Edgewise Therapeutics’ sevasemten shows efficacy in Becker disorder

Edgewise Therapeutics (EWTX) presented long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy, a genetic disorder with no approved therapeutic interventions. The data show participants on sevasemten experienced stabilization of function over 3.5 years in marked contrast to the functional decline expected from Becker natural history data. Key findings reported after 12 months of ARCH and CANYON participation and further follow-ups in MESA: sustained stabilization over 3.5 years; divergence from Becker natural history. Sevasemten continued to show a favorable safety profile in MESA with long-term exposure for 3+ years. The company expects to announce top-line results of the sevasemten placebo-controlled pivotal cohort, GRAND CANYON, in 4Q. If data are positive, the company will advance sevasemten toward a marketing application to seek approval as the first targeted therapy for this underserved population.