January | February | March | April | May | June | July | August | September | October | November | December |
| Company | Product | Description | Indication | Phase II status | Date | Therapeutic area |
| Affimed NV, of Heidelberg, Germany | AFM-13 | Innate cell engager specifically targets CD30 | Relapsed or refractory CD30-positive peripheral T-cell lymphoma | Completed enrollment in Redirect study; top-line data expected in second half of 2022 | 1/6/22 | Cancer |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland | Derazantinib | FGFR kinase inhibitor | Metastatic intrahepatic cholangiocarcinoma | Interim results from Fides-01 study (n=23) with a median follow-up period of 5.2 months; disease control rate was 74%; 2 patients with confirmed objective response and 15 patients with stable disease; tumor shrinkage in most of patients; median progression-free survival was 7.3 months; study continues to enroll; top-line results for cohort 2 in the first half of 2022 | 1/24/22 | Cancer |
| Biosight Ltd., of Airport City, Israel | Aspacytarabine (BST-236) | Anti-metabolite composed of cytarabine covalently bound to asparagine; prodrug of cytarabine | Acute myeloid leukemia | Final results showed well-tolerated at 4.5 g/m2/d; complete remission rates were 37% in all evaluable patients (n=65); 27% in patients with prior HMA ± venetoclax therapy; 44% in de novo AML patients; 27% in secondary AML patients; 35% in patients ? 75 years old and 32% in patients with adverse ELN risk scores; rapid complete hematological recovery in patients achieving complete bone marrow response with a median time of 25 days for complete neutrophil recovery, 26 days (range 18-40) for complete platelet recovery; median duration of response and median overall survival follow-up is ongoing | 1/6/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Relatlimab and Opdivo (nivolumab) | LAG-3-blocking antibody and PD-1-blocking antibody | Advanced melanoma | Study results published in The New England Journal of Medicine showed improved progression-free survival (PFS) compared to nivolumab monotherapy; median PFS was 10.12 months vs. 4.63 months in the monotherapy arm; patients with a LAG-3 expression of 1% or greater had median PFS with the combination of 12.58 months vs. 4.76 months; LAG-3 expression of less than 1% had median PFS with the combination of 4.83 months vs. 2.79 months; 3 deaths in combination arm and 2 deaths in the monotherapy arm | 1/6/22 | Cancer |
| Evaxion Biotech A/S, of Copenhagen | EVX-01 | Patient-specific cancer neoepitope immunotherapy | Melanoma | Results published in OncoImmunology showed improved antitumor activity in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); safe in 5 patients | 1/26/22 | Cancer |
| Gritstone Bio Inc., of Emeryville, Calif. | Granite | Personalized immunotherapy of 20 tumor-specific neoantigens | Metastatic, microsatellite-stable colorectal cancer after fluoropyrimidine, oxaliplatin and bevacizumab induction therapy | Started the phase II/III Granite-CRC-1L study; the phase II portion will measure changes in ctDNA over time; the phase III portion will measure progression-free survival | 1/13/22 | Cancer |
| Hookipa Pharma Inc., of New York | HB-201 and HB-202 | Live-attenuated lymphocytic choriomeningitis virus expressing E6/E7 tumor antigens of human papillomavirus | HPV16+ squamous cell head and neck cancers | First patient dosed in combination with pembrolizumab | 1/18/22 | Cancer |
| I-Mab Biopharma Co. Ltd., of Shanghai | Efineptakin alfa (TJ-107) | Long-acting recombinant human interleukin-7 | Advanced solid tumors | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co.) | 1/12/22 | Cancer |
| I-Mab Biopharma Ltd., of Shanghai | Lemzoparlimab | CD47 antibody | Advanced solid tumors | First patient dosed in combination with toripalimab | 1/18/22 | Cancer |
| IMV Inc., of Dartmouth, Nova Scotia | Maveropepimut-S (DPX-Survivac) | T cell-activating immunotherapy; survivin-based peptide | Relapsed/refractory diffuse large B-cell lymphoma | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co.) | 1/12/22 | Cancer |
| Mina Therapeutics Ltd., of London | MTL-CEBPA | Small activating RNA oligonucleotide | Advanced hepatocellular carcinoma | First of 150 patients treated in the Outreach-2 trial evaluating MTL-CEBPA plus sorafenib compared to sorafenib alone; primary endpoint is progression-free survival; data read out expected in early 2024 | 1/25/22 | Cancer |
| Mirati Therapeutics Inc., of San Diego | MRTX-849/adagrasib | Oral small-molecule inhibitor of KRASG12C | Pancreatic ductal adenocarcinoma and other gastrointestinal tumors | Results from Krystal-1 study at 600 mg BID showed significant clinical activity in 27 evaluable patients; objective response rate was 41% and disease control rate was 100%; median progression-free survival in patients with pancreatic cancer was 6.6 months; in patients with other GI tumors, the mPFS was 7.9 months; well-tolerated, with manageable safety profile | 1/21/22 | Cancer |
| Nordic Nanovector ASA, of Oslo, Norway | Betalutin (177Lu lilotomab satetraxetan) | Lutetium-177 radioimmunoconjugate of the CD37-directed murine monoclonal antibody lilotomab | Follicular lymphoma | 106 patients enrolled in Paradigme study; preliminary 3-month data readout expected in the second half of 2022 | 1/7/22 | Cancer |
| Obi Pharma Inc., of Taipei, Taiwan | OBI-999 | Antibody-drug conjugate targeting Globo H | Advanced solid tumors | First patient enrolled | 1/24/22 | Cancer |
| Obi Pharma Inc., of Taipei, Taiwan | OBI-3424 | Prodrug targeting the enzyme aldo-keto reductase 1C3 | Advanced solid tumors | First patient enrolled | 1/24/22 | Cancer |
| OS Therapies LLC, of Cambridge, Md. | OST-HER2 | Live-attenuated Listeria monocytogenes strain genetically engineered to encode a chimeric human HER2/neu fusion protein | Osteosarcoma | Data safety and monitoring committee approved open enrollment for AOST-2121 phase IIb study; trial to enroll 36 to 42 patients | 1/27/22 | Cancer |
| Panbela Therapeutics Inc., of Minneapolis | SBP-101 | Polyamine analogue | Metastatic pancreatic ductal adenocarcinoma | Initiated phase II Aspire study in combination with gemcitabine and nab-paclitaxel | 1/26/22 | Cancer |
| Pieris Pharmaceuticals Inc., of Boston | Cinrebafusp alfa (PRS-343) | Bispecific HER2-targeting monoclonal antibody-Anticalin fusion protein specific for the immune co-stimulatory TNF family receptor 4-1BB (CD137) | Gastric cancer | First patient dosed | 1/13/22 | Cancer |
| Radiomedix Inc., of Houston, and Orano Med of Plano, U.S. | Alphamedix | Peptide receptor radionuclide therapy | Neuroendocrine tumors | Initiated phase II study to evaluate the safety and effectiveness; enroll 34 patients | 1/3/22 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | Devimistat (CPI-613) | Targets the mitochondrial tricarboxylic acid | Biliary tract cancer | Successful enrollment rate in combination with gemcitabine and cisplatin | 1/18/22 | Cancer |
| Revolution Medicines Inc., of Redwood City, Calif. | RMC-4630 | Small-molecule inhibitor of SHP2 | Advanced non-small-cell lung cancer | First patient dosed in combination with Lumakras (sotorasib, Amgen Inc.) | 1/11/22 | Cancer |
| Sapience Therapeutics Inc., of Harrison, N.Y. | ST-101 | Peptide antagonist of C/EBP? | Glioblastoma | First patient dosed | 1/6/22 | Cancer |
| Tempus Labs Inc., of Chicago, and London-based Glaxosmithkline plc | Zejula (niraparib) | Poly (ADP-ribose) polymerase inhibitor | Advanced or metastatic solid tumors and a germline or somatic PALB2 mutation | Opened enrollment in the Pavo study that will identify patients with PALB2 somatic and germline mutations through Tempus’ genomic sequencing platform; primary endpoint is overall response rate by independent central review; secondary endpoints include duration of response, progression-free survival, ORR by investigator review and others | 1/13/22 | Cancer |
| Tyme Technologies Inc., of Bedminster, N.J. | SM-88 | Tyrosine derivative that interrupts the metabolic processes of cancer cells | Metastatic pancreatic cancer | Study terminated for futility on primary endpoint of overall survival vs. control | 1/26/22 | Cancer |
| Vigeo Therapeutics Inc., of Cambridge, Mass. | VT-1021 | Dual-CD47/CD36 modulating agent | Recurrent glioblastoma | Advanced to phase II/III registrational study | 1/20/22 | Cancer |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-40 | Fatty acid synthase inhibitor | Moderate to severe acne | Treated first patient in the study comparing 3 dose levels of ASC-40 to placebo; primary endpoints include percentage change of total lesion count at week 12 compared to baseline and percent of subjects whose Investigator's Global Assessment grades are decreased by ?2 grades at week 12 compared to baseline | 1/13/22 | Dermatologic |
| Aslan Pharmaceuticals Ltd., of Singapore | ASLAN-004 (eblasakimab) | Antibody targeting the IL-13 receptor | Moderate-to-severe atopic dermatitis | Screened first patient; expected to enroll 300 patients; top-line data in first half of 2023 | 1/20/22 | Dermatologic |
| Connect Biopharma Inc., of San Diego | CBP-201 | IL-4R? antibody | Moderate to severe atopic dermatitis | Study met the primary and key secondary efficacy endpoints; favorable safety data with low incidences of injection site reactions, conjunctivitis and herpes infections; significant improvements in skin clearance, disease severity and itch compared to placebo | 1/24/22 | Dermatologic |
| Connect Biopharmaceuticals Ltd., of Taicang, China | CBP-201 | Antibody designed to target interleukin-4 receptor alpha | Moderate to severe atopic dermatitis | Data showed study met multiple key secondary endpoints; favorable safety data; greater reduction of EASI score from baseline and a lower placebo response in patients with higher disease severity at baseline based on EASI score (n=69); planning to initiate phase III study in second half of 2022 | 1/5/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | KX-826/pyrilutamide | Androgen receptor antagonist | Acne vulgaris | First patient dosed | 1/24/22 | Dermatologic |
| Mediwound Ltd., of Yavne, Israel | Escharex | Bioactive therapy; concentrate of proteolytic enzymes enriched in bromelain for topical use | Debridement of venous leg ulcers | Top-line data showed study met its primary endpoint; statistically significant higher incidence of complete debridement compared to gel vehicle( p=0.004); well-tolerated and overall safety was comparable between the arms; no serious adverse events | 1/24/22 | Dermatologic |
| Nimbus Therapeutics LLC, of Cambridge, U.S. | NDI-034858 | Oral allosteric TYK2 inhibitor | Moderate to severe plaque psoriasis | Initiated phase IIb study | 1/6/22 | Dermatologic |
| Union Therapeutics A/S, of Hellerup, Denmark | Orismilast | Selective next-generation PDE4 inhibitor | Moderate to severe psoriasis | First patient enrolled in phase Iib dose-ranging IASOS study | 1/4/22 | Dermatologic |
| First Wave Biopharma Inc., of Boca Raton, Fla. | Adrulipase | Recombinant lipase | Exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis | Plans to start a phase IIb pilot study with a new formulation in the second half of 2022 | 1/13/22 | Endocrine/Metabolic |
| Imcyse SA, of Liege, Belgium | IMCY-0098 | Proinsulin-derived Imotope | Type 1 diabetes | Interim analysis of biomarker data from 17 patients in the Impact study showed an expansion of a population of CD4+ T cells expressing Granzyme B and anti-inflammatory markers, no observation of treatment-induced FOXP3+ regulatory T cells and a smaller population of antigen-specific CD4+ T cells with pathogenic signature compared to placebo; half of the patients have been dosed; recruitment scheduled to be complete in mid-2022 | 1/13/22 | Endocrine/Metabolic |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Severe obesity and hyperphagia potentially caused by a genetic variant that impairs the MC4R pathway | First of approximately 500 patients treated in the Daybreak study that includes a 16-week open-label run-in period followed by a double-blind, placebo-controlled withdrawal study; primary endpoint is the proportion of patients treated with setmelanotide who achieve a clinically meaningful weight reduction at the end of treatment compared to patients treated with placebo | 1/13/22 | Endocrine/Metabolic |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | Oral small-molecule drug; highly specific and selective agonist at A3 adenosine receptor | Nonalcoholic steatohepatitis | First patient enrolled in phase IIIb study | 1/31/22 | Gastrointestinal |
| Orphomed Inc., of San Francisco | ORP-101 | New chemical entity designed to target peripheral partial mu agonist and full kappa receptor antagonist | Irritable bowel syndrome with diarrhea | Results from 12-week response adaptive randomization study showed ORP-101 was safe and well tolerated in all patients; 100 mg was superior to placebo (p=0.008); significantly better than placebo on secondary and exploratory endpoints; strong trend toward efficacy with a treatment difference of 8.5 % points; robust effect with an approximately 18 % point difference in composite responder rate between ORP-101 100-mg and placebo, p=0.04 in IBS-D post-cholecystectomy patients (n=85); favorable safety profile | 1/4/22 | Gastrointestinal |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | Humanized monoclonal antibody targeting the CCR5 receptor | Nonalcoholic steatohepatitis | Study met primary and secondary endpoint at 350-mg weekly dose; no significant differences in treatment-emergent adverse events between leronlimab and placebo groups | 1/5/22 | Gastrointestinal |
| Angion Biomedica Corp., of Uniondale, N.Y. | ANG-3070 | Oral tyrosine kinase receptor inhibitor | Primary proteinuric kidney diseases | First patient dosed | 1/4/22 | Genitourinary/Sexual Function |
| Provention Bio Inc., of Red Bank, N.J. | PRV-3279 | Humanized bispecific DART molecule targeting the B-cell surface proteins CD32B and CD79B | Systemic lupus erythematosus | Initiated phase IIa Prevail-2 study | 1/20/22 | Immune |
| Remegen Ltd., of Yantai, China | RC-18 (telitacicept) | TACI-Fc fusion protein | Primary Sjögren's syndrome | The 160 mg dose improved ESSDAI scores compared to placebo in the full-set analysis; Per-protocol-set analysis showed statistical improvements for both the 160 mg and and 240 mg doses compared to placebo | 1/24/22 | Immune |
| Ascletis Pharma Inc., of China | ASC-42 | Selective, potent farnesoid X receptor agonist; Anti-viral agent target transcription of HBV cccDNA | Chronic hepatitis B | First patient dosed | 1/10/22 | Infection |
| Calcimedica Inc., of La Jolla | Auxora | Small-molecule CRAC channel inhibitor | Critical COVID-19 pneumonia | Initiated Cardea plus phase Iib study in U.S. and Canada; to evaluate dosing, safety and efficacy of Auxora in combination with both tocilizumab and corticosteroids | 1/20/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Chengdu, China | SCB-2019 (CpG 1018/Alum) | COVID-19 S-Trimer subunit vaccine; vaccine comprising recombinant SARS-CoV-2 spike glycoprotein | COVID-19 prophylaxis | First participants dosed; homologous booster dose following primary vaccination in SPECTRA study | 1/5/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Chengdu, China | SCB-2019 (CpG 1018/Alum) | COVID-19 S-Trimer subunit vaccine; vaccine comprising recombinant SARS-CoV-2 spike glycoprotein | COVID-19 prophylaxis | Data from Spectra study published in The Lancet showed the vaccine achieved the primary efficacy endpoint and secondary efficacy endpoints; 100% efficacy against severe COVID-19 and hospitalization caused by any strain of SARS-CoV-2; favorable safety profile with no significant differences vs. placebo | 1/20/22 | Infection |
| Deinove SA, of Paris | DNV-3837 | Prodrug of DNV-3681; narrow-spectrum, hybrid oxazolidinone-quinolone synthetic antibiotic | Clostridioides difficile infection | Independent data safety monitoring board completed its review of the safety data; benefit-risk balance of antibiotic therapy with DNV-3837 was in favor of continuing trial | 1/6/22 | Infection |
| First Wave Biopharma Inc., of Boca Raton, Fla. | FW-COV | Oral, tablet formulation of micronized niclosamide | COVID-19-related gastrointestinal infections | Completed enrollment for part 2 of reservoir study; 150 patients enrolled; no drug related serious adverse events; top-line data expected in first half of 2022 | 1/6/22 | Infection |
| Galecto Inc., of Boston | GB-0139 | Galectin-3 inhibitor | COVID-19 infection requiring oxygen but not mechanical ventilation | Study met its primary endpoint of safety; no observed treatment-related adverse events; confirmed target engagement with a statistically significant reduction (p<0.01) in serum galectin-3 levels; decreased markers of inflammation linked to the cytokine storm, inflammation-associated microthrombosis and short- or long-term fibrosis; significantly greater rate decline oxygen requirement vs. standard care alone showed other signs reduced organ damage; multiple improved immune-mediated viral responses as well lymphocyte exhaustion level profibrotic macrophage; 21% reduction mortality was observed subgroup patients at high clinical risk< td> | 1/27/22 | Infection |
| Golden Biotechnology Corp., of Taipei, Taiwan | Hocena (antroquinonol) | Antioxidant compound extracted from Antrodia camphorata | COVID-19 | Top-line results showed recovery ratio was 97.9% at the day 14 visit; no death or respiratory failure; recovery ratio of 100% at day 28; median duration of ICU stay on antroquinonol was 9.5 days shorter than that in the placebo group; median duration of hospitalization was 4 days; median time to virological clearance was 14 days; good tolerability and safety results | 1/7/22 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | Hospitalized COVID-19 | Reached target enrollment in phase II/III ACTIV-5/BET-B study; around 400 patients enrolled; top-line data expected in first quarter or early in the second quarter of 2022 | 1/5/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273/Spikevax | Omicron-specific booster candidate (mRNA-1273.529) | COVID-19 prophylaxis | First subject dosed; trial to assess the potential susceptibility and neutralization of the omicron variant | 1/26/22 | Infection |
| Novartis AG, of Basel, Switzerland, and Molecular Partners AG, of Zurich-Schlieren, Switzerland | Ensovibep/MP-0420 | DARPin therapeutic candidate designed to block the receptor binding domains of SARS-CoV-2 spike protein | COVID-19 | Top-line data of part A of Empathy study met primary endpoint of viral load reduction over 8 days; clinically meaningful benefit over placebo for 2 secondary endpoints; 78% reduction in risk of events across ensovibep arms compared to placebo; safe and well-tolerated at all doses (75 mg, 225 mg and 600 mg); no deaths in treatment arm | 1/10/22 | Infection |
| Ocugen Inc., of Malvern, Pa., and Bharat Biotech International Ltd., of Hyderabad, India | BBV-152 (Covaxin) | Whole-virion inactivated COVID-19 vaccine manufactured using a vero cell platform | COVID-19 | Results published in Medrxiv showed significant increase in neutralizing titers (>10-fold across alpha, beta, delta and delta plus variants) in booster dose 6 months after second dose; no serious adverse events | 1/8/22 | Infection |
| Quantum Leap Healthcare Collaborative, of San Francisco | Pulmozyme (dornase alfa) | Highly purified solution of recombinant human deoxyribonuclease I | COVID-19 | I-SPY COVID trial terminated; interim analysis from critically ill patients not achieved statistically significant improvement in efficacy | 1/11/22 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Opaganib | Sphingosine kinase-2 selective inhibitor | Severe COVID-19 pneumonia | In patients who were positive for SARS-CoV-2 by PCR at screening, opaganib produced a median time to viral RNA clearance of 10 days, while clearance median was not reached for the placebo group at the end of the 14-day treatment (HR=1.34, p=0.43) | 1/13/22 | Infection |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | Small-molecule selective BET inhibitor | COVID-19 | Initated enrollment and dosing of patient | 1/18/22 | Infection |
| Revelation Biosciences Inc., of San Diego | REVTx-99 | Intranasal drop formulation | H3N2 Influenza infection | 15 patients enrolled and dosed | 1/20/22 | Infection |
| SAB Biotherapeutics Inc., of Sioux Falls, S.D. | SAB-185 | Fully human, specifically targeted, broadly neutralizing polyclonal antibody | COVID-19 | Study met criteria for advancement to phase III study with day 3 viral load data from the prespecified interim analysis; nasopharyngeal viral qRT-PCR reductions of 1.48 log10 and 0.67 log10 vs. placebo; viral load reductions of 0.5 log10 vs. placebo for both the low and high doses; safe and well-tolerated; enrolled more than 700 patients of the 1,200 total targeted for the phase III | 1/24/22 | Infection |
| Shionogi and Co. Ltd., of Osaka, Japan | S-217622 | 3CL protease inhibitor | COVID-19 | Results from phase IIa part study from Japanese adults showed rapid virus reduction effect compared to placebo; rapid decrease in proportion of patients with viral titer positively in the S-217622 group compared to placebo; no high-grade or serious adverse events | 1/31/22 | Infection |
| Sorrento Therapeutics Inc., of San Diego | Covi-Drops | Single low dose intranasal instillation | COVID-19 | Enrolled 72 adult outpatients in U.S.; viral load reduction from baseline as primary endpoint; no significant safety events reported during study; interim analysis from U.K. phase II study (n=175) found to be well-tolerated | 1/20/22 | Infection |
| Todos Medical Ltd., of Rehovot, Israel, and NLC Pharma Ltd., of Tel Aviv, Israel | Tollovir | 3CL protease inhibitor and anti-cytokine therapeutic candidate | Hospitalized severe/critical COVID-19 | Data lock for interim data analysis of phase II study; data will be released on Jan. 27, 2022 | 1/24/22 | Infection |
| Todos Medical Ltd., of Rehovot, Israel, and NLC Pharma Ltd., of Tel Aviv, Israel | Tollovir | 3CL protease inhibitor and anti-cytokine therapeutic candidate | Hospitalized severe/critical COVID-19 | Interim data showed study met primary and several key secondary clinical endpoints; time to clinical improvement reached with average reduction of 2.7 days in Tollovir vs. placebo group; no deaths in Tollovir vs. 22% in placebo | 1/27/22 | Infection |
| Vasomune Therapeutics Inc., of Toronto, and Anges Inc., of Ibaraki, Japan | AV-001 | Pegylated peptide targeting Tie2 receptor | COVID-19 | First patient dosed | 1/18/22 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass., and Brii Biosciences Ltd., of Durham, N.C. | VBI-2601 (BRII-179) | Virus-like particle vaccine | Chronic hepatitis B virus infection | First patient dosed; assessing the safety and efficacy as an add-on therapy to standard-of-care treatment; initial data expected in first half of 2023 | 1/5/22 | Infection |
| AI Therapeutics Inc., of Guilford, Conn. | AIT-101 | PIKfyve kinase inhibitor | Amyotrophic lateral sclerosis | Started the 12-week study that will enroll 12 patients to evaluate the safety, tolerability, pharmacokinetics and effect of AIT-101 on biomarkers of target engagement, toxic protein aggregates and neurodegeneration; Revised ALS Functional Rating Scale score, vital capacity and ALS Cognitive Behavioral Screen score will also be assessed | 1/13/22 | Musculoskeletal |
| Khondrion BV, of Nijmegen, the Netherlands | Sonlicromanol | Redox modulator with anti-inflammatory properties | MELAS spectrum disorders | Last patient dosed in Khenergyze study | 1/10/22 | Musculoskeletal |
| Lynk Pharmaceuticals Co. Ltd., of Hangzhou, China | LNK-01001 | JAK1 selective inhibitor | Ankylosing spondylitis | First patient dosed | 1/19/22 | Musculoskeletal |
| Prilenia Therapeutics BV, of Naarden, the Netherlands | Pridopidine | Oral, highly selective and potent Sigma-1 receptor agonist | Amyotrophic lateral sclerosis | Completed patient enrollment in HEALEY ALS platform trial | 1/5/22 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass., and Roche Holding AG, of Basel, Switzerland | SRP-9001 (delandistrogene moxeparvovec) | Gene therapy expressing microdystrophin | Duchenne muscular dystrophy | Top-line data from 41 patients showed SRP-9001 scored a statistically significant 2 points higher on the mean North Star Ambulatory Assessment at 48 weeks compared to propensity-score weighted external controls (p=0.0009); mean NSAA scores from these part 2 participants improved 1.3 points from baseline for the SRP-9001-treated group and the NSAA scores in the external control group (n=103) declined 0.7 points from baseline; no new safety signals | 1/10/22 | Musculoskeletal |
| Annexon Inc., of South San Francisco | ANX-005 | Monoclonal antibody target C1Q | Huntington’s disease | Interim data showed well-tolerated; 5 patients discontinued and 3 patients discontinued due to a drug-related adverse event; full target engagement of C1q in both serum and CSF; patients maintained clinical function relative to baseline in cUHDRS after 6 months; 56% of patients showed improvement from baseline in cUHDRS and several subdomains of cUHDRS over 6 months of treatment; 75% of patients with excess baseline complement activity demonstrated a statistical improvement in cUHDRS over 6 months of treatment vs. 36% with low baseline complement activity | 1/4/22 | Neurology/Psychiatric |
| Athira Pharma Inc., of Seattle | ATH-1017 | HGFR activator | Parkinson's disease dementia and dementia with Lewy bodies | Started dosing of the 75-patient Shape study comparing 2 dose levels of ATH-1017 to placebo; primary endpoint is the composite Global Statistical Test, which combines the scores from the cognitive assessment and change in Event-Related-Potential P300 latency | 1/25/22 | Neurology/Psychiatric |
| Bionomics Ltd., of Adelaide, Australia | BNC-210 | Oral, selective negative allosteric modulator of the alpha7 nicotinic acetylcholine receptor | Social anxiety disorder | Initiated phase II study and results expected by the end of 2022 | 1/3/22 | Neurology/Psychiatric |
| Biovie Inc., of Santa Monica, Calif. | NE-3107 | Orally bioavailable small-molecule adrenal steroid hormone and insulin sensitizer; NFKB activation inhibitor; selective inhibitor of inflammatory ERK signaling | Parkinson’s disease | First patient treated | 1/20/22 | Neurology/Psychiatric |
| Cyclerion Therapeutics Inc., of Cambridge, Mass. | CY-6463 | CNS-penetrant sGC stimulator | Alzheimer’s disease with vascular pathology | Initiated patient dosing | 1/26/22 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | E-2814 | Anti-microtubule binding region tau antibody | Dominantly inherited Alzheimer's disease | Enrolled first subject in Tau Nexgen study | 1/18/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-810 | Prodrug of the bioactive lipid palmitoylethanolamide | Diabetic peripheral neuropathic pain | Achieved full enrollment in phase Iia study; top-line data expected in first half of 2022 | 1/18/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-810 | Prodrug of the bioactive lipid palmitoylethanolamide | Sciatica | Enrollment is ongoing; expected to complete enrollment in the first half of 2022 and top-line data in second half of 2022 | 1/18/22 | Neurology/Psychiatric |
| Intelgenx Corp., of Quebec | Montelukast Versafilm | Leukotriene receptor antagonist, as an oral film | Mild to moderate Alzheimer’s Disease | Resumed patient dosing | 1/20/22 | Neurology/Psychiatric |
| Longeveron Inc., of Miami | Lomecel-B | Allogeneic, bone marrow-derived medicinal signaling cell therapy | Alzheimer’s disease | Initiated phase Iia study; patient screening began | 1/5/22 | Neurology/Psychiatric |
| Onquality Pharmaceuticals LLC, of Seattle | OQL-011 | Ointment designed to locally activate VEGF downstream signaling pathways | Hand-foot skin reaction (HFSR)-related pain | Top-line data from part-1 NOVA-II met safety, efficacy and patient reported outcome; planning to initiate part-2 NOVA-II study in first quarter of 2022 | 1/4/22 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Tominersen | Antisense drug targeting the huntingtin protein | Huntington’s disease | Planning phase II study design in younger adult patients with lower disease burden | 1/18/22 | Neurology/Psychiatric |
| Scisparc Ltd., of Tel Aviv, Israel | SCI-110, THX-110 | Fixed-dose combination of dronabinol and palmitoylethanolamide | Alzheimer's disease | First patient enrolled in study to evaluate the safety, tolerability and efficacy | 1/6/22 | Neurology/Psychiatric |
| Yamo Pharmaceuticals LLC., of New York | L1-79 | Tyrosine hydroxylase inhibitor | Autism spectrum disorder | First patient treated | 1/27/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Small-molecule modulator of RASP | Dry eye disease | Top-line data showed reproxalap statistically lower than Xiidra (lifitegrast, Novartis AG) for the two endpoints as ocular discomfort (p=0.002) and ocular itching (p=0.01); no treatment-related discontinuations or moderate or serious adverse events related to drug; mild instillation site discomfort as common adverse event | 1/11/22 | Ocular |
| Gemini Therapeutics Inc., of Cambridge, Mass. | GEM-103 | Recombinant form of human complement factor H protein (CFH) | Age-related macular degeneration | Regatta study (n=62) showed GEM-103 found to be well-tolerated; reduced biomarkers of complement activation; maintain supraphysiological levels of CFH; consistent safety profile | 1/10/22 | Ocular |
| Glaukos Corp., of San Clemente, Calif. | GLK-302 | Cream-based pilocarpine formulations | Presbyopia | First patient enrolled | 1/11/22 | Ocular |
| Oxurion NV, of Leuven, Belgium | THR-149 | Plasma kallikrein inhibitor | Diabetic macular edema | U.S. institutional review board approved protocol amendment to part B of Kalahari trial | 1/7/22 | Ocular |
| Proqr Therapeutics NV, of Leiden, the Netherlands | Sepofarsen | RNA therapy | CEP290-mediated Leber congenital amaurosis 10 due to the p.Cys998X mutation | Last patient completed their last visit (month 12) in phase II/III Illuminate trial; top-line data expected in first quarter of 2022 | 1/4/22 | Ocular |
| Reneuron Group plc., of Pencoed, U.K. | Human retinal progenitor cell therapy | Cell-based therapeutic | Retinitis pigmentosa | Initial study treated with 10 patients at 1 million cell administration; proposed for 9 patients with a higher level 2 million cell dose |
1/18/22 | Ocular |
| Stuart Therapeutics Inc., of Stuart, Fla. | ST-100 | Topical ophthalmic therapeutics; targeted tissue reparative technology restoring the helical collagen structures to their primary structural and cell signaling ligand binding site role | Dry eye disease | Top-line results showed ST-100 achieved a pre-approved primary endpoint of Schirmer's test responder rate; statistically significant difference between the percentage of patients achieving a 10mm increase or more in Schirmer's tear test scores at 28 days (p=0.0266); significant results in several symptoms and ocular surface staining scores as early as treatment day 7 in both the intent to treat population, as well as the treated patient sub-populations; ocular discomfort improvement was observed at treatment day 14 (p=0.0332);well tolerated; comfort scores for the eyedrops compare favorably with artificial tear product; no observed serious drug-related adverse events | 1/3/22 | Ocular |
| Glaukos Corp., of San Clemente, Calif. | GLK-302 | Cream-based pilocarpine formulations | Dry eye disease | First patient enrolled | 1/11/22 | Ocular |
| Oxurion NV, of Leuven, Belgium | THR-687 | Small-molecule pan-RGD integrin antagonist | Diabetic macular edema | Completed patient enrollment for part A phase II Integral study in treatment-naïve subjects | 1/7/22 | Ocular |
| Sciwind Biosciences Co. Ltd., of Hangzhou, China | XW-003 | Long-lasting glucagon-like peptide-1 peptide derivative | Obesity | Initiated patient dosing | 1/17/22 | Other/Miscellaneous |
| Sensorion SA, of Paris | SENS-401 | Small-molecule drug to preserve residual acoustic hearing with cochlear implant | Sudden sensorineural hearing loss | Preliminary data from 115 patient showed safe and well-tolerated; primary endpoint of 15 dB not met; significant improvement in pure tone audiometry in the affected ear from baseline compared to placebo at the end of the 4-week treatment period; better response compared to placebo at the 2 doses for hearing threshold > 80dB; secondary endpoints results expected in mid-march of 2022 | 1/17/22 | Other/Miscellaneous |
| Pieris Pharmaceutcals Inc., of Boston,and Astrazeneca plc, of Cambridge, U.K. | PRS-060/AZD-1402 | Inhaled IL-4 receptor alpha inhibitor | Moderate to severe asthma | Completed sponsor safety review of part 1a phase II study (n=31); planning to initiate part2a and part1b study | 1/3/22 | Respiratory |
| Verona Pharma plc, of London | Ensifentrine | Bronchodilator with anti-inflammatory activities | Chronic obstructive pulmonary disease | Completed patient enrollment in Enhance-1 study; completed screening, with 788 subjects randomized in Enhance-2 study and full enrollment expected at end of January 2022 | 1/5/22 | Respiratory |
| Horizon Therapeutics plc, of Dublin | HZN-825 | Lysophosphatidic acid receptor 1 antagonist | Idiopathic pulmonary fibrosis | First patient enrolled | 1/24/22 | Respiratory |
| Anebulo Pharmaceuticals Inc., of Austin, Texas | ANEB-001 | Human cannabinoid receptor type 1 competitive antagonist | Acute cannabinoid intoxication | First patient dosed; top-line results expected in first half of 2022 | 1/3/22 | Toxicity and Intoxication |
| Awakn Life Sciences Corp., Toronto | Ketamine | NMDA receptor antagonist | Alcohol use disorder | Study achieved primary and secondary endpoints when combined with KARE therapy; resulted in total abstinence in 162 of 180 days in following 6-month period; achieved an increase in abstinence from around 2% prior to the trial to 86% post trial; risk of relapse was 2.7 times less than the placebo plus alcohol education group; no serious adverse events | 1/11/22 | Toxicity and Intoxication |
| Opiant Pharmaceuticals Inc., of Santa Monica, Calif. | OPNT-002 (naltrexone hydrochloride, intranasal) | Opioid receptor antagonist | Alcohol use disorder | First patient dosed in study expected to enroll about 300 patients; results expected in 2023 | 1/20/22 | Toxicity and Intoxication |
| Ayala Pharmaceuticals Inc., of Rehovot, Israel | AL-102 | Selective, oral gamma-secretase inhibitor | Desmoid tumors | Completed patient enrollment in part A of Ringside studies; interim results expected in mid-2022 | 2/23/22 | Cancer |
| Celsion Corp., of Lawrenceville, N.J. | GEN-1 | Plasmid encoding human interleukin-12 | Advanced ovarian cancer | Data safety monitoring board recommended ovation 2 study at dose of 100 mg/m2; full enrollment expected by mid-2022; no dose-limiting toxicity | 2/17/22 | Cancer |
| Clarity Pharmaceuticals Ltd., of Sydney | 64Cu-SARTATE and 67Cu-SARTATE | Theranostics | Neuroblastoma | Completed cohort 1 study at 75 megabecquerels (MBq)/kg; no adverse event reported; increased dose level of 175MBq/kg | 2/1/22 | Cancer |
| Cyteir Therapeutics Inc., of Lexington, Mass. | CYT-0851 | Inhibits RAD51-mediated homologous recombination and the repair of double-strand DNA breaks | Hematologic malignancies and solid tumors | First patient enrolled in the phase II portion of the ongoing phase I/II study; antitumor activity will be measured in 6 cohorts (relapsed and/or refractory diffuse large B-cell lymphoma, follicular lymphoma, multiple myeloma, recurrent metastatic or locally advanced pancreatic cancer, progressive ovarian cancer and metastatic soft tissue sarcoma) | 2/8/22 | Cancer |
| Genmab A/S, of Copenhagen, Denmark, and Seagen Inc., of Bothell, Wash. | Tisotumab vedotin | Antibody-drug conjugate targeting tissue factor | Head and neck squamous cell carcinoma | Preliminary data from monotherapy study showed manageable safety profile; antitumor activity in patient population with the primary endpoint of confirmed objective response rate per investigator achieved by 16 % of patients; disease control rate of 58.1%; median PFS was 4.2 months; median follow-up was 10.0 months; median OS was 9.4 months | 2/24/22 | Cancer |
| Imcheck Therapeutics SAS, of Marseille, France | ICT-01 | Monoclonal antibody targeting BTN3A | Ovarian cancer or head and neck squamous cell carcinoma | First patient dosed in phase IIa Eviction monotherapy arm | 2/3/22 | Cancer |
| Inflarx NV, of Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Cutaneous squamous cell carcinoma | Started enrollment of the second dosing cohort testing vilobelimab in combination with Keytruda (pembrolizumab Merck& Co. Inc.) to determine the maximum tolerated or recommended dose, safety and antitumor activity of the combination; treatment with the combination for 36 days in the first dosing cohort of 3 patients produced no safety concerns | 2/16/22 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib | PI3K inhibitor | Glioblastoma | The first patient was enrolled in a phase II study investigating it in combination with metformin and a ketogenic diet | 2/28/22 | Cancer |
| Marker Therapeutics Inc., of Houston | MT-401 | Multitumor-associated antigen (MultiTAA)-specific T-cell product | Acute myeloid leukemia | Results from Safety study showed no dose-limiting toxicity; no cytokine release syndrome or neurotoxicity; no objective responses from the frank relapse patients; well-tolerated; eliminated measurable residual disease in 1 MRD+ patient and induced epitope spreading | 2/16/22 | Cancer |
| Modra Pharmaceuticals BV, of Amsterdam, the Netherlands | ModraDoc-006/r | Oral tablet formulation of docetaxel co-administered with ritonavir | Metastatic castration-resistant prostate cancer | Data showed similar efficacy and an improved tolerability profile compared to I.V. docetaxel; reduced neutropenia, neuropathy and alopecia; overall response rate of 44% vs. 39% for I.V. docetaxel; prostate-specific antigen responses were comparable at 50% vs. 57%, respectively | 2/17/22 | Cancer |
| Monopar Therapeutics Inc., of Wilmette, Ill. | Validive | Mucobuccal tablet formulation of clonidine | Chemoradiotherapy-induced severe oral mucositis in patients with oropharyngeal cancer | Trial actively enrolling at 43 sites in U.S. and EU; interim analysis anticipated in mid-2022 | 2/15/22 | Cancer |
| Neoimmunetech Inc., of Rockville, Md. | Efineptakin alfa (TJ-107/GX-I7/NT-I7) | Long-acting recombinant human interleukin-7 | Advanced high-risk skin cancers | First patient dosed in combination with Tecentriq (atezolizumab, Roche Holding AG) | 2/7/22 | Cancer |
| Nykode Therapeutics AS of Oslo, Norway | VB-10.16 | Cancer neoantigen vaccine | Advanced cervical cancer | Completed patient enrollment in combination with Tecentriq (atezolizumab, Roche Holding AG); interim efficacy and safety data expected in first half of 2022 | 2/11/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 (Versamune-HPV16) | Protein subunit vaccine | Advanced human papillomavirus-associated head and neck cancer | Results from Versatile-002 study in combination with pembrolizumab (Keytruda, Merck & Co. Inc.) achieved preliminary objective response benchmarks; progress to full enrollment of 54 patients | 2/2/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 (Versamune-HPV16) | Protein subunit vaccine | Advanced human papillomavirus-associated head and neck cancer | Investigator initiated trial to test whether PDS-0101 alone or in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) will increase HPV-specific anti-tumor responses before surgery | 2/15/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 (Versamune-HPV16) | Protein subunit vaccine | Recurrent or metastatic HPV16-positive head and neck cancer | Preliminary data in combination with Keytruda (pembrolizumab, Merck & Co.) showed well-tolerated; safe in 18 patients; without evidence of enhanced or significant toxicity | 2/24/22 | Cancer |
| Rakuten Medical Inc., of San Mateo, Calif. | ASP-1929 (cetuximab sarotalocan) | Antibody-dye conjugate targeting EGFR | Operable primary or recurrent head and neck squamous cell carcinoma or cutaneous squamous cell carcinoma | First of 22 patients enrolled in the study to assess the efficacy and safety of a single ASP-1929 photoimmunotherapy treatment administered prior to standard of care surgical tumor resection | 2/15/22 | Cancer |
| Seagen Inc., of Bothell, Wash. | Adcetris (brentuximab vedotin) | Antibody-drug conjugate comprising anti-CD30 monoclonal antibody attached by protease-cleavable linker to microtubule disrupting agent, monomethyl auristatin E | Hodgkin lymphoma | Data from Echelon-1 study demonstrated statistically significant improvement in overall survival (p=0.009) in combination with chemotherapy; 6 years median follow up, patients receiving Adcetris plus doxorubicin, vinblastine, and dacarbazine (A+AVD) had a 41% reduction in the risk of death compared with patients receiving doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD); no new safety signals | 2/3/22 | Cancer |
| Secura Bio Inc., of Summerlin, Nev. | Copiktra (duvelisib) | Oral inhibitor of phosphoinositide 3-kinase | Relapsed or refractory peripheral T-cell lymphoma | Completed enrollment in Primo study; enrolled 157 patients | 2/9/22 | Cancer |
| Sirnaomics Inc., of Gaithersburg, Md. | STP-705 | Nanoparticle-encapsulated formulation of siRNAs targeting TGFB1 and COX2 | Cutaneous basal cell carcinoma | Interim data from 3 cohorts with 15 total subjects showed dose response with complete response; improved or stable cosmetic result with no significant cutaneous skin reactions; favorable safety profile; no drug related adverse events or serious events | 2/23/22 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | TRC-102/methoxyamine | Small-molecule inhibitor of the DNA base excision repair pathway | Stage III non-squamous non-small-cell lung cancer | Initiated phase II study in combination with chemoradiation; planning to enroll 78 patients | 2/2/22 | Cancer |
| Transcenta Holding Ltd., of Suzhou, China | ST-001 | Claudin18.2 monoclonal antibody, combined with Cisplatin and Gemcitabine | Biliary tract cancer | First participant dosed | 2/28/22 | Cancer |
| Aobiome Therapeutics Inc., of Cambridge, Mass. | B-244 | Live topical biotherapeutics | Pruritus associated with atopic dermatitis | Results from 547 patients met all primary and secondary endpoints; reduced enrolled patients mean Worst Itch Numeric Rating Scale (WI-NRS) score by 34.3% placebo, p=0.0143 and p=0.0148 for high and low dose,respectively) from a baseline; achieved statistical significance of 29.3% and 27.7% of patients in the high and low dose groups of B-244 achieved EASI-75 success vs. 15.8% in placebo group (p=0.0035 and p=0.0086, respectively); 26.2% and 21.7% of patients in the high and low dose groups of B-244 achieved IGA success (?2-point improvement in IGA to clear or almost clear) vs. 12.3% in the placebo group (p=0.0015 and p=0.0228, respectively); well-tolerated with no SAE | 2/22/22 | Dermatologic |
| Evelo Biosciences Inc., of Cambridge, Mass. | EDP-1815 | Orally administered monoclonal microbial strain of Prevotella histicola | Mild and moderate psoriasis | Results showed statistically significant reduction in the release of cytokines compared to placebo: IL-6 (p=0.0003), IL-8 (p=0.0007) and TNF (p=0.0037); no observed distribution of EDP-1815 outside the gut | 2/7/22 | Dermatologic |
| Evelo Biosciences Inc., of Cambridge, Mass. | EDP-1815 | Non-live pharmaceutical preparation of a strain of Prevotella histicola | Atopic dermatitis | First patient dosed | 2/17/22 | Dermatologic |
| Evelo Biosciences Inc., of Cambridge, Mass. | EDP-1815 | Non-live pharmaceutical preparation of a strain of Prevotella histicola | Atopic dermatitis | Data from the post-treatment follow-up of its phase II trial included durable and deeper clinical responses; 18 out of 30 patients maintained a PASI-50 (50% reduction in Psoriasis Area and Severity Index score from baseline) or greater response at 24 weeks post-treatment; 9 of 20 patients experienced a deepening of response from PASI-50 to at least PASI-75 during post-treatment period; no flare or rebound observed following discontinuation of treatment (which are often seen with other therapies for psoriasis) | 2/28/22 | Dermatologic |
| Lynk Pharmaceuticals Co. Ltd., of Hangzhou, China | LNK-01001 | Selective JAK1 inhibitor | Atopic dermatitis | Dosed the study's first patient | 2/28/22 | Dermatologic |
| Promore Pharma AB, of Stockholm | Ensereptide (PXL-01) | Peptide-based drug | Skin scar | The 24-patient study will evaluate local tolerance, the application process and the preliminary effect on scar prevention after experimentally induced wounds; biopsies to be collected and evaluated during fall of 2022; study results expected in winter of 2022/2023 | 2/16/22 | Dermatologic |
| Timber Pharmaceuticals Inc., of Basking Ridge, N.J. | TMB-001 | Topical formulation of the vitamin A derivative isotretinoin | Moderate to severe congenital ichthyosis | Data showed median time to response to treatment with TMB-001 0.05% in the ITT population was significantly shorter than vehicle (28 days for TMB-001 0.05% vs. 63.5 days for vehicle); no safety signals; statistically significant more rapid relief of scaling and fissuring, with half of patients acheiving relief in 28 days or less; per-protocol (PP) population had 100%, 40%, and 40% of patients receiving TMB-001 0.05%, TMB-001 0.1%, and vehicle achieved VIIS-50, respectively (p= 0.04 for TMB-001 0.05% vs. vehicle); ITT population, 64%, 40%, and 33% of patients receiving TMB-001 0.05%, TMB-001 0.1%, and vehicle achieved VIIS-50, respectively (p=0.17 for TMB-001 0.05% vs. vehicle); PP population had 100%, 60%, and 10% of patients receiving TMB-001 0.05%, TMB-001 0.1%, and vehicle, respectively, reported a ?2-grade IGA score improvement (p=0.002 for TMB-001 0.05% vs vehicle); ITT population, improvement of ?2-grade IGA score was observed in 55%, 40%, and 8% of patients receiving TMB-001 0.05%, TMB-001 0.1%, and vehicle, respectively (p=0.02 for TMB-001 0.05% vs vehicle) | 2/3/22 | Dermatologic |
| Trevi Therapeutics Inc., of New Haven, Conn. | Haduvio (nalbuphine ER) | Opioid receptor mu antagonist; opioid receptor kappa agonist | Pruritus associated with prurigo nodularis | Completed enrollment in phase IIb/III Prism study; expected top-line data in second quarter of 2022 | 2/1/22 | Dermatologic |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-ANG3 | Double-stranded interference RNA trigger targeting angiopoietin-like protein 3 | Mixed dyslipidemia | Reached full enrollment of more than 180 participants; top-line data expected in first half of 2023 | 2/24/22 | Endocrine/Metabolic |
| Astellas Pharma Inc., of Tokyo | AT-845 | Adeno-associated virus gene replacement therapy | Late-onset Pompe disease | Interim safety data from Fortis study showed safe and well-tolerated; no serious adverse events | 2/7/22 | Endocrine/Metabolic |
| Daewoong Pharmaceutical Ltd., of Seoul, South Korea | Enavogliflozin | SGLT-2 inhibitor | Type 2 diabetes | Top-line results in combination with metformin and gemigliptin from 270 patients showed decrease in HbA1c level by 0.92% and by 0.86% ; no unexpected adverse drug reactions | 2/25/22 | Endocrine/Metabolic |
| Diurnal Group plc, of Cardiff, U.K. | DNL-0200 (previously chronocort) | Modified-release hydrocortisone | Adrenal insufficiency | First of up to 50 patients dosed in the Champain study comparing the efficacy, safety and tolerability of DML-0200 to Plenadren (hydrocortisone) over 16 weeks; data expected in second half of 2022 | 2/10/22 | Endocrine/Metabolic |
| Lysogene SA, of Paris, and Sarepta Therapeutics Inc., of Cambridge, Mass. | LYS-SAF302 | Gene therapy | Mucopolysaccharidosis type IIIA | Top-line data expected in third quarter of 2022; white matter abnormalities have stabilized or diminished in size in all patients; no clinically significant symptoms | 2/24/22 | Endocrine/Metabolic |
| Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Small-molecule phosphoinositide 3-kinase delta inhibitor | Activated phosphoinositide 3-kinase delta syndrome | Study met both co-primary endpoints; statistically significant reduction from baseline in the log10 transformed sum of product of diameters in the index lymphadenopathy lesions (p=0.0012); normalization of immune dysfunction, as evidenced by increased proportion of naïve B cells from baseline (p<0.0001); well-tolerated; mild adverse events in both treatment groups; no deaths and serious events< td> | 2/2/22 | Endocrine/Metabolic |
| Calliditas Therapeutics AB, of Stockholm | Setanaxib | NOX inhibitor | Primary biliary cholangitis | First patient randomized in the Transform study; primary endpoint is alkaline phosphatase; secondary endpoints include change from baseline in liver stiffness and change from baseline in fatigue; interim analysis to be conducted once the 99th randomized patient has completed week 24, which is expected in the first half of 2023 | 2/15/22 | Gastrointestinal |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Moderately to severely active ulcerative colitis | Results showed patients achieved clinical remission (25.7% and 25.2% at 200-mg and 400-mg, respectively vs. 9.5% in placebo group) and other major secondary endpoints at week 12 compared with placebo in QUASAR study; 50.5% and 47.7% at 200 mg and 400 mg, respectively for symptomatic remission rates vs. 20% placebo; endoscopic improvement rates were achieved in 30.7% and 30.8% of patients at 200 mg and 400 mg compared with 12.4% in placebo; histo-endoscopic mucosal improvement rates were achieved in 19.8% and 27.1% of patients at 200 mg and 400 mg compared with 8.6 % in placebo group; endoscopic normalization rates were achieved in 17.8% and 14% of patients at 200 mg and 400 mg compared with 6.7 % in the placebo group; no serious infections reported | 2/18/22 | Gastrointestinal |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Tremfya (guselkumab) | IL-23A inhibitor | Moderately to severely active Crohn's disease | Results from Galaxi I study achieved clinical remission ranged from 57.4% to 73% across 3 doses at week 48; majority of patients in clinical remission were also in corticosteroid-free remission with rates ranging from 55.7% to 71.4 %; achieved PRO-2 remission; consistent with the known safety profile | 2/18/22 | Gastrointestinal |
| Akero Therapeutics Inc., of South San Francisco | Efruxifermin | Differentiated Fc-FGF21 fusion protein | Nonalcoholic steatohepatitis | Completed enrollment in phase IIb study | 2/8/22 | Gastrointestinal |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Oral factor D inhibitor | C3 glomerulopathy, immunoglobulin A nephropathy and primary membranous nephropathy | First patient enrolled in Renew proof-of-concept basket study | 2/22/22 | Genitourinary/Sexual Function |
| Humacyte Inc., of Durham, N.C. | Human acellular vessel (HAV) | Engineered off-the-shelf replacement vessels | Dialysis access in patients with end-stage renal disease | 5-year data from a phase II trial for arteriovenous (AV) access in hemodialysis were published in EJVES Vascular Forum; at month 60, patency allowing for reliable dialysis access cannulation was estimated at 58.2% of evaluable patients, and the most prevalent method of providing hemodialysis access indicate an approximately 30% functional patency of evaluable patients at 5 years; no infections of the HAV were reported during the follow-up period, with the HAV being well-tolerated and non-immunogenic | 2/28/22 | Genitourinary/Sexual Function |
| Regulus Therapeutics Inc., of San Diego | Lademirsen/RG-012 | Single-stranded, chemically modified oligonucleotide that binds to and inhibits the function of miR-21 | Alport Syndrome | Completed enrollment in HERA study | 2/24/22 | Genitourinary/Sexual Function |
| Innocare Pharma Ltd., of Beijing | Orelabrutinib | BTK inhibitor | Primary immune thrombocytopenia | First patient dosed | 2/22/22 | Hematologic |
| Prota Therapeutics Pty Ltd., of Melbourne, Australia | PRT-120 | Oral immunotherapy | Peanut allergy | Data published in The Lancet Child & Adolescent Health showed 51% of participants achieved sustained unresponsiveness (clinical remission) to peanut after 18 months of treatment; improved quality of life | 2/9/22 | Immune |
| Acurx Pharmaceuticals Inc., of Staten Island, N.Y. | Ibezapolstat | Antibiotic | C. difficile infection | Study showed 100% met primary and secondary efficacy endpoints of clinical cure at end of treatment and sustained clinical cure of no recurrence of CDI at the 28-day follow-up visit; low plasma and high fecal concentrations; favorable microbiome changes included regrowth of Actinobacteria and Firmicutes phylum species; good tolerability | 2/7/22 | Infection |
| Adamis Pharmaceuticals Corp., of San Diego | Tempol | Antiviral, anti-inflammatory and antioxidant activity | COVID-19 | Due to the acceleration of patient enrollment in the phase II/III trial, an ad-hoc meeting of the Data Safety Monitoring Board (DSMB) was held to evaluate interim clinical and safety data; the DSMB determined that the study can continue, as there were no safety or clinical concerns identified | 2/28/22 | Infection |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | Capsid-virus-like particle vaccine | COVID-19 | Additional phase II results from a group of 66 seropositive subjects showed that, comparing the induced levels of neutralizing titres by taking into account the starting titre (pre-booster) and/or the time since the last vaccination, the higher booster dose of ABNCoV2 trended toward inducing stronger levels of neutralizing titres against SARS-CoV-2; the vaccine was generally well-tolerated, with no related serious adverse events reported and no relevant difference in the safety profile between subjects receiving either the low- or high-dose, which will be used in the upcoming phase III trial | 2/28/22 | Infection |
| Enanta Pharmaceuticals Inc., of Watertown, Mass. | EDP-938 | N-protein inhibitor | Respiratory syncytial virus infection | Data published in The New England Journal of Medicine showed primary efficacy endpoint was met with a highly statistically significant reduction (p<0.001) 5 28 in viral load auc observed; statistically significant reduction was observed total symptom score for edp-938 dosing groups compared placebo arm (p<0.001); good pharmacokinetics; safe and well-tolerated; favorable safety profile over days of through day follow-up; no serious adverse events; discontinuations< td> | 2/17/22 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Nucleotide derivative | COVID-19 | The 53-patient pediatric study showed the drug was well tolerated; 85% of patients showed clinical improvement based on the clinical ordinal scale; recovery rate was 83% at last assessment | 2/11/22 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Lenacapavir | HIV capsid inhibitor | HIV | 1-year data in heavily treatment-experienced patients administered subcutaneously every 6 months in combination with other antiretrovirals achieved high rates of virologic suppression and clinically meaningful increases in CD4 counts; 83% of patients achieved an undetectable viral load (<50 copies ml) at week 52; clinically meaningful viral load reduction of least 0.5 log10 ml from baseline compared with randomly allocated to receive placebo during the 14-day functional monotherapy period (88% vs. 17%, p<0.0001); statistically significant greater mean decrease in than those who received (-1.93 -0.29 ml, well-tolerated< td> | 2/16/22 | Infection |
| Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Oral small-molecule potassium-competitive acid blocker | Gastroesophageal reflux disease (GERD) due to H. pylori infection | In the Phalcon-NERD study, 56%, 60.6% and 70% of patients taking 10-mg, 20-mg and 40-mg doses, respectively, had complete relief of heartburn within 3 hours and sustained relief over 24 hours compared to 27.3% of patients taking placebo (p<0.0001); plans to initiate phase iii nerd-301 study with top-line results expected in 2023< td> | 2/9/22 | Infection |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone (RVX-208) | BD2 (bromodomain) selective BET (bromodomain and extra-terminal) inhibitor | COVID-19 | First Brazilian and Canadian sites initiated | 2/17/22 | Infection |
| Russian Direct Investment Fund, of Moscow and Astrazeneca plc, of Cambridge, U.K. | Sputnik V and Astrazeneca vaccines | Adenoviral-based vaccines against SARS-CoV-2 | COVID-19 prophylaxis | Combination of the Astrazeneca vaccine and the first component of Sputnik V vaccine had a good safety profile with no serious adverse events related to the combined therapy | 2/14/22 | Infection |
