Company Product Description Indication Status
Phase I
Alligator Bioscience AB, of Lund, Sweden ATOR-1017 4-1BB antibody  Metastasized cancer Data review committee cleared the 100-mg dose and approved starting dosing at 200 mg; data from the study expected in the first half of 2021
Genetx Biotherapeutics LLC, of Sarasota, Fla., and Ultragenyx Pharmaceutical Inc., of Novato, Calif. GTX-102 Antisense oligonucleotide targeting UBE3A antisense transcript Angelman syndrome At day 128 in phase I/II trial, mean change in global rating on the Clinical Global Impression of Improvement Scale for Angelman Syndrome was 2.4 with all patients having at least 2 symptom domains that were assessed as +3 "very much improved" or  +2 "much improved"
Iveena Delivery Systems Inc., of Salt Lake City Ivmed-80 eyedrops Up-regulates lysyl oxidase and induces corneal crosslinking pharmacologically Keratoconus Top-line results from phase I/IIa study showed at 6-month observation period, group mean reduction of 1.8 D in Kmax; paired longitudinal analysis, baseline adjusted, resulted in statistically significant reduction in Kmax relative to placebo (p=0.0199); 11.3-letter improvement in BCVA over baseline
Oncolytics Biotech Inc., of San Diego Pelareorep  Oncolytic retrovirus vaccine Gastrointestinal cancer Phase I/II Goblet study will assess pelareorep in combination with PD-L1 inhibitor Tecentriq (atezolizumab, Roche Holding AG) in about 55 people with metastatic pancreatic or colorectal and advanced anal cancer, enrolled across 4 cohorts; trial will use master clinical supply agreement with Roche, which will provide Tecentriq for development plan; safety is primary endpoint, with overall response rate and blood-based biomarkers as exploratory endpoints
Samus Therapeutics Inc., of Boston PU-H71 Epichaperome inhibitor Myelofibrosis Study testing drug with concomitant ruxolitinib terminated; company focusing efforts on new formulation of PU-H71
Syntrix Pharmaceuticals Inc., of Auburn, Wash. SX-682 Inhibitor of CXCR1 and CXCR2 Myelodysplastic syndromes and metastatic melanoma Completed initial dosing in phase I/II trial; drug was well-tolerated and absorbed with dose-proportional drug levels in blood; adding cohorts of patients with pancreatic cancer, colorectal cancer and advanced tumors including breast and head and neck cancers 
Transgene SA, of Strasbourg, France TG-4001 HPV16-targeted therapeutic vaccine HPV16-positive recurrent and/or metastatic malignancies TG-4001 plus Bavencio (avelumab, EMD Serono/Pfizer Inc.) produced a 23.5% overall response rate in phase Ib/II study; ORR was 34.8% in patients without liver metastases with a median progression-free survival of 5.6 months
Phase II
Aerpio Pharmaceuticals Inc., of Cincinnati Razuprotafib  Vascular endothelial protein tyrosine phosphatase inhibitor Acute respiratory distress syndrome in moderate to severe COVID-19 Treated first patient in the study; primary endpoint is safety; secondary endpoints include patients alive and free of respiratory failure at day 7 and day 28; length of hospitalization and improvement on the NIAID 8-point ordinal scale
Alzheon Inc., of Framingham, Mass. ALZ-801 Anti-amyloid drug Early Alzheimer’s disease with 1 or 2 copies of APOE4 Treated first patient in the study measuring fluid and imaging biomarkers shown to be sensitive early markers of disease progression
Bridgebio Pharma Inc. and its affiliate, Phoenix Tissue Repair, both of Boston BBP-589 (PTR-01) Recombinant collagen 7 Dystrophic epidermolysis bullosa First of 6 patients dosed in the study measuring the drug's effect on wound healing and various systemic endpoints as well as long-term safety and tolerability
Ellodi Pharmaceuticals Inc., of Lawrenceville, N.J. APT-1011 (fluticasone propionate oral disintegrating tablet) Corticosteroid agonist Eosinophilic esophagitis Results of phase IIb study showed all dose arms had clinically meaningful and statistically significant achievement of histological remission with reduction of eosinophil counts in the esophageal mucosa, compared to placebo, at 12 weeks; that was accompanied by symptomatic improvement, as demonstrated by reduction in patient-reported disease symptoms, such as dysphagia events, which were maintained over 52 weeks 
