagus, with a reduction 3.5 points compared to an increase 0.3 for placebo (p<0.0001); numerical improvement the proportion days children experienced disease symptoms from baseline as reported by caregivers placebo, not statistically significant; 3.09 percentile body weight age baseline, 0.29 placebo; consistent safety profile< td> | 10/11/22 | Gastrointestinal |
| Tricida Inc., of South San Francisco | Veverimer | Hydrochloric acid binder | Metabolic acidosis and chronic kidney disease | The Valor-CKD study didn’t meet its primary endpoint; 20.2% of patients taking veverimer had an event, defined as renal death, end-stage renal disease or a confirmed greater than or equal to 40% reduction in estimated glomerular filtration rate, compared to 20.1% of patients who received placebo (p=0.898, HR=0.99) | 10/24/22 | Genitourinary/Sexual Function |
| Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Conditioning regimen prior to bone marrow transplant | Sierra study met its primary endpoint of percent of patients who had a durable complete remission of 6 months post initial remission for Iomab-B compared to conventional care (p<0.0001)< td> | 10/31/22 | Immune |
| Biogen Inc., of Cambridge, Mass. | Vumerity (diroximel fumarate) | Monomethyl fumarate prodrug | Relapsing-remitting multiple sclerosis | Final results from EVOLVE-MS-1 study showed decreased disease activity and favorable tolerability; reduction in annualized relapse rate (ARR) was 81.6%; estimated proportion of patients who were relapse-free was 82.4%; estimated proportion with no evidence of disease activity (NEDA-3) was 41.1%; 24.3% of patients discontinued treatment due to GI adverse events | 10/26/22 | Immune |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Relapsing-remitting multiple sclerosis | Interim analysis from Daybreak open-label extension study showed 68% of participants were relapse-free and demonstrated an adjusted annualized relapse rate (ARR) of 0.099 at up to 74 months of treatment; long-term efficacy was sustained on MRI measures after 60 months of treatment; 6-month confirmed disability progression of 14% | 10/26/22 | Immune |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Multiple sclerosis | Post-hoc analysis of Sunbeam, Radiance and Daybreak open-label extension studies demonstrated a greater proportion of patients treated with Zeposia vs. interferon beta-1a had a lower annualized rate of brain volume loss; Sunbeam study at month 12 showed 50.9% vs. 37.5%, respectively; Radiance study at month 24 showed 63.1% vs. 50%, respectively | 10/26/22 | Immune |
| Genentech Inc., of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Multiple sclerosis | Interim data of open-label Ensemble study (96 weeks) showed 77% of patients achieved no evidence of disease activity; majority of patients had no relapses (93%); no MRI lesion activity (89%); no 24-week confirmed disability progression (91%); average annualized relapse rate in all patients was low; EDSS score from baseline significantly improved from 1.8 to 1.67 (p<0.0001); favorable safety profile< td> | 10/26/22 | Immune |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy product | Immunosuppression | Reported patient death in Freedom-1 study; triggered prespecified, temporary stopping requirement and review by the data monitoring committee | 10/20/22 | Immune |
| Agenus Inc., of Lexington, Mass. | QS-21 Stimulon adjuvant | Saponin extracted from the bark of the Quillaja saponaria | Respiratory syncytial virus infection | Data showed overall vaccine efficacy of 82.6% against RSV-lower respiratory tract disease in adults aged 60 years and above; robust neutralizing antibody response generated against both subtypes; well-tolerated; favorable safety profile | 10/13/22 | Infection |
| Allecra Therapeutics GmbH, of Saint-Louis, France, and Weil Am Rhein, Germany | Cefepime/enmetazobactam | Penicillin binding protein inhibitor; beta-lactamase inhibitor | Complicated urinary tract infections | Data from the Allium trial published in the Journal of the American Medical Association showed combo treatment met criteria for noninferiority and superiority vs. piperacillin/tazobactam in primary outcome of clinical cure and microbiological eradication, which occurred in 79.1% (273/345) compared to 58.9% (196/333); in 20.9% (142/678) of patients with ESBL-producing baseline pathogen, 73.7% (56/76) in cefepime/enmetazobactam group and 51.5% (34/66) in piperacillin/tazobactam group achieved composite outcome (difference 30.2% [95% CI, 13.4% to 45.1%]) | 10/5/22 | 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 | Revival study stopped per recommendation of data monitoring committee after per-protocol pre-specified interim futility analysis on the primary endpoint; no safety concern; significant reduction of events was observed in the combined population p<0.05; reduction in 28-day all-cause mortality compared to those treated with placebo (p<0.01)< td> | 10/20/22 | Infection |
| Aridis Pharmaceuticals Inc., of Los Gatos, Calif. | AR-301 | Monoclonal antibody targeting S. aureus alpha-toxin | Ventilator associated pneumonia caused by S. aureus | Completed enrollment of the AR-301-002 ASAP-1 study; top-line data expected in December 2022 | 10/4/22 | Infection |
| Codagenix Inc., of Farmingdale, New York | Coviliv | Intranasal, live-attenuated virus vaccine | COVID-19 | Initiated dosing; study to evaluate the safety, efficacy and immunogenicity | 10/26/22 | Infection |
| Evofem Biosciences Inc., of San Diego | EVO-100 (Phexxi) | Contraceptive vaginal gel | Chlamydia and gonorrhea infection | Evoguard study in women did not achieve its endpoints; consistent safety profile; 2 women in the study discontinued due to adverse events | 10/11/22 | Infection |
| Iterum Therapeutics plc, of Dublin | Sulopenem | Beta-lactam antibiotic; anti-inflammatory/sulopenem etzadroxil combined with probenecid in a bilayer tablet | Uncomplicated urinary tract infections | First patient dosed | 10/20/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.214 (Spikevax bivalent original/omicron) | Omicron-containing bivalent booster vaccine | COVID-19 | Data from 90 days after administration as a fourth booster dose (50 mcg) showed superior neutralizing antibody response against omicron BA.1 compared to a 50-mcg booster dose of mRNA-1273 in all participants; durable with higher antibody titers sustained through 3 months; significantly higher neutralizing antibody responses against omicron BA.4/BA.5 compared to mRNA-1273; no new safety concerns | 10/19/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | Nuvaxovid (NVX-CoV2373) | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Data from Prevent-19 trial achieved its prespecified immunologic endpoint; neutralizing titers were 2.7-fold higher than those with primary vaccination; significant boost observed for antibody against omicron BA.1, BA.2 and BA.5; well-tolerated in both adults and adolescents; mild to moderate reactogenicity | 10/12/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | Nuvaxovid (NVX-CoV2373) | SARS-CoV-2 recombinant spike protein nanoparticle vaccine containing saponin-based Matrix-M adjuvant | COVID-19 prophylaxis | Data from study 307 achieved its primary endpoint; no serious related treatment-emergent adverse events | 10/12/22 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Movantik (naloxegol) | Opioid antagonist | Opioid-induced constipation | Study showed clinically meaningful symptom improvement; rapid and clinically meaningful constipation-related symptom improvement in the overall population; achieved clinically meaningful quality-of-life improvement; well-tolerated and favorable safety profile | 10/26/22 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Anti-inflammatory | Mild to moderate COVID-19 | Finalized an amended protocol that will be submitted to the U.S. FDA with a new primary endpoint measuring the proportion of participants with improvement in at least 2 COVID-19-related clinical symptoms on or before day 14 compared with baseline for bucillamine compared to placebo | 10/14/22 | Infection |
| Siga Technologies Inc., of New York | Tpoxx (tecovirimat) | Antiviral | Monkeypox | Started 3 clinical trials; one in the U.S. testing the drug in more than 500 patients, including children and those who are pregnant or breastfeeding; one in the U.K. of at least 500 patients including children weighing ?13 kg; one in the Democratic Republic of Congo enrolling more than 450 patients, including children weighing ?3 kg and those who are pregnant or breastfeeding | 10/12/22 | Infection |
| Summit Therapeutics Inc., of Menlo Park, Calif. | Ridinilazole | Antibacterial | Clostridioides difficile infection | Data from the Ri-Codify study showed ridinilazole produced a 53% relative risk reduction in recurrence of C. difficile infection compared to treatment with vancomycin | 10/20/22 | Infection |
| Vaxxinity Inc., of Dallas | UB-612 | COVID-19 vaccine | COVID-19 prophylaxis | Completed enrollment in the study testing the vaccine as a booster; top-line data expected in the fourth quarter of 2022 | 10/24/22 | Infection |
| Venatorx Pharmaceuticals Inc., of Malvern, Pa. | Cefepime-taniborbactam | Cephalosporin derivative; beta-lactamase inhibitor | Complicated urinary tract infections (cUTI), including acute pyelonephritis | Study met the prospectively defined non-inferiority primary endpoint of composite microbiologic and clinical response versus meropenem at the test of cure; 70.6% response rate for cefepime-taniborbactam vs. 58% for meropenem; statistically superior to meropenem for the composite endpoint p=0.0088; microbiologic endpoint p=0.0085; 63.8% response rate for cefepime-taniborbactam vs. 51.7% for meropenem p=0.0157; consistent safety profile | 10/20/22 | Infection |
| Viiv Healthcare Ltd., of London | Vocabria (cabotegravir) | Integrase strand transfer inhibitor | HIV infection | In the Carisel study, there was high acceptability, appropriateness and feasibility of Vocabria plus Rekambys (rilpivirine, Johnson & Johnson); 56% of 70 staff reported optimal implementation within 1-3 months | 10/24/22 | Infection |
| GSK plc, of London | Otilimab (MOR-103/GSK-3196165) | Monoclonal antibody targeting granulocyte-macrophage colony-stimulating factor | Moderate to severe rheumatoid arthritis | Data from Contrast-3 trial did not demonstrate statistical significance on the primary endpoint of ACR20 response vs. placebo at week 12; full results expected in 2023 | 10/27/22 | Inflammatory |
