Company Product Description Indication Status
Phase I
Cynata Therapeutics Ltd., of Melbourne, Australia CYP-001 Mesenchymoangioblast-derived mesenchymal stem cell therapy Graft-vs.-host disease In 2-year follow-up, overall survival was 60% (9/15), comparing favorably to previous outcomes in patient population; no treatment-related serious adverse events or safety concerns identified
Enterome SA, of Paris EO-2401  Microbiome-derived vaccine Glioblastoma multiforme Phase I/II Rosalie combination trial with immune checkpoint inhibitor initiated, aiming to enroll 32 people with progressive or first recurrent disease; data expected in 2022
Genocea Biosciences Inc., of Cambridge, Mass. GEN-009 CD4 agonist; T-cell surface glycoprotein CD8 stimulator Advanced solid tumors Initial data on first 5 participants from part B of ongoing phase I/IIa combination study with checkpoint inhibitor-based regimens showed 3 achieved independent RECIST responses starting from first dose; overall, 2 achieved complete response and 3 showed partial response
Sab Biotherapeutics Inc., of Sioux Falls, S.D. SAB-176 Human IgG immunotherapy  Influenza virus infection First of 27 healthy volunteers dosed in trial to inform dosing for phase II; study expected to complete in early 2021
Salarius Pharmaceuticals Inc., of Houston Seclidemstat LSD1 inhibitor Ewing sarcoma Phase I/II trial in relapsed/refractory disease to expand into second cohort of about 30 people with Ewing-related sarcomas after refractory Ewing participant on study drug for 6 months showed reduction of >80% in prospectively defined target lesions but, at 8 weeks, increase in non-target lesions resulted in overall RECIST classification of progressive disease
Terns Pharmaceuticals Inc., of Foster City, Calif. TERN-201 Semicarbazide-sensitive amine oxidase inhibitor Nonalcoholic steatohepatitis In healthy volunteers, single and multiple oral doses were well-tolerated with no significant safety findings; each dose level showed potent target engagement and achieved near complete inhibition of plasma SSAO activity, maintained up to 1 week post-dosing
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole (inhalation powder) Lanosterol-14 demethylase inhibitor Invasive pulmonary aspergillosis infection Dosing completed, with repeated doses tolerated in healthy volunteers
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-621 Immunoglobulin gamma Fc receptor agonist Cutaneous T-cell lymphoma Dose-limiting toxicity evaluation of cohort 4 (1.4 mg/kg) in phase Ib study completed; dose escalation continuing at 2 mg/kg
Tychan Pte Ltd., of Singapore TY-014 Monoclonal antibody Yellow fever Data published in The New England Journal of Medicine demonstrate safety and efficacy and complete eradication of yellow fever virus in the bloodstream of all participants within 48 hours of infusion
Xeris Pharmaceuticals Inc., of Chicago XP-0863 (liquid diazepam) Benzodiazepine receptor agonist Epilepsy Additional data from phase Ib study in healthy adults showed comparable pharmacokinetics to Diastat (Bausch Health Companies Inc.), with similar partial AUCs of XP=0863 (0.25 mg/kg) to Diastat after dosing and increased overall exposure compared to Diastat (18,800 h*ng/mL vs. 10,900 h*ng/mL, respectively); study drug (0.25 mg/kg) also had comparable Cmax vs. Diastat (355 ng/mL vs. 384 ng/mL, respectively); program expected to advance directly into phase III registration study in children and adults with epilepsy
Phase II
Astrazeneca plc, of Cambridge, U.K., and Sanofi SA, of Paris Nirsevimab Human IgG1-kappa monoclonal antibody Respiratory syncytial virus infection Phase IIb results, published in The New England Journal of Medicine, showed study drug achieved statistically significant 70.1% reduction of medically attended RSV lower respiratory tract infection (LRTI) vs. placebo in healthy preterm infants through 150 days post-dose; on secondary endpoint, nirsevimab achieved 78.4% relative reduction in hospitalizations due to RSV LRTI vs. placebo through 150 days post-dose
Eisai Co. Ltd., of Tokyo BAN-2401 Beta-amyloid antagonist Alzheimer's disease Bioarctic AB said Eisai presented data from phase IIb open-label extension showing decrease in brain amyloid levels measured at 3, 6 and 12 months in those who received placebo during active study, with observed effect comparable to results in those randomized initially to highest dose of study drug
Redhill Biopharma Ltd., of Tel Aviv, Israel Yeliva (opaganib, ABC-294640) Sphingosine kinase-2 inhibitor  COVID-19 infection Global phase II/III study initiated in individuals hospitalized with severe infection and pneumonia requiring supplemental oxygen; trial expected to enroll up to 270 participants, with primary endpoint of proportion requiring intubation and mechanical ventilation by day 14; unblinded interim futility analysis by data safety monitoring board planned when about 100 evaluated for primary endpoint
Theralase Technologies Inc., of Toronto TLD-1433 Light-activated photodynamic compound Bladder cancer First 12 participants with non-muscle invasive disease showed 58.3% response 90 days following initial treatment with drug/device; Canadian sites remain closed to new enrollment but sites that already enrolled and treated participants providing second treatments to eligible (n=8) participants
Phase III
Boehringer Ingelheim International GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) SGLT2 inhibitor Heart failure Emperor-Reduced trial in 3,730 adults with heart failure with reduced ejection fraction, with or without diabetes, met primary endpoint in reducing risk of cardiovascular death or hospitalization vs. placebo
Onconova Therapeutics Inc., of Newtown, Pa. Rigosertib Small-molecule RAS mimetic Myelodysplastic syndrome Required number of survival events in pivotal Inspire trial reached to begin data analysis; top-line results expected by end of third quarter of 2020


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