Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne Muscular Dystrophy (Duchenne) - Seite 2

“Having provided care for individuals with Duchenne for over 20 years, I’m excited to see Edgewise expand its clinical studies with EDG-5506 to include boys with Duchenne who are not currently treated with steroids as well as those who have received gene therapy and remain symptomatic,” said Barry J. Byrne, M.D., Ph.D., Director, Powell Gene Therapy Center, University of Florida and Chief Medical Adviser for MDA. “The patient community is desperate to find additional treatment options to curb the effects of this devastating disease.”

“We have listened to the Duchenne community and thoughtfully initiated FOX and expanded LYNX, both important advances in our EDG-5506 clinical development program. We anticipate moving towards a Phase 3 trial start in Duchenne in the second half of 2024,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We are thrilled to open our studies of EDG-5506 to broader groups of individuals with Duchenne exploring the potential of a muscle-targeted therapeutic against the evolving treatment landscape.”

About FOX Phase 2 Trial in Children with Duchenne

FOX is a Phase 2 placebo-controlled trial to assess the effect of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage in children and adolescents with Duchenne who have been previously treated with gene therapy. Approximately 24 participants, aged 6 to 14 years, are expected to be enrolled in the trial at multiple sites across the United States, which is expected to start in early 2024. 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. Go to clinicaltrials.gov to learn more about this trial (NCT06100887).

About LYNX Phase 2 Trial in Children with Duchenne

LYNX is a Phase 2 placebo-controlled trial to assess the effect of multiple doses of EDG-5506 over 12 weeks on safety, 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 45 children with Duchenne aged 4 to 9 years are expected to be enrolled in this trial. Participants will then continue in an open-label extension portion of the trial for a total of 24 months to gain further insights into safety and functional measures. Importantly, this trial is designed to identify a dose of EDG-5506 that will reduce biomarkers of muscle damage and has the potential to provide functional benefit to patients in a Phase 3 trial. Go to clinicaltrials.gov to learn more about this trial (NCT05540860).