Olatec Therapeutics LLC, of New York Dapansutrile NLRP3 inhibitor Moderate COVID-19 Enrolled first of approximately 80 patients in the study; primary endpoint is the complete resolution of fever symptoms and shortness of breath 
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162 Vaccine COVID-19 Pfizer reported in third-quarter earnings that, as of Oct. 26, the phase II/III study has enrolled more than 42,000 participants, with nearly 36,000 having receiving their second vaccinations
Reata Pharmaceuticals Inc., of Plano, Texas Omaveloxolone  Activator of Nrf2 Friedreich’s ataxia Data from the Moxie study published in the Annals of Neurology showed omaveloxolone produced a placebo-corrected mean improvement in mFARS of 2.40 points after 48 weeks of treatment (p=0.014)
Scholar Rock Inc., of Cambridge, Mass. SRK-015 Inhibitor of the activation of myostatin Type 2 and type 3 spinal muscular atrophy Interim analysis at 6 months of the Topaz study showed 67% of patients achieved ≥1 point improvement in Hammersmith scores; 35% of patients achieved ≥3 point increase in Hammersmith scores
Sumitomo Dainippon Pharma Oncology Inc., of Cambridge, Mass. DSP-7888 Immunotherapeutic cancer vaccine targeting Wilms tumor 1 Platinum-resistant ovarian cancer First patient dosed in the phase II expansion portion of the study testing the drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); primary endpoint is objective response rate; duration of response, disease control rate, progression-free survival, 6-month PFS rate and overall survival will also be measured
Phase III
Catabasis Pharmaceuticals Inc., of Boston Edasalonexent  Nuclear factor kappa B inhibitor Duchenne muscular dystrophy PolarisDMD study didn't meet the primary endpoint of change in North Star Ambulatory Assessment over 1 year compared to placebo; study also didn't meet secondary endpoints of time to stand, 10-meter walk/run and 4-stair climb; stopping development of the drug
Eli Lilly and Co., of Indianapolis Bamlanivimab (LY-CoV555) COVID-19 Spike glycoprotein modulator COVID-19 NIH halted Activ-3 trial testing drug in hospitalized patients; recommendation followed review of data suggesting drug is unlikely to help hospitalized patients recover from advanced stage of disease
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 Protein-based vaccine using nanoparticle technology and the Matri-M adjuvant COVID-19 prophylaxis U.K. study has enrolled 5,500 participants; study expanded to 15,000 patients which is expected to be fully enrolled by end of November; interim data expected as soon as early first quarter 2021; U.S. pivotal study in up to 30,000 participants will begin by the end of November
Prilenia Therapeutics BV, of Naarden, the Netherlands Pridopidine  S1R agonist Early stage Huntington’s disease Enrolled first of 480 patients in study; primary endpoint is change in Unified Huntington Disease Rating Scale-Total functional capacity score at week 65
Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Targets the mitochondrial tricarboxylic acid cycle Relapsed or refractory acute myeloid leukemia Enrolled 100 patients in the study testing the drug in combination with high-dose cytarabine and mitoxantrone
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan TAK-721 (budesonide oral suspension) Glucocorticoid receptor agonist Eosinophilic esophagitis Post hoc analyses in patients, ages 11-55, showed treatment improved dysphagia symptoms and endoscopic appearance while reducing eosinophil counts
Phase IV
Amgen Inc., of Thousand Oaks, Calif., and Eisai Co. Ltd., of Tokyo Apremilast and eritoran Phosphodiesterase 4 inhibitor and Toll-like receptor 4 antagonist Hospitalized COVID-19 Drugs being added to the REMAP-COVID adaptive platform trial


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