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Edgewise Therapeutics

Edgewise Therapeutics: Initiation of GRAND CANYON, a Global Study of EDG-5506 in Becker Muscular Dystrophy

October 2, 2023

Edgewise Therapeutics has announced the start of enrollment of GRAND CANYON, a global study of EDG-5506 in individuals with Becker muscular dystrophy. GRAND CANYON is an expansion of the CANYON study which was over-enrolled and includes 39 adults and 24 adolescents.

What is EDG-5506?

EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Becker and Duchenne muscular dystrophy. There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need.

What is the Grand Canyon Study?

GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. Data from GRAND CANYON, if positive, could support a marketing application. The primary endpoint of GRAND CANYON is the North Star Ambulatory Assessment (NSAA). In addition, other functional assessments, biomarkers of muscle damage and safety will be assessed. GRAND CANYON is anticipated to recruit approximately 120 individuals with Becker, aged between 18 and 50 years old, at up to 50 sites in 10 countries. The treatment period for participants will be 18 months.

“Based on the strength of safety, functional and biomarker results from our ARCH open label study, we have rapidly initiated this ground-breaking clinical study. This study is critical for the Becker community, as there currently are no approved treatments for Becker, a condition that for far too long has been neglected.”

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

“It is exciting to move to the next stage of testing of the clinical hypothesis that a reduction in contraction-induced muscle damage has the potential to benefit people living with muscular dystrophies. Having seen promising preclinical results with EDG-5506 translate clinically, I am thrilled we are entering into a pivotal study.”

Alan Russell, Ph.D., Co-Founder and Chief Scientific Officer