| Valneva SE, of Saint Herblain, France, and Pfizer Inc., of New York | VLA-15 | Multivalent protein subunit vaccine targets the outer surface protein A of Borrelia burgdorferi | Lyme disease | VLA-15 found to be immunogenic with both vaccination schedules tested; induction of anti-OspA IgG antibody titers was higher in participants who received the three-dose primary series compared to those who received the two-dose primary series; consistent with acceptable safety and tolerability profile; no vaccine-related serious adverse events; pediatric study ongoing with intial data expected in first half of 2022 | 2/4/22 | Infection |
| Windtree Therapeutics Inc., of Warrington, Pa. | Lucinactant | Aerosolized KL4 surfactant therapy | COVID-19 associated-lung injury and acute respiratory distress syndrome | Completed enrollment; data expected in first quarter of 2022 | 2/1/22 | Infection |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Nurtec ODT (rimegepant) | Oral CGRP receptor antagonist | Chronic rhinosinusitis with or without nasal polyps | Commenced enrollment in phase II/III study | 2/22/22 | Inflammatory |
| Medivir AB, of Stockholm | MIV-711 | Selective cathepsin K inhibitor | Osteoarthritis | Subgroup analysis showed a statistically significant reduction in osteoarthritis pain | 2/28/22 | Inflammatory |
| Aptinyx Inc., of Evanston, Ill. | NYX-2925 | Oral NMDA receptor positive allosteric modulator | Fibromyalgia | Completed enrollment of 305 patients in the phase IIb study | 2/28/22 | Musculoskeletal |
| Neuvivo Inc., of Palo Alto, Calif. | NP-001 | Disease-modifying, anti-inflammatory drug | Amyotrophic lateral sclerosis | Results from phase IIb study published in the journal Muscle & Nerve showed NP-001 significantly slowed or halted in a subset of patients during a 6-month period; 36% slower rate of decline of ALSFRS-R scores; 51% slower rate of decline of respiratory function; 34% of NP-001 treated vs. 11% of placebo patients did not progress over the 6-month studies (p = 0.004); higher levels of inflammation showed a 10-fold higher rate of non-progression than placebo with NP-001 treatment (p = 0.001); safe and well-tolerated | 2/1/22 | Musculoskeletal |
| Seelos Therapeutics Inc., of New York | SLS-005 | Low molecular weight disaccharide | Amyotrophic lateral sclerosis | First participants dosed | 2/28/22 | Musculoskeletal |
| Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Inhibits the formation of neurotoxic soluble amyloid oligomers | Alzheimer’s disease | Data from 84 enrolled patients with APOE4/APOE3/4 genotype showed significant 29% reduction in plasma p-tau181 (p=0.028) at 26 weeks, and 18% reduction at 13 weeks (p=0.038); significantly reduced the plasma p-tau181/A?42 ratio by 30% at 26 weeks (p=0.022), 21% at 13 weeks (p=0.018); RAVLT total memory scores included both immediate and delayed recall, showed a significant improvement from baseline to week 26 (p=0.002); nausea as adverse events; no evidence of vasogenic edema and no drug-related serious events; safety and tolerability profile was favorable | 2/8/22 | Neurology/Psychiatric |
| Cyclerion Therapeutics Inc., of Cambridge, Mass. | CY-6463 | CNS-penetrant sGC stimulator | Mitochondrial encephalomyopathy, Lactic acidosis, and Stroke-like episodes | Completed enrollment; top-line data expected in second quarter of 2022 | 2/24/22 | Neurology/Psychiatric |
| Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab-cdon) | Anti-CD19 B-cell-depleting humanized monoclonal antibody | Neuromyelitis optica spectrum disorder | During the 28-week treatment in the phase II/III study, 89% of patients taking Uplizna remained attack-free compared to 58% of patients taking placebo; a post-hoc analysis showed 12 attacks in patients taking Uplizna (67%) were minor and six (33%) were major, compared to 12 (55%) minor attacks and 10 (45%) major attacks in patients taking placebo | 2/16/22 | Neurology/Psychiatric |
| Noema Pharma AG, of Basel, Switzerland | NOE-101 | mGluR5 inhibitor | Trigeminal neuralgia | First patient dosed | 2/22/22 | Neurology/Psychiatric |
| Olipass Corp., of Seoul | OLP-1002 | SCN9A antisense pain killer | Osteoarthritis patients with pain | Initiated dosing in phase IIa study with 2 stages; first stage is an open label study to identify an optimum dose range of the efficacy profile(1 mcg to 80 mcg s.c.); second stage a placebo-controlled double blind study | 2/18/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol formulated as a permeation-enhanced clear gel | 22q11.2 deletion syndrome in children and adolescents | Enrollment completed | 2/28/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Small-molecule modulator of RASP | Allergic conjunctivitis | Results from phase II study published in the Journal Clinical Ophthalmology showed rapid and durable improvement in the symptoms and signs; statistically significant improvement in patient-reported ocular itching and tearing and investigator-assessed ocular redness; improvement in signs and symptoms persisted over the entire chamber and all post-chamber time points | 2/1/22 | Ocular |
| Kodiak Sciences Inc., of Palo Alto, Calif. | KSI-301 | Anti-VEGF antibody biopolymer conjugate | neovascular age-related macular degeneration | Top-line data showed study didn't meet primary endpoint of showing non-inferior visual acuity gains compared to aflibercept given every 8 weeks; 59% of patients achieved every-5-month dosing at year 1 with visual acuity gains and anatomic improvements comparable to the overall aflibercept group; safe and well tolerated; no new or unexpected safety signals | 2/23/22 | Ocular |
| Nanoscope Therapeutics Inc., of Bedford, Texas | MCO-010 | Multicharacteristic opsin ambient-light activatable optogenetic monotherapy | Retinitis pigmentosa | Completed enrollment; top-line data expected in first quarter of 2023 | 2/7/22 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Extracellular domains of human VEGFR-3 antibodies fused to the Fc-domain | Polypoidal choroidal vasculopathy (PCV) | OPT-302 plus Lucentis (ranibizumab, Roche Holding AG) produced +6.7 letters in best-corrected visual acuity compared to Lucentis alone (p = 0.0253) in 66 patients with PCV – a subtype of neovascular age-related macular degeneration | 2/14/22 | Ocular |
| Oxurion NV, of Leuven, Belgium | THR-149 | Plasma kallikrein inhibitor | Diabetic macular edema | Post-hoc analysis that excluded 2 patients with abnormal optical coherence tomography biomarker data showed THR-149 produced an improvement of 9.3 letters in mean best-corrected visual acuity at 3 months, which was maintained for the remaining 3 months of the trial | 2/14/22 | Ocular |
| Polyactiva Pty Ltd., of Melbourne, Australia | PA-5108 | Ocular implant with Prezia; sustained drug delivery technology | Primary open-angle glaucoma | Study met primary and secondary efficacy endpoints; statistically significant intraocular pressure (IOP) reduction vs. baseline of a >20% IOP-lowering effect (p<0.001 12 26 and p<0.01) across all assessment visits; statistically significant clinically meaningful mean change in diurnal iop at weeks (p<0.001); well-tolerated with no ocular serious adverse events related to product; endothelial cell loss implant movement< td> | 2/17/22 | Ocular |
| Proqr Therapeutics NV, of Leiden, the Netherlands | Sepofarsen | RNA therapy | CEP290-mediated Leber congenital amaurosis 10 | Top-line data from phase II/III illuminate study did not meet its primary endpoint of best corrected visual activity (BCVA) at month 12; well-tolerated; mean change from baseline in BCVA in the 160/80-?g dose group was -0.11 logMAR (p=0.96), in the 80/40 ?g group -0.13 logMAR (p=0.97), and in the sham group -0.12 logMAR. P values are treatment group vs. sham at month 12; no difference between the treated and sham groups for full-field stimulus test and mobility; | 2/11/22 | Ocular |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Aflibercept 8-mg | VEGF inhibitor | Wet age-related macular degeneration | Trial met primary safety endpoint and no new safety signals seen through week 44; 40% of patients treated with aflibercept 8-mg dose did not have fluid in the center subfield compared to 28% of patients treated with Eylea ( p=0.2185); no macular fluid compared to patients treated with Eylea (p=0.0395); 7.9 average letter improvement from baseline in the aflibercept compared to 5.1 letters in the Eylea; achieved at least a 10-letter gain, and more than a quarter achieved more than 15 letters comapred to Eylea; no serious adverse events | 2/11/22 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | NAV AAV8 vector encoding antibody fragment designed to inhibit VEGF | Diabetic retinopathy without center-involved diabetic macular edema | In Altitude trial, 6 months after treatment, 47% of cohort 1 patients had a ?2 step improvement from baseline on the ETDRS-DRSS (up from 33% of patients at 3 months), compared to 0% of patients in observational control; in-office suprachoroidal delivery showed good tolerability in 15 patients treated in cohort 1 with no drug-related serious adverse events at six months and no intraocular inflammation observed; cohorts 2 and 3 continue to enroll patients at an increased dose level of 5x1011 GC/eye | 2/12/22 | Ocular |
| Stealth Biotherapeutics Corp., of Boston | Elamipretide | Mitochondria-targeted compound | Geographic atrophy associated with dry age-related macular degeneration | Final of 176 patients in the Reclaim-2 study completed treatment; top-line results expected during the second quarter of 2022 | 2/15/22 | Ocular |
| Rivus Pharmaceuticals Inc., of Charlottesville, Va. | HU-6 | Controlled metabolic accelerator therapy | Obesity | Results showed significant reductions in liver, visceral and total body fat; statistically significant (p<0.0001 3 6 10 by ancova) reductions in liver fat at all dose levels; weight and loss showed a response with participants losing an average of pounds (p<0.001, high vs. placebo); patients elevated hba1c levels experienced greater loss, (p<0.0001, significant dose-dependent reduction glycated albumin; inflammation marker sensitivity c-reactive protein; well-tolerated no serious adverse events or deaths< td> | 2/9/22 | Other |
| Aardvark Therapeutics Inc., of San Diego | ARD-101 | Small-molecule bitter taste receptor (TAS2R) pan-agonist | Obesity | Initiated enrollment in phase II studies; trials to evaluate the safety, tolerability and efficacy of oral drug in adults | 2/2/22 | Other/Miscellaneous |
| Aardvark Therapeutics Inc., of San Diego | ARD-101 | Small-molecule bitter taste receptor (TAS2R) pan-agonist | Refractory weight gain post-bariatric surgery | Initiated enrollment in phase II studies; planning to enroll 30 patients | 2/2/22 | Other/Miscellaneous |
| Aardvark Therapeutics Inc., of San Diego | ARD-101 | Small-molecule bitter taste receptor (TAS2R) pan-agonist | Prader-Willi syndrome | Initiated enrollment in phase II studies; planning to enroll 12 patients | 2/2/22 | Other/Miscellaneous |
| Otonomy Inc., of San Diego | OTO-313 | Sustained-exposure formulation of NMDA receptor antagonist gacyclidine | Tinnitus | Completed patient enrollment; top-line results for all timepoints expected in mid-2022 | 2/22/22 | Other/Miscellaneous |
| Pliant Therapeutics Inc., of South San Francisco | PLN-74809 | Oral small-molecule dual selective inhibitor of ?v?6/?v?1 | Idiopathic pulmonary fibrosis | DSMB recommended trial without modification and proceed to evaluate dosing at 320mg; no drug-related serious adverse events or drug-related severe adverse advents reported | 2/24/22 | Respiratory |
| Trevi Therapeutics Inc., of New Haven, Conn. | Haduvio (nalbuphine ER) | Opioid receptor mu antagonist; opioid receptor kappa agonist | Idiopathic pulmonary fibrosis | Interim analysis showed statistically significant on the primary efficacy endpoint of cough reduction; 52% placebo-adjusted reduction in the geometric mean percent change in daytime cough events (p<0.0001); no new safety signals; well-tolerated< td> | 2/24/22 | Respiratory |
| Vicore Pharma Holding AB, of Gothenburg, Sweden | C-21 | Angiotensin II type 2 receptor agonist | Idiopathic pulmonary fibrosis | Interim analysis of Air study demonstrated that forced vital capacity was increased by an average of 251 ml over baseline at 24 weeks; plans to start a phase IIb study | 2/10/22 | Respiratory |
| Aadi Bioscience Inc., of Pacific Palisades, Calif. | Nab-sirolimus | Sirolimus albumin-bound nanoparticles for injectable suspension | Solid tumors with pathogenic inactivating alterations in TSC1 and TSC2 genes | First patient dosed | 3/31/22 | Cancer |
| Actuate Therapeutics Inc., of Fort Worth, Texas, and the University of Kansas Cancer Center | Elraglusib (9-ING-41) | Small-molecule glycogen synthase kinase-3 beta (GSK-3?) inhibitor | Advanced pancreatic cancer | The first patient was enrolled in a phase II clinical trial plus retifanlimab combined with gemcitabine/nab-paclitaxel | 3/1/22 | Cancer |
| Alligator Bioscience AB, of Lund, Sweden | Mitazalimab | CD40 agonist | Metastatic pancreatic ductal adenocarcinoma | Completed second dose-escalation cohort (900 µg/kg of mitazalimab in combination with mFOLFIRINOX); safe and recommended dosing level for phase II study; initiated enrollment | 3/23/22 | Cancer |
| Ammax Bio Inc., of Redwood City, Calif. | AMB-05X | Monoclonal antibody against CSF1R | Tenosynovial giant cell tumor | Results from the local delivery phase II study were positive and showed that intra-articular injection strategy showed efficacy across multiple clinical endpoints and favorable safety profile after 12 weeks of therapy | 3/2/22 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | Lisaftoclax (APG-2575) | Bcl-2 selective inhibitor | Relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma | First patient dosed in China | 3/15/22 | Cancer |
| Bold Therapeutics Inc., of Vancouver, Brisith Columbia | BOLD-100 | Ruthenium(III) complex that inhibits the expression of endoplasmic reticulum chaperone BiP (GRP78) | Advanced gastrointestinal cancers | Started phase II trial | 3/2/22 | Cancer |
| Bold Therapeutics Inc., of Vancouver, British Columbia | BOLD-100 | Ruthenium(III) complex that inhibits the expression of endoplasmic reticulum chaperone BiP (GRP78) | Advanced gastrointestinal cancers (colorectal, pancreatic, gastric and bile duct) | Completed the phase Ib (dose-escalation) portion of its seamless adaptive oncology trial of BOLD-100 in combination with FOLFOX | 3/1/22 | Cancer |
| Carsgen Therapeutics Holdings Ltd., of Shanghai | CT-041 | CLDN18.2-targeted CAR T cells | Advanced gastric or gastroesophageal junction adenocarcinoma | Completed first patient enrollment | 3/23/22 | Cancer |
| Catalym GmbH, of Munich, Germany | CTL-002 | Humanized MAb designed to neutralize the tumor-produced Growth Differentiation Factor-15 | Solid tumors | Started trial | 3/2/22 | Cancer |
| Enzychem Lifesciences Corp., of Englewood Cliffs, N.J. | EC-18 | Small-molecule oral immunomodulator | Chemoradiation-induced oral mucositis | Completed final clinical study report; no safety issues; median duration of severe oral mucositis (SOM) was 0 days vs. 13.5 days in placebo group (100% reduction); incidence of SOM from baseline through the active treatment period was reduced by 37.1% in EC-18 group vs. placebo group (40.9% vs. 65%); incidence of SOM from baseline through short-term follow-up period was reduced by 35% in comparison to placebo group (45.5% vs. 70%); median delay of 11.5 days in time to the first use of opioid analgesics vs. placebo group (37 days vs. 25.5 days) | 3/9/22 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha | Soluble LAG-3 fusion protein | Non-small-cell lung cancer | Data from TACTI-002 study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed 73.7% of evaluable patients had tumor shrinkage; 73% of patients were alive at 6 months; disease control rate of 36.1%, with 26% being progression-free at the 6-month landmark; 5.6% had confirmed and durable partial responses in the study for over 9 months and 23 months, respectively; 6 patients still under therapy in the trial; median overall survival not reached; safe; well-tolerated in combination therapy | 3/30/22 | Cancer |
| Isa Pharmaceuticals BV, of Leiden, the Netherlands | ISA-101b | Immunotherapy targeting oncogenic E6 and E7 proteins of HPV16 | Head and neck cancer | Extension data from a key study were published in the Journal of Immunotherapy of Cancer and confirmed efficacy of the combination treatment (ISA-101b and nivolumab); 2 patients displayed a complete response to treatment and patients survived disease-free for more than 44 months; improved tumor infiltration by immune cells predicted response to therapy | 3/1/22 | Cancer |
| Jazz Pharmaceuticals plc, of Dublin | Zepzelca (lurbinectedin) | Alkylating drug that binds guanine residues within DNA | Solid tumors | First patient enrolled in Emerge-201 study as a monotherapy in 3 cohorts of patients | 3/24/22 | Cancer |
| Oncolytic Biotech Inc., of San Diego | Pelareorep-paclitaxel combination therapy | Intravenously delivered immunotherapeutic agent; taxane | Advanced or metastatic breast cancer | Advanced to third and final dose-escalation cohort of the bridging study; to evaluate safety, tolerability and preliminary efficacy in Chinese patients; first 2 cohorts completed; well-tolerated; no new safety signals | 3/24/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 (Versamune-HPV16) | Protein subunit vaccine | Advanced HPV-associated cancers | Achieved intended enrollment of 30 patients in the checkpoint inhibitor (CPI) refractory arm of the NCI-led phase II study; study enrolled 45 patients; continue to enroll both CPI refractory and CPI naïve patients until the total enrollment of 56 achieved | 3/15/22 | Cancer |
| Sanofi SA, of Paris | Amcenestrant | Oral selective estrogen receptor degrader | ER+/HER2- advanced or metastatic breast cancer | Ameera-3 study did not meet primary endpoint of improving progression-free survival | 3/14/22 | Cancer |
| Spectrum Pharmaceuticals Inc., of Henderson, Nev. | Poziotinib | EGFR tyrosine kinase inhibitor | Non-small-cell lung cancer | Results from Zenith20 study met its primary endpoint as the observed lower bound of 30% exceeded the prespecified lower bound of 20%; confirmed objective response rate (ORR) of 41%; disease control rate was 73%; median progression-free survival was 5.6 months; 90% of patients had dose interruptions and 79% had reductions from the 16-mg QD dose; 64% had reductions from the 8-mg BID dose; consistent safety profile | 3/7/22 | Cancer |
| XNK Therapeutics AB, of Stockholm | Autologous natural killer cells | Autologous natural killer cells | Multiple myeloma | First patient treated in combination with Sarclisa (Isatuximab, Sanofi SA) | 3/23/22 | Cancer |
| Cytokinetics Inc., of South San Francisco | Aficamten | Next-generation cardiac myosin inhibitor | Hypertrophic cardiomyopathy | Cohort 4 opened enrollment | 3/2/22 | Cardiovascular |
| Windtree Therapeutics Inc., of Warrington, Pa. | Istaroxime | Na+ K+ ATPase inhibitor; sarco endoplasmic calcium ATPase 2a stimulator | Cardiogenic shock | Completed enrollment; top-line data in Apr. 2022 | 3/15/22 | Cardiovascular |
| Aldeyra Therapeutics Inc., Lexington,Mass. | ADX-629 | Orally administered RASP modulator | Psoriasis | Top-line data showed statistically significantly decrease in PASI scores (p=0.0008 vs. baseline at week 12); peak PASI 50% and PASI 75% responder percentages were 57% (p=0.001) and 25% (p=0.051), respectively; investigator global assessment scores decreased over the duration of treatment (p=0.01 vs. baseline at week 12); normalization of global gene expression patterns; no gene expression pathways in lesional tissue were dysregulated compared to non-lesional skin; pro-inflammatory RASP malondialdehyde reduced relative to baseline as soon as four weeks after initiation of treatment (p=0.02) | 3/29/22 | Dermatologic |
| Anaptysbio Inc., of San Diego | Imsidolimab | Anti-interleukin-36 receptor monoclonal antibody | Moderate to severe acne | Top-line data from Acorn trial did not demonstrate efficacy over placebo on primary or secondary endpoints; well-tolerated; no imsidolimab-related serious or severe adverse events; mean baseline facial inflammatory lesion counts for imsidolimab high, low dose arm and placebo arm were 30, 29 and 27, respectively; discontinue imsidolimab clinical development in acne | 3/15/22 | Dermatologic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Donidalorsen | Antisense medicine designed to reduce the production of prekallikrein | Hereditary angioedema | Results published in the New England Journal of Medicine showed 90% reduction in angioedema attacks compared with placebo at 80 mg; significant improvement in quality of life (P=0.002); safe and well-tolerated; mean monthly rate of angioedema attacks was 0.23 and 2.21 in patients receiving donidalorsen and placebo, respectively (P<0.001); no deaths or serious adverse events< td> | 3/16/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | KX-826 (pyrilutamide) | Androgen receptor antagonist | Androgenic alopecia | Dosed the first patient in U.S.-based trial | 3/1/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | KX-826 (pyrilutamide) | Androgen receptor antagonist | Androgenic alopecia | Completed enrollment of 160 patients | 3/6/22 | Dermatologic |
| Moonlake Immunotherapeutics AG., of Zug, Switzerland | Sonelokimab | Nanobody; inhibits IL-17A and IL-17F | Moderate to severe hidradenitis suppurativa | Initiated phase II study in patients (n=200) | 3/24/22 | Dermatologic |
| Promore Pharma AB, of Stockholm | Ensereptide (PXL-01) | Peptide-based drug | Skin scar | Completed recruitment goal in phase II study (n=24) | 3/17/22 | Dermatologic |
| Recursion Inc., of Salt Lake City | REC-3599 | Orally bioavailable, small molecule inhibitor of protein kinase C and glycogen synthase kinase 3 beta | Infantile GM2 gangliosidosis | Company decided that conducting a dose optimization study in a sheep efficacy model of Tay-Sachs disease is prudent and in the best interest of patients before enrolling infants | 3/3/22 | Endocrine/Metabolic |
| Rezolute Inc., of Redwood City, Calif. | RZ-358 | Monoclonal antibody targeting an allosteric site on the insulin receptor | Congenital hyperinsulinism | Data from Rize study showed highly significant improvements in hypoglycemia; good safe and tolerability | 3/23/22 | Endocrine/Metabolic |
| Valbiotis SA, of La Rochelle, France | TOTUM-070 | Combination of plant extracts | Hypercholesterolemia | Data showed presence of 22 metabolites of interest in the serum of volunteers after oral ingestion of 5 g of TOTUM-070; results expected in the second quarter of 2022 | 3/29/22 | Endocrine/Metabolic |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-42 | Selective farnesoid X receptor agonist; antiviral agent targeting transcription of HBV cccDNA | Chronic hepatitis B | Completed patient enrollment; safe and well-tolerated at 10-mg and 15-mg doses; no severe adverse events | 3/30/22 | Gastrointestinal |
| Durect Corp., of Cupertino, Calif. | DUR-928 (larsucosterol) | Inhibits DNA methyltransferases | Alcohol-associated hepatitis | Dosed first patient in Europe as part of its phase IIb trial | 3/2/22 | Gastrointestinal |
| Morphic Therapeutic Inc., of Waltham, Mass. | MORF-057 | Integrin alpha4 beta7 inhibitor | Moderate to severe ulcerative colitis | Initiated MORF-057-201 Emerald 1 study | 3/25/22 | Gastrointestinal |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin capsule | Nonalcoholic steatohepatitis | Completed enrollment of patients; top-line data expected in first half of 2022 | 3/16/22 | Gastrointestinal |
| Acer Therapeutics Inc., of Newton, Mass. | ACER-801 (osanetant) | Non-hormonal, neurokinin 3 receptor antagonist | Moderate to severe vasomotor symptoms associated with menopause | First patient enrolled; results expected in the second half of 2022 | 3/30/22 | Genitourinary/Sexual Function |
| Evofem Biosciences Inc., of San Diego | EVO-100 (Phexxi) | Contraceptive vaginal gel | Contraception | Data based on phase IIb/III results showed gel provided significant decrease in gonorrhea (by 78%) and chlamydia (by 50%) infections | 3/31/22 | Genitourinary/Sexual Function |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes | Anti-glomerular basement membrane (anti-GBM) disease | Results published in the Journal of the American Society of Nephrology showed improved kidney function at 6 months, better than previously published cohorts; no safety concerns; 67% of the included patients were dialysis-independent vs. 18% in a historical control cohort | 3/8/22 | Genitourinary/Sexual Function |
| Urovant Sciences Inc., of Irvine, Calif. | URO-902 | locally injected gene therapy product;plasmid human cDNA encoding maxi-K channel | Overactive bladder and urge urinary incontinence | Top-line data showed clinically meaningful and statistically significant effect at week 12 on a number of relevant outcome measures in number of micturitions, urgency episodes and quality of life indicators compared to placebo; well-tolerated | 3/7/22 | Genitourinary/Sexual Function |
| Vertex Pharmaceuticals Inc., of Boston | VX-147 | Therapy targeting APOL1 | APOL1 gene-mediated kidney disease | Advanced to pivotal phase II/III study; reduction in the rate of decline of kidney function as measured by estimated glomerular filtration rate slope vs. placebo at two years as primary endpoint; also designed to have a pre-planned interim analysis at week 48; pivotal study will open for enrollment later March 2022 | 3/22/22 | Genitourinary/Sexual Function |
| Cellphire Therapeutics Inc., of Rockville, Md. | DMSO cryopreserved platelets | DMSO cryopreserved platelets | Acute hemorrhage in patients with a platelet deficiency or a platelet dysfunction | First patient treated | 3/29/22 | Hematologic |
| Rigel Pharmaceuticals Inc., of South San Francisco | Fostamatinib disodium hexahydrate | SYK inhibitor | Warm antibody autoimmune hemolytic anemia | Results published in the American Journal of Hematology showed 46% of patients achieved the primary endpoint; increase in median Hgb were detected at week 2 and sustained over time; manageable and consistent adverse events; no new safety signals | 3/8/22 | Hematologic |
| Abivax SA, of Paris | ABX-464 | Rev protein modulator | Rheumatoid arthritis | Results from continued daily treatment with 50 mg showed 57% of the patients were in remission at week 52; 100% completed 52 weeks of treatment, achieved at least an ACR20 response (58% in full analysis set); 83% and 52% achieved ACR50 and ACR70 responses, respectively; 17 patients discontinued the study due to either mild to moderate adverse events; safety and tolerability was met | 3/10/22 | Immune |
| Neurogenesis Ltd., of Jerusalem, and Hadassah Medical Center, of Jerusalem | NG-01 | Autologous cell therapy candidate | Progressive multiple sclerosis | Phase II results published in Stem Cells Translational Medicine, show significant reduction of NF-L, a biomarker for neuronal damage, in patients after treatment | 3/1/22 | Immune |
| Adagio Therapeutics Inc., of Waltham, Mass. | Adintrevimab (ADG-20) | Monoclonal antibody | COVID-19 | Study met its primary endpoint; 71% relative risk reduction in favor of adintrevimab through 3 months in PrEP cohort; lower incidence of symptomatic COVID-19 compared with placebo through month 3 or the emergence of omicron(p<0.0001); 28 56 77 relative risk reduction of 59% and 47% with a median follow-up duration days, respectively (nominal p<0.05); associated lower incidence symptomatic covid-19 through day compared placebo in pep cohort (p="0.0135;" 75% favor adintrevimab days; similar safety profiles< td> | 3/30/22 | Infection |
| Adamis Pharmaceuticals Corp., of San Diego | Tempol | Antiviral, anti-inflammatory and antioxidant activity | COVID-19 | Data safety monitoring board met to evaluate the clinical and safety data from the first planned interim analysis; recommended the study continue without modification | 3/14/22 | Infection |
| Aldeyra Therapeutics Inc., Lexington,Mass. | ADX-629 | Orally administered RASP modulator | COVID-19 | Data showed reductions in plasma levels of the cytokines CXCL9 (p=0.0008), IFN? (p=0.02), and TNF? (p=0.07);no serious adverse events observed | 3/29/22 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | rNAPc2 (AB-201) | Small recombinant protein; selective inhibitor of tissue factor | Hospitalized COVID-19 | Top-line data from 160 patients demonstrated a treatment benefit; achieved statistical significance for the primary efficacy endpoint of change in D-dimer level from baseline to day 8 compared to standard-of-care heparin; change in D-dimer of p=0.41 compared to p=0.91 in the heparin group (between groups p=0.47); D-dimer levels as percent change decreased at day 8 or hospital discharge in both the pooled high- and low-dose rNAPc2 arms (p=0.009) and in the heparin arm (p= 0.010); statistically significant in the higher-dose rNAPc2 group (p=0.016); non-significant secondary endpoints; well-tolerated at both doses | 3/31/22 | Infection |
| Atriva Therapeutics GmbH, of Tübingen and Frankfurt, Germany | Zapnometinib | Blocks RNA-virus replication; immune modulator | Moderate to severe COVID-19 | Enrolling patients in phase II study | 3/28/22 | Infection |
| Brii Biosciences Ltd., of Beijing | BRII-835 (VIR-2218) | HBV-targeting siRNA | Chronic hepatitis B virus (HBV) infection | Data from Chinese patients showed dose-dependent reduction in hepatitis B surface antigen; well-tolerated; mild or moderate adverse events; no clinically significant alanine transaminase elevations | 3/31/22 | Infection |
| Capricor Therapeutics Inc., of Los Angeles | CAP-1002 | Allogeneic cardiac-derived cell therapy | Hospitalized patients with advanced symptoms of COVID-19 | Results of Inspire study met primary objective of safety; safe and well-tolerated; overall mortality in the study was 20%; 6 deaths in placebo group and 5 deaths in the CAP-1002 group | 3/28/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Chengdu, China | SCB-2019 (CpG 1018/Alum) | COVID-19 S-Trimer subunit vaccine; vaccine comprising recombinant SARS-CoV-2 spike glycoprotein | COVID-19 prophylaxis | Preliminary data showed high and durable protection against COVID-19 as well as strong immune responses against Omicron; no safety concerns; 2-fold higher levels of neutralizing antibodies against the omicron variant in individuals previously receiving 2 doses of Astrazeneca plc's COVID-19 vaccine, Vaxzevria; 4-fold higher levels of neutralizing antibodies against the Omicron variant in previously infected; multi-fold higher levels of neutralizing antibodies against the omicron variant when compared to individuals receiving 3 doses of Vaxzevria | 3/17/22 | Infection |
| Ena Respiratory Pty Ltd., of Sydney | INNA-051 | TLR2/6 agonist; topical, broad-spectrum antiviral immunomodulator | H3N2 influenza virus infection | First healthy adult dosed; to evaluate the safety, tolerability, and antiviral efficacy; enroll up to 123 participants | 3/15/22 | Infection |
| Moderna Inc., Cambridge, Mass. | mRNA-1273.214; mRNA-1273.529; mRNA-1273 | mRNA booster dose vaccine; omicron-specific bivalent booster candidate | COVID-19 | First participant dosed; expected to enroll 375 subjects | 3/10/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 (Spikevax) | mRNA vaccine | COVID-19 | Interim data in participants 6 months to under 6 years met primary endpoint; robust neutralizing antibody titers similar to adults; well-tolerated at 25 mcg; favorable safety profile; statistically significant vaccine efficacy against omicron wave | 3/23/22 | Infection |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | Zyesami (aviptadil) | Synthetic form of vasoactive intestinal polypeptide; nebulized form | COVID-19 | I-SPY COVID Trial has been stopped; met the predefined futility criterion; greater than 90% probability that the hazard rate for recovery is less than 1.5 when compared to standard treatment (Pr(HR < 1.5) ? 0.9) | 3/31/22 | Infection |
| Pfizer Inc., of New York | Paxlovid (nirmatrelvir/ritonavir) | Inhibits a SARS-CoV-2 protein; protease inhibitor, slows nirmatrelvir’s breakdown | COVID-19 | Initiated phase II/III study in pediatric patients | 3/9/22 | Infection |
| Revelation Biosciences Inc., of San Diego | REVTx-99 | Intranasal drop formulation; TLR4 agonist | H3N2 influenza virus infection | Completed enrollment and dosing in phase IIb viral challenge study (RVL-VRL01); evaluating the efficacy of intranasal administration in healthy volunteers | 3/15/22 | Infection |
| Revelation Biosciences Inc., of San Diego | REVTx-99a | Intranasal therapy activates a nonspecific immune response | Preventive treatment of H3N2 influenza (influenza A) | Enrollment and dosing completed; primary endpoint analysis expected early in the second quarter of 2022 | 3/24/22 | Infection |
| Revelation Biosciences Inc., of San Diego | REVTx-99a | Intranasal therapy that activates a nonspecific immune response | Preventive treatment of H3N2 influenza (influenza A) | Results from RVL-VRL01 study did not meet primary endpoint in healthy individuals; no serious adverse events | 3/30/22 | Infection |
| Windtree Therapeutics Inc., of Warrington, Pa. | Lucinactant | Aerosolized KL4 surfactant therapy | COVID-19 associated lung injury and acute respiratory distress syndrome | Results showed safe and well-tolerated; Oxygenation and other physiological outcomes were generally stable and improved in critically ill patients | 3/22/22 | Infection |
| Atom Bioscience (also known as Jiangsu Atom Bioscience and Pharmaceutical Co. Ltd.), of Jiangsu, China | APB-671 | Small-molecule inhibitor of the urate transporter | Chronic gout | Results showed APB-671 achieved its primary endpoint of reducing serum uric acid levels to less than 6 mg/dL; no safety concerns reported | 3/21/22 | Inflammatory |
| Taiwan Liposome Co., of Taipei, Taiwan | TLC-599 | Sustained-release formulation of dexamethasone sodium phosphate | Osteoarthritis of the knee | Results published in Arthritis Research & Therapy showed statistically significantly reduced pain scores and oral pain medication use for up to 6 months; statistically significantly greater reduction in WOMAC pain and function as well as VAS scores compared to placebo through 24 weeks | 3/7/22 | Inflammatory |
| Capricor Therapeutics Inc., of Los Angeles | CAP-1002 | Allogeneic cardiac-derived cell therapy | Duchenne muscular dystrophy | Results published in The Lancet for Hope-2 study showed improvements in both skeletal and cardiac muscle function after 4 doses; safe and well-tolerated; no serious safety signals | 3/11/22 | Musculoskeletal |
| Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | SMN2 splicing modulator | Spinal muscular atrophy | Interim results of Rainbowfish study showed majority of babies treated with Evrysdi for at least 12 months were able to stand and walk within timeframes typical of healthy babies | 3/16/22 | Musculoskeletal |
| Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | SMN2 splicing modulator | Spinal muscular atrophy | Long-term 3-year data from Sunfish study confirmed sustained increase in motor function with decreased adverse event; well-tolerated over the 3-year time period; improvement (change ?3 points) or stabilization (change ?0 points) in Evrysdi for 24 months vs. untreated comparator group (p=0.025 and p=0.002, respectively) | 3/16/22 | Musculoskeletal |
| Santhera Pharmaceuticals AG, of Pratteln, Switzerland, and Reveragen Biopharma Inc., of Rockville, Md. | Vamorolone | Dissociative agonist | Duchenne muscular dystrophy | Results from VISION-DMD study met the primary endpoint at week 24 at 6 mg/kg/day; sustained efficacy across multiple endpoints over 48 weeks; well-tolerated with a favorable safety profile | 3/15/22 | Musculoskeletal |
| Xalud Therapeutics Inc., of New York | XT-150 | Locally-injectable plasmid DNA gene therapy expressing IL-10v | Osteoarthritis of the knee and neuropathic pain | First patient dosed | 3/2/22 | Musculoskeletal |
| Bridgebio Pharma Inc., of San Francisco | BBP-418 | Glycosylation substrate prodrug | Limb-girdle muscular dystrophy type 2i | Results from phase II study (n=12) showed 43% increase in the ratio of glycosylated alpha-dystroglycan (?DG) to total ?DG from baseline, measured across all 3 dosing cohorts; 70% reduction in creatine kinase and 77% reduction after 180 days; increase in velocity in the 10-meter walk test; well-tolerated; no treatment-related serious adverse events | 3/14/22 | Musculoskeletal |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | Selective p38?/? mitogen activated protein kinase (MAPK) inhibitor | Facioscapulohumeral muscular dystrophy | Data showed losmapimod preserved or improved function in patients measured by Reachable Workspace | 3/14/22 | Musculoskeletal |
| Anavex Life Sciences Corp., of New York | ANAVEX 2-73 (blarcamesine) | Oral sigma-1 receptor activator | Parkinson’s disease with dementia | Results from phase II study showed MDS-UPDRS part I, II, III, IV sub-scores improved, including substantial majority of individual items between 71% and 92%; SIGMAR1 mRNA expression significantly increased in patients vs placebo (p=0.035); statistically significant improvements in CDR system cognitive domain of attention assessed by Choice Reaction Time (p = 0.039) and digital vigilance (p = 0.008) and CDR system episodic memory (p = 0.047); does not impair sleep and has a positive effect on REM sleep behavior disorder; well-tolerated in oral doses up to 50-mg | 3/15/22 | Neurology/Psychiatric |
| Apnimed Inc., of Cambridge, Mass. | AD-109 (atomoxetine + aroxybutynin) | Norepinephrine reuptake inhibitor + antimuscarinic | Obstructive sleep apnea | Study showed statistically significant and clinically meaningful difference from placebo in hypoxic burden (HB); median HB for participants on placebo was significantly higher than for patients on the high dose (p<0.001) and low dose (p<0.01); statistically significant clinically meaningful median reduction in ahi [high (p<0.001) the (p<0.05)]; favorable safety at both doses< td> | 3/9/22 | 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 | Preliminary data from 57 patients who completed the study (n=77); top-line data expected in second quarter of 2022 | 3/20/22 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Lecanemab (BAN-2401) | Anti-amyloid beta protofibril antibody | Early Alzheimer’s disease | Results showed dose- and time-dependent change of central and blood amyloid and p-tau biomarkers; robust changes in A? 42/40 and p-tau181 | 3/21/22 | Neurology/Psychiatric |
| Cali Biosciences Co Ltd., of San Diego | CPL-01 | Extended-release injectable version of Naropin (ropivacaine hydrochloride) | Postoperative surgical pain | 14 participants who received CPL-01 showed mean AUC through 72 hours of wWOCF adjusted NRS-A of 286.8 with a trend towards significance vs. 13 subjects who received placebo (p=0.08); no meaningful safety findings; About two-thirds of CPL-01 subjects (9/14) required no opioids at all after the first 72 hours after the operation vs. 30% of patients given placebo | 3/21/22 | Neurology/Psychiatric |
| Cerevance Inc., of Boston | CVN-424 | Non-dopaminergic protein modulator | Parkinson's disease | Results showed CVN-424 met all safety objectives; well-tolerated; 1.3-hour reduction in OFF time compared to placebo at high dose | 3/31/22 | Neurology/Psychiatric |
| Cerevance Inc., of Boston | CVN-424 | Non-dopaminergic protein modulator | Parkinson's disease | At high dose, CVN-424 showed a 1.3-hour improvement in “off” time vs. placebo (p=0.042) at 4 weeks, accompanied by increase in “on” time without dyskinesia, without meaningful worsening of “on” time with dyskinesia; efficacy improved at 4 weeks vs. at 2 weeks, and daytime sleepiness as measured by the Epworth Sleepiness Scale was reduced compared to placebo; at lower dose, CVN-424 also demonstrated meaningful improvement in “off” time and “on” time without dyskinesia vs. placebo | 3/31/22 | Neurology/Psychiatric |
| Cortexyme Inc., of South San Francisco | Atuzaginstat (COR-388) | Lysine gingipain inhibitor; brain-penetrant oral small molecule targeting P. gingivalis infection | Mild to moderate Alzheimer’s disease | Results from Gain trial showed atuzaginstat inhibited lysine gingipain; 30% to 50% slowing of cognitive decline in the participants with high P. gingivalis load; numerical trends to benefit in tau biomarkers | 3/21/22 | Neurology/Psychiatric |
| Grifols SA subsidiary Araclon Biotech SL, of Zaragoza, Spain | Abvac-40 | Vaccine targeting the C terminus of A?40 | Alzheimer's disease | Top-line results showed study met the primary objectives on safety and efficacy; strong immune response in patients | 3/15/22 | Neurology/Psychiatric |
| HMNC Brain Health of Munich, Germany | KET-01 | Prolonged-release formulation of ketamine | Treatment-resistant depression | Top-line results showed larger improvement on the montgomery-asberg depression rating scale (MADRS) compared with placebo (p=0.1499) at 240 mg/day; safe and well-tolerated; scores on the clinician-administered dissociative states scale (CADSS) similar to those for placebo; 7 days suggestive of rapid onset of efficacy | 3/31/22 | Neurology/Psychiatric |
| HMNC Brain Health, of Munich, Germany, and Develco Pharma Schweiz AG, of Pratteln, Switzerland | KET-01 | Oral, prolonged-release formulation of ketamine | Treatment-resistant depression | Top-line results showed positive trend in efficacy; after 15 days, highest dose of 240 mg/day was associated with larger improvement on Montgomery-Åsberg Depression Rating Scale vs. placebo (?=-4.99, p=0.1499); safe and well-tolerated, with limited dissociative side effects, and scores on Clinician-Administered Dissociative States Scale similar to those for placebo | 3/31/22 | Neurology/Psychiatric |
| Incannex Healthcare Ltd., of Docklands, Australia | IHL-42X | Cannabinoid combination product | Obstructive sleep apnea | Preliminary data showed 20% of trial participants experienced reduction in apnea hypopnea index (AHI) of greater than 80% with at least 1 treatment compared to baseline; 60% of trial participants experienced a reduction in AHI of greater than 50% during at least 1 treatment compared to baseline; well-tolerated; reduced disease severity, improved sleep quality and increased quality of life | 3/10/22 | Neurology/Psychiatric |
| Neuroplast Beheer BV, of Geleen, Netherlands | Neuro-Cells | Non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells from patient's own bone marrow | Traumatic spinal cord injury | First patient enrolled | 3/29/22 | Neurology/Psychiatric |
| NLS Pharma Ltd., of Stans, Switzerland | Quilience | Mazindol extended release | Narcolepsy | Interim top-line data (n=60) showed drug was safe and well-tolerated; no serious adverse events; 39% reduction in Epworth Sleepiness Scale (ESS) from baseline (-4.3 points placebo-adjusted); mean decrease of 7.3 points on the ESS compared to a 3-point decrease for patients on placebo | 3/16/22 | Neurology/Psychiatric |
| Novamind Inc., of Toronto | Psilocybin | Psychedelic medicines | Major depressive disorder | Completed first patient randomization | 3/9/22 | Neurology/Psychiatric |
| Pharmather Holdings Ltd., of Toronto | Ketamine | N-methyl-D-aspartate receptor-modulating agent | Levodopa-induced dyskinesia | Top-line data showed 100% of patients treated with ketamine demonstrated a reduction in dyskinesias; well-tolerated with no serious adverse events | 3/23/22 | Neurology/Psychiatric |
| PTC Therapeutics Inc, of South Plainfield, N.J. | PTC-518 | Orally bioavailable small-molecule splicing modifier | Huntington's disease | Initiated phase II study; designed in 2 parts; an initial 12-week placebo-controlled phase; followed by a 9-month placebo-controlled phase | 3/30/22 | Neurology/Psychiatric |
| Recursion Pharmaceuticals Inc., of Salt Lake City | REC-994 | Orally bioavailable small molecule | Cerebral cavernous malformation | First patient enrolled | 3/18/22 | Neurology/Psychiatric |
| Sage Therapeutics Inc., of Cambridge, Mass. | SAGE-718 | Positive allosteric modulator of N-methyl-D-aspartate receptor | Mild cognitive impairment (MCI) due to Parkinson’s disease | Results from Paradigm study showed SAGE-718 was associated with improved performance at day 14 compared to baseline; improved performance on tests of learning and memory; sustained effects and improving trends were seen out to day 28 for assessments completed at the follow-up visit; well-tolerated; no serious adverse events; no treatment emergent adverse events | 3/15/22 | Neurology/Psychiatric |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | Oxytocin (TNX-1900) | Intranasal potentiated oxytocin; drug-device combination product | Binge eating disorder | Investigator-initiated phase II study expected to start in the second half of 2022 | 3/7/22 | Neurology/Psychiatric |
| Tryp Therapeutics Inc., of San Diego | TRP-8802 | Synthetic psilocybin | Binge eating disorder | First patient enrolled | 3/23/22 | Neurology/Psychiatric |
| Vertex Pharmaceuticals Inc., of Boston | VX-548 | NaV1.8 inhibitor | Acute pain | Results from bunionectomy and abdominoplasty trial showed rapid, statistically significant and clinically meaningful improvement in the primary endpoint of the time-weighted Sum of Pain Intensity Difference over 48 hours; well-tolerated; mild to moderate adverse events; no serious adverse events | 3/31/22 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-2191 | Intravitreal methotrexate 0.8% | Retinitis pigmentosa | Initiated phase II study; to evaluate the safety and tolerability; trial expected to enroll 8 patients; top-line data expected in the second half of 2022 | 3/8/22 | Ocular |
| Aramis Biosciences Inc., of Boston | A-197 | Topical immunomodulatory agent | Dry eye disease | The first patient was dosed in a phase II trial | 3/2/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | APX-3330 | Oral Ref-1 inhibitor | Diabetic retinopathy | Completed enrollment of 103 patients | 3/16/22 | Ocular |
| Ribomic Inc., of Tokyo | RBM-007 | Oligonucleotide-based aptamer targeting FGF2 | Wet age-related macular degeneration | Results showed positive trend in best corrected visual acuity and central subfield thickness (improved by 200 micron); 1 patient had improvement in retinal anatomy; subject gained 12 and 15 letters at 3 months and 4 months, respectively | 3/23/22 | Ocular |
| Vivavision Biotech Inc., of Shanghai | VVN-001 | Second-generation LFA-1 inhibitor | Dry eye disease | Top-line data showed 1% and 5% treatment group improvement in primary efficacy endpoint of inferior corneal staining; statistically and clinically significant improvement; dose and treatment duration-related improvement relative to vehicle; safe and well-tolerated; no significant treatment-related safety findings | 3/22/22 | Ocular |
| Aristea Therapeutics Inc., of San Diego | RIST-4721 | CXCR2 antagonist | Palmoplantar pustulosis | Dosed first patient | 3/28/22 | Other/Miscellaneous |
| Concentric Analgesics Inc., of San Francisco | Vocacapsaicin (CA-008) | Vanilloid VR1 agonist | Open laparotomy for ventral hernia repair | Study showed vocacapsaicin administered during surgery at a dose of 24 mg (0.3 mg/mL solution) reduced pain after coughing by 46% compared to placebo control during the first 96 hours after surgery (p=0.02); pain with ambulation was reduced by 35% (p=0.08); positive trends were seen in the reduction of pain at rest and reduction of opioid consumption; safe and well-tolerated compared to control group | 3/30/22 | Other/Miscellaneous |
| Evolus Inc., of Newport Beach, Calif. | Jeuveau/prabotulinumtoxinA | Neurotoxins | Treatment of glabellar lines | First patient enrolled | 3/31/22 | Other/Miscellaneous |
| Kalvista Pharmaceuticals Inc., of Cambridge, Mass. | KVD-900 | Small molecule protease inhibitor | Hereditary angioedema | Data show rapid suppression of plasma kallikrein activity after KVD-900 administration and its relationship with symptomatic relief | 3/2/22 | Other/Miscellaneous |
| Mydecine Innovations Group, of Denver | MYCO-001 | Small molecule; neuroprotectant | Smoking cessation | Received conditional approval from the institutional review board for its multisite phase IIb study | 3/24/22 | Other/Miscellaneous |
| Saniona AB, of Copenhagen, Denmark | Tesomet | Fixed-dose combination therapy of tesofensine and metoprolol | Hypothalamic obesity and Prader-Willi syndrome | Voluntarily paused phase II study; not related to the safety or efficacy; due to funding limitations | 3/29/22 | Other/Miscellaneous |
| Aldeyra Therapeutics Inc., Lexington,Mass. | ADX-629 | Orally administered RASP modulator | Asthma | Data showed asthma symptom scores and sputum eosinophil cell counts were numerically reduced in 8 mild asthma patients; statistically significant reductions in plasma levels of the pro-inflammatory cytokines IL-5 (p=0.02) and TNF? (p<0.0001); numerical reductions in plasma levels of malondialdehyde< td> | 3/29/22 | Respiratory |
| Knopp Biosciences LLC, of Pittsburgh | Dexpramipexole | Selective inhibitor of eosinophil maturation | Asthma | Presented new phase II data demonstrating that oral dexpramipexole improves airflow obstruction as measured by forced expiry volume largely through its effect on forced vital capacity | 3/1/22 | Respiratory |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody designed to block TNF-like ligand 1A | Systemic sclerosis-associated interstitial lung disease | First patient enrolled; top-line results expected in first half of 2024 | 3/30/22 | Respiratory |
| Trevi Therapeutics Inc., of New Haven, Conn. | Haduvio (nalbuphine ER) | Opioid receptor mu antagonist; opioid receptor kappa agonist | Idiopathic pulmonary fibrosis | Completed enrollment and no additional recruitment was required; trial achieved statistical significance in the interim analysis; 40 subjects enrolled in the study; full reports expected in the third quarter of 2022 | 3/15/22 | Respiratory |
| Hibercell Inc., of New York | Odetiglucan | Dectin-1, pattern recognition receptor agonist | Metastatic, hormone-refractory breast cancer | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 4/28/22 | Cancer |
| Immunitybio Inc., of Culver City, Calif. | Anktiva (N-803, Nant cancer vaccine) | IL-15 receptor agonist | Non-small-cell lung cancer | First participant enrolled in Lung-Map trial in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 4/25/22 | Cancer |
| IO Biotech Inc., of New York | IO-102/IO-103 | Dual IDO-derived peptide vaccine/PD-L1-derived peptide vaccine | Solid tumors | Dosed first patient in IOB-022/KN-D38 study testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in previously untreated patients with 3 different tumor types – non-small-cell lung cancer, squamous cell carcinoma of the head and neck and urothelial bladder cancer – in first-line setting | 4/12/22 | Cancer |
| Maat Pharma SA, of Lyon, France | MaaT-013 | Off-the-shelf, standardized, pooled-donor, Microbiome Ecosystem Therapy | Melanoma | Initiation of phase IIa study with combination of immune checkpoint inhibitors | 4/7/22 | Cancer |
| Oncternal Therapeutics Inc., of San Diego | Zilovertamab | Anti-ROR1 monoclonal antibody | Ewing sarcoma | Discontinued enrollment of phase I/II trial; planning to initiate phase III study in third quarter of 2022 | 4/13/22 | Cancer |
| Phosplatin Therapeutics Inc., of New York | PT-112 | Small-molecule conjugate of pyrophosphate | Recurrent thymic epithelial tumor | First patient treated | 4/13/22 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Giredestrant | Selective estrogen receptor (ER) degrader | ER-positive, HER2-negative locally advanced or metastatic breast cancer | Trial of giredestrant vs. physician's choice endocrine monotherapy found it missed PFS endpoint in Acelera trial; still being being investigated in further trials for patients with 1L metastatic breast cancer and early breast cancer | 4/24/22 | Cancer |
| Surface Oncology Inc., of Cambridge, Mass. | SRF-388 | Antibody against IL-27 | Hepatocellular carcinoma and non-small-cell-lung cancer | Initiation of 2 phase II study | 4/14/22 | Cancer |
| Syneurx International Corp., of Taiwan | Pentarlandir | Antiviral candidate | COVID-19 | Completed enrollment | 4/13/22 | Cancer |
| Verrica Pharmaceuticals Inc., of West Chester, Pa. | LTX-315 | Oncolytic peptide | Basal cell carcinoma | First patient dosed | 4/5/22 | Cancer |
| Zymeworks Inc., of Vancouver, British Columbia, and Beigene Ltd., of Cambridge, Mass. and Beijing | Zanidatamab | Bispecific antibody binding 2 non-overlapping epitopes of HER2 | Advanced or metastatic HER2-amplified biliary tract cancers | Completed enrollment in HERIZON-BTC-01 trial; results expected in early 2023 | 4/28/22 | Cancer |
| Bridgebio Pharma Inc., of Palo Alto, Calif. | Acoramidis (AG-10) | Stabilizes tetrameric transthyretin | Transthyretin amyloid cardiomyopathy | In the open-label extension study, median change from baseline at month 30 in N-terminal pro-brain natriuretic peptide (NT-proBNP) was -437 pg/mL; 68% of participants had NT-proBNP levels below their baseline; mean serum TTR levels increased from baseline by approximately 41% at month 30; top-line data from ongoing phase III trial of acoramidis expected in mid-2023 | 4/3/22 | Cardiovascular |
| Gossamer Bio Inc., of San Diego | Seralutinib (GB-002) | Inhaled PDGFR, CSF1R, c-KIT inhibitor | Pulmonary arterial hypertension | Target enrollment reached in Torrey study; top-line data expected in the fourth quarter of 2022 | 4/25/22 | Cardiovascular |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa., and San Diego | Bentracimab | Monoclonal antibody antigen-binding fragment targeting the antiplatelet activity of ticagrelor | Uncontrolled major or life-threatening bleeding | In the phase IIb study, bentracimab significantly restored platelet function within 5 minutes of administration (p<0.001) in older volunteers (50-80 years old) pretreated with ticagrelor and low-dose aspirin< td> | 4/4/22 | Cardiovascular |
| Respira Therapeutics Inc., of Palo Alto, Calif. | RT234-PAH (vardenafil, dry powder inhaler treatment) | PDE5i vasodilator | Pulmonary arterial hypertension | First patient in U.S. dosed in multicenter phase IIb VIPAH-PRN trial | 4/5/22 | Cardiovascular |
| Windtree Therapeutics Inc., of Warrington, Pa. | Istaroxime | Na+ K+ ATPase inhibitor; sarco endoplasmic calcium ATPase 2a stimulator | Cardiogenic shock | Top-line data showed study met its primary endpoint in systolic blood pressures profile over 6 hours compared to the control group | 4/20/22 | Cardiovascular |
| Edesa Biotech Inc., of Toronto | EB-01 | non-steroidal anti-inflammatory compound ;sPLA2 inhibitor | Chronic allergic contact dermatitis | Completed patient recruitment ahead of schedule | 4/28/22 | Dermatologic |
| Lakewood-Amedex Inc., of Sarasota, Fla. | Bisphosphocin Nu-3 | Antimicrobial | Diabetic foot ulcers | Started second phase II study to test topically applied gel in type 1 or type 2 diabetes mellitus patients with chronic DFU | 4/5/22 | Dermatologic |
| Meji Seika Pharma Co. Ltd., of Tokyo | ME-3183 | Selective PDE4 inhibitor | Plaque psoriasis | First patient dosed | 4/7/22 | Dermatologic |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-ANG3 | Double-stranded interference RNA trigger targeting angiopoietin-like protein 3 | Homozygous familial hypercholesterolemia | First patient dosed | 4/26/22 | Endocrine/Metabolic |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Avexitide | GLP-1 receptor antagonist | Congenital hyperinsulinism | Data showed 76% reduction in fasting hypoglycemia in the mid-dose (0.44 mg/kg) group and an 84% reduction in hypoglycemia in the high-dose (0.6 mg/kg) group; 20% increase in fasting glucose at mid-dose; 28% increase in glucose after a meal at high dose; 30% increase in glucose after protein challenge | 4/25/22 | Endocrine/Metabolic |
| Hightide Therapeutics Inc., of Shenzhen, China | HTD-1801 | Ionic salt of berberine and ursodeoxycholic acid | Type 2 diabetes | First patient dosed | 4/28/22 | Endocrine/Metabolic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif., and Astrazeneca plc, of Cambridge, U.K. | ION-449 (AZD-8233) | Antisense targeting proprotein convertase subtilisin/kexin type 9 | High-risk hypercholesterolemia | In the phase IIb Etesian study, ION-449 reduced serum LDL-cholesterol levels from baseline by 73% and 79% for the 50-mg and 90-mg doses, respectively, compared to a 2% reduction for placebo; PCSK9 levels were reduced by 89% and 94% for the 50-mg and 90-mg doses, respectively, compared to a 5% decline for placebo | 4/4/22 | Endocrine/Metabolic |
| Lumos Pharma Inc., of Austin, Texas | LUM-201 | Oral growth hormone secretagogue | Pediatric growth hormone deficiency | Reached 50% enrollment in the Oragrowht210 study; interim data expected by the end of 2022; complete 6-month, primary outcome data anticipated in the second half of 2023 | 4/11/22 | Endocrine/Metabolic |
