EDG-5506

EDG-5506 is a small molecule designed to halt the damage in a broad range of rare skeletal muscle disorders, including Duchenne and Becker. EDG-5506 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.

EDG-5506 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 EDG-5506 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 EDG-5506 demonstrated improvements in cardiac fibrosis and hypertrophy. To learn more about how EDG-5506 might work in Duchenne and Becker, watch our video here.

Edgewise is advancing a set of clinical trials of EDG-5506 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 EDG-5506, click here.