Edgewise Therapeutics has announced the initiation of the CANYON Phase 2 clinical trial evaluating EDG-5506 in individuals with Becker muscular dystrophy (BMD). EDG-5506 is an orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibres in dystrophinopathies such as BMD and Duchenne muscular dystrophy (DMD).

CANYON will assess the effect of EDG-5506 over a 12-month period on safety, pharmacokinetics (PK), biomarkers such as creatine kinase (CK), and functional measures in individuals with BMD aged 12 years and above. This placebo-controlled trial is anticipated to recruit approximately 66 individuals at up to 14 sites in the United States, United Kingdom and the Netherlands.

“We are delighted to advance EDG-5506 into Phase 2, based on the safety and positive biomarker data observed in our Phase 1 and ARCH studies. In the CANYON trial, we aim to understand the effect of EDG-5506 on circulating biomarkers, muscle MRI, and functional measures, all of which could provide indications of a treatment effect.”

Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise

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About EDG-5506 for DMD and BMD

EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action designed 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 (FDA) granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.

The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate the safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center trial assesses the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). The company anticipates initiating a Phase 2 trial in individuals with DMD in the second half of 2022.