Company Product Description Indication Status
Phase I
Biomarin Pharmaceutical Inc., of San Rafael, Calif. BMN-307 AAV5-phenylalanine hydroxylase gene therapy  Phenylketonuria First patient dosed
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia INM-755 Cannabinol topical cream Epidermolysis bullosa All subjects completed treatment
Medicinova Inc., of La Jolla, Calif. MN-166 (ibudilast) Small-molecule macrophage migration inhibitory factor inhibitor and PDE4/10 inhibitor  Chemotherapy-induced peripheral neuropathy Across all neurotoxicity measures, a majority of participants experienced either an improvement or no worsening of neurotoxicity; data published in Cancer Chemotherapy and Pharmacology
Millendo Therapeutics Inc., of Ann Arbor, Mich. MLE-301 Selective neurokinin 3 receptor antagonist Hot flashes and night sweats in menopausal women First patient dosed
Neomatrix Therapeutics Inc., of New York NMT-cP12 Platelet-derived growth factor modulator  Skin burns Safe and well-tolerated in 5 cohorts of healthy volunteers; started designing phase II trial to launch in 2021
Phase II
Akero Therapeutics Inc., of South San Francisco Efruxifermin FGF-21 ligand Nonalcoholic steatohepatitis Planned adaptive phase IIb/III trial will evaluate 2 doses in a 24-week phase IIb part to inform selection of a single dose for phase III
Calithera Biosciences Inc., of South San Francisco Telaglenastat Glutaminase inhibitor Non-small-cell lung cancer Started Keapsake trial, evaluating telaglenastat plus standard-of-care immunotherapy as first-line therapy for stage IV nonsquamous NSCLC patients whose tumors have a KEAP1 or NRF2 mutation
Cortexyme Inc., of South San Francisco Atuzaginstat Gingipain inhibitor Alzheimer's disease Interim analysis of the phase II/III GAIN trial expected to occur before year-end 2020; top-line results expected in Q4 2021
Theralase Technologies Inc., of Toronto TLD-1433 Ruthenium- based photodynamic compound Bladder cancer New patient enrollment and treatment in trial has restarted
Phase III
Aerie Pharmaceuticals Inc., of Durham, N.C. Roclanda (netarsudil and latanoprost ophthalmic solution) Rho associated protein kinase inhibitor; PGF2 alpha agonist Open-angle glaucoma or ocular hypertension Interim 90-day top-line data showed 6-month phase IIIb Mercury 3 trial met overall objective by demonstrating noninferiority to Ganfort, a fixed-dose combination of PGA bimatoprost and beta-blocker timolol, across 9 timepoints; demonstrated consistent intraocular pressure (IOP) reduction through the day of about 9.5 mmHg, for average reduction from baseline IOPs of about 37%
Axsome Therapeutics Inc., of New York AXS-07 5-HT 1d receptor agonist; cyclooxygenase 2 inhibitor; 5-HT 1b receptor agonist Acute migraine Drug rapidly relieved and substantially reduced relapse of migraine pain, as compared to active comparator rizatriptan, in Momentum trial; probability of achieving pain relief was greater vs. rizatriptan within 30 minutes after dosing and at every time point thereafter, with median time to migraine pain relief that was nearly 3x faster (1.5 vs. 4 hours, p<0.001); drug substantially and significantly reduced relapse of migraine pain vs. rizatriptan, with 45.2% of rizatriptan-treated patients experiencing relapse compared to 21.2% of AXS-07 patients over 48 hours after dosing (p=0.001)
Oncimmune Ltd., of Rockville, Md. Saccovid (CD24Fc) Immunomodulator targeting innate immune system COVID-19 Closed enrollment of trial and released top-line results from preplanned efficacy analysis from 203 participants, showing severe or critical patients treated with drug exhibited faster recovery and significantly reduced disease progression to death or respiratory failure vs. those receiving placebo when used with standard of care; results showed 60% better chance to achieve clinical recovery (p=0.005), with median time to recovery of 6 days vs. 10 days for placebo; risk of death or respiratory failure reduced by more than 50%
Verrica Pharmaceuticals Inc., of West Chester, Pa. VP-102 Drug-device combination containing cantharidin 0.7% Molluscum  Results from two pivotal studies, Camp-1 and Camp-2, published in the Journal of the American Medical Association Dermatology, showed treatment demonstrated superior results vs. vehicle in percentage of participants with complete clearance of molluscum lesions at the end of trial (day 84); in CAMP-1, 46% achieved complete clearance of molluscum lesions compared to 18% in vehicle group (p<0.001); in CAMP-2, 54% achieved complete clearance vs. 13% in vehicle group (p<0.001)
Phase IV
Vifor Pharma Group, of St. Gallen, Switzerland Ferinject (intravenous ferric carboxymaltose) Blood system agent; iron uptake stimulator Iron deficiency in acute heart failure Top-line data from Affirm-AHF study in patients hospitalized for acute heart failure missed statistical significance on its composite primary endpoint of reducing the risk of total heart failure hospitalizations and cardiovascular death; a prespecified sensitivity analysis considering the impact of the COVID-19 pandemic, however, revealed statistically significant difference in favor of Ferinject on cardiovascular mortality and hospitalization for heart failure


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