Company Product Description Indication Status
Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion intra-articular injection Stem cell antigen-1 inhibitor Severe osteoarthritis of the knee Received a positive response from FDA on plans for its AP-013 phase III trial; FDA's response provides guidance and flexibility on how to maintain the special protocol assessment; the study was paused in early 2020 because of the COVID-19 pandemic
Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion intra-articular injection Stem cell antigen-1 inhibitor Respiratory symptoms experienced by COVID-19 long haulers (post-acute sequelae of SARS-CoV-2) Phase I study is awaiting IRB approval and is expected to commence enrollment shortly
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo (hydroxypropyl beta cyclodextrin) Lipid metabolism modulator; cholesterol binding agent  Niemann-Pick disease type C1  Announced the design of its phase III study; FDA and EMA are in agreement with the protocol and have given clearance to proceed with the study
Eledon Pharmaceuticals Inc., of Irvine, Calif. AT-1501 Monoclonal antibody targeting CD40L Renal transplantation FDA requested that Eledon provide renal transplant data in nonhuman primates prior to initiating a phase II trial, so Eledon plans to initiate an evaluation of AT-1501 in a standard nonhuman primate model of renal transplantation, which is expected to be completed by late 2022; the company will continue to explore potentially conducting a trial outside the U.S.
Enzyvant Therapeutics Inc., of Cambridge, Mass. RVT-802 Cultured human thymus tissue Pediatric congenital athymia Resubmitted BLA to FDA; FDA had made several regulatory requests related to chemistry, manufacturing and controls in a December 2019 complete response letter that followed the initial BLA submission in April 2019; Enzyvant has been working to address each of the FDA requests and the expected PDUFA date is Oct. 8, 2021
Hillhurst Biopharmaceuticals Inc., of San Diego HBI-002 Oral carbon monoxide therapeutic Sickle cell disease Received a study may proceed letter from FDA to initiate a phase I trial 
Instil Bio Inc., of Dallas ITIL-168 Autologous cell therapy made from tumor infiltrating lymphocytes Melanoma stages IIb to IV FDA granted orphan drug designation
Krystal Biotech Inc., of Pittsburgh Beremagene geperpavec (B-VEC) COL7A1 gene stimulator Dystrophic epidermolysis bullosa Announced modifications to the statistical analysis plan in the ongoing phase III study based on feedback from FDA; to account for paired binary data in the study design and the within-pair correlation in the GEM-3 study, Krystal will use the Mcnemar test for primary efficacy analysis and the primary outcome measure is complete wound healing in B-VEC-treated wounds vs. placebo
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Orally administered activator of Nrf2 Chronic kidney disease caused by Alport syndrome FDA accepted for filing the NDA, and the PDUFA date is scheduled for Feb. 25, 2022
Revive Therapeutics Ltd., of Toronto Psilocybin Psilocybe mushroom product Moderate to severe traumatic brain injury Filed for FDA orphan drug designation 
Rockwell Medical Inc., of Wixom, Mich. Triferic Avnu (ferric pyrophosphate citrate) Iron replacement Hemodialysis-dependent chronic kidney disease Received notice of marketing approval from Health Canada 
Russian Direct Investment Fund, of Moscow Sputnik V Adenovirus-based vaccine COVID-19 prophylaxis Approved in Bangladesh
Russian Direct Investment Fund, of Moscow Sputnik V Adenovirus-based vaccine COVID-19 prophylaxis Brazil’s Anvisa said its collegiate board (Dicol) unanimously decided not to authorize the exceptional import of the vaccine; decision based on data collected and evaluated 
Sol-Gel Technologies Ltd., of Ness Ziona, Israel Epsolay (benzoyl peroxide) 5% topical cream Microencapsulated benzoyl peroxide cream Inflammatory lesions of rosacea Announced an update regarding the FDA approval process and said the final content of the labeling was discussed and agreed to; received email confirmation that the action on the NDA could not be taken by April 26, 2021, PDUFA date, since a pre-approval inspection of the production site still needs to be conducted

Notes

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