Capricor Therapeutics announced that its first-in-class cell therapy candidate, deramiocel, met both the primary and key secondary endpoints in the pivotal Phase III HOPE-3 trial for Duchenne muscular dystrophy (DMD). The news triggered a 371