| Medicinova Inc., of La Jolla, Calif. | MN-001 (tipelukast) | Small molecule compound with anti-inflammatory and anti-fibrotic activity | Non-alcoholic fatty liver disease, type 2 diabetes mellitus and hypertriglyceridemia | FDA completed review of the protocol for the 40-patient study; co-primary endpoints are change from baseline in liver fat content measured by MRI proton density fat fraction at week 24 and change from baseline in fasting serum triglycerides at week 24; secondary endpoints include safety and tolerability and changes in HDL-C, LDL-C and total cholesterol | 4/11/22 | Endocrine/Metabolic |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor | Reducing low-density lipoprotein cholesterol | Dose administration for first patient in Rose2 trial | 4/5/22 | Endocrine/Metabolic |
| Abivax SA, of Paris | ABX-464 | Rev protein modulator | Ulcerative colitis | Interim results (n=217) showed clinical remission of 55.3%; endoscopic improvement of 61.8%; endoscopic remission was 33.6% with clinical response of 80.6%; 73.1% maintained clinical remission at week 48; good safety and tolerability profile; planning to launch pivotal phase III study | 4/6/22 | Gastrointestinal |
| Applied Molecular Transport Inc., of South San Francisco | AMT-101 | Fusion protein comprising human interleukin 10 fused to a non-toxic immunogenic fragment of cholix exotoxin | Chronic pouchitis | Top-line data showed Fillmore study achieved prespecified efficacy endpoints; 36.4% of patients achieved stool frequency response; reduction of ? 3 stools and ? 30% from baseline; symptomatic improvements in fecal urgency, incontinence and abdominal cramps; safe and well-tolerated; achieved clinically meaningful responses in stool frequency and histologic healing in both 3-mg and 10-mg dosage groups; independent data monitoring committee recommends phase III study | 4/25/22 | Gastrointestinal |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Halo-Lido | Topical formulation of halobetasol and lidocaine | Hemorrhoids | First patient enrolled | 4/26/22 | Gastrointestinal |
| Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | Orally active PPAR-delta agonist | Primary biliary cholangitis | The 52-week study's interim primary efficacy analysis of serum alkaline phosphatase reductions from baseline to week 8 were 26%, 33% and 41% for the 2-mg, 5-mg and 10-mg doses, respectively (all p<0.005); 5 10 pruritus visual analogue scale score decreased for patients taking mg and mg< td> | 4/4/22 | Gastrointestinal |
| Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | Oral, selective PPAR? agonist | Primary biliary cholangitis | Results, published in the Journal of Hepatology, showed interim analysis of ALP change from baseline at week 8 found treatment provided 26%, 33% and 41% reductions for 2-mg, 5-mg and 10-mg doses, respectively (all p<0.005); responses maintained or improved at week 52, which included dose escalation in 91% and 80% of the 2-mg 5-mg cohorts, respectively; composite biochemical response (alp <1.67×uln, ?15% alp decrease from baseline, normal total bilirubin) rates were 64%, 53% 67%, normalization 9%, 13% 33%, pruritus visual analogue scale score also decreased 10-mg cohorts< td> | 4/4/22 | Gastrointestinal |
| Gannex Pharma Co. Ltd., of Shanghai, a wholly owned company of Ascletis Pharma Inc. | ASC-42 | Nonsteroidal, selective FXR agonist | Primary biliary cholangitis | First patient dosed; 100-patient study expected to be completed by end of 2022 | 4/10/22 | Gastrointestinal |
| Gossamer Bio Inc., of San Diego | GB-004 | Hypoxia-inducible factor 1-alpha stabilizer | Ulcerative colitis | Top-line data showed study did not meet primary or secondary endpoints at week 12; safety and tolerability profile consistent; study terminated for lack of treatment benefit | 4/25/22 | Gastrointestinal |
| Protagonist Therapeutics Inc., of Newark, Calif. | PN-943 | Oral, gut-restricted, alpha-4-beta-7-integrin antagonist | Ulcerative colitis | Top-line data from Ideal study achieved 27.5% clinical remission with a delta of 13% vs. placebo p=0.08; 16% delta in biologic-naïve population (p=0.04); statistically significant differences of key secondary endpoints in histologic remission/improvement and endoscopic improvement; 450-mg twice-daily dose did not meet the prespecified primary endpoint; similar safety analysis at 150 mg twice daily | 4/25/22 | Gastrointestinal |
| Bioinvent International AB, of Lund, Sweden | HMI-115 | Anti-prolactin receptor antibody | Endometriosis | First patient enrolled | 4/28/22 | Genitourinary/Sexual Function |
| Cellectar Biosciences Inc., of Florham Park, N.J. | Iopofosine I-131 (CLR-131) | Phospholipid-drug conjugate | Waldenstrom’s macroglobulinemia | Data monitoring committee completed its planned futility/efficacy assessment of phase IIb study; recommended continuation of the trial as planned | 4/26/22 | Hematologic |
| Imara Inc., of Boston | Tovinontrine (IMR-687) | Small-molecule inhibitor of PDE9 | Sickle cell disease | Interim analysis from phase IIb Ardent trial showed no statistically significant data for annualized vaso-occlusive crisis rate vs. placebo and no meaningful difference observed in fetal hemoglobin response; trial to be discontinued | 4/5/22 | Hematologic |
| Imara Inc., of Boston | Tovinontrine (IMR-687) | Small-molecule inhibitor of PDE9 | Beta-thalassemia | Interim analysis from phase IIb Forte trial showed no meaningful improvements observed in most disease-related biomarkers, including total hemoglobin; trial to be discontinued | 4/5/22 | Hematologic |
| Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Small-molecule inhibitor of delta isoform of 110 kDa catalytic subunit of class IA PI3K | Activated phosphoinositide 3-kinase delta syndrome | Co-primary endpoint data from pivotal phase II/III study showed, in index lymphadenopathy lesions, a statistically significant adjusted mean change in log10 transformed sum of product of diameters (SPD) of -0.30 vs. -0.06 for placebo (p=0.0012); from a baseline level of ?48%, an increase of 34.76% in proportion of naïve B cells vs. -5.37% decrease for placebo (p?0.0001) | 4/1/22 | Immune |
| Pharming Group NV, of Leiden, the Netherlands, and Novartis AG, of Basel, Switzerland | Leniolisib | Small-molecule phosphoinositide 3-kinase delta inhibitor | Activated phosphoinositide 3-kinase delta syndrome | At day 85 of the phase III portion of the phase II/III study, patients who received leniolisib had an index lymphadenopathy lesions transformed sum of product of diameters of -0.30 compared with -0.06 for patients who received placebo (p=0.0012); there was an increase of 34.76% in the proportion of naïve B cells in patients who received leniolisib compared to a -5.37% decrease in patients who received placebo (p?0.0001) | 4/1/22 | Immune |
| AB Science SA, of Paris | Masitinib + isoquercetin | Tyrosine kinase inhibitor | COVID-19 | DSMB recommended continuing the study without restrictions in moderate patients | 4/28/22 | Infection |
| Akston Biosciences Inc., of Beverly, Mass. | AKS-452 | Vaccine containing the receptor binding domain of SARS-CoV2 | COVID-19 prophylaxis | First volunteer dosed in India | 4/7/22 | Infection |
| Bergenbio ASA, of Bergen, Norway | Bemcentinib | Oral AXL kinase inhibitor | Hospitalized COVID-19 | Data in combination with standard of care therapy showed study met primary efficacy endpoint; 90% of patients treated with bemcentinib + SoC experienced a statistical significant clinical response by day 29 (median 7 days) compared to SoC ; 69% (median 9.5 days); statistically significant improvements for key secondary endpoints; 97% alive compared to 81% for SoC at day 29; well-tolerated; no clinically relevant safety signals | 4/26/22 | Infection |
| Calcimedica Inc., of La Jolla, Calif. | Auxora | Small-molecule CRAC channel inhibitor | Critical COVID-19 pneumonia | Results showed time to recovery was 7 days vs. 10 days (p=0.0979) for patients who received Auxora vs. placebo, respectively; mortality was 7.7% with Auxora vs. 17.6% placebo (p=0.0165) at day 30; mortality was 13.8% with Auxora vs. 20.6% with placebo (p=0.1449) at day 60; serious adverse events occurred in 24.1% of patients treated with Auxora vs. 35% of patients receiving placebo (p=0.0616) | 4/14/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Chengdu, China | SCB-2019 (CpG 1018/Alum) | COVID-19 S-Trimer subunit vaccine; vaccine comprising recombinant SARS-CoV-2 spike glycoprotein | COVID-19 prophylaxis | Data showed durable protection through approximately 6 months following primary vaccination; robust immune responses in neutralizing Omicron and other variants of concern after a booster dose; efficacy against any SARS-CoV-2 strain was maintained at 100% for severe COVID-19 and 95% against hospitalizations due to COVID-19; efficacy against any SARS-CoV-2 strain was maintained at 100% for severe COVID-19 and 100% against hospitalizations in elderly population (>60 years of age) | 4/19/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Chengdu, China | SCB-2019 (CpG 1018/Alum) | COVID-19 S-Trimer subunit vaccine; vaccine comprising recombinant SARS-CoV-2 spike glycoprotein | COVID-19 prophylaxis | Data from the Lancet Infectious Disease showed 89.7% cumulative protective effect in individuals previously infected with SARS-CoV-2 after 1 dose; 93.8% after 2 doses against COVID-19 of any severity; 49.9% after 1 dose and 64.2% after 2 doses in incremental risk reduction against COVID-19 vs. placebo; favorable safety profile with infrequent severe and serious adverse events | 4/20/22 | Infection |
| Deinove SA, of Paris | DNV-3837 | Prodrug of DNV-3681; narrow-spectrum, hybrid oxazolidinone-quinolone synthetic antibiotic | Clostridioides difficile infection | Second part of trial will enroll patients at 5 new sites in Canada in addition to the sites in the U.S. used in the first part of the study | 4/7/22 | Infection |
| Direct Biologics LLC, of Austin, Texas | Exoflo | Human allogeneic bone marrow mesenchymal stem or stromal cell-derived exosomes | COVID-19 related acute respiratory distress syndrome | Top-line data showed robust safety and efficacy role; reduction in 60-day mortality rate of 37.6% in comparison to placebo; no reportable adverse events | 4/6/22 | Infection |
| First Wave Biopharma Inc., of Boca Raton, Fla. | FW-COV | An oral formulation of niclosamide | COVID-19 | The efficacy endpoint, removing SARS-CoV-2 from the digestive tract, did not demonstrate statistical significance compared to placebo | 4/29/22 | Infection |
| Lumen Bioscience Inc., of Seattle | LMN-201 | Cocktail of 2 classes of therapeutic proteins | C. difficile infection | Completed phase I study; initiating phase II study in spring 2022 | 4/18/22 | Infection |
| Medicinova Inc., of La Jolla, Calif. | MN-166 (ibudilast) | Small-molecule compound that inhibits PDE4 | Hospitalized COVID-19 patients at risk for developing acute respiratory distress syndrome | Completed enrollment | 4/13/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.211 | Multivalent candidate combining the Beta-specific variant and mRNA-1273 | COVID-19 prophylaxis | Data from 50-µg booster dose showed superiority against Beta, Delta and Omicron variants of concern one month after administration; well-tolerated with a reactogenicity profile comparable to a booster dose of mRNA-1273 at the 50 µg dose level | 4/19/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Comirnaty (BNT-162b2; tozinameran) | mRNA vaccine | COVID-19 | Results from 10-mcg booster dose in healthy children 5 through 11 years of age showed 36-fold increase in SARS-CoV-2 Omicron neutralizing titers; increased neutralizing antibodies by 6-fold against the SARS-CoV-2 wild-type strain | 4/14/22 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | S-217622 | 3CL protease inhibitor | COVID-19 | Results showed rapid clearance of SARS-CoV-2 virus; proportion of patients with positive viral titer decreased by about 90% vs. placebo; shortened infectious virus shedding by 1-2 days vs. placebo; significant reduction in viral RNA on days 2, 4, 6 and 9 vs. placebo; no significant difference in total score of 12 COVID-19 symptoms between treatment arms; post-hoc showed improvement in composite score of 5 respiratory and feverish symptoms; well-tolerated | 4/23/22 | Infection |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy | Kidney transplant | Data showed low rate of COVID-19 infection observed in vaccinated; durably chimeric patients off immunosuppression; no evidence of acute kidney injury or impaired renal function in FCR-001-treated patients with COVID-19 infection; no patient lost chimerism | 4/6/22 | Infection |
| Valneva SE, of Saint Herblain, France, and Pfizer Inc., of New York | VLA-15 | Multivalent protein subunit vaccine targets the outer surface protein A of Borrelia burgdorferi | Lyme disease | Results showed strong immunogenicity profile observed in study participants aged 5-17 years, 1 month after the primary vaccination series; similar safety profiles to adult; no vaccine-related serious adverse events | 4/26/22 | Infection |
| Veru Inc., of Miami | Sabizabulin (VERU-111) | Cytoskeleton disruptor | COVID-19 patients at high risk for acute respiratory distress syndrome | Treatment resulted in 82% relative reduction in deaths (p=0.0442) in intent-to-treat population; treatment also resulted in reduction in mean days in the ICU from 9.6 ± 12.40 days in placebo group to 2.6 ± 5.78 days (p=0.0261) in sabizabulin group, a 73% relative reduction; reduction in mean days on mechanical ventilation from 5.1 ± 11.24 days in placebo group to 1.2 ± 6.06 days (p=0.1437), a 78% relative reduction; safe and well-tolerated | 4/25/22 | Infection |
| Abionyx Pharma SA, of Toulouse, France | CER-001 | Negatively charged lipoprotein particle containing human recombinant apoA-I, the natural HDL protein and natural phospholipids sphingomyelin and dipalmitoylphosphatidylglycerol | Septic patients at high risk of developing acute kidney injury | Interim results showed rapidly improved biomarkers of inflammation such as leukocytosis and endothelial dysfunction; well-tolerated at all dose levels; no treatment-related serious side effects; rapid reversal of cytokine storm in septic patients | 4/7/22 | Inflammatory |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-220 | Delivers 6 months of continuous anti-inflammatory medication | Chronic rhinosinusitis | First patient treated; top-line results of part 1 Beacon study expected around year-end | 4/25/22 | Inflammatory |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Local, intra-nasal, anti-inflammatory therapy | Chronic rhinosinusitis | Data showed participants treated with 7500-?g dose of LYR-210 reported dose-dependent global symptom improvement (p<0.05) in each snot-22 subdomain vs. control at week 24; decreased rhinologic, ear facial, extra-nasal psychological dysfunction, and sleep dysfunction domain scores by an average of 10, 8, 5.2, 15.2, 10.3 points from baseline, respectively< td> | 4/29/22 | Inflammatory |
| Navidea Biopharmaceuticals Inc., of Dublin, Ohio | Tc99m Tilmanocept/Lymphoaim/ Lymphoseek | Imaging agent | Rheumatoid arthritis joint inflammation | Preliminary data (n=11) showed uptake in the hands and wrists of patients is proportional to the amount of macrophage involvement in an individual patient; 7 patients had had relatively lower levels of uptake | 4/20/22 | Inflammatory |
| ABVC Biopharma Inc., of Fremont, Calif. | PDC-1421 | Capsule is a botanical investigational new drug containing the extract of Radix Polygalae (Polygala tenuifolia Willd.) | Attention-deficit hyperactivity disorder | Completed site initiation visits; intiated enrollment | 4/28/22 | Musculoskeletal |
| Neurosense Therapeutics Ltd., of Cambridge, Mass. | PrimeC | Extended-release oral formulation composed of a fixed-dose combination of ciprofloxacin and celecoxib | Amyotrophic lateral sclerosis | Dosed first healthy volunteer in the study comparing the bioavailability of PrimeC to the bioavailability of co-administered ciprofloxacin tablets and celecoxib capsules; results expected in the third quarter of 2022 | 4/11/22 | Musculoskeletal |
| Ultragenyx Pharmaceutical Inc., of Novato, Calif. | UX-143 (setrusumab) | Fully human monoclonal antibody that inhibits sclerostin | Osteogenesis imperfecta | First patient dosed | 4/20/22 | Musculoskeletal |
| Acadia Pharmaceuticals Inc., of San Diego | ACP-044 | Redox modulator; non-opioid | Post-operative pain | Top-line data showed primary endpoint not achieved; difference of -10.5 points compared to placebo (p = 0.1683; effect size = 0.219; mild to moderate adverse events; no serious events | 4/19/22 | Neurology/Psychiatric |
| Aeon Biopharma Inc., of Newport Beach, Calif. | ABP-450 (prabotulinumtoxinA) | Botulinum toxin complex | Cervical dystonia | Patient enrollment for study completed; top-line data expected in the second half of 2022 | 4/11/22 | Neurology/Psychiatric |
| Alzamend Neuro Inc., of Atlanta | AL-001 | Lithium-salicylate-L-proline engineered ionic co-crystal | Mild to moderate Alzheimer’s disease | Study to evaluate the safety and tolerability of multiple-dose, steady-state conditions of AL-001 is expected to start enrollment in May 2022 | 4/11/22 | Neurology/Psychiatric |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 | Sodium phenylbutyrate and taurursodiol | Amyotrophic lateral sclerosis and Alzheimer’s disease | Safety and tolerability data from Centaur and Pegasus studies showed adverse event incidence generally similar between treatment and placebo groups | 4/2/22 | Neurology/Psychiatric |
| Aptinyx Inc., of Evanston, Ill. | NYX-2925 | Oral NMDA receptor positive allosteric modulator | Painful diabetic peripheral neuropathy | Study did not achieve statistically significant separation from placebo on the primary endpoint; well-tolerated; no safety issues | 4/7/22 | Neurology/Psychiatric |
| Athersys Inc., of Cleveland, and Healios K.K., of Tokyo | Multistem (invimestrocel, HLCM-051) | Bone marrow-derived regenerative stem cell therapy | Ischemic stroke | Last patient in the phase II/III Treasure study completed the 365-day follow-up visit; top-line results anticipated in May 2022 | 4/4/22 | Neurology/Psychiatric |
| Corcept Therapeutics Inc., of Menlo Park, Calif. | Miricorilant | Cortisol modulator | Weight gain associated with antipsychotic medication | Completed enrollment in Gratitude trial; data expected in fourth quarter 2022 | 4/5/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-810 | Prodrug of the bioactive lipid palmitoylethanolamide | Diabetic peripheral neuropathic pain | Study did not achieve statistically significant separation from placebo on the primary endpoint, defined as change from baseline to week 4 in weekly average of daily pain score as measured by Pain Intensity Numerical Rating Scale | 4/25/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-810 | Prodrug of the bioactive lipid palmitoylethanolamide | Lumbosacral radicular pain | Study fully enrolled; top-line data expected in third quarter of 2022 | 4/25/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-155 | Neuroactive steroid GABAA receptor positive allosteric modulator | Major depressive disorder and perimenopausal depression | Enrollment in phase IIa study delayed due to interim analysis of pharmacokinetic data in phase Ib in photosensitive epilepsy patients | 4/25/22 | Neurology/Psychiatric |
| Inmune Bio Inc., of La Jolla, Calif. | Xpro-1595 (Xpro, pegipanermin) | Next-generation TNF inhibitor | Alzheimer's disease | First patient dosed | 4/13/22 | Neurology/Psychiatric |
| Neuraly Inc., of Germantown, Md. | NLY-01 | GLP-1R agonist | Parkinson's disease | Completed enrollment; enrolled 255 patients; top-line data expected in first half of 2023 | 4/19/22 | Neurology/Psychiatric |
| Neuroderm Ltd., of Rehovot, Israel | ND-0612 | Continuous delivery of carbidopa/levodopa via subcutaneous infusion | Parkinson's disease | The 114-patient phase IIb study demonstrated a positive long-term safety and tolerability profile; 95% of study participants who completed 1 year of treatment enrolled in the extension period | 4/4/22 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | SMN2 splicing modulator | Spinal muscular atrophy | Results from Firefish open label extension study showed 91% of infants treated with Evrysdi were alive after 3 years of treatment; continued to improve or maintain motor functions; overall continued reductions in serious adverse events | 4/29/22 | Neurology/Psychiatric |
| Sage Therapeutics Inc., of Cambridge, Mass. | SAGE-718 | Oral, positive allosteric modulator of NMDA receptor | Alzheimer’s disease | Data from Luminary study showed drug well-tolerated and associated with improvement on multiple tests of executive performance and learning and memory in patients with mild cognitive impairment and mild dementia due to Alzheimer’s disease; statistically significant improvement in Montreal Cognitive Assessment (+2.3 points vs. baseline) observed at day 28 | 4/1/22 | Neurology/Psychiatric |
| Sanbio Co Ltd., of Tokyo | SB-623 | Allogeneic modified and cultured adult bone marrow-derived mesenchymal stem cells | Chronic motor deficit from traumatic brain injury | Study met primary endpoint (p=0.04); demonstrated trend toward maintaining the improvement of function and activities of daily living in the final 1-year analysis; well-tolerated; no new safety concerns | 4/5/22 | Neurology/Psychiatric |
| Synaptogenix Inc. (formerly Neurotrope Bioscience Inc.), of New York | Bryostatin-1 | Activator of protein kinase C | Alzheimer's disease | Completed enrollment of 100 patients; top-line data expected in the fourth quarter of 2022 | 4/19/22 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Olinvyk (oliceridine) | G protein-selective mu-opioid receptor agonist | Acute pain | Results showed both Olinvyk and morphine achieved comparable levels of pain relief; statistically significantly reduced impact on respiratory function compared to morphine as measured by mean respiratory ventilation profiles over time (p < 0.0001); less respiratory depression over 6 hours; peak level was lower for Olinvyk compared to morphine, difference was not statistically significant (p > 0.05); respiratory function at the higher dose of Olinvyk rapidly returned toward baseline from 3 hours onwards (p < 0.05) | 4/20/22 | Neurology/Psychiatric |
| Tryp Therapeutics Inc., of San Diego | TRP-8802 | Synthetic psilocybin | Binge eating disorder | First patient dosed in combination with psychotherapy | 4/28/22 | Neurology/Psychiatric |
| Virios Therapeutics Inc., of Atlanta | IMC-1 (famciclovir + celecoxib) | Dual COX-2/COX-1 inhibitor | Fibromyalgia | Completed enrollment in phase IIb Fortress study; 425 patients enrolled; top-line data expected in September 2022 | 4/28/22 | Neurology/Psychiatric |
| ABVC Biopharma Inc., of Fremont, Calif. | Vitargus | Next-generation vitreous substitute | Ocular Endotamponade | Study planning to enroll 40 patients; to evaluate safety and effectiveness vs. ocular endotamponade with sulfur hexafluoride | 4/26/22 | Ocular |
| Addex Therapeutics Ltd., of Geneva | Dipraglurant | mGlu5 negative allosteric modulator | Blepharospasm | Completed patient enrollment | 4/13/22 | Ocular |
| Annexon Inc., of South San Francisco | ANX-007 | Complement C1q subcomponent inhibitor | Geographic atrophy | Completed enrollment in Archer trial; top-line data expected in the first half of 2023 | 4/7/22 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating prostaglandin analogue | Glaucoma | Results of the 433-patient study published in the Journal of Glaucoma; all 3 tested concentrations were statistically non-inferior to latanoprost with a dose response of improved intraocular pressure (IOP) lowering with each incremental concentration; 0.065% dose was statistically superior to latanoprost for IOP | 4/11/22 | Ocular |
| Orasis Pharmaceuticals Ltd., of Herzliya, Israel | CSF-1 | Eye drops; pilocarpine hydrochloride 0.4% | Presbyopia | Post-hoc analysis showed phase IIb study met its primary endpoint of sustained improvement; 47% of participants showed an improvement of 20/40 visual acuity or better consistently over an 8-hour period on day 15; sustained DCNVA improvements higher in the CSF-1 group vs. vehicle for participants assessed for sustained 20/40 vision across all time points (p<0.05) 20 40 in both monocular and binocular measurements; achieved sustained functional near vision of or better compared to vehicle when tested binocularly< td> | 4/25/22 | Ocular |
| Altimmune Inc., of Gaithersburg, Md. | Pemvidutide | Glucagon-like peptide-1/glucagon dual receptor agonist | Obesity | Enrolled first subject in the 48-week phase II Momentum study | 4/1/22 | Other/Miscellaneous |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Oral Factor D inhibitor | Complement-mediated diseases | Company has paused enrollment in clinical trials after elevated serum creatinine levels seen in some patients; no enrollement of new patients in Redeem-1 Redeem-2 and Renew studies | 4/8/22 | Other/Miscellaneous |
| Biorestorative Therapies Inc., of Melville, N.Y. | BRTX-100 | Product formulated from autologous cultured mesenchymal stem cells | Chronic lumbar disc disease | Selected 10 clinical sites for phase II study | 4/18/22 | Other/Miscellaneous |
| Erytech Pharma SA, of Lyon, France | Eryaspase | L-asparaginase encapsulated inside donor-derived red blood cells | Acute lymphoblastic leukemia with hypersensitivity to peg-asparaginase | Results published in the British Journal of Haematology showed trial achieved primary endpoints; eryaspase sustained asparaginase enzyme activity above the threshold of >100 U/L at trough levels 14 days after first infusion in 92.5% of patients; well-tolerated; 2 patients had severe allergic reaction and withdrew eryaspase treatment | 4/6/22 | Other/Miscellaneous |
| Otonomy Inc., of San Diego | OTO-413 | Sustained-exposure formulation of brain-derived neurotrophic factor | Hearing loss | Top-line data demonstrated sustained exposure formulation of brain-derived neurotrophic factor in single intratympanic injection of 0.3 mg; clinically meaningful treatment benefit vs. placebo across multiple speech-in-noise hearing tests as well as the Patient Global Impression of Change (PGIC) at consecutive time points (day 57 and day 85); well-tolerated; no serious adverse events and no discontinuations due to adverse events | 4/20/22 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Pro-opiomelanocortin-derived peptide that is an agonist of melanocortin-4 receptor | Obesity | Modified enrollment criteria of Daybreak trial to focus initially on rare variants associated with 10 prioritized MC4R-relevant genes; paused enrollment of patients with variants in additional MC4R pathway genes; company will evaluate expansion of trial to those genes based on early clinical data from prioritized genes | 4/6/22 | Other/Miscellaneous |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | Trans sodium crocetinate | Oxygen-enhancing agent | Hypoxia | Final participant dosed in the Altitude study; top-line data expected within two months | 4/11/22 | Respiratory |
| Galecto Inc., of Boston | GB-0139 | Inhaled once-daily small molecule galectin-3 inhibitor | Idiopathic pulmonary fibrosis | Completed enrollment in GALACTIC-1 study | 4/26/22 | Respiratory |
| Anebulo Pharmaceuticals Inc., of Austin, Texas | ANEB-001 | Human cannabinoid receptor type 1 competitive antagonist | Acute cannabinoid intoxication | Dosed more than 60 subjects in part A of ongoing phase II study | 4/1/22 | Toxicity and Intoxication |
| Medicinova Inc., of La Jolla, Calif. | MN-166 (ibudilast) | Small molecule compound that inhibits phosphodiesterase type-4 | Alcohol use disorder | Results from secondary analysis of phase II study published in the journal Alcoholism: Clinical and Experimental Research showed the reduction rates of heavy drinking and measures of alcohol craving; completed daily diary assessments during the 2-week period; did not significantly alter mean levels of stimulation or sedation; moderated the effect of daily stimulation on same-day number of drinks consumed (p=0.045); attenuated alcohol-induced increases in craving compared with placebo (p=0.047) but no other subjective response measures; reduce the acute and chronic proinflammatory effects of alcohol; significantly tempered daily alcohol-induced changes in urge to drink (p=0.021) and positive mood (p=0.001) | 4/7/22 | Toxicity and Intoxication |
| Amphera BV, of s-Hertogenbosch, the Netherlands | Mesopher | Comprises autologous dendritic cells loaded with Pheralys | Resected pancreatic cancer | Reactive data published in European Journal of Cancer showed an average overall survival after surgery of 36 months; 8/10 patients alive; no disease recurrence or any tumor progression in 7/10 patients; 3 patients with disease recurrence did not demonstrate rapid tumor growth; favorable safety profile; transient grade 1 and 2 adverse events | 5/4/22 | Cancer |
| Arcus Biosciences Inc., of Hayward, Calif. | Domvanalimab (AB-154) | Fc-silent anti-TIGIT monoclonal antibody | Metastatic, PD-L1-high non-small-cell lung cancer | Interim data from ARC-7 study showed meaningful differentiation compared to zimberelimab alone across multiple efficacy measures; ORR for the doublet continued to increase and further separate from zimberelimab alone; data are still immature; no unexpected safety signals | 5/9/22 | Cancer |
| Calliditas Therapeutics AB, of Stockholm | Setanaxib | NOX 1 and 4 inhibitor | Squamous cell carcinoma of the head and neck | First patient randomized | 5/17/22 | Cancer |
| Compass Therapeutics Inc., of Boston | CTX-009 | Bispecific antibody that blocks Delta-like ligand 4/Notch (DLL4) and VEGF-A | Biliary tract cancer | Interim data in combination with paclitaxel showed 42% overall response rate; 10 patients with partial response; antitumor activity with a clinical benefit rate of 92%; well-tolerated | 5/4/22 | Cancer |
| Compass Therapeutics Inc., of Boston, and Elpiscience Biopharmaceuticals Inc., of Shanghai | CTX-009/ES-104 | DLL4/VEGF bispecific antibody | Biliary tract cancer | CTX-009/ES-104 plus paclitaxel produced a 42% overall response rate; in 24 previously treated patients, the clinical benefit rate was 92% | 5/5/22 | Cancer |
| Idera Pharmaceuticals Inc., of Exton, Pa. | Tilsotolimod | Synthetic Toll-like receptor 9 agonist | Melanoma | Interim data showed sentinel lymph node (SLN) positivity rates showed a 70% lower SLN+ compared to placebo 40%; statistical significance exceeded the pre-specified p-value of 0.008; trial stopped early | 5/17/22 | Cancer |
| Immunicum AB, of Stockholm | DCP-001 | Allogeneic, off-the shelf cell therapy | Acute myeloid leukemia | Interim data showed analysis of primary study endpoint was completed in 7 patients with measurable residual disease (MRD) response; 5 patients converted from MRD+ to MRD-negative 2 patients showed a substantial; 10-fold reduction in MRD following treatment; median follow-up period of 14.3 months; median relapse-free survival and overall survival not reached; estimated 6-month RFS based on the currently available data 83.7% and the estimated 6-month OS 97%; well-tolerated; no serious events |
5/16/22 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha (IMP-321) | Soluble LAG-3 fusion protein | HER2-negative/HR+ metastatic breast cancer | Exploratory analysis data from AIPAC phase IIb study showed statistically significant improvements in overall survival; significantly increased the number of circulating immune cells compared to baseline in combination with paclitaxel; increase in activated CD4 cells; increase in monocytes (p=0.025), CD8 T cells (p=0.027) and CXCL10 markers (p=0.006) | 5/4/22 | Cancer |
| Kintor Pharmaceutical Ltd., of Suzhou, China | GT-90001 | ALK-1 antibody | Advanced hepatocellular carcinoma | First patient dosed in combination with nivolumab (Opdivo, Bristol Myers Squibb Co.) | 5/4/22 | Cancer |
| Nykode Therapeutics A/S, of Oslo, Norway | VB-10.16 | Cancer neoantigen vaccine | HPV16-positive advanced cervical cancer | Interim results from 39 patients in combination with atezolizumab with a median follow-up of 6 months showed objective response rate (ORR) of 21%; 2 achieved a complete response and 6 achieved partial response; disease control rate (DCR) of 64%; antitumor activity was observed in both PD-L1-positive (ORR of 27% and DCR of 77%) and PD-L1-negative patients (ORR of 17% and DCR of 58%); DCR of 71% in patients with non-inflamed tumors; safe and well-tolerated | 5/9/22 | Cancer |
| Rhizen Pharmaceuticals AG, of Basel, Switzerland | Tenalisib (RP-6530) | Isoform-selective dual PI3K-delta/gamma inhibitor | Advanced or metastatic breast cancer | Interim data (n=40) showed well-tolerated; median duration of treatment was 4 months; about 60% of patients continuing on the study; median time to response was 1.8 months; disease control rate of 67.5% | 5/4/22 | Cancer |
| Rhovac AB, of Stockholm | Onilcamotide (RV-001) | RhoC GTPase modulator | Prostate cancer | Reached database lock | 5/10/22 | Cancer |
| Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | HER2-positive metastatic colorectal cancer | Results in combination with trastuzumab showed 38.1% confirmed objective response rate; median duration of response was 12.4 months; well-tolerated; low-grade adverse events | 5/23/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX-250 (77Lu-DOTA-girentuximab) | Monoclonal antibody targeting carbonic anhydrase IX | Renal cancer | First of 30 patients dosed in Starlite 2 trial testing the safety and efficacy of the drug combined with Opdivo (nivolumab, Bristol Myers Squibb) as assessed by the tumor response; imaging agent TLX250-CDx (89Zr-DFO-girentuximab) will also be used in the study | 5/4/22 | Cancer |
| Athersys Inc., of Cleveland, and Healios K.K., of Tokyo | Multistem (invimestrocel, HLCM-051) | Bone marrow-derived regenerative stem cell therapy | Ischemic stroke | Top-line data showed improvement in prespecified measures of functional independence and good outcomes, such as mRS ?2, Barthel Index ?95 and Global Recovery; primary endpoint did not reach statistical significance in this population; consistent improvement in essentially all measured functional outcomes over time through 1 year, supporting long-term impact on and continued improvement in the quality of life of treated patients; global recovery 27.9% vs. 15.7% for placebo (p<0.05); barthel index ?95 of 35.6% vs. 22.5% (p="0.05);" 15.4% multistem-treated patients acheived "excellent outcome" 10.8% for placebo< td> | 5/19/22 | Cardiovascular |
| Caladrius Biosciences Inc., of Basking Ridge, N.J. | CLBS-201 (Xowna) | CD34-positive cell therapy | Coronary microvascular dysfunction | Suspended patient enrollment in Freedom trial | 5/23/22 | Cardiovascular |
| Recardio Inc., of San Francisco | Dutogliptin | DPPIV inhibitor | Acute myocardial infarction | Data showed dutogliptin in co-administration with filgrastim was safe; none of the subjects experienced non-fatal stroke; stent thrombosis or cardiovascular death; no statistically significant difference between the groups with respect to serious or severe treatment-emergent adverse events; positive changes in left ventricular parameters over time compared to the placebo group; improvement in cardiac tissue | 5/23/22 | Cardiovascular |
| Tenax Therapeutics Inc., of Morrisville | TNX-201 | Oral imatinib modified release tablets | Pulmonary arterial hypertension | Initiated clinical sites; patient enrollment expected to start in the second half of 2022 | 5/23/22 | Cardiovascular |
| Windtree Therapeutics Inc., of Warrington, Pa. | Istaroxime | Positive inotropic agent; Na+/K+- ATPase inhibitor | Cardiogenic shock | Study met its primary endpoint of improved systolic blood pressure (SBP) profile at 6 hours, significantly better compared to the control group (p =0.017); statistically significant improvement persisted through the 24-hour SBP profile (p=0.025); SBP increased rapidly within the first hour and sustained throughout the 96-hour post-infusion measure; improvement in cardiac index compared to the control (p=0.016); significant improvements, including left atrial area and left ventricular end systolic volume; substantial increase in stroke volume; maintained renal function; well-tolerated | 5/23/22 | Cardiovascular |
| Akari Therapeutics plc, of New York | Nomacopan | LTB4 and complement C5 inhibitors | Bullous pemphigoid | Results published in JAMA Dermatology showed 7 of 9 patients responded to nomacopan, with 3 as complete responders; >80% reduction in BP disease activity index score; 4 patients showed >70% reduction in pruritis by day 42; 2 of 9 were non-responders | 5/10/22 | Dermatologic |
| Argenx SE, of Breda, the Netherlands | Efgartigimod | Antibody fragment designed to reduce pathogenic immunoglobulin G | Pemphigus | Secondary analysis of subset study showed sustained reduction of antigen-specific B-cells; sustained clinical improvement while total IgG returned to near baseline levels; median CD19+ B-cells remained within normal limits; no new safety signals; no observed effect on total leukocytes, neutrophils, monocytes, or lymphocytes; efficiently rescue the loss of keratinocyte adhesion upon anti-Dsg-3 antibodies and PV IgG treatment | 5/19/22 | Dermatologic |
| Bridgebio Pharma Inc., of Palo Alto, Calif. | PTR-01 | I.V.-administered recombinant collagen 7 (rC7) protein replacement therapy | Recessive dystrophic epidermolysis bullosa | Data showed rapid, consistent, and durable wound healing as observed in reduction of wound surface area and clinician-reported assessments; 80% of target wounds demonstrated a 50% or greater reduction in wound surface area at the end of treatment (day 120) compared to baseline; 58% of patients met the response criteria of ?2-point increase on the wound-specific scale at day 15; 69% met the response criteria at day 120; rapid deposition of rC7 at the DEJ during the loading phase (first 28 days of treatment) and remained present up to 3 months after treatment; well-tolerated | 5/20/22 | Dermatologic |
| Clinuvel Pharmaceuticals Ltd., of Perth, Australia | Afamelanotide (Scenesse) | Melanocyte stimulating hormone ligand | Vitiligo | Around 11,000 doses of afamelanotide administered to over 1,600 individuals | 5/9/22 | Dermatologic |
| Horizon Therapeutics plc, of Dublin | Daxdilimab (HZN-7734) | Fully human monoclonal antibody targeting immunoglobulin-like transcript 7 | Alopecia areata | First patient enrolled | 5/17/22 | Dermatologic |
| Mediwound Ltd., of Yavne, Israel | Escharex | Bioactive therapy; concentrate of proteolytic enzymes enriched in bromelain for topical use | Debridement of chronic wounds | Study met its primary and key secondary endpoints with statistically significant results compared to control; statistically significant higher incidence of complete debridement during the 14-day measurement period within up to 8 applications compared to gel vehicle (Escharex: 63% vs. gel vehicle: 30%, p=0.004); median time to complete debridement was 9 days vs. 59 days for patients treated with NSSOC (p=0.016); significantly higher incidence of at least 75% granulation tissue at the end of the treatment period compared to gel vehicle (p<0.0001); safe and well-tolerated< td> | 5/12/22 | Dermatologic |
| Moonlake Immunotherapeutics AG, of Zug, Switzerland | Sonelokimab | Nanobody; inhibits IL-17A and IL-17F | Moderate to severe plaque psoriasis | Results published in the British Journal of Dermatology showed more patients achieved complete skin clearance at week 24 vs. patients treated with secukinumab (56.9% vs. 34%, respectively, p= 0.019); 20% maintained complete skin clearance for 24 weeks and no further treatment; rapid and durable clinical response | 5/9/22 | Dermatologic |
| Moonlake Immunotherapeutics AG, of Zug, Switzerland | Sonelokimab | Nanobody; inhibits IL-17A and IL-17F | Moderate to severe hidradenitis suppurativa | First patient randomized and dosed | 5/12/22 | Dermatologic |
| Rapt Therapeutics Inc., of South San Francisco | RPT-193 | Small-molecule oral therapy; selectively inhibits migration of Th2 cells in inflamed tissues by blocking CCR4 | Moderate to severe atopic dermatitis | Initiated 16-week phase IIb study in patients as monotherapy | 5/23/22 | Dermatologic |
| Lysogene SA, of Paris | LYS-SAF302 | Gene therapy | Mucopolysaccharidosis type IIIA | Data showed persistent increase or stabilization in three main developmental domains in all patients enrolled under the age of 30 months; full results expected in Q3 2022 | 5/18/22 | Endocrine/Metabolic |
| Rezolute Inc., of Redwood City, Calif. | RZ-358 | Human monoclonal antibody that binds to an allosteric site on insulin receptors in the liver, fat, and muscle | Congenital hyperinsulinism | Exceeded expectations for correcting hypoglycemia, including a highly significant reduction of ~75% in hypoglycemia events by blood glucometer as well as time in hypoglycemia by continuous glucose monitoring | 5/1/22 | Endocrine/Metabolic |
| Anji Pharma Inc., of Cambridge, Mass., and Shanghai | Pradigastat (ANJ-908) | DGAT1 inhibitor | Functional constipation | Completed enrollment in proof-of-concept study; top-line data expected in second half of 2022 | 5/18/22 | Gastrointestinal |
| Connect Biopharmaceuticals Ltd., of Taicang, China | CBP-307 | Small molecule designed to modulate sphingosine 1-phosphate receptor 1 | Moderate to severe ulcerative colitis | Top-line data at 12 weeks showed numerical reduction for the primary endpoint of least squares mean change from baseline in adapted Mayo Score that did not meet statistical significance at 0.2 mg (p=0.103); achieved clinical remission (p=0.016) vs. placebo; reductions in lymphocyte counts by 51.2%; no cases of progressive multifocal leukoencephalopathy and no deaths; well-tolerated | 5/3/22 | Gastrointestinal |
| Galera Therapeutics Inc., of Malvern, Pa. | Avasopasem | Selective small molecule dismutase mimetic | Severe acute radiation-induced esophagitis in patients with lung cancer receiving concurrent chemoradiotherapy | The incidence of Grade 3 esophagitis was substantially reduced in patients treated with avasopasem compared to literature | 5/2/22 | Gastrointestinal |
| Lipocine Inc., of Salt Lake City | LPCN-1144 | Orally delivered prodrug of testosterone | Nonalcoholic steatohepatitis | Top-line data showed 23 subjects completed the study; 1 discontinued due to a non-drug related treatment emergent adverse event; 6 elected for an optional liver biopsy at the end of thestudy at week 72; well-tolerated over 72-week exposure with no observed safety signals; significantly reduced liver injury markers (p<0.05) and maintained with extended treatment; observed liver histology improvements support further development< td> | 5/12/22 | Gastrointestinal |
| Chinook Therapeutics Inc., of Seattle | Atrasentan | Endothelin A receptor inhibitor | Primary IgA nephropathy | Results showed drug well-tolerated with no treatment-related serious adverse events; 38% proteinuria reduction at 6 weeks of treatment, 49.9% proteinuria reduction at 12 weeks of treatment and 58.5% reduction at 24 weeks of treatment; no meaningful changes in blood pressure or acute eGFR; no increase in brain natriuretic peptide or mean bodyweight | 5/23/22 | Genitourinary/Sexual Function |
| Eledon Pharmaceuticals Inc., of Irvine, Calif. | AT-1501 (tegoprubart) | Targets CD40 ligand | IgA nephropathy | First patient dosed; initial data from study expected in late 2022 | 5/9/22 | Genitourinary/Sexual Function |
| Kibow Pharmaceuticals Llc., of Newtown Square, Pa. | US-APR2020 | Oral therapy; natural probiotics formulation that metabolizes nitrogenous waste | Chronic Kidney Disease stage4 | Recruited about half of the targeted 630 patients; first patient completed 6 months of study | 5/17/22 | Genitourinary/Sexual Function |
| Urovant Sciences Inc., of Irvine, Calif. | URO-902 | Locally injected gene therapy product; plasmid human cDNA encoding maxi-K channel | Overactive bladder and urge urinary incontinence | Interim data showed safe and well-tolerated; clinically relevant improvement in the common symptoms of OAB compared to placebo; clinically relevant improvement in efficacy at 24-mg and 48-mg doses in women with OAB; urinary tract infection as adverse event | 5/13/22 | Genitourinary/Sexual Function |
| Imago Biosciences Inc., of South San Francisco | Bomedemstat | Lysine-specific histone demethylase-1 inhibitor | Essential thrombocythemia | Completed enrollment; 73 patients enrolled in trial to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics | 5/3/22 | Hematologic |
| Rocket Pharmaceuticals Inc., of Cranbury, N.J. | RP-L102 | Gene therapy | Fanconi anemia | Data showed 5/9 evaluable patients had increased resistance to mitomycin-C (MMC) in bone marrow-derived colony-forming cells, ranging from 21% to 42% at 12 to 18 months, compared to 51% to 94% at 18 to 21 months; achieved primary endpoint with statistical and clinical significance; 3/12 treated patients have not yet reached the 12-month time point; no signs of dysplasia, clonal dominance or oncogenic integrations | 5/16/22 | Hematologic |
| Acelyrin Inc., of Los Angeles, Affibody AB, of Solna, Sweden, and Inmagene Biopharmaceuticals Co. Ltd., of Shanghai | Izokibep | IL-17A-inhibiting antibody mimetic | Psoriatic arthritis | Results showed 16-week phase II study in 135 patients met its primary endpoint of ACR50 and achieved secondary endpoints; clinically meaningful improvement in disease-specific quality of life was achieved; no new safety issues | 5/3/22 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes | Active and chronic active antibody-mediated rejection episodes | Completed enrollment; first readout expected in the second half of 2022 | 5/23/22 | Immune |
| Horizon Therapeutics plc, of Dublin | Dazodalibep (HZN-4920) | CD40 ligand antagonist | Rheumatoid arthritis | Top-line data showed study met the primary endpoint of change from baseline in DAS28-CRP at day 113 in all 4 dazodalibep dosing arms | 5/3/22 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-107 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | Interim data showed all 23 treated patients are alive at 2.6-year median follow-up without evidence of malignant transformation; well-tolerated; all patients experienced complete hematopoietic recovery | 5/19/22 | Immune |
| Protara Therapeutics Inc., of New York | OK-432 | Originator compound for TARA-002; immunopotentiator | Lymphatic malformations | Data showed 69% of patients in the randomized study immediate treatment group achieved clinical success after 6 months vs. 7.5% of patients in the delayed treatment group showed spontaneous resolution in the same time period (p<0.0001); 1 73.1% of patients in the open-label study achieved clinical success; well-tolerated; serious event relaed to ok-432 led discontinuation; no significant safety concerns< td> | 5/12/22 | Immune |
| Akston Biosciences Inc., of Beverly, Mass. | AKS-452 | Vaccine containing the receptor binding domain of SARS-CoV2 | COVID-19 prophylaxis | Dosed first partipant of anticipated 600 volunteers | 5/12/22 | Infection |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | Capsid-virus-like particle vaccine | COVID-19 prophylaxis | Vaccination of patients who had previously received mRNA or adenoviral vaccines induced a significant boost in neutralizing antibodies against the omicron variant with a fold increase in the same range as reported for the original Wuhan SARS-CoV2 virus | 5/5/22 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody designed to inhibit TLR4 | COVID-19-induced acute respiratory distress syndrome | Initiated enrollment for second cohort of patients for phase III part of phase II/III study | 5/24/22 | Infection |
| Enanta Pharmaceuticals Inc., of Watertown, Mass. | EDP-938 | N-protein inhibitor | Respiratory syncytial virus infection | Top-line data showed study did not meet primary endpoint of clinical symptom reduction compared to placebo, or the secondary antiviral endpoints; AUC for the mean total symptom score was not statistically different between the two groups (p=0.256); statistically significant difference in the number of subjects achieving undetectable RSV RNA at the end of treatment at day 5 was observed with EDP-938 compared to placebo (p=0.033); favorable safety profile; safe and well-tolerated | 5/18/22 | Infection |
| Hyundai Bioscience Co. Ltd., of Seoul, South Korea | CP-COV03 | Oral; niclosamide | COVID-19 | Launched phase II study | 5/12/22 | Infection |
| Karolinska Development AB, of Stockholm | BSG-005 | Antifungal compound | Mucormycosis | Initiating phase II study in India | 5/3/22 | Infection |
| KVK-Tech Inc., of Newtown, Pa. and Sen-Jam Pharmaceutical LLC., of Huntington, New York | SJP-002C | Oral therapy | COVID-19 | Launched study in Nepal; initiated enrollment | 5/5/22 | Infection |
| Regencell Bioscience Holdings Ltd., of Hong Kong | RGC-COV19/RGCA-CV0 | Natural, orally administered liquid formulation | Mild to moderate COVID-19 | Results showed 48/51 patients had all their mild to moderate COVID-19 symptoms eliminated within the 6-day treatment period; 11 patients recovered their sensory functions while the remaining 6 showed improvements at the end of the 6-day treatment period | 5/18/22 | Infection |
| Kezar Life Sciences Inc., of South San Francisco | Zetomipzomib (KZR-616) | Selective immunoproteasome inhibitor | Dermatomyositis and polymyositis | Top-line data of Presidio study showed clinically meaningful improvements in total improvement score (TIS); no significant differentiation from placebo; achieved a mean TIS of 25.5 vs. the control group mean TIS of 25 at week 16; well-tolerated | 5/3/22 | Inflammatory |
| 180 Life Sciences Corp., of Palo Alto, Calif. | Adalimumab | Anti-TNF treatment | Dupuytren’s disease | Phase IIb data published in The Lancet Rheumatology showed nodule hardness was lower in anti-TNF arm vs. placebo (-4.6AU; 95% CI -7.1 to -2.2; p=<0.0002) 9 12 18 at months and decreased further (-5.8au; 95% ci -8.7 to -3.0; p="<0.0001)," after the last injection; nodule size, measured using ultrasound scan, was lower in anti-tnf group vs. placebo (-8.4 mm2; -13.8 -2.9; (-14.4 -19.9 -9.0; no treatment-related side effects< td> | 5/2/22 | Musculoskeletal |
| ABVC Biopharma Inc., of Fremont, CA. | ABV-1505 | Norepinephrine inhibitor; plant based drug | Attention-deficit hyperactivity disorder | First subject dosed | 5/10/22 | Musculoskeletal |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 (ursodoxicoltaurine + sodium phenylbutyrate) | Fixed dose combination | Amyotrophic lateral sclerosis | Long-term survival of Centaur study published in Muscle and Nerve showed larger survival benefit compared to placebo crossover ranging from 10.6 months to 18.8 months compared with 6.9 months seen in the original prespecified intent-to-treat analysis (p=.023); significantly lower hazard of death and longer median survival duration of 4.8 months in AMX-0035 vs. placebo (p=.048) | 5/5/22 | Musculoskeletal |
| Denali Therapeutics Inc., of South San Francisco | DNL-788 | Small molecule inhibitor of RIPK1 | Amyotrophic lateral sclerosis | Commenced dosing | 5/2/22 | Musculoskeletal |
| Saol Therapeutics Inc., of Roswell, Ga. | SL-1002 | Nerve-blocking agent | Limb spasticity | First patient enrolled; top-line data expected in 2023 | 5/3/22 | Musculoskeletal |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 (ursodoxicoltaurine + sodium phenylbutyrate) | Fixed dose combination | Amyotrophic lateral sclerosis | Results published in the Journal of Neurology, Neurosurgery and Psychiatry showed 47% lower of risk of any event compared to placebo (71%), p=.003; risk of death or tracheostomy/PAV was 49% lower, p=.007; risk of first hospitalization was 44% lower, p=.03; median key event-free survival duration was 4.8 months longer vs. placebo; median tracheostomy/PAV-free survival duration was 7.3 months longer; median time to first hospitalization was not yet reached vs. 14.1 months in the group originally randomized to placebo | 5/17/22 | Musculoskeletal |
| Alzamend Neuro Inc., of Atlanta | AL-001 | Lithium-salicylate-L-proline engineered ionic co-crystal | Mild to moderate Alzheimer’s disease | First patient dosed in a 12-month, multiple ascending-dose study to test the safety and tolerability of AL-001 under steady-state conditions and to determine the maximum tolerated dose; top-line data expected in December 2022 | 5/5/22 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-05 | Oral NMDA receptor antagonist; 45-mg dextromethorphan and 105-mg bupropion | Major depressive disorder | Results published in the American Journal of Psychiatry showed rapid, substantial, and statistically significant improvement in depressive symptoms and induction of remission compared with bupropion; mean change from baseline in MADRS score over weeks 1-6 was significantly greater with AXS-05 than with bupropion (-13.7 points vs. -8.8 points; least-squares mean difference=-4.9; p<0.001); 2 madrs score change with axs-05 was significantly greater than bupropion at week and every time point (week 6: -17.3 vs. -12.1 points; least-squares mean difference="-5.2;" p="0.013);" remission rates were 46.5% 16.2%; well-tolerated< td> | 5/18/22 | Neurology/Psychiatric |
| Cognition Therapeutics Inc., of Pittsburgh | CT-1812 | Binds to a receptor on neurons that regulates cellular damage response pathways | Mild to moderate Alzheimer’s disease | Dosing commenced in the second cohort of the Shine study | 5/11/22 | Neurology/Psychiatric |
| Compass pathway plc, of London | COMP-360 | Psilocybin therapy | Treatment-resistant depression | Results showed statistically significant reduction in depressive symptoms after 3 weeks (p<0.001), 12 with a rapid and durable response for up to weeks; sustained at week 12, 20.3% of patients in the 25-mg group vs. 10.1% 1-mg group; well-tolerated< td> | 5/23/22 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | Zyrnelef | Extended-release solution of bupivacaine and meloxicam | Postpartum pain in women undergoing a planned C-section | Results showed well-tolerated; 27% had an opioid-free recovery; 69% less opioid rescue medication for pain compared to the lower dose group and reported fewer opioid-related adverse events; low microgram levels of bupivacaine and meloxicam in breast milk after single postpartum dose | 5/16/22 | Neurology/Psychiatric |
| Idorsia Ltd., of Allschwil, Switzerland | ACT-539313 | Orexin-1 receptor antagonist | Binge eating disorder | Results showed primary endpoint was not met; well-tolerated over the treatment period of 12 weeks | 5/10/22 | Neurology/Psychiatric |
| Mind Medicine Inc., of New York | LSD | LSD | Anxiety disorders | Top-line data showed significant and strong reductions of STAI-G scores 16 weeks after treatment in the between-subjects analysis (p=0.007); reduction in STAI-G scores (?30% vs. 9% in placebo, p=0.003); rapid and similar responses in HAM-D-21, BDI and SCL-90-R; well-tolerated | 5/11/22 | Neurology/Psychiatric |
| NLS Pharma Ltd., of Stans, Switzerland | Quilience | Mazindol extended release | Narcolepsy | Achieved 90% patient enrollment in phase IIa study | 5/4/22 | Neurology/Psychiatric |
| Nrx Pharmaceuticals Inc., of Radnor, Pa. | NRX-101 | Oral; Fixed-dose combination product of D-cycloserine and lurasidone | Bipolar depression and sub-acute suicidality | Enrolled first patient | 5/12/22 | Neurology/Psychiatric |
| Praxis Precision Medicines Inc., of Boston | PRAX-944 | T-type calcium channel blocker | Essential tremor | Top-line data showed clinically meaningful improvements in function; PRAX-944 demonstrated mean improvement from baseline of 42% in the modified activities of daily living score at day 42 p<0.05; clinically and statistically significant compared to placebo; well-tolerated; no new safety findings< td> | 5/9/22 | Neurology/Psychiatric |
| Scilex Holding Company of Palo Alto, Calif. | SP-103 | Non-aqueous lidocaine topical system | Acute low back pain | First subject dosed | 5/18/22 | Neurology/Psychiatric |
| Vasopharm GmbH, of Würzburg, Germany | Ronopterin (formerly VAS-203) | iNOS inhibitor | Traumatic brain injury | Combined analysis of Nostra and Nostra III (phase III) showed improved neurologic outcome in all patients with significantly increased proportion of patients with Good Recovery (GOS 5) at 6 months (ronopterin: 39% vs. placebo: 18%, p=0.03); proportion of patients with low therapy intensity level was significantly decreased (77% vs 67%, p<0.004)< td> | 5/10/22 | Neurology/Psychiatric |
| Vasopharm GmbH, of Würzburg, Germany | Ronopterin (formerly VAS-203) | iNOS inhibitor | Traumatic brain injury | Results from Nostra study showed primary endpoint was eGOS at 6 months for which the median was 6 with Ronopterin vs 4.5 in the placebo arm; therapy intensity level was significantly reduced (p<0.04)< td> | 5/10/22 | Neurology/Psychiatric |
| Xenon Pharmaceuticals Inc., of Burnaby, British Columbia | XEN-1101 | TargetsKv7 neuronal potassium channels | Major depressive disorder | Initiation of phase II study to evaluate the safety, tolerability and efficacy | 5/3/22 | Neurology/Psychiatric |
