US-based biotechnology company Edgewise Therapeutics has reported positive two-year topline data from the ARCH clinical trial of sevasemten (EDG-5506) in adults with Becker muscular dystrophy.

Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies such as Becker and Duchenne muscular dystrophy.

The open-label, single-centre trial evaluated the drug’s safety, pharmacokinetics (PK) and tolerability.

The trial also assessed various other outcomes, including changes in biomarkers of muscle damage such as creatine kinase (CK) and fast skeletal muscle troponin I, measures of function and subject-reported outcomes.

It involved 12 adult males with Becker muscular dystrophy, who were administered sevasemten daily over two years.

According to the findings, sevasemten was found to be well-tolerated in all participants without any discontinuations or dose reductions due to adverse events.

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The trial also focused on the North Star Ambulatory Assessment (NSAA) functions, which are crucial for daily living activities for individuals with Becker.

Participants’ NSAA scores remained stable over the two-year treatment period with sevasemten, in contrast with the functional declines observed in multiple Becker natural history studies, where mean decreases of 2.4 NSAA points are typically reported over two years.

In addition, participants treated with sevasemten exhibited significant decreases in key biomarkers of muscle damage, including CK and fast skeletal muscle troponin I (TNNI2).

These results demonstrated that the sevasemten treatment to reduce contraction-induced muscle damage could preserve function and potentially halt disease progression.

Edgewise chief medical officer Joanne Donovan said: “We are pleased by the promising and consistent functional results observed over two years of treatment with sevasemten, together with the favourable safety and tolerability profile.

“We thank the Becker community for engaging with us on this promising therapy.”

In September last year, Edgewise began dosing healthy adults in a Phase I study of EDG-7500 to treat hypertrophic cardiomyopathy and other diseases of cardiac diastolic dysfunction.