Defeat Duchenne Canada is pleased to share the news that Edgewise Therapeutics has announced positive topline results from the EDG-5506 Phase 1b clinical trial in adults with Becker muscular dystrophy (BMD).

“The completion of this clinical trial in BMD adults is an important milestone for Edgewise. We’re excited as we advance into Phase 2 clinical trials in BMD and DMD,” said their Chief Medical Officer, Joanne Donovan, M.D., Ph.D.

Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise said “We expect EDG-5506 treatment to lead to reduced muscle damage, the root cause of dystrophinopathies, and ultimately improve function in individuals with BMD and DMD.”

Edgewise Phase 1b study in Becker is currently recruiting in Atlanta, Georgia.Click the button below to learn more about the clinical trial:

We look forward to more progress coming from Edgewise. In the meantime, you can read the complete press release here:

About EDG-5506 for DMD and BMD

EDG-5506 is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.

EDG-5506 has been studied in a Phase 1 clinical trial designed to evaluate safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b).