Edgewise Therapeutics has announced an expansion of their clinical program of EDG-5506 in Duchenne muscular dystrophy. They are initiating FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy.
FOX will assess the effect of EDG-5506 over 12 weeks on safety, pharmacokinetics (PK) and biomarkers of muscle damage. The study will also explore changes in functional measures, such as the North Star Ambulatory Assessment (NSAA) and self-reported/caregiver-reported outcomes. Approximately 24 participants, aged 6 to 14 years, are expected to be enrolled in the trial at multiple sites across the US. Participants will then continue in an open-label extension portion of the trial for a total of 12 months to gain further insights into safety, PK, function and biomarker measures.
Additionally, in the LYNX trial, Edgewise is continuing dose escalation and initiating a cohort of boys with Duchenne who are not currently treated with corticosteroids. These advances have resulted from thoughtfully listening to the Duchenne community, and they anticipate moving towards a Phase 3 trial in Duchenne in the second half of 2024.
Hear more from Edgewise Therapeutics at our national annual education conference for Canadians living with Duchenne and Becker muscular dystrophy – the Defeat Duchenne Canada Family Forum from November 4-5, 2023: defeatduchenne.ca/familyforum.