| Octapharma AG, of Lachen, Switzerland | Octagam | Liquid preparation of highly purified immunoglobulin G | Dermatomyositis | Proderm results published in The New England Journal of Medicine showed study achieved primary endpoint (79% vs. 44%; p <0.001) at week 16; well-tolerated< td> | 10/6/22 | Inflammatory |
| Biogen Inc., of Cambridge, Mass. | BIIB-122 | Selective, central nervous system-penetrant small molecule inhibitor of LRRK2 | Parkinson's disease | Dosing commenced | 10/3/22 | Musculoskeletal |
| Genentech Inc., of South San Francisco, part of the Roche Group | Evrysdi (risdiplam) | SMN2 splicing modulator | Spinal muscular atrophy | New 2-year data showed Evrysdi improved or maintained motor function; rapid increase in SMN protein levels; consistent safety data | 10/12/22 | Musculoskeletal |
| Acelrx Pharmaceuticals Inc., of Hayward, Calif. | Dsuvia (sufentanil) | Sublingual formulation of the opioid | Pain from complex plastic surgery procedures | Study of 324 patients showed that treatment with Dsuvia initially and on an as-needed basis during surgery allowed doctors to perform procedures without general anesthesia or intravenous opioids; patient recovery time averaged 19 minutes | 10/31/22 | Neurology/Psychiatric |
| Astrazeneca plc, of Cambridge, U.K. | Ultomiris (ravulizumab) | Long-acting C5 complement inhibitor | Neuromyelitis optica spectrum disorder | Study achieved primary and secondary endpoint; Ultomiris significantly reduced relapse risk in adults with anti-aquaporin-4 antibody-positive patients compared to placebo; median treatment duration of 73 weeks; relapse risk reduction of 98.6% p<0.0001; 48 relaspse-free at weeks compared to 63% of patients in placebo; annualized relapse rate p<0.0001; clinically important change from baseline mobility measured by hauser ambulation index p="0.0228;" eq-5d score vas edss> | 10/27/22 | Neurology/Psychiatric |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous MSC-NTF cells | Amyotrophic lateral sclerosis | Data showed Nurown decreased biomarkers associated with neuroinflammation and neurodegeneration; increased neuroprotective biomarkers over 20 weeks; key secondary endpoint was nominally statistically significant (p=0.050) | 10/7/22 | 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 | Initiated open-label extension study to generate long-term safety and tolerability data at 100 mg twice daily over 52 weeks; expected to enroll 1,600 patients | 10/13/22 | Neurology/Psychiatric |
| Compass Pathways plc, of London | COMP-360 | Psilocybin therapy | Treatment resistant depression | Designed phase III study; two pivotal trials and one long-term follow-up; planning to initiate study at the end of 2022; primary endpoint in both pivotal trials is the change from baseline in MADRS total score at week 6 | 10/12/22 | Neurology/Psychiatric |
| Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab) | B-lymphocyte antigen CD19 inhibitor | Neuromyelitis optica spectrum disorder | Data showed increase in plasma cells at the preceding visit relative to baseline (p< 0.01); no significant increases in any B-cell subsets at time of attack; significant increase in AQP4-IgG+ titers were observed at time of attack relative to baseline (p=0.02) but not in those treated with Uplizna (p=0.76); changes in AQP4-IgG titers from baseline to attack were not significantly different between treatment groups (p=0.15); decreased AQP4-IgG+ titer relative to placebo; ?2-fold decrease in AQP4-IgG titers from baseline compared to 18% placebo (p=0.01) | 10/26/22 | Neurology/Psychiatric |
| Idorsia Ltd., of Allschwil, Switzerland | Daridoexant | Dual orexin receptor antagonist | Insomnia | Demonstrated significant improvement in the subjective measures of Total Sleep Time and Latency for Sleep Onset measures in Japanese patients | 10/3/22 | Neurology/Psychiatric |
| Pharmazz Inc., of Willowbrook, Ill. | Sovateltide | Endothelin B receptor agonist | Acute ischemic stroke | Change in mean mRS was 1.48 for patients receiving sovateltide and 2.1 for placebo (p=0.008); 76.1% of patients receiving sovateltide and 52.9% of patients who got placebo had a mRS change greater than 2 (p=0.005) | 10/31/22 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor (NMDAR) channel blocker | Major depressive disorder | Reliance III study did not achieve primary endpoint; MADRS reduction of 14.8 points at day 28 vs. 13.9 points for the placebo arm; favorable tolerability and safety | 10/13/22 | Neurology/Psychiatric |
| Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. | Zuranolone | GABAA receptor modulator | Postpartum depression | Data from the 195-patient Skylark study showed the drug improved the 17-item Hamilton Rating Scale for Depression total score least-squares mean by -15.6 points compared to -11.6 for placebo (p=0.0007) | 10/17/22 | Neurology/Psychiatric |
| Taurx Pharmaceuticals Ltd., of Singapore | Hydromethylthionine mesylate | Hydromethylthionine mesylate | Alzheimer's disease | Study (n=105) at 16 mg/day showed statistically significant cognitive improvement of 2 units over the pre-treatment baseline at 6 months (p=0.0002), 12 months (p=0.0391) and 18 months (p=0.0473) on the ADAS-cog13 scale; statistically significant reductions in disease progression as measured by change in cognitive function (p=0.0008) and brain atrophy (p<0.0001); rate of progression brain atrophy was significantly less than in adni mci subjects (p<0.0001) and comparable to that seen healthy aging subjects< td> | 10/6/22 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Austedo (deutetrabenazine) | Synaptic vesicular amine transporter inhibitor | Chorea associated with Huntington’s disease | Results from 3-year open-label, single-arm, 2-cohort, multicenter extension study showed medication compliance rates were greater than 90%; improved and maintained chorea control in both the rollover cohort and the switch cohort; reduction in total motor score (7.1-point and 2.4-point in 2 cohorts); no new safety concerns; results published in CNS Drugs | 10/18/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-2191 | Intravitreal methotrexate 0.8% | Proliferative vitreoretinopathy | Study achieved primary endpoint; statistically superior to historical control for the prevention of retinal detachment due to PVR over 6 months (p=0.024); not statistically powered for secondary or exploratory endpoints; complete retinal attachment by 6 months, macular attachment by 6 months and epiretinal membrane formation (overall p=0.047); well-tolerated; no observed treatment-emergent serious adverse events | 10/6/22 | Ocular |
| Kubota Vision Inc., of Seattle, a unit of Tokyo-based Kubota Pharmaceutical Holdings Co. Ltd. | Emixustat | Retinal pigment epithelium protein 65 inhibitor | Stargardt disease | A post hoc analysis showed that emixustat treatment produced a 40.8% reduction in lesion progression compared to placebo at 24 months for the subgroup of patients with smaller lesions at baseline | 10/3/22 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating bimatoprost eye drop | Open-angle glaucoma or ocular hypertension | NCX-470 produced an intraocular pressure reduction of 8 to 9.7 mmHg, compared to 7.1 to 9.4 mmHg for latanoprost, meeting the test for noninferiority; comparing time-matched changes, NCX-470 was superior to latanoprost (p<0.049) 4 6 at of timepoints but did not achieve statistical superiority on prespecified secondary endpoint< td> | 10/31/22 | Ocular |
| Roche Holding AG, of Basel, Switzerland, and its subsidiary Genentech Inc. | Vabysmo (faricimab) | Bispecific antibody for the eye targeting Ang-2 and VEGF-A | Macular edema due to branch and central retinal vein occlusion | Top-line results of Balaton and Comino study met their primary endpoints; rapid drying of retinal fluid from baseline through week 24; reduction in central subfield thickness; well-tolerated | 10/27/22 | Ocular |
| Vyluma Inc., of Bridgewater, N.J. | NVK-002 | Low-dose, preservative-free atropine eye drop | Myopia progression in children | Data showed 0.01% atropine achieved statistically significant and clinically meaningful differences from placebo in every key outcome measure; 0.02% atropine demonstrated efficacy at several time points; strong safety and tolerability; no ocular serious adverse events | 10/27/22 | Ocular |
| Albireo Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Alagille syndrome | Top-line study met its primary endpoint of improvement in pruritus (p=0.002) and its key secondary endpoint of reduction in serum bile acids (p=0.001); substantial improvements in multiple sleep parameters; no patient discontinuation; well-tolerated; low rates of drug-related diarrhea | 10/11/22 | Other/Miscallaneous |
| Altamira Therapeutics Ltd., of Hamilton, Bermuda | Bentrio | Nasal spray gel | Seasonal allergic rhinitis | Enrollment resumed in Australia | 10/3/22 | Respiratory |
| Boehringer Ingelheim Pharmaceuticals Inc., a unit of Boehringer Ingelheim GmbH, of Ingelheim, Germany | BI-1015550 | PDE4B inhibitor | Idiopathic pulmonary fibrosis | First patient enrolled | 10/26/22 | Respiratory |
| Covis Pharma GmbH, of Zug, Switzerland | Eklira (aclidinium bromide) and Duaklir (aclidinium bromide/formoterol) | Inhaled respiratory medicines | Moderate to severe stable chronic obstructive pulmonary disease | Top-line results from Avant study achieved statistically significant and clinically important outcomes for all key endpoint measures of efficacy in adult patients; no new safety concerns | 10/25/22 | Respiratory |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso DPI (inhaled treprostinil) | PGI2 agonist | Idiopathic pulmonary fibrosis | First patient enrolled | 10/11/22 | Respiratory |
| Akeso Inc., of Hong Kong | Ivonescimab (AK-112) | PD-1/VEGF bispecific antibody | EGFR-mutant locally advanced or metastatic non-squamous non-small-cell-lung cancer | Completed patient enrollment | 11/4/22 | Cancer |