| Iveric Bio Inc., of New York | Zimura (avacincaptad pegol) | Pegylated aptamer targeting the C5 component of the complement cascade | Geographic atrophy | Post-hoc analysis of Gather1 study showed reduction of GA lesion growth in patients compared to sham; no additional safety analysis | 5/4/22 | Ocular |
| Oxurion NV, of Leuven, Belgium | THR-687 | Small-molecule pan-RGD integrin antagonist | Diabetic macular edema | Top-line data showed safe and well-tolerated with no serious adverse events; insufficient evidence of efficacy on the key endpoints; company decided not to advance part B of integral study | 5/9/22 | Ocular |
| Qlaris Bio Inc., of Wellesley, Mass. | QLS-101 | Adenosine triphosphate-sensitive potassium channel modulator | Glaucoma | Data showed favorable safety and tolerability profile; no evidence of hyperemia | 5/18/22 | Ocular |
| Rocket Pharmaceuticals Inc., of Cranbury, N.J. | RP-L201 | Gene therapy | Leukocyte adhesion deficiency-I | Top-line data showed sustained CD18 restoration and expression on more than 10% of neutrophils (median 56%); overall survival without allogeneic hematopoietic stem cell transplantation across the cohort is 100% based on the Kaplan-Meier estimate; statistically significant reduction in the rate of all-cause hospitalizations and severe infections, relative to pre-treatment; favorable in all patients with no RP-L201-related serious adverse events | 5/18/22 | Ocular |
| Stealth Biotherapeutics Corp., of Boston | Elamipretide | Mitochondria-targeted compound | Geographic atrophy secondary to dry age-related macular degeneration | Study did not meet primary endpoints of mean change in low luminance visual acuity and geographic atrophy progression | 5/2/22 | Ocular |
| Stealth Biotherapeutics Corp., of Boston | Elamipretide | Peptide compound targets the inner mitochondrial membrane and binds reversibly to cardiolipin | Geographic atrophy secondary to dry age-related macular degeneration | Results from Reclaim-2 study showed elamipretide ameliorated progressive decline, or attenuation of the mitochondrial rich ellipsoid zone layer of the photoreceptors (p<0.01); categorical>2-line improvement in low luminance visual acuity (LLVA) (p=0.04); primary endpoints of mean change in LLVA and GA progression were not met; well-tolerated; rate of new-onset exudations was 5.3% in the elamipretide treated group vs. 6.9% in placebo | 5/23/22 | Ocular |
| Mereo Biopharma Group plc, of London | Alvelestat | Neutrophil elastase inhibitor | Severe alpha-1 antitrypsin deficiency-associated emphysema | Top-line Astraeus study (n=99) data showed statistically significant changes vs. placebo in all 3 primary biomarker endpoints; statistically significant inhibition of blood neutrophil elastase activity of up to 90% in patients in both high- and low-dose alvelestat groups throughout the 12-week dosing period; statistically significant reductions in the biomarkers A?-val360 and desmosine at the high dose; no safety signals | 5/9/22 | Other/Miscellaneous |
| Prota Therapeutics Pty Ltd., of Melbourne, Australia | PRT-120 | Oral immunotherapy | Peanut allergy | Data showed 51% of treated children ages 1-10 achieved clinical remission; significant and substantial improvement in quality of life compared to placebo at 12 months post-treatment (p= 0.002) | 5/16/22 | Other/Miscellaneous |
| Saniona AB, of Copenhagen, Denmark | Tesomet | Fixed-dose combination therapy of tesofensine and metoprolol | Hypothalamic obesity | Results published in European Journal of Endocrinology showed favorable safety and tolerability profile | 5/23/22 | Other/Miscellaneous |
| Avalo Therapeutics Inc., of Wayne, Pa. | AVTX-002 | Fully human anti-LIGHT monoclonal antibody | Non-eosinophilic asthma | First patient dosed | 5/18/22 | Respiratory |
| Boehringer Ingelheim Pharmaceuticals Inc., a unit of Boehringer Ingelheim GmbH, of Ingelheim, Germany | BI-1015550 | PDE4B inhibitor | Idiopathic pulmonary fibrosis | Data published in The New England Journal of Medicine showed slight improvement in forced vital capacity (FVC) (+5.7 mL for BI-1015550 and -81.7 mL for placebo) at week 12; median changes in FVC were +2.7 mL for BI-1015550 and -59.2 mL in the placebo arm in patients already taking antifibrotic therapy; trial met its secondary endpoint; acceptable safety and tolerability | 5/16/22 | Respiratory |
| Insmed Inc., of Bridgewater, N.J. | Brensocatib | Oral, reversible inhibitor of dipeptidyl peptidase 1 | Non-cystic fibrosis bronchiectasis | Post-hoc data of Willow study showed favorable benefit-risk profile; significantly prolonged time to first pulmonary exacerbation over 24-week treatment ranging from 6 to 7; reduced the rate of pulmonary exacerbations | 5/17/22 | Respiratory |
| Adverum Biotechnologies Inc., of Redwood City, Calif. | ADVM-022 | Gene therapy | Wet age-related macular degeneration | Post-hoc analysis of Optic study showed, in all subjects, single intravitreal injection gene therapy at 2 X 10^11 vg/eye-dose demonstrated greater than 80% reduction in annualized anti-VEGF injections; in subjects with baseline NAbs <1:125, the reductions in mean annualized injection frequency increased to 94%< td> | 5/1/22 | Ocular |
| Applied Genetic Technologies Corp., of Gainesville, Fla. | AGTC-501 | Recombinant AAV vector-based gene therapy | X-linked retinitis pigmentosa | Data from 18-month safety and efficacy phase I/II study showed improvements to the ellipsoid zone (EZ); sustained durability of improved visual function; two-thirds patients showed recovery of foveal EZ; half had improved EZ appearance; improvement in macular sensitivity; well-tolerated | 5/4/22 | Ocular |
| Applied Genetic Technologies Corp., of Gainesville, Fla. | AGTC-501 | Recombinant AAV vector-based gene therapy | X-linked retinitis pigmentosa | Interim results showed improvements in visual sensitivity after 3 months dosing in 62.5% of patient; mean visual sensitivity of entire macula increased and the area of the macula with visual sensitivity also increased | 5/16/22 | Ocular |
| Ashvattha Therapeutics Inc., of Redwood City, Calif. | D-4517.2 | VEGFR/PDGFR tyrosine kinase inhibitor conjugated to a hydroxyl dendrimer | Age-related macular degeneration and diabetic macular edema | Safety data from phase I study showed safe and well-tolerated in healthy subjects at dose levels of 0.25 mg/kg, 0.50 mg/kg and 1 mg/kg; no significant changes in safety labs or other adverse reactions to the treatment | 5/2/22 | Ocular |
| Belite Bio Inc., of San Diego | LBS-008 (tinlarebant) | Plasma retinol binding protein inhibitor | Adolescent Stargardt disease | Drug had an acceptable safety profile; 8 of 13 patients recorded a gain in best corrected visual acuity; subjects from the phase Ib study are participating in the 2-year, phase II extension | 5/5/22 | Ocular |
| Kala Pharmaceuticals Inc., of Arlington, Mass. | KPI-012 | Cell-free secretome therapy | Severe ocular diseases driven by impaired healing | Data from phase Ib trial in patients with persistent corneal epithelial defect etiologies showed rapid and complete wound healing, with 6/8 (75%) achieving complete healing within 4 weeks; all patients with pain at baseline reported zero pain by week 3; well-tolerated | 5/1/22 | Ocular |
| Luxa Biotechnology LLC, of Fort Lee, N.J. | RPESC-RPE-4W | Cell product derived from the retinal pigment epithelium stem cell | Dry age-related macular degeneration | First subject dosed | 5/24/22 | Ocular |
| Acousia Therapeutics GmbH, of Tübingen, Germany | ACOU-085 | Small-molecule, otoprotective drug | Presbycusis | First patient dosed in the phase Ib study to test the safety and tolerability of the drug; subjective and objective hearing tests will also be measured | 5/5/22 | Other/Miscellaneous |
| Inhibrx Inc., of San Diego | INBRX-101 | Optimized recombinant human AAT-Fc fusion protein | Alpha-1 antitrypsin (AAT) deficiency | INBRX-101 dosed at 80 mg/kg produced a mean C-avg of functional AAT of 40.4 µM over the 21-day dosing interval following the third dose; INBRX-101 was found in the lung fluid of both patients with data | 5/16/22 | Other/Miscellaneous |
| 4D Pharma plc, of Leeds, U.K. | MRx-4DP0004 | Immunomodulatory, single strain live biotherapeutic | Asthma | Data showed safe and well-tolerated; no treatment-related severe adverse events; no serious events; 83.3% of subjects receiving MRx-4DP0004 had improved ACQ-6 scores vs. 56.3% of placebo group (p=0.088); 50% reduced their use of SABA rescue medication v.s 18.8% of placebo group; 50% of patients saw clinically meaningful improvements in Asthma Quality of Life Questionnaire scores of ?0.5 from baseline vs. 31.3% of placebo group; mean measures of lung function remained stable from baseline and throughout treatment period | 5/17/22 | Respiratory |
| Atyr Pharma Inc., of San Diego | Efzofitimod (ATYR-1923) | Immunomodulator; selective modulation of neuropilin-2 | Pulmonary sarcoidosis | Data showed improvements in percent-predicted forced vital capacity and percent-predicted diffusing capacity of the lungs for carbon monoxide compared to placebo; clinically meaningful and statistically significant improvements at week 24 at 5 mg/kg; dose-dependent improvements in key physiologic and quality of life measures; reduced pro-inflammatory serum biomarkers | 5/17/22 | Respiratory |
| Blade Therapeutics Inc., of South San Francisco | Cudetaxestat | Non-competitive autotaxin inhibitor | Idiopathic pulmonary fibrosis | Data from drug-drug interaction study showed neither nintedanib nor pirfenidone significantly alter the exposure of cudetaxestat; cudetaxestat does not affect the PK of pirfenidone and does not significantly increase the exposure of nintedanib (AUC[0-12]); no treatment-related serious adverse events; well-tolerated | 5/16/22 | Respiratory |
| Epiendo Pharmaceuticals ehf, of Reykjavik, Iceland | EP-395 | Oral, non-antibiotic macrolide | Chronic obstructive pulmonary disease | Completed first-in-human study showing drug well-tolerated in healthy subjects, with pharmacokinetics consistent with once-daily dosing | 5/18/22 | Respiratory |
| Puretech Health plc, of Boston | LYT-100 (deupirfenidone) | Deuterated form of pirfenidone | Idiopathic pulmonary fibrosis | Data showed lower incidence of adverse events compared to pirfenidone at comparable exposure levels in healthy older adults; LYT-100 at 550-mg (fed state) dose met the criteria for bioequivalence for exposure compared to the pirfenidone; improved tolerability profile of LYT-100 compared to pirfenidone; top-line results expected in 2023 | 5/16/22 | Respiratory |
| Mind Medicine Inc., of New York | MM-110 (zolunicant) | Alpha-3-beta-4 nicotinic cholinergic receptor antagonist and non-hallucinogenic proprietary congener of ibogaine | Opioid withdrawal | Results showed favorable safety and tolerability; support advancement of MM-110, and guided phase IIa dose, schedule and design in individuals undergoing supervised opioid withdrawal | 5/19/22 | Toxicity and Intoxication |
| Active Biotech AB, of Lund, Sweden, and Neotx Therapeutics Ltd., of Dublin | Naptumomab estafenatox | Fusion protein targeting 5T4 tumor antigen with immunomodulating and antineoplastic activities | Advanced or metastatic non-small-cell lung cancer | Completed first stage of a Simon 2-stage phase IIa study in combination with docetaxel | 6/1/22 | Cancer |
| Aileron Therapeutics Inc., of Boston | ALRN-6924 | MDM2/MDMX dual inhibitor | p53-mutated non-small-cell-lung cancer | Company planning to stop further enrollment and apply key learnings from the interim analysis to strengthen the phase Ib breast cancer trial | 6/29/22 | Cancer |
| Aristea Therapeutics Inc., of San Diego | RIST-4721 | CXCR2 antagonist | Palmoplantar pustulosis | First subject dosed | 6/30/22 | Cancer |
| Calithera Biosciences Inc., of South San Francisco | Mivavotinib (CB-659) | SYK inhibitor | Relapsed/refractory non-germinal center B-cell like (non-GCB) diffuse large B-cell lymphoma | First patient enrolled | 6/24/22 | Cancer |
| Celsion Corp., of Lawrenceville, N.J. | GEN-1 | Plasmid encoding human interleukin-12 | Stage III/IV ovarian cancer | Data safety monitoring board recommended Ovation2 study at dose of 100 mg/m2; well-tolerated; no dose-limiting toxicity; full enrollment expected in the third quarter of 2022 | 6/21/22 | Cancer |
| Cend Therapeutics Inc., of San Diego | CEND-1 | Targeted to tumor vasculature by its affinity for alpha-v integrins | Metastatic pancreatic ductal adenocarcinoma | First patient treated | 6/9/22 | Cancer |
| Day One Biopharmaceuticals Inc., of South San Francisco | Tovorafenib (DAY-101) | Oral, brain-penetrant, type II pan-RAF kinase inhibitor | Relapsed or progressive pediatric low-grade glioma | Initial data from 22 response-evaluable patients in Firefly-1 study showed 64% objective response rate and 91% clinical benefit rate (partial response/unconfirmed partial response + stable disease), with 14 partial responses and 6 stable disease; all patients with stable disease were noted to have tumor shrinkage ranging between 19% and 43%; responses observed in patients with both BRAF fusions and BRAF V600E mutations who received prior MAPK-targeted therapy; median time to response was 2.8 months | 6/12/22 | Cancer |
| Galecto Inc., of Boston | GB-1211 | Oral small-molecule galectin-3 inhibitor | Non-small-cell lung cancer | First patient enrolled in Gallant-1 study in combination with atezolizumab (Tecentriq, Roche Holding AG) | 6/16/22 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib | Brain-penetrant small-molecule phosphoinositide-3 kinase inhibitor of PI3K/AKT/mTOR pathway | Breast cancer | Paxalisib arm of the Alliance study (A071701) fully recruited cohort of breast cancer patients with brain metastases for initial prespecified interim analysis and has met the threshold for transition to the expansion stage of the study | 6/7/22 | Cancer |
| Merck KGaA, of Darmstadt, Germany | Berzosertib | Serine threonine protein kinase ATR inhibitor | Relapsed, platinum-resistant small-cell-lung cancer | Interim analysis in combination with topotecan showed to discontinue the study due to low probability of meeting the pre-defined objective of this trial | 6/3/22 | Cancer |
| Nordic Nanovector ASA, of Oslo, Norway | Betalutin (177Lu lilotomab satetraxetan) | Lutetium-177 radioimmunoconjugate of the CD37-directed murine monoclonal antibody lilotomab | Relapsed rituximab/anti-CD20 refractory follicular lymphoma | Data showed attractive safety profile at 15 MBq/kg after a pre-dose of 40 mg lilotomab; study will remain open for recruitment | 6/21/22 | Cancer |
| Nykode Therapeutics ASA, of Norway | VB10.16 | DNA-based therapeutic cancer vaccine delivered via PharmaJet needle-free injection technology | Advanced cervical cancer | Interim data showed anti-tumor activity in a population of heavily pre-treated patients; durable responses; clinical benefit | 6/21/22 | Cancer |
| Rakuten Medical Inc., of San Mateo, Calif. | ASP-1929 (cetuximab sarotalocan) | Antibody-dye conjugate targeting EGFR | Head and neck squamous cell carcinoma | Enrolled and treated first patient | 6/21/22 | Cancer |
| Sapience Therapeutics Inc., of Harrison, New York | ST-101 | Antagonist of C/EBP? | Recurrent glioblastoma | Interim data (n=14) showed 1 patient with confirmed partial response after 18 weeks of therapy; 7 patients have not reached the first on-study assessment; 6 patients progressed; favorable safety profile; manageable mild-moderate infusion-related reaction | 6/2/22 | Cancer |
| Scancell Holdings plc, of Nottingham, U.K. | SCIB1 vaccine | Immunotherapy | Metastatic melanoma | Additional cohort to test vaccine in combination with checkpoint doublet therapy | 6/15/22 | Cancer |
| Sirnaomics Ltd., of Suzhou, China | STP-705 | siRNA-inhibiting polymeric nanoparticle-enhanced delivery to directly knock down both TGF-?1 and COX2 gene expression | Skin cancer | Results published in Journal of Drugs in Dermatology showed STP-705 was safe and well-tolerated; 76% of patients achieved histological clearance; 80% and 100% of patients achieved histological clearance at 30-mcg and 60-mcg doses, respectively | 6/16/22 | Cancer |
| Ultimovacs ASA, of Oslo | UV-1 | Targets human telomerase | Metastatic malignant melanoma | Completed recruitment of 154 patients in the Initium trial in combination with Yervoy (ipilimumab, Bristol-Myers Squibb Co.) and Opdivo (nivolumab, BMS); top-line progression-free survival data expected in first half of 2023 | 6/30/22 | Cancer |
| Verastem Inc., of Boston | VS-6766; defactinib | Oral RAF and MEK dual inhibitor; FAK inhibitor | Low-grade serous ovarian cancer patients | Interim analyses supports continued evaluation of monotherapy and combination therapy; independently confirmed responses achieved in both KRAS-mutant and KRAS wild-type tumors; approximately 80% of patients remain on therapy; company plans to complete enrollment of all 4 36-patient cohorts in the second half of 2022 | 6/6/22 | Cancer |
| Zenith Epigenetics Ltd., of Calgary, Alberta | ZEN-3694 | BET bromodomain inhibitor | Advanced breast cancer | Data in combination with talazoparib(Talzenna,Pfizer Inc.) showed clinically meaningful response rate of 32% in a defined population;manageable safety profile | 6/21/22 | Cancer |
| Huyabio International LLC, of San Diego | HBI-3000 | Multi-ion channel blocker | Cardioversion of atrial fibrillation | First 3 patients dosed | 6/14/22 | Cardiovascular |
| Xylocor Therapeutics Inc., of Wayne, Pa. | XC-001 | Gene therapy | Refractory angina | Achieved target enrollment; top-line data expected in Feb. 2023 | 6/28/22 | Cardiovascular |
| Cincor Pharma Inc., of Waltham, Mass. | Baxdrostat (CIN-107) | Highly selective aldosterone synthase inhibitor | Uncontrolled hypertension and chronic kidney disease | First patient dosed | 6/1/22 | Cardiovascular/Genitourinary/Sexual Function |
| Amytrx Therapeutics Inc., of Germantown, Md. | AMTX-100 CF3 | Peptide in a topical ointment formulation | Atopic dermatitis | Initiated phase II study to evaluate safety and efficacy of treatment over 28 days | 6/9/22 | Dermatologic |
| Cara Therapeutics Inc., of Stamford, Conn. | Difelikefalin | Analgesic opioid peptide | Moderate-to-severe pruritus in patients with notalgia paresthetica | Top-line data showed oral difelikefalin achieved the primary endpoint p=0.001 with significant improvement observed as early as week 1 and sustained through week 8; statistically significantly greater proportion of patients treated with oral difelikefalin achieved a ?4-point improvement in WI-NRS score at week 8 vs. placebo (41% difelikefalin vs. 18% placebo, p=0.007); well-tolerated | 6/30/22 | Dermatologic |
| Celldex Therapeutics Inc., of Hampton, N.J. | Barzolvolimab | Humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT | Chronic spontaneous urticaria | First patient dosed | 6/21/22 | Dermatologic |
| Dermata Therapeutics Inc., of San Diego | DMT-310 | Multifactorial, naturally derived product candidate from Spongilla lacustris | Moderate to severe rosacea | Completed enrollment; top-line results expected in second half of 2022 | 6/13/22 | Dermatologic |
| Dermbiont Inc., of Boston | SM-020 | AKT inhibitor | Seborrheic keratoses (SKs) | 52% of SKs lesions rated PLA 2 and PLA3 treated for 28 days were cleared completely, reaching PLA 0; 100% of SKs had at least a 1-point drop in their PLA score; plans to start a larger phase II study in early 2023 | 6/23/22 | Dermatologic |
| OliX Pharmaceuticals Inc., of Suwon, South Korea | OLX-101A | Targets Connect tissue growth factor | Hypertrophic scar | Completed patient enrollment | 6/3/22 | Dermatologic |
| Promore Pharma AB, of Stockholm | Ensereptide | Lactoferrin peptide | Prevention of skin scars | Target of 24 treated patients reached; results expected around the end of 2022 or start of 2023 | 6/13/22 | Dermatologic |
| Trevi Therapeutics Inc., of New Haven, Conn. | Haduvio (nalbuphine) | Oral, extended-release nalbuphine | Prurigo nodularis | Prism trial showed 25% of Haduvio subjects evaluated at week 14 met the primary endpoint of a 4-point reduction in WI-NRS from baseline compared to 14% of placebo subjects (p=0.0157); significantly greater improvements in ItchyQoL vs. placebo (p=0.0002) at week 14; statistically significant across each of the three domains (symptoms, functional limitations, and emotions); 55% of Haduvio subjects saw at least a 1-category improvement in the 5-point scale in their Prurigo Activity Scale (pruriginous lesions with excoriations), vs. 38% on placebo (p=0.006) as evaluated at week 14 | 6/29/22 | Dermatologic |
| Vyne Therapeutics Inc., of Bridgewater, N.J. | FMX-114 | Combination gel formulation of tofacitinib and fingolimod | Mild-to-moderate atopic dermatitis | Completed enrollment in phase IIa study | 6/17/22 | Dermatologic |
| 89bio Inc., of San Francisco | Pegozafermin | Specifically engineered glyco-Pegylated analog of fibroblast growth factor 21 | Severe hypertriglyceridemia | Top-line data showed trial met primary endpoint; statistically significant and clinically meaningful reductions across all doses (63% at the 27-mg; p<0.001) in triglycerides (tg) from baseline; significant and potent reductions atherogenic lipids (non-hdl-c apo b), liver fat, improvements enzymes glycemic control markers; well-tolerated; favorable safety profile< td> | 6/29/22 | Endocrine/Metabolic |
| Amolyt Pharma SA, of Lyon, France | AZP-3601 | Therapeutic peptide targeting parathyroid hormone receptor | Hypoparathyroidism | First cohort showed drug well-tolerated, with no safety concerns; daily administration to patients enabled discontinuation of the standard of care, while mean serum calcium was maintained within the target range; sustained reduction and normalization in 24-hour urinary calcium in all patients; mid-normal range increase in bone turnover as assessed by bone biomarkers | 6/1/22 | Endocrine/Metabolic |
| Cannalean Biotechs Ltd., of Tel Aviv, Israel | Formulation of cannabidiol and chitosan | Non-psychotropic component of cannabis; cholesterol and triglycerides lowering agents | Hypercholesterolemia in patients with hyperlipidemia | First patient enrolled | 6/21/22 | Endocrine/Metabolic |
| Novaremed AG, of Basel, Switzerland | NRD.E1 | Non-opioid development compound | Diabetic peripheral neuropathy | Study of 88 patients testing 3 weeks of treatment at 3 dose levels showed reductions in pain across primary and secondary endpoints; a phase IIb study is expected to begin recruitment in the third quarter of 2022 | 6/23/22 | Endocrine/Metabolic |
| 9 Meters Biopharma Inc., of Raleigh, N.C. | NM-002 (vurolenatide) | Long-acting injectable GLP-1 receptor agonist | Short bowel syndrome | Top-line data from Vibrant study showed well-tolerated; no adverse events; 7 of 11 patients met the primary efficacy definition of TSO responder (greater than or equal to 10% reduction from baseline in 24-hour mean TSO) over the 6-week efficacy evaluation period | 6/30/22 | Gastrointestinal |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | Spesolimab | Humanized, selective antibody that blocks the activation of the interleukin-36 receptor | Crohn's disease | Study terminated | 6/23/22 | Gastrointestinal |
| Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Results after 2-year treatment showed improvement in GLOBE score; younger patients tended to have numerically greater improvements; differences did not achieve significance; improved transplant-free survival | 6/22/22 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody that blocks TNF-like ligand 1A (TL1A) | Crohn's disease | Completed enrollment for Apollo-CD study; top-line data expected in fourth quarter 2022 | 6/8/22 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody that blocks TNF-like ligand 1A (TL1A) | Ulcerative colitis | Enrollment remain on track with enrollment completion of cohort 1 expected in the third quarter 2022; top-line data expected in the fourth quarter 2022 | 6/8/22 | Gastrointestinal |
| Sagimet Biosciences Inc., of San Mateo, Calif. | TVB-2640 (denifanstat) | Selective fatty acid synthase inhibitors | Nonalcoholic steatohepatitis | Trial met both primary endpoints of efficacy and safety; 28% relative reduction in liver fat at 50 mg; 56% responder rate; statistically significant improvements in inflammation/lipotoxicity, fibrosis and metabolic biomarkers; well-tolerated | 6/8/22 | Gastrointestinal |
| Takeda Pharmaceutical Co. Ltd., of Japan, and Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-AAT/TAK-999 (fazirsiran) | RNA interference therapeutics | Alpha-1 antitrypsin deficiency associated liver diseases | Results published in the New England Journal of Medicine showed reductions in accumulated total mutant AAT protein (Z-AAT) in the liver median percentage change at week 24 or 48, -83.3%; substantial mean reduction from baseline in serum Z-AAT concentration was observed in all cohorts with a nadir of -90±5% in the 200-mg cohort and -87±6% in the 100-mg cohort at week 6; decreased globule burden by 69% reduction mean score decreasing to 2.3 at week 24 or 48; liver injury biomarkers reduced; ALT concentrations decreased in all cohorts from week 16 through week 52; reduced ?-glutamyltransferase concentrations | 6/27/22 | Gastrointestinal |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Cemdisiran | RNAi therapeutic targeting the C5 component | IgA Nephropathy | Data at week 32 showed 37% mean reduction from baseline in the 24-hour urine protein to creatinine ratio relative to placebo; no significant drug-related safety signals; no serious or severe adverse events | 6/9/22 | Genitourinary/Sexual Function |
| Angion Biomedica Corp., of Uniondale, N.Y. | ANG-3070 | Oral tyrosine kinase inhibitor | Focal segmental glomerulosclerosis and immunoglobulin A nephropathy | Discontiuned Juniper trial in interest of patient safety based upon a reassessment of the risk/benefit profile | 6/29/22 | Genitourinary/Sexual Function |
| Global Blood Therapeutics Inc., of South San Francisco, Calif. | GBT-601 | Sickle hemoglobin (HbS) polymerization inhibitor | Sickle cell disease | Initiated phase II portion of planned phase II/III study; | 6/29/22 | Hematologic |
| Acelyrin Inc., of Los Angeles, Affibody AB, of Solna, Sweden, and Inmagene Biopharmaceuticals Co. Ltd., of Shanghai | Izokibep | IL-17A-inhibiting antibody mimetic | Psoriatic arthritis | Results from 16-week study showed 52% on 80-mg dose and 48% of those on 40-mg dose saw 50% improvement in RA symptoms (p=0.0006, p=0.0014) vs. 13% for placebo; Leeds Enthesitis Index showed 88% and 63% improvements for 80-mg and 40-mg doses, respectively (p=0.0003 and 0.0095) vs. 10% for placebo; 75% or greater reduction Psoriasis Area and Severity Index scores (PASI75) for 85% and 83% for 80-mg and 40-mg doses, respectively vs. 14% for placebo; as well as improvement in Psoriatic Arthritis Impact of Disease, a patient-reported measure of disease impact | 6/3/22 | Immune |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-109 | Polymer matrix-based epinephrine prodrug; oral drug | Anaphylaxis, severe allergy | Top-line data showed rapid absorption with favorable pharmacokinetics; median time to maximum concentration was observed to be 12 minutes vs. 50 minutes for the epinephrine 0.3-mg intra-muscular (IM) injection; consistent maximum plasma concentration vs. 0.3-mg IM injection; safe and well-tolerated | 6/16/22 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Pooled analysis showed 78.4%, 66% and 52.6% of patients in the voclosporin group and 62.4%, 47% and 33.1% of patients in the control group achieved ?25%, ?50% reduction in UPCR and achieved UPCR ?0.7 mg/mg (p<0.0001 6 12 at 3, and months, respectively); 89.6% of the voclosporin group 82.8% control achieved recommended steroid dose ?0.75 mg day according to protocol-defined taper (p="0.0396)" months< td> | 6/3/22 | Immune |
| Immunic Inc., of New York | Vidofludimus calcium (IMU-838) | Selective oral DHODH inhibitor | Relapsing multiple sclerosis | Top-line data from Caldose-1 study did not achieve the primary endpoint of clinical remission for the pooled 30-mg/day and 45-mg/day doses vs. placebo at week 10; no meaningful differences were observed between the 3 active doses (10 mg/day: 14.9%; 30 mg/day: 10.6%; 45 mg/day: 13.6%, placebo: 12.5%) | 6/2/22 | Immune |
| Immunic Inc., of New York | Vidofludimus calcium (IMU-838) | Selective oral DHODH inhibitor | Relapsing multiple sclerosis | Trial achieved all primary and key secondary endpoints; statistically significant reduction in the cumulative number of combined unique active (CUA) MRI lesions up to week 24 in patients receiving 45 mg of vidofludimus calcium once daily, by 62% (p=0.0002) as compared to placebo; statistically significant reduction in the cumulative number of CUA MRI lesions for the 30-mg once-daily dose, by 70% (p<0.0001) compared to placebo; well-tolerated; results published in annals of clinical and translational neurology< td> | 6/15/22 | Immune |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy product | Immunosuppression | Real-world, retrospective analysis of phase II patients vs. matched controls found FCR001-treated patients have improved kidney function and fewer cardiometabolic complications than patients on immunosuppression after 5 years; eGFR significantly higher in patients treated with FCR-001 at 5 years (p=0.02) | 6/7/22 | Immune |
| Vectivbio Holding AG, of Basel, Switzerland | Apraglutide | Long-acting GLP-2 analogue | Acute graft-vs.-host disease | First 2 patients dosed; interim data expected in the first half of 2023 | 6/2/22 | Immune |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel (ZYN-002) | Cannabidiol transdermal gel | 22q11.2 deletion syndrome | Top-line study showed trial achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments; total score and all 5 subscales of the Anxiety, Depression and Mood Scale (ADAMS) showed statistically significant improvements at 14 weeks of treatment compared to baseline; PARS-R showed statistically significant improvements at 14 weeks of treatment compared to baseline; majority of patients showed clinically meaningful improvements at week 14 as measured by CGI-I; well tolerated; consistent safety profile | 6/21/22 | Immune |
| 60 Degrees Pharmaceuticals LLC, of Washington | Arakoda (tafenoquine) | 8-aminoquinoline chemically derived from primaquine | Mild to moderate COVID-19 | Results published in New Microbes and New Infections showed drug increased the proportion of clinically recovered patients by between 9% (intent to treat population) and 14 % (per protocol population); decreased the proportion of clinically unrecovered patients by between 27% (intent to treat population) and 47% (per protocol population); time to clinical recovery from COVID-19 symptoms was accelerated by about 2-2.5 days | 6/21/22 | Infection |
| Aceragen Inc., of Raleigh-Durham, N.C. | ACG-701/ARV-1801 | Oral formulation of sodium fusidate | Melioidosis | First patient dosed | 6/15/22 | Infection |
| Adamis Pharmaceuticals Corp., of San Diego | Tempol | Membrane-permeable radical scavenger | COVID-19 | Data safety monitoring board recommended that the study continue without modification | 6/1/22 | Infection |
| Akston Biosciences Inc., of Beverly, Mass., and Biolexis Pvt Ltd., of Bangalore, India | AKS-452 | SARS-CoV-2 vaccine | COVID-19 | Results from 1,600 healthy volunteers showed no significant safety issues; 91% seroconversion rate at day 56; statistically-significant high antibody titers were maintained for six months; well-tolerated | 6/24/22 | Infection |
| Allovir Inc., of Waltham, Mass. | Viralym-M (posoleucel) | Allogeneic, off-the-shelf, mutivirus-specific T cell therapy | BK viremia in adult kidney transplant patients | Well-tolerated with no cases of graft rejection observed to date; declines in median BK viral load were observed in this blinded dataset irrespective of the baseline BK viral load | 6/7/22 | Infection |
| AN2 Therapeutics Inc., of Menlo Park, Calif. | Epetraborole | Boron-containing, orally-available, small molecule inhibitor of bacterial leucyl-tRNA synthetase | Treatment-refractory Mycobacterium avium complex (MAC) lung disease | Screened first patient | 6/29/22 | Infection |
| Arbutus Biopharma Corp., of Warminster, Pa., and Vaccitech Ltd., of Oxford, U.K. | AB-729; VTP-300 | N-acetylgalactosamine small interfering RNA conjugate targeting hepatitis B surface antigen; prime-boost HBV vaccine | Chronic hepatitis B infection | First of 40 patients dosed in trial testing the combination therapy for safety, antiviral activity and immunogenicity of VTP-300 administered after AB-729 in virologically suppressed chronic HBV patients | 6/6/22 | Infection |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-22 (envafolimab) | Single domain antibody against PD-L1 | HIV-1 infection | First subject dosed in combination with anti-retroviral therapy | 6/28/22 | Infection |
| Assembly Biosciences Inc., of South San Francisco | Vebicorvir | First-generation hepatitis B core inhibitor | Chronic hepatitis B | Phase IIa studies showed no clinically significant drug-drug interactions in patients | 6/22/22 | Infection |
| Dompe Farmaceutici SpA, of Milan, Italy | Reparixin | IL-8 receptor antagonist | Severe COVID-19 | Results, published in Infectious Diseases and Therapy, showed improvement in clinical outcomes vs. standard of care; rate of clinical events was statistically significantly lower in Reparixin group compared to standard of care (27% vs. 42.1%, p=0.02); treatment was well-tolerated in terms of treatment-emergent adverse events, laboratory test and vital sign parameters | 6/13/22 | Infection |
| Emergent BioSolutions Inc., of Gaithersburg, Md. | PXVX-0317 | Aluminum hydroxide-adjuvanted chikungunya virus-like particle vaccine | Chikungunya infection | Results published in the The Lancet Infectious Diseases showed well-tolerated; durable serum neutralizing antibody immune response against the Chikungunya virus up to two years | 6/21/22 | Infection |
| Kinarus Therapeutics Holding AG, of Basel, Switzerland | KIN-001 | Combination of pamapimod and pioglitazone | COVID-19 | Study enrolled 130 hospitalized COVID-19 patients; interim data expected in third quarter of 2022 | 6/20/22 | Infection |
| Medicinova Inc., of La Jolla, Calif. | MN-166 (ibudilast) | Small-molecule compound that inhibits PDE4 | Hospitalized COVID-19 patients at risk for developing acute respiratory distress syndrome | Top-line results showed achieved co-primary endpoint of the proportion of subjects free from respiratory failure at day 7; 71% of subjects in the MN-166 group and 35% of subjects in the placebo group were free of respiratory failure at day 7 (p=0.02); 71% of subjects in the MN-166 group and 47% of subjects in the placebo group had improved clinical status at day 7 (p=0.08); 65% of subjects in the MN-166 group and 29% of subjects in the placebo group were discharged from the hospital at day 7 (p=0.02); 0% of subjects in the MN-166 group and 24% of subjects in the placebo had worsened clinical status at day 7 (p=0.05); 2 deaths in the placebo group and no deaths in the MN-166; no serious adverse events | 6/8/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.214 containing mRNA-1273 (Spikevax) | mRNA vaccine | COVID-19 | Data showed mRNA-1273.214 boosted neutralizing titers against BA.4/BA.5 by 5.4-fold and by 6.3-fold in the subset of seronegative participants; neutralizing titers against BA.4/BA.5 were approximately 3-fold lower than previously reported neutralizing titers against BA.1 | 6/22/22 | Infection |
| Ocugen Inc., of Malvern, Pa., and Bharat Biotech International Ltd., of Hyderabad, India | Covaxin (BBV-152) | Whole-virion inactivated COVID-19 vaccine manufactured using a vero cell platform | COVID-19 | Results published in The Lancet Infectious Diseases showed safe and superior response to that shown in adults in children aged 2-18 years old | 6/21/22 | Infection |
| Pfizer Inc., of New York | Paxlovid | Nirmatrelvir (PF-07321332) tablets and ritonavir tablets | Severe COVID-19 | Updated analysis from 1,153 patients showed a non-significant 51% relative risk reduction; non-significant 57% relative risk reduction in hospitalization or death in subgroup analysis of 721 vaccinated adults; nominally significant 62% decrease in COVID-19-related medical visits per day across all patients relative to placebo (p=0.0228); 67% reduction in medical visits per day vs. placebo (p<0.0001, statistically significant); 72% reduction in the average number of days hospital among paxlovid-treated patients vs. placebo epic-sr; no deaths< td> | 6/14/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Comirnaty (tozinameran) | mRNA vaccine | COVID-19 | Data from booster dose of both omicron-adapted vaccine candidates showed substantially higher immune response against omicron BA.1 as compared to the other vaccine; monovalent omicron-adapted vaccine 30-µg and 60-µg doses achieved a lower bound 95% confidence interval for GMR of >1.5; booster dose of the omicron-adapted bivalent candidates conferred a 9.1 and 10.9-fold increase in neutralizing GMTs against omicron BA.1; well-tolerated | 6/25/22 | Infection |
| Prothione LLC, of Franklin, Tenn. | Prothione capsules | Pro-glutathione therapy | Mild to moderate COVID-19 | Data showed safe and well-tolerated; statistically significant decrease in viral load; mean change in viral load of COVID-19 from baseline to day 14 compared with placebo (p=0.0347); marked increase in red blood cell GSH levels from baseline to day 29 in Prothione of 156.9 vs. 107.3 for placebo | 6/14/22 | Infection |
| Puretech Health plc, of Boston | LYT-100-COV (deupirfenidone) | Selectively deuterated form of pirfenidone | Post-acute long COVID with respiratory complications | Data showed no treatment effect observed in patient population; substantial improvement in 6-minute walk test seen in both placebo and active groups; no statistically significant differences between treatment groups were observed; well-tolerated; no serious events | 6/14/22 | Infection |
| Todos Medical Ltd., of New York | Tollovir | Oral 3CL protease inhibitor and anti-cytokine therapeutic candidates | Hospitalized COVID-19 patients | Data showed no signals of treatment-associated risk | 6/30/22 | Infection |
| ABVC Biopharma Inc., of Fremont, Calif. | ABV-1505 | Plant-based drug; NET inhibitor | Attention-deficit hyperactivity disorder | 13 additional subjects enrolled in the study from a total of 23 subjects screened | 6/1/22 | Musculoskeletal |
| Biorestorative Therapies Inc., of Melville, N.Y. | BRTX-100 | Autologous stem cell-based therapeutic | Chronic lumbar disc disease | Site initiation for study, expected to enroll up to 99 patients | 6/13/22 | Musculoskeletal |
| Biorestorative Therapies Inc., of Melville,N.Y. | BRTX-100 | Autologous bone marrow-derived mesenchymal stem cells | Chronic lumbar disc disease | First patient enrolled | 6/30/22 | Musculoskeletal |
| Cyclerion Therapeutics Inc., of Cambridge, Mass. | CY-6463 | CNS-penetrant soluble guanylate cyclase stimulator | Mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes | Data from 8-participant open-label study showed well-tolerated; no reports of serious adverse events; showed improvements across multiple mitochondrial disease-associated biomarkers, inflammatory biomarkers, cerebral blood flow, and functional connectivity between neural networks; reductions in lactate were observed in 6 of 8 participants with a mean percentage change of 24% and a range of 7% to 46%; reductions in GDF-15 concentrations were observed in 4 of 8 participants with the greatest reductions (up to 39%) observed in participants with higher concentrations of GDF-15 at baseline; increase from baseline in cerebral blood flow were observed for 5 of 8 participants, ranging from 19% to 60% (mean change of 42%) | 6/28/22 | Musculoskeletal |
| Innocare Pharma Ltd., of Beijing | Orelabrutinib | BTK inhibitor | Systemic lupus erythematosus | SLE response index -4 response rates at week 12 were 50% (7/14), 61.5% (8/13) and 64.3% (9/14) for patients treated with 50 mg, 80 mg and 100 mg of orelabrutinib, respectively, compared to 35.7% (5/14) for placebo treatment | 6/6/22 | Musculoskeletal |
| Kangpu Biopharmaceuticals Ltd., of Shanghai | KPG-818 | Orally bioavailable Neomides molecular glue | Systemic lupus erythematosus | First patient dosed in U.S. phase IIa study | 6/12/22 | Musculoskeletal |
| Neurosense Therapeutics Ltd., of Cambridge, Mass. | PrimeC | Extended-release oral formulation composed of a fixed-dose combination of ciprofloxacin and celecoxib | Amyotrophic lateral sclerosis | First patient enrolled; expects to complete enrollment by the end of 2022 and to report top-line results in the second quarter of 2023 | 6/1/22 | Musculoskeletal |
| Neurosense Therapeutics Ltd., of Cambridge, Mass. | PrimeC (ciprofloxacin + celecoxib) | Extended-release oral formulation composed of a fixed-dose combination of ciprofloxacin and celecoxib | Amyotrophic lateral sclerosis | Study met its primary endpoint; 12 months of administration with PrimeC showed statistically significant changes in key ALS-related biomarkers were observed in patients | 6/27/22 | Musculoskeletal |
| Neuvivo Inc., of Palo Alto, Calif. | NP-001 | Disease-modifying, anti-inflammatory drug | Amyotrophic lateral sclerosis | Data showed significantly reduced baseline markers of microbial translocation (LPS, LBP, sCD163, IL-18) compared to placebo(p = 0.04, 0.006, 0.02, 0.02, respectively); plasma levels of wound healing epidermal growth factor and neopterin increased in patients treated with NP001 (p = 0.04 and 0.02, respectively) | 6/2/22 | Musculoskeletal |
| Scholar Rock Holding Corp., of Cambridge, Mass. | Apitegromab | Selective inhibitor of the activation of latent myostatin | Type 2 and type 3 spinal muscular atrophy | Data from 24-months showed sizable and sustained improvement in Hammersmith Functional Motor Scale-Expanded scores and substantial increase in Revised Upper Limb Module scores; no serious risks; enrollment progressing in Sapphire study | 6/17/22 | Musculoskeletal |
| Actinogen Medical Ltd., of Sydney | Xanamem | Blocks the excess production of cortisol and 11?-HSD1 enzyme | Alzheimer's disease | Dose-ranging phase Ib trial (n=107) in healthy older adults met primary safety, pharmacodynamic and efficacy endpoints; enhanced attention and working memory with statistically significant Cohen's d effect size achieved; high levels of target engagement in the brain; initiating phase II study in patients | 6/14/22 | Neurology/Psychiatric |
| Addex Therapeutics Ltd., of Geneva, Switzerland | Dipraglurant | Orally available mGlu5 NAM | Dyskinesia associated with Parkinson’s disease | Terminated study due to slow recruitment rate; COVID-19 related challenges negatively impacted patient recruitment | 6/17/22 | Neurology/Psychiatric |
| Alterity Therapeutics Ltd., of Melbourne, Australia | ATH-434 | Small molecule designed to inhibit the aggregation of pathological proteins | Multiple system atrophy | Initiated enrollment in New Zealand; expected to enroll 60 adult patients | 6/2/22 | Neurology/Psychiatric |
| Alzecure Pharma AB, of Stockholm | ACD-440 | TRPV1 antagonist | Peripheral neuropathic pain | First patient enrolled | 6/21/22 | Neurology/Psychiatric |
| Annexon Inc., of Brisbane, Calif. | ANX-005 | IgG4 mAB | Huntington's disease | Final data showed ANX-005 led to complete, durable target engagement of C1q; disease progression stabilized for all 9 months of study; generally well-tolerated, though three discontinuations were determined to be potentially drug-related | 6/7/22 | Neurology/Psychiatric |
| Apnimed Inc., of Cambridge, Mass. | AD-504 and AD-182 | Atomoxetine and trazodone combination; atomoxetine and an orexin antagonist combination | Obstructive sleep apnea | AD-504 showed significant reduction of the Apnea Hypopnea Index (p=0.024) and hypoxic burden (HB) p=0.003; AD-182 showed trend toward reduction in HB relative to placebo; favorable safety profile | 6/2/22 | Neurology/Psychiatric |
| Apnimed Inc., of Cambridge, Mass. | AD-109 | Combination of atomoxetine + aroxybutynin | Obstructive sleep apnea | FDA granted fast track designation | 6/28/22 | Neurology/Psychiatric |
| Athira Pharma Inc., of Bothell, Wash. | Fosgonimeton (ATH-1017) | Small molecule designed to enhance the activity of HGF and its receptor MET | Mild to moderate Alzheimer’s disease | Top-line data showed primary endpoint of change in biomarker ERP P300 latency was not statistically significant for the full study population in combination with standard of care; monotherapy suggested improvement in both ERP P300 latency and ADAS-Cog11 at week 26 compared to placebo in prespecified subgroup analysis; ADAS-Cog11, ADCS-CGIC and ADCS-ADL23 were not significant in treated subjects compared with placebo at 26 weeks; favorable safety profile over 26 weeks; well-tolerated | 6/22/22 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-05 (dextromethorphan-bupropion) | Oral NMDA receptor antagonist with multimodal activity | Major depressive disorder | Evolve trial showed drug substantially improved depressive symptoms, induced remission of depression; improved functioning in patients with at least 1 prior antidepressant treatment failure; mean MADRS total score at baseline was 32.2; mean improvements from baseline to weeks 1, 2 and 6 in MADRS total scores were -9.1 points, -13.3 points and -20.4 points, respectively (p<0.001 4 6 9 12 for all); madrs improvement were durable through month (-23.3 points, p<0.001) and (-24.5 p<0.001); remission of depression (madrs ?10) was achieved by 16%, 32% 46% patients at weeks 2, 6, respectively; substantial improvements on the sds seen all timepoints from a baseline value 17.5 (p<0.001 substantially reduced anxiety, induced anxiety in proportion patients; durable, with 75% remitting 78% 12; well-tolerated< td> | 6/1/22 | Neurology/Psychiatric |
| Braxia Scientific Corp., of Toronto | Psilocybin | Naturally occurring psychedelic compound | Resistant depression | Data showed clinically meaningful improvements in depression severity observed; 93% of participants retained to primary endpoint; no serious adverse events; zero suicide attempts to date | 6/2/22 | Neurology/Psychiatric |
| Cognition Therapeutics Inc., of Pittsburgh | CT-1812 | Binds to a receptor on neurons that regulates cellular damage response pathways | Dementia with Lewy bodies | First patient dosed in the 120-patient Shimmer trial testing 2 dose levels of CT-1812 for 6 months; endpoints include changes in cognitive performance, physical activity, pharmacokinetic and pharmacodynamic biomarkers and alpha-synuclein in the cerebrospinal fluid | 6/23/22 | Neurology/Psychiatric |
| Dyve Biosciences Inc., of Thousand Oaks, Calif. | DYV-702 | Transdermal cream formulation of sodium bicarbonate | Pain | Data showed significantly better median time to pain resolution of 24 hr vs. 72 hr for control (p=0.03); responder rate at 24 hr of 58% in the DYV-702 vs. 24% in the control (p=0.01); significant decrease in rescue medication usage in the full analyses set with only 6.3% in the active arm compared to 20% for the control arm (p=0.02); significant improvement in PROMIS PF-20 scores at 24 hours with 16.7 for DYV-702 vs. 9.4 for control (p<0.01); acceptable adverse event profile< td> | 6/2/22 | Neurology/Psychiatric |
| HMNC Brain Health, of Munich, Germany | KET-01 | Oral, prolonged-release formulation of ketamine | Treatment-resistant depression | First patient dosed | 6/8/22 | Neurology/Psychiatric |
| Incannex Healthcare Ltd., of Melbourne, Australia | IHL-42X | Cannabinoid combination | Obstructive sleep apnoea | Data showed IHL-42X reduced primary endpoint apnea hypopnea index relative to baseline at all three doses; group level the difference relative to baseline with low dose and medium dose was statistically significant (p<0.05); low dose ihl-42x reduced ahi by> 50% relative to baseline in 62.5% of subjects and by > 80% in 25% of subjects; reduced oxygen desaturation index compared to baseline to a greater extent than placebo p<0.05; improved sleep efficiency ; well-tolerated< td> | 6/3/22 | Neurology/Psychiatric |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | LX-9211 | Orally delivered, selective small molecule inhibitor of AAK1 | Painful diabetic neuropathy | Study achieved the primary endpoint; statistically significant reduction in average daily pain score (ADPS) at week 6 compared to placebo; statistically significant reduction in ADPS at week 6 compared to placebo | 6/29/22 | Neurology/Psychiatric |
| Newron Pharmaceuticals S.p.A., of Milan, Italy | Evenamide | Voltage gated sodium channel inhibitor | Treatment-resistant schizophrenia (TRS) | Addition of evenamide (7.5 mg, 15 mg, or 30 mg, twice daily) improved symptoms of psychosis in patients with chronic TRS, reflected by an approximately 12% reduction in the PANSS score, CGI-S improvement of 0.7, and CGI-C ratings indicating 77% of patients responded to the treatment | 6/7/22 | Neurology/Psychiatric |
| NLS Pharma Ltd., of Stans, Switzerland | Quilience | Mazindol extended release | Narcolepsy | Phase IIa fully enrolled; top-line results expected in September 2022; 90% of patients completing the trial to date have rolled over into the open-label extension study | 6/2/22 | Neurology/Psychiatric |
| Praxis Precision Medicines Inc., of Cambridge, Mass. | PRAX-114 | Positive allosteric modulator extrasynaptic GABAA receptor | Major depressive disorder | Aria study didn't meet statistical significance on primary endpoint of change from baseline in the 17-item Hamilton Depression Rating Scale Total Score at day 15, or on any secondary endpoints | 6/6/22 | Neurology/Psychiatric |
| Puretech Health plc, of Boston | LYT-300 | Oral prodrug of natural allopregnanolone | Neurological and neuropsychological conditions | Results from healthy adults showed LYT-300 achieved systemic blood levels approximately 9-fold greater compared to previous reports with oral allopregnanolone | 6/14/22 | Neurology/Psychiatric |
| Receptor Life Sciences Inc., of Seattle | RLS-103 | Dry powder inhaled cannabidiol | Acute anxiety within social anxiety disorder | First patient dosed | 6/29/22 | Neurology/Psychiatric |
| Scisparc Ltd., of Tel Aviv, Israel | Palmitoylethanolamide + dronabinol (SCI-110) | Combination of synthetic cannabinoid analog and THC | Tourette Syndrome | Launch of the trial is pending approvals by the Ministry of Health in both Germany and Israel | 6/29/22 | Neurology/Psychiatric |
| Tryp Therapeutics Inc., of San Diego | TRP-8802 | Psilocybin-based drug candidate | Binge eating disorder | First patient dosed | 6/9/22 | Neurology/Psychiatric |
| Vivoryon Therapeutics NV, of Halle, Germany | Varoglutamstat (PQ-912) | Targets glutaminyl cyclase | Alzheimer's disease | Independent data safety monitoring board for the Viviad study recommended using the 600-mg twice daily dose for the second half of the study; all subjects randomized to the treatment arm will receive the selected dose for 48 or 96 weeks; data to be presented at an upcoming medical conference | 6/23/22 | Neurology/Psychiatric |
| Xenon Pharmaceuticals Inc., of Burnaby, British Columbia | XEN-1101 | Kv7 potassium channel opener | Focal onset seizures | Subgroup analysis of the X-Tole study showed median reduction in monthly focal onset seizure frequency at week 1 was 55.4% for the 25-mg group (p<0.001), 3 6 12 41.5% for the 20-mg group (p="0.039)," and 39.1% 10-mg compared to 20.2% placebo group; in open-label extension study, patients remaining study at least months experienced a greater than 70% 80% reduction monthly focal onset seizure frequency, respectively; 19.6% 9.5% of experience freedom from seizures, respectively< td> | 6/22/22 | Neurology/Psychiatric |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Voxzogo (vosoritide) | C-type natriuretic peptide analogue; positive regulator of the signaling pathway downstream of FGFR3 | Achondroplasia | Study results from infants and children ages 0 to less than 5 years showed an improvement in height Z-score compared to placebo at 52 weeks; improvement in annualized growth velocity by 0.92cm/year; did not significantly impact upper-to-lower body segment ratio; consistent safety profile | 6/13/22 | Ocular |
| Iveric Bio Inc., of Parsippany, N.J. | Zimura (avacincaptad pegol) | Target and inhibit the cleavage of complement protein C5 | Geographic atrophy (GA) | Post-hoc analysis showed 30% reduction was observed in OCT-measured GA growth with Zimura at 12 months; 22% reduction in progressive ellipsoid zone loss/attenuation at 18 months was observed with Zimura compared to sham | 6/10/22 | Ocular |
| Altamira Therapeutics Ltd., of Hamilton, Bermuda | AM-125 (intranasal betahistine) | Histamine H3 receptor antagonist; histamine H1 receptor partial agonist | Acute vertigo | Top-line data from exploratory Travers trial showed good safety and tolerability at doses up to 20 mg administered 3 times daily for 4 weeks; resulted in dose- and time-dependent improvement in balance and vestibular function; at the end of treatment period, patients treated with AM-125 20 mg on average managed to maintain balance for 12.5 seconds vs. 7.5 seconds for placebo-treated patients (p=0.0242; least square means in repeated-measure ANCOVA model, per protocol population); while active group maintained that level during subsequent observation period, placebo group continued recovery course and ultimately converged with the former, resulting in a miss of the primary endpoint, which had been set at 6 weeks | 6/13/22 | Other/Miscellaneous |
| Innovent Biologics Inc., of San Francisco | Mazdutide (IBI-362) | GLP-1R and GCGR dual agonist | Overweight or obesity | Results showed primary and all key secondary endpoints were met; least squares (LS) mean percent change (absolute change) from baseline in body weight were -7.21%, -10.56% and -11.57% with mazdutide compared with 1.05% with placebo (p < 0.0001 for all comparisons); proportion of participants with ? 5% body weight loss from baseline was 58.1% (3 mg), 82.5% (4.5 mg), and 80.3% (6 mg) with mazdutide, compared with 4.8% with placebo; proportion of participants with ? 10% body weight loss from baseline was 19.4% (3 mg), 49.2% (4.5 mg), and 50.8% (6 mg) with mazdutide, compared with 0% with placebo; reduced body mass index, waist circumference, liver fat content and blood pressure; lowered lipid, transaminase and serum uric acid levels, and improved insulin sensitivity | 6/8/22 | Other/Miscellaneous |
| Mymd Pharmaceuticals Inc., of Baltimore | MYMD-1 | TNF-alpha production inhibitor | Aging | Initiated enrollment; efficacy data expected in the second half of 2022 | 6/21/22 | Other/Miscellaneous |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor | Elevated low-density lipoprotein cholesterol | 92.5% of patients treated with the 10-mg dose of obicetrapib plus high-intensity statin (HIS) achieved the apolipoprotein B (Apo) target of <80 mg dl, compared to 27.5% of patients treated with placebo plus his; 67.5% obicetrapib his achieved the apob target <65 10% 82.5% at 10-mg dose ldl cholesterol <70 20% his< td> | 6/6/22 | Other/Miscellaneous |
| Renibus Therapeutics Inc., of Dallas | RBT-1 | Stannous protoporphyrin and iron sucrose fixed-combination agent | Postoperative complications following cardiothoracic surgery | Prespecified interim data when compared to placebo showed trial achieved primary endpoint; statistically significant (p<0.0001) 2 increase in biomarkers of cytoprotective preconditioning; statistically significant (p<0.05) reduction days the icu and on ventilator among patients receiving active treatment; clinically meaningful rates aki (-52%) make30 (-78%); overall length hospital stay by days; (p<0.003) 30-day re-admission rates; well-tolerated safety profile< td> | 6/15/22 | Other/Miscellaneous |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NMDA receptor antagonist | Idiopathic pulmonary fibrosis and chronic cough | Proof-of-concept study reached database lock; top-line data expected in July 2022 | 6/13/22 | Respiratory |
| Axalbion SA, of Manchester, U.K. | AX-8 | Transient receptor potential melastatin 8 agonist | Refractory or unexplained chronic cough | Data showed reduction in cough frequency within the first 15 minutes after treatment and lasting for more than 4 hours at 40 mg; 44% reduction in cough frequency over 2 hours vs. 18% with placebo and a 35% reduction over 4 hours vs. 20% with placebo; improvements in patient- and clinician-reported outcomes over 14 days; no serious adverse events | 6/29/22 | Respiratory |
| Indivior plc, of Slough, U.K. | AEF-0117 | Synthetic signaling specific inhibitor engineered to inhibit the cannabinoid type 1 | Cannabis use disorder | First patient enrolled | 6/1/22 | Toxicity and Intoxication |
| ADC Therapeutics SA, of Lausanne, Switzerland | Zynlonta (loncastuximab tesirine) | CD19-targeted antibody-drug conjugate | Diffuse large B-cell lymphoma | First patient dosed in Lotis-9 study in combination with rituximab in unfit and frail patients; planning to enroll 80 patients | 7/20/22 | Cancer |
