Therapeutic Potential of EDG-5506 in Treating Becker Muscular Dystrophy: Joanne Donovan, MD, PhD

WATCH TIME: 6 minutes

"EDG-5506 is a promising therapy for Becker Muscular Dystrophy, as it focuses on reducing muscle stress during physical activity, which is key in preserving muscle strength and slowing the progression of the disease. The results so far have been highly encouraging, with reductions in muscle damage biomarkers and physical improvements seen in patients."

Becker muscular dystrophy (BMD) is caused by mutations in the dystrophin gene, which leads to reduced production of the dystrophin protein, which is critical for maintaining muscle cell structure and function. It is inherited in an x-linked recessive pattern, meaning it primarily affects males. Currently, there are no FDA-approved therapies considered disease-modifying, rather only symptomatic strategies, such as physical therapy, to try to improve quality of life.

One agent in development, Edgewide Therapeutics’ EDG-5506, is currently being investigated in a phase 2 trial, dubbed GRAND CANYON (NCT05291091), which includes 120 patients with BMD. The study, an 18-month trial further assessing efficacy and safety of the agent, includes ambulatory patients with a mutation in the DMD gene with Becker phenotype who have a North Star Ambulatory Assessment (NSAA) score between 5-32. GRAND CANYON will last a total of up to 20 months, with a screening period of up to 4 weeks and a 4-week follow-up period after the 18-month treatment period.

In a recent interview with NeurologyLive^®, Joanne Donovan, MD, PhD, chief medical officer at Edgewise, discussed the mechanism of action of EDG-5506 and its potential in treating BMD. She noted that unlike therapies targeting specific genetic mutations, EDG-5506 focuses on the muscle’s sarcomere to protect the most vulnerable muscle fibers. In addition, she commented on how initial trials, such as the ARCH study (NCT05160415), sparked hope for future trials like the GRAND CANYON study. Furthermore, she gave thoughts on the lessons learned from ARCH, the promising results on EDG-5506 early on, and the need for continued awareness toward BMD going forward.