Sevasemten

Sevasemten is a small molecule designed to halt the damage in a broad range of rare skeletal muscle disorders, including Duchenne and Becker. Sevasemten presents a novel mechanism of action to selectively reduce injurious mechanical stress caused by the absence of functional dystrophin, allowing a normal, healthy range of muscle contraction. We believe this intervention will limit other negative events, including inflammation, muscle degeneration, exhaustion of the regenerative process, fibrosis, adipogenesis and loss of function. Our goal is to ultimately provide enhanced function due to improved muscle health.

Sevasemten has been characterized in a comprehensive set of short- and long-term preclinical studies of muscular dystrophy, looking at muscle function and fibrosis. Treatment with sevasemten in these studies was shown to protect muscle fibers from injury; with longer term treatment, reduced muscle wasting and fibrosis resulted in improvements in strength. Additionally, treatment with sevasemten demonstrated improvements in cardiac fibrosis and hypertrophy. To learn more about how sevasemten might work in Duchenne and Becker, watch our video here.

Edgewise is advancing a set of clinical trials of sevasemten to determine if protection of fast fibers improves muscle health while preventing further muscle degeneration and slowing disease progression in individuals with Duchenne and Becker.  For more information on our clinical trials for sevasemten, click here.