| Astellas Pharma Inc., of Tokyo, and Seagen Inc., of Bothell, Wash. | Padcev (enfortumab vedotin) | Antibody-drug conjugate targeting Nectin-4 | Unresectable locally advanced or metastatic urothelial cancer | Top-line results of EV-103 study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed 64.5% confirmed objective response rate; median duration of response not reached; consistent safety profile | 7/26/22 | Cancer |
| Athenex Inc., of Buffalo, N.Y. | Oral paclitaxel and encequidar | Taxane; P-glycoprotein inhibitor | Advanced breast cancer | Data showed significantly improved tumor response in patients compared to intravenous administration; positive trends in progression-free survival and overall survival | 7/25/22 | Cancer |
| Ayala Pharmaceuticals Inc., of Rehovot, Israel | AL-102 | Oral gamma-secretase inhibitor | Desmoid tumors | Completed dosing in monotherapy cohorts; MRI scan showed decrease in tumor size in most of the 13 patients who had reached the 16-week time point; 1 patient reached unconfirmed partial response at week 16 per RECIST; well-tolerated; no dose-limiting toxicities and no grade 4/5 adverse events | 7/5/22 | Cancer |
| Brooklyn Immunotherapeutics Inc., of Brooklyn, N.Y. | IRX-2 | Primary cell-derived multicytokine biologic immunotherapy | Head and neck cancer | Inspire study showed median event-free survival was 48.3 months and not reached in the control arm (p=0.62); no new safety signals; well-tolerated | 7/26/22 | Cancer |
| Calithera Biosciences Inc., of South San Francisco | Sapanisertib (CB-228) | Dual mTORC 1/2 inhibitor | Relapsed/refractory NRF2 (NFE2L2)-mutated squamous non-small-cell lung cancer | Enrolled first patient | 7/6/22 | Cancer |
| Cytomx Therapeutics Inc., of South San Francisco | Praluzatamab ravtansine | Conditionally activated antibody-drug conjugate comprising CD166-directed humanized monoclonal antibody conjugated to maytansinoid DM4 | HR+/HER2-non-amplified breast cancer | Study met its primary efficacy endpoint of confirmed objective response rate (ORR) of greater than 10% by central radiology review; clinical benefit rate was 40% at 24 weeks; median progression-free survival was 2.6 months; arm B did not pass protocol-defined futility boundary in triple-negative breast cancer; enrollment to arms B and C to be discontinued | 7/6/22 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Sarah Cannon Research Institute | DS-7300 | Antibody-drug conjugate targeting B7-H3 | Pretreated extensive-stage small-cell lung cancer | First patient dosed | 7/20/22 | Cancer |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | Trans sodium crocetinate (TSC) | Oxygen-enhancing therapeutic | Glioblastoma | Expects to initiate the trial by the end of 2022; dosing the first patient in the trial in the first quarter of 2023 | 7/26/22 | Cancer |
| Hutchmed Ltd., of Hong Kong, and Astrazeneca plc, of Cambridge, U.K. | Savolitinib (orpathys) and Tagrisso (osimertinib) | MET tyrosine kinase inhibitor and irreversible EGFR inhibitor | Non-small-cell lung cancer | Results from 193 efficacy evaluable patients showed trend toward improved response rates with increasing level of MET aberration; overall response rate (ORR) of 32%; median duration of response (DoR) was 8.3 months; median progression-free survival (mPFS) was 5.3 months; ORR was 49, DoR was 9.3 months and mPFS was 7.1 months in Savannah patients who met the criteria for higher cut-off levels of MET aberration | 7/12/22 | Cancer |
| Immvira Group Co., of Shenzhen, China | MVR-T3011 I.V. | Genetic engineered oncolytic herpes simplex virus | Cancer | Completed first 3 cohort escalation study; 1 subject at low-dose cohort had continuous 17-week stable disease with significant increase in IFN?+ CD8 cells in peripheral blood | 7/20/22 | Cancer |
| Leap Therapeutics Inc., of Cambridge, Mass. | DKN-01 | Anti-SSEDickkopf 1 (DKK1) antibody | Gastric or gastroesophageal junction cancer | Initiated part C of ongoing Distinguish study in combination with tislelizumab and chemotherapy | 7/12/22 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Enoblituzumab | Fc-optimized B7-H3-directed monoclonal antibody | Recurrent or metastatic squamous cell carcinoma of the head and neck | Closed phase II study in combination with checkpoint inhibitor; will discontinue the study based on an internal review of safety data; 6/7 fatal events; hemorrhagic events in both arms | 7/8/22 | Cancer |
| Maia Biotechnology Inc., of Chicago | Thio/6-thio-dG or 6-thio-2’-deoxyguanosine | Telomere-targeting agent | Advanced non-small-cell lung cancer | First patient dosed in combination with cemiplimab | 7/18/22 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | Next-generation anthracycline | Soft tissue sarcoma | Data suggested below 390 mg/m2 as recommended phase II dose; study to explore the efficacy as monotherapy; planning to initiate phase II recruitment | 7/28/22 | Cancer |
| Nordic Nanovector ASA, of Oslo, Norway | Betalutin (177Lu lilotomab satetraxetan) | Lutetium-177 radioimmunoconjugate of the CD37-directed murine monoclonal antibody lilotomab | Relapsed rituximab/anti-CD20 refractory follicular lymphoma | Following independent data evaluation, company will discontinue the study; no further patients to be enrolled; observed profile does not fully meet the objectives set | 7/5/22 | Cancer |
| Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | HER2-positive metastatic colorectal cancer | Study in combination with trastuzumab showed drug well-tolerated; median duration of follow-up of 20.7 months showed 38.1% confirmed objective response rate in HER2-positive patients; median duration of response was 12.4 months; median progression-free survival was 8.2 months and overall survival was 24.1 months; monotherapy showed 3.3% objective response rate and disease control rate of 80% | 7/2/22 | Cancer |
| Sotio Biotech Inc., of Cambridge, Mass. | SOT-101 | IL-15 superagonist | Advanced/metastatic solid tumors | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); planning to enroll 320 patients | 7/26/22 | Cancer |
| Spectrum Pharmaceuticals Inc., of Henderson, Nev. | Poziotinib | EGFR tyrosine kinase inhibitor | Non-small-cell lung cancer | Results published in Cancer Cell showed study met primary endpoint with manageable safety profile; overall response rate of 32% across all patients with EGFR exon 20 mutations; 46% overall response rate was observed for near-loop insertions and a 0% response rate was observed for far-loop insertions; objective response rate of 30% or greater; 32% and 31% ORR as assessed by the investigator and blinded independent review, respectively; median progression-free survival was 5.5 months; median duration of response was 8.6 months; median overall survival was 19.2 months; grade 1 or 2 toxicities | 7/11/22 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab (KN-035) | Single-domain antibody against PD-L1 | Solid tumors | Enrollment of 36th patient at the 600-mg dose; interim analysis expected in the fourth quarter of 2022 | 7/26/22 | Cancer |
| Cardior Pharmaceuticals GmbH, of Hanover, Germany | CDR-132L | Targets microRNA-13 | Heart failure | First patient dosed; study to evaluate the safety and efficacy in 280 patients; study consists of a 6-month double-blind period and 6-month extension period | 7/20/22 | Cardiovascular |
| Cardurion Pharmaceuticals Inc., of Boston | CRD-740 | PDE9 inhibitor | Heart failure | Opened multiple clinical sites and initiated dosing in patients | 7/19/22 | Cardiovascular |
| Cincor Pharma Inc., of Waltham, Mass. | Baxdrostat (CIN-107) | Highly selective aldosterone synthase inhibitor | Uncontrolled hypertension | Initiated open-label extension Halo study to evaluate the long-term safety for up to 52-weeks; last patient randomized; top-line data in the second half of 2022 | 7/26/22 | Cardiovascular |
| Scpharmaceuticals Inc., of Burlington, Mass. | Furoscix (furosemide injection) | Furosemide solution | Heart failure | Results from At Home-HF pilot study (n=51) showed a positive trend in the Finkelstein-Schoenfeld win ratio of the hierarchal primary composite endpoint; 37% reduction in the risk of heart failure hospitalization relative to patients randomized to treatment as usual at day 30; improvement in predefined secondary endpoints measuring symptoms of congestion, quality of life and functional status; 2-kg greater weight loss at day 3 and a 12-point increase in the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12) summary scores at day 7 and day 30; acceptable tolerability profile; 99.6% bioavailability | 7/12/22 | Cardiovascular |
| Sequana Medical NV, of Ghent, Belgium | First-generation DSR | Direct sodium removal agent | Congestive heart failure | Completed enrollment in phase IIa Sahara study | 7/19/22 | Cardiovascular |
| Celldex Therapeutics Inc., of Hampton, N.J. | Barzolvolimab | Humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT | Chronic inducible urticaria | First patient dosed; planning to enroll 180 patients in 2 cohorts | 7/21/22 | Dermatologic |
| Connect Biopharma Holdings Ltd., of San Diego | CBP-201 | Antibody designed to target interleukin-4 receptor alpha | Moderate to severe atopic dermatitis | Enrollment of 255 adult patients completed in the first half of 2022; top-line results expected by year-end 2022 | 7/11/22 | Dermatologic |
| Inmed Pharmaceuticals Inc., of Vancouver, British Columbia | INM-755 | Cannabinol cream | Epidermolysis bullosa | Independent data monitoring committee agreed to allow enrollment of adolescent patients for phase II study | 7/25/22 | Dermatologic |
| Merakris Therapeutics Inc., of Research Triangle Park, N.C. | Dermacyte amniotic wound care liquid | Cell-free amniotic therapy | Chronic venous stasis ulcers | First patient dosed in phase II trial | 7/27/22 | Dermatologic |
| Allena Pharmaceuticals Inc., of Newton, Mass. | ALLN-346 | Bioengineered enzyme specifically designed to degrade urate | Hyperuricemia | Completed enrollment in first 2 cohorts of study in gout and chronic kidney patients; top-line results expected in the third quarter 2022 | 7/19/22 | Endocrine/Metabolic |
| George Medicines Pty Ltd., of London | GMRx4 | Single pill, triple-component combination of metformin, dapagliflozin and sitagliptin | Type 2 diabetes | Advanced to phase II study; expected to commence later this year | 7/20/22 | Endocrine/Metabolic |
| Innovent Biologics Inc., of San Francisco and Suzhou, China | Mazdutide (IBI-362) | GLP-1 and glucagon receptor dual agonist | Diabetes type 2 | Study met primary endpoint in Chinese patients; significantly reduced HbA1c levels compared with placebo (p<0.0001); 20 proportion of patients with hba1c less than 7% at week were 62.8%; reduced body weight in a dose-dependent manner (p<0.0001); safe and well-tolerated< td> | 7/18/22 | Endocrine/Metabolic |
| Medicinova Inc., of La Jolla, Calif. | MN-001 (tipelukast) | Orally bioavailable, small-molecule compound | Nonalcoholic fatty liver disease, type 2 diabetes mellitus and hypertriglyceridemia | Initiated phase II study; planning to enroll 40 patients | 7/26/22 | Endocrine/Metabolic |
| Canbridge Pharmaceuticals Inc., of Beijing | CAN-108 | Maralixibat oral solution | Biliary atresia | First patient dosed; study to enroll up to 20 patients in China and 72 patients globally | 7/14/22 | Gastrointestinal |
| Innovent Biologics Inc., of San Francisco | IBI-112 | Recombinant anti-interleukin 23p19 subunit antibody | Moderate to severe active ulcerative colitis | First patient dosed | 7/3/22 | Gastrointestinal |
| Inventiva SA, of Daix, France | Lanifibranor | PPAR agonist | Noncirrhotic nonalcoholic steatohepatitis and type 2 diabetes | First patient screened in combination trial with empagliflozin; top-line data expected in the second half of 2023 | 7/7/22 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody that targets TNF-like ligand 1A (TL1A) | Ulcerative colitis | Completed enrollment for cohort 1 of Artemis-UC global phase II study; top-line results of Artemis-UC and Apollo-CD trials expected in the fourth quarter of 2022 | 7/18/22 | Gastrointestinal |
| Ovoca Bio plc, of Dublin | Orenetide | Synthetic peptide administered through a nasal spray | Premenopausal women with hypoactive sexual desire disorder | Completed enrollment of participants | 7/29/22 | Genitourinary/Sexual Function |
| Regulus Therapeutics Inc., of San Diego, and Sanofi SA, of Paris | Lademirsen (RG-012) | Single-stranded, chemically modified oligonucleotide that binds to and inhibits the function of miR-21 | Alport syndrome | Terminated phase II study for failure to meet Sanofi’s predefined futility criteria in the HERA study | 7/12/22 | Genitourinary/Sexual Function |
| Vera Therapeutics Inc., of Brisbane , Calif. | Atacicept | Recombinant fusion protein that contains the soluble transmembrane activator and calcium-modulating cyclophilin ligand interactor receptor that binds to the cytokines B lymphocyte stimulator and a proliferation-inducing ligand | IgA nephropathy | Completed patient enrollment ; top-line results expected in the fourth quarter 2022 | 7/6/22 | Genitourinary/Sexual Function |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Fesomersen | Antisense medicine | End-stage renal disease on hemodialysis/clotting disorders | Top-line data showed fesomersen achieved its primary outcome measure of no increase in incidence of major bleeding and clinically relevant non-major bleeding as compared to placebo; safe and well-tolerated; substantial and statistically significant reductions in factor XI activity levels | 7/28/22 | Hematologic |
| Aisa Pharma Inc., of Boston | Profervia | Once-daily, oral calcium channel antagonist | Secondary Raynaud’s disease associated with scleroderma | Data safety monitoring board (DSMB) for Reconnoiter study unanimously recommended continuing to next stage and approved a go-forward dose level; DSMB found no safety issues of concern and noted encouraging early efficacy trends | 7/27/22 | Immune |
| Atara Biotherapeutics Inc., of South San Francisco | ATA-188 | T-cell immunotherapy targeting EBV antigens | Progressive multiple sclerosis | Completed planned interim analysis; independent data and safety monitoring committee recommended continuing study without sample size adjustment; no safety issues; target enrollment achieved; final data expected in October 2023 | 7/12/22 | Immune |
| Biogen Inc., of Cambridge, Mass. | BIIB-059 (litifilimab) | Humanized IgG1 monoclonal antibody targeting blood dendritic cell antigen 2 | Cutaneous lupus erythematosus | Results published in The New England Journal of Medicine showed study met its primary endpoint; significantly reduced skin disease activity in patients compared to placebo at week 16; well-tolerated; mild to moderate adverse events | 7/28/22 | Immune |
| Diamyd Medical AB, of Stockholm | Diamyd | Diabetes vaccine | Latent autoimmune diabetes | Open-label investigator study initiated; positive immunological response to the treatment; good safety; no treatment-related severe adverse events; well-tolerated; completed the study with no drop-outs | 7/7/22 | Immune |
| Siolta Therapeutics Inc., of San Carlos, Calif. | STMC-103H | Live biotherapeutic product | Atopic disease | First newborn subject dosed | 7/20/22 | Immune |
| Adamis Pharmaceuticals Corp., of San Diego | Tempol | Membrane-permeable radical scavenger | COVID-19 | Data safety monitoring board to review interim data results has been scheduled for late September | 7/29/22 | Infection |
| Arbutus Biopharma Corp., of Warminster, Pa., and Assembly Biosciences Inc., of South San Francisco | AB-729 | RNAi therapeutic | Chronic hepatitis B infection | Continued dosing patients in triple-combination study of AB-729, vebicorvir and nucleoside derivative; preliminary data expected in the second half of 2022 | 7/20/22 | Infection |
| Ashvattha Therapeutics Inc., of Redwood City, Calif. | OP-101 | Conjugation of N-acetyl cysteine to hydroxyl dendrimer | Severe COVID-19 | Results published in Science Translational Medicine showed improved survival (2 mg/kg: 67%; 4 mg/kg: 100%; 8 mg/kg: 80%) vs. placebo (43%); risk for the composite outcome of mechanical ventilation or death at 30 and 60 days after treatment was 71% for placebo and 18% for the pooled OP-101 treatment arms; significantly decreased markers of hyperinflammation (TNF-?, IL-6 and IL-8 at 4 mg/kg); no drug-related adverse events | 7/25/22 | Infection |
| Hillevax Inc., of Boston | HIL-214 | Bivalent vaccine candidate; consists of virus-like particles designed to mimic the structure of 2 major genotypes of norovirus, GI.1 and GII.4 | Moderate to severe acute gastroenteritis caused by norovirus infection | Results from long-term immunogenicity follow-up study in combination with 500-µg alum showed no adverse events; no new safety risks; Pan-Ig and HBGA-blocking antibody responses persisted to year 5 | 7/20/22 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Antibody targeting GM-CSF | COVID-19 | Study in combination with remdesivir did not achieve statistical significance on the primary endpoint; non-significant trend toward a reduction in mortality in the overall patient population; no new safety signals | 7/12/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.214 containing mRNA-1273 (Spikevax) | mRNA vaccine | COVID-19 | New data after 1 month administration in previously vaccinated and boosted participants (50 mcg) showed significantly higher neutralizing antibody responses against the omicron subvariants BA.4 and BA.5 compared to the currently authorized booster (mRNA-1273) in adults; BA.4/5 geometric mean fold rise (GMFR) from pre-booster levels was 6.3-fold vs. 3.5-fold; well-tolerated; consistent safety profile | 7/11/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b5 | Next-generation bivalent COVID-19 mRNA-based vaccine | COVID-19 | Initiated study to evaluate safety, tolerability and immune response at 30-µg dose level | 7/27/22 | Infection |
| Syneurx International Corp., of Taiwan | Pentarlandir (SNB-01) | Antiviral candidate | COVID-19 | Results showed Pentarlandir reduced inflammation and showed greater reduction in inflammatory markers; improvement in overall health; improved secondary endpoints; greater reduction in total COVID-19 symptoms in the longer run (2-8 weeks) compared to placebo; well-tolerated in both high- and low-dose groups with no serious adverse events | 7/31/22 | Infection |
| Vaxart Inc., of South San Francisco | VXA-CoV2-1 | Spike/nucleocapsid (S+N) oral tablet | COVID-19 | Results from 35 healthy adult volunteers published in Medrxiv showed vaccine induced long-lasting mucosal IgA antibodies against SARS-CoV-2; higher neutralizing activity compared with convalescent samples; well-tolerated | 7/20/22 | Infection |
| Vaxcyte Inc., of San Carlos, Calif. | Vax-24 | 24-valent pneumococcal conjugate vaccine | Invasive pneumococcal disease and pneumonia | Completed enrollment; first subject dosed | 7/12/22 | Infection |
| Viiv Healthcare Ltd., of London | Cabotegravir | Long-acting agent; integrase strand transfer inhibitor | HIV infection | Results from pre-exposure prophylaxis (PrEP) in women in sub-Saharan Africa showed superior efficacy in the prevention of new HIV infections when compared to daily oral emtricitabine/tenofovir disoproxil fumarate; 89% lower rate of HIV acquisition; well-tolerated; no new safety issues | 7/28/22 | Infection |
| Mymd Pharmaceuticals Inc., of Baltimore | MYMD-1 | TNF-alpha production inhibitor | Chronic inflammation associated with sarcopenia | Completed dosing for the first patient; safety review committee confirmed no safety issues and no toxicity; voted unanimously to escalate to the next higher dose | 7/26/22 | Inflammatory |
| Bone Therapeutics SA, of Mont-Saint-Guibert, Belgium | Allob | Off-the-shelf allogeneic cell therapy platform consisting of human allogeneic bone-forming cells derived from cultured bone marrow mesenchymal stromal cells from healthy adult donors | High-risk tibial fractures | Interim analysis showed good tolerability; increased bone formation and other clinical benefits; improved statistical analysis, the number of required patients reduced by 20% | 7/15/22 | Musculoskeletal |
| Clene Inc., of Salt Lake City | CNM-Au8 | Oral suspension of gold nanocrystals | Amyotrophic lateral sclerosis | Data from long-term open-label extension for its Rescue-ALS trial showed median survival from randomization for the CNM-Au8 group was undefined due to insufficient mortality events and median survival for the placebo group was 23.1 months; significant survival benefit with participants compared to placebo (70% decreased risk of death, p = 0.0143); well-tolerated and no significant safety findings | 7/14/22 | Musculoskeletal |
| Endo International plc, of Dublin | Collagenase clostridium histolyticum | Collagenase enzyme | Adhesive capsulitis (frozen shoulder) | Study results from 3 doses showed difference compared to placebo was not statistically significant for improvement in the change from baseline in the adapted American Shoulder and Elbow Surgeons Standardized Shoulder Form composite score for the affected shoulder at day 95 visit | 7/1/22 | Musculoskeletal |
| Neuraptive Therapeutics Inc., of Philadelphia | NTX-001 | Undisclosed drug + nerve fusion technology | Facial paralysis | First patient enrolled; top-line data expected in the second half of 2023 | 7/21/22 | Musculoskeletal |
| Seelos Therapeutics Inc., of New York | SLS-005 | Trehalose injection, 90.5 mg/mL for intravenous infusion | Amyotrophic lateral sclerosis | First patient dosed in Australia; expected to complete enrollment this quarter in Healey ALS platform study; top-line data expected in mid-2023 | 7/18/22 | Musculoskeletal |
| Alterity Therapeutics Ltd., of San Francisco | ATH-434 | Small molecule designed to inhibit the aggregation of pathological proteins | Multiple system atrophy | First patient dosed | 7/6/22 | Neurology/Psychiatric |
| Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Oral agent to fully inhibit the formation of neurotoxic soluble amyloid oligomers | Alzheimer’s disease | Interim data showed significant memory improvement from baseline over 6 months in parallel with the p-tau181 reduction; favorable safety profile and no events of vasogenic edema; several-fold higher brain burden of neurotoxic amyloid oligomers | 7/26/22 | Neurology/Psychiatric |
| Compass pathway plc, of London | COMP-360 | Psilocybin therapy | Anorexia nervosa | Launched a multicenter, double-blind randomized controlled phase II study | 7/28/22 | Neurology/Psychiatric |
| Denovo Biopharma LLC, of San Diego | DB-104 (liafensine) | Triple reuptake inhibitor targeting transporters for dopamine, serotonin and norepinephrine | Treatment-resistant depression | First patient dosed | 7/28/22 | Neurology/Psychiatric |
| Impel Pharmaceuticals Inc., of Seattle | INP-105 | Nasal olanzapine; second-generation antipsychotic | Agitation in autism spectrum disorder | First subject dosed; results expected in first quarter of 2023 | 7/19/22 | Neurology/Psychiatric |
| Olipass Corp., of Seoul, South Korea | OLP-1002 | SCN9A antisense peptide nucleic acid; selectively inhibits the expression of Nav1.7 sodium channel | Osteoarthritis patients with pain | Moved to the second stage of the phase IIa study in Australia; open-label study showed pain reduction by 60% to 85% over the period of 30 days post dose | 7/18/22 | Neurology/Psychiatric |
| Synaptogenix Inc. (formerly Neurotrope Bioscience Inc.), of New York | Bryostatin-1 | Activator of protein kinase C | Alzheimer's disease | First patient dosed; top-line data expected in the fourth quarter of 2022 | 7/20/22 | Neurology/Psychiatric |
| Tetra Therapeutics, of Grand Rapids, Mich., a unit of Osaka, Japan-based Shionogi & Co. Ltd. | BPN-14770 (zatolmilast) | Phosphodiesterase?4D allosteric inhibitor | Fragile X syndrome | Initiated 3 parts of phase IIb/III study; studies comprise 2 separate 150 subjects of adolescent male population, adult male population (18-45) and open-label extension study | 7/12/22 | Neurology/Psychiatric |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-601 ER | Tianeptine oxalate extended-release tablet | Major depressive disorder | Planning to initiate phase II study in the first half of 2023 | 7/11/22 | Neurology/Psychiatric |
| Vivoryon Therapeutics NV, of Halle, Germany | Varoglutamstat (PQ-912) | Targets glutaminyl cyclase | Alzheimer's disease | Data showed well-tolerated; 14% overall reported adverse events; no clinical signs of on-target toxicity; no amyloid-related imaging abnormalities; enrolling patients at 22 study centers to evaluate its primary and secondary outcome measures | 7/31/22 | Neurology/Psychiatric |
| Bridgebio Pharma Inc., of Palo Alto, Calif. | Infigratinib | FGFR3 inhibitor | Achondroplasia | Interim data showed mean increase in annualized height velocity (AHV) was 1.52 cm/yr over baseline (p=0.02) at highest dose level (0.128 mg/kg); 64% were responders (defined with a strict criterion of an increase ?25% in AHV from baseline) and the average percent change from baseline in AHV was 60%; increase in AHV over baseline of 0.22 cm/yr (p=0.54) in children 5 and older; no-treatment related adverse events; well-tolerated; no serious adverse events; median duration of follow-up of 48.1 weeks | 7/26/22 | Ocular |
| Kiora Pharmaceuticals Inc., of Salt Lake City | KIO-201 | Topical eye drops; chemically modified form of the natural polymer hyaluronic acid | Persistent corneal epithelial defect | Enrolled first patient | 7/5/22 | Ocular |
| Nanoscope Therapeutics Inc., of Bedford, Texas | MCO-010 | Multicharacteristic opsin ambient-light activatable optogenetic monotherapy | Stargardt disease | First patient dosed; six-month safety and efficacy data are expected in the first quarter of 2023 | 7/25/22 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | Suprachoroidal delivery; consisting of NAV AAV8 vector encoding antibody fragment designed to inhibit VEGF | Wet age-related macular degeneration | Completed enrollment in cohort 5 of the phase II Aaviate trial | 7/8/22 | Ocular |
| Yuyu Pharma Inc., of Jecheon, South Korea | YP-P10 | Patented synthetic peptide | Dry eye disease | Enrolled first patient | 7/20/22 | Ocular |
| Anebulo Pharmaceuticals Inc., of Austin, Texas | ANEB-001 | Small molecule; competitive CB1 antagonist | Acute cannabinoid intoxication | Top-line data showed study met primary endpoint; statistically significant reduction in key symptoms; 10% of subjects in 50-mg and 30% in 100-mg groups reporting feeling high compared to 75% of subjects in placebo group (p<0.001); well-tolerated in volunteers; improvement subjective cns symptoms using visual analogue scale assessment (p<0.01)< td> | 7/5/22 | Other/Miscellaneous |
| Clairvoyant Therapeutics Inc., of Vancouver, British Canada | Psilocybin | Psychedelics | Alcoholism | Initiated the first of 15 planned sites | 7/6/22 | Other/Miscellaneous |
| Evolus Inc., of Newport Beach, Calif. | Jeuveau (prabotulinumtoxinA) | Neurotoxins | Treatment of glabellar lines | Completed patient enrollment; study to evaluate an extra-strength dose for extended duration | 7/12/22 | Other/Miscellaneous |
| Radius Health Inc., of Boston | RAD-011 | Synthetic cannabidiol oral solution | Hyperphagia and related neurobehavioral symptoms in Prader-Willi syndrome | First patient randomized in Scout-15 study | 7/7/22 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | MC4R agonist | Severe obesity and hyperphagia in people with hypothalamic obesity | Interim results (n=11) showed -17.2% mean percentage change in BMI; -15.9-kg or -35.1-lb mean weight loss from baseline; results from 9 patients showed -19.5% mean percent change in BMI and -17.8-kg or -37.7-lb mean weight loss from baseline; meaningful reduction in hunger scores | 7/12/22 | Other/Miscellaneous |
| Sling Therapeutics Inc., of Ann Arbor, Mich. | Linsitinib | Small molecule IGF-1R inhibitor | Thyroid eye disease | Initiated enrollment in global phase IIb study to evaluate the safety, pharmacokinetics and efficacy | 7/25/22 | Other/Miscellaneous |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | Ifenprodil | NMDA receptor antagonist | idiopathic pulmonary fibrosis and chronic cough | Data showed 65% of patients had stable or improved forced vital capacity over the 12-week treatment period with statistical significance when compared to an anticipated placebo effect (40%); 30% of subjects achieved the endpoint of a 50% reduction in the average number of coughs per hour over 24 hours from baseline to week 12; study did not achieve statistical significance of primary endpoint compared to placebo; 24% relative reduction from baseline in mean cough count and 38% relative reduction from baseline in median cough count in 24-hour cough count per hour at week 12 (p=0.0344); 75% of subjects saw improvements in their cough over 12 weeks; no new safety signals | 7/18/22 | Respiratory |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | Ifenprodil (NP-120) | NMDA receptor antagonist | Idiopathic pulmonary fibrosis and chronic cough | Results showed significant improvement in mean objective 24-hour and waking cough counts after 4 and 12 weeks; geometric mean 24-hour cough counts were reduced by 32% at 4 weeks (p=0.023) and 39.5% at 12 weeks (p=0.001) compared to baseline; geometric mean awake cough counts were reduced by 30.2% at 4 weeks (p=0.038) and 37.4% at 12 weeks (p=0.002) compared to baseline | 7/28/22 | Respiratory |
| Pliant Therapeutics Inc., of South San Francisco | PLN-74809 | Oral, small-molecule, dual-selective inhibitor of ?v?6 and ?v?1 | Idiopathic pulmonary fibrosis | Study met its primary and secondary endpoints; well-tolerated over a 12-week treatment period; favorable pharmacokinetic profile; no discontinuations due to adverse events; no deaths or serious events; dose-proportional increases in plasma concentrations; 80% reduction in forced vital capacity decline at 12 weeks (38% and 66% at 40-mg and 160-mg doses, respectively); mean percentage change in QLF at 12 weeks was 3.15%, 0.70% and 0% in the 40-mg, 80-mg and 160-mg treatment groups, respectively, vs. 1.15% in placebo arm | 7/10/22 | Respiratory |
| ABVC Biopharma Inc., of Fremont, Calif. | ABV-1703 | Extract from Grifola frondosa mushroom | Pancreatic cancer | Planning to initiate phase II study in the second quarter of 2023 | 8/22/22 | Cancer |
| Aim Immunotech Inc., of Ocala, Fla. | Ampligen ( rintatolimod) | Interferon?-2b | Pancreatic cancer | Institutional review board approved trial protocol; initiated study; expected to enroll 90 subjects | 8/18/22 | Cancer |
| Aim Immunotech Inc., of Ocala, Fla. | Type-1 polarized dendritic cells, Ampligen (rintatolimod) and celecoxib | Cell therapy, derivative of inosinic acid, NSAID | HLA-A2+ refractory melanoma | Started enrollment in the 24-patient study testing Roswell Park Comprehensive Cancer Center’s dendritic cell therapy with Aim’s Ampligen; primary endpoint is objective response rate at 12 weeks; progression-free survival and overall survival will also be measured | 8/30/22 | Cancer |
| Alpha Biopharma Ltd., of Shanghai | Zorifertinib | Next-generation EGFR-TKI | Non-small-cell lung cancer | Completed last patient visit in Everest phase II/III study; top-line data expected in the end of 2022 | 8/24/22 | Cancer |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-046 | PD-L1/CTLA4 bispecific antibody | Advanced non-small-cell-lung cancer | First patient dosed in combination with axitinib | 8/9/22 | Cancer |
| Alx Oncology Holdings Inc., of South San Francisco | Evorpacept | CD47 blocker | Refractory microsatellite stable metastatic colorectal cancer | First patent dosed in combination with cetuximab and pembrolizumab | 8/11/22 | Cancer |
| Amphera BV, of 's-Hertogenbosch, the Netherlands | Mesopher | Comprises autologous dendritic cells loaded with Pheralys | Pleural mesothelioma | Last patient completed in the active follow-up in the Denim study; top-line results expected in the early fourth quarter 2022 | 8/1/22 | Cancer |
| Artios Pharma Ltd., of Cambridge, U.K. | ART-4215 | Polymerase theta inhibitor; oral small molecule | BRCA-deficient breast cancer | Initiated phase II study in combination with talazoparib; enrolling up to 206 patients | 8/10/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Hutchmed Ltd., of Hong Kong | Savolitinib (orpathys) and Tagrisso (osimertinib) | MET tyrosine kinase inhibitor and irreversible EGFR inhibitor | Non-small-cell lung cancer | Results from Savannah study showed an objective response rate (ORR) of 49%; ORR of 52% in patients with high levels of MET who were not treated with prior chemotherapy; ORR was 9% in patients whose tumors did not show high levels of MET; disease control rate of 75% in patients with high level of MET; no new safety signals | 8/8/22 | Cancer |
| Beyondspring Inc., of New York | Plinabulin | Selective immunomodulating microtubule-binding agent; antigen presenting cell inducer | Multiple myeloma | Data showed well-tolerated in combination with pegfilgrastim; ongoing enrollment; full results at later date | 8/29/22 | Cancer |
| Bioatla Inc., of San Diego | Mecbotamab vedotin (BA-3011) | Conditionally and reversibly active antibody-drug conjugate targeting the receptor tyrosine kinase AXL | Non-small-cell lung cancer | Data from 9 efficacy-evaluable patients showed 1 complete response and 2 partial responses; combined objective response rate of 33%; safe and well-tolerated in both monotherapy and combination with nivolumab; no new safety signals; progression-free survival rate of 60% and 50% in liposarcoma and synovial sarcoma patients, respectively | 8/10/22 | Cancer |
| Bioatla Inc., of San Diego | Ozuriftamab vedotin (BA-3021) | Conditionally and reversibly active antibody-drug conjugate directed against ROR2 | Non-small-cell lung cancer | Interim data expected in the second half of 2022 in ROR2-postive patients; ongoing trial in melanoma patients | 8/10/22 | Cancer |
| Genexine Inc., of Seoul, South Korea | GX-188E, GX-I7 and Opdivo (nivolumab) | DNA vaccine, long-acting interleukin 7 and PD-1 immune checkpoint inhibitor | Head and neck squamous cell carcinoma | First patient dosed; to evaluate the safety and efficacy in 21 participants | 8/17/22 | Cancer |
| Immunogen Inc., of Waltham, Mass. | Pivekimab sunirine | Antibody-drug conjugate targeting CD123 | Blastic plasmacytoid dendritic cell neoplasm | Data from Cadenza study showed 2/4 de novo patients achieved complete response; 4/6 patients with prior or concomitant hematologic malignancy achieved complete response; top-line data expected in 2024 | 8/31/22 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha | Soluble LAG-3 fusion protein | Non-small-cell lung cancer | Interim data from TACTI-002 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed 36.5% of patients survived for at least 18 months; median overall survival of 9.7 months; 36.1% disease control rate; disease control (progression-free) in 25% of patients at 6 months; durable responses of 10+ months in 5.6% of patients, with both patients continuing in the trial for over 11 months and 24+ months; safe and well-tolerated; no new safety signals | 8/1/22 | Cancer |
| Janssen Pharmaceutical Co., a unit of Johnson & Johnson, of New Brunswick, N.J. | Darzalex (daratumumab) | Monoclonal antibody targeting CD38 | Newly diagnosed, transplant-eligible multiple myeloma | In the Griffin study, patient treated with Darzalex plus Revlimid, Velcade and dexamethasone (RVd) followed by maintenance therapy of Darzalex and Revlimid produced a stringent complete response rate of 42.4% compared to 32% for RVd followed by Revlimid maintenance therapy (p=0.0680); Darzalex-RVd plus Darzalex-R reduced the likelihood of progression-free survival by 55% compared to RVd plus R (p=0.0324) | 8/27/22 | Cancer |
| Jounce Therapeutics Inc., of Cambridge, Mass. | Vopratelimab | Monoclonal antibody targeting inducible costimulator | Immunotherapy naïve, TIS-vopra biomarker-selected, second line non-small cell lung cancer | In the select trial, vopratelimab plus pimivalimab didn’t meet its primary endpoint of mean tumor change averaged over 9 and 18 weeks compared to pimivalimab alone with an absolute difference of 7%; overall response rate was 40% for combination compared to 25% for pimivalimab alone | 8/30/22 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib | Brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway | Glioblastoma | Completed recruitment; arm did not meet predefined criteria; study will not open to the paxalisib arm in Germany or China | 8/1/22 | Cancer |
| Merrimack Pharmaceuticals Inc., of Cambridge, Mass. | Onivyde | Liposomal injection of irinotecan | Second-line small-cell lung-cancer | Results of phase III primary endpoint of overall survival was not met in patients treated with Onivyde vs. topotecan; secondary endpoint of objective response rate was favorable; no new safety concerns emerged | 8/3/22 | Cancer |
| Olema Pharmaceuticals Inc., of San Francisco | OP-1250 | Complete estrogen receptor (ER) antagonist; selective ER degrader | ER+/HER2-negative advanced breast cancer | Advanced to phase II study; recommended phase II dose of 120 mg; initiated enrollment for 3 cohorts; planning to initiate a pivotal monotherapy trial in mid-2023 | 8/10/22 | Cancer |
| Oncoinvent A/S, of Oslo, Norway | Radspherin | Alpha-emitting radioactive microsphere suspension | Peritoneal carcinomatosis from colorectal carcinoma | First patient treated; trial designed to assess the safety, tolerability and antitumor activity | 8/25/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 | T-cell HPV-specific immunotherapy | Recurrent or metastatic HPV16-positive head and neck cancer | Independent data monitoring committee met for its scheduled review for trial testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.); safe and well-tolerated in 43 patients; no drug discontinuations related to toxicity; recommended continuing the trial with no modifications | 8/4/22 | Cancer |
| Sirnaomics Ltd., of Suzhou, China | STP-705 | siRNA oligonucleotides targeting TGF-?1 and COX-2 mRNA formulated in nanoparticles with a histidine-lysine co-polymer | Cutaneous basal cell carcinoma | Data showed 100% complete response at 180-mcg dose; excellent safety profile; no significant cutaneous skin reactions; final report expected in the first quarter of 2023 | 8/29/22 | Cancer |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Oral ethyl-eicosapentaenoic acid | Coronary artery disease | Data from Reduce-IT study shouwed significantly reduced ST-segment elevation myocardial infarction (STEMI) (p=0.0008) and non-ST segment elevated myocardial infarction (NSTEMI) (p=0.001) by 40% and 27%, respectively; reduced the primary and secondary composite endpoint by 25% and 26%, respectively; significantly reduced myocardia infarction P<0.0001; statistically significant reductions in infarction subtypes leading to cardiac arrest p="0.01;" resuscitated significantly reduced the overall burden of total stemi and nstemi (p="0.0006" 0.002) vs. placebo< td> | 8/26/22 | Cardiovascular |
| Cardiol Therapeutics Inc., of Oakville, Ontario | Cardiolrx | Oral cannabidiol formulation | Acute myocarditis | First patient enrolled in Archer study | 8/3/22 | Cardiovascular |
| Cincor Pharma Inc., of Waltham, Mass. | Baxdrostat (CIN-107) | Highly selective aldosterone synthase inhibitor | Treatment-resistant hypertension | Top-line data showed baxdrostat met its primary endpoint and achieved statistically significant placebo-adjusted reduction in systolic blood pressure (SBP), including 11 mmHg (p< 0.0001) at 2 mg; 17.5-mmHg SBP reduction at the 1-mg dose, or 8.1 mmHg placebo-adjusted decline (p=0.0030); 12.1-mmHg SBP reduction at the 0.5-mg dose, or 2.7-mmHg placebo-adjusted decline (p=0.3110); baxdrostat significantly lowered diastolic blood pressure by 5.2 mmHg in the 2-mg dose; well-tolerated; no clinical safety concerns | 8/8/22 | Cardiovascular |
| Hemostemix Inc., of Calgary, Alberta | ACP-01 | Angiogenic cell precursors | Heart failure | Four months after ACP-01 implantation, ejection fraction of the left ventricle (LVEF) increased by 4.6 percentage points (p=0.0011); 12 months after implantation LVEF improved by 7.69 percentage points (p=0.003); ischemic heart failure patients had an 8.37 percentage point improvement in LVEF at 12 months (p=0.003); non-ischemic dilated cardiomyopathy patients had a 14.35 percentage point improvement in LVEF at 12 months | 8/30/22 | Cardiovascular |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Cholesteryl ester transfer protein inhibitor; oral, low-dose | High risk for cardiovascular disease | Results published in the Nature Medicine showed clinically meaningful and significantly lowered low density lipoprotein cholesterol (LDL-C) in patients on high-intensity statins; dose-dependent statistically significant changes from baseline in apolipoprotein B, non-high density lipoprotein cholesterol concentration, HDL-C concentration and lipoprotein(a); well-tolerated | 8/11/22 | Cardiovascular |
| Anaptysbio Inc., of San Diego | Imsidolimab | Anti-interleukin-36 receptor monoclonal antibody | Moderate to severe hidradenitis suppurativa | Top-line data from its Harp study showed safe and well-tolerated; did not demonstrate improvement over placebo in the primary endpoint and key secondary endpoints; no imsidolimab-related serious or severe adverse events reported; discontinued clinical devolpment; | 8/31/22 | Dermatologic |
| Aristea Therapeutics Inc., of San Diego | RIST-4721 | CXCR2 antagonist | Hidradenitis suppurativa | Treated first of 33 patients in the study comparing treatment with RIST-4721 for 12 weeks to placebo; primary endpoint is safety; secondary endpoint is proportion of patients achieving HiSCR50 at week 12 | 8/30/22 | Dermatologic |
| Innovent Biologics Inc., of San Francisco | IBI-112 (picankibart) | Recombinant anti-interleukin 23p19 subunit antibody | Moderate to severe plaque psoriasis | Study met primary endpoint; 98% completed primary endpoint at week 16; 94.4% completed the visit at week 28; 52% to 54.9% of the subjects achieved PASI 90 after picankibart treatment at week 16; achieved PASI 75 and PASI 100 significantly higher than placebo group (all p-values?0.001); well-tolerated; no new safety signals; significant increase from baseline in the dermatology life quality index (DLQI) of subjects treated with each dose of picankibart compared to placebo (all p-values?0.0001) | 8/10/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | KX-826 (pyrilutamide) | Androgen receptor antagonist | Male androgenetic alopecia | Completed the enrollment of 121 patients in the U.S.; trial to evaluate the efficacy and safety in adult males; primary endpoint is the change from baseline in non-vellus target area hair counts (TAHC) at week 24 compared with placebo | 8/3/22 | Dermatologic |
| Timber Pharmaceuticals Inc., of Basking Ridge, N.J. | TMB-001 | Topical isotretinoin formulated using IPEG delivery system | Moderate to severe congenital ichthyosis | Results published in the Journal of the American Academy of Dermatology showed clinically meaningful efficacy with a favorable safety profile; 100%, 40%, and 40% of patients receiving 0.05%, 0.1%, and vehicle achieved VIIS-50, respectively (p=0.04 for TMB-001 0.05% vs. vehicle) in per-protocol population (PP); 64%, 40%, and 33% of patients receiving 0.05%, 0.1%, and vehicle achieved VIIS-50, respectively (p=0.17 for TMB-001 0.05% vs. vehicle) in intent to treat population (ITT); 100%, 60%, and 10% of patients receiving 0.05%, 0.1%, and vehicle, respectively, reported a ?2-grade IGA score improvement (p=0.002 for TMB-001 0.05% vs vehicle) in PP; improvement of ?2-grade IGA score was observed in 55%, 40%, and 8% of patients receiving 0.05%, 0.1%, and vehicle, respectively (p=0.02 for TMB-001 0.05% vs. vehicle) in ITT; mild or moderate in severity of local skin irritations; no serious adverse events | 8/26/22 | Dermatologic |
| Union Therapeutics A/S, of Hellerup, Denmark | Orismilast | Selective next-generation PDE4 inhibitor; modified release tablet | Moderate to severe atopic dermatitis | First patient enrolled in Adesos study | 8/4/22 | Dermatologic |
| Vyne Therapeutics Inc., of Bridgewater, N.J. | FMX-114 | Combination gel formulation of tofacitinib and fingolimod | Mild to moderate atopic dermatitis | Study did not meet its primary endpoint (n=21); mean ADSI scores for FMX-114 and vehicle-treated lesions were 6.6 and 6.9, respectively, at baseline; mean reduction in ADSI score from baseline was -4.05 for FMX-114-treated lesions compared to -3.48 for vehicle-treated lesions at week 4 (p=0.228) | 8/10/22 | Dermatologic |
| 89bio Inc., of San Francisco | Pegozafermin | Engineered FGF21 derivative | Severe hypertriglyceridemia | Study met primary and secondary endpoints; statistically significant reductions in median triglyceriodes from baseline in patients across all dose groups vs. placebo after 8 weeks; improvements in atherogenic lipoproteins, metabolic measures and liver fat; well-tolerated with a favorable safety profile across different doses | 8/26/22 | Endocrine/Metabolic |
| Disc Medicine Inc., of Cambridge, Mass. | Bitopertin | Oral, selective inhibitor of glycine transporter 1 | Erythropoietic protoporphyria or X-linked protoporphyria | Initiated study to evaluate the safety, tolerability and efficacy; planning to enroll 20 patients | 8/10/22 | Endocrine/Metabolic |
| Hightide Therapeutics Inc., of Shenzhen, China | HTD-1801 | Ionic salt of berberine and ursodeoxycholic acid | Type 2 diabetes | Enrollment completed with 113 subjects | 8/30/22 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsværd, Denmark | Cagrisema | 2.4-mg semaglutide and 2.4-mg cagrilintide | Type 2 diabetes | Headline results showed mean baseline HbA1c was 8.4% and the mean baseline body weight was 106 kg; achieved a numerically higher HbA1c reduction of 2.18%-points compared to a reduction of 1.79%-points for people treated with semaglutide and 0.93%-points with cagrilintide alone; numerically higher body weight reduction of 15.6% compared to a reduction of 5.1% for people treated with semaglutide and 8.1% with cagrilintide alone; safe and well-tolerated profile | 8/22/22 | Endocrine/Metabolic |
| Sciwind Biosciences Co. Ltd., of San Francisco | XW-003 | Long-lasting GLP-1 peptide ecnoglutide in injectable form | Type 2 diabetes | Results of 20-week study in Chinese adults showed subcutaneous administration safe and well-tolerated; achieved robust reductions in HbA1c; statistically significant HbA1c reductions from baseline of 1.8%, 1.9% and 2.4%, respectively, compared to 0.5% in participants receiving placebo (p<0.0001); 88% of participants in the 1.2-mg xw-003 cohort achieved hba1c ? 7%; significantly reduced participants' body weight a dose-dependent fashion; improvements additional cardiometabolic parameters< td> | 8/2/22 | Endocrine/Metabolic |
| 89bio Inc., of San Francisco | Pegozafermin | Specifically engineered FGF21 derivative | Nonalcoholic steatohepatitis | Completed enrollment of 219 patients; top-line data expected in the first quarter of 2023 | 8/18/22 | Gastrointestinal |
| Akero Therapeutics Inc., of South San Francisco | Efruxifermin | Differentiated Fc-FGF21 fusion protein | Nonalcoholic steatohepatitis | Results from cohort C of Balanced trial published in JHEP Reports showed drug well-tolerated; liver stiffness and serum markers of fibrosis significantly decreased compared to baseline at 16 weeks of treatment; improved whole-body metabolism; significant improvement in lipoprotein profile and decrease in HbA1c | 8/24/22 | Gastrointestinal |
| Hepion Pharmaceuticals Inc., of Edison, N.J. | Rencofilstat | Cyclophilin inhibitor | Nonalcoholic steatohepatitis | Screened first subject | 8/31/22 | Gastrointestinal |
| Immunogenx Inc., of Newport Beach, Calif. | Latiglutenase (IMGX-003) | Oral mixture of 2 gluten-specific recombinant proteases | Celiac disease | Data from the Celiacshield study to be published in Gastroentrerology showed latiglutenase eliminated approximately 95% of gluten in the stomachs of patients as measured by gluten-immunogenic peptide in urine (p=0.0001) | 8/15/22 | Gastrointestinal |
| Poxel SA, of Lyon, France | PXL-065 | Deuterium-stabilized R-stereoisomer of pioglitazone | Non-alcoholic steatohepatitis | In the 117-patient Destiny study, mean relative decrease from baseline to 36 weeks in liver fat content vs. placebo was 21% to 25% for the 4 dose levels (p=0.024 to p=0.008) | 8/30/22 | Gastrointestinal |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass., and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Cemdisiran | RNAi therapeutic targeting the C5 component | IgA nephropathy | Data showed 36% reduction in 24-hour urine total protein vs. placebo; well-tolerated; no adverse events; no drug-related adverse events | 8/29/22 | Genitourinary/Sexual Function |
| Xortx Therapeutics Inc., of Calgary, Alberta | Oxypurinol formulation | Oxypurinol formulation | Autosomal dominant polycystic kidney disease | Top-line data from pharmacokinetics bridging study showed improved bioavailability; clean safety and pharmacologic profile with no drug-related adverse or serious adverse events; planning to initiate phase III study in the first half of 2023 | 8/22/22 | Genitourinary/Sexual Function |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Pyrukynd (mitapivat) | Pyruvate kinase activator | Non-transfusion-dependent ?- or ?-thalassemia | Results published in The Lancet showed 16 of 20 patients (80%; p<0.0001), 5 11 13 15 16 including of (100%) with ?-thalassemia and (73%) achieved the primary endpoint hemoglobin response, a ?1 g dl increase in concentration from baseline at one or more assessment between weeks 4–12; mean time to response was 4.5 weeks; had sustained endpoint; decrease erythropoietin concentrations were also observed both ?- patients< td> | 8/11/22 | Hematologic |
| Avalo Therapeutics Inc., of Wayne, Pa. | AVTX-803 | Oral formulation of L-fucose | Leukocyte adhesion deficiency type II | First patient dosed in the Ladder trial | 8/2/22 | Hematologic |
| Fibrogen Inc., of San Francisco | Roxadustat | Oral small-molecule hypoxia-inducible factor prolyl hydroxylase inhibitor | Lower risk transfusion-dependent myelodysplastic syndromes | Completed patient enrollment in Matterhorn study; top-line data expected in the first half of 2023; 141 subjects enrolled; primary endpoint of the study is transfusion independence for ? 56 consecutive days in the first 28 weeks of treatment; secondary endpoint is reduction of red blood cell transfusion | 8/26/22 | Hematologic |
| Clene Inc., of Salt Lake City | CNM-Au8 | Gold nanocrystal suspension | Stable relapsing remitting multiple sclerosis with chronic optic neuropathy | In the Visionary-MS study, LCLA letter change in the clinically affected eye was 3.13 (p=0.056); study was stopped early due to COVID-19 pandemic operational challenges | 8/15/22 | Immune |
| Acurx Pharmaceuticals Inc., of New York | Ibezapolstat | Antibiotic | C. difficile infection | Completed phase IIa study; 100% clinical cure and 100% sustained clinical cure in patients; beneficial effects on bile acid metabolism | 8/2/22 | Infection |
| Aivita Biomedical Inc., of Irvine, Calif. | AV-COVID-19 | Dendritic cell-based vaccine against SARS-CoV-2 consisting of autologous dendritic cells ex vivo-loaded with the SARS-CoV-2's spike protein | COVID-19 | Data from 3 formulations in phase II study showed well-tolerated; no high-grade or serious adverse events; 94.4% of subjects increased ELISPOTS by day 14 and 122 (96.8%) by day 28 | 8/29/22 | Infection |
| Axcella Health Inc., of Cambridge, Mass. | AXA-1125 | EMM composition of 6 amino acids and derivatives | Long COVID | Top-line data of AXA-1125 showed improvements in measures of mental and physical fatigue that were both highly statistically significant and clinically relevant compared to those who received placebo; mean changes in total, physical and mental scores in the CFQ-11 vs. placebo (p=0.0039), (p=0.0097) and (p=0.0097), respectively; statistically significant correlation of improvement in fatigue score and greater distance achieved in the 6MWT (p=0.0027); significant improvement in serum lactate levels after a 6MWT (p=0.0730); safe and well-tolerated with no significant adverse events | 8/2/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 (CpG 1018/Alum) | Protein recombinant S-Trimer vaccine | COVID-19 | Study met the primary endpoint; 2-fold higher neutralizing antibody titers in adolescents compared to young adults; favorable safety and reactogenicity profile; mild and transient adverse events | 8/25/22 | Infection |
| Cocrystal Pharma Inc., of Bothell, Wash. | CC-42344 | Oral antiviral binds to PB2 site of influenza A polymerase complex | Pandemic and seasonal influenza A | Initiated phase IIa study to evaluate safety, viral and clinical measures of oral administration | 8/8/22 | Infection |
| Hillevax Inc., of Boston | HIL-214 | Bivalent vaccine candidate; consists of virus-like particles designed to mimic the structure of 2 major genotypes of norovirus, GI.1 and GII.4 | Moderate to severe acute gastroenteritis caused by norovirus infection | Independent data safety monitoring committee completed a prespecified review of safety data (n=203); recommended continuation of study without modification; enrollment has subsequently resumed; top-line data expected in the second half of 2023 | 8/31/22 | Infection |
| Jiangsu Recbio Technology Co. Ltd., of Taizhou, China | Recov | Recombinant protein | COVID-19 vaccine | Completed the subject enrollment and dosing; 600 subjects enrolled | 8/18/22 | Infection |
| Kinarus Therapeutics Holding AG, of Basel, Switzerland | KIN-001 | Combination of pamapimod and pioglitazone | COVID-19 | First patient dosed | 8/29/22 | 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 | Initiated phase IIb/III Hummingbird study to evaluate the safety, effectiveness (immunogenicity), and efficacy of 2 doses in younger children ages 6 months-11 years | 8/4/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Pfizer-Biontech COVID-19 vaccine | mRNA vaccine | COVID-19 | Updated results at 3-µg dose series in children 6 months through 4 years of age showed vaccine efficacy was 73.2%; efficacy was 75.8% in children ages 6 through 23 months; well-tolerated | 8/23/22 | Infection |
| Pneumagen Ltd., of St. Andrews, Scotland | Neumifil | Intranasal spray; multivalent carbohydrate binding molecule | Influenza | First patient dosed | 8/16/22 | Infection |
| Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Sublingual formulation of muscle relaxant cyclobenzaprine hydrochloride | Long COVID syndrome whose symptoms overlap with fibromyalgia | First subject enrolled in Prevail study; 14-week double-blind, randomized, multicenter, placebo-controlled study to evaluate efficacy and safety | 8/22/22 | Infection |
| Virios Therapeutics Inc., of Atlanta | IMC-2 (valacyclovir + celecoxib) | Herpes virus antiviral combination | Long-COVID | Started enrollment in the exploratory study testing the drug’s effect on fatigue, pain, sleep disruption, anxiety, depression and cognitive function as well as overall health improvement | 8/15/22 | Infection |
| Saol Therapeutics Inc., of Roswell, Ga. | SL-1002 | Nerve-blocking agent | Knee pain associated with osteoarthritis | First patient enrolled in Compass trial | 8/9/22 | Inflammatory |
| Arthrosi Therapeutics Inc., of San Diego | AR-882 | Uricosuric agent | Chronic gout | Completed enrollment; exceeded the target enrollment of 120 patients | 8/23/22 | Inflammatory |
| Alvotech Holdings SA, of Reykjavik, Iceland | Denosumab (AVT-03) | Biosimilar Prolia; antibody binding to RANK-L | Postmenopausal women with osteoporosis | Initiated a confirmatory patient study | 8/25/22 | Musculoskeletal |
| Santhera Pharmaceuticals AG, of Pratteln, Switzerland, and Reveragen Biopharma Inc., of Rockville, Md. | Vamorolone | Dissociative agonist | Becker muscular dystrophy | First patient dosed; to evaluate the safety, tolerability and exploratory clinical efficacy on motor function outcomes of vamorolone compared to placebo over a treatment period of 24 weeks in 39 males | 8/22/22 | Musculoskeletal |
| AC Immune SA, of Lausanne, Switzerland | Crenezumab | Antibody targeting beta-amyloid | Alzheimer’s disease | Data showed safe and well-tolerated; did not hit statistical significance on clinical endpoints; API ADAD composite 22.9% (p=0.43); FCSRT 19.9% (p=0.16); RBANS total score 43.8% (p=0.55); time to MCI/dementia due to AD 20.8% (p=0.48); time to non-zero in CDR-GS 8.1% (p=0.76); CDR Sum of Boxes 8.8% (p=0.64); favored biomarker results; A? PET SUVR 3.6% (p=0.69); tau-PET SUVR 51.1% (p=0.20); FDG PET SUVR 18.1% (p=0.25); t-tau 28.7% (p=0.53); p-tau-181 37.4% (p=0.28); NfL 18.2% (p=0.46) | 8/2/22 | Neurology/Psychiatric |
