The small molecule myosin modulator, EDG-5506 can be administered orally and has been designed to offer protection to injury-susceptible fast skeletal muscle fibers in BMD and Duchenne muscular dystrophy (DMD).
At present, no approved treatments are available for BMD.
The placebo-controlled CANYON trial is expected to enrol around 66 subjects at up to 14 study sites in the UK, US and the Netherlands.
In this trial, the effect of EDG-5506 will be evaluated for 12 months on pharmacokinetics (PK), safety, biomarkers such as creatine kinase (CK), and functional measures in BMD individuals aged 12 years and above.
Edgewise Therapeutics chief medical officer Joanne Donovan said: “We are delighted to advance EDG-5506 into Phase II, based on the safety and positive biomarker data observed in our Phase I 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.”
The US Food and Drug Administration granted Fast Track designation to EDG-5506 in August last year to treat individuals with BMD.
EDG-5506, which is anticipated to be used as a monotherapy, exhibits a new mechanism of action designed to selectively restrict the exaggerated muscle damage that is caused due to the absence of functional dystrophin.
BMD is a progressively debilitating, serious as well as potentially fatal inherited X-linked neuromuscular disorder and is also linked to early mortality from cardiac disease.