Fulcrum Starts Pivotal Global Phase 3 Trial of Losmapimod for Facioscapulohumeral Muscular Dystrophy

 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.

https://finance.yahoo.com/news/fulcrum-therapeutics-enrolls-first-patient-110000087.html