| Alterity Therapeutics Ltd., of San Francisco | ATH-434 | Small molecule designed to inhibit aggregation of pathological proteins | Multiple system atrophy | Study open for enrollment in the U.K. | 8/25/22 | Neurology/Psychiatric |
| Aptinyx Inc., of Evanston, Ill. | NYX-2925 | Small molecule that modulates the N-methyl-D-aspartate receptor | Fibromyalgia | Study did not achieve statistically significant separation from placebo on the study’s primary endpoint at week 12; clinically meaningful improvement in pain and secondary endpoints; well-tolerated; no safety issues | 8/12/22 | 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 | Interim analysis (n=100) showed safe and well-tolerated; ADAS-Cog11 scores improved an average of 1.5 points (p<0.05); 5 100 63% of the patients showed an improvement in adas-cog11 scores; 21% declined less than points on adas-cog11; average 2.7 points< td> | 8/3/22 | Neurology/Psychiatric |
| Eliem Therapeutics Inc., of Cambridge, U.K. | ETX-810 | New chemical entity prodrug of the bioactive lipid palmitoylethanolamide | Lumbosacral radicular pain | Results did not achieve statistically significant separation from placebo on the trial’s primary endpoint; discontinued further development of ETX-810 | 8/2/22 | Neurology/Psychiatric |
| Inhibikase Therapeutics Inc., of Boston and Atlanta | IkT-148009 | c-Abl inhibitor | Parkinson’s disease | First patient dosed | 8/23/22 | Neurology/Psychiatric |
| Mind Medicine Inc., of New York | MM-120 | Optimized form of lysergic acid diethylamide | Generalized anxiety disorder | First patient dosed | 8/25/22 | Neurology/Psychiatric |
| Noema Pharma AG, of Basel, Switzerland | NOE-105 (gemlapodect) | PDE10A inhibitor | Childhood onset fluency disorder/stuttering | First patient dosed in Orpheus study; top-line data expected in fourth quarter 2023 | 8/4/22 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Branaplam (LMI-070) | SMN2 RNA splicing modulator | Huntington`s disease | Company temporarily suspended dosing in Vibrant-HD study; decision based on recommendation from the independent data monitoring committee following a planned data review; committee did not recommend terminating the study at this time; participants will continue to have regular study assessments per the protocol | 8/25/22 | Neurology/Psychiatric |
| Regenacy Pharmaceuticals Inc., of Waltham, Mass. | Ricolinostat | Oral HDAC6 inhibitor | Peripheral diabetic neuropathy | Completed enrollment | 8/24/22 | Neurology/Psychiatric |
| Vaccinex Inc., of Rochester, N.Y. | Pepinemab | Humanized IgG4 monoclonal antibody that inhibits SEMA4D | Cognitive impairment in Huntington’s Disease | Results published in Nature Medicine showed trial did not meet its prespecified primary efficacy endpoints; significantly improved cognition in patients with early manifest disease, as reflected in the HD-Cognitive Assessment Battery of 6 different cognitive measures (p=0.007); significantly reduced apathy severity (p=0.02); reduced atrophy (p=0.017) in the caudate nucleus; significantly improved brain metabolic activity; well-tolerated with a low frequency of treatment-emergent adverse events; low treatment discontinuation rate | 8/8/22 | Neurology/Psychiatric |
| Eyepoint Pharmaceuticals Inc., of Watertown, Mass. | EYP-1901 | Sustained delivery anti-VEGF therapy; bioerodible formulation of Durasert delivery technology with vorolanib | Wet age-related macular degeneration | First patient dosed; top-line data expected in the second half of 2023 | 8/1/22 | Ocular |
| Oculis SA, of Lausanne, Switzerland | OCS-02/licaminlimab | Anti-TNF alpha antibody fragment | Dry eye disease | Results published in Clinical Ophthalmology showed statistically significant in the change from baseline to day 29 in the global ocular discomfort score (p=0.041); statistically greater on secondary endpoints (p=0.018); well-tolerated; no increase in intra-ocular pressure and no other safety issues | 8/23/22 | Ocular |
| Ocuterra Therapeutics Inc., of Boston | OTT-166 | Non-invasive selective RGD integrin inhibitor; eye drop | Moderately severe to severe non-proliferative diabetic retinopathy | First patient dosed | 8/2/22 | Ocular |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif. | TP-03 | Topical ophthalmic solution of lotilaner 0.25% | Meibomian gland disease in patients with Demodex mites | First patient enrolled | 8/5/22 | Ocular |
| Unity Biotechnology Inc., of South San Francisco,Calif. | UBX-1325 | Senolytic Bcl-xL inhibitor | Diabetic macular edema | Data from 12 weeks of Behold study maintained central subfield thickness (CST) compared to sham-treated patients (p=0.0747); mean improvement in mean Best Corrected Visual Acuity (BCVA) of +4.7 Early Treatment Diabetic Retinopathy (ETDRS) letters from baseline compared to +1.3 ETDRS letters in sham-treated patients (p=0.1148); at 18 week showed mean improvement in BCVA of +6.1 ETDRS letters from baseline compared to +1.1 EDTRS letters in sham-treated patients (p=0.0368); mean change in CST of +3.2 microns from baseline compared to +53.5 microns in sham-treated patients (p=0.0719); favourable safety and tolerability profile | 8/12/22 | Ocular |
| Valohealth Inc., of Boston | OPL-0401 | ROCK 1/2 inhibitor | Nonproliferative diabetic retinopathy | Initiated dosing | 8/18/22 | Ocular |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Diketopiperazine | Phelan-McDermid syndrome and Pitt Hopkins syndrome |
Initiated phase II study | 8/8/22 | Other/Miscellaneous |
| Otonomy Inc., of San Diego | OTO-313 | Sustained-exposure formulation of NMDA receptor antagonist gacyclidine | Tinnitus | Study showed no clinically meaningful benefit vs. placebo for primary and secondary endpoints across all timepoints; company to discontinue development of OTO-313 | 8/1/22 | Other/Miscellaneous |
| Akebia Therapeutics Inc., of Cambridge, Mass. | Vadadustat | Hypoxia-inducible factor prolyl hydroxylase inhibitor | Acute respiratory distress syndrome in patients with COVID-19 and hypoxemia | Trial failed to meet its primary superiority threshold of >95% probability; proportions of subjects who had a 6, 7 or 8 on the NIAID-OS were 25.4% (20.7%, 30.5%) for vadadustat vs. 29.7% (24.5%, 35.3%) for placebo with a relative risk of 0.86 and 97% probability that vadadustat was superior to placebo at day 7 | 8/4/22 | Respiratory |
| Agenus Inc., of Lexington, Mass. | Botensilimab | Fc-enhanced anti-CTLA4 | Microsatellite stable colorectal cancer and melanoma | Initiated phase II study; to evaluate as monotherapy and in combination with balstilimab | 9/12/22 | Cancer |
| Bioatla Inc., of San Diego | Mecbotamab vedotin (HTBA-3011) | Conditionally and reversibly active antibody-drug conjugate targeting the receptor tyrosine kinase AXL | Non-small-cell lung cancer and Sarcoma | Top-line data showed objective response rate of 43% in 7 non-squamous patients; safe and well-tolerated in both monotherapy and in combination with nivolumab; PFS for osteosarcoma patients at 3 months was 4 of 7 patients, or 57%; full data expected in the fourth quarter 2022 | 9/12/22 | Cancer |
| Biomed Valley Discoveries Inc., of Kansas City, Mo. | Ulixertinib (BVD-523) | ERK inhibitor | Advanced gastrointestinal malignancies | First patient dosed | 9/12/22 | Cancer |
| Carsgen Therapeutics Holdings Ltd., of Shanghai | Zevorcabtagene autoleucel | BCMA-targeting autologous CAR T-cell therapy | Relapsed and/or refractory multiple myeloma | A dose of 1.8×108 CAR T cells was well-tolerated in the initial 17 phase II patients, with promising efficacy and MRD negativity; outpatient treatment undergoing further exploration | 9/21/22 | Cancer |
| Clarity Pharmaceuticals Ltd., of Sydney | 64Cu SAR-bombesin | Targeted pan-cancer radiopharmaceutical | PSMA-negative prostate cancer | Initiated recruitment in multi-centre, single arm, non-randomised, open-label trial | 9/5/22 | Cancer |
| Evaxion Biotech A/S, of Copenhagen | EVX-01 | Personalized cancer therapy | Melanoma | Enrolled first patient | 9/21/22 | Cancer |
| Galecto Inc., of Boston | GB-2064 | LOXL2 inhibitor | Myelofibrosis | Four out of five evaluable patients who received GB-2064 monotherapy for at least six months experienced a ? 1-grade reduction in collagen fibrosis of the bone marrow | 9/29/22 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha | Soluble LAG-3 fusion protein | Soft tissue sarcoma | Initiated phase II study in combination with pembrolizumab and radiotherapy in the neoadjuvant setting; first patient dose expected in the first half of 2023 | 9/6/22 | Cancer |
| IMV Inc., of Dartmouth, Nova Scotia, | MVP-S (maveropepimut) | MVP-S combined with pembrolizumab and intermittent low-dose cyclophosphamid | Relapsed/refractory diffuse large B cell lymphoma | Site activation and enrollment have increased substantially in the last few months | 9/30/22 | Cancer |
| Innocare Pharma Ltd., of Beijing | Tafasitamab | Humanized Fc-modified cytolytic CD19-targeting monoclonal antibody | Relapsed or refractory diffuse large B-cell lymphoma | First patient dosed in combination with lenalidomide | 9/8/22 | Cancer |
| Inxmed Co. Ltd., of Nanjing, China | IN-10018 | Oral inhibitor of focal adhesion kinase | Platinum-resistant recurrent ovarian cancer | First patient dosed in combination with pegylated liposomal doxorubicin | 9/7/22 | Cancer |
| Jacobio Pharmaceuticals Co. Ltd., of Shanghai | JAB-21822 | KRAS G12C inhibitor | Non-small-cell lung cancer | First patient dosed | 9/2/22 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide crystal nanoparticles | Head and neck cancer | Data showed recommended phase II dose for all 3 cohorts was determined to be 33% of gross tumor volume; expected to enroll 141 patients in combined dose-escalation and dose-expansion parts of study 1100.; updated results expected in the fourth quarter 2022 | 9/21/22 | Cancer |
| Oncxerna Therapeutics Inc., of Waltham, Mass. | Navicixizumab | Anti-DLL4/VEGF bispecific antibody | Advanced solid tumors | First patient dosed | 9/8/22 | Cancer |
| PDS Biotechnology Corp., Florham Park, N.J. | PDS-0101-based triple combination therapy | DOTAP lipid and the E7 antigen from HPV 16 | Advanced human papilloma virus-based cancers | Optimal patient group identified | 9/21/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX-591 (177Lu-DOTA-rosopatamab) | Radiopharmaceutical targeting prostate-specific membrane antigen (PSA) | First recurrence of PSA increase after initial therapy for prostate cancer | Enrolled first of 50 patients in the Prostact Target study testing the drug plus external beam radiation therapy in patients with 5 or fewer metastases; primary endpoint is biological progression-free survival | 9/26/22 | Cancer |
| Yingli Pharmaceuticals Ltd., of Shanghai | Linperlisib | PI3K-delta inhibitor | Relapsed/refractory peripheral T/NK cell lymphoma | First patient dosed | 9/3/22 | Cancer |
| Alumis Inc., of South San Francisco | ESK-001 | Allosteric tyrosine kinase 2 inhibitor | Moderate to severe plaque psoriasis | First patient dosed in Stride trial | 9/28/22 | Dermatologic |
| Aobiome Therapeutics Inc., of Cambridge, Mass. | B-244 | Live topical biotherapeutics | Moderate-to-severe itch and mild-to-moderate atopic dermatitis | Results showed no adverse events; reductions in itch were seen within the first hour of application; 57% reduction of itch scores at day 14 in adults; 69% reduction of itch scores at day 14 in pediatric; 57.9% of adult subjects showed a 4-point improvement in itch (Visual Analog Scale) at day 14; 81.8% of pediatric subjects showed a 2-point improvement in itch (Itch Man Scale) at day 14 (5-point scale); improvement in the appearance of eczema | 9/20/22 | Dermatologic |
| Cara Therapeutics Inc., of Stamford, Conn. | Difelikefalin (Korsuva) | Analgesic opioid peptide | Moderate to severe pruritus in notalgia paresthetica | Results from Komfort study achieved primary endpoint of Worst Itch-Numeric Rating Scale score (WI-NRS) change from baseline at week 8 (p=0.001); significant reduction of itch intensity was evident at day 1 and maintained through week 8; achieved a ?4-point improvement in WI-NRS score at week 8 with oral difelikefalin vs. placebo (41% difelikefalin vs. 18% placebo, p=0.007); met the complete response endpoint (22% difelikefalin vs. 5% placebo, p<0.01); well-tolerated; mild or moderate in severity adverse events< td> | 9/8/22 | Dermatologic |
| Edesa Biotech Inc., of Toronto | EB-01 | Nonsteroidal anti-inflammatory compound; sPLA2 inhibitor | Chronic allergic contact dermatitis | Completed patient recruitment; top-line data by the end of 2022 | 9/7/22 | Dermatologic |
| Evommune Inc., of Palo Alto, Calif. | EVO101 topical cream | Small molecule inhibitor of IRAK4 | Atopic dermatitis | First patient enrolled in phase IIa proof of concept trial | 9/28/22 | Dermatologic |
| MC2 Therapeutics A/S., of Copenhagen, Denmark | MC2-25 | Inhibits carbamylation in skin | Chronic kidney disease-associated pruritus | First patient dosed | 9/14/22 | Dermatologic |
| Moonlake Immunotherapeutics AG, of Zug, Switzerland | Nanobody sonelokimab | Trivalent nanobody comprising monovalent camelid-derived nanobodies specific to human interleukin (IL)-17A, IL-17F and human serum albumin | Active psoriatic arthritis | Initiated phase II study; expected to enroll 200 patients; to assess the efficacy and safety of sonelokimab compared to placebo | 9/26/22 | Dermatologic |
| Amolyt Pharma SA, of Lyon, France | AZP-3601 | Therapeutic peptide targeting parathyroid hormone receptor | Hypoparathyroidism | Data showed mean serum calcium levels were maintained within the target range; discontinuation of oral calcium and active vitamin D supplementation; well-tolerated and demonstrated a favorable safety profile; 24-hour urinary excretion of calcium was rapidly normalized in all cases including in patients with hypercalciuria at baseline; P1NP and CTX slightly increased after 2 weeks of treatment and remained within their mid-normal range through the end of the study; | 9/12/22 | Endocrine/Metabolic |
| Ascendis Pharma Inc., of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Hypoparathyroidism | Data from week 110 from Path forward trial showed sustained release of active parathyroid hormone (PTH) at stable levels in the physiological range for 24 hours/day; continued normalization of serum calcium; 93% achieving independence from conventional therapy; higher baseline bone mineral density (BMD) Z-scores and larger decreases in BMD Z-scores; well-tolerated; no discontinuations due to treatment-emergent adverse events | 9/12/22 | Endocrine/Metabolic |
| Glyscend Therapeutics Inc., of Boston | GLY-200 | Orally administered, gut-restricted therapeutic agent | Non-insulin dependent diabetes | Initiated phase II study in patients; top-line data in early 2023 | 9/20/22 | Endocrine/Metabolic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif., and Astrazeneca plc, of Cambridge, U.K. | ION-449 (AZD-8233) | Antisense targeting proprotein convertase subtilisin/kexin type 9 | High-risk hypercholesterolemia | Top-line study showed AZD-8233 achieved a statistically significant 62.3% (p<0.001) 28 reduction in low-density lipoprotein cholesterol levels after weeks vs. placebo; safe and well-tolerated; results did not achieve prespecified efficacy criteria < td> | 9/23/22 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin candidate | Type 2 diabetes patients with non-alcoholic steatohepatitis | Study met primary and secondary endpoint; safe and well-tolerated at 8 mg twice daily; clinically meaningful reduction of liver fat from baseline at 12 weeks | 9/13/22 | Endocrine/Metabolic |
| Poxel SA, of Lyon, France | PXL-065 | Deuterium-stabilized R-pioglitazone | Non-alcoholic steatohepatitis | Met primary efficacy endpoint for liver fat content reduction at 36 weeks for all doses. | 9/21/22 | Endocrine/Metabolic |
| Sparrow Pharmaceuticals Inc., of Portland, Ore. | SPI-62 | Selective HSD-1 inhibitor | ACTH-dependent Cushing’s syndrome | First patient dosed in phase II Rescue trial | 9/28/22 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | BI-456906 | Glucagon receptor/glucagon-like peptide-1 receptor agonist | Non-insulin dependent diabetes | Lowered HbA1c up to -1.88% at week 16 | 9/21/22 | 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 | Results published in the Lancet Gastroenterology & Hepatology showed study met the primary endpoint at 25-mg, 50-mg and 100-mg compared to placebo; statistically significant reduction of Modified Mayo Score; reduction of fecal calprotectin showed significant difference in patients dosed with obefazimod compared to placebo; good tolerability profile | 9/6/22 | Gastrointestinal |
| Akero Therapeutics Inc., of South San Francisco | Efruxifermin | Fc-FGF21 fusion protein | Pre-cirrhotic nonalcoholic steatohepatitis and fibrosis stage 2 or 3 | Harmony study achieved primary and secondary endpoints; 41% (50 mg) and 39% (28 mg) of EFX-treated patients, respectively, experiencing at least a 1-stage improvement in liver fibrosis with no worsening of NASH by week 24, compared with 20% for the placebo arm; 76% and 47% of patients treated with 50-mg and 28-mg doses, respectively, achieving NASH resolution without worsening of fibrosis, compared with 15% for placebo; 41% and 29% of patients treated with 50-mg and 28-mg doses, respectively achieved both endpoints (NASH resolution and fibrosis improvement ?1 stage), compared with 5% for placebo; well-tolerated; statistically significant improvements in liver fat, liver enzymes, noninvasive fibrosis markers, glycemic control, lipoproteins, and body weight | 9/13/22 | Gastrointestinal |
| Altimmune Inc., of Gaithersburg, Md. | Pemvidutide | GLP-1/glucagon dual receptor agonist | Nonalcoholic steatohepatitis | Completion of first dosing of all subjects in Momentum trial | 9/28/22 | Gastrointestinal |
| Applied Molecular Transport Inc., of South San Francisco | AMT-101 | GI-selective, oral fusion of IL-10 and carrier molecule | Moderate to severe ulcerative colitis | Completed enrollment in Lombard study; top-line data expected in late 2022 or early 2023 | 9/20/22 | Gastrointestinal |
| Palatin Technologies Inc., of Cranbury, N.J. | PL-8177 | Melanocortin-1 receptor agonist | Ulcerative colitis | Initiated phase II study to evaluate the safety, tolerability, efficacy, pharmacokinetics and biomarkers | 9/8/22 | Gastrointestinal |
| Recursion Inc., of Salt Lake City | REC-4881 | Non-ATP-competitive allosteric small-molecule inhibitor of MEK1 and MEK2 | Familial adenomatous polyposis | Initiated phase II Tupelo study in patients; to evaluate efficacy, safety, and pharmacokinetics | 9/13/22 | Gastrointestinal |
| Revolo Biotherapeutics Inc., of New Orleans | IRL-201104 ('1104) | Peptide derived from mTB Chaperonin 60.1 | Eosinophilic esophagitis | Completed enrollment and randomized all patients; top-line data expected in the end of 2022 | 9/12/22 | Gastrointestinal |
| Sagimet Biosciences Inc., of San Mateo, Calif. | Denifanstat (TVB-2640) | FASN inhibitor | Nonalcoholic steatohepatitis | Completed enrollment | 9/13/22 | Gastrointestinal |
| Pfizer Inc., of New York, and Sangamo Therapeutics Inc., of Brisbane, Calif. | Giroctocogene fitelparvovec | Gene therapy | Moderately severe to severe hemophilia A | Re-opened recruitment; dosing expected to resume in October; read-out expected in the first half of 2024 | 9/22/22 | Hematologic |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-109 | Epinephrine sublingual film | Acute anaphylaxis | After 1 dose of AQST-109, maximum mean effects on systolic blood pressure occurred within 5 minutes of dosing compared to 8 minutes for Epipen; maximum mean effects in heart rate occurred within 8 minutes for AQST-109 compared to an average of 5 minutes for Epipen; median time to maximum concentration was 12 minutes for AQST-109 and 22.5 minutes for Epipen | 9/27/22 | Immune |
| Biogen Inc., of Cambridge, Mass. | BIIB-059 (litifilimab) | Humanized IgG1 monoclonal antibody targeting blood dendritic cell antigen 2 | Cutaneous lupus erythematosus and systemic lupus erythematosus | Results from phase II LILAC study published in The New England Journal of Medicine showed litifilimab met primary endpoint; significantly reduced the total number of swollen and tender joints in participants with SLE from baseline compared to placebo over 24 weeks; well-tolerated; mild or moderate adverse events | 9/7/22 | Immune |
| Biogen Inc., of Cambridge, Mass., and The Feinstein Institutes for Medical Research | Litifilimab (BIIB-059) | Anti-BDCA2 monoclonal antibody | Systemic lupus erythematosus | Biogen-sponsored Lilac (part A) trial showed greater reduction of swollen and tender joints vs. placebo over 24 weeks | 9/19/22 | Immune |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous MSC-NTF cells | Progressive multiple sclerosis (MS) | Results published in Multiple Sclerosis Journal showed clinically meaningful improvements in some patients across all endpoints measured; 19% of treated trial participants were responders vs. 4% of the matched CLIMB registry patients; 38% of treated trial participants showed a ?10-point improvement from baseline in the 12-item MSWS ; 27% of treated trial participants showed a ?8-letter improvement in LCLA binocular at a 2.5% contrast threshold vs. 6% of the matched CLIMB registry patients; 67% of treated trial participants showed a ?3-point improvement in the SDMT, compared to 18% of the matched CLIMB registry patients; 47% of treated trial participants showed a ?8-point improvement in LCLA binocular at a 1.25% contrast threshold; no adverse events related to worsening of MS disease and no clinically significant changes; favorable safety profile | 9/15/22 | Immune |
| Navidea Biopharmaceuticals Inc., of Dublin, Ohio | Tc99m Tilmanocept | Imaging agent | Rheumatoid arthritis joint inflammation | Preliminary results showed quantitative TIL uptake in the hands and wrists of patients is proportional to the amount of macrophage involvement in 11 patients; high affinity ligand to CD206 expressed on activated macrophages | 9/15/22 | Immune |
| Sparrow Pharmaceuticals Inc., of Portland, Ore. | SPI-62 | Selective HSD-1 inhibitor | Polymyalgia rheumatica | First patient dosed in combination with prednisolone | 9/9/22 | Immune |
| AB Science SA, of Paris | Alsitek (masitinib) | Protein kinase inhibitor | COVID-19 | Following safety assesment based on first half of trial particcipants, data and safety monitoring board recommended continuation of study in non-hospitalized patients at risk of developing severe COVID-19 and in hospitalized patients with need of oxygen | 9/14/22 | Infection |
| Adamis Pharmaceuticals Co., of San Diego | Tempol | Superoxide dismutase modulator | High-risk subjects with early COVID-19 infection | Trial halted after missing primary endpoint of clinical resolution of COVID-19 symptoms | 9/21/22 | Infection |
| Adamis Pharmaceuticals Corp., of San Diego | Tempol | Membrane-permeable radical scavenger | COVID-19 | Reached the initial planned enrollment of 248 subjects | 9/13/22 | Infection |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-22 (envafolimab) | Single domain antibody against PD-L1 | HIV-1 infection | Completed enrollment of 15 HIV infected patients; to evaluate the efficacy in combination with Chidamide | 9/15/22 | Infection |
| Ascletis Pharma Inc., of Hangzhou, China | ASC22 (envafolimab) | Subcutaneously administered PD-L1 antibody | Chronic hepatitis B | First patient dosed in phase IIb extension cohort | 9/28/22 | Infection |
| Atriva Therapeutics GmbH, of Tübingen and Frankfurt, Germany | Zapnometinib | Blocks RNA-virus replication; immune modulator | Moderate to severe COVID-19 | Top-line results from Respire study showed clinically relevant efficacy profile; favorable safety profile; company planning to terminate the study early based on low recruitment projection as well as the high prevalence of patients infected with the Omicron variant | 9/20/22 | Infection |
| Bergenbio ASA, of Bergen Norway | Bemcentinib | AXL inhibitor | Hospitalized COVID-19 | First patient enrolled in the phase IIb EU-Solidact adaptive study | 9/27/22 | Infection |
| Biophytis SA, of Paris | Sarconeos (BIO-101) | Proto-oncogene Mas agonist | Severe respiratory failure in COVID-19 patients | Top-line data from COVA study showed drug reduced by 39% risk of respiratory failure or early death at 28 days compared to placebo (p=0.07); reduced both the proportion of patients with respiratory failure (12.7% vs. 21.5%) and early death (0.8% vs. 2.8%); significantly (p=0.03) delayed the progression of respiratory failure or early death over 28 days maximum treatment period; good safety profile; similar proportion of adverse events compared to placebo | 9/7/22 | Infection |
| Cidara Therapeutics Inc., of San Diego | CD-388 | Long-acting antiviral immunotherapy | Prevention of influenza | Initiated phase IIa study to evaluate the pre-exposure prophylactic activity | 9/13/22 | Infection |
| Curevo Inc., of Seattle | CRV-101 | Adjuvanted subunit vaccine | Varicella zoster virus (shingles) infection | Completed enrollment; enrolled 678 participants in 6 months; top-line data expected in early 2023 | 9/15/22 | Infection |
| Dynavax Technologies Corp., of Emeryville, Calif. | rF1V vaccine and CpG 1018 adjuvant | Recombinant plague vaccine | Plague | First subject dosed; to evaluate the immunogenicity, safety and tolerability | 9/12/22 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody targeting TLR4 | COVID-19-induced acute respiratory distress syndrome | Results from phase II portion of ongoing phase II/III trial in hospitalized patients with or at risk of developed ARDS showed statistically significant and clinically meaningful trend for mortality and survival time for all randomized subjects; company reported revised 28-day death rate of 7.7% in the EB-05 plus standard of care (SOC) arm vs. 40% in placebo/SOC arm in critically severe patients on extracorporeal membrane oxygenation or invasive mechanical ventilation plus organ support with ARDS at baseline (p=0.04); revised Survival Analysis using Cox's Proportional Hazard Model demonstrated those treated with EB-05/SOC had an 84% reduction in risk of dying vs. placebo/SOC at 28 days | 9/30/22 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody | COVID-19 | Showed an 84% reduction in the risk of dying vs. placebo in critically ill patients at 28 days | 9/30/22 | Infection |
| Innate Pharma SA., of Marseille, France | Lacutamab | Anti-KIR3DL2 antibody | Mycosis fungoides | Tellomak study showed lacutamab produced a global objective response rate of 28.6%; 2 complete responses; 4 partial responses | 9/23/22 | Infection |
| Kinarus Therapeutics Holding AG, of Basel, Switzerland | KIN-001 | Combination of pamapimod, a small molecule inhibitor of the p38 MAPK signaling pathway, and pioglitazone | COVID-19 | Independent data and safety monitoring board recommended discontinuing the study, saying there is low probability to show a statistically significant benefit with a reasonable number of hospitalized COVID-19 patients "in light of the lower than anticipated incidence of the primary endpoint due to evolution of the current treatment landscape" | 9/30/22 | Infection |
| Locus Biosciences Inc., of Research Triangle Park, N.C. | LBP-EC01 | CRISPR-enhanced bacteriophage precision medicine | Urinary tract infections caused by Escherichia coli | First patient dosed | 9/13/22 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Islatravir | Nucleoside reverse transcriptase translocation inhibitor; oral | HIV-1 infection | Initiated phase III study in combination with doravirine 100-mg | 9/20/22 | Infection |
| Omeros Corp., of Seattle | Narsoplimab | MASP-2 inhibitor | Critically ill COVID-19 | Data showed narsoplimab reduced mortality risk; Narsoplimab was not observed to shorten the time to recovery in critically ill patients; did not identify any new safety signals | 9/15/22 | Infection |
| Polypid Ltd., of Petach Tikva, Israel | D-Plex100 | Doxycycline (broad-spectrum antibiotic) | Prevention of surgical site infections in abdominal surgery | Data, published in Techniques in Coloproctology, from a study in patients undergoing elective colorectal surgery, showed the SSI rate within 30-day post-index surgery resulted in 64% statistically significant relative risk reduction for D-Plex100 plus standard of care (SoC) arm at 8% infection rate compared to 22% infection rate in the SoC-alone cohort (p=0.0115); no statistically significant difference in incidence of treatment-emergent events between the 2 groups | 9/19/22 | Infection |
| SAB Biotherapeutics Inc., of Sioux Falls, S.D. | SAB-176 | Fully human polyclonal antibody | H1N1 influenza virus infection | Phase IIa challenge trial showed SAB-176 reduced the viral load in subjects exposed to H1N1 influenza virus, improved symptoms by day 4 and shortened the timeframe for viral shedding. | 9/28/22 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Formulation for inhalation containing the broad-spectrum antiviral protein interferon-beta | Chronic obstructive pulmonary disease patients with a confirmed respiratory viral infection | Results showed human rhinovirus clearance more rapidly in patients treated with SNG-001 than placebo, with a statistically significant difference in the proportion of patients with detectable HRV in sputum at day 7 | 9/7/22 | Infection |
| Vaxart Inc., of South San Francisco | VXA-CoV2-1.1-S | Oral COVID-19 vaccine | COVID-19 | Top-line data showed study met its primary safety and secondary immunogenicity endpoints; improved antibody responses compared with VXA-CoV2-1 and boosted immune responses in subjects who previously received an mRNA vaccine; safe and well-tolerated; no vaccine related adverse events; increased levels of SARS-CoV-2-specific serum IgG and IgA antibodies at days 29 and 57 | 9/1/22 | Infection |
| Vaxcyte Inc., of San Carlos, Calif. | VAX-24 | 24-valent pneumococcal conjugate vaccine | Invasive pneumococcal disease | Completed enrollment in phase II study; enrolled 200 healthy adults 65 and older to evaluate the safety, tolerability and immunogenicity | 9/6/22 | Infection |
| Vir Biotechnology Inc., of San Francisco | VIR-3434 and VIR-2218 | Antibody designed to block entry of HBV and HDV viruses into hepatocytes and to reduce the level of virions and subviral particles in the blood; HBV-targeting siRNA | Chronic hepatitis D virus | First patient dosed in SOLSTICE study testing monotherapy and combination therapy | 9/22/22 | Infection |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Local, intranasal, anti-inflammatory therapy | Chronic rhinosinusitis | Lantern study significantly improved symptom severity from baseline of 3 cardinal symptoms at 7,500 mcg; improved from moderate or severe at baseline to mild or none at week 24 in nasal blockage, nasal discharge and facial pain/pressure (p<0.05)< td> | 9/10/22 | Inflammatory |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-220 | Delivers 6 months of continuous anti-inflammatory medication | Chronic rhinosinusitis | First patient dosed in part 2 of randomized stage of Beacon study; 21-point (37%) mean improvement in SNOT-22 total score at 6 weeks during the uncontrolled part 1 stage of the 24-week Beacon study | 9/13/22 | Inflammatory |
| Biojiva LLC, of Los Altos, Calif. | RT-001 | Synthetic linoleic acid | Amyotrophic lateral sclerosis | Patients treated with RT-001 experienced less worsening in ALSFRS-R score vs. placebo group at 24 weeks (3.3 point reduction vs. 4.6-point reduction); less worsening in their score on the 40-item ALS assessment questionnaire as compared to the placebo group (13.3-point increase from baseline vs. 17.2-point increase); did not achieve statistical significance due to the small size of the study; well-tolerated; will advancd to larger targeted study | 9/15/22 | Musculoskeletal |
| Coya Therapeutics Inc., of Houston | COYA-101 | Autologous, expanded Treg cell therapy | Amyotrophic lateral sclerosis | Data published in Neurology, Neuroimmunology, & Neuroinflammation showed safe and tolerated; increased Treg suppressive function; 8 patients in the open-label extension study completed the 24-week study; 75% of patient exhibited slow to no progression as measured by the ALSFRS-R; sub-analysis of 4/6 patients exhibited no decline over the 24-week treatment period; well-tolerated | 9/1/22 | Musculoskeletal |
| Entera Bio Ltd., of Jerusalem | EB-613 | Oral formulation of PTH (1-34) teriparatide | Postmenopausal women with osteoporosis | Data showed excellent correlations between the dose of the oral formulation of EB-613 and teriparatide hPTH(1-34) plasma concentrations at the 15-minute time point (R=0.996); linear dose response for the change in lumbar spine BMD after 6 months of treatment (R=0.998) | 9/12/22 | Musculoskeletal |
| Neurosense Therapeutics Ltd., of Cambridge, Mass. | Primec (ciprofloxacin + celecoxib) | Endoribonuclease DICER stimulator + COX2 inhibitor | Amyotrophic lateral sclerosis | Phase IIa data published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration showed combination product met the primary endpoint of safety in 12-month study of 15 participants; exploratory efficacy analyses showed treatment resulted in statistically significant changes in ALS-related biomarkers of serum neuron-derived exosomes such as TDP-43 and LC3, as measured by Exosort, indicating positive biological activity | 9/19/22 | Musculoskeletal |
| Santhera Pharmaceuticals AG, of Pratteln, Switzerland, and Reveragen Biopharma Inc., of Rockville, Md. | Vamorolone | Dissociative agonist | Duchenne muscular dystrophy | Results from 24-week analysis from the Vision-DMD study met its primary endpoint; statistically significant and clinically relevant improvement in time to stand from floor compared to placebo; no negative impact on biomarkers of bone health and no loss of linear growth; mild to moderate severity adverse events | 9/1/22 | Musculoskeletal |
| Virios Therapeutics Inc., of Atlanta | IMC-1 | Fixed-dose combination of famciclovir and celecoxib | Fibromyalgia | Top-line results showed Fortress study did not achieve statistical significance on prespecified primary efficacy endpoint of change from baseline to week 14 in the weekly average of daily self-reported average pain severity scores vs. placebo (p=0.302); analysis of data suggests a bifurcation of response based on timing of patient enrollment, with first half of study enrolling when the delta variant of COVID-19 was the dominant strain in the U.S. and second half enrolling when vaccination rates improved and less severe omicron variant was the dominant strain; IMC-1 demonstrated no improvement vs. placebo for patients in first half, while showing statistically significant improvement on primary pain reduction endpoint (p=0.03) at week 14, as well as a statistically significant improvement in the key secondary PROMIS Fatigue assessment (p=0.006) and Fibromyalgia Impact Questionnaire-Revised symptoms domain score (p=0.015) for patients in second half | 9/19/22 | Musculoskeletal |
| ABVC Biopharma Inc., of Fremont, Calif. | ABV-1505 | Plant-based drug; NET inhibitor | Attention deficit hyperactivity disorder | Enrollment progressing in phase II part 2 study | 9/23/22 | Neurology/Psychiatric |
| ABVC Biopharma Inc., of Fremont, Calif. | PDC-1421 | Capsule is a botanical investigational new drug containing the extract of Radix Polygalae (Polygala tenuifolia Willd.) | Attention deficit hyperactivity disorder | Planning to enroll 100 phase II study participants in Taiwan and U.S. | 9/23/22 | Neurology/Psychiatric |
| Acticor Biotech SAS, of Paris | Glenzocimab | Monoclonal antibody targeting platelet glycoprotein VI | Acute ischemic stroke | First patient in the U.S. enrolled in the ongoing phase II/III Actisave study | 9/26/22 | Neurology/Psychiatric |
| Aeon Biopharma Inc., of Irvine, Calif. | PrabotulinumtoxinA (ABP-450) | 900 kDa botulinum toxin type A complex produced by the bacterium Clostridium botulinum | Cervical dystonia | Top-line data showed study met primary and secondary endpoints with statistical significance in reduced signs and symptoms; safe and well-tolerated; mean improvement from baseline in Toronto Western Spasmodic Torticollis Rating Scale Total Score (p=0.0864); Clinicians Global Impression of Change Score p=0.0095; Patients Global Impression of Change Score (p=0.0028) | 9/23/22 | Neurology/Psychiatric |
| Algo Theraprutics SAS, of Suresnes, France | ATX-01 | Topical formulation of amitriptyline | Chemotherapy-induced peripheral neuropathy | Initiated phase II study; planning to enroll 240 patients; initial results expected in early 2024 | 9/1/22 | Neurology/Psychiatric |
| Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Prodrug of tramiprosate which inhibits the formation of toxic amyloid beta 42 oligomers | Alzheimer’s disease | Result showed rapid and robust plasma p-tau181 reductions by 41% (p=0.016) at 52 weeks; significantly reduced the plasma p-tau181/A?42 ratio by 37% at 52 weeks (p=0.032); improvement from baseline on memory tests at 3 and 6 months and maintained above baseline at 1 year | 9/20/22 | Neurology/Psychiatric |
| Biomind Labs Inc., of Toronto | BMND-01 | Inhaled version of N, N-dimethyltryptamine (DMT) | Treatment-resistant depression | Completion of dosing | 9/21/22 | Neurology/Psychiatric |
| Biovie Inc., of Santa Monica, Calif. | NE-3107 | Orally bioavailable small-molecule adrenal steroid hormone and insulin sensitizer; NFKB activation inhibitor; selective inhibitor of inflammatory ERK signaling | Alzheimer’s disease | Top-line data showed significant improvements in the global rating of change (overall impression of patient’s daily abilities) with NE-3107 treatment (p<0.0001 17 to p<0.05); significant improvements in cognition adas-cog12 scale; 82% of patients with mmse>=20 experienced a 2.6-point decrease in ADAS-Cog12 (p=0.0046); reductions in TNF-alpha; trending improvements in ratio of p-tau:Ab (p=0.055); no drug-related adverse events | 9/7/22 | Neurology/Psychiatric |
| Charsire Biotechnology Corp., of Taiwan | BAC | Multi-glycan complex and soybeans | Alzheimer's disease | Trial completed | 9/21/22 | Neurology/Psychiatric |
| Clene Inc., of Salt Lake City | CNM-Au8 | Catalytically active gold nanocrystal suspension | Amyotrophic lateral sclerosis | Significantly improved long-term survival with approximately a 70% decreased risk of mortality vs. original placebo randomization | 9/21/22 | Neurology/Psychiatric |
| Curasen Therapeutics Inc., of San Mateo, Calif. | CST-2032 | Oral beta-2 adrenoceptor agonist | Mild cognitive impairment or mild dementia due to either Parkinson’s or Alzheimer’s disease | Initiated dosing in patients in combination with CST-107; expected to enroll approximately 40 patients; top-line data expected in the third quarter of 2022 | 9/7/22 | Neurology/Psychiatric |
| EIP Pharma Inc., of Boston | Neflamapimod | Oral, brain-penetrating small molecule drug that inhibits the intra-cellular enzyme p38MAP kinase alpha | Dementia with Lewy bodies | Product generally safe and well tolerated, and improved vs. placebo dementia and motor function; results published in Nature Communications | 9/21/22 | Neurology/Psychiatric |
| Mind Medicine Inc., of New York | MM-120 | Optimized form of lysergic acid diethylamide | Generalized anxiety disorder | Patient dosing is ongoing; expected to enroll 200 participants; primary objective is to determine the reduction in anxiety symptoms 4 weeks after a single administration | 9/8/22 | Neurology/Psychiatric |
| Sorrento Therapeutics Inc., of San Diego | Resiniferatoxin | TRPV1 agonist | Pain associated with knee osteoarthritis | Completed enrollment; no dose-limiting toxicity observed; efficacy data expected in the second quarter of 2023 | 9/26/22 | Neurology/Psychiatric |
| ABVC Biopharma Inc., of Fremont, Calif. | Vitargus | Hyaluronic acid derived hydrogel; bio-degradable vitreous substitute | Ocular disorders | Initiated new clinical site in Australia | 9/9/22 | Ocular |
| Adverum Biotechnologies Inc., of Redwood City, Calif. | ADVM-022 (ixo-vec/ixoberogene soroparvovec) | AAV.7m8, carrying an aflibercept coding sequence | Wet age-related macular degeneration | First subject dosed in Luna study; interim data in 2023 | 9/14/22 | Ocular |
| Adverum Biotechnologies Inc., of Redwood City, Calif. | Ixoberogene soroparvovec (ADVM-022) | Gene therapy | Wet age-related macular degeneration | Post-hoc analysis of Optic trial showed single injection of 2E11 dose resulted in 93% and 55% reduction in mean intraretinal (IRF) and subretinal fluid (SRF) volume, respectively, from baseline to week 48; percentage of subjects with dry SRF, across both 6E11 and 2E11 dose groups, increased through week 48 with a 200% increase in the 2E11 dose group; 60% of Optic participants receiving 2E11 dose had dry IRF at week 48, representing a 50% increase from baseline | 9/30/22 | Ocular |
| Ashvattha Therapeutics Inc., of Redwood City, Calif. | D-4517.2 | VEGFR/PDGFR tyrosine kinase inhibitor conjugated to a hydroxyl dendrimer | Wet age-related macular degeneration or diabetic macular edema | First patient enrolled; to evaluate the safety and relative pharmacodynamic effect of different doses of subcutaneously compared to intravitreal injection | 9/7/22 | Ocular |
| Nanoscope Therapeutics Inc., of Bedford, Texas | MCO-010 | Multicharacteristic opsin ambient-light activatable optogenetic monotherapy | Stargardt disease | Completed enrollment; 6-month data from the phase II Starlight trial expected in the first half of 2023 | 9/13/22 | Ocular |
| Ocuphire Pharma Inc., of Farmington Hills, Mich. | APX-3330 | Small-molecule, oral inhibitor of the transcription factor regulator Ref-1 | Diabetic retinopathy | Last patient completed visit; top-line results expected in the fourth quarter of 2022; interim results showed favorable safety and tolerability profile | 9/8/22 | Ocular |
| Trefoil Therapeutics Inc., of Chicago | TTHX-1114 | Engineered variant of fibroblast growth factor-1 | Fuchs endothelial corneal dystrophy | Recovery of vision to 20/40 in a mean time of 4.2 weeks; significant dose-dependent effect was found on the recovery of central corneal thickness | 9/29/22 | Ocular |
| Innovent Biologics Inc., of San Francisco and Suzhou, China | IBI-362 (mazdutide) | GLP-1 and glucagon receptor dual agonist | Obesity | First subject dosed; to evaluate the efficacy and safety of mazdutide in Chinese participants; | 9/6/22 | Other/Miscellaneous |
| Revance Therapeutics Inc., of Nashville, Tenn. | Daxxify (daxibotulinumtoxinA-lanm) | Botulinum toxin A stimulator | Forehead lines and glabellar lines | Data published in Aesthetic Surgery Journal reported impact of injection on brow position and frontalis muscle activity from post-hoc analysis in phase IIa trial, and open-label Sakura safety study showed treatment of frown lines results in overall positive change in eyebrow position even with modest reduction in forehead muscle activity | 9/19/22 | Other/Miscellaneous |
| Sensorion SA, of Paris | SENS-401 (arazasetron) | 5-HT3 receptor antagonist; calcineurin inhibitor | Hearing loss | First patient enrolled | 9/14/22 | Other/Miscellaneous |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel (ZYN-002) | Cannabidiol transdermal gel | 22q11.2 deletion syndrome | Results showed statistically significant improvements from baseline in anxiety-related and behavioral symptoms in children and adolescents; well-tolerated; mild application site adverse events; 1 patient discontinued treatment due to adverse events not related to Zygel | 9/8/22 | Other/Miscellaneous |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | Ifenprodil (NP-120) | NMDA receptor antagonist | Idiopathic pulmonary fibrosis (IPF) and chronic cough | Data from secondary endpoints of IPF showed unchanged diffusing capacity for carbon monoxide (DLCO) over 12 weeks (p>0.99); no change in dyspnea scale over 12 weeks; increase of 7 points in the King’s Brief Interstitial Lung Disease Questionnaire (p=0.1209); secondary endpoints of cough data showed significant improvements; reduction of 23.6 mm from baseline in the cough visual analogue scale (p=0.0005); improvement of 10.05 points in the mean score on the Leicester Cough Questionnaire (p=0.0296); well-tolerated with a high overall compliance rate; no new safety concerns | 9/1/22 | Respiratory |
| Eloxx PharmaceuticalsInc., of Watertown, Mass. | ELX-02 | Small molecule drug candidate designed to restore production of full-length functional proteins | Cystic fibrosis Class 1 | Study testing combo of ELX-02 with Kalydeco (ivacaftor, Vertex Phamaceuticals Inc.) did not achieve statistical significance for efficacy endpoints including changes from baseline in sweat chloride content (SCC) and FEV1; no incremental improvement; higher baseline SCC levels demonstrated increased responses as indicated by SCC (p=0.00013 at day 35); lung drug exposure with inhaled delivery of ELX-02 expected to be at least 50-fold greater than with subcutaneous delivery; combination was well-tolerated; no treatment-related serious adverse events | 9/14/22 | Respiratory |
| Pliant Therapeutics Inc., of South San Francisco | PLN-74809 | Oral small-molecule dual selective inhibitor of ?vß6 and ?vß1 integrins | Idiopathic pulmonary fibrosis (IPF) | Data safety monitoring board recommended phase IIa study without modification; interim 12-week data expected in early 2023; completed enrollment of 320-mg cohort | 9/1/22 | Respiratory |
| Trevi Therapeutics Inc., of New Haven, Conn. | Haduvio (oral nalbuphine ER) | Opioid receptor kappa agonist; opioid receptor mu antagonist | Chronic cough in idiopathic pulmonary fibrosis | Data from the full set of subjects in the Canal trial showed the study was statistically significant for the trial's primary endpoint and showed a 52.5% change compared to placebo (p<0.0001), with a 75.1% reduction in geometric mean percent change daytime cough frequency for haduvio< td> | 9/19/22 | Respiratory |
| Anebulo Pharmaceuticals Inc., of Austin, Texas | ANEB-001 | Small-molecule cannabinoid receptor antagonist | Acute cannabinoid intoxication and substance addiction | Interim data showed lower doses reduced the negative effects of higher doses of THC; substantial increase in feeling high and body sway, decreased alertness, and slightly increased heart rate compared to baseline; significant and sustained reductions in the visual analogue scale (VAS) feeling high score (p<0.001), improvement in the vas alertness scale (p<0.01); reduction thc-induced body sway (p<0.01) compared to placebo; 100% of subjects met threshold for feeling high; rapid absorption and dose-related plasma exposure< td> | 9/26/22 | Toxicity and Intoxication |
| Aivita Biomedical Inc., of Irvine, Calif. | AV-GBM-1 | Personal dendritic cell vaccine | Glioblastoma | Data published in the Journal of Oncology Research and Therapy showed 50% improvement in progression-free survival vs. standard of care in patients with newly diagnosed disease | 10/4/22 | Cancer |
| Allogene Therapeutics Inc., of South San Francisco | ALLO-501A | Anti-CD19 products | Relapsed or refractory large B-cell lymphoma | Initiated single-dose phase II study | 10/7/22 | Cancer |
| Anaptysbio Inc., of San Diego, and GSK plc, of London | Jemperli (dostarlimab) | Anti-PD-1 inhibitor | Metastatic nonsquamous non-small-cell lung cancer | Head-to-head trial vs. Keytruda (pembrolizumab, Merck & Co. Inc.), both in combination with chemotherapy, met primary endpoint of objective response rate as assessed via RECIST1.1 | 10/5/22 | Cancer |
| Anaptysbio Inc., of San Diego, and GSK plc, of London | Jemperli (dostarlimab) and cobolimab | Anti-PD-1 inhibitor and anti T-cell immunoglobulin mucin protein-3 antibody | Metastatic nonsquamous non-small-cell lung cancer | GSK advancing both arms of Costar Lung trial to test both doublet and triplet combination of dostarlimab/chemotherapy and cobolimab/dostarlimab/chemotherapy | 10/5/22 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Camizestrant | Next-generation oral selective estrogen receptor degrader | ER-positive locally advanced or metastatic breast cancer | Study met primary endpoint; statistically significant and clinically meaningful progression-free survival at 75-mg and 150-mg dose levels vs. fulvestrant; well-tolerated; no new safety signals identified | 10/26/22 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdualag (relatlimab + nivolumab) | Fixed-dose combination of anti-PD-1 and anti-LAG-3 antibodies | Unresectable or metastatic melanoma | Achieved primary endpoint; completely cleared all viable tumor in 57% of patients; pathologic response rate was 70%; median of 24.4 months follow-up in 29 patients; rate of recurrence-free survival (RFS) was 97% at 1 year; 82% at 2 year; overall survival rates for all patients were 93% and 88% at 1 and 2 year, respectively; results published in Nature | 10/25/22 | Cancer |
| Can-Fite BioPharma Ltd., of Petach Tikva, Israel | Namodenoson | A3 adenosine receptor agonist | Advanced hepatocellular carcinoma | Case study of a patient in the study to be presented at The Liver Meeting; after 4 treatments, tumor mass, ascites and peritoneal carcinomatosis disappeared; alanine transaminase and aspartate aminotransferase levels normalized and were maintained for 5 years | 10/25/22 | Cancer |
| Hutchmed Ltd., of Hong Kong | Fruquintinib | Inhibits VEGFR 1, 2 and 3 | Advanced renal cell carcinoma | Intiated phase II/III study in combination with sintilimab; first patient dosed | 10/27/22 | Cancer |
| Imaginab Inc., of Los Angles | CD8 ImmunoPET | Diagnostic imaging of CD8 | Melanoma | First patient dosed at the Macquarie University Hospital in Sydney, Australia in the ongoing Ipredict phase IIb study | 10/25/22 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha (IMP-321) | Soluble LAG-3Ig fusion protein | Head and neck squamous cell carcinoma | Independent data monitoring committee reviewed initial safety data and recommended continuing the trial with no modifications | 10/26/22 | Cancer |
| Innocare Pharma Ltd., of Beijing | ICP-723 | Pan-TRK Inhibitor | Advanced or metastatic solid tumors harboring NTRK fusion genes | Enrolled first adolescent patient in the study | 10/31/22 | Cancer |
| Instil Bio Inc., of Dallas | ITIL-168 and ITIL-306 | Autologous cell therapies made from tumor infiltrating lymphocytes | Solid tumors | Pausing enrollment in the phase II Delta-1 study of ITIL-168 and phase I study of ITIL-306 due to a decrease in the rate of successful manufacturing of ITIL-168; no safety issues found in Delta-1; plans to evaluate manufacturing protocol and update investors by early in first quarter of 2023 | 10/31/22 | Cancer |
| Istari Oncology Inc., of Durham, N.C. | Lerapolturev (PVSRIPO) | Genetically engineered, live-attenuated oncolytic poliovirus derived from a Sabin serotype 1; targeting poliovirus receptor CD155 | Metastatic melanoma | Data in combination with anti-PD-1 infusions showed 71% of clinical benefit rate; anti-tumor responses in both injected and non-injected lesions; complete resolution of a non-injected lung metastasis; patient enrollment ongoing to the enhanced dosing regimen; data expected in the second quarter 2023 | 10/18/22 | Cancer |
| Jaguar Health Inc., of San Francisco | Crofelemer | Oral plant-based non-opioid agent | Chemotherapy-induced diarrhea | Results from investigator-initiated HALT-D trial published in Breast Cancer Research and Treatment | 10/26/22 | Cancer |
| Leap Therapeutics Inc., of Cambridge, Mass. | DKN-01 | Anti-Dickkopf-1 (DKK1) antibody | Gastric or gastroesophageal junction cancer | First patient enrolled in Distinguish study in combination with tislelizumab (Beigene Ltd.) | 10/12/22 | Cancer |
| Leap Therapeutics Inc., of Cambridge, Mass. | DKN-01 | Anti-Dickkopf-1 antibody | Second-line advanced colorectal cancer | First patient enrolled in the study testing DKN-01 combined with bevacizumab and chemotherapy; initial cohort will have 20 patients that will be expanded into a 130-patient randomized controlled trial against the combination of bevacizumab and chemotherapy; primary endpoint is progression-free survival; secondary endpoints include overall response rate, duration of response and overall survival | 10/17/22 | Cancer |
| Morphosys AG., of Munich | Tulmimetostat (CPI-0209) | Selective dual inhibitor of EZH2 and EZH1 | Advanced tumor | Preliminary results from 10 evaluable patients with ovarian clear cell carcinoma showed 4 patients with partial responses and 3 with stable disease; 5 cases of stable disease in patients with metastatic castration-resistant prostate cancer; 2 partial responses in endometrial carcinoma; tulmimetostat treatment tied to thrombocytopenia in 47.1% of patients | 10/27/22 | Cancer |
| Oblato Inc., of Princeton, N.J. | OKN-007 (disufenton sodium) | Neuroprotectant with free radical-scavenger properties | Recurrent glioblastoma multiforme | Completed enrollment in the in the study testing OKN-007 in combination with temozolomide; data expected in 2023 | 10/25/22 | Cancer |
| PDS Biotechnology Corp., Florham Park, N.J. | PDS-0101-based triple combination therapy | DOTAP lipid and the E7 antigen from HPV 16 | Advanced human papilloma virus-based cancers | Interim data showed 66% of patients in the cohort were alive at a median follow up of 16 months; median overall survival of 3-4 months; 48% of patients experienced grade 3 treatment-related adverse events; no grade 5 treatment-related adverse events; 75% of CPI naïve patients were alive at a median of 25 months of follow up; 38% (3/8) of responders had a complete response | 10/11/22 | Cancer |
| Prolynx LLC, of San Francisco | PLX-038 | Long-acting prodrug of topoisomerase 1 inhibitor SN-38 | Platinum-resistant ovarian cancer | First patient treated | 10/7/22 | Cancer |
| Puma Biotechnology Inc., of Los Angeles | Neratinib (PB-272, Nerlynx) | Pan-EGFR inhibitor | EGFR exon 18-mutant non-small-cell-lung cancer | Interim efficacy results showed objective response rate was 35%; stable disease lasting for ? 48 weeks was observed in 7 patients | 10/27/22 | Cancer |
| Radiopharm Theranostics Ltd., of Australia | F-18 Pivalate | Targets fatty acid synthetase | Brain metastases/PET imaging | Data showed superior imaging performance; significant tumor uptake | 10/18/22 | Cancer |
| Sanofi SA, of Paris | THOR-707 (SAR-444245) | Pegylated version of interleukin-2 | Tumors | Discontinued 3-week dose schedule for phase II study due to lower data than projected efficacy; decision not based on any safety-related issues | 10/28/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agent | Triple negative breast cancer | Preliminary data showed 83% patient lesions had a CAIX strong expression; best detected metastatic sites are bone | 10/18/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agent | Non-muscle invasive bladder cancer | Data showed improved tumor targeting and biodistribution; no systemic distribution of radiation | 10/18/22 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab (KN-035) | Single-domain antibody against PD-L1 | Sarcoma | Data safety monitoring board recommended trial proceed as planned following the review of 12-week safety data; well-tolerated at 600-mg dose | 10/6/22 | Cancer |
| Ultimovacs ASA, of Oslo, Norway | UV-1 | Cancer vaccine | Non-small-cell lung cancer | Enrolled first of 138 patients in the Lungvac study testing UV-1 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) compared to Keytruda alone; primary endpoint is progression-free survival; secondary endpoints include overall survival, response rate, duration of response and safety; top-line data expected by the end of 2024 | 10/25/22 | Cancer |
| Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Oral, once-daily CETP inhibitor | Cardiovascular disease | Started dose-finding study testing drug as adjunct to stable statin therapy in Japanese patients with dyslipidemia | 10/5/22 | Cardiovascular |
| Quantum Genomics SA, of Paris | Firibastat | Brain aminopeptidase A inhibitor; prodrug | Cardiovascular | Failed to showed significant greater efficacy than placebo, so prematurely discontinued Refresh study for ethical reasons | 10/28/22 | Cardiovascular |
| Valbiotis SA, of La Rochelle, France | Totum-070 | Combination of plant extracts | LDL cholesterol | Data from Heart study showed, in commercially targeted population with cholesterol levels above 130 mg/dl at randomization, drug reduced blood LDL cholesterol levels by 13.7% at 3 months and by 14.3% at 6 months vs. placebo, with a very high response rate; new data confirmed intestinal and hepatic mode of action | 10/3/22 | Cardiovascular |
| Artiva Biotherapeutics Inc., of San Diego | AB-101 | Optimized and cryopreserved off-the-shelf NK cell therapy | Mild-to-moderate atopic dermatitis | Enrollment passed the halfway point | 10/18/22 | Dermatologic |
