Company Product Description Indication Status
Phase I
Ampio Pharmaceuticals Inc., of Englewood, Calif. Ampion (AP-014) Stem cell antigen-1 inhibitor COVID-19 Study is 55% enrolled
Atyr Pharma Inc., of San Diego, and Kyorin Pharmaceutical Co. Ltd., of Tokyo ATYR-1923 (KRP-R120) Fusion protein combining the immuno-modulatory domain of histidyl tRNA synthetase fused to the FC region of a human antibody Healthy volunteers (eventually interstitial lung disease) Completed last subject visit in the study testing the safety, pharmacokinetics and immunogenicity of the drug in 32 healthy Japanese male volunteers
Bicycle Therapeutics plc, of Cambridge, U.K. BT-5528 Bicyclic peptide-toxin conjugate targeting EphA2 Solid tumors Drug tolerated up to 8.5 mg/m2 weekly, which is expected to be toward the top of the therapeutic range; a EphA2-selected urothelial patient had a 43% reduction in target lesions; an ovarian cancer patient retrospectively determined to be positive for EphA2 has an ongoing 25% reduction in target lesions
Phase II
Knopp Biosciences LLC, of Pittsburgh, Pa. Dexpramipexole  Dopamine D3 receptor agonist Asthma Exhale study met primary endpoint, showing statistically significant, dose-dependent reduction in blood absolute eosinophil count from baseline to week 12 vs. placebo when added to standard of care in people with moderate to severe eosinophilic disease
Mirum Pharmaceuticals Inc., of Foster City, Calif. Volixibat Ileal sodium bile acid cotransporter inhibitor Primary sclerosing cholangitis First participant enrolled in phase IIb Vistas study; primary endpoint is change in pruritus from baseline vs. placebo using Adult ItchRO tool
Nantkwest Inc., of El Segundo, Calif., and Immunitybio Inc., of Culver City, Calif.
PD-L1 t-haNK 
PD-L1 modulator Metastatic pancreatic cancer Early interim results for cohort C of ongoing Quilt 88 trial, initiated in October 2020 with overall survival as primary endpoint, showed 15/18 (83%) with second-line or greater disease remained alive; cohorts A and B, with progression-free survival as primary endpoint, enrolling people with first- and second-line disease
United Therapeutics Corp., of Silver Spring, Md. Tyvaso (treprostinil) PGI2 agonist Pulmonary hypertension associated with interstitial lung disease The New England Journal of Medicine published online results from Increase trial that showed inhaled study drug was well-tolerated and improved exercise capacity measure of 6-minute walk distance by 31 meters vs. placebo at week 16 (p<0.001) 
Verona Pharma plc, of London Ensifentrine (via pressurized metered-dose inhaler) Dual PDE3/PDE4 inhibitor  Hospitalized COVID-19 Completed enrollment of 45 patients; top-line results expected in the second quarter of 2021
Phase III
Alexion Pharmaceuticals Inc., of Boston Ultomiris (ravulizumab-cwvz) Complement C5 factor inhibitor COVID-19 infection Enrollment paused in study of people with severe infection requiring mechanical ventilation based on independent data monitoring committee interim finding of lack of efficacy when added to standard of care
Organogenesis Holdings Inc., of Canton, Mass. Renu Cryopreserved amniotic suspension allograft  Knee osteoarthritis First of 474 participants with moderate to severe symptomatic disease enrolled in pivotal trial; primary efficacy endpoint is difference in pain from baseline to 6 months vs. placebo as assessed by WOMAC index


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