| Astellas Pharma Inc., of Tokyo | Zolbetuximab | Monoclonal antibody targeting Claudin 18.2 (CLDN18.2) | HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | Top-line results from Spotlight study (n=566) in combination with mFOLFOX6 (regimen of oxaliplatin, leucovorin and fluorouracil) met its primary endpoint with statistical significance in progression-free survival for patients compared to placebo; achieved secondary endpoint of overall survival with statistical significance compared to placebo; nausea, vomiting and decreased appetite as adverse events | 11/17/22 | Cancer |
| Aura Biosciences Inc., of Boston | Belzupacap sarotalocan (AU-011) | Virus-like drug conjugate | Early stage choroidal melanoma | Initiated startup activities for the global phase III study; evaluate the efficacy and safety in suprachoroidal administration; three arm randomized and masked design; planning to enroll 75 adult patients | 11/10/22 | Cancer |
| Ayala Pharmaceuticals Inc., of Rehovot, Israel | AL-102 | Oral gamma-secretase inhibitor | Desmoid tumors | First patient dosed | 11/16/22 | Cancer |
| CNS Pharmaceuticals Inc., of Houston | Berubicin | Anthracycline for crossing the blood-brain barrier | Recurrent glioblastoma multiforme | First patient enrolled in Europe and dosed in France | 11/2/22 | Cancer |
| GSK plc, of London | Blenrep (belantamab mafodotin) | Antibody-drug conjugate comprising a humanized BCMA monoclonal antibody covalently linked to the cytotoxic agent auristatin F via non-cleavable linker | Relapsed or refractory multiple myeloma | Dreamm-3 study did not meet its primary endpoint of progression-free survival (PFS); median progression-free survival was longer for belantamab mafodotin vs. dexamethasone/pomalidomide (PomDex) at 11.2 months vs. 7 months; overall response rate was 41% for belantamab mafodotin and 36% for PomDex; deeper response rate when compared with PomDex (25% VGPR or better with belantamab mafodotin compared to 8% with PomDex); median follow-up was 11.5 months for belantamab mafodotin and 10.8 months for PomDex; duration of response (DOR) was not reached; DOR rates at 12 months were 76.8% and 48.4% for belantamab mafodotin and PomDex, respectively; no new safety signals | 11/7/22 | Cancer |
| Henlius Biotech Inc., of Shanghai | Hansizhuang (serplulimab) | Anti-PD-1 monoclonal antibody | Extensive-stage small-cell lung cancer | First patient dosed in bridging head-to-head trial against Tecentriq (atezolizumab, Roche Holding AG) | 11/30/22 | Cancer |
| Hutchmed Ltd., of Hong Kong | Fruquintinib | Inhibits VEGFR 1, 2 and 3 | Advanced gastric or F17gastroesophageal junction adenocarcinoma | Trial met 1 of the primary endpoints of progression-free survival; not statistically significant per the prespecified statistical plan on overall survival; statistically significant improvement in secondary endpoints, including objective response rate, disease control rate and improved duration of response; consistent safety profile | 11/13/22 | Cancer |
| Immix Biopharma Inc., of Los Angeles | IMX-110 | Doxorubicin and curcumin; nanoparticle formulation | Rhabdomyosarcoma | Received institutional review board approval to enroll pediatric patients in upcoming trial | 11/11/22 | Cancer |
| Infinity Pharmaceuticals Inc., of Cambridge, Mass. | Eganelisib | PI3K-gamma inhibitor | Metastatic triple-negative breast cancer | Update from Mario-3 study showed 57/62 evaluable patients with a median duration of follow-up of 10 months had progression-free survival of 36%, 37.5% and 34.7% in ITT, PD-L1 (+)and PD-L1 (-) patients, respectively; complete response of 16.7% and 5.7% in PD-L1 (+) and PD-L1 (-) patients, respectively; overall response rate of 66.7% and 54.3%, respectively; median PFS of 6.4 months and 7.3 months; no new safety signals | 11/14/22 | Cancer |
| Menarini Group, of Florence, Italy, and subsidiary Stemline Therapeutics | Elacestrant | Oral SERD | ER+/HER2- metastatic breast cancer | Post-hoc analysis from Emerald study, based on the duration of prior CDK4/6i usage, showed clinically meaningful results which favor monotherapy elacestrant, both in the total patient population as well as in patients with ESR1-mutation; increased duration of prior CDK4/6i in metastatic patients was positively associated with longer progression-free survival on elacestrant but not with standard-of-care endocrine monotherapy | 11/30/22 | Cancer |
| Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 | HER2-negative locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma. | Keynote-859 study met its primary endpoint of overall survival in combination with chemotherapy; statistically significant and clinically meaningful improvements in progression-free survival and overall response rate in the all-randomized patient population; no new safety signals | 11/22/22 | Cancer |
| Merrimack Pharmaceuticals Inc., of Cambridge, Mass., and Ipsen SA, of Paris | Onivyde | Liposomal injection of irinotecan | Metastatic pancreatic ductal adenocarcinoma | Study met its primary endpoint; clinically meaningful and statistically significant improvement in overall survival compared to nab-paclitaxel plus gemcitabine; improvement in progression-free survival | 11/9/22 | Cancer |
| Northwest Biotherapeutics Inc., of Bethesda, Md. | Dcvax-L | Dendritic cell-based vaccine | Newly diagnosed and recurrent glioblastoma multiforme | Top-line results showed study achieved primary and secondary endpoint; median overall survival (mOS) was 19.3 months vs. 16.5 months for placebo (p=0.002); survival 13% vs. 5.7% at 60 months (p<0.001) 24 30 in recurrent gbm; survival at and months post-recurrence was 20.7% vs. 9.6%, 11.1% 5.1%, respectively; mos 30.2 21.3 (p="0.027)" newly diagnosed gbm patients with methylated mgmt< td> | 11/18/22 | Cancer |
| Panbela Therapeutics Inc., of Minneapolis | Ivospemin (SBP-101) | Polyamine analogue | Metastatic pancreatic ductal adenocarcinoma | First patient enrolled in Aspire study | 11/28/22 | Cancer |
| Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Independent data monitoring committee recommended for phase III Regal study protocol update; increase from 116 patients to range of 125-140 patients; primary endpoint is overall survival; interim analysis at 60 events; final analysis at 80 events | 11/14/22 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Iclusig (ponatinib) | Kinase inhibitor targeting BCR-ABL1 | Philadelphia chromosome-positive acute lymphoblastic leukemia | Phallcon study met its primary endpoint; achieved higher rates of minimal residual disease-negative complete remission in combination with reduced-intensity chemotherapy compared to imatinib | 11/17/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agent | Clear cell renal cell carcinoma | Study met all of its primary and secondary endpoints; 86% sensitivity and 87% specificity; 93% positive predictive value | 11/7/22 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Repatha (evolocumab) | Human monoclonal antibody that inhibits PCSK9 | Atherosclerotic cardiovascular disease | Analysis of Fourier and fourier open-label extension study from 3500 patients achieved LDL-C levels of <20 mg dl ; 39% patient achieved ldl-c levels of <40 dl; key secondary efficacy endpoint p for trend<0.0001; well-tolerated; no new safety signals< td> | 11/7/22 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Heart failure | Pre-specified analysis of Deliver phase III study improved symptom burden, physical limitations and quality of life as measured by mean KCCQ scores; sustained benefits at 8 months; p <0.001 for all; published in journal of the american college cardiology< td> | 11/7/22 | Cardiovascular |
| Idorsia Ltd., of Allschwil, Switzerland, and Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson | Aprocitentan | Dual endothelin receptor antagonist | Resistant hypertension/difficult-to-control hypertension | Precision study met primary (p=0.0046 at 25-mg) and secondary endpoint (p<0.0001 25 at 25-mg); 28% and 37% of the patients treated with 12.5 mg aprocitentan, respectively vs. 19% in placebo for treatment related adverse events; simultaneously published lancet< td> | 11/8/22 | Cardiovascular |
| Milestone Pharmaceuticals Inc., of Montreal | Etripamil | Calcium channel blocker | Paroxysmal supraventricular tachycardia | Rapid study achieved its primary endpoint; statistically significant and clinically meaningful difference in time to PSVT conversion compared to placebo, converted in 30 minutes (64.3% vs. 31.2%, p<0.001); 90 80.6% of etripamil patients converted vs. 60.7% placebo by minutes post-study p<0.001; statistical significance was maintained throughout the 5-hour observation window; mild-to-moderate randomized treatment emergent adverse events; no serious additional medical interventions less frequently compared to 15% 25%; p="0.103;" fewer emergency department visits (14% 21%; than placebo< td> | 11/7/22 | Cardiovascular |
| Abeona Therapeutics Inc., of New York | EB-101 | Autologous, engineered cell therapy | Recessive dystrophic epidermolysis bullosa | Top-line results of study met its 2 co-primary efficacy endpoints; significantly improved wound healing vs. control at 6 months (p<0.0001); 6 significant pain reduction associated with wound dressing changes vs. control at months (p="0.0002);" well-tolerated; no serious treatment-related adverse events< td> | 11/3/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream 0.3% | Topical inhibitor of phosphodiesterase-4 | Mild-to-moderate atopic dermatitis | Top-line results of the study achieved primary and all secondary endpoints; rapid and statistically significant improvements compared to vehicle on all secondary endpoints; 3.2% of individuals treated with roflumilast cream 0.15% achieved 75% reduction in EASI-75 at week 4 compared to vehicle 22% (p<0.0001); 4 33.6% of patients achieved 4-point reduction in worst itch numeric scale (wi-nrs) at week (vs. 20.7% for vehicle-treated subjects, [p<0.0001]); well-tolerated; favorable safety profile< td> | 11/15/22 | Dermatologic |
| Biofrontera Inc., of Woburn, Mass. | Ameluz | Aminolevulinic acid hydrochloride gel | Actinic keratosis on the extremities, neck and trunk | Launched phase III study; planning to enroll 165 patients | 11/21/22 | Dermatologic |
| Biothera Solutions Ltd., of China | BAT-2306 | Cosentyx (secukinumab) biosimilar | Moderate to severe plaque psoriasis | Initiated dosing in patients | 11/1/22 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 (deuruxolitinib) | JAK1 and JAK2 inhibitor | Moderate to severe alopecia areata | Thrive-AA1 study measuring absolute Severity of Alopecia Tool (SALT) score, showed 43% and 57% of the 8-mg twice-daily and 12-mg twice-daily dose groups, respectively, achieved a SALT score of 20 or less at week 24 compared to 1% of patients in the placebo group (p<0.0001); well-tolerated< td> | 11/21/22 | Dermatologic |