| Connect Biopharma Holdings Ltd., of San Diego and Taicang, China | CBP-201 | Antibody targeting interleukin-4 receptor alpha | Atopic dermatitis | Top-line data from trial in Chinese patients with moderate to severe AD showed primary endpoint of IGA of 0 or 1 with at least 2 grades of reduction at week 16 from baseline was 30.3% in treatment group vs. 7.5% for placebo group (p<0.001); 4 cbp-201 also met key secondary endpoints, including 83.1%, 62.9% and 35.8% of patients achieving a 50%, 75%, 90% reduction in eczema area severity index score (easi-50, easi-75, easi-90) from baseline compared to 41.1%, 23.4% 6.3% for placebo (p<0.001), respectively; significant improvement pruritus with 35% experiencing or greater on peak pruritus-numerical rating scale vs. 9.6% (p< .001); generally well-tolerated< td> | 10/4/22 | Dermatologic |
| Devonian Health Group Inc., of Quebec City | Thykamine (PUR-0110) | IL-1 beta ligand modulator; TNF alpha ligand modulator | Atopic dermatitis | Results published in the Journal of Drugs in Dermatology showed trial achieved an IGA of clear/almost clear; at least a 2-grade improvement from baseline in IGA (30.8%) compared to placebo (6.7%; p=0.014); changes differentiated significantly from placebo from day 14 until day 29 (p? 0.001); rapidly improved itch from baseline by day 7 with a statistically difference vs. placebo at day 21 and day 29 (p? 0.01); statistically significant in POEM outcome from placebo at day 21 and 29 (p? 0.01); well-tolerated | 10/6/22 | Dermatologic |
| Kalvista Pharmaceuticals Inc., of Cambridge, Mass. | KVD-824 | Plasma kallikrein inhibitor | Hereditary angioedema | The Komplete study was terminated due to elevation in liver enzymes (ALT/AST) | 10/4/22 | Dermatologic |
| Q32 Bio Inc., of Waltham, Mass. | ADX-914 | Fully human anti-IL-7R? antibody | Persistent, moderate-to-severe atopic dermatitis | First patient randomized | 10/27/22 | Dermatologic |
| Amolyt Pharma SA, of Lyon, France | AZP-3601 | Therapeutic peptide targeting parathyroid hormone receptor | Hypoparathyroidism | Data showed 93% of patients had discontinued both active vitamin D and oral calcium supplementation at the end of the 3-month treatment period; mean albumin-adjusted serum calcium remained within the target range; 24-hour urinary excretion of calcium was rapidly normalized in all patients except 1; P1NP and CTX increased after 2 weeks of treatment and remained within their mid-normal range through the end of the study; bone mineral density was stable; well-tolerated; no serious adverse events; mild to moderate adverse events | 10/12/22 | Endocrine/Metabolic |
| Disc Medicine Inc., of Watertown, Mass. | Bitopertin | Glycine transporter 1 inhibitor | Erythropoietic protoporphyria | Started the Aurora study assessing changes in protoporphyrin IX levels, safety, tolerability, photosensitivity and other measurements; top-line data expected in 2023 | 10/31/22 | Endocrine/Metabolic |
| First Wave Biopharma Inc., of Boca Raton, Fla. | Adrulipase | Microgranule delivery formulation of the lipase enzyme | Exocrine pancreatic insufficiency | Contracted with Rho to run the clinical trial that is scheduled to begin before end of 2022 | 10/31/22 | Endocrine/Metabolic |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-41 | Thyroid hormone receptor beta agonist | Nonalcoholic steatohepatitis | Dosed first patient in 52-week trial enrolling liver biopsy-proven patients | 10/4/22 | Gastrointestinal |
| Durect Corp., of Cupertino, Calif. | DUR-928 (larsucosterol) | Inhibits DNA methyltransferases | Alcohol-associated hepatitis | Achieved enrollment of more than 200 of the planned 300 patients in AHFIRM study; completion of enrollment expected in the second quarter of 2023 | 10/6/22 | Gastrointestinal |
| Eupraxia Pharmaceuticals Inc., of Victoria, British, Columbia | EP-104IAR | Encapsulated fluticasone propionate within a microns-thin polymer membrane | Eosinophilic esophagitis | Initiated phase II study; initial data readout expected in the first half of 2023 | 10/12/22 | Gastrointestinal |
| Galectin Therapeutics Inc., of Norcross, Ga. | Belapectin | Carbohydrate-based drug that inhibits the galectin-3 protein | Liver cirrhosis caused by nonalcoholic steatohepatitis | Data safety monitoring board concluded Navigate study continue as designed without modifications; treatment safe and well-tolerated | 10/6/22 | Gastrointestinal |
| Hepion Pharmaceuticals Inc., of Edison, N.J. | Rencofilstat | Cyclophilin inhibitor | Nonalcoholic steatohepatitis | Results published in Hepatology Communications showed study met safety, tolerability and pharmacokinetics primary endpoints; reductions in ALT, ProC3 and C6M levels with longer treatment duration; enrollment initiated in phase IIb Ascend-NASH study | 10/26/22 | Gastrointestinal |
| Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Guselkumab and golimumab | Fully human monoclonal antibody that selectively binds to the p19 subunit of IL-23 and inhibits its interaction with the IL-23 receptor; human monoclonal antibody targeting TNF-alpha | Moderately to severely active ulcerative colitis | Data showed rate of clinical remission was 47.9% in patients who received combination induction therapy compared with either treatment alone (31% and 20.8%, respectively) at 38 weeks (p=<0.05); improvement in endoscopic and composite histologic-endoscopic endpoints compared to either treatment alone; comparable rates of adverse events< td> | 10/10/22 | Gastrointestinal |
| Morphic Therapeutic Inc., of Waltham, Mass. | MORF-057 | Integrin alpha4 beta7 inhibitor | Moderate to severe ulcerative colitis | Completed enrollment of 30 patients in the main cohort of the Emerald-1 phase IIa study; additional patients undergoing screening may be added | 10/24/22 | Gastrointestinal |
| Palatin Technologies Inc., of Cranbury, N.J. | PL-8177 | Melanocortin-1 receptor agonist | Ulcerative colitis | First patient enrolled; interim data expected in the first quarter of 2023; top-line data in the third quarter of 2023 | 10/20/22 | Gastrointestinal |
| Vectivbio Holding AG, of Basel, Switzerland | Apraglutide | Long-acting synthetic GLP-2 agonist | Short bowel syndrome with intestinal failure and colon-in-continuity | Interim data showed 50% reduction in parenteral support (PS) volume at 6 months; 47% reduction in parenteral energy content; 80% of patients were clinical responders;achieved at least one day off PS at 6 months | 10/13/22 | Gastrointestinal |
| Muvon Therapeutics AG, of Zurich, Switzerland | Autologous skeletal muscle injections | Regenerative cell therapy | Stress urinary incontinence | First female patient enrolled; to evaluate safety and efficacy of a low and high dose of injections | 10/27/22 | Genitourinary/Sexual Function |
| Hutchmed Ltd., of Hong Kong | Sovleplenib | Syk inhibitor; small molecule | Autoimmune hemolytic anemia | Initiated phase II/III study; first patient dosed on Sept. 30, 2022 | 10/10/22 | Hematologic |
| Merck KGaA, of Darmstadt, Germany | Evobrutinib | BTK inhibitor | Multiple sclerosis | Data showed evobrutinib demonstrated low disease activity and stable EDSS; sustained and ongoing reductions in blood NfL levels | 10/26/22 | Immune |
| Abionyx Pharma SA, of Toulouse, France | CER-001 | Negatively charged lipoprotein particle containing human recombinant apoA-I, the natural HDL protein and natural phospholipids sphingomyelin and dipalmitoylphosphatidylglycerol | Sepsis | Last patient enrolled in phase IIa trial in patients with sepsis at high risk of developing acute kidney injury; top-line results due by late fall 2022 | 10/5/22 | Infection |
| Apogenix AG, of Heidelberg, Germany | Asunercept | CD95 antagonist | COVID-19 | Results from 435 patients showed statistically significant faster clinical improvement compared to SOC group (p= 0.038); marked reduction of all-cause mortality at all time points; favorable safety profile; pivotal phase III study initiated | 10/19/22 | Infection |
| Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | VLP-based, non-adjuvanted COVID-19 booster vaccine | COVID-19 prophylaxis | Neutralization titers were 6 times higher than pre-boost titers against Wuhan strain and nearly 10 times higher than the pre-boost titers for omicron BA.1 in 39 participants, 6 months after booster | 10/17/22 | Infection |
| Cardiol Therapeutics Inc., of Oakville, Ontario | Cardiolrx | Oral cannabidiol formulation | Hospitalized COVID-19 with prior history of, or risk factors for, cardiovascular disease | Discontinuing the Lancer study due to lack of eligible patients to support recruitment | 10/25/22 | Infection |
| Enanta Pharmaceuticals Inc., of Watertown, Mass. | EDP-938 | N-protein inhibitor | Acute respiratory syncytial virus | Started phase IIb study in adults with RSV at high risk of complications, including elderly and/or those with congestive heart failure, chronic obstructive pulmonary disease or asthma; primary endpoint is time to resolution of RSV lower respiratory tract disease symptoms as assessed by Respiratory Infection Intensity and Impact Questionnaire symptom scale through day 33 | 10/3/22 | Infection |
| Everest Medicines of Shanghai, and Providence Therapeutics Inc., of Calgary, Alberta | PTX-COVID19-B | mRNA vaccine designed to encode the S protein of SARS-CoV-2 encapsulated in a lipid nanoparticle | COVID-19 | Top-line data showed study met the criteria for non-inferiority compared to Comirnaty (tozinameran, Pfizer Inc. and Biontech SE) for analysis of immune responses; well-tolerated; safety and tolerability profile similar to Comirnaty | 10/19/22 | Infection |
| Inotrem SA, of Paris | Nangibotide | Synthetic 12-mer peptide acting as a TREM-1 inhibitor | Septic shock | Study (n=361) showed clinically and statistically significant at higher concentrations of sTREM-1; improved respiratory, cardiovascular and renal function; improvement in all-cause mortality at day 28 | 10/13/22 | Infection |
| Inotrem SA, of Paris | Nangibotide | Synthetic 12-mer peptide acting as a TREM-1 inhibitor | Hospitalized COVID-19 in critical care units and experiencing acute respiratory distress | Drug improved 7-point clinical status ordinal scale from baseline to day 28 (p=0.040) and produced a 43% relative reduction in mortality (p=0.030) | 10/25/22 | Infection |
| Kintor Pharmaceutical Ltd., of Suzhou, China | KX-826 (pyrilutamide) | Androgen receptor antagonist | Acne vulgaris | Completed enrollment in the 160-patient study measuring changes in the Investigator Global Assessment | 10/16/22 | Infection |
| Matinas Biopharma Holdings Inc., of Bedminster, N.J. | MAT-2203 | Lipid nanocrystal formulation of amphotericin B | Cryptococcal meningitis | Interim results from 40 patients in cohort 4 of the Enact study showed 2-week survival was 95%, and 90% of patients observed to-date were alive; plans to start a phase III study in the first quarter of 2023 | 10/21/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Omicron BA.4/BA.5-adapted bivalent | COVID-19 vaccine mRNA vaccine | COVID-19 | Data showed substantial increase in the Omicron BA.4/BA.5 neutralizing antibody response above pre-booster levels at 30-mcg; well-tolerated in individuals aged 18 to 55 years of age and those older than 55 years of age | 10/13/22 | Infection |
| Recce Pharmaceuticals Ltd., of Sydney | R-327 | Broad-spectrum, synthetic polymer anti-infective | Urinary tract infections | Expects to initiate dosing in first patient in early 2023 | 10/3/22 | Infection |
| Recce Pharmaceuticals Ltd., of Sydney | R-327 | Broad-spectrum, synthetic polymer anti-infective | Diabetic foot ulcer infections | Trial to assess R-327 as spray-on (topical) broad-spectrum antibiotic therapy for mild skin and soft tissue DFUs, with first patient expected to be dosed in fourth quarter of 2022 | 10/3/22 | Infection |
| Sifi SpA, of Catania, Italy | Akantior (polyhexanide) | Anti-amoebic polymer | Acanthamoeba keratitis | Achieved pre-defined non-inferiority vs. combination regimen of polihexanide 0.2 mg/mL + propamidine 1 mg/mL; 84.8% of patients reached a clinical resolution of acanthamoeba keratitis and associated inflammation within a median of 4 months of treatment vs. 88.5% of the control arm; similar safety and tolerability profile reported in healthy volunteers | 10/13/22 | Infection |
| Synairgen plc, Southampton, U.K. | SNG-001 | Interferon-beta for inhalation | COVID-19 | There was 1 hospitalization out of 110 patients given SNG-001 compared to 7 out of 110 for patients treated with placebo (p=0.07) | 10/4/22 | Infection |
| Vaccitech plc, of Oxford, U.K. | VTP-300 | Nonreplicating viral vectors encoding hepatitis B virus antigens | Chronic hepatitis B infection | Treated first of 120 patients in the HBV002 study testing 3 dose regimens of VTP-300 plus a low-dose anti-PD-1 antibody | 10/31/22 | Infection |
| Viiv Healthcare Ltd., of London | N6LS (VH-3810109) | Neutralizing antibody | HIV infection | Single infusion showed strong antiviral efficacy; well-tolerated; median viral count reduction from baseline; robust decline in viral load consistent with antiviral activity; no adverse events leading to discontinuation | 10/25/22 | Infection |
| Vir Biotechnology Inc., of San Francisco | VIR-2482 | Intramuscularly administered influenza A-neutralizing monoclonal antibody | Influenza | First subject dosed | 10/18/22 | Infection |
| Vivacelle Bio Inc., of Fort Wayne, Ind. | VBI-S | Intravenously injected fluid that is comprised of phospholipid nanoparticles | Hypovolemia due to Sepsis | Study (n=13) met the primary goal; reached the secondary endpoint of the trial; clinically significant improvement in lung, kidney and liver function; no drug related adverse events | 10/20/22 | Infection |
| Eupraxia Pharmaceuticals Inc., of Victoria, British, Columbia | EP-104IAR | Encapsulated fluticasone propionate within a microns-thin polymer membrane | Osteoarthritis | Completed data safety monitoring board review; no drug-related serious events; clean safety profile; reductions in inflammation | 10/11/22 | Inflammatory |
| Paradigm Biopharmaceuticals Ltd., of New York | iPPS | Injectable pentosan polysulfate sodium | Osteoarthritis | In the PARA_OA_008 study, 73% of patients achieved a ?30% improvement in the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) and 60% achieved a ?50% improvement in WOMAC | 10/4/22 | Inflammatory |
| Bridgebio Pharma Inc., of San Francisco | BBP-418 | Glycosylation substrate prodrug | Limb-girdle muscular dystrophy type 2i | Data from 12-month phase II study showed increased glycosylation of ?DG in all dose cohorts at day 90; greater than 75% reduction in creatine kinase sustained over 12 months; no dose limiting toxicities; no treatment related adverse events | 10/14/22 | Musculoskeletal |
| Corcept Therapeutics Inc., of Menlo Park, Calif. | Dazucorilant | Selective cortisol modulator | Amyotrophic lateral sclerosis | Initiated phase II Dazals study | 10/11/22 | Musculoskeletal |
| Edgewise Therapeutics Inc., of Boulder, Colo. | EDG-5506 | Small-molecule myosin modulator | Duchenne muscular dystrophy | Initiated phase II study in children; placebo-controlled trial will assess the effect of 3 doses | 10/27/22 | Musculoskeletal |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | Small molecule; p38 inhibition | Facioscapulohumeral muscular dystrophy | Data from open-label extension portion showed 97% participants remained on treatment at week 96; improved muscle function; favorable safety profile; well-tolerated; no serious events | 10/12/22 | Musculoskeletal |
| NS Pharma Inc., of Paramus, N.J. | Viltepso (viltolarsen) | Antisense morpholino oligonucleotide | Duchenne muscular dystrophy | Data from the open-label extension study at week 205 showed time to stand, time to run/walk 10 meters and time to climb 4 stairs were better for patients treated with Viltepso compared to matched patients in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study | 10/24/22 | Musculoskeletal |
| Clene Inc., of Salt Lake City | CNM-Au8 | Gold nanocrystal suspension | Amyotrophic lateral sclerosis | Top-line results from Healey ALS Platform trial showed primary endpoint of slope of change in ALS Functional Rating Scale Revised scores adjusted for mortality was not significant (2% slowing, 95% CI: -20% to +19%) at 24 weeks; secondary endpoints of Combined Assessment of Function and Survival and slow vital capacity were also not met at 24 weeks across the combined 30-mg and 60-mg doses; prespecified exploratory analyses of secondary survival endpoint demonstrated a >90% reduction in risk of death alone or in risk of death/permanently assisted ventilation at 24 weeks, when adjusted for baseline imbalances in risk (p=0.028 to p=0.075, unadjusted for multiple comparisons) with 30-mg dose | 10/3/22 | Musculoskeletal |
| Actinogen Medical Ltd., of Sydney | Xanamem | Blocks the excess production of cortisol and 11?-HSD1 enzyme | Alzheimer's disease | Data showed blood pTau levels above the median value of 6.74 pg/mL (p = 0.09); CDR-SB effect of 0.6-0.8 points is larger than the 0.45 points reported; 60%-80% reduction in progression over 12 weeks compared with placebo | 10/10/22 | Neurology/Psychiatric |
| Alterity Therapeutics Ltd., of San Francisco | ATH-434 | Small molecule designed to inhibit aggregation of pathological proteins | Multiple system atrophy | Received approval in Australia from the St. Vincent’s Hospital Melbourne Human Research Ethics Committee for phase II study | 10/13/22 | Neurology/Psychiatric |
| Alzamend Neuro Inc., of Atlanta | AL-001 | Lithium-salicylate-L-proline engineered ionic cocrystal lithium delivery system | Dementia related to Alzheimer’s disease | Dosed first healthy subject in phase IIa multiple ascending-dose study | 10/5/22 | Neurology/Psychiatric |
| Annovis Bio Inc., of Berwyn, Pa. | Buntanetap | Oral translational inhibitor of neurotoxic aggregating proteins | Parkinson's and Alzheimer's diseases | Results published in the Journal of Prevention of Alzheimer's Disease showed well-tolerated; safe; significantly improved cognition and motor functions | 10/11/22 | Neurology/Psychiatric |
| Apnimed Inc., of Cambridge, Mass. | AD-109 (atomoxetine + aroxybutynin) | Norepinephrine reuptake inhibitor and anti-muscarinic agent | Obstructive sleep apnea | In the Mariposa phase IIb study, Apnea-Hypopnea Index was improved for both dose groups compared to placebo (p<0.001)< td> | 10/17/22 | Neurology/Psychiatric |
| Arrivo Bioventures LLC, of Morrisville, N.C. | SP-624 | Sirtuin 6 activator | Major depressive disorder | The 319-patient SP-624-201 study wasn’t positive on the primary endpoint of the Montgomery-Asberg Depression Rating Scale (MADRS) score; post-hoc analysis showed female patients had a 3.9-point reduction on the MADRS score after 4 weeks of treatment compared to placebo (p=0.008) | 10/17/22 | Neurology/Psychiatric |
| Atai Life Sciences NV, of Berlin | PCN-101 (R-ketamine) | NMDA receptor antagonist | Treatment-resistant depression | Completed enrollment in the study; top-line data expected near end of 2022 | 10/25/22 | Neurology/Psychiatric |
| Athira Pharma Inc., of Bothell, Wash. | Fosgonimeton (ATH-1017) | Enhancer of hepatocyte growth factor and its receptor MET | Mild to moderate Alzheimer’s disease | Independent data monitoring committee recommended continuation of the LIFT-AD phase II/III study with the addition of fewer than 150 patients for a total enrollment of less than 300 patients; enrollment expected to be completed in mid-2023, with top-line data expected in early 2024 | 10/17/22 | 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 | Parkinson’s disease | Completed enrollment of 44 patients; top-line data expected in December 2022 | 10/19/22 | Neurology/Psychiatric |
| Cerecin Inc., of Singapore | CER-0001 | Medium chain triglyceride | Migraine prevention | Data from the 62-patient Relief study produced an efficacy signal; data to be presented at CNS Summit; plans to run a pivotal phase II/III study | 10/31/22 | Neurology/Psychiatric |
| Cognition Therapeutics Inc., of Pittsburgh | CT-1812 | Binds to a receptor on neurons that regulates cellular damage response pathways | Lewy body dementia | Recruitment ongoing in Shimmer study | 10/12/22 | Neurology/Psychiatric |
| Cyclo Therapeutics Inc., of Gainesville, Fla. | Trappsol Cyclo | Hydroxy-propyl-beta-cyclodextrin | Alzheimer’s disease | Started phase IIb study to treat early disease, targeting reduction of amyloid beta and tau | 10/4/22 | Neurology/Psychiatric |
| Medicane Health Inc., of Kfar Sava, Israel | T3:C3 oral medical cannabis oil | T3:C3 oral medical cannabis oil | Behavioral and psychological symptoms of Dementia | First patient enrolled; planning to enroll 15 participants in part 1 of study for safety and dose-range finding and 40 subjects in part 2 to evaluate safety and efficacy | 10/20/22 | Neurology/Psychiatric |
| Melt Pharmaceuticals Inc., of Bentwood, Tenn. | Melt-300 | Fixed-dose combination of midazolam and ketamine; sublingual, needle- and opioid-free patented formulation | Procedural sedation and analgesia during cataract surgery | Last patient dosed; top-line results expected by the end of 2022 | 10/27/22 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | NBI-1117568 | Muscarinic M4 selective agonist | Schizophrenia | First patient randomized | 10/27/22 | Neurology/Psychiatric |
| PTC Therapeutics Inc, of South Plainfield, N.J. | PTC-518 | Orally bioavailable small-molecule splicing modifier | Huntington's disease | Enrollment in ongoing study is active; data from 12-week portion of trial expected to be shared in first half of 2023 | 10/18/22 | Neurology/Psychiatric |
| Scholar Rock Holding Corp., of Cambridge, Mass. | Apitegromab | Inhibitor of the activation of myostatin | Nonambulatory patients with types 2 and 3 spinal muscular atrophy | In the Topaz study, type 2 patients under 5 years of age had a stabilization or continuous improvements up to a mean change from baseline of 3 points in Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT) scores and up to a mean change from baseline of 5 points in Reported Outcome Measurement Information System (PROMIS) scores over 24 months of treatment; type 2 and 3 combined over 5 had ADL stabilization or increases up to a mean change from baseline of 0.7 points in PEDI-CAT and up to a mean change from baseline of 3.5 points in PROMIS scores | 10/22/22 | Neurology/Psychiatric |
| Seelos Therapeutics Inc., of New York | SLS-005 (trehalose) | Low molecular weight disaccharide | Spinocerebellar ataxia | First of up to 245 patients dosed in the study that will focus on patients with type 3 disease; primary efficacy endpoint is change from baseline in the Modified Scale for Assessment and Rating of Ataxia total score at week 52; Secondary endpoints include change from baseline in a blood-based biomarker for neurodegeneration, clinical global impression of severity, patient global impression of severity and activities of daily living score | 10/25/22 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol transdermal gel | 22q11.2 deletion syndrome | Data from the 20-patient Inspire study showed the drug produced statistically significant improvements from baseline in the Pediatric Anxiety Rating Scale, the total score and all 5 subscales of the Anxiety, Depression and Mood Scale and all 5 subscales of the Aberrant Behavior Checklist – Community | 10/17/22 | Neurology/Psychiatric |
| Aura Biosciences Inc., of Boston | Belzupacap sarotalocan (AU-011) | Virus-like drug conjugate | Early stage choroidal melanoma | Interim data, with an average of 6 months follow-up in patients receiving 3 cycles of therapy in cohorts 5 and 6, showed statistically significant reduction in tumor growth rate (-0.296 mm/yr, p= 0.0007) compared to each patient’s documented growth rate at study entry, and 88.9% (8/9) tumor control rate; visual acuity preservation rate was 88.9% (8/9), with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk; overall safety profile generally favorable, with no dose-limiting toxicities or treatment-related severe adverse events reported as of Aug. 19, 2022 | 10/3/22 | Ocular |
| Belite Bio Inc., of San Diego | Tinlarebant | Reduces and maintains levels of serum retinol binding protein 4 | Adolescent Stargardt disease | 1-year data from ongoing 2-year study showed stabilization of retinal thickness in many subjects; fundus autofluorescence imaging showed no autofluorescence expansion (QDAF) in 7 of 13 (53.8%) subjects, and 12 of 13 (92.3%) subjects showed no atrophic lesion (DDAF) after 1 year; 9 of 13 (69.2%) subjects showed stabilization or improvement in visual acuity throughout treatment period | 10/1/22 | Ocular |
| Lenz Therapeutics Inc., of San Diego | LNZ-100 and LNZ-101 | Formulations of aceclidine; aceclidine + brimonidine | Presbyopia | Study achieved the primary endpoint of 3-line or greater improvement in near visual acuity, without losing one-line or more in distance visual acuity at 1 hour; 71% for LNZ-100 (p<0.0001) 1 10 and 56% for lnz-101 (p<0.0001) at hour post treatment compared to 6% vehicle; maintained statistical significance of three-line or greater improvement vehicle all timepoints including the last measured hours (37% lnz-100 (p<0.0012) 48% (p<0.0002) 4% vehicle.); achieved key secondary endpoint two-line in near visual acuity without losing one-line (5 letters) more distance vision; 86% 78% after treatment, 55% 58% treatment; an average pupil size 1.5-2mm hours; well-tolerated; no serious drug related adverse events< td> | 10/18/22 | Ocular |
| NGM Biopharmaceuticals Inc., of South San Francisco | NGM-621 | Humanized monoclonal IgG1 antibody targeting complement C3 | Geographic atrophy secondary to age-related macular degeneration | Catalina study didn’t meet the primary endpoint of a statistically significant improvement in the rate of change in GA lesion area using slope analysis over 52 weeks for NGM-621 vs. sham; rate of change compared to sham was 6.3% and 6.5% for patients treated every 4 weeks and every 8 weeks, respectively | 10/17/22 | Ocular |
| Aphaia Pharma AG, of Zug, Switzerland | APH-012 | Glucose formulation designed to be released at discrete parts of the small intestine | Obesity | First patient enrolled | 10/27/22 | Other/Miscellaneous |
| Frequency Therapeutics Inc, of Lexington, Mass. | FX-322 | Small-molecule combination product | Acquired sensorineural hearing loss | Completed enrollment; enrolled 142 individuals; readout expected in the first quarter of 2023 | 10/12/22 | Other/Miscellaneous |
| Monopar Therapeutics Inc., of Wilmette, Ill. | Validive | Clonidine HCl mucoadhesive buccal tablet | Prevention of severe oral mucositis | Enrolled more than 130 patients in phase IIb/III Voice study testing drug for prevention of SOM in patients undergoing chemoradiotherapy for oropharyngeal cancer; began enrolling in phase III portion | 10/5/22 | Other/Miscellaneous |
| Sciwind Biosciences Co. Ltd., of San Francisco | XW-003 | Long-lasting GLP-1 peptide ecnoglutide in injectable form | Obesity | Interim data after 18 weeks of treatment showed XW-003 (2.4-mg) achieved a mean body weight reduction from baseline of 11.1% compared to 7.9% in liraglutide (3-mg); ?5% body weight loss from baseline was 88.5% for 2.4 mg XW-003 and 70.4% for liraglutide; ?10% body weight loss from baseline was 57.7% for 2.4 mg XW-003 and 33.3% for liraglutide; safe and well-tolerated | 10/27/22 | Other/Miscellaneous |
| Redx Pharma plc, of Alderley Park, U.K. | RXC-007 | ROCK2 selective inhibitor | Idiopathic pulmonary fibrosis | First patient dosed | 10/11/22 | Respiratory |
| Verona Pharma plc, of London, and Nuance Pharma Co. Ltd., of Shanghai | Ensifentrine | Dual inhibitor of the enzymes phosphodiesterase 3 and 4; CFTR activator | Chronic obstructive pulmonary disease (COPD) | Sub-group analysis showed exacerbation rate reduction in overall population (42%); 43% in background medication; 41% in no background medication; inhaled corticosteroids (44% and 42% in yes and no, respectively); current smoker (56% and 26% in yes and no, respectively); 43% in blood eosinophils >150 cells/µL; 42% in Blood eosinophils ?150 cells/µL; 59% and 31% in Europe and North America patients, respectively |
10/14/22 | Respiratory |
| Opiant Pharmaceuticals Inc., of Santa Monica, Calif. | OPNT-002 (naltrexone hydrochloride, intranasal) | Opioid receptor antagonist | Alcohol use disorder | Last patient enrolled, expected to complete the study in early 2023; top-line data in mid-2023 | 10/6/22 | Toxicity and Intoxication |
| Adaptimmune Therapeutics plc, of Philadelphia and Oxfordshire, U.K. | Afami-cel (formerly ADP-A2M4) | CAR T cell targeting MAGE-A4 | Synovial sarcoma and myxoid/round cell liposarcoma | Results showed durable responses with a median duration of 50 weeks; reversible hematologic toxicities and cytokine release syndrome; acceptable safety profile; overall response rate in synovial sarcoma of 39% | 11/18/22 | Cancer |
| Ammax Bio Inc., of Redwood City, Calif. | AMB-05X | Monoclonal antibody against CSF1R | Tenosynovial giant cell tumor | Study achieved overall response rate targets; tumor shrinkage beyond the dosing period; minimal systemic exposure and significantly fewer adverse events | 11/18/22 | Cancer |
| Apexigen Inc., of San Carlos, Calif. | Sotigalimab | CD40 agonist antibody | Liposarcoma | Interim data showed median progression-free survival was 12.45 months in 10 evaluable patients in combination with doxorubicin; favorable safety profile | 11/14/22 | Cancer |
| Arvinas Inc., of New Haven, Conn. | ARV-471 | Estrogen receptor protein degrader | Locally advanced or metastatic ER+/HER2-negative breast cancer | Initial results showed favorable tolerability profile; clinical benefit rate of 38%; antitumor activity in 100% of CDK4/6 inhibitor-pretreated patients; median progression-free survival (mPFS) of 3.7 months in all evaluable patients; mPFS of 5.7 months in patients with mutant ESR1 tumors | 11/22/22 | Cancer |
| Ascentage Pharma Group Inc., of Suzhou, China | Lisaftoclax (APG-2575) | Bcl-2 inhibitor | Relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma | Data showed interim objective response rate of 98% in combination with acalabrutinib; 87% with rituximab; favorable safety profile; combination therapies maintained low incidence of tumor lysis syndrome and and hematologic adverse events | 11/3/22 | Cancer |
| Bicycle Therapeutics Ltd., of Cambridge, U.K. | BT-8009 | Second-generation BTC targeting nectin-4 | Solid tumors | First patient dosed; RP2Ds of 5mg/m2 and 7.5mg/m2 | 11/8/22 | Cancer |
| Bioatla Inc., of San Diego | Mecbotamab vedotin (BA-3011) | Conditionally and reversibly active ADC targeting the receptor tyrosine kinase AXL | Non-small-cell lung cancer | Results showed 4 partial responses in monotherapy group; 1 complete response in combination therapy; no new safety signals | 11/3/22 | Cancer |
| Blaze Bioscience Inc., of Seattle | Tozuleristide (BLZ-100) | Targeting peptide and a fluorescent dye | Pediatric central nervous system tumors | completed enrollment; trial enrolled 123 subjects | 11/2/22 | Cancer |
| Excellthera Inc., of Montreal | ECT-001 | Cell therapy | High risk leukemia and myelodysplastic syndromes | Completed patient enrollment; enrolled 50 patients | 11/15/22 | Cancer |
| G1 Therapeutics Inc., of Research Triangle Park, N.C. | Cosela (trilaciclib) | CDK4/6 inhibitor | Bladder cancer | Initial data showed reduction in the rates of adverse events >50% related to Trodelvy (sacituzumab govitecan, Gilead Sciences Inc.); efficacy data expected in the second quarter of 2023 | 11/2/22 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif., and Arcus Biosciences Inc., of Hayward, Calif. | Domvanalimab (AB-154) | Fc-silent anti-TIGIT monoclonal antibody | Metastatic, PD-L1-high non-small-cell lung cancer | Interim analysis of ARC-7 study showed clinically meaningful differentiation compared to zimberelimab monotherapy across multiple efficacy measures such as objective response rate and progression-free survival | 11/28/22 | Cancer |
| Immunitybio Inc., of Culver City, Calif. | N-803 (nogapendekin alfa inbakicept) | IL-15 superagonist complex | BCG-unresponsive non-muscle invasive bladder cancer | Data published in NEJM Evidence from the Quilt 3.032 study testing combination with BCG in patients with or without TaT1 papillary disease showed complete responses achieved in patients with papillary disease with a persistence of effect with 90% probably of avoiding cystectomies in responders, and 100% bladder cancer-specific survival at 24 months; in patients with BCG-unresponsive high-grade Ta/T1 papillary NMIBC who received N-803 plus BCG, the Kaplan-Meier-estimated disease-free survival rate (DFS) was 55.4% at 12 months, with median DFS of 19.3 months | 11/10/22 | Cancer |
| ISA Pharmaceuticals BV, of Oegstgeest, the Netherlands | ISA-101b | Immunotherapy | HPV16-positive oropharyngeal cancer | Completed enrollment in OpcemISA trial in patients with metastatic/advanced first- and second-line disease | 11/30/22 | Cancer |
| MEI Pharma Inc., of San Diego, and Kyowa Kirin Co. Inc., of Tokyo | Zandelisib | PI3K? inhibitor | Indolent B-cell non-Hodgkin’s lymphoma | Top-line study showed 75.4% objective response rate; 24.6% of patients achieved a complete response; data are not sufficiently mature for duration of response | 11/17/22 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide crystal nanoparticles | Locally advanced pancreatic adenocarcinoma | Data showed radiotherapy-activated NBTXR-3 was feasible and well-tolerated; 1 patient had partial response 9 months | 11/14/22 | Cancer |
| Nanology LLC, of Fort Worth, Texas | LSAM-PTX | Microparticle paclitaxel | Nonoperable lung cancer | Preliminary data from single-arm, 18-patient study in primary or recurrent nonoperable locally advanced stages II or III with nodal disease or stage IV advanced disease showed drug well-tolerated, with encouraging signs of tumor and immune response; final data expected in third quarter 2023 | 11/11/22 | Cancer |
| Netris Pharma of Lyon, France | NP-137 | Humanized monoclonal antibody of isotype IgG1 directed against netrin-1 | Advanced/metastatic solid tumors | First patient dosed; study designed to enroll up to 90 patients | 11/29/22 | Cancer |
| Nouscom AG., of Basel, Switzerland | NOUS-209 | Off-the-shelf immunotherapy targeting 209 specific neoantigens | dMMR/MSI-high metastatic colorectal cancer | First patient dosed | 11/15/22 | Cancer |
| Nykode Therapeutics ASA, of Norway | VB10.16 | DNA-based therapeutic cancer vaccine delivered via Pharmajet needle-free injection technology | Advanced cervical cancer | Combination with atezolizumab showed ORR of 40% and DCR of 60% by 2 prior systemic treatments; 100% DCR by 4 prior systemic treatments; ORR of 25% and DCR 100% based on 0 extrapelvic metastases; ORR of 27% and DCR of 73% based on 1-5 extrapelvic metastases | 11/7/22 | Cancer |
| Oncosec Medical Inc., of Pennington, N.J., and San Diego | Tavo | IL-12-encoding plasmid delivered via intratumoral electroporation | Melanoma | Data from Keynote-695 study testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with unresectable or metastatic disease who progressed on immediate prior anti-PD-1 therapy showed the key secondary endpoint was met; investigator assessment of overall response rate per RECIST v1.1 from 101 evaluable patients with at least 1 post-baseline tumor assessment showed confirmed ORR of 18.8%, exceeding prespecified clinically meaningful ORR of ?17%; 3 patients achieved a complete response and 16 patients had a partial response | 11/11/22 | Cancer |
| Oncosec Medical Inc., of Pennington, N.J., and San Diego | Tavo-EP | Intratumoral tavokinogene telseplasmid (TAVO) plus electroporation | Operable locoregionally advanced melanoma | Data in combination with intravenous Opdivo (nivolumab, Bristol Myers Squibb Co.) showed 70% as preoperative overall response rate; 4 patients with complete response; 3 patients with partial responses; 2 patients had stable disease and 1 showed progressive disease; no disease recurrence | 11/15/22 | Cancer |
| Onquality Pharmaceuticals LLC, of Seattle | OQL-011 | Ointment designed to locally activate VEGF downstream signaling pathways | Hand-foot skin reaction in cancer patient | First patient enrolled in part 2 of the NOVA-II phase II study | 11/8/22 | Cancer |
| Rain Therapeutics Inc., of Newark, Calif. | Milademetan | Oral mouse double minute 2 (MDM2) inhibitor | Advanced solid tumors | Interim data showed 2 unconfirmed partial responses with tumor regression of 34% and 30% in pancreatic and lung cancer, respectively; tumor regression of 29% and 27% in biliary tract and breast cancer, respectively; consistent safety profile | 11/4/22 | Cancer |
| Redx Pharma plc, of Alderley Park, U.K. | RXC-004 | Porcupine inhibitor | Microsatellite stable metastatic colorectal cancer | Opening enrollment in Porcupine study testing RXC-004 with Opdivo (nivolumab, Bristol Myers Squibb Co.) and the Porcupine 2 study combining the drug with Keytruda (pembrolizumab, Merck & Co. Inc.); data expected in the first half of 2023 | 11/10/22 | Cancer |
| Surface Oncology Inc., of Cambridge, Mass. | SRF-388 | Antibody against IL-27 | Non-small-cell lung cancer | Monotherapy data showed 2 confirmed partial responses; durable disease stabilization in patient with adenocarcinoma; initiated a single-arm phase II study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); expected to enroll 40 patients in the second stage of the trial | 11/2/22 | Cancer |
| TC Biopharm Ltd., of Glasgow, U.K. | Omnimmune | Unmodified allogeneic gamma delta T-cell product | Acute myeloid leukemia | Initiated dosing in phase IIb study | 11/18/22 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX-101 | (4-L-[ 131I] iodo-phenylalanine in combination with external beam radiation therapy | Recurrent glioblastoma multiforme | First patient dosed in combination with external beam radiation therapy | 11/21/22 | Cancer |
| Transgene SA, of Strasbourg, France | TG-4001 | Therapeutic cancer vaccine | HPV+ anogenital cancers | Independent data monitoring committee recommended the study continue; expected to enroll an additional 66 patients | 11/2/22 | Cancer |
| Abbvie Inc., of North Chicago | AGN-151607 | Botulinum toxin type A | Prevention of postoperative atrial fibrillation in cardiac surgery patients | Primary endpoint was not met for the modified intent-to-treat (mITT) population; relative risk reduction in specific study populations (p=0.15) and overall lower rates of rehospitalization within 30 days compared to placebo 8.7% vs. 15.7%, respectively; greater risk reduction at 51% compared to placebo (nominal p<0.01); 62.9% of patients were atrial fibrillation-free and anticoagulation-free vs. placebo (45.1%) (nominal p<0.05)< td> | 11/7/22 | Cardiovascular |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | Olpasiran | TRiM-enabled investigationa agent | Atherosclerotic cardiovascular disease | Data showed substantial reductions of Lp(a) levels in all olpasiran arms at week 36; 70.5%, 97.4%, and 101.1% at 10-mg, 75-mg and225-mg | 11/7/22 | Cardiovascular |
| Cincor Pharma Inc., of Waltham, Mass. | Baxdrostat | Aldosterone synthase inhibitor | Treatment-resistant hypertension | Brightn Study met its primary endpoint; dose-dependent reduction in both plasma and urine aldosterone; dose-dependent changes in renin activity; no meaningful impact on cortisol; statistically significant change from baseline in mean seated systolic blood pressure vs. placebo; significantly lowering diastolic blood pressure by 5.2 mmHg in the 2-mg; no drug related serious adverse events; well-tolerated profile | 11/7/22 | Cardiovascular |
| Cincor Pharma Inc., of Waltham, Mass. | Baxdrostat | Aldosterone synthase inhibitor | Uncontrolled hypertension | Top-line data of Halo study did not achieve statistical significance on its primary endpoint evaluating change from baseline in mean seated systolic blood pressure (SBP) in intent-to=treat population; placebo-adjusted reduction in SBP of 12.6 mmHg (nominal p-value = 0.001) at the 2-mg dose; no drug-related adverse events; no patients discontinued the study due to treatment-related adverse events; favorable safety and tolerability profile | 11/28/22 | Cardiovascular |
| Mineralys Therapeutics Inc., of Philadelphia | MLS-101 | Aldosterone synthase inhibitor | Uncontrolled and resistant hypertension | Top-line study met its primary and secondary endpoints; statistically significant and clinically meaningful reduction in systolic blood pressure SBP (p=0.010 and p=0.037 at 50 mg and 100 mg, respectively); robust placebo-adjusted reductions in SBP (p=0.010 and p=0.028) and diastolic blood pressure; no notable effects on serum cortisol; serious events in 3 subjects | 11/16/22 | Cardiovascular |
| Que Oncology Inc., of Melbourne, Australia | Q-122 | Non-hormonal therapy | Vasomotor symptoms | Data published in The Lancet from the study in 131 women taking endocrine therapy following breast cancer showed drug significantly reduced frequency and severity of moderate and severe vasomotor symptoms; mean percentage change in msVMS-SS from baseline over 28 days of treatment vs. placebo (least squares mean: Q-122, –39% [95% CI –46 to –31] vs. placebo –26% [–33 to –18]; p=0.018); well-tolerated with no serious adverse effects | 11/10/22 | Cardiovascular |
| Alphyn Biologics Inc., of Annapolis, Md. | AB-101a | Topical therapeutic candidate | Mild-to-moderate atopic dermatitis | Completed first cohort; top-line results expected in 8 weeks from Nov. 2022 | 11/15/22 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast cream 0.3% | Topical inhibitor of phosphodiesterase-4 | Plaque psoriasis | Data published in the American Journal of Clinical Dermatology showed significantly greater improvements in itch compared to those treated with vehicle by week 2 as assessed by WI-NRS (p?0.002) and PSD (p<0.012 2 and p<0.010 at weeks 12, respectively); improvements in itch-related sleep loss as measured by nrs (p?0.022); improvement dlqi (p="0.045) | 11/28/22 | Dermatologic |
| Ascletis Inc., of Hangzhou and Shaoxing, China | ASC-40 (denifanstat) | Oral, selective small-molecule inhibitor of fatty acid synthase | Moderate to severe acne | Completed 180-patient enrollment | 11/30/22 | Dermatologic |
| Dermavant Sciences Ltd., of Long Beach, Calif. | Vtama (tapinarof) | Nonsteroidal topical cream/aryl hydrocarbon receptor agonist | Atopic dermatitis | Top-line data showed favorable safety, PK and clinical improvement; minimal-to-no systemic exposure despite maximal use as 1% cream | 11/8/22 | Dermatologic |
| Equillium Inc., of La Jolla, Calif. | EQ-101 | IL-2, IL-9 and IL-15 inhibitor | Severe alopecia areata | Started an open-label study to evaluate the efficacy, safety, tolerability and pharmacokinetic/pharmacodynamic properties of EQ-101 over 24 weeks | 11/10/22 | Dermatologic |
| Escient Pharmaceuticals Inc., of San Diego | EP-547 | MRGPRX4 antagonist | Cholestatic pruritus | Initiated phase II study to enroll 58 patients; primary efficacy endpoint is the change from baseline to week 6 in itch intensity | 11/17/22 | Dermatologic |
| Intellia Therapeutics Inc., of Cambridge, Mass. | NTLA-2002 | CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene | Hereditary angioedema | Interim data showed dose-dependent reductions in plasma kallikrein; 64%, 81% and 92% at 25-mg, 50-mg and 75-mg doses, respectively; mean HAE attack rate reduction from week 1 to 16 (91% and 78%) and from week 5 to 16 (89% at both doses) at 25 mg and 75 mg; attack-free interval (5.5 months – 10.6 months) and (2.3 months – 4.2 months) at 25 mg and 75 mg; well-tolerated | 11/12/22 | Dermatologic |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Donidalorsen | Antisense medicine designed to reduce the production of prekallikrein | Hereditary angioedema | Data showed mean reduction in attack rate of 75.6% from baseline at 80 mg; mean monthly attack rate was 0.28; 5 patients remained attack-free over the 1-year duration; 3 patients returned to 80 mg every 4 weeks; no serious events; no treatment-emergent adverse events; no clinically relevant abnormalities in any laboratory measurement | 11/13/22 | Dermatologic |
| Nimbus Therapeutics Inc., of Boston | NDI-034858 | Allosteric TYK2 inhibitor | Moderate to severe plaque psoriasis | Top-line data showed phase IIb study achieved primary endpoint, with statistically significant greater proportion of patients reaching PASI-75 vs. placebo at 12 weeks; additional endpoints achieved; safety consistent with other leading allosteric TYK2 inhibitors | 11/30/22 | Dermatologic |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-APOC3 | RNAi therapeutic targeting apolipoprotein C-III (APOC3) | Severe hypertriglyceridemia | Shasta-2 study showed ARO-APOC3 durably decreased APOC3 up to 87%; triglycerides to 86%; non-HDL-C up to 45%; increased HDL-C up to 99% through the week 16 in patients with severe hypertriglyceridemia; well-tolerated | 11/7/22 | Endocrine/Metabolic |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-ANG3 | RNAi therapeutic designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3) | Mixed dyslipidemia | Arches-2 study showed ARO-ANG3 decreased ANGPTL3 up to 71% at week 8; triglycerides to 59%; LDL-C up to 32% at week 16; relative reduction in liver fat fraction at week 24; well-tolerated; no adverse events in in patients with mixed dyslipidemia | 11/7/22 | Endocrine/Metabolic |
| Lumos Pharma Inc., of Austin, Texas | LUM-201 | Oral growth hormone-stimulating small molecule | Pediatric growth hormone deficiency | Interim results met phase II study expectations; 50% enrollment showed mean annualized height velocity (AHV) of 8.6 cm at 6 months for 1.6 mg/kg/day; favorable safety profile; no treatment-related serious events; oragrowth210 trial enrolled 80%; primary outcome readout expected in the second half of 2023 | 11/14/22 | Endocrine/Metabolic |
| Lysogene SA, of Paris | LYS-SAF302 | Gene therapy | Mucopolysaccharidosis type IIIA | Top-line results from phase II/III AAVance study in subjects enrolled at <30 24 30 months of age showed statistically significant improvement in cognitive development vs. natural history at post-dosing; subjects also achieved key secondary efficacy criteria; trial did not meet primary endpoint for main cohort (12 enrolled ? age) demonstrating the developmental quotient post-dosing as assessed by bsid-iii, compared to nh cohort< td> | 11/29/22 | Endocrine/Metabolic |
| Novartis AG, of Basel, Switzerland, and Alnylam Pharmaceutical Inc., of Cambridge, Mass. | Leqvio (inclisiran) | siRNA therapy | Elevated low-density lipoprotein cholesterol | Open-label Orion trial showed effective and sustained reductions in low-density lipoprotein cholesterol (LDL-C) over four years of treatment ; 47.5% reduction in LDL-C from baseline (day 1 of ORION-1) to day 210; time-averaged reduction in LDL-C of 44.2% over the 4 years through twice-yearly dosing; 80% of patients reached an LDL-C level of <70mg dl2; safety-benefit profile consistent with findings in previous 18-month phase iii study< td> | 11/7/22 | Endocrine/Metabolic |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Volixibat | Oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter | Intrahepatic cholestasis of pregnancy | Discontinued Ohana study due to enrollment feasibility issues | 11/29/22 | Gastrointestinal |
| Morphic Therapeutic Inc., of Waltham, Mass. | MORF-057 | Integrin alpha4 beta7 inhibitor | Ulcerative colitis | First patient dosed in Emerald-2 study | 11/22/22 | Gastrointestinal |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin candidate | Type 2 diabetes patients with nonalcoholic steatohepatitis | Study achieved primary endpoint of safety and tolerability; no serious adverse events; reduced liver fat content, fibrosis and steatosis; decreased lipid levels | 11/17/22 | Gastrointestinal |
| Poxel SA, of Lyon, France | PXL-065 | Deuterium-stabilized R-pioglitazone | Nonalcoholic steatohepatitis | Study achieved primary endpoint; statistically significant (p=0.024 to p=0.008) mean relative decrease vs. placebo of 21% to 25% in liver fat content from baseline to 36 weeks; achieved a > 30% relative reduction in liver fat content in 40% of patients at 22.5-mg; decrease in ALT by 38% to 54% vs. placebo (26%); statistically significant dose-dependent decreases in PIIINP fibrogenesis biomarker, p=0.02 at 22.5 mg; fibrosis score p=0.04; dose-dependent decrease in HbA1c (up to 0.41 % placebo-adjusted; p=0.003 at 22.5 mg); plasma adiponectin level increase (p<0.0001 vs. placebo at 22.5 mg); improvements in insulin levels and sensitivity indices (homa-ir, adipo-ir, quicki); no dose-dependent increase body weight; safe well tolerated < td> | 11/7/22 | Gastrointestinal |
| Revolo Biotherapeutics Inc., of New Orleans | IRL-201104 ('1104) | Peptide derived from mTB Chaperonin 60.1 | Eosinophilic esophagitis | Last patient completed; top-line data expected in early 2023 | 11/9/22 | Gastrointestinal |
| Sagimet Biosciences Inc., of San Mateo, Calif. | Denifanstat (TVB-2640) | FASN inhibitor | Nonalcoholic steatohepatitis | Interim data showed statistically significant improvements across key markers; statistically significant reductions in markers of liver fat (p<0.002), inflammation and fibrosis; ?30% reduction of liver fat (p<0.002); alt, enhanced fibrosis ldl cholesterol (p<0.05 for all); no treatment-related serious adverse events< td> | 11/3/22 | Gastrointestinal |
| Chinook Therapeutics Inc., of Seattle | Atrasentan | Endothelin A receptor inhibitor | Primary IgA nephropathy | Data showed no treatment-related adverse events; well-tolerated; no meaningful changes in blood pressure or acute eGFR effects; no increases in BNP or mean bodyweight; mean reductions in 24-hour urine protein creatinine ratio (UPCR) of 38.1% at 6 weeks of treatment, 48.3% at 12 weeks of treatment and 54.7% at 24 weeks of treatment | 11/3/22 | Genitourinary/Sexual Function |
| Inversago Pharma Inc., of Montreal | INV-202 | Peripherally-acting CB1 inverse agonist | Diabetic kidney disease | First patient dosed | 11/29/22 | Genitourinary/Sexual Function |
| Edigene Inc., of Beijing | ET-01 | Autologous CD34-positive hematopoietic stem cells ex vivo modified with a CRISPR/Cas9 gene editing system targeting BCL11A erythroid enhancer | Transfusion-dependent beta-thalassemia | Completed last patient dosing | 11/18/22 | Hematologic |
| Kira Pharmaceuticals Inc., of Cambridge, Mass. | KP-104 | Bifunctional biologic | Paroxysmal nocturnal hemoglobinuria | First cohort dosed in trial in China | 11/30/22 | Hematologic |
| Acelyrin Inc., of Los Angeles, Affibody AB, of Solna, Sweden, and Inmagene Biopharmaceuticals Co. Ltd., of Shanghai | Izokibep | IL-17A-inhibiting antibody mimetic | Psoriatic arthritis | Data showed statistically significant and clinically important improvements across multiple measures of disease activity including DAS28-CRP, DAPSA and MDA; improvements in efficacy and reduced disease activity drove improvements across health-related quality of life and physical function; | 11/8/22 | Immune |
| Biosenic SA., of Mont-Saint-Guibert, Belgium | Arsenic trioxide (ATO) | Immunomodulators | Chronic graft-vs.-host disease | Completed phase IIb study; demonstrated efficacy of more than 75% | 11/8/22 | Immune |
| Clene Inc., of Salt Lake City | CNM-Au8 | Gold nanocrystal suspension | Multiple sclerosis | Data showed significant improvement in low contrast vision improvement and global neurological improvement; improvement in primary (p=0.056) and secondary endpoints (p=0.0207); well-tolerated; no significant safety findings | 11/28/22 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes | Active and chronic active antibody mediated rejection episodes | Top-line results showed significantly superior efficacy compared to plasma exchange in reducing donor specific antibodies during the 5 days following the start of treatment; full results in 2023 | 11/28/22 | Immune |
| Immunic Inc., of New York | Vidofludimus calcium (IMU-838) | DHODH inhibitors | Relapsing-remitting multiple sclerosis | Long-term study showed proportion of patients free from 12wCDW was 97.6% after 48 weeks and 94.5% after 96 weeks compared to the start of trial; low relapse activity | 11/17/22 | Immune |
| Kezar Life Sciences Inc., of South San Francisco | Zetomipzomib | Selective immunoproteasome inhibitor | Active lupus nephritis | Data showed overall renal response (ORR) as 50% or greater reduction in urine protein-to-creatinine ratio at the end of treatment; 58.8% achieved an ORR (10/17 patients) at week 13; 5 of 17 patients (29.4%) achieved complete renal response rate as early as week 13; reduction in proteinuria; well-tolerated; favorable safety profile | 11/14/22 | Immune |
| Ose Immunotherapeutics SA, of Nantes, France, and Servier Pharmaceuticals, of Boston, part of Servier Group | OSE-127 (S-95011) | Humanized monoclonal antibody targeting interleukin-7 receptor | Primary Sjögren’s syndrome | Completed patient enrollment; results expected in 2023 | 11/3/22 | Immune |
| Akston Biosciences Inc., of Beverly, Mass. | AKS-452 | Second-generation vaccine based on Akston’s Fc-fusion platform | COVID-19 | Top-line results showed 93% of those previously vaccinated had higher neutralizing antibody titers after a single dose; increased 4-fold against the Wuhan strain and 5-fold against omicron variants at day 28; no safety issues reported; well-tolerated; no adverse effects and no serious adverse events | 11/1/22 | Infection |
| Biophytis SA, of Paris | Sarconeos (BIO-101) | Proto-oncogene Mas agonist | COVID-19-related respiratory failure | Post-hoc analysis showed reduction in the risk of early death or respiratory failure at day 28 of 45% (p=0.037) in intent-to-treat (ITT) population and 53% (p=0.051) in per protocol (PP) population; reduction in the risk of death at day 90 of 43% (p=0.076) in the ITT population and 70% (p=0.016) in the PP population; good safety profile | 11/3/22 | Infection |
| Bioxytran Inc., of Boston | Prolectin-M | Galectin antagonist | COVID-19 | Top-line data from 34 patients as chewable tablet showed statistically significant reduction in viral load measured by the number of patients reaching a below threshold PCR value (Ct value ? 29) by day 7; achieved endpoint with a 100% response rate by day 7 vs. 6% in placebo (p=0.001); 88% response rate by day 3 (p=0.001); no drug-related serious adverse events | 11/16/22 | Infection |
| Clover Biopharmaceuticals Ltd., of Shanghai | SCB-2019 (CpG 1018/Alum) | Vaccine | COVID-19 | Data published in Clinical Infectious Diseases from a phase II/III trial showed vaccine reduced risk of transmitting SARS-CoV-2 infection to household members, compared to placebo participants; household contact was 84% less likely to infection when the infected household member received SCB-2019 vs. households where infected household member was not vaccinated; among 134 household contacts of infected household members who had received SCB-2019, there was 1 case of COVID-19; among 250 household contacts of infected household members who were not vaccinated, there were 12 cases | 11/30/22 | Infection |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | CHIKV VLP | Chikungunya virus virus-like particle vaccine | Chikungunya disease | Study showed well-tolerated; immunogenic in both alphavirus vaccine-naïve participants and participants previously vaccinated against the Venezuelan equine encephalitis virus | 11/1/22 | Infection |
| Enanta Pharmaceuticals Inc., of Watertown, Mass. | EDP-235 | Coronavirus 3CL protease inhibitor | COVID-19 | Initiated SPRINT phase II study; to evaluate the safety, tolerability and antiviral activity of 200-mg and 400-mg once-daily doses | 11/9/22 | Infection |
| Geneuro SA, of Geneva | Temelimab | Monoclonal antibody targeting pathogenic retroviral envelope protein pHERV-W Env | Long COVID | First patient enrolled | 11/16/22 | Infection |
| GSK plc, of London | GSK-3036656 | Leucyl tRNA synthetase inhibitor | Drug-sensitive pulmonary tuberculosis | Data showed drug well-tolerated with no serious adverse events; reduction in TB disease over 14 days in lungs | 11/14/22 | Infection |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | IONIS-HBVRx (bepirovirsen) | Antisense medicine | Chronic hepatitis B | Study resulted in sustained clearance of hepatitis B surface antigen and hepatitis B virus DNA for 24 weeks as monotherapy and or in combination with nucleoside/nucleotide analogue | 11/8/22 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273.222 (Spikevax bivalent original/omicron BA.4-5) | Omicron-targeting bivalent COVID-19 booster | COVID-19 | Study achieved primary endpoint; induced significantly higher neutralizing antibody titers against BA.4/BA.5 compared to a booster dose of mRNA-1273; superior neutralizing antibody response against omicron BA.1; similar safety and tolerability profile; results published in The New England Journal of Medicine | 11/14/22 | Infection |
