Edgewise reports ‘positive’ two-year topline results from ARCH trial

Edgewise Therapeutics announced positive two-year topline results from the ARCH trial. ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics and functional measures with sevasemten in adults with Becker. Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy. The ARCH trial evaluated sevasemten administered daily over two years in adults with Becker. Sevasemten was well-tolerated in all 12 participants with no discontinuations or dose reductions due to adverse events. Preserving NSAA functions that correlate to activities of daily living are important to individuals living with Becker. As seen in Figure 1, during two years of sevasemten treatment, participants’ NSAA scores stabilized and continued to diverge relative to functional declines reported across multiple Becker natural history studies, in which two-year mean decreases of 2.4 NSAA points were reported. In addition, significant decreases in key biomarkers of muscle damage including CK and TNNI2 were observed in participants treated with sevasemten, which are consistent with prior observations. “We are pleased by the promising and consistent functional results observed over two years of treatment with sevasemten, together with the favorable safety and tolerability profile,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We thank the Becker community for engaging with us on this promising therapy.”