Edgewise Therapeutics Reports Positive Phase 2 Sevasemten Trial Results for Becker Muscular Dystrophy

Edgewise Therapeutics, Inc., a prominent biopharmaceutical company specializing in muscle diseases, has announced promising results from its Phase 2 CANYON trial. The study focused on the efficacy of sevasemten, a pioneering oral treatment designed to mitigate muscle damage in individuals with Becker muscular dystrophy. This trial is noted as the largest interventional study for Becker muscular dystrophy and the first to successfully meet its primary endpoint.

The primary goal evaluated was the change from baseline in creatine kinase (CK) levels, an enzyme indicative of muscle damage. The results revealed a significant reduction in CK levels in patients treated with sevasemten compared to those receiving a placebo. Specifically, there was an average decrease of 28% in CK levels over a six to twelve-month period in the sevasemten group, highlighting the drug's potential effectiveness.

Additionally, the trial assessed the North Star Ambulatory Assessment (NSAA) as a key secondary endpoint. This functional scale showed a trend towards improvement in the sevasemten group over time. Although a statistically significant difference was not achieved, the NSAA scores remained stable in the treatment group, contrasting with the decline observed in the placebo group.

Further secondary endpoints included plasma fast skeletal muscle troponin I (TNNI2) levels, which significantly decreased by 77% in the sevasemten group compared to placebo. Functional assessments, such as the 10-meter walk/run, 4-stair climb, and 100-meter timed test, also indicated trends towards improvement in the sevasemten-treated participants.

Importantly, sevasemten was well-tolerated with no new safety concerns arising in either adult or adolescent participants. The study enrolled a total of 69 individuals, comprising 40 adults and 29 adolescents, with a randomization ratio of 3:1 for adults and 2:1 for adolescents in favor of sevasemten.

Edgewise is also conducting an open-label extension trial named MESA, in which 99% of eligible participants from the CANYON and other sevasemten trials have enrolled. This underscores the continued interest and optimism surrounding sevasemten's potential benefits.

Experts involved in the study, like Dr. Craig M. McDonald from UC Davis Health, expressed encouragement about the findings. He emphasized the trial's significance given the lack of approved therapies for Becker muscular dystrophy and highlighted the potential of sevasemten to stabilize muscle function.

Joanne Donovan, Chief Medical Officer at Edgewise, conveyed enthusiasm about the CANYON results, noting the consistency of these findings with previous studies, such as the ARCH trial. She reiterated the potential of sevasemten to preserve muscle function in patients with Becker muscular dystrophy.

Looking ahead, Edgewise plans to complete recruitment for the GRAND CANYON cohort by the first quarter of 2025. Based on the positive outcomes of the Phase 2 trial, the company intends to engage with regulatory authorities in the U.S. and Europe to discuss marketing authorization strategies for sevasemten.

Sevasemten, also known as EDG-5506, has already achieved several regulatory milestones, including Orphan Drug Designation from both the U.S. Food and Drug Administration and the European Medicines Agency. These designations further bolster the potential of sevasemten as a transformative treatment for Becker and Duchenne muscular dystrophies.

The CANYON study's promising data will be submitted for publication at an upcoming medical congress, marking another step forward in the journey to potentially providing new therapeutic options for individuals suffering from these debilitating neuromuscular disorders.