Edgewise Therapeutics, Inc.

Edgewise discovers new medicines to improve the lives of people with serious muscle diseases. The company uses a proprietary, muscle-focused drug discovery platform that combines expertise in muscle biology and small molecule engineering. This platform utilizes custom-built high throughput and translatable systems to measure integrated muscle function in whole organ extracts. Its purpose is to identify small molecule precision medicines that regulate key proteins in muscle tissue, initially targeting rare neuromuscular and cardiac diseases. Edgewise has developed a library of novel sarcomere modulators with various pharmacological and pharmacokinetic properties that regulate disease-related muscle biology. The company is advancing multiple clinical-stage programs for muscular dystrophies and severe cardiac diseases, alongside several preclinical programs. Its muscular dystrophy program includes sevasemten, an orally administered allosteric, selective, fast myofiber (type II) myosin small molecule inhibitor. Sevasemten is designed to address contraction-induced muscle injury, which is the root cause of dystrophinopathies such as Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker). These are rare and often debilitating diseases. Edgewise estimates approximately 35,000 individuals live with Duchenne and 12,000 individuals live with Becker in the US, EU-5, and Japan. There are currently no approved therapies for individuals with Becker. Sevasemten is designed to selectively limit injurious stress caused by the lack of dystrophin by moderating fast skeletal muscle myosin force development, thereby compensating for the absence of functional dystrophin.