Edgewise Announces Positive Long-Term Sevasemten Data Demonstrating Sustained Functional Stabilization in Becker Muscular Dystrophy Through 3.5 Years of Follow-Up

Core Insights - Edgewise Therapeutics presented long-term data from the MESA study showing that sevasemten leads to sustained functional stabilization in patients with Becker muscular dystrophy over 3.5 years, contrasting with expected functional decline from natural history studies [1] - The company is on track to report pivotal data from the GRAND CANYON placebo-controlled cohort in Q4 2026, which could lead to the first marketing application for sevasemten as a targeted therapy for Becker muscular dystrophy [1] Group 1: Study Findings - In the MESA study, participants on sevasemten showed a +0.1 improvement in NSAA scores over 3.5 years, compared to a -5.3 decline predicted from natural history data [1] - In the CANYON study, participants also showed a +0.1 improvement over 2 years, against a predicted decline of -2.9 [1] - Nearly all eligible participants (99%) from prior sevasemten studies chose to enroll in the MESA open-label extension study [1] Group 2: Drug Profile - Sevasemten is an investigational fast skeletal myosin inhibitor designed to protect muscle from contraction-induced damage, currently in late-stage clinical trials for Becker and Duchenne muscular dystrophy [1] - The drug has demonstrated a favorable safety profile with long-term exposure exceeding 3 years in the MESA study [1] Group 3: Company Overview - Edgewise Therapeutics focuses on developing novel therapeutics for muscular dystrophies and serious cardiac conditions, leveraging deep expertise in muscle physiology [1] - The company is also developing EDG-7500, a cardiac sarcomere modulator for hypertrophic cardiomyopathy, and EDG-15400 for heart failure, both in clinical development [1]