Company Product Description Indication Status
Phase I
Aravive Biologics Inc., of Houston AVB-500 GAS6/AXL inhibitor Advanced urothelial carcinoma First patient dosed in investigator-sponsored trial in combination with avelumab (Bavencio, EMD Serono Inc./Pfizer Inc.)
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod Selective p38α/β mitogen activated protein kinase inhibitor Facioscapulohumeral muscular dystrophy Complete results showed dose-dependent and sustained target engagement in blood; evidence of target engagement observed in skeletal muscle biopsies; data support selection of 15-mg dose twice daily in ongoing phase IIb trial and phase II open-label study
Novartis AG, of Basel, Switzerland AVXS-101 Gene therapy Spinal muscular atrophy type 2 Met the primary efficacy endpoint, achieving a mean increase from baseline of 6 points in the Hammersmith Functional Motor Scale-Expanded 
Oncolytics Biotech Inc., of San Diego Pelareorep Oncolytic retrovirus Breast cancer Safety committee provided favorable assessment from ongoing Aware-1 combination study with Tecentriq (atezolizumab, Roche Holding AG) in individuals with early stage disease; viral replication in majority of tumors seen in those enrolled in cohort 1, creating pro-inflammatory effect in tumor microenvironment
Oncopeptides AB, of Stockholm Melflufen Caspase-3 stimulator; peptidase modulator Multiple myeloma Findings from phase I/II O-12-M1 combination study with dexamethasone in individuals with relapsed/refractory disease, published in The Lancet Haematology, showed overall response rate of 31%, median duration of response of 8.4 months, median overall survival of 20.7 months and median progression-free survival of 5.7 months
Partner Therapeutics Inc., of Lexington, Mass. Leukine (sargramostim)  CSF-1 agonist; GM-CSF receptor agonist COVID-19 Sarpac trial initiated at University Hospital Ghent to assess efficacy of approved drug in nebulized form to treat respiratory illness associated with COVID-19, measured by lung function and patient outcomes
Seelos Therapeutics Inc., of New York Intranasal ketamine (SLS-002)
NMDA receptor antagonist
Suicidal ideation  Additional interim data showed all vital signs-related adverse events (AEs) seen in combined studies were mild and transient in nature; no serious AEs related to vital signs were observed
Phase II
Leading Biosciences Inc., of Carlsbad, Calif. LB-1148 (tranexamic acid) Serine protease inhibitor Bowel function Top-line data showed study drug provided 30% improvement in time to normal bowel function following cardiovascular surgery (p<0.001) vs. placebo, resulting in 1.1-day reduction in average ICU length of stay (LOS) and 1.2-day reduction in average hospital LOS
Oncoquest Inc., of Edmonton, Alberta Oregovomab Anti-CA-125 antibody Ovarian cancer Report in Gynecologic Oncology at median of 42 months follow-up showed risk of progression and death were reduced by > 50% with study drug plus carboplatin and paclitaxel vs. carboplatin/paclitaxel alone; report in Cancer Immunology, Immunotherapy from same study showed chemoimmunotherapy combo increased presence of CA125-specific CD8+T lymphocytes measured in peripheral blood compared to chemotherapy, correlating with clinical outcomes
Phase III
Avexis, unit of Novartis AG, of Basel, Switzerland Zolgensma (onasemnogene abeparvovec-xioi) SMN1 gene stimulator Spinal muscular atrophy Interim data from ongoing Spr1nt study continued to show participants achieved age-appropriate motor milestones when treated presymptomatically; 7 of 8 participants with 2 copies of SMN2 who achieved ability to sit independently did so within WHO window of normal development
Milestone Pharmaceuticals Inc., of Montreal Etripamil Calcium channel inhibitor Paroxysmal supraventricular tachycardia Node-301 trial evaluating termination of SVT episodes in at-home setting missed primary endpoint of time to conversion of SVT to sinus rhythm vs. placebo over 5-hour period following study drug administration (median time to conversion of 25 minutes for etripamil vs. 50 minutes for placebo, p=0.12)
Novavax Inc., of Gaithersburg, Md. Nanoflu Recombinant quadrivalent seasonal influenza vaccine Influenza virus infection Pivotal trial in adults 65 and older achieved primary endpoints of day 28 ratio of geometric mean titers (24% to 66% higher) responses and difference in seroconversion rates (11.4 to 20.4 percentage points higher) for vaccine's 4 strains vs. Fluzone Quadrivalent (Sanofi SA); Nanoflu was well-tolerated and safety profile was comparable to comparator with modest increase in local adverse events; BLA filing expected under FDA accelerated approval pathway
Rafael Pharmaceuticals Inc., of Cranbury, N.J. Devimistat (CPI-613) 2 oxoglutarate dehydrogenase inhibitor; dual pyruvate dehydrogenase inhibitor/kinase stimulator Metastatic pancreatic cancer > 75% of expected 500 participants enrolled ahead of scheduled in pivotal Avenger 500 combination trial with modified Folfirinox as first-line therapy; COVID-19 task force monitoring trials to make changes, as needed


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