Company Product Description Indication Status
Phase I
Astex Pharmaceuticals Inc., of Pleasanton, Calif., a subsidiary of Otsuka Pharmaceutical Co. Ltd., and Forma Therapeutics Inc., of Watertown, Mass. ASTX-727 and FT-2102 Oral DNMT inhibitor and IDH1m inhibitor Recurrent/refractory myelodysplastic syndrome of acute myeloid leukemia Phase I/II study in patients with IDHI-mutated disease paused due to COVID-19
Beyond Air Inc., of Garden City, N.Y. Inhaled nitric oxide Inhaled nitric oxide Bronchiolitis Top-line results from third pilot study showed, on an intent-to-treat basis, 150 parts per million (ppm) NO is statistically significant compared to both the 85 ppm NO and control arms
Cyclo Therapeutics Inc., of Gainesville, Fa. Trappsol Cyclo Cyclodextrin-based product  Niemann-Pick disease type C1 Top-line data showed favorable safety and tolerability profile; drug crossed the blood-brain barrier as measured in cerebrospinal fluid (CSF) taken at intervals following the onset of intravenous infusion, with CSF:plasma ratio at 8 hours of 2% increasing to 11%-16% at 12 hours, suggesting significant persistence of the drug in CSF for several hours after the end of infusion; doses in both treatment groups affected cholesterol synthesis and metabolism as measured by cholesterol precursors, such as lathosterol, and cholesterol metabolites, such as 4-beta-hydroxysterol, in serum
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. TSC Trans sodium crocetinate COVID-19 Entered agreement with Romanian National Institute of Infectious Disease to begin trial program in hospitalized patients presenting with lowered blood oxygen levels
Phase II
Arch Biopartners Inc., of Toronto Metablok LSALT peptide COVID-19 Expanded primary endpoint to prevent organ inflammation and injury experienced by patients with COVID-19; in addition to acute respiratory distress syndrome, primary endpoint now includes acute kidney injury, cardiomyopathy, acute liver injury, coagulopathy and all-cause mortality as components of a composite endpoint in hospitalized patients
KBP Biosciences Inc., of Princeton, N.J. KBP-5074 Nonsteroidal mineralocorticoid receptor antagonist Moderate to severe chronic kidney disease and uncontrolled hypertension Completed patient enrollment in Block CKD phase IIb trial; top-line data expected in fourth quarter of 2020
Phase III
Alkermes plc, of Dublin ALKS-5461 Acts as opioid system modulator Refractory major depressive disorder Study terminated due to decision of sponsor
Cidara Therapeutics Inc., of San Diego Rezafungin Echinocandin antifungal Prevention of invasive fungal disease in allogeneic blood and marrow transplantation Dosed first patient in Respect pivotal trial; drug dosed once weekly will be compared to daily regimens containing multiple drugs, including fluconazole or posaconazole, and trimethoprim-sulfamethoxazole, for 90 days, at which time fungal-free survival will be measured as primary efficacy outcome
Roche Holding AG, of Basel, Switzerland Ranibizumab Port delivery system for anti-VEGF antibody Diabetic macular edema Enrollment in study comparing port delivery system to intravitreal administration temporarily paused due to COVID-19
Santhera Pharmaceuticals AG, of Pratteln, Switzerland Idebenone Synthetic short-chain benzoquinone and a cofactor for the enzyme NQO1 Duchenne muscular dystrophy Full recruitment in Sideros study; company is assessing potential of conducting interim analysis due to study’s high power (>99%)


For more information about individual companies and/or products, see Cortellis.

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