| Osteal Therapeutics Inc., of Dallas | VT-X7 | Drug-device combo comprised of spacer plus vancomycin hydrochloride and tobramycin sulfate | Periprosthetic joint infection | Completed enrollment of 76 subjects | 11/4/22 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine | mRNA vaccine | COVID-19 | Updated data from one-month after a 30-µg booster dose showed 4-fold higher neutralizing antibody titers against omicron BA.4/BA.5 sublineages compared to the original in individuals older than 55 years of age; increased 13.2-fold from pre-booster levels in adults older than 55 years of age and 9.5-fold in adults 18 to 55 years of age, compared to a 2.9-fold increase in adults older than 55 years or age who received the original booster vaccine | 11/4/22 | Infection |
| Providence Therapeutics Inc., of Calgary, Alberta | PTX-COVID19-B | mRNA vaccine designed to encode the S protein of SARS-CoV-2 encapsulated in a lipid nanoparticle | COVID-19 | Data showed well-tolerated; no safety signals; noninferior to comparator vaccine Comirnaty (Pfizer Inc./Biontech SE) | 11/28/22 | Infection |
| Resolve Therapeutics LLC, of St. Petersburg, Fla. | RSLV-132 | Human RNase moiety fused to a human IgG1 Fc domain | Long COVID | Completed enrollment in the 113-patient study; top-line data expected in the first quarter of 2023 | 11/9/22 | Infection |
| Sanofi Sa, of Paris | Acoziborole | Boron-based antiparasitic compound | Sleeping sickness | Success rates of up to 95% in phase II/III study, published in The Lancet Infectious Diseases; 18-month success rate was 95% in late-stage patients with gambiense strain of human African trypanosomiasis (g-HAT); 100% of 41 patients with early stage g-HAT were considered treatment successes at all timepoints; favorable safety profile, with no significant drug-related safety signals reported | 11/30/22 | Infection |
| Atom Bioscience and Pharmaceutical Co. Ltd., of Jiangsu, China | ABP-671 | URAT1 inhibitor | Chronic gout | After 4 weeks of treatment, mean serum uric acid levels were 3.1-5.3 mg/dL for the 6 ABP-671 dose cohorts compared to 9.1 mg/dL for the combined placebo cohorts | 11/10/22 | Inflammatory |
| Kinevant Sciences Holdings Ltd., of New York | Namilumab | Human monoclonal antibody that targets granulocyte-macrophage colony stimulating factor | Pulmonary sarcoidosis | First patient dosed | 11/17/22 | Inflammatory |
| 180 Life Sciences Corp., of Palo Alto, Calif. | Adalimumab | Anti-TNF | Dupuytren’s disease | Results showed adalimumab injections significantly reduced hardness and size of early stage nodules | 11/15/22 | Musculoskeletal |
| Athira Pharma Inc., of Bothell, Wash. | Fosgonimeton (ATH-1017) | Small molecule designed to enhance activity of HGF and MET | Mild to moderate Alzheimer’s disease | Biomarker data from exploratory ACT-AD study showed baseline NfL (neurodegeneration) levels may predict functional decline in patients, as assessed by the change from baseline measures of ADCS-ADL23; treatment significantly reduced levels of NfL (p=0.0241) and numerically reduced levels of GFAP and YKL-40 (neuroinflammation); showed directional improvements in biomarkers of AD-associated protein pathologies (A? 42/40 ratio and p-Tau181) compared to placebo; treatment-related reductions in NfL and GFAP significantly correlated with improvements in clinical outcomes, as assessed by the Global Statistical Test | 11/30/22 | Neurology/Psychiatric |
| Biomind Labs Inc., of Toronto | BMND-01 | Liquid inhaled version of N, N-dimethyltryptamine (DMT) | Treatment-resistant depression | Data showed no serious adverse events; mild, transient and self-limited increase in blood pressure; heart rate of up to 30% compared to baseline values with no clinical repercussions | 11/4/22 | Neurology/Psychiatric |
| Compass Pathways plc, of London | COMP-360 | Psilocybin therapy | Treatment-resistant depression | Results published in The New England Journal of Medicine showed 30% of patients were in remission at week 3 (p<0.002); 12 sustained response seen through week at 25-mg dose; 20.3% of patients in the group vs. 10.1% 1-mg group; well-tolerated; suicidal ideation and intentional self-injury as treatment-related adverse events< td> | 11/3/22 | Neurology/Psychiatric |
| Embarkneuro Inc., of Oakland, Calif. | ANC-501 | Vasopressin 1b (V1b) receptor antagonist | Major depressive disorder | First patient dosed | 11/19/22 | Neurology/Psychiatric |
| Khondrion BV, of Nijmegen, the Netherlands | Sonlicromanol | Redox modulator with anti-inflammatory properties | MELAS spectrum disorders | Placebo-controlled phase IIb study and ongoing phase IIb open-label extension study showed statistically significant and clinically meaningful results in multiple outcome measures with acceptable safety profile; 28-day phase IIb study did not meet primary endpoint of the attention domain score of cognitive functioning; statistically significant and clinically meaningful treatment effects vs. placebo on Beck Depression Inventory (p=0.01) and Cognitive Failure Questionnaire (p=0.007) | 11/22/22 | Neurology/Psychiatric |
| Longeveron Inc., of Miami | Lomevel-B | Allogeneic medicinal signaling cell therapy | Alzheimer’s disease | Completed enrollment in the 48-patient Clear Mind phase IIa study | 11/10/22 | Neurology/Psychiatric |
| Lysogene SA, of Paris | LYS-SAF302 | Gene therapy | Mucopolysaccharidosis type IIIA | Top-line results of the study did not meet its primary efficacy and secondary endpoint for the main cohort; statistically significant improvement in the cognitive developmental quotient at 24 months post-dosing; improvement or stabilization of cognitive, language and motor developmental age; no statistically significant decrease in mean cortical gray matter volume from baseline to 24 months post-treatment | 11/18/22 | Neurology/Psychiatric |
| NLS Pharma Ltd., of Stans, Switzerland | Quilience | Mazindol extended release | Narcolepsy | Open-label extension (OLE) study improved wakefulness; improved 1.8 points in the Epworth sleepiness scale; mean weekly cataplexy episodes for these patients declined to 2.1; remained relatively stable in the 2 to 4 range through week 12; similar safety and tolerability profile between the randomized trial and OLE | 11/7/22 | Neurology/Psychiatric |
| Praxis Precision Medicines Inc., of Boston | PRAX-562 | Inhibitor of persistent sodium current | Developmental and epileptic encephalopathies | Initiated phase II Embold study in pediatric patients; top-line results expected in the second half of 2023 | 11/28/22 | Neurology/Psychiatric |
| Scisparc Ltd., of Tel-Aviv, Israel | SCI-110 | Combination of dronabinol and cannamide | Alzheimer’s disease and agitation | Interim results from 8 patients showed study met its primary endpoints of safety; no delirium, oversedation, hypotension or falls at high dose; increased appetite in 6/8 patients; no changes in cognitive measurements and sleep quality; well-tolerated | 11/7/22 | Neurology/Psychiatric |
| Adverum Biotechnologies Inc., of Redwood City, Calif. | ADVM-022 (ixo-vec/ixoberogene soroparvovec) | AAV.7m8, carrying an aflibercept coding sequence | Wet age-related macular degeneration | Long-term follow-up data from the Optic study found drug to be well-tolerated; participants were inflammation-free and did not require steroids to treat inflammation at the end of the study; 81%-98% reduction in annualized anti-VEGF injections; BCVA and CST were maintained to improved through at least 2 years at both the 6E11 and 2E11 dose levels | 11/4/22 | Ocular |
| Allysta Pharmaceuticals Inc., of Bellevue, Wash. | ALY-688 | Sterile and preservative-free eye drop formulation containing ALY-688 peptide | Dry eye disease | Completed enrollment; randomized 922 subjects; top-line data expected in the second quarter of 2023 | 11/8/22 | Ocular |
| Aramis Biosciences Inc., of Boston | A-197 | Topical immunomodulatory agent | Dry eye disease | Completed enrollment; top-line data expected in the first quarter of 2023 | 11/3/22 | Ocular |
| Azura Ophthalmics Ltd., of Tel Aviv, Israel, and Melbourne, Australia | AZR-MD-001 | Selenium sulfide | Meibomian gland dysfunction | Study met its co-primary and secondary endpoints; 0.5% achieved statistically significant differences compared to vehicle in both signs and symptoms at month 3; significant improvements in MGYLS score, with patients experiencing an average increase of 1.8 more open glands secreting meibum from baseline (p = 0.0004); improvements in OSDI score, with patients reporting an average improvement of 3.5 from baseline (p = 0.0438); 46.9% of patients became asymptomatic as measured by Total OSDI; 45.7% of patients had at least 5 more glands opened from baseline of 1.7, as measured by MGYLS responder rate; 68.7% of patients had their meibum quality return to normal levels, as measured by MGS responder rate; significant improvements in SPEED (p<0.0001) and average vas (visual analogue scale) (p<0.0001); significant improvements in eye discomfort (p<0.0001), dryness (p<0.0001) ocular itch mild transient adverse events; no serious treatment-related events< td> | 11/17/22 | Ocular |
| Lineage Cell Therapeutics Inc., of Carlsbad, Calif. | RG-6501 (Opregen) | Retinal pigment epithelial cell therapy | Geographic atrophy secondary to age-related macular degeneration | Launched phase IIa study; to optimize subretinal surgical delivery and evaluate the safety and activity | 11/28/22 | Ocular |
| NGM Biopharmaceuticals Inc., of South San Francisco | NGM-621 | Humanized monoclonal IgG1 antibody targeting complement C3 | Geographic atrophy (GA) secondary to age-related macular degeneration | Post-hoc analysis of NGM-621 showed a reduction in the rate of change in GA lesion area (slope) of 21.9% (Q4W) and 16.8% (Q8W) compared to sham; reduction in change from baseline in GA at 52 weeks was 20.6% (Q4W) and 16.6.% (Q8W) in MMRM analysis with the adjusted treatment arm | 11/3/22 | Ocular |
| Oculis SA, of Lausanne, Switzerland | OCS-02 (licaminlimab) | Anti-TNF-alpha antibody fragment | Acute anterior uveitis | Results published in the Translational Vision Science &Technology ARVO Journal showed study met its primary objective according to prospectively specified criteria; response rate on day 15 of 56% as a reduction from baseline in AC cell grade of ?2; well-tolerated; no increase in intra-ocular pressure related to licaminlimab | 11/22/22 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | Suprachoroidal delivery; consisting of NAV AAV8 vector encoding antibody fragment designed to inhibit VEGF | Diabetic retinopathy, wet age-related macular degeneration and other chronic retinal conditions | Data showed well-tolerated; no drug-related serious adverse events; clinically meaningful improvements in disease severity and less disease worsening vs. observation control at 6 months; 20% of patients achieved ?2-step DRSS improvement vs. 10% in control; 54% of patients achieved any DRSS improvement vs. 20% in control; 0% of patients worsened ?2 steps vs. 20% in control; no meaningful differences in safety outcomes at 6 months for patients who are NAb-positive | 11/3/22 | Ocular |
| Achieve Life Sciences Inc., of Seattle | Cytisinicline | Nicotinic acetylcholine receptor partial agonist | Nicotine dependence | Target enrollment reached in the ORCA-V1 study; results expected in the first half of 2023 | 11/8/22 | Other/Miscellaneous |
| Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Prader-Willi syndrome | Initial top-line results showed 91% completed treatment; 64 of 65 patients opted to continue to the open-label extension; mean baseline ESS-CHAD ranged from 14.7 to 15.7; clinically meaningful change in mean change from baseline to EOT on the ESS-CHAD; response rates of 70% in the high dose pitolisant group; 55.6% in the low dose pitolisant group, and 52.6% in the placebo group in the responder analysis; 57% of patients on pitolisant showed adverse events vs. 65% on placebo | 11/1/22 | Other/Miscellaneous |
| Innovent Biologics Inc., of San Francisco and Suzhou, China | Mazdutide (IBI-362) | GLP-1R and GCGR dual agonist | Obesity | First participant dosed | 11/14/22 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Melanocortin-4 receptor agonist | Hypothalamic obesity | Data showed 89% of patients evaluable for assessment had ?5% reduction in BMI (p<0.0001); 16 29 41 78% patients had a 10% or greater reduction in bmi at weeks; 14.5 mean percent bmi; 12.6 body weight; of 1.3 points from baseline for z score; hunger score was 6.6 compared with 3.7 week 16; long-term data showed setmelanotide therapy achieved 21.1% 26.7% weeks< td> | 11/2/22 | Other/Miscellaneous |
| Aridis Pharmaceuticals Inc., of Los Gatos, Calif. | AR-501 | Inhalable broad-spectrum anti-infective | Cystic fibrosis | Closed patient enrollment in multiple ascending-dose and dose-ranging cohorts in phase IIa trial; top-line data due first quarter 2023 | 11/30/22 | Respiratory |
| Bridge Biotherapeutics Inc., of Seongnam, South Korea | BBT-877 | Autotaxin inhibitor | Idiopathic pulmonary fibrosis | Initiated phase II study; planning to enroll 120 patients | 11/7/22 | Respiratory |
| Epiendo Pharmaceuticals ehf, of Reykjavik, Iceland | EP-395 | Oral, non-antibiotic macrolide | Chronic obstructive pulmonary disease | Planning to initiate phase II study to enroll 60 adult patients; results expected in the second half of 2023 | 11/14/22 | Respiratory |
| Laurent Pharmaceuticals Inc., of Montreal | Fenretinide (LAU-7b) | Oral, once-daily formulation of fenretinide, a synthetic retinoid derivative and a lipid modulator | Cystic fibrosis | Study achieved a statistically significant treatment difference in LAU-7b arm in the absolute change in the percent predicted forced expiratory volume in one second (ppFEV1) through 24 weeks (p=0.0486); clinically meaningful reduction of 49% in the loss of lung function at 24 weeks relative to placebo; statistically significant clinical benefit was also observed in the relative change in ppFEV1 through 24 weeks (p=0.0351); plasma levels of C-reactive protein (p=0.029); higher benefit in subjects with ppFEV1 greater than 70% at baseline in pre-specified subgroup; predictable and acceptable safety profile; no unexpected serious adverse events reported | 11/3/22 | Respiratory |
| Renovion Inc., of Chapel Hill, N.C. | ARINA-1 | Nebulized therapy | Non-CF bronchiectasis | First patient enrolled | 11/2/22 | Respiratory |
| Vicore Pharma Holding AB, of Gothenburg, Sweden | C-21 | Angiotensin II type 2 receptor agonist | Idiopathic pulmonary fibrosis | Data showed increase in forced vital capacity (FVC) of 28 mL from baseline after 12 weeks of treatment; increase in FVC was 213 mL after 24 weeks; at week 36 the increase was 633 mL; linear regression analysis showed statistical significance vs. the expected mean for untreated patients (p<0.05 21 28 for weeks and 32; p<0.01 36)< td> | 11/3/22 | Respiratory |
| Clairvoyant Therapeutics Inc. and Origin Therapeutics Holdings Inc., both of Vancouver, British Canada | Psilocybin | Psychedelics | Alcohol use disorder | First patient dosed | 11/3/22 | Toxicity and Intoxication |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-026 | HER2-targeted bispecific antibody | First-line, HER2-positive recurrent/metastatic breast cancer | Of the 55 evaluable patients treated with KN-026 plus docetaxel, confirmed objective response rate was 76.4%, median progression-free survival was 25.4 months and median overall survival (OS) wasn’t met yet; 2-, 18- and 24-month OS rates were 93%, 91.2% and 91.2%, respectively | 12/12/22 | Cancer |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-026 | HER2-targeted bispecific antibody | HER2-positive early or locally advanced breast cancer | Treatment of 20 evaluable patients with KN-026 plus docetaxel as a neoadjuvant after surgery resulted in a total pathological complete response rate of 50%, a breast pathological complete response rate of 55% and an objective response rate of 100% | 12/12/22 | Cancer |
| Amphera BV, of 's-Hertogenbosch, the Netherlands | Mesopher | Autologous dendritic cells loaded with Pheralys, a lysate of tumor cell lines | Mesothelioma with stable disease or better after platin-pemetrexed chemotherapy | In the phase II/III Denim study, treatment with Mesopher didn’t improve overall survival compared to best supportive care | 12/12/22 | Cancer |
| Amphera BV, of 's-Hertogenbosch, the Netherlands | Mesopher | Autologous dendritic cells loaded with Pheralys, a lysate of tumor cell lines | Resected pancreatic cancer | In the Reactive study of 38 patients who were treated after completion of standard-of-care chemotherapy, 2-year recurrence free survival was 60% | 12/12/22 | Cancer |
| Arcus Biosciences Inc., of Hayward, Calif., and Gilead Sciences Inc., of Foster City, Calif. | Domvanalimab + zimberelimab + etrumadenant | Monoclonal antibody targeting TIGIT + targeting PD1 + adenosine A2A and A2B receptor antagonist | First-line, metastatic non-small-cell lung cancer with PD-L1 tumor proportion score ?50% without epidermal growth factor receptor or anaplastic lymphoma kinase mutations | Zimberelimab monotherapy, domvanalimab + zimberelimab and domvanalimab + etrumadenant + zimberelimab produced a median progression-free survival of 5.4, 12.0 and 10.9 months, respectively; objective response rate was 27%, 41% and 40%, respectively |
12/19/22 | Cancer |
| Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1202 | MetAP2 Inhibitor | Muscular invasive bladder cancer | Enrolled first patient in the phase II portion of the phase I/II study testing APL-1202 plus PD-1 inhibitor tislelizumab (Beigene Ltd.) as neoadjuvant therapy | 12/12/22 | Cancer |
| Autolus Therapeutics plc, of London | Obecabtagene autoleucel | CD19 CAR T-cell therapy | Acute lymphoblastic leukemia | Study met its primary endpoint; overall remission rate of 70%; well-tolerated | 12/9/22 | Cancer |
| Can-Fite Biopharma Ltd., of Petah-Tikva, Israel | Namodenoson | Drug binds to A3 adenosine receptor | Hepatocellular carcinoma (HCC) | Showed that median overall survival in the HCC Child-Pugh B7 (CPB7) patient population was 6.8 months for those treated with Namodenoson vs. 4.3 months for those treated with placebo; the < 1-year survival in the whole patient population was 32% in the namodenoson treated group vs. 14% (p=0.058) in the placebo treated patients, whereas in the CPB7 population 44% survival was found in the namodenoson treated group vs. 18% in the placebo treated one (p=0.028) | 12/28/22 | Cancer |
| Catalym GmbH, of Munich, Germany | Visugromab | Monoclonal antibody targeting GDF-15 | Advanced-stage tumors are relapsed or refractory to anti-PD-1/-PD-L1 treatment | Expanding 2 of the tumor-specific cohorts based on promising initial data, as well as a tumor-agnostic, biomarker-selected cohort; initial data expected in mid-2023 | 12/14/22 | Cancer |
| Dragonfly Therapeutics Inc., of Waltham, Mass. | DF-1001 | HER2-targeted NK cell engager therapy | Advanced solid tumors | First patient dosed | 12/6/22 | Cancer |
| Enterome SA, of Paris | EO-2401 | Microbiome-derived, peptide-based cancer vaccine | Recurrent glioblastoma | Completed enrollment of 100 patients in the Rosalie study; further data to be presented in 2023 | 12/12/22 | Cancer |
| Epicentrx Inc., of San Diego | RRx-001 | NLRP3 inhibitor | Advanced colorectal cancer | Data from the Rocket study published in Clinical Colorectal Cancer showed RRx-001 plus irinotecan produced a median overall survival of 8.6 months compared to 4.7 months for regorafenib; median progression-free survival was 6.1 months for RRx-001 plus irinotecan and 1.7 months for regorafenib (p=0.0030); overall response rate was 20.8% for RRx-001 plus irinotecan compared to 0% for regorafenib | 12/14/22 | Cancer |
| Imaginab Inc., of Los Angeles | Zr 89 crefmirlimab berdoxam | CD8 ImmunoPET agent | Melanoma or renal cell carcinoma | Initiated phase II study; trial enroll total of 12 patients | 12/6/22 | Cancer |
| Imaginab Inc., of Los Angeles | 89Zr crefmirlimab berdoxam | CD8 T-cell imaging agent | Neoadjuvant immune checkpoint inhibitor treatment of stage III surgically resectable melanoma | Investigator-sponsored C-TI Neo study started to test whether the imaging agent can allow for earlier surgical resection | 12/14/22 | Cancer |
| Immuneoncia Therapeutics Inc., of Seoul, South Korea | IMC-001 | PD-L1 monoclonal antibody | NK/T-cell lymphoma | Data showed 60% of patients achieved an objective response with complete remission | 12/5/22 | Cancer |
| IMV Inc., of Dartmouth, Nova Scotia, and Cambridge, Mass. | MVP-S | Delivers antigenic peptides from survivin | Relapse/refractory diffuse large B-cell lymphoma | Initial data from Vitalize phase IIb trial testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed complete responses in patients who received at least 3 lines of previous treatment, including CAR Ts; further data to be presented in February 2023 | 12/15/22 | Cancer |
| Maia Biotechnology Inc., of Chicago | THIO | Telomere-targeting agent | Non-small-cell lung cancer | Ongoing study testing THIO plus Libtayo (cemiplimab, Regeneron Pharmaceuticals Inc.) expanding into Hungary, Poland and Bulgaria | 12/13/22 | Cancer |
| Mimivax LLC, of Buffalo, N.Y. | Survaxm | Peptide vaccine | Glioblastoma | Study showed 51% of patients survived at least 2 years; 41% survived at 3 years; results published in the Journal of Clinical Oncology; 95.2%of patients remained progression-free 6 months after diagnosis; endpoints of median PFS of 11.4 months and median OS of 25.9 months; patients with the strongest antibody responses had a mOS of 43.1 months; safe and well-tolerated | 12/15/22 | Cancer |
| Mirati Therapeutics Inc., of San Diego | Adagrasib | KRAS G12C inhibitor | Advanced /metastatic non-small-cell-lung cancer | Krystal-7 study with a median follow-up of 3.5 months; objective response rate (ORR) of 49% in combination with pembrolizumab; ORR was 56% in subset of response-evaluable patients enrolled at least 6 months; Krystal-1 phase Ib study showed ORR of 57%; disease control rate (DCR) of 100%; manageable safety profile with no Grade 4-5 adverse events | 12/5/22 | Cancer |
| Moderna Inc., of Cambridge, Mass., and Merck & Co. Inc., of Rahway, N.J. | mRNA-4157/V940 | Personalized mRNA cancer vaccine | Stage III/IV melanoma following complete resection | In the Keynote-942/mRNA-4157-P201 trial, adjuvant treatment with mRNA-4157/V940 plus Keytruda (pembrolizumab) reduced the risk of recurrence or death by 44% compared to Keytruda alone | 12/13/22 | Cancer |
| Nanology LLC, of Fort Worth, Texas | LSAM-PTX | Large surface area microparticle paclitaxel | Mucinous pancreatic cysts | Data published in Endoscopy International Open showed cyst volume reduction of 10% to 78% was seen in 71% of the 19 patients treated for 24 weeks | 12/20/22 | Cancer |
| Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | HR+/HER2-negative advanced or metastatic breast cancer | In Right Choice study, Kisqali plus endocrine therapy (ET) showed statistically significant progression-free survival (PFS) benefit of 1 year vs. combination combination chemotherapy; Kisqali plus ET doubled the median PFS vs. combination CT at 24 months compared to 12.3 months (p=.0007) in the first-line setting; median time to treatment failure with Kisqali plus ET was 18.6 months compared to 8.5 months with combination CT; lower rates of treatment-related serious adverse events | 12/6/22 | Cancer |
| Nykode Therapeutics ASA, of Oslo, Norway | VB-10.16 | Therapeutic cancer vaccine targeting HPV16 | Checkpoint inhibitor refractory advanced cervical cancer | Plans to start the VB-C-04 study with potential registrational intent in the fourth quarter of 2023 | 12/20/22 | Cancer |
| Nykode Therapeutics ASA, of Oslo, Norway | VB-10.16 | Therapeutic cancer vaccine targeting HPV16 | Anal, penile, vaginal and/or vulvar cancer | Plans to collaborate on an investigator-initiated basket trial testing VB-10.16 + a PD-L1 inhibitor | 12/20/22 | Cancer |
| Oncoc4 Inc., of Rockville, Md. | ONC-392 | Next-generation target-preserving anti-CTLA4 antibody | Ovarian cancer | First patient dosed in combination Preserve-004 study with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with platinum-resistant disease; primary endpoints are efficacy, as measured by objective response rate per RECIST1.1, and safety | 12/30/22 | Cancer |
| PDS Biotechnology Corp., of Florham Park, N.J. | PDS-0101 | Protein subunit vaccine; apoptosis stimulator | Recurrent and/or metastatic HPV16-positive head and neck cancer | Completed enrollment in first stage of checkpoint inhibitor-refractory group of Versatile-002 study | 12/15/22 | Cancer |
| PDS Biotechnology Corp., of North Brunswick, N.J. | PDS-0101 combined with M-9241 and bintrafusp alfa | Immunotherapy based on the Versamune platform combined with the IL-12 fusion protein formerly known as NHS-IL12 and a bifunctional fusion protein targeting PD-L1 and TGF-? | Advanced human papillomavirus (HPV)-positive cancers | In new interim results, median overall survival (OS) is 21 months in 29 checkpoint inhibitor refractory patients who received the triple combination; in immune checkpoint inhibitor (CPI) naïve subjects, 75% remain alive at a median follow-up of 27 months; median OS has not yet been reached; objective response rate in CPI refractory patients who received the optimal dose of the triple combination is 63% (5/8) | 12/28/22 | Cancer |
| Precision Biologics Inc., of Bethesda, Md. | NEO-201 | Monoclonal antibody | Metastatic non-small-cell lung, head and neck, endometrial and cervical cancers | Completed safety phase and enrollment of new patients into expansion trial testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 12/14/22 | Cancer |
| Qilu Pharmaceutical Co. Ltd., of Jinan, China | QL-1706 | Dual immune checkpoint blockade containing a mixture of anti-PD-1 IgG4 and anti-CTLA-4 IgG1 antibodies | Non-small-cell lung cancer (NSCLC) | Data in combination with chemotherapy at median follow-up was about 9.2 months; objective response rate was 58.6%; squamous NSCLC cohort: 70.6%; non-squamous NSCLC cohort: 41.7%; disease control rate was 93.1%; median progression-free survival (mPFS) was about 7 months in 29 patients with advanced NSCLC with wild-type EGFR and naïve to systemic treatment ; median follow-up was 5.8 months; ORR was 64.5%, DCR was 93.5% ; PFS not yet matured; 6-month PFS rate was 61.3% in 31 patients with advanced NSCLC with mutated EGFR; good safety profile; manageable adverse events | 12/2/22 | Cancer |
| Qilu Pharmaceutical Co. Ltd., of Jinan, China | QL-1604 | Humanized monoclonal antibody that binds to PD-1 | Cervical cancer | Data showed median follow-up time was 12.91 months; 17.4% of patients achieved complete response and 41.3% achieved partial response; objective response rate of 58.7%; disease control rate was 84.8%; median duration of response was 9.6 months; median progression-free survival was 8.1 months; overall survival was not reached; manageable safety profile | 12/4/22 | Cancer |
| Quantum Leap Healthcare Collaborative, of San Francisco, and Athenex Inc., of Buffalo, N.Y. | Oral paclitaxel + encequidar | Taxane + P-glycoprotein inhibitor | Triple-negative subset of high-risk early-stage breast cance | In the I-Spy 2 adaptive study, oral paclitaxel was associated with less neuropathy and was not associated with an increase in febrile neutropenia relative to intravenous paclitaxel; data expected to be presented at medical meeting in the second quarter of 2023 | 12/20/22 | Cancer |
| Replimune Group Inc., of Woburn, Mass. | RP-1 (vusolimogene oderparepvec) | GM-CSF receptor agonist | Melanoma | Ignyte studies in combination with nivolumab showed deep and durable responses in patients with anti-PD1 failed melanoma; overall response rate of 36% and complete response rate of 20% | 12/7/22 | Cancer |
| Sirnaomics Inc., of Germantown, Md. | STP-705 | siRNA therapeutic; dual-targeted inhibitor of TGF-?1 and COX-2 | Cutaneous squamous cell carcinoma in situ | Interim results from part 1 of ongoing phase IIb trial showed majority of 32 treated patients (78%) achieved histological clearance; 1 of 3 treatment cohorts achieved 89% histological clearance | 12/14/22 | Cancer |
| Tessa Therapeutics Ltd., of Singapore | TT-11 | Autologous CAR T targeting CD30 | Relapsed or refractory CD30-positive classical Hodgkin lymphoma | Overall response rate was 73.3%, including 60% complete responses, in 15 heavily pretreated patients | 12/12/22 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Single-domain antibody against PD-L1 | Undifferentiated pleomorphic sarcoma or myxofibrosarcoma | Independent data monitoring committee for ongoing pivotal Envasarc trial recommended continued accrual as planned in both cohorts: single agent and in combination with Yervoy (ipilimumab, Bristol Myers Squibb Co.) | 12/14/22 | Cancer |
| Ultimovacs ASA, of Oslo, Norway | UV-1 | Cancer vaccine consisting of p719-20, p728, and p725 synthetic peptides, against human telomerase | Advanced or metastatic non-small-cell lung cancer (NSCLC) | The ongoing Lungvac study will start testing UV-1 with Libtayo (cemiplimab, Regeneron Pharmaceuticals Inc.) rather than Keytruda (pembrolizumab, Merck & Co. Inc.) starting on Jan. 1, 2023 following the decision of the Norwegian Health Authorities to reimburse for Libtayo for NSCLC | 12/20/22 | Cancer |
| Cardiol Therapeutics Inc., of Oakville, Ontario | Cardiolrx | Pharmaceutically manufactured cannabidiol | Recurrent pericarditis | Started an open-label pilot study to evaluate safety and objective measures of disease; extension period will asses feasibility of weaning concomitant background therapy including corticosteroids while continuing to take Cardiolrx | 12/12/22 | Cardiovascular |
| Gossamer Bio Inc., of San Diego | Seralutinib (GB-002) | Inhaled PDGFR, CSF1R, c-KIT inhibitor | Pulmonary arterial hypertension | Study achieved primary endpoint; statistically significant reduction in NT-proBNP increasing to a 408.3 ng/L mean difference from placebo at week 24 (p = 0.0012); mean difference in pulmonary vascular resistance (PVR) between the placebo and seralutinib arms of -96.1 dynes (p = 0.0310), equating to a placebo-corrected improvement of 14.3%; observed mean difference in 6MWD between placebo and seralutinib of 6.5 meters; 21% reduction in PVR (p = 0.0427) and 37m improvement in 6MWD (p = 0.0476) in FC III patients; 23% reduction in PVR (p = 0.0134) and 22m improvement in 6MWD (p = 0.2482) in patients with a baseline REVEAL 2 Risk Score of 6 or greater; well-tolerated; no cases of subdural hematoma were reported | 12/6/22 | Cardiovascular |
| Aslan Pharmaceuticals Ltd., of Singapore | Eblasakimab | Monoclonal antibody targeting the IL-13 receptor | Moderate to severe atopic dermatitis after treatment with dupilumab | Screened first of 75 patients in the Trek-Dx study testing 400 mg of eblasakimab weekly for 16 weeks; primary efficacy endpoint is percentage change in Eczema Area Severity Index (EASI) score from baseline to week 16; key secondary efficacy endpoints at week 16 include the proportion of patients achieving Investigator Global Assessment score of 0 or 1, proportion of patients with a 75% or greater reduction in EASI (EASI-75), EASI-50 and EASI-90 and changes in peak pruritus | 12/15/22 | Dermatologic |
| Kintor Pharmaceutical Ltd., of Suzhou, China | Pyrilutamide (KX-826) | Androgen receptor antagonist | Adult female androgenetic alopecia | Target area non-vellus hair count for the 5-mg, 4-times daily dose group increased by 11.39 hair counts per cm2 compared to placebo (p=0.0087); plans to use that dose in the phase III study | 12/1/22 | Dermatologic |
| Pharvaris BV, of Zug, Switzerland | PHVS-416 | Bradykinin B2 receptor antagonist | Hereditary angioedema | Top-line results showed study met primary and secondary endpoints; well-tolerated at all dose levels; mean VAS-3 changes at 4 hours vs. placebo (p=<0.0001 20 for all doses at 10, and 30-mg)< td> | 12/8/22 | Dermatologic |
| Ventyx Biosciences Inc., of Encinitas, Calif. | VTX-958 | Allosteric TYK2 inhibitor | Moderate to severe plaque psoriasis | First patient dosed | 12/1/22 | Dermatologic |
| Medicinova Inc., of La Jolla, Calif. | MN-001 (tipelukast) | Orally bioavailable, small-molecule compound | Hypertriglyceridemia | Subgroup analysis of completed phase II study showed greater reduction in serum triglyceride levels at week 8 (50.8% reduction for with T2DM vs 17.8% reduction for without T2DM, p=0.098); mean HDL increase was significantly greater in subjects with T2DM than subjects without T2DM at Week 8 (15.8% versus 1.0%, p<0.0002); 8 greater reduction in serum ldl levels at week (15.4% vs 6.7%)< td> | 12/6/22 | Endocrine/Metabolic |
| Sosei Group Corp., of Tokyo, and Pfizer Inc., of New York | PF-07081532 | GLP-1 receptor agonist | Type 2 diabetes and obesity | First patient treated in study | 12/20/22 | Endocrine/Metabolic |
| Akero Therapeutics Inc., of South San Francisco | Efruxifermin | Engineered to mimic the biological activity profile of native FGF21 | Non-alcoholic steatohepatitis in patients with compensated cirrhosis fibrosis stage 4 | Enrollment completed in the phase IIb Symmetry study | 12/21/22 | Gastrointestinal |
| Applied Molecular Transport Inc. (AMT), of South San Francisco | AMT-101 | Once-daily, GI-selective, oral fusion of IL-10 and AMT’s proprietary carrier molecule | Moderate-to-severe ulcerative colitis (UC) | Results from Lombard trial in biologic-naïve and experienced patients found clinical remission (CR) rate in patients treated with AMT-101 monotherapy was 17.1% vs. CR rate of 20% with placebo, which was above company’s baseline assumption for placebo CR rates based on published data in moderate-to-severe UC patients | 12/22/22 | Gastrointestinal |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-41 | Thyroid hormone receptor ? agonist | Non-alcoholic steatohepatitis | Enrollment is progressing and on target to complete enrollment by the end of the 2023 Q3 | 12/20/22 | Gastrointestinal |
| Chemomab Therapeutics Ltd., of Tel Aviv | CM-101 | Monoclonal antibody targeting CCL24 | Primary sclerosing cholangitis | After review of safety, the independent data monitoring committee for the Spring study cleared enrollment of patients in the 20 mg/kg dosing cohort; top-line data expected in the second half of 2024 | 12/21/22 | Gastrointestinal |
| Equillium Inc., of La Jolla, Calif. | Itolizumab | Monoclonal antibody targeting CD6 | Ulcerative colitis | Initiated phase II study; expected to enroll 90 patients | 12/16/22 | Gastrointestinal |
| First Wave Biopharma Inc., of Boca Raton, Fla. | Adrulipase | Enteric microgranule delivery formulation of adrulipase | Exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis | Initiated study; expected to report top-line data by mid-2023 | 12/5/22 | Gastrointestinal |
| Hepion Pharmaceuticals Inc., of Edison, N.J. | Rencofilstat | Cyclophilin inhibitor | Nonalcoholic steatohepatitis | Completed enrollment; results expected in the second quarter of 2023 | 12/5/22 | Gastrointestinal |
| Hillevax Inc., of Boston | HIL-214 | Virus-like particle (VLP) based vaccine | Moderate to severe norovirus-related acute gastroenteritis | Results from prespecified immunogenicity analysis of the 203 subjects showed geometric mean fold rise (GMFR) vs. baseline of more than 18-fold for HIL-214; 4-fold increase in pan-Ig; 99% for GI.1 and 86.9% for GII.4; top-line safety and efficacy data due in the second half of 2023 | 12/5/22 | Gastrointestinal |
| Lynk Pharmaceuticals Co. Ltd., of Hangzhou, China | LNK-01003 | Oral small-molecule JAK inhibitor | Active ulcerative colitis | First patient dosed | 12/5/22 | Gastrointestinal |
| Palisade Bio Inc., of Carlsbad, Calif. | LB-1148 | Oral formulation of broad-spectrum serine protease inhibitor | Reduction in intra-abdominal adhesions following elective bowel resection | Completed surgery for 31 patients; voluntarily ceasing enrollment in the trial; expecting top-line results of 35 patients in the first half of 2023 | 12/16/22 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody that targets TNF-like ligand 1A (TL1A) | Ulcerative colitis | Top-line results from Artemis-UC study met primary endpoint with 26.5% of patients on PRA-023 achieving clinical remission compared to 1.5% of patients on placebo at Week 12 (p<0.0001); 36.8% of patients reached the secondary endpoint endoscopic improvement (mayo endoscopy subscore ? 1) compared to 6.0% on placebo for a placebo-adjusted rate 30.8% (p<0.0001); all endpoints met with statistical significance; well-tolerated; no treatment-emergent serious adverse events< td> | 12/6/22 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | IgG1 humanized monoclonal antibody that targets TNF-like ligand 1A (TL1A) | Crohn's disease | Apollo-CD study showed 26% of patients on PRA-023 achieved endoscopic response (p=0.002) compared to 12% prespecified historical placebo rate; 49.1% of patients on PRA-023 achieved clinical remission (p<0.001 compared to 16% prespecified historical placebo rate); well-tolerated; no treatment-emergent serious adverse events< td> | 12/6/22 | Gastrointestinal |
| Karolinska Development AB, of Stockholm, and its portfolio company Dilafor AB, of Solna, Sweden | Tafoxiparin | Heparin derivative | Labor complication | Dilafor enrolled last patient to an extension of the phase IIb trial, intended to document the effect of tafoxiparin at further doses | 12/27/22 | Genitourinary/Sexual Function |
| Spago Nanomedical AB, Lund, Sweden | Spagopix (SN-132D) | Contrast agent | Endometriosis | First patient dosed | 12/8/22 | Genitourinary/Sexual Function |
| Imago Biosciences Inc., of Redwood City, Calif. | Bomedemstat | Lysine-specific demethylase 1 inhibitor | Myelofibrosis | First of approximately 20 patients dosed with bomedemstat plus Jakafi (ruxolitinib, Incyte Corp.) in the investigator-sponsored trial | 12/20/22 | Hematologic |
| Regimmune Ltd., of Taipei, Taiwan | RGI-2001 | Liposomal formulation of an alpha-galactosylceramide analog | Acute graft-versus-host disease (aGvHD) following allogeneic hematopoietic cell transplantation | Through day 100, the incidence of grades II-IV aGvHD was 20.4% in patients treated with RGI-2001 plus standard of care tacrolimus/methotrexate; at day 180, estimates of overall survival, grades II-IV aGvHD-free survival and disease relapse were 93.6%, 75.2% and 4.1%, respectively | 12/20/22 | Immune |
| Arbutus Biopharma Corp., of Warminster, Pa. | AB-729 | RNA interference targeting hepatitis B virus | Chronic hepatitis B virus infection | Completed enrollment of 43 patients; first 15 patients who received AB-729 plus nucleos(t)ide analogue therapy had an HBsAg decline of 1.51 log | 12/13/22 | Infection |
| Bioxytran Inc., of Needham, Mass. | Galectin antagonist | Orally administered in the form of a chewable tablet | COVID-19 | Randomized trial in 34 patients met its endpoint with a 100% response rate by day 7 vs. 6% in placebo, which was statistically significant (p=.001); showed 88% response rate by day 3, which was statistically significant (p=.001) | 12/28/22 | Infection |
| Clene Inc., of Salt Lake City | CNM-Znag | Ionic solution of zinc and silver | COVID-19 | Study in Brazil did not meet primary endpoint of time to substantial symptom resolution in acutely symptomatic, non-hospitalized COVID-19 patients; CNM-ZnAg was safe and well-tolerated | 12/27/22 | Infection |
| Jiangsu Recbio Technology Co. Ltd., of Taizhou, China | ReCOV | COVID-19 spike glycoprotein modulator | COVID-19 | 14 days after booster vaccination, seroconversion rate (SCR) of neutralizing antibody against omicron BF.7 of the ReCOV group and the mRNA vaccine group were 91.1% and 88.4%, respectively, and the SCR of the ReCOV group was higher than that of the mRNA vaccine group; geometric mean titers (GMT) of neutralizing antibodies in the ReCOV group and the mRNA vaccine group were 6,549.1 and 4,529.6, respectively, and the GMT in the ReCOV group was significantly higher than that in the mRNA vaccine group (p<0.001)< td> | 12/25/22 | Infection |
| Kinarus Therapeutics Holding AG, of Basel, Switzerland | KIN-001 | Orally available patented combination of pamapimod and pioglitazone | COVID-19 | Discontinued study after data safety and monitoring board concluded there was low probability to show statistically significant benefit with a reasonable number of hospitalized COVID-19 patients, due to a lower-than-anticipated incidence of the primary endpoint due to evolution of the current treatment landscape | 12/6/22 | Infection |
| Novavax Inc., of Gaithersburg, Md. | COVID-19-Influenza Combination (CIC) and influenza standalone vaccine candidates | Recombinant protein-based COVID-19 vaccine (NVX-CoV2373), quadrivalent influenza vaccine candidate, and saponin-based Matrix-M adjuvant | COVID-19 and influenza | Started dose-confirming trial to test immunogenicity of different formulations of CIC and influenza vaccines in adults, ages 50-80; primary and secondary objectives are to assess safety, tolerability and immune responses to various formulations; 2-part study to enroll about 2,300 participants in Australia and New Zealand, with initial results expected midyear 2023 | 12/30/22 | Infection |
| Ondine Biomedical Inc., of Vancouver, British Columbia | Nasal photodisinfection treatment | Uses non-thermal light to activate a photosensitive agent | Staphylococcus aureus and multidrug-resistant S. aureus (MRSA) | Study met the primary and secondary endpoint; eliminated or significantly decreased S. aureus in 86% of carriers (p<0.001); substantially lower surgical site infection rate than the u.s. national average (0.6% vs. 3%); no treatment-related reportable adverse events< td> | 12/2/22 | Infection |
| Osivax SAS, of Lyon, France | OVX-836 | Vaccine to elicit immune response to nucleoprotein | Influenza prophylaxis | Completed last patient last visit for the 2 phase IIa studies; top-line data expected in 2023 H1 | 12/21/22 | Infection |
| Pfizer Inc., of New York, and Valneva SE, of Saint-Herblain, France | VLA-15 | Lyme disease vaccine | Lyme disease prophylaxis | Geometric mean fold rises of antibodies were 1.9-fold for serotype 1 and 3.2-fold serotype 2 compared to baseline, 6 months after the final dose of the vaccine given at 0, 2 and 6 months | 12/1/22 | Infection |
| Recbio Technology Co. Ltd., of Taizhou, China | Recov | Recombinant 2-component vaccine | COVID-19 | Booster vaccination trial showed neutralizing antibody titer levels against omicron BA.5 and BA.2 and prototype strain induced by sequential vaccination of Recov were significantly better than those of mRNA vaccine group (with significant differences statistically); upon 14 days after booster, seroconversion rate (SCR) of neutralizing antibody against prototype strain was 96% and 91%, respectively (p=0.039); geometric mean titers (GMT) of neutralizing antibodies were 7,781.8 and 5,605.3, respectively (p<0.001); neutralizing antibody level induced by recov increased 25.1 times vs. baseline, compared to 15.7 for mrna vaccine< td> | 12/14/22 | Infection |
| Simcere Pharmaceutical Group Ltd., of Nanjing, China | Xiannuoxin (SIM-0417) | 3CL protease inhibitor | COVID-19 | Completed enrollment of 1,208 patients in the study | 12/20/22 | Infection |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif. | TP-05 | Oral formulation of lotilaner that inhibits parasite-specific GABA-CI channels | Lyme disease prophylaxis | First patient enrolled in the phase IIa Carpo study that will study the ability of the drug to kill sterile, non-disease carrying ticks after they have attached to the skin of healthy volunteers | 12/15/22 | Infection |
| Vir Biotechnology Inc., of San Francisco | VIR-2482 | Monoclonal antibody targeting influenza A | Influenza A infection | Completed enrollment in the Peninsula study; initial data expected in mid-2023 | 12/21/22 | Infection |
| Eupraxia Pharmaceuticals Inc., of Victoria, British Columbia | EP-104 | Encapsulates a highly potent corticosteroid (fluticasone propionate) | Osteoarthritis | Completed enrollment, randomization and dosing of the last patient; top-line results expected in the second quarter of 2023 | 12/7/22 | Inflammatory |
| Moonlake Immunotherapeutics AG, of Zug, Switzerland | Nanobody sonelokimab | Binds with high affinity to IL-17A and IL-17F | Active psoriatic arthritis | First subject screened; about 200 patients to be enrolled; primary endpoint is American College of Rheumatology 50 response, compared to placebo | 12/14/22 | Inflammatory |
| Amo Pharma Ltd., of London | AMO-02 (tideglusib) | Disrupts pathogenic RNA repeat in CDM1 and inhibits excess levels of the kinase GSK3beta | Congenital myotonic dystrophy | Completed patient enrollment in the Reach-CDM study | 12/6/22 | Musculoskeletal |
| Clinigen Group plc., of U.K. | Proleukin (aldesleukin) | Low dose IL-2 | Amyotrophic lateral sclerosis | Results from MIROCALS trial (n=220) showed not statistically significant analysis of the primary endpoint; 19% reduction in risk of death in IL2 treated patients compared with placebo; statistically significant decrease (73%) in the risk of death for the IL2 group over the 21-month trial period; well-tolerated; mild to moderate adverse events | 12/8/22 | Musculoskeletal |
| Neuvivo Inc., of Palo Alto, Calif. | Sodium chlorite (NP-001) | Anti-inflammatory drug | Amyotrophic lateral sclerosis | Data published in Biomedicines showed biomarkers associated with harmful microbial translocation (LPS, LBP and HGF), monocyte trafficking (sCD163) and inflammasome activation (IL-18) were significantly decreased in patients treated with NP-001 compared to placebo, while biomarkers associated with suppression of inflammation (IL-10, neopterin) and wound healing (EGF) increased | 12/1/22 | Musculoskeletal |
| Neurocrine Biosciences Inc., of San Diego | NBI-827104 | Orally active brain-penetrating T-type calcium channel blocker (Cav 3.1, Cav 3.2, Cav 3.3) | Epileptic encephalopathy with continuous spike-and-wave during sleep | Study did not meet its primary endpoint in pediatric patients ; well-tolerated | 12/6/22 | Neurology/Psychiatric |
| Actinogen Medical Ltd., of Sydney | Xanamem | 11?-HSD1 inhibitor | Major depressive disorder and cognitive impairment inadequately treated by current antidepressant medication | Treated first of approximately 160 patients in the 6-week study comparing the drug to placebo using the Cogstate Cognitive Test Battery and the Montgomery Asberg Depression Rating Scale; data expected in late 2023 or early 2024 | 12/14/22 | Neurology/Psychiatric |
| Aeon Biopharma Inc., of Irvine, Calif. | ABP-450 (prabotulinumtoxinA) | Cleaves SNAP-25 | Episodic migraine prevention | Completed enrollment in study; top-line data expected in 2023 H2 | 12/21/22 | Neurology/Psychiatric |
| Anavex Life Sciences Corp., of New York | ANAVEX 2-73 (blarcamesine) | Oral sigma-1 receptor activator | Mild cognitive impairment due to Alzheimer’s disease | Top-line results from the study met the primary endpoints ADAS-Cog (p=0.015) and ADCS-ADL (p=0.0255); statistically significant results in CDR-SB (p=0.040); safe and well-tolerated; no clinically significant changes in vital signs, laboratory values and ECG parameters | 12/1/22 | Neurology/Psychiatric |
| Baudaxbio Inc., of Malvern, Pa. | BX-1000 | Neuromuscular blocking agents | Neuromuscular blockade in patients undergoing elective surgery | Initiated phase II study; to evaluate safety, tolerability profile, and intubation conditions | 12/8/22 | Neurology/Psychiatric |
| Beckley Psytech Ltd., of Oxford, U.K. | BPL-003 | Synthetic intranasal formulation of the psychedelic 5-methoxy-N, N-dimethyltryptamine (5-MeO-DMT) | Treatment resistant depression | Started study testing the safety, pharmacokinetic and efficacy of BPL-003; first patient expected to enroll in January 2023 | 12/21/22 | 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 | Data showed NE-3107 experienced enhanced cognition as measured by multiple assessment tools, including a 2.1 points improvement on the modified ADAS-Cog12 scale (p=0.0173); reduced CSF phospho-tau levels by -1.66 pg/mL (p=0.0343) and the ratio of p-tau to A?42 by -0.0024 (p=0.0401); no drug-related adverse events | 12/5/22 | 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 | Parkinson's disease | Results in combination with levodopa achieved primary objective; improvements in UPDRS Part 3 (motor) score (3+ points superior to patients treated with levodopa alone) | 12/5/22 | Neurology/Psychiatric |
| Canaquest Medical Corp., of Toronto | CQ-001 | Cannabidiol molecules | Epilepsy | Study showed statistically significant efficacy results at higher doses compared to CBD; no negative side effects | 12/8/22 | Neurology/Psychiatric |
| Cognition Therapeutics Inc., of Purchase, N.Y. | CT-1812 | Modulates sigma-2 | Mild to moderate Alzheimer’s disease | Shine study expanded into sites in Spain, the Netherlands and the Czech Republic | 12/15/22 | Neurology/Psychiatric |
| Compass Pathways plc, of London | COMP-360 | Psilocybin therapy | Treatment-resistant depression | Results showed significantly better at reducing depressive symptoms at 25-mg compared to a 1-mg dose; 10-mg dose was not found to be significantly better than 1-mg dose | 12/8/22 | Neurology/Psychiatric |
| EIP Pharma Inc., of Boston | Neflamapimod | Oral, brain-penetrating small-molecule drug that inhibits the intracellular enzyme p38MAP kinase alpha | Early Alzheimer's disease | Analysis from 12 weeks showed increase in the volume of the nucleus basalis of Meynert (p=0.03, median 4.5% volume increase) | 12/2/22 | Neurology/Psychiatric |
| Imbrium Therapeutics LP, a unit of Stamford, Conn.-based Purdue Pharma LP | Sunobinop (IMB-115/V-117957) | Partial agonist of nociceptin/orphanin-FQ peptide receptors | Insomnia during recovery from alcohol use disorder | On nights 1 and 2, least squares mean (LSM) difference between sunobinop 1 mg and placebo for wakefulness after sleep onset was -12.03 minutes (p=0.05) and was -18.35 minutes for LSM difference between sunobinop 2 mg and placebo (p=0.003); on nights 20 and 21, LSM difference between drug and placebo was -12.35 minutes (p=0.099) and -15.80 (p=0.043) for the 1-mg and 2-mg doses, respectively | 12/12/22 | Neurology/Psychiatric |
| India Globalization Capital Inc., of Potomac, Md. | IGC-AD1 | Low-dose THC | Agitation in dementia due to Alzheimer’s disease | Started the 146-patient study comparing IGC-AD1 to placebo; primary endpoint is mean Cohen Mansfield Agitation Inventory score at week 6; secondary endpoint is change in score at week 2 | 12/1/22 | Neurology/Psychiatric |
| Irlab Therapeutics AB, of Gothenburg, Sweden | Mesdopetam | Oral dopamine D3-receptor antagonist | Levodopa-induced dyskinesia in Parkinson’s disease | Final patient completed treatment period and follow-up visit in phase IIb study; top-line results expected in mid-January 2023 | 12/14/22 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-1077 | Prodrug of the stimulant d-methylphenidate | Idiopathic hypersomnia | Started the randomized withdrawal study of approximately 48 patients testing the efficacy and safety of the drug; primary endpoint is safety and tolerability; major secondary efficacy endpoint is the change in Epworth Sleepiness Scale total score | 12/21/22 | Neurology/Psychiatric |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | LX-9211 | Oral, selective small molecule inhibitor of adaptor-associated kinase 1 | Postherpetic neuralgia | Results did not reach statistical significance on the primary endpoint; LX-9211 achieved a reduction in average daily pain score (ADPS) of 2.42 from baseline at week 6 compared to a reduction of 1.62 in the placebo arm, with a placebo adjusted difference of 0.80 (p=0.12); separation of LX-9211 from placebo on ADPS was seen at week 1 and maintained consistently thereafter, with an average placebo adjusted reduction over the 6-week dosing period of 0.80 (p=0.03) | 12/21/22 | Neurology/Psychiatric |
| Melt Pharmaceuticals Inc., of Nashville, Tenn. | MELT-300 | Fixed-dose combination of the benzodiazepine midazolam and the anesthetic ketamine | Sedation during cataract surgery | MELT-300 produced superior procedural sedation compared to 3mg midazolam (p=0.0129) and 50 mg ketamine (p=0.0096); patients taking MELT-300 were 50% less likely to require rescue sedation compared to midazolam (p=0.0198)) and 66% less likely to require rescue sedation pre-operatively | 12/21/22 | Neurology/Psychiatric |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Oral dipeptide | Phelan-McDermid, Angelman and Pitt Hopkins syndromes | Open label phase II trials in each indication are each enrolling up to 20 children each to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment; in Phelan-McDermid syndrome trial and in Angelman syndrome trial, the first subject in the oldest age group has now completed the treatment period of 13 weeks, with a good safety and tolerability profile | 12/22/22 | Neurology/Psychiatric |
| Small Pharma Inc., of London | SPL-026 | Intravenous N,N-dimethyltryptamine | Major depressive disorder | Last patient completed final visit | 12/21/22 | Neurology/Psychiatric |
| Synaptogenix Inc. (formerly Neurotrope Bioscience Inc.), of New York | Bryostatin-1 | Activator of protein kinase C | Alzheimer's disease | Top-line results showed primary endpoint of change from baseline in the Severe Impairment Battery (SIB) total score assessment obtained after completion of the second course of treatment (week 28) was not met with statistical significance; average increase in the SIB total score of 1.4 points and 0.6 points in Bryostatin-1 and placebo groups, respectively, at week 28 | 12/16/22 | Neurology/Psychiatric |
| Vanda Pharmaceuticals Inc., of Washington | VQW-765 | Small molecule alpha 7 nicotinic acetylcholine receptor partial agonist | Acute performance anxiety | Results showed numerically lower stress levels compared to those who received placebo; larger magnitude and statistically significant response to VQW-765 | 12/2/22 | Neurology/Psychiatric |
| Azura Ophthalmics Ltd., of Tel Aviv, Israel | AZR-MD-001 | Ophthalmic ointment | Meibomian gland dysfunction | Study achieved statistically significant differences compared to vehicle in both signs and symptoms at month 3; significant improvements in MGYLS score with patients experiencing an average increase of 1.8 more open glands secreting meibum from baseline (p=0.0004); significant improvements in OSDI score, with patients reporting an average improvement of 3.5 from baseline (p=0.0438); 46.9% of patients became asymptomatic as measured by total OSDI responder rate; 45.7% of patients had at least 5 more glands opened from baseline of 1.7, as measured by MGYLS responder rate; 68.7% of patients had their meibum quality return to normal levels, as measured by MGS responder rate; significant improvements in SPEED (p<0.0001) and average visual analogue scale (p<0.0001); significant improvements in eye discomfort (p<0.0001), dryness (p<0.0001) ocular itch no serious treatment-related adverse events< td> | 12/9/22 | Ocular |
| Cognition Therapeutics Inc., of Purchase, N.J. | CT-1812 | Sigma-2 receptor modulator | Geographic atrophy secondary to dry age-related macular degeneration | Company is in discussions with the U.S. FDA on design of a study testing CT-1812 in over 200 patients | 12/1/22 | Ocular |
| Oxurion NV, of Leuven, Belgium | THR-149 | Bicyclic peptide that selectively inhibits human plasma kallikrein | Diabetic macular edema | Independent data monitoring committee completed planned interim analysis of Kalahari part B trial and recommended continuation; top-line data expected in second half of 2023 | 12/14/22 | Ocular |
| Rezolute Inc., of Redwood City, Calif. | RZ-402 | Plasma kallikrein inhibitor | Diabetic macular edema | Initiated proof-of-concept study of oral therapy; study to enroll 100 patients; primary endpoints include change in central subfield thickness, change in visual acuity by ETDRS scale, repeat-dose pharmacokinetics and safety/tolerability | 12/15/22 | Ocular |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol formulated as permeation-enhanced clear gel | 22q11.2 deletion syndrome | Inspire 38-week data showed improvement in anxiety-related and behavioral symptoms; statistically significant improvements from baseline were seen in the PARS-R; well-tolerated | 12/5/22 | Other/Miscellaneous |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-629 | Reactive aldehyde species modulator | Alcohol intoxication | ADX-629 reduced dermal flushing (p=0.0007), increased Romberg test balance time (p=0.02) and lowered levels of acetaldehyde (p=0.03) compared to placebo following acute exposure to alcohol | 12/13/22 | Toxicity and Intoxication |
| Medicinova Inc., of La Jolla, Calif. | MN-166 (ibudilast) | Small-molecule compound; PDE4 inhibitor; parenteral formulation | Alcohol use disorder | Top-line secondary analysis results published in the American Journal of Drug and Alcohol Abuse showed ibudilast significantly fewer drinks per drinking day (DPDD) compared to the low baseline CRP group (p=0.007) who received MN-166 (ibudilast); significant interaction between MN-166 (ibudilast) and continuous log CRP levels on DPDD (p = 0.03); reward signaling in several brain regions relative to the high baseline CRP group treated with MN-166 (p <0.001) in low baseline crp group patients< td> | 12/5/22 | Toxicity and Intoxication |