Company Product Description Indication Status
Phase I
Context Therapeutics LLC, of Philadelphia Onapristone extended release Oral progesterone receptor antagonist Progesterone receptor-positive cancers Phase I/II results published in Drug Safety showed drug, at escalating doses of 10 mg to 50 mg twice-a-day, had favorable safety and tolerability profile at all doses; no subject receiving ONA-ER developed liver test elevations meeting Hy’s Law criteria or other clinically significant hepatic injury considered to be drug-related
Daiichi Sankyo Co. Ltd., of Tokyo DS-1062 Anti-TROP2 monoclonal antibody conjugated to topoisomerase I inhibitor exatecan Breast tumor First patient with triple-negative breast cancer dosed after preliminary results in patients with non-small-cell lung cancer
Ipsen SA, of Paris Onivyde (irinotecan liposome injection) + 5-FU/LV and oxaliplatin Encapsulated nanoliposomal formulation of irinotecan sucrosofate combo Metastatic pancreatic ductal adenocarcinoma No new safety signals observed in 32 patients evaluated from recommended NALIRIFOX 50/60 mg/m2 dose (primary endpoint); patients achieved median progression-free survival of 9.2 months and median overall survival of 12.6 months (secondary endpoints)
Oncolys Biopharma Inc., of Tokyo Telomelysin (OBP-301)  Telomerase modulator Hepatocellular carcinoma 18 patients evaluable for safety were found to tolerate drug; next steps to be decided with licensee Chugai Pharmaceutical Co. Ltd.
Pfizer Inc., of New York, and Biontech, of Mainz, Germany BNT-162b1 COVID-19 Spike glycoprotein modulator COVID-19 Companies announced positive preliminary data from ongoing phase I/II trial of vaccine candidate, with participants in each of 3 dose groups mounting immune responses that were greater than seen in patients who recovered from COVID-19 infections
Transcenta Holding Ltd., of Suzhou, China TST-001  Humanized Claudin18.2 monoclonal antibody Advanced or metastatic solid tumors Dosed first subject in trial to evaluate safety, tolerability and recommended phase II dose 
Phase II
Akero Therapeutics Inc., of South San Francisco Efruxifermin (AKR-001) FGF receptor agonist; FGF-21 ligand Nonalcoholic steatohepatitis In phase IIa Balanced study, 40 participants who had end-of-treatment biopsies achieved 48% fibrosis improvement of at least 1 stage without worsening of NAFLD activity score across dose groups, with 62% response rate for 50-mg group; 28% fibrosis improvement of at least 2 stages across dose groups, with 38% response rate for 50-mg group; 48% NASH resolution without worsening of fibrosis across dose groups, with 54% response rate for 50-mg group
Anavex Life Sciences Corp., of New York Blarcamesine (ANAVEX-2-73) Muscarinic receptor modulator; opioid receptor sigma agonist 1 Rett syndrome First of at least 69 participants dosed in phase II/III Excellence trial assessing once-daily oral liquid formulation over 12 weeks; enrollment in open-label extension will be offered
Durect Corp., of Cupertino, Calif. DUR-928 Liver X receptor antagonist COVID-19 infection Enrollment opened among individuals hospitalized with COVID-19 infection who have acute liver or kidney injury; trial expected to enroll about 80 participants, who will receive 150 mg of study drug or placebo by intravenous infusion on day 1 and day 4 in combination with standard of care; primary efficacy endpoint is composite of survival and freedom from acute organ failure at day 28
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 (icosapent + docosapentaenoic acid + heneicosapentaenoic acid) Omega-3 fatty acid-based therapeutic Hypertriglyceridemia Enrollment resumed following pause due to COVID-19 pandemic and dosing begun in Enhance-It trial in Uganda comparing study drug vs. Vascepa (icosapent ethyl, Amarin Corp. plc); enrollment expected to complete in August 2020, with top-line data available in first quarter of 2021
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-2203 Lipid-crystal nanoparticle cochleate formulation of amphotericin B Cryptococcus neoformans meningitis Uganda National Drug Authority cleared restart of Enact study following pause due to COVID-19 pandemic, with dosing expected to begin imminently; first cohort expected to complete enrollment by early September 2020, with data evaluation of cohort by independent DSMB and expected progression to second cohort expected during fourth quarter of 2020
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA Acetylcholine receptor antagonist; botulinum toxin A stimulator Upper limb spasticity Final participant enrolled in modified Juniper trial in adults following stroke or traumatic brain injury; screening ended early, with 76 participants enrolled and several still under evaluation, due to ongoing concerns related to COVID-19 pandemic; 73 individuals dosed before enrollment paused in March due to COVID-19 will be followed for up to 36 weeks, while those enrolled subsequently will be followed to week 12; top-line data expected in early 2021
Timber Pharmaceuticals Inc., of Woodcliff Lake, N.J. TMB-001 (isotretinoin, topical) Retinoic acid receptor agonist Congenital ichthyosis All 11 sites across U.S. and Australia open in phase IIb Control study, expected to enroll about 45 individuals with moderate to severe disease
Timber Pharmaceuticals Inc., of Woodcliff Lake, N.J. TMB-002 (rapamycin, topical) mTOR inhibitor Tuberous sclerosis complex 70% of sites open in phase IIb trial assessing treatment of individuals with facial angiofibromas; study expected to enroll 120 participants
Phase III
Biondvax Pharmaceuticals Ltd., of Jerusalem M-001  Non-flagellin-based, multimeric, multiepitope, single-protein vaccine Influenza virus infection Last of 12,400 participants completed final visit in universal flu vaccine pivotal trial; results expected by year-end 2020
Corcept Therapeutics Inc., of Menlo Park, Calif. Relacorilant Glucocorticoid receptor antagonist Pancreatic cancer First of 80 participants with metastatic disease enrolled in open-label Reliant combination trial with nab-paclitaxel (Abraxane, Celgene Corp.); interim analysis planned on data from first 40; primary endpoint is objective response rate, with secondary endpoints including progression-free survival and duration of response
Eyenovia Inc., of New York Micropine (atropine) Muscarinic receptor antagonist Myopia Enrollment resumed following temporary pause due to COVID-19 pandemic in Chaperone study expected to enroll >400 children aged 3 to 12 with progressive disease 
Ipsen SA, of Paris Onivyde (irinotecan liposome injection) + 5-FU/LV and oxaliplatin Encapsulated nanoliposomal formulation of irinotecan sucrosofate combo Metastatic pancreatic ductal adenocarcinoma Initiated Napoli-3 study comparing safety and efficacy of combo to gemcitabine + nab-paclitaxel in first-line setting 
Polypid Ltd., of Petah Tikva, Israel D‐PLEX100 Drug-device using controlled-release antibiotic  Post-abdominal surgical site infection First of up to 900 participants enrolled and randomized in Shield I trial assessing intervention plus standard of care (SOC) vs. SOC alone; primary endpoint is infection rate measured by proportion of those with at least 1 abdominal target incisional infection event within 30 days following abdominal surgery
Vasopharm GmbH, of Würzburg, Germany Ronopterin (VAS-203) Nitric oxide synthase inhibitor Traumatic brain injury Last of 223 participants completed last visit in pivotal European Nostra III trial; top-line data on schedule to report in fourth quarter of 2020

Notes

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