Company Product Description Indication Status
Phase I
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 Gene therapy delivered via intravitreal injection Wet age-related macular degeneration Interim data from cohorts 1-4 of Optic trial further show effect in reducing treatment burden in patients requiring frequent anti-VEGF injections; long-term durability beyond 15 months from single IVT injection with 0 rescue injections in cohort 1 (high dose); substantial reduction in annualized anti-VEGF injection rate following administration of ADVM-022 (100% for high dose and 87% for low dose); therapy continues to be well-tolerated
Arcturus Therapeutics Holdings Inc., of San Diego ARCT-021 Self-replicating mRNA-based vaccine COVID-19 All subjects in first cohort dosed in phase I/II study; initial data from study expected in fourth quarter of 2020
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. ARO-ENaC RNAi therapeutic Cystic fibrosis Dosed first subject in phase I/II Aroenac1001 trial
Byondis BV, of Nijmegen, the Netherlands SYD-1875 Antibody-drug conjugate Solid tumors First patients started treatment testing drug in 5T4-expressing, locally advanced or metastatic solid tumors
Equillium Inc., of La Jolla, Calif. Itolizumab (EQ-001) Monoclonal antibody targeting CD6-ALCAM pathway Acute graft-vs.-host disease Interim data from first 2 cohorts of phase Ib portion of Equate study showed 5 of 7 patients achieved complete response by day 29
Indalo Therapeutics Inc., of Cambridge, Mass. IDL-2965 Integrin antagonist Idiopathic pulmonary fibrosis Study in healthy participants and IPF patients terminated due to development challenges associated with COVID-19 and emerging nonclinical data
SAB Biotherapeutics Inc., of Sioux Falls, S.D. SAB-185 Polyclonal antibody therapeutic COVID-19 Dosed first healthy participant
Phase II
Destiny Pharma plc, of Brighton, U.K. XF-73 nasal gel Antibacterial Prevention of postsurgical bacterial infections Interim safety review completed by independent data monitoring committee for ongoing phase IIb study in first 75 cardiac surgery patients, with recommendation that study continue without any modifications; recruitment remains on track to complete by end of 2020
Eisai Inc., of Woodcliff Lake, N.J. Lenvima (lenvatinib) Kinase inhibitor Radioactive iodine-refractory differentiated thyroid cancer Top-line results from Study 211 indicate the lower starting dose (18 mg) did not meet the noninferiority requirement vs. approved starting dose (24 mg) as measured by objective response rate at week 24; data support selection of 24 mg as appropriate starting dose; study was conducted as postmarketing commitment to FDA, EMA and other regulators following priority review designation and approval
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod Selective p38α/β mitogen activated protein kinase inhibitor Facioscapulohumeral muscular dystrophy Results from prespecified interim analysis of Redux4 trial from first 29 subjects indicate that DUX4-driven gene expression did not show separation from placebo at 16 weeks; however, in a prespecified sensitivity analysis, those with highest pre-treatment DUX4-driven gene expression in their muscle biopsy sample showed 38-fold reduction in DUX4-driven gene expression following treatment vs. a 5.4-fold reduction with placebo
Kezar Life Sciences Inc., of South San Francisco KZR-616 Proteasome inhibitor Active autoimmune hemolytic anemia or immune thrombocytopenia Marina study withdrawn due to COVID-19-related activity slowdown, high screen fail rate and lack of enrollment
Kymab Ltd., of Cambridge, U.K. KY-1005 Monoclonal antibody binding to OX40L Moderate to severe atopic dermatitis Met primary endpoints in phase IIa study in adults whose disease is not adequately controlled with topical corticosteroids
Tyme Technologies Inc., of Bedminster, N.J. SM-88 Cancer metabolism-based therapy Ewing’s sarcoma and other high-risk sarcomas Interim futility review for Hopes Sarcoma trial indicated study will proceed with current trial design as planned
Phase III
Bristol Myers Squibb Co., of New York Opdivo (nivolumab) PD-1 inhibitor Esophageal and gastroesophageal junction cancer CheckMate-577 trial evaluating study drug as adjuvant therapy met primary endpoint of disease-free survival at prespecified interim analysis, showing statistically significant improvement vs. placebo in randomized population following neoadjuvant chemoradiation therapy and complete resection
Bristol Myers Squibb Co., of New York Opdivo (nivolumab) PD-1 inhibitor Gastric and esophageal cancer Pivotal CheckMate-649 trial evaluating study drug + chemotherapy vs. chemotherapy alone in first-line metastatic disease met co-primary endpoints of overall survival (OS) at prespecified interim analysis and progression-free survival at final analysis in tumors expressing PD-L1 with combined positive score ≥ 5; OS benefit also seen in all-randomized population
Catalyst Pharmaceuticals Inc., of Coral Gables, Fla. Firdapse (amifampridine) Potassium channel inhibitor Myasthenia gravis Musk-MG trial missed statistical significance on primary endpoint, Myasthenia Gravis Activities of Daily Living assessment (p=0.2196), and secondary endpoint, Quantitative Myasthenia Gravis assessment (p=0.3736)
Fibrogen Inc., of San Francisco Pamrevlumab Connective tissue growth factor ligand inhibitor Duchenne muscular dystrophy First of about 90 non-ambulatory participants enrolled in Lelantos trial assessing study drug vs. placebo, both in combination with systemic corticosteroids; primary efficacy endpoint is change in total score of performance of upper limb assessment from baseline to week 52; additional endpoints include pulmonary and cardiac function tests
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA (injectable) Acetylcholine receptor antagonist; botulinum toxin A stimulator Glabellar (frown) lines Separate articles in Dermatologic Surgery on results from Sakura long-term safety study of single and repeat treatments in 2,691 participants confirmed consistent efficacy in response rates and duration of effect and results from pivotal trials; adverse events also were consistent across treatments, with no new safety signals
Romark Laboratories LC, of Tampa, Fla. NT-300 (nitazoxanide extended-release) Viral replication inhibitor COVID-19 infection Trial targeting treatment of mild or moderate infection initiated in up to 800 people 12 and older with fever and respiratory symptoms; primary endpoint in those with laboratory-confirmed infection is reduction in time to sustained response vs. placebo; secondary endpoint is reduction in rate of progression to severe infection vs. placebo


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