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

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne. The Company is initiating FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy. Further, the Company is continuing dose escalation and expanding enrollment in their Phase 2 placebo-controlled LYNX trial; one of the new LYNX cohorts will study EDG-5506 in boys with Duchenne not currently treated with corticosteroids.

With strong interest from the Duchenne community and supportive initial safety data from LYNX, the Company initiated the FOX trial, a new Phase 2 placebo-controlled trial in children and adolescents with Duchenne who have been previously treated with gene therapy. The FOX trial 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 United States. 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.

The LYNX trial in children with Duchenne has rapidly enrolled at 14 sites across the United States, with the first three cohorts over-enrolled. Based on the safety profile observed to date, the Company added an additional cohort to continue dose escalation of EDG-5506. LYNX 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. The Company expects to report Phase 2 interim data in the first half of 2024, with the Phase 3 dose identified. Additionally, the Company is adding a new cohort to LYNX to include children aged 4 to 9 years with Duchenne who are not currently treated with corticosteroids. The Company also extended the open label portion of the trial for a total of 24 months.