| Promore Pharma AB, of Stockholm | Ensereptide | Lactoferrin peptide | Prevention of skin scars | Final reports expected in April 2023 | 11/1/22 | Dermatologic |
| Reistone Biopharma Ltd., of Shanghai | Ivarmacitinib | JAK1 inhibitor | Moderate-to-severe atopic dermatitis | Study achieved co-primary endpoints as monotherapy at 8 mg once daily or 4 mg once daily; significantly improved IGA 0/1 response rate and EASI-75 response rate vs. placebo at week 16 (p<0.001); well-tolerated; without new safety findings< td> | 11/15/22 | Dermatologic |
| Ardelyx Inc., of Waltham, Mass. | Xphozah (tenapanor) | Phosphate absorption inhibitor | Hyperphosphatemia with chronic kidney disease | Monotherapy lowered serum phosphorus, with early responders maintaining response with continued treatment; meaningfully reduced serum phosphorus in patients with severe cases; in combination with phosphate binders, drug reduced patients interdialytic weight gain compared to binders alone | 11/3/22 | Endocrine/Metabolic |
| Daewoong Pharmaceutical Ltd., of Seoul, South Korea | Enavogliflozin | SGLT-2 inhibitor | Type 2 diabetes | Monotherapy showed statistically significant reduction in HbA1c by 1% over placebo group; significant improvements in weight, blood pressure, low-density lipoprotein cholesterol and high-density lipoprotein cholesterol compared to the placebo group; noninferiority in HbAc1 change compared to dapagliflozin 10 mg in combination studies of metformin and gemigliptin | 11/2/22 | Endocrine/Metabolic |
| Intrabio Inc., of Oxford, U.K. | IB-1001 | N-acetyl-L-leucine | Niemann-Pick disease type C | 100% target enrollment reached; readout expected in the second quarter of 2023 | 11/22/22 | Endocrine/Metabolic |
| Visen Pharmaceuticals Co. Ltd., of Shanghai | Lonapegsomatropin | Long-acting prodrug of unmodified somatropin | Growth hormone deficiency | Results achieved primary endpoint; an annualized height velocity (AHV) of 10.66 cm/year compared to 9.75 cm/year for the daily hGH at 52 weeks (p=0.0010); noninferior to the daily hGH; well-tolerated | 11/17/22 | Endocrine/Metabolic |
| Xeris Biopharma Holdings Inc., of Chicago | Recorlev (levoketonconazole) | Cortisol synthesis inhibitor | Cushings syndrome | Results published in the European Journal of Endocrinology from 60 patients who entered extended evaluation study showed 61% exhibited normal mean urinary free cortisol at month 6; 55% and 41% at months 9 and 12, respectively; reductions in late-night salivary cortisol and random serum cortisol were observed at month 6, 9, and 12; improved mean fasting glucose, total and LDL-cholesterol, body weight, body mass index, abdominal girth, Cushing QoL, and BDI-II scores at months 9 and 12; mean decrease in hirsutism score in female patients; mean testosterone levels decreased significantly at month 6; tumor diameter was stable in 27 patients, decreased in 1 patients and increased in 3 patients; well-tolerated; no new drug-related safety signals | 11/1/22 | Endocrine/Metabolic |
| 9 Meters Biopharma Inc., of Raleigh, N.C. | Vurolenatide | Long-acting injectable GLP-1 receptor agonist | Short bowel syndrome | Disclosed design of phase III study called Vibrant, which will test 50 mg given subcutaneously every 2 weeks for 12 weeks; about 105 patients to be enrolled; will assess reduction in weekly parenteral support volume requirements and the impact on malabsorptive diarrhea as measured by total stool output volume | 11/29/22 | Gastrointestinal |
| Albireo Inc., of Boston | Bylvay (odevixibat) | Ileal bile acid transport inhibitor | Biliary atresia | Completed patient enrollment; top-line results expected by the end of 2024 | 11/2/22 | Gastrointestinal |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Thyroid hormone receptor-beta selective agonist | Nonalcoholic steatohepatitis | Data showed resmetirom lowered markers of cardiovascular risk and NASH fibrosis; patients achieved reductions in MRI-PDFF, CAP,VCTE, MRE, liver and spleen volume, ALT, AST, GGT, LDL-C, triglycerides, ApoB and lipoprotein (a) at week 52; safe and well-tolerated | 11/3/22 | Gastrointestinal |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor | Progressive familial intrahepatic cholestasis | Top-line results of randomized placebo-controlled study met primary and secondary endpoints with statistical significance; significant and rapid improvements in pruritus and serum bile acids; primary endpoint P=0.0098 and secondary endpoint P=0.0013 in PFIC2; secondary endpoint P<0.0001 in all type of pfic; common treatment emergent adverse event as diarrhea; mild and transient; no serious events< td> | 11/7/22 | Gastrointestinal |
| Vectivbio Holding AG, of Basel, Switzerland | Apraglutide | Long-acting synthetic GLP-2 analogue | Short bowel syndrome with intestinal failure | Completed enrollment; top-line results expected in the end of 2023 | 11/3/22 | Gastrointestinal |
| Aimmune Therapeutics UK Ltd., of London | Palforzia(peanut (Arachis hypogaea) allergen powder) | Oral immunotherapy | Peanut allergy | Study showed 73.5% and 68.4% of patients met the primary outcome at 12 months compared with 6.3% and 4.2% of placebo at 600-mg and 100-mg single doses; 61.2% compared with 2.1% of placebo-treated patient tolerated the highest exit double-blind, placebo-controlled exit food challenge (DBPCFC) dose level of 2000-mg; median highest tolerated dose of peanut increased 66-fold between baseline and exit DBPCFC; favorable safety profile; no serious events | 11/10/22 | Immune |
| Janssen Pharmaceuticals Cos., part of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Binds to p19 subunit of IL-23 and inhibits its interaction with IL-23 receptor | Active psoriatic arthritis | New post-hoc analysis from Discover program (Discover-1 and Discover-2) showed early skin and enthesitis responses predicted longer-term clinical response, including disease remission, at week 52; another post-hoc analysis of Discover-2, a diverse population of bio-naïve, Tremfya-treated patients sustained several PsA disease control endpoints through 2 years, regardless of baseline characteristics or dosing regimen, including minimal disease activity response, skin clearance (IGA 0) and resolution of dactylitis | 11/11/22 | Immune |
| Mesoblast Ltd., of New York | Remestemcel-L | Allogeneic stromal cell | Children with steroid-refractory acute graft-vs.-host disease | Top-line data from long term survival studies showed durable survival through 4 years of follow-up; overall survival was 63% at 1 year, 51% at 2 years, and 49% at 4 years; median survival of 2 to 3 years | 11/22/22 | Immune |
| Mesoblast Ltd., of New York | Remestemcel-L | Allogeneic stromal cell | Children with steroid-refractory acute graft-vs.-host disease | Top-line data from long-term survival studies showed durable survival through 4 years of follow-up; overall survival was 63% at 1 year, 51% at 2 years and 49% at 4 years; median survival of 2 to 3 years | 11/22/22 | Immune |
| Remegen Ltd., of Yantai, China | RC-18 (telitacicept) | TACI-Fc fusion protein | Myasthenia gravis | Results showed a mean reduction of 7.7 points and 9.6 points at 160-mg and 240-mg doses, respectively; good safety | 11/1/22 | Immune |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Psoriatic arthritis | Data from 52-week data of Be Optimal study showed 54.5% of patients continuously treated with bimekizumab, 53% of patients who switched from placebo to bimekizumab at week 16, and 50% of patients in reference arm (adalimumab) achieved ACR50; 60.8% of patients continuously treated with bimekizumab, 65% of patients who switched from placebo to bimekizumab at week 16, and 48.5% of patients in adalimumab arm achieved PASI 100; 55% of patients continuously treated with bimekizumab, 53.7% of patients who switched from placebo to bimekizumab at week 16, and 52.9% of patients in adalimumab arm achieved minimal disease activity | 11/10/22 | Immune |
| X4 Pharmaceuticals Inc., of Boston | Mavorixafor | CXCR4 antagonist | WHIM syndrome | Top-line results showed pivotal 4WHIM study met primary endpoint, achieving clinical and statistical superiority vs. placebo on TATANC, or the length of time that participants’ absolute neutrophil counts (ANC) remained above a clinically meaningful threshold of 500 cells per microliter, over 24-hour periods at 4 time points throughout 52-week trial; mean TATANC was 15.04 hours in the treatment group vs. 2.75 hours for placebo (p<0.0001); 4 52 also met key secondary endpoint, with statistical superiority over placebo when measuring tatalc, or the length of time that participants’ absolute lymphocyte counts (alc) remained above clinically meaningful threshold 1,000 cells per microliter, 24-hour periods at points throughout trial; increases in both tatanc and tatalc maintained vs. baseline across weeks; generally well-tolerated< td> | 11/29/22 | Immune |
| Atea Pharmaceuticals Inc., of Boston | Bemnifosbuvir | Purine nucleos(t)ide prodrug | COVID-19 | First patient dosed in Sunrise-3 study; planning to enroll 1500 patients | 11/29/22 | Infection |
| Daiichi Sankyo Co. Ltd., of Tokyo, Japan | DS-5670 | mRNA vaccine | COVID-19 | Study achieved primary endpoint in Japanese healthy adults and elderly subjects who completed the primary series (two doses) of mRNA vaccines; no safety concerns | 11/15/22 | Infection |
| Ferring Pharmaceuticals Inc., of Saint-Prex, Switzerland | RBX-2660 | Microbiota-based live biotherapeutic | Clostridium difficile infection | Data showed statistically significant treatment success; reduced CDI recurrence at 8 weeks compared to placebo (70.6% vs. 57.5%); mild to moderate adverse events; no treatment-related serious adverse events; well-tolerated | 11/7/22 | Infection |
| GSK plc, of London | Gepotidacin | Triazaacenaphthylene antibiotic | Uncomplicated urinary tract infections | Stopped enrollment based on recommendation of independent data monitoring committee; review did not identify any safety concerns; study met the primary efficacy endpoint of combined clinical and microbiological resolution | 11/3/22 | Infection |
