Core Insights - Capricor Therapeutics Inc's shares surged over 293% following the positive results from the Phase 3 HOPE-3 trial for its experimental cell therapy, Deramiocel, aimed at treating Duchenne muscular dystrophy (DMD) [1][9][12] Trial Overview - The HOPE-3 trial involved 106 participants across 20 U.S. centers, administering intravenous Deramiocel infusions of 150 million cells every three months over a 12-month period [3][10] - The average age of participants was approximately 15 years, with all participants continuing their standard corticosteroid therapy [4][11] Patient Condition and Results - At the trial's start, about 90% of participants were on cardiac medications, and over 75% had clinical cardiomyopathy [4][11] - Results indicated that nearly 54% of patients experienced a slowing of skeletal muscle disease progression, while around 91% showed improvement in heart function [6][11] Expert Commentary - Craig McDonald, an investigator in the trial, highlighted that this was the first Phase 3 trial in a largely non-ambulatory DMD population to meet its primary endpoint, marking a significant achievement for innovative therapy development [7][11] - Jonathan Soslow, a Professor of Pediatrics at Vanderbilt University, emphasized the significant preservation of left ventricular ejection fraction in patients treated with Deramiocel, underscoring its potential to address critical aspects of DMD [8][11] Regulatory Context - The positive trial results come after Capricor received a Complete Response Letter from the FDA for its previous Biologics License Application, which requested additional clinical data [8][11]
CAPR stock price: Why is Capricor Therapeutics stock surging over 293% today?