Fulcrum Therapeutics Enrolls First Patient in Pivotal Global Phase 3 Clinical Trial of Losmapimod for Facioscapulohumeral Muscular Dystrophy (FSHD)

– REACH is the first Phase 3 trial for this rare, progressive, and debilitating muscular disease –
– Losmapimod is an oral small molecule that has the potential to be the first therapy to treat FSHD, the second most common form of muscular dystrophy –
– U.S. Food and Drug Administration (FDA) granted Fast Track Designation in 2021 –

Excerpt from the Press Release:

CAMBRIDGE, Mass., July 05, 2022 (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 first patient has been dosed in REACH, a Phase 3 clinical trial designed to evaluate the safety and efficacy of losmapimod for the treatment of FSHD. The randomized, double-blind, placebo-controlled, multi-national Phase 3 trial will enroll approximately 230 adults at over 30 sites in North America and Europe. The pivotal trial is designed to support the U.S. FDA and European Medicines Agency (EMA) regulatory applications of lospmapimod for the treatment of FSHD. If approved, losmapimod would be the first and only therapy for the disease.

FSHD is a rare, serious, progressive, and debilitating disease that is caused by the aberrant expression of the DUX4 gene, which leads to skeletal muscle cell death and fat infiltration. Patients with FSHD experience progressive muscle weakness leading to significant impairment in function, including the inability to use their upper limbs, communicate via facial expression, and walk unassisted.

“The progression of FSHD can make it increasingly difficult to pursue work, hobbies, social and family life,” said Mark Stone, CEO of the FSHD Society.

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