| Novartis AG., of | Ganaplacide/lumefantrine- | Solid dispersion formulation | Acute uncomplicated malaria due to Plasmodium falciparum | Planning to start phase III in 2023 | 11/23/22 | Infection |
| Novartis AG., of Basel, Switzerland | Ganaplacide (lumefantrine-SDF) | Solid dispersion formulation; combination therapy | Acute uncomplicated malaria due to Plasmodium falciparum | Planning to start phase III in 2023 | 11/23/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | BA.1 vaccine candidate (NVX-CoV2515) | Protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2 | COVID-19 | Top-line results of phase III Boosting study met the primary strain-change endpoint; vaccine induced a broad immune response against original prototype, BA.1, and BA.5 strains; induced robust IgG responses to both BA.1 and the matched prototype strain; well-tolerated; well-established safety profile; majority of reactions being mild or moderate | 11/8/22 | Infection |
| Pfizer Inc., of New York | RSVpreF | Bivalent RSV prefusion F vaccine candidate | Respiratory syncytial virus | Top-line data in pre-planned interim efficacy analysis from pregnant participants met success criterion for one of two primary endpoints; observed efficacy for severe medically attended lower respiratory tract illness was 81.8%; substantial efficacy of 69.4% for infants over the 6-month follow-up period | 11/1/22 | Infection |
| Rigel Pharmaceuticals Inc., of South San Francisco | Fostamatinib | Oral spleen tyrosine kinase inhibitor | Hospitalized COVID-19 | Trial did not meet statistical significance (p=0.0603) in the primary efficacy endpoint of the number of days on oxygen through day 29; numerically favored prespecified secondary endpoints for fostamatinib over placebo; reduced patient all-cause mortality by 50% (p=0.4521); mean change from baseline in clinical status score to day 15 using the 8-point ordinal scale was -2.4 for fostamatinib and -1.9 for placebo (p=0.0428); median number of days to first sustained hospital discharge by day 29 was 6 days for fostamatinib and 7 days for placebo (p=0.2371); proportion of patients alive and oxygen free on day 29 was 85.1% for fostamatinib and 73.4% for placebo (p=0.0653); number of days in the ICU was 2.2 for fostamatinib and 3.3 for placebo (p=0.1883); comparable and consistent safety profile | 11/1/22 | Infection |
| Biosplice Therapeutics Inc., of San Diego | Lorecivivint | Wnt pathway modulator | Osteoarthritis of the knee | During first year of OA-07 long-term extension study, data showed clinically significant improvement in medial joint space width relative to placebo, with an effect size of 0.13 mm and 0.20 mm in overall KL2 and early stage KL2 patients, respectively; data also showed signifcant improvement in WOMAC Pain and trends in Pain NRS (daily pain score) as well as WOMAC function in all subjects | 11/11/22 | Inflammatory |
| Horizon Therapeutics plc, of Dublin | Krystexxa (pegloticase) | Pegylated uric acid specific enzyme | Uncontrolled gout | Mirror randomized controlled study in combination with methotrexate achieved serum uric acid (sUA) level less than 6 mg/dL for at least 80% of of the time during month 12 treatment vs 30.8% for those randomized to receive achieved Krystexxa with placebo; 53.8% in the Krystexxa with methotrexate group had complete resolution of at least 1 tophus, no new tophus and no single tophus showing progression at week 52 compared with 31% in the Krystexxa with placebo group; monosodium urate volume decreased 93.8% vs. 95.7% at week 52; statistically significant decrease in arterial inflammation after treatment [standard uptake value mean (p=0.0003) and SUV max (p=0.0090)] across all arteries studied | 11/8/22 | Inflammatory |
| UCB SA, of Brussels | Bimekizumab | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Axial Spondyloarthritis | 52-week data from Be Mobile 1 and Be Mobile 2 studies showed 60.9% of patients with active non-radiographic axial spondyloarthritis (nr-axSpA) and 58.4% of ankylosing spondylitis (AS) patients continuously treated with bimekizumab achieved ASAS40, with consistent outcomes across both TNFi-naïve and TNFi-inadequate responder populations; 1.6 % of nr-axSpA patients and 57.1 % of AS patients continuously treated with bimekizumab achieved low disease activity (ASDAS<2.1); at week 52; inactive disease or clinical remission (asdas<1.3) was achieved by 25.2% of nr-axspa patients and 23.4% as continuously treated with bimekizumab< td> | 11/10/22 | Musculoskeletal |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous MSC-NTF cells | Amyotrophic lateral sclerosis | Primary endpoint of ALSFRS-R improvement in slope post-treatment vs. pre-treatment was not met; post-hoc analysis showed Nurown had a higher rate of clinical response and less function lost across 28 weeks compared to placebo; statistically significant benefit in all subgroups with ALSFRS-R baseline total score of at least 26 to 35 (p?0.050) | 11/7/22 | Musculoskeletal |
| Axsome Therapeutics Inc., of New York | AXS-05 (dextromethorphan-bupropion) | Oral NMDA receptor antagonist with multimodal activity | Alzheimer’s disease | Study met the primary and key secondary endpoints, substantially and statistically significantly delaying the time to relapse of agitation symptoms as compared to placebo (p=0.014); 3.6-fold lower risk of relapse compared to placebo; relapse prevention results showed 7.5% of AXS-05 patients vs. 25.9% of placebo patients (p=0.018); statistically significant improvement on the Cohen Mansfield Agitation Inventory at week 1 (p<0.001); 2 4 5 11 mean reductions from baseline of points at week (p<0.001), and 20.6 (p<0.001) ; improved quality life (p="0.013)" 8< td> | 11/28/22 | Neurology/Psychiatric |
| Biovie Inc., of Santa Monica, Calif. | NE-3107 | Orally bioavailable small-molecule adrenal steroid hormone and insulin sensitizer; NFKB activation inhibitor; inhibitor of inflammatory ERK signaling | Alzheimer’s disease | Enrolled more than 316 patients; Company opted to continue enrolling up to 400 patients; top-line results expected in 2023 Q3 | 11/29/22 | Neurology/Psychiatric |
| Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 (dexmedetomidine) | Orally dissolving film formulation of selective alpha-2 adrenergic receptor agonist | Agitation associated with bipolar I or II disorder or schizophrenia | First 13 patients dosed in part 1 of pivotal Serenity trial testing at-home use | 11/30/22 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo, and Biogen Inc., of Cambridge, Mass. | Lecanemab (BAN-2401) | Anti-amyloid beta protofibril antibody | Mild cognitive impairment due to Alzheimer’s disease and mild AD with confirmed presence of amyloid pathology in the brain | Data from confirmatory Clarity AD study in 1,795 people with early AD showed mean change of Clinical Dementia Rating Sum of Boxes (CDR-SB) from baseline at 18 months of 1.21 and 1.66 for lecanemab and placebo groups, respectively; treatment resulted in highly statistically significant results, reducing clinical decline on the global cognitive and functional scale, compared with placebo at 18 months by -0.45 (p=0.00005), representing a 27% slowing of decline; starting as early as 6 months (p<0.01), 3 and increasing in absolute difference over time across all points every months, treatment showed highly statistically significant changes cdr-sb from baseline vs. placebo (all p-values less than 0.01); key secondary endpoints results compared with (p<0.001)< td> | 11/29/22 | Neurology/Psychiatric |
| Eli Lilly and Co., of Indianapolis | Donanemab | Monoclonal antibody targeting N3pG beta amyloid plaques | Alzheimer’s disease | Donanemab reduced brain amyloid plaque levels compared to baseline by 65.2% at 6 months compared with 17% for Aduhelm (aducanumab-avwa); 37.9% of donanemab-treated patients achieved brain amyloid plaque levels of <24.1 6 centiloids at months compared to 1.6% of patient taking aduhelm< td> | 11/30/22 | Neurology/Psychiatric |
| Genentech, a unit of Roche Holding AG, of Basel, Switzerland | Gantenerumab | Monoclonal antibody targeting beta-amyloid | Mild cognitive impairment due to Alzheimer's disease | Study did not meet their primary endpoints of slowing clinical decline; well-tolerated; slowing of clinical decline in GRADUATE I and GRADUATE II of -0.31 (p0.0954) and -0.19 (p=0.2998), respectively, from baseline score on the CDR-SB; relative reduction in clinical decline of 8% in GRADUATE I and 6% in GRADUATE II compared with placebo | 11/14/22 | Neurology/Psychiatric |
| Janssen Pharmaceutical Cos., of Beerse, Belgium, unit of Johnson & Johnson | Spravato (esketamine nasal spray) | NMDA antagonist | Treatment-resistant major depressive disorder | Escape-TRD study met its primary and secondary endpoint; significantly more participants achieved remission in the esketamine NS arm compared to the quetiapine XR arm (27.1% vs. 17.6% respectively; p=0.003); achieved remission at week 8 with no relapse at week 32 21.7% vs. 14.1% respectively; p=0.008; remission rates continued to increase in both arms after the primary endpoint at week 8 with a significantly greater proportion of patients in remission at week 32 in the esketamine NS arm vs the quetiapine XR arm (55% vs 37%; p<0.001)< td> | 11/23/22 | Neurology/Psychiatric |
| Janssen Pharmaceutical Cos., of Beerse, Belgium, unit of Johnson & Johnson | Spravato (esketamine nasal spray) | NMDA antagonist | Treatment-resistant major depressive disorder | Escape-TRD study met its primary and secondary endpoints; significantly more participants achieved remission in the esketamine NS arm compared to the quetiapine XR arm (27.1% vs. 17.6%; p=0.003); achieved remission at week 8 with no relapse at week 32 (21.7% vs. 14.1%, respectively; p=0.008); remission rates continued to increase in both arms after the primary endpoint at week 8 with a significantly greater proportion of patients in remission at week 32 in the esketamine NS arm vs. the quetiapine XR arm (55% vs. 37%; p<0.001)< td> | 11/23/22 | Neurology/Psychiatric |
