Edgewise Therapeutics has shared its winter newsletter for the Duchenne and Becker muscular dystrophy communities, including the following updates:
Edgewise Initiates GRAND CANYON, a Global Study of EDG-5506 in Becker Muscular Dystrophy (Becker)
They recently announced the start of enrollment of GRAND CANYON, a global study of EDG-5506 in individuals with Becker. GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. EDG-5506 is orally administered and is designed to prevent contraction-induced muscle damage, which we hypothesize will prevent muscle damage or possibly improve muscle function and delay the progression of disease. This will be evaluated using the North Star Ambulatory Assessment in addition to other functional assessments. This trial is anticipated to recruit approximately 120 ambulatory adults with Becker between the ages of 18 and 50 years old at up to 50 sites in 10 countries. The treatment period for participants will be 18 months.
They also presented 12-month results from the ARCH open-label study of EDG-5506 in adults with Becker. The ARCH study has been evaluating different doses of EDG-5506 administered daily in 12 adults with Becker. After 12 months, there were significant decreases in biomarkers of muscle damage and a trend toward improvement in the North Star Ambulatory Assessment score, compared to a predicted decline from natural history studies. EDG-5506 was well-tolerated, with no one reducing dose or discontinuing use due to adverse events.
Edgewise Expands the EDG-5506 Clinical Program in Duchenne muscular dystrophy (Duchenne)
Earlier this fall, Edgewise announced FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy. This study will enroll approximately 24 participants between the ages of 6 and 14 in multiple states across the U.S. Participants will then continue in an open-label extension for a total of 12 months. The study will examine safety, changes in biomarkers of muscle damage, changes in functional measures, such as the North Star Ambulatory Assessment, and self-reported/caregiver-reported outcomes.