FOSTER CITY, Calif. –Sep. 1, 2020– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases today announced that it has submitted the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter (ASBT) inhibitor being evaluated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum expects to complete the NDA submission in the first quarter of 2021.
Art Pappas will moderate a virtual fireside chat with Amy Abernethy, MD, Principal Deputy Commissioner, US Food & Drug Administration, on Wednesday, October 14, 2020 at 11:00 AM EDT.
Kyle Rasbach will serve on the panel “Evaluating investment opportunities in the rare disease & orphan space” at the World Orphan Drug Congress USA 2020.
4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of 4D-125 by Intravitreal Injection for the Treatment of X-Linked Retinitis Pigmentosa
EMERYVILLE, CA — 4D Molecular Therapeutics (4DMT), a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-125 for X-Linked Retinitis Pigmentosa (XLRP), a blinding and currently untreatable inherited retinal disease. 4D-125 is an AAV gene therapy with an optimized and proprietary vector designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina. This vector enables both administration by intravitreal injection, a safe and routine clinical route, plus the potential to treat broader and earlier-stage patient populations as compared to subretinal approaches. 4D-125 is a 4DMT-declared ophthalmology therapeutic candidate under 4DMT’s collaboration with Roche, where Roche has an option to take an exclusive world-wide license prior to initiation of a pivotal trial.
Amplyx Doses First Patient in Phase 2 Clinical Trial of Anti-BKV Monoclonal Antibody in Renal Transplant Recipients
SAN DIEGO, Aug. 17, 2020 — Amplyx Pharmaceuticals, a clinical-stage biopharmaceutical company developing innovative therapies for debilitating and life-threatening diseases in patients with compromised immune systems, today announced that the first patient has been dosed in its Phase 2 clinical trial evaluating the efficacy and safety of MAU868 for the treatment of BK viremia in kidney transplant recipients. MAU868 is a novel, human monoclonal antibody that potently neutralizes all four major genotypes of BK virus (BKV). BKV-associated nephropathy is a leading cause of kidney allograft loss.