Company Product Description Indication Status
Phase I
Basilea Pharmaceutica Ltd., of Basel, Switzerland Derazantinib FGFR inhibitor Advanced gastric cancer Started phase I/II Fides-03 study in patients whose disease has FGFR genetic aberrations; trial will assess drug as monotherapy and in combination with other treatments, including PD-L1 inhibitor Tecentriq (atezolizumab, Roche Holding AG)
Covaxx Inc., of Hauppauge, N.Y., and Hsinchu, Taiwan UB-612 Multitope peptide-based vaccine COVID-19 Dosed first healthy volunteers in open-label, dose-escalation trial in Taiwan
Covistat Inc., of San Diego, and parent company Ensysce Inc. Nafamostat Protease inhibitor COVID-19 Started study after receiving IND clearance; trial in healthy volunteers
Sarepta Therapeutics Inc., of Cambridge, Mass. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) DMD gene stimulator Duchenne muscular dystrophy 2-year data from phase I/II trial showed participants (n=4) sustained functional improvement, with mean 7-point improvement on North Star Ambulatory Assessment compared to baseline
Sarepta Therapeutics Inc., of Cambridge, Mass. SRP-9003 (rAAVrh74.MHCK7.hSGCB) SGCB gene stimulator Limb girdle muscular dystrophy 18-month data from low-dose cohort (n=3) in ongoing phase I/II trial showed improvements from baseline across all functional measures, including mean improvement from baseline of 3 at 6 months and 5.7 at 18 months in North Star Assessment for Dysferlinopathies (NSAD) as well as time-to-rise, 4-stair climb, 100-meter walk test and 10-meter walk test; 6-month data from high-dose cohort (n=3) also showed improvements from baseline across all functional measures, including mean NSAD improvement from baseline of 3.7
Sorrento Therapeutics Inc., of San Diego Resiniferatoxin Vanilloid VR1 agonist Osteoarthritis  Phase Ib trial showed no dose-limiting toxicities at doses tested (up to 30 ug) at 6 months post-administration; pain control in advanced disease (Kellgren-Lawrence grade 3/4) showed persistent relief >6 months; responder analysis at day 84 of those with baseline combined WOMAC A pain subscale and WOMAC C function subscale >=23 composite showed 68% (34/50) improved by at least 20% vs. 20% (2/10) for control
Tilt Biotherapeutics Oy, of Helsinki, Finland TILT-123 IL-2 agonist; TNF alpha ligand Metastatic melanoma First 2 participants dosed in Tunintil trial passed 36-day primary safety endpoint
Valirx plc, of London VAL-201 Src tyrosine kinase inhibitor Metastatic prostate cancer With 11/12 participants eligible for analysis, phase I/II trial showed overall response rate of 54.5%; maximum tolerated dose not reached
Phase II
Aptinyx Inc., of Evanston, Ill. NYX-2925 NMDA modulator Fibromyalgia Recruitment resumed following suspension in March 2020 due to COVID-19 pandemic
Aurinia Pharmaceuticals Inc., of Victoria, British Columbia Voclosporin Calcineurin inhibitor Dry eye disease Final participant dosed in phase II/III Audrey trial; top-line results expected in fourth quarter of 2020
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Peginterferon lambda-1a Interferon receptor modulator; interleukin-29 ligand COVID-19 infection Investigator-sponsored study in 120 outpatients with mild and uncomplicated infection showed no difference vs. placebo in duration of SARS-CoV-2 viral shedding, at median of 7 days, and time to symptom resolution
Immunic Inc., of New York IMU-838 Dihydroorotate dehydrogenase inhibitor COVID-19 infection At preplanned interim safety analysis, data monitoring committee recommended Calvid-1 trial in people with moderate infection continue without changes; 110 of expected 200 participants enrolled to date
Tilray Inc., of Nanaimo, British Columbia Cannabidiol + dronabinol Cannabinoid receptor agonist Chemotherapy-induced nausea and vomiting Investigator-sponsored study in 81 people found complete response was improved to 25% with study drug vs. 14% for placebo (p=0.041), with similar effects on absence of emesis, use of rescue medications, absence of significant nausea and summary scores for Functional Living Index-Emesis; study moving into phase III plans to recruit additional 170 people
Phase III
Innovent Biologics Inc., of Suzhou, China Tyvyt (sintilimab) + Byvasda (bevacizumab biosimilar, or IBI-305) PD-1 inhibitor + VEGF ligand inhibitor Hepatocellular carcinoma At interim analysis, Orient-32 study in first-line advanced disease met primary endpoints of progression-free and overall survival
PTC Therapeutics Inc., of South Plainfield, N.J. Evrysdi (risdiplam) Survival of motor neuron 2 splicing modifier Spinal muscular atrophy 2-year data from part 1 of Firefish study showed 88% (17/21) of treated infants remained alive and required no permanent ventilation, with motor function and developmental milestones showing continued improvement

Notes

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