Press release
Fulcrum Therapeutics Announces Publication of Results from Phase 2b Clinical Trial of Losmapimod in Facioscapulohumeral muscular dystrophy (ReDUX4) in The Lancet Neurology
― Phase 2b ReDUX4 clinical trial indicates improvements in functional, structural, and patient-reported outcomes; findings informed the design and choice of
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"― Phase 2b ReDUX4 clinical trial indicates improvements in functional, structural, and patient-reported outcomes; findings informed the design and choice of efficacy endpoints in the ongoing Phase 3 clinical trial― ― Topline data for the Phase 3 REACH clinical trial of losmapimod in FSHD expected during 4Q’24― CAMBRIDGE, Mass., May 08, 2024 (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 the publication of results from its Phase 2b clinical trial of losmapimod for the treatment of facioscapulopumeral muscular dystrophy (FSHD). The data are published in the peer-reviewed journal The Lancet Neurology. FSHD is a serious, rare, and progressively disabling disease characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, shoulder girdle, upper arms, abdomen, and lower limbs. It is caused by the aberrant expression of the DUX4 protein. It is characterized by accumulating muscle weakness and functional impairment. There are no approved disease-modifying treatments for FSHD. “The publication of these results, which informed the design and choice of efficacy endpoints in our Phase 3 clinical trial, also provide important validation for the therapeutic potential of losmapimod,\" said Patrick Horn M.D., Ph.D., Fulcrum’s chief medical officer. \"Looking ahead, we remain on track to report topline data for REACH in the fourth quarter of 2024, which will bring us one step closer to addressing the high unmet needs of the FSHD patient community. With an unwavering commitment to our patients, we continue to take important steps toward a potential NDA filing and commercial launch for losmapimod.” While the primary endpoint of change in DUX4-driven gene expression in muscle biopsies did not show significant differences between the treatment and placebo groups, losmapimod was associated with improvements in structural and functional outcomes, including muscle fat infiltration and reachable workspace – a measure of shoulder girdle function, and patient-reported global impression of change when compared to placebo. Losmapimod was also found to be well tolerated by trial participants. No serious adverse events related to the drug were reported,...