Capricor’s Deramiocel Shows Long-Term Benefits in Duchenne Muscular Dystrophy

Capricor Therapeutics has unveiled promising long-term data from its HOPE-2 open-label extension (OLE) clinical trial, showing that its lead therapy, deramiocel, significantly slows disease progression in Duchenne muscular dystrophy (DMD). Presented at the Muscular Dystrophy Association Clinical and Scientific Conference, the study revealed a 52% reduction in disease progression compared to external controls. Over three years, patients treated with deramiocel experienced a decline of 3.46 points in the Performance of the Upper Limb (PUL 2.0) score, compared to a 7.19-point decline in untreated patients. The treatment effect increased yearly, with slowing progression rates and sustained muscle function, reinforcing deramiocel’s potential as a disease-modifying therapy.

Capricor CEO Linda Marbán, Ph.D., emphasized the therapy’s transformative impact, stating, "For patients and families battling DMD, time is muscle. This data reinforces that deramiocel is not just slowing the disease—it is rewriting the trajectory of what’s possible." The company also announced that the FDA has accepted its Biologics License Application (BLA) for the cardiomyopathy associated with DMD, with a target action date set for August 31, 2025. If approved, deramiocel could provide new hope for those living with this devastating disease.