Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today 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).

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Figure 1: NSAA stabilized with sevasemten treatment and continues to diverge from natural history studies at 2 years (Photo: Business Wire)

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^{1, 2,3}.

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.”

“Becker is a devastating neuromuscular disease that currently has no treatment,” said Barry J. Byrne, M.D., Ph.D., Director, Powell Gene Therapy Center, University of Florida and Chief Medical Adviser for MDA. “Once symptoms of muscle weakness occur, individuals with Becker have a relentless course of disease progression. In my experience, each one-point decrease in NSAA represents a loss of function important to daily activities. I have been closely following the progress of sevasemten in the clinic and am encouraged by the two-year results and the potential of this novel muscle-targeted therapy for individuals with Becker.”