| Medincell SA, of Jacou, France | F-14 (mdc-CWM) | Sustained-release formulation of celecoxib | Pain and inflammation after total knee replacement surgery | Initiated phase III study; expected to enroll first patient in November 2022 | 11/7/22 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor | Chorea associated with Huntington disease | Kinect-HD study met its primary endpoint of change in chorea severity using the total maximal chorea score; statistically significant secondary endpoints of CGI-C/PGI-C response status; placebo-adjusted lean-squares mean change (LSM) reduction of 3.2 units (p<0.0001); reduction in the secondary endpoints of 42.9% vs. 13.2% (p< 0.001), for cgi-c, 52.7% 26.4% (p<0.01), pgi-c; did not reach statistical significance neuroqol< td> | 11/3/22 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 | Nasal powder formulation of dihydroergotamine mesylate | Acute migraine | Top-line study of STS-101 was not statistically superior to placebo at 2 hours post-administration on the co-primary endpoints of freedom from pain and most bothersome symptom; proportion of subjects with pain relief at 2 hours post-dose (p=0.0017) and at all timepoints; proportion of subjects requiring rescue medication within 24 hours and 48 hours post-treatment (p<0.0001 and p="0.0001," respectively); proportion of subjects with 24-hour sustained freedom from pain (p="0.0479);" favorable safety tolerability< td> | 11/14/22 | Neurology/Psychiatric |
| UCB SA, of Brussels, Belgium | Fintepla (fenfluramine) | Serotonin-releasing agent | Lennox-Gastaut Syndrome | Long-term open-label extension study showed sustained reduction in the frequency of motor seizures; median percentage change in monthly frequency of seizures associated with a drop from pre-randomized baseline was -28.6% over the entire OLE (P<0.0001) 15 and -50.5% at month (p<0.0001); median percentage change in the frequency of all motor seizures was 45.9% (p="0.0038);" tonic -35.8% over entire ole (p<0.0001) < td> | 11/23/22 | Neurology/Psychiatric |
| UCB SA, of Brussels, Belgium | Fintepla (fenfluramine) | Serotonin-releasing agent | Lennox-Gastaut syndrome | Long-term open-label extension (OLE) study showed sustained reduction in the frequency of motor seizures; median percentage change in monthly frequency of seizures associated with a drop from pre-randomized baseline was -28.6% over the entire OLE (p<0.0001) 15 and -50.5% at month (p<0.0001); median percentage change in the frequency of all motor seizures was 45.9% (p="0.0038);" tonic -35.8% over entire ole (p<0.0001) < td> | 11/23/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol transdermal gel | Fragile X syndrome | Results published in the Journal of Neurodevelopmental Disorders showed well-tolerated; statistical significance for the primary endpoint was not achieved in the full cohort; significant improvement was demonstrated in patients with ?90% methylation of FMR1 (nominal p=0.020); achieved statistically significant improvements in Caregiver Global Impression-Change in SA and Isolation, Irritable and Disruptive Behaviors, and Social Interactions (nominal p-values: p=0.038, p=0.028 and p=0.002, respectively) | 11/28/22 | Neurology/Psychiatric |
| Belite Bio Inc., of San Diego | Tinlarebant (LBS-008) | Reduces and maintains levels of serum retinol binding protein 4 | Geographic atrophy associated with dry age-related macular degeneration | Finalized study design for planned phase III study; enrollment expected to begin in the first half of 2023 | 11/18/22 | Ocular |
| Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab-trbw) | Monoclonal antibody targeting insulin-like growth factor-1 receptor | Thyroid eye disease | Completed enrollment in Japan | 11/16/22 | Ocular |
| Lianbio Co. Ltd., of Shanghai | TP-03 | Lotilaner ophthalmic solution, 0.25% | Demodex blepharitis | First patient dosed; top-line results expected in the fourth quarter of 2023 | 11/1/22 | Ocular |
| Neurotech Pharmaceuticals Inc., of Cumberland, R.I. | NT-501 | Implant; encapsulated cell-based delivery of CNTF | Macular telangiectasia type 2 | Top-line data showed statistical significance for the prespecified primary endpoint of rate of change in ellipsoid zone (EZ) area loss from baseline through 24 months; 56.4% rate of reduction in protocol A (p<0.0001) and 29.2% rate of reduction in protocol b (p="0.021);" safe well-tolerated< td> | 11/2/22 | Ocular |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Aflibercept 8 mg | VEGF inhibitor | Diabetic macular edema and wet age-related macular degeneration | Data showed no retinal fluid in the center subfield (71% and 67%) compared to 59% for Eylea (aflibercept injection, 2 mg) in a prespecified analysis of PULSAR study; median time to a fluid-free subfield was 4 weeks for aflibercept 8 mg compared to 8 weeks for Eylea; mean reduction of 14 mm2 and 9 mm2 in DME patients in the total area of fluorescein leakage from baseline compared to 9 mm2 for Eylea; reductions in fluorescein leakage | 11/4/22 | Ocular |
| Unity Biotechnology Inc., of South San Francisco | UBX-1325 | Senolytic Bcl-xL inhibitor | Diabetic macular edema | Behold study met key safety and efficacy endpoints at 24 weeks; increase of +6.2 ETDRS letters; improvement of +7.6 ETDRS letters compared to sham-treated subjects from baseline (p=0.0084); favorable safety and tolerability profile; no cases of intraocular inflammation, retinal artery occlusion, endophthalmitis, or vasculitis; mean change in CST of -5.4 microns from baseline compared to an increase (worsening) of +34.6 microns in sham-treated patients for a total difference of 40.0 microns (p=0.1244) | 11/1/22 | Ocular |
| 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 showed no statistically significant impact on eradication outcomes; favorable safety and tolerability across all subjects | 11/4/22 | Other/Miscallaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Melanocortin-4 receptor agonist | Bardet-Biedl syndrome | Results published in The Lancet Diabetes and Endocrinology showed study met its primary endpoint and all key secondary endpoints; statistically significant reductions in weight and hunger at 52 weeks on therapy; primary endpoint was achieved by 32.3% p=0.0006 in patients ?12 years old; 15 patients ?18 years achieved a mean (SD) percent reduction in BMI of -9.1%; 14 patients <18 2 12 14 years achieved a mean (sd) change in bmi z score of ?0.8; patients (85.7%) ?0.2-point reduction z; ?12 who reported hunger scores -30.5% maximal score; hyperpigmentation skin as adverse events; serious events< td> | 11/8/22 | Other/Miscallaneous |
| Anaptysbio Inc., of San Diego, and GSK plc, of London | Jemperli (dostarlimab) | Anti-PD-1 inhibitor | Endometrial cancer | Results met its primary endpoint of investigator-assessed progression-free survival (PFS); overall survival immature; favorable trend was observed in the overall population | 12/2/22 | Cancer |
| Astellas Pharma Inc., of Tokyo | Zolbetuximab | Monoclonal antibody targeting Claudin 18.2 (CLDN18.2) | HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | Top-line results showed study achieved primary and secondary endpoint in combination with capecitabine and oxaliplatin | 12/15/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Anti-PD-L1 antibody | Stage IV non-small-cell lung cancer | Pearl study in patients whose tumor cells express high levels of PD-L1, or in subgroup of patients at low risk of early mortality, did not achieve statistical significance of improving overall survival (OS) vs. platinum-based chemotherapy as a monotherapy; improvement in OS with monotherapy, which was clinically meaningful in subset of patients with PD-L1 tumor expression greater than 50%, a secondary endpoint | 12/19/22 | Cancer |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | Small orally bioavailable drug that binds to A3 adenosine receptor | Hepatocellular carcinoma | Pivotal phase III registration study is open for enrollment for the most advanced liver cancer patients | 12/28/22 | Cancer |
| Celcuity Inc., of Minneapolis | Gedatolisib | Pan-PI3K/mTOR inhibitor | HR+/HER2- advanced breast cancer | First patient dosed | 12/7/22 | Cancer |
| Chimerix Inc., of Durham, N.C. | ONC-201 | Small-molecule dopamine receptor D2 antagonist and caseinolytic protease (ClpP) agonist | Glioma | Data from Action study showed median overall survival 26.3 months (p<0.0001); median overall survival of 16.2 months compared to 8.1 for placebo (p="0.05)" in the recurrent setting< td> | 12/8/22 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate targeting HER2 | HER2 positive unresectable and/or metastatic breast cancer | Study achieved 36% reduction in risk of death vs. T-DM1 (p=0.0037); median OS was not yet reached; median duration of follow-up of 28.4 months for Enhertu and 26.5 months for T-DM1; estimated 77.4% of patients were alive at 2 years compared to 69.9% of patients treated with T-DM1; median PFS was 28.8 months compared to 6.8 months for T-DM1; confirmed objective response rate was 78.5% vs. 21.1% | 12/7/22 | Cancer |
| Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK4/6 inhibitor; non-chemotherapy oral tablet | Hormone receptor-positive, HER2-negative, node-positive early breast cancer | Results published in the Lancet Oncology showed median follow-up of 3.5 years; absolute increase in invasive disease-free survival (IDFS) and distance relapse-free survival (DRFS) continued to deepen in magnitude at four years, to 6.4% and 5.9%, respectively; overall survival data immature; no new safety findings; invasive disease was reduced by 33.6% (nominal p<0.0001); four-year idfs rate was 85.8% for patients treated with verzenio plus et compared to 79.4% alone; reduced the risk of developing metastatic disease by 34.1% (p<0.0001)< td> | 12/7/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | XL-092 (zanzalintinib) | Tyrosine kinase inhibitor | Advanced non-clear cell renal cell carcinoma | Initiated the Stellar-30 study | 12/22/22 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabozantinib (Cabometyx) | Inhibitor of tyrosine kinases | Metastatic non-small-cell lung cancer | Contact-01 study did not meet its primary endpoint of overall survival at the final analysis in combination with atezolizumab vs. docetaxel in patients; no new safety signals | 12/8/22 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Trodelvy (sacituzumab govitecan) | Antibody-drug conjugate targeting Trop-2 | HR+/HER2- metastatic breast cancer | Post-hoc analysis from TROPiCS-02 study improved progression-free survival (PFS), overall survival (OS) and objective response rate (ORR) vs. physicians’ choice of chemotherapy across Trop-2 expression levels; no new safety signals; For patients with a Trop-2 expression H-score <100, 4 pfs="5.3" months vs. months; median os="14.6" 11.3 for trodelvy v.s physicians’ choice of chemotherapy, respectively< td> | 12/6/22 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex (daratumumab) | Monoclonal antibody targeting CD38 | Newly diagnosed, transplant-ineligible patients with multiple myeloma | A post-hoc analysis of the Maia study testing Darzalex plus lenalidomide and dexamethasone (Rd) showed patients under 75 taking the triple regimen had a 48% reduction in the likelihood of progression-free survival compared to Rd alone; overall response rate was 95.2% and 81.7%, respectively; in patients under 70, PFS risk reduction was 65% with ORR of 93.6% and 80.5%, respectively | 12/12/22 | Cancer |
| Mirati Therapeutics Inc., of San Diego | Sitravatinib | Oral receptor tyrosine kinase inhibitor | Non-squamous non-small-cell lung cancer | Final analysis expected to be reached in mid-2023 | 12/2/22 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide crystal nanoparticles | Locally advanced head and neck squamous cell carcinoma | First patient randomized in Nanoray-312 | 12/27/22 | Cancer |
| Novartis AG, of Basel, Switzerland | Pluvicto (lutetium (177Lu) vipivotide tetraxetan) | PSMA ligand labeled to Lu-177 | PSMA?positive metastatic castration-resistant prostate cancer | Study met its primary endpoint; statistically significant and clinically meaningful improvement in radiographic progression-free survival in patients; no unexpected safety findings | 12/5/22 | Cancer |
| Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Independent data monitoring committee recommended to continue without modifications | 12/8/22 | Cancer |
| Urogen Pharma Ltd., of Princeton, N.J. | Jelmyto (mitomycin) | Pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel | Lower grade upper tract urothelial cancer | Results showed no patient had progressed to high-grade disease; median duration of response among the 16 patients was 28.9 months; 13 patients remained in complete response | 12/2/22 | 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 | Data showed median duration of response of 24.4 months in 15 patients; 65% complete response at 3 months; well-tolerated; mild to moderate adverse events reported | 12/2/22 | Cancer |
| Urogen Pharma Ltd., of Princeton, N.J. | UGN-102 (mitomycin) | DNA synthesis inhibitor; anticancer alkylating agent | Low-grade, intermediate-risk, non-muscle invasive bladder cancer | 220-patient Envision study fully enrolled | 12/19/22 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Nilemdo (bempedoic acid) | Inhibits ATP citrate lyase | Reduction of risk of serious cardiovascular events | Study met primary endpoint; statistically significant relative risk reduction in major adverse CV events at 180 mg/day compared to placebo | 12/8/22 | Cardiovascular |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream | Nonsteroidal topical phosphodiesterase-4 inhibitor | Mild to moderate atopic dermatitis | In the Integument-2 study, 28.9% of patients treated with roflumilast achieved Investigator Global Assessment Success, defined as a score of clear or almost clear plus a 2-grade improvement from baseline at week 4, compared to 12% of patients treated with vehicle (p<0.0001); 42% of patients treated with roflumilast achieved a 75% improvement in eczema area and severity index compared to 19.7% vehicle (p<0.0001)< td> | 12/12/22 | Dermatologic |
| Journey Medical Corp., of Scottsdale, Ariz. | DFD-29 (minocycline modified-release capsules) | Tetracycline antibiotic | Papulopustular rosacea | Pharmacokinetic comparability data show safety on par with Solodyn (minocycline modified-release tablets); enrollment of pivotal program reached 96%, with top-line data anticipated in first half of 2023 | 12/20/22 | Dermatologic |
| Krystal Biotech Inc., of Pittsburgh | Beremagene geperpavec | Gene therapy | Dystrophic epidermolysis bullosa | Results published in the New England Journal of Medicine achieved primary and secondary endpoints; complete wound healing at 6 months occurred in 67.4% compared to 21.6% for placebo in 31 patients (p=0.002); complete wound healing at 3 months occurred in 70.6% vs. 19.7% (p=0.0005); well-tolerated; no drug-related serious adverse events or discontinuations | 12/14/22 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib cream | Pan-JAK inhibitor | Moderate to severe chronic hand eczema (CHE) | Study achieved primary and all key secondary endpoints; statistically significant improvement in CHE after 16 weeks of treatment compared to vehicle; well-tolerate; reduction of itch and pain scores of ?4 points measured by the Hand Eczema Symptom Diary (HESD) from baseline to Week 16; 75% improvement from baseline and at least 90% improvement from baseline on the Hand Eczema Severity Index | 12/6/22 | Dermatologic |
| UCB SA, of Brussels | Bimzelx (bimekizumab) | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Moderate to severe hidradenitis suppurativa | Top-line results showed Be Heard I and Be Heard II studies met their primary and key secondary endpoints with statistical significance and consistent clinical relevance at week 16; showed depth of response with statistically significant improvements at week 16 over placebo in the percentage of patients achieving HiSCR75; no new observed safety signals | 12/9/22 | Dermatologic |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | SGLT2 inhibitor | Diabetes type 2 | Study met its primary endpoint; HbA1c was reduced by 0.84% compared with placebo at week 26 (p=0.012); reduced fasting plasma glucose (p=0.0035) | 12/7/22 | Endocrine/Metabolic |
| Bridgebio Pharma Inc., of Palo Alto, Calif. | Encaleret | Oral, small molecule that selectively antagonizes the calcium sensing receptor targeting ADH1 | Autosomal dominant hypocalcemia type 1 | Study initiated | 12/22/22 | Endocrine/Metabolic |
| Intrabio Inc., of Oxford, U.K. | IB-1001/N-Acetyl-L-Leucine | Neuroprotective agents | Niemann-Pick disease type C | Completed recruitment; enrolled 130% of target patients; results expected in the end of 2023 Q2 | 12/8/22 | Endocrine/Metabolic |
| Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Oral, selective phosphoinositide 3-kinase delta inhibitor | Activated PI3K-delta syndrome | Results published in American Society of Hematology journal showed improvement over placebo was significant in the co-primary endpoints which evaluated reduction in lymph node size and increase in naïve B cells; adjusted mean change between leniolisib and placebo for lymph node size was -0.25 (p=0.0006); well-tolerated | 12/6/22 | Endocrine/Metabolic |
| Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Liver-directed THR-beta agonist | Nonalcoholic steatohepatitis | Top-line results showed pivotal Maestro-NASH biopsy trial achieved both liver histological improvement endpoints; 26% response rate for the 80-mg dose and 30% response rate for 100-mg dose for NASH resolution endpoint vs. 10% for placebo (p<0.0001 2 24 for both); on fibrosis improvement endpoint, responses of 24% and 26% seen resmetirom dose groups, respectively, vs. 14% placebo (p="0002" p<0.0001, respectively); secondary ldl-c-lowering at weeks, groups showed response rates 12% 16% 80-mg 100-mg doses, 1% (p<0.0001 both)< td> | 12/19/22 | Gastrointestinal |
| Xortx Therapeutics Inc., of Calgary, Alberta | Xorlo | Oral oxypurinol formulation | Progressive kidney disease | Completed dosing in the OXR-OXY-101 pharmacokinetics bridging study; consolidated top-line results from each part of study anticipated in January 2023 | 12/19/22 | Genitourinary/Sexual Function |
| Novartis AG, of Basel, Switzerland | Iptacopan | Factor B inhibitor of the alternative complement pathway | Paroxysmal nocturnal hemoglobinuria | Top-line results from Appoint-PNH study met primary endpoint; significant proportion of patients treated with iptacopan (200-mg twice daily) achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks; consistent safety profile | 12/8/22 | Hematologic |
| Pfizer Inc., of New York | Fidanacogene elaparvovec | Gene therapy | Moderately severe to severe hemophilia B | Top-line results from Benegene-2 study in adult males met primary endpoint of noninferiority and superiority in annualized bleeding rate (ABR)of total bleeds post infusion vs. prophylaxis regimen with factor IX; superiority with mean ABR for all bleeds of 1.3 for 12 months from week 12 to month 15 compared to ABR of 4.43 during lead-in pretreatment period of at least 6 months, resulting in 71% reduction in ABR (p<0.0001) 15 24 after a single dose of 5e11 vg kg; secondary endpoints demonstrated 78% reduction in treated abr (p="0.0001)" and 92% annualized infusion rate (p<0.0001); mean fix activity was 27% at months by 1-stage synthasil assay 25% months; steady-state fix:c higher than prespecified threshold 5% (p<0.0001)< td> | 12/29/22 | Hematologic |
| Idorsia Ltd., of Allschwil, Switzerland | Cenerimod | S1P receptor modulator | Moderate to severe systemic lupus erythematosus | First patient entered screening for Opus program, which consists of 2 phase III trial testing drug on top of background therapy; primary endpoint is change from baseline to month 12 in modified Systemic Lupus Erythematosus Disease Activity Index 2000 score | 12/15/22 | Immune |
| UCB SA, of Brussels | Bimzelx (bimekizumab) | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Psoriatic arthritis | Results published in The Lancet from Be Optimal and Be Complete studies met their primary and all ranked secondary endpoints; bimekizumab achieved improvements in joint symptoms at week 16 compared with placebo; clinical response observed in both biologic-naïve and TNFi-IR populations (p<0.0001 for each-acr50); achieved high levels of skin clearance (p<0.0001 each-pasi90); consistent safety profile< td> | 12/7/22 | Immune |
| Bavarian Nordic A/S, of Copenhagen | MVA-BN RSV | Vaccine incorporating five distinct RSV antigens to stimulate a broad immune response against RSV subtypes A and B | Lower-respiratory tract disease caused by respiratory syncytial virus | Patient enrollment complete | 12/21/22 | Infection |
| Bioved Pharmaceuticals Inc., of San Jose, Calif. | Artovid-22 | Natural plant based oral product | COVID-19 | Results published in the peer-reviewed journal Phytotherapy Research, showed statistically significant reduction in duration of illness and severity scores of the symptoms | 12/15/22 | Infection |
| Contrafect Corp., of Yonkers, N.Y. | Exebacase | Lysin | Staph aureus bacteremia | All patients in the Disrupt study were independently adjudicated and the database was locked | 12/19/22 | Infection |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Zokinvy (lonafarnib) | Prenylation inhibitor | Hepatitis delta virus infection | Top-line week 48 results achieved statistical significance over placebo in the composite primary endpoint; oral therapy and combination therapy with ritonavir+peginterferon alfa showed a composite response of 10.1% (p=0.0044) and 19.2% (p <0.0001), 8 30 35 107 respectively, compared to placebo (1.9%); statistically significant improved rates of alt normalization 24.7% (p="0.003)" and 34.4% (p<0.0001), (7.7%); secondary histological endpoint (53%, p="0.0139)" with statistical significance in the combination arm vs. patients (27%) receiving placebo; response was demonstrated (33%, all-oral full endpoints results expected be reported mid-2023< td> | 12/8/22 | Infection |
| Junshi Biosciences Co. Ltd., of Shanghai | VV-116 (JT-001) | Remdesivir derivative; SARS coronavirus replication inhibitor; antiviral | COVID-19 | Data published in The New England Journal of Medicine comparing drug to Paxlovid (nirmatrelvir/ritonavir, Pfizer Inc.) in symptomatic patients with mild to moderate infection who are at high risk for progression to severe COVID-19, including death, indicated primary endpoint realized noninferiority endpoint; VV-116 group had shorter time to sustained clinical recovery with less safety concerns vs. Paxlovid | 12/29/22 | Infection |
| Takeda Pharmaceuticals Co. Ltd., of Osaka, Japan | Maribavir | Antiviral targeting UL97 protein kinase | Cytomegalovirus infection | Aurora trial comparing maribavir to valganciclovir in hematopoietic stem cell transplant (HSCT) recipients, demonstrated clinically meaningful efficacy in confirmed CMV viremia clearance, but did not meet primary endpoint of noninferiority (maribavir 69.6% vs. valganciclovir 77.4%); at week 16, key secondary endpoint, 8 weeks after end of treatment, showed numerically higher proportion of patients treated with maribavir (52.7%) maintained CMV viremia clearance and symptom control achieved at week 8 compared to valganciclovir (48.5%), and comparable maintenance was consistent at all post-treatment evaluations at week 12 (maribavir 59.3% vs. valganciclovir 57.3%) and week 20 (43.2% vs. 42.3%) | 12/19/22 | Infection |
| Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine | Chikungunya virus infection | Study met primary endpoint; 99% seroresponse rate 12 months after single-dose vaccination; antibody levels remained stable from month 6 to month 12; no safety concerns | 12/5/22 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-2001 | Inactivated viral vaccine with CpG 1018 adjuvant | COVID-19 prophylaxis | Booster dose showed well-tolerated; increased neutralizing antibody response; favorable safety profile | 12/30/22 | Infection |
| Vaxxinity Inc., of Dallas | UB-612 | COVID-19 vaccine | COVID-19 prophylaxis | Study achieved primary and secondary endpoint; UB-612 shown to be statistically non-inferior to Comirnaty (tozinameran, Pfizer Inc. and Biontech SE) with 1.04 geometric mean ratio (GMR) non-inferiority against Wuhan variant (p=0.6147), 1.11 GMR against omicron BA.5 (p=0.2171); UB-612 also shown to be statistically superior to Vaxzevria (ChAdOx1-S, Astrazeneca plc): 1.92-fold higher geometric mean titers against Wuhan with UB-612 (p<0.0001), 2.85-fold higher against omicron ba.5 (p<0.0001); statistically superior to bbibp-corv (beijing institute of biological products co. ltd.): 5.77-fold geometric mean titers wuhan with ub-612 (p<0.0001), 5.93-fold well-tolertaed; no serious adverse events< td> | 12/2/22 | Infection |
| Biosplice Therapeutics Inc., of San Diego | Lorecivivint | CLK2/DYRK1A inhibitor | Osteoarthritis | Results from ongoing long-term extension study, OA-07, continue to support potential efficacy of multiple injections in delaying structural progression and providing symptomatic benefit | 12/15/22 | Inflammatory |
| Ocugen Inc., of Malvern, Pa. | Neocart | Autologous chondrocyte-derived neocartilage | Repair of full-thickness lesions of the knee cartilage | U.S. FDA agreed to proposed control and overall design of phase III study, a randomized, controlled trial to demonstrate the superiority over standard of care | 12/16/22 | Musculoskeletal |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Cyclobenzaprine HCl sublingual tablets | Fibromyalgia | First 50% of participants randomized in Resilient study; interim analysis by data monitoring committee expected in second quarter 2023 | 12/19/22 | Musculoskeletal |
| Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Prodrug of tramiprosate which inhibits the formation of toxic amyloid beta 42 oligomers | Alzheimer’s disease | Achieved the enrollment target of 325 patients | 12/6/22 | Neurology/Psychiatric |
| Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 (dexmedetomidine) sublingual film | Orally dissolving film formulation of alpha-2 adrenergic receptor agonist | Agitation in Alzheimer’s disease | First patient dosed in pivotal Tranquility trial; primary endpoint is change in Positive and Negative Syndrome Scale-Excitatory Component total score from baseline measured at 2 hours after initial dose and subsequent doses | 12/19/22 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Small molecule drug | Alzheimer’s disease | Completed dosing in an open-label study | 12/6/22 | Neurology/Psychiatric |
| Idorsia Ltd., of Allschwil, Switzerland | Quviviq (daridorexant ) | Dual orexin receptor antagonist | Insomnia | Data published in CNS Drugs showed taking daridorexant for up to 12 months in the extension study was well-tolerated with no signs of tolerance or physical dependence; no evidence of withdrawal or rebound insomnia upon treatment discontinuation was observed; efficacy was similar to initial 12-week study | 12/12/22 | Neurology/Psychiatric |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Rexulti (brexpiprazole) | Partial agonist of serotonin 5-HT1A and dopamine D2 receptors | Agitation in Alzheimer’s dementia | Data showed significantly (p<0.05) 2 12 improved symptoms of agitation in patients at or 3-mg; statistically-significant improvement vs. placebo from baseline to week the cmai total (lsmd, -5.32; p="0.0026)" and cgi-s score as related -0.27;> | 12/1/22 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor channel blocker; chemical molecule | Major depressive disorder | Reliance I study showed a MADRS reduction of 15.1 points at day 28 vs. 12.9 points for the placebo arm; clinically meaningful difference of 2.2 points on the MADRS; statistically significant difference in the response rate; response rate of 27.2% on placebo vs 39.8% in the REL-1017 arm (p<0.05); 28 post-hoc analysis of study showed a madrs reduction 16.7 points at day vs 12.6 for the placebo arm (n="88)," 4.1 point difference with p<0.02; favorable tolerability and safety profile; enrolling patients in reliance ii study< td> | 12/7/22 | Neurology/Psychiatric |
| Satsuma Pharmaceuticals Inc., of South San Francisco | STS-101 (dihydroergotamine nasal powder) | 5-HT 1d receptor agonist | Acute treatment of migraine | Data from Summit study showed high proportions of subjects’ treated migraine attacks had symptoms predictive of poor response to treatment; STS-101 demonstrated numerical but not statistically significant differences vs. placebo on co-primary endpoints (% of subjects free from pain and % of subjects free from most-bothersome-symptom [MBS] at 2 hours post-dose); showed robust and sustained effects (p<0.001) 2 24 48 on key study endpoints, freedom from pain and mbs, at all post-dose timepoints after hours (3, 4, 6, 12, hours)< td> | 12/20/22 | Neurology/Psychiatric |
| SK Life Science Inc., of Paramus, N.J. | Xcopri (cenobamate) | Positive allosteric modulator of the ?-aminobutyric acid (GABAA) ion channel | Partial-onset (focal) seizures | Post-hoc analysis showed reduction of concomitant anti-seizure medications; maintained ?50% and 100% seizure reduction response rates | 12/2/22 | Neurology/Psychiatric |
| Taurx Pharmaceuticals Ltd., of Aberdeen, U.K. | Hydromethylthionine mesylate (HMTM) | Tau aggregation inhibitor | Alzheimer’s disease | In the Lucidity study, at 18 months, patients with mild cognitive impairment had an improvement of 2 points over the pretreatment baseline at 6 months (p=0.0002), 12 months (p=0.0391) and 18 months (p=0.0473) on the 13-item Alzheimer’s Disease Assessment Scale; in patients with mild to moderate disease, there was a 2.5-point cognitive decline in the first 9 months that continued to 18 months | 12/1/22 | Neurology/Psychiatric |
| Vanda Pharmaceuticals Inc., of Washington | Fanapt (iloperidone tablets) | Atypical antipsychotic | Acute manic and mixed episodes associated with bipolar I disorder | Results from study VP-VYV-683-3201 in about 400 volunteers with history of bipolar I suffering from current episode of mania showed Fanapt-treated patients had larger improvement vs. placebo on primary endpoint assessed by Young Mania Rating Scale at week 4 (p=0.000008); statistically significant benefit over placebo observed as early as week 2; outcomes such as Clinician Global Impression of Severity and Clinician Global Impression of Change also achieved statistical significance (p=0.0005 and p=0.0002, respectively) | 12/19/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Pharmaceutically manufactured cannabidiol | Fragile X syndrome | Revised target for announcing top-line results from 2023 H2 to the 2024 H1 | 12/21/22 | Neurology/Psychiatric |
| Viridian Therapeutics Inc., of Waltham, Mass. | VRDN-001 | Differentiated monoclonal antibody targeting insulin-like growth factor-1 receptor | Active thyroid eye disease | First patient enrolled in Thrive study | 12/21/22 | Ocular |
| Verona Pharma plc, of London | Ensifentrine (nebulized) | Inhibits PDE3/4 | Chronic obstructive pulmonary disease | Enhance-1 trial met primary and key secondary endpoints demonstrating significant improvements in lung function, symptoms and quality of life measures; reduced rate and risk of COPD exacerbations by 36% over 24 weeks | 12/20/22 | Respiratory |