Edgewise Therapeutics, Inc., has announced positive two-year topline results from the ARCH open label study of sevasemten (EDG-5506) in adults with Becker. Sevasemten is an orally administered small molecule inhibitor designed to prevent contraction-induced muscle damage in dystrophinopathies, including Becker and Duchenne.

Results showed that during two years of sevasemten treatment, participants’ North Star Ambulatory Assessment (NSAA) scores stabilized, in contrast to functional declines reported across multiple Becker natural history studies. In addition, significant decreases in key biomarkers of muscle damage including Creatine Kinase (CK) and fast skeletal muscle troponin I (TNNI2) were observed. Sevasemten was also well-tolerated in all 12 participants with no discontinuations or dose reductions due to adverse events. 

Edgewise notes that these results support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies has the potential to preserve function.

PPMD is pleased to learn these encouraging results and we look forward to further updates from Edgewise.

Read Edgewise’s press release here.