Edgewise Therapeutics, Inc. 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 (PK) and functional measures with sevasemten (EDG-5506) 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 (Duchenne).

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.

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.

The positive results from the two-year ARCH trial further support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies has the potential to preserve function and halt disease progression in Becker.