Capricor Therapeutics Duchenne Treatment Shows Strong Heart And Muscle Results In Trial

Core Insights - Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is experiencing a significant rally following the release of topline results from the pivotal Phase 3 HOPE-3 trial for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) [1][2] Study Details - The HOPE-3 trial involved 106 randomized participants who received either intravenous Deramiocel at 150 million cells per infusion or a placebo every three months for 12 months [2] - The average age of participants was approximately 15 years [3] Efficacy Results - Nearly 54% of patients demonstrated a slowing of skeletal muscle disease progression, while around 91% showed a treatment effect on cardiomyopathy [3] - The study is noted as the first Phase 3 trial in a largely non-ambulatory DMD population to successfully meet its primary endpoint, indicating a significant impact on the development of innovative therapies [4] Clinical Significance - The preservation of left ventricular ejection fraction in patients treated with Deramiocel highlights its potential to address critical aspects of DMD [5] Regulatory Background - In July, Capricor Therapeutics received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, indicating that additional clinical data is required to meet the statutory effectiveness requirements [6] Market Reaction - Following the announcement, Capricor Therapeutics shares rose by 8.96%, reaching $6.93 during premarket trading [7]