Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy

Core Insights - Capricor Therapeutics presented positive long-term data from its HOPE-2 clinical trial, showing that deramiocel can significantly slow disease progression in patients with Duchenne muscular dystrophy (DMD) [1][2] - The data indicates a 52% reduction in disease progression, with an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points over three years for treated patients compared to 7.19 points in the external comparator group [2] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead asset [1][8] - The company has received FDA acceptance for its Biologics License Application (BLA) for deramiocel, targeting cardiomyopathy associated with DMD, with a PDUFA action date set for August 31, 2025 [2][10] Treatment Efficacy - Patients treated with deramiocel showed a mean annual decline in PUL 2.0 scores of 1.8 points in Year 1, 1.2 points in Year 2, and 1.1 points in Year 3, indicating a year-over-year improvement in treatment effect [6] - During a one-year gap in treatment, patients originally on deramiocel experienced a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year) [6] Safety Profile - Deramiocel has demonstrated a favorable safety profile, being well tolerated with no new safety signals identified [6] Background on DMD - Duchenne muscular dystrophy is a severe genetic disorder leading to progressive muscle weakness and has a median mortality age of approximately 30 years, affecting about 15,000-20,000 individuals in the U.S. [5][7]