Press release
Fulcrum Therapeutics Announces U.S. Food and Drug Administration Grants Fast Track Designation to Losmapimod for the Potential Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy (FSHD). “There are no approved therapies to treat patients with FSHD, and losmapimod is currently the only drug in clinical development for this serious and debilitating disease,” said Judith Dunn, Ph.D., Fulcrum’s president of research and development. “We are pleased that the FDA has granted Fast Track designation, which we believe demonstrates the potential for losmapimod to address unmet medical needs for people living with FSHD.” Fast Track Designation is intended to facilitate development and expedite the review of drugs that treat serious conditions so an approved product can reach the market expeditiously. It enables the company to have more frequent interactions with the FDA throughout the drug development process and allows for eligibility for priority review and accelerated approval in certain cases, as well as a rolling review. Fulcrum is on track to report full data from ReDUX4, a Phase 2b randomized, double-blind, placebo-controlled trial of losmapimod in FSHD patients, at the virtual FSHD International Research Congress taking place June 24-25, 2021. Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression. Additional data to be reported include secondary endpoints evaluating disease progression via skeletal muscle MRI, exploratory endpoints assessing muscle function measures and patient reported outcomes. Losmapimod previously received Orphan Drug Designation for FSHD. About LosmapimodLosmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of...