Edgewise Therapeutics (EWTX) said Tuesday its experimental drug, sevasemten, maintained patient function for up to 3.5 years in an open-label extension study for Becker muscular dystrophy, a disorder with no approved therapies.
The company said the study showed that North Star Ambulatory Assessment scores remained stable in patients previously treated in clinical trials, contrasting with the typical annual decline seen in the disease's natural progression.
Edgewise said it expects to release top-line results from the placebo-controlled cohort in Q4, adding if positive, the company plans to pursue regulatory approval for sevasemten as the first targeted therapy for this underserved population.