Capricor Therapeutics announced positive three-year data from its open-label extension of the HOPE-2 clinical trial for deramiocel, a cell-based therapy for Duchenne muscular dystrophy (DMD). The trial demonstrated a 52% slower decline in upper limb function for patients treated with deramiocel compared to an external comparator group. This positive data was presented at the 2025 Muscular Dystrophy Association Conference.
This news holds significant promise for the DMD community. Current treatment options for DMD are limited, and the disease inevitably leads to progressive muscle degeneration and premature death. Deramiocel’s demonstrated ability to not only slow but potentially modify the disease course offers a new level of hope for improving patients’ quality of life and extending lifespan. The increasing treatment effect observed year over year, coupled with a favorable safety profile, further underscores deramiocel’s therapeutic potential.
The Performance of the Upper Limb (PUL 2.0) score, a key measure of disease progression in DMD, declined by an average of 3.46 points over three years in deramiocel-treated patients, compared to a 7.19-point decline in the comparator group. Notably, even during a one-year treatment gap, patients previously treated with deramiocel maintained a slower rate of decline than untreated patients, suggesting a potential disease-modifying effect. Furthermore, the annual decline in PUL 2.0 scores lessened each year for deramiocel patients (1.8 points in Year 1, 1.2 points in Year 2, and 1.1 points in Year 3), indicating accumulating benefits over time.
This long-term data reinforces the potential of deramiocel to become a transformative therapy for DMD. With the FDA having accepted Capricor’s Biologics License Application for deramiocel for DMD-associated cardiomyopathy, and a PDUFA date set for August 2025, the future of DMD treatment appears promising. Positive results from the BLA review could lead to a significant advancement in DMD care, offering patients a new therapeutic option that may fundamentally alter the disease’s progression. Further research and development of deramiocel, including exploring its application to other aspects of DMD, will be crucial for realizing its full therapeutic potential.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
