Company Product Description Indication Status
Phase I
4D Molecular Therapeutics Inc., of Emeryville, Calif. 4D-310 Gene therapy Fabry disease Dosed first patient in phase I/II trial
Canbridge Pharmaceuticals Inc., of Beijing and Cambridge, Mass. CAN-106 Recombinant human monoclonal antibody that binds to and neutralizes C5 Paroxysmal nocturnal hemoglobinuria Dosed first subject in healthy volunteer trial to assess safety, pharmacokinetics and key biomarkers for C5 inhibition
Molecular Partners AG, of Zurich, Switzerland, and Novartis AG, of Basel, Switzerland Ensovibep (MP-0420) Trispecific antiviral COVID-19 Data from healthy volunteer study show drug safe and well-tolerated, with no significant adverse events reported; preliminary results showed extended exposure of candidate in serum, with half-life of 2-3 weeks, as expected from preclinical experiments
Oncosynergy Inc., of Stamford, Conn. OS-2966 Immunotherapy; anti-CD29 therapeutic Recurrent glioblastoma First patient treated
Orca Therapeutics BV, of ‘S-Hertogenbosch, the Netherlands ORCA-010 Integrin modulator Prostate cancer Independent data safety monitoring board recommended phase I/IIa trial in patients with localized disease continue without modification, based on review of all safety data from low- and mid-dose cohorts; company will initiate administration of highest schedule dose in single-dose part of study
Rocket Pharmaceuticals Inc., of Cranbury, N.J. RP-L201 ITGB2 gene stimulator Leukocyte adhesion deficiency-I Phase I/II trial fully enrolled; data expected in second quarter of 2021
VBI Vaccines Inc., of Ottawa, Ontario VBI-2902 Monovalent enveloped virus-like particle vaccine COVID-19 Initiated enrollment of phase I/II trial
Phase II
Aeon Biopharma Inc., of Newport Beach, Calif. ABP-450 (prabotulinumtoxinA) Botulinum toxin complex Migraine Started enrollment in trial for preventive treatment
Biovie Inc., of Santa Monica, Calif. BIV-201 Continuous infusion terlipressin Refractory ascites Started patient screening
Edesa Biotech Inc., of Toronto EB-01 sPLA2 inhibitor Chronic allergic contact dermatitis Completed enrollment of first cohort in phase IIb study
Gannex Pharma Co. Ltd., wholly owned unit of Ascletis Pharma Inc., of Shanghai, and Sagimet Biosciences Inc., of San Mateo, Calif. ASC-40 (TVB-2640 outside of China) Fatty acid synthase inhibitor Nonalcoholic steatohepatitis Top-line results from China cohort showed reduction of 28.2% for study drug vs. 11.1% for placebo in primary efficacy endpoint of liver fat, with 50% responder rate (≥30% reduction); statistically significant decrease of 29.8% (p=0.0499) in ALT (mean decrease of 33 U/L at week 12)
Graybug Vision Inc., of Redwood City, Calif. GB-102 Microparticle depot formulation of the pan-VEGF inhibitor sunitinib malate Wet age-related macular degeneration Preliminary top-line data from 12-month treatment phase of phase IIb Altissimo trial showed primary endpoint of median time to first supportive therapy was 5 months; 48% of patients did not require supportive therapy for at least 6 months, and 62% of patients for at least 4 months or more, at least once during the trial; overall, the 1-mg dose performed better than the 2-mg dose
Irlab Therapeutics AB, of Stockholm  Mesdopetam Dopamine D3 receptor antagonist Levodopa-induced dyskinesias in Parkinson's disease Dosed first patients in phase IIb/III study in Europe
Metacrine Inc., of San Diego MET-642 Non-bile acid farnesoid X receptor agonist Nonalcoholic steatohepatitis First patient treated in phase IIa trial
Oryzon Genomics SA, of Madrid Vafidemstat LSD1 inhibitor Alzheimer's disease 12-month data showed phase IIa Etheral trial met primary safety endpoint in 140 AD participants, with reduced levels of proinflammatory protein YKL40 maintained, but no cognitive improvement signals seen; 12-month data for 2 people with moderate AD in phase IIa Reimagine-AD trial showed reduction in agitation-aggression
Rhovac AB, of Stockholm RV-001 RhoC GTPase modulator Prostate cancer Phase IIb Bravac study delayed as COVID-19 vaccinations are rolled out to study population
TG Therapeutics Inc., of New York Ukoniq (umbralisib) Dual PI3K-delta/CK1-epsilon inhibitor Non-Hodgkin lymphoma  Results from phase IIb Unity-NHL monotherapy trial in people with relapsed/refractory indolent disease, previously presented at ASH 2020 conference, published in Journal of Clinical Oncology
Xenon Pharmaceuticals Inc., of Burnaby, British Columbia XEN-1101 Kv7 potassium channel modulator Major depressive disorder Icahn School of Medicine at Mount Sinai will facilitate investigator-sponsored trial, expected to begin in second quarter of 2021
Phase III
Clene Inc., of Salt Lake City  CNM-Au8 Bioenergetic nanocatalyst Amyotrophic lateral sclerosis >50% of participants enrolled in Healey ALS platform trial at sites of Northeast ALS consortium; in CNM-Au8 treatment regimen, 160 participants are randomized 3:1, with 120 across 2 active arms (30 mg and 60 mg) and 40 in placebo
Novartis AG, of Basel, Switzerland Canakinumab (ACZ-885)  IL-1beta inhibitor Non-small-cell lung cancer Canopy-2 combination study with docetaxel in 237 adults with locally advanced or metastatic disease following platinum-based chemotherapy and PD-(L)1 inhibitor missed primary endpoint of overall survival; NSCLC trials continue in first-line and adjuvant settings
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany Comirnaty (BNT-162b2, tozinameran) COVID-19 spike glycoprotein modulator COVID-19 infection The New England Journal of Medicine published updated report on neutralizing activity of BNT-162b2-elicited serum from patient samples obtained from pivotal trial, showing effectiveness against SARS-CoV-2 variants originating in U.K. (B.1.1.7), South Africa (B1.351) and Brazil (P.1)

Notes

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