Core Insights - Edgewise Therapeutics, Inc. announced positive topline results from the Phase 2 CANYON trial of sevasemten for Becker muscular dystrophy (BMD), marking it as the largest interventional trial in this area and the first to meet its primary endpoint [1][2] Group 1: Trial Results - The trial met its primary endpoint with a significant 28% average decrease in creatine kinase (CK) levels in the sevasemten-treated group compared to placebo over months 6 through 12 (p=0.02) [2] - Secondary endpoint, North Star Ambulatory Assessment (NSAA), indicated a trend towards improvement in motor function in the sevasemten-treated group [2] - Plasma fast skeletal muscle troponin I (TNNI2) showed a significant reduction in the treatment group compared to placebo, indicating muscle damage reduction [3] Group 2: Functional Measures - Additional functional measures, including the 10-meter walk/run, 4-stair climb, and 100-meter timed test, also showed trends towards improvement in the sevasemten group compared to placebo [3] Group 3: Patient Population and Safety - The treatment population had more advanced disease compared to the placebo group, suggesting a challenging patient demographic [4] - Sevasemten was well-tolerated with no new safety concerns reported during the trial [5] Group 4: Future Plans and Market Reaction - The company is on track to complete recruitment for the GRAND CANYON cohort by the first quarter of 2025 [6] - Following the announcement, EWTX stock rose by 8.62% to $30 [7]
Edgewise Therapeutics' Lead Candidate Reduces Biomarker Levels In Mid-Stage Study In Patients With Muscle Weakness Disease