Edgewise Therapeutics, Inc. (EWTX), a biopharmaceutical firm specializing in muscle diseases, announced encouraging topline results on Monday from their Phase 2 CANYON trial of sevasemten, a treatment for Becker muscular dystrophy.
Sevasemten is an innovative, orally administered fast skeletal myosin inhibitor, aimed at protecting muscle tissue from damage caused by contractions in individuals suffering from muscular dystrophies.
The CANYON trial achieved its primary goal by demonstrating a reduction in circulating creatine kinase (CK) levels—a biomarker indicative of skeletal muscle damage. Notably, this is the largest interventional study conducted for Becker muscular dystrophy and the first to meet its primary endpoint successfully.
Regarding the key secondary endpoint, patients treated with sevasemten exhibited stabilization in the North Star Ambulatory Assessment (NSAA), with a positive trend towards improvement after 12 months when compared to the placebo group.
Additionally, sevasemten was well-tolerated with no new safety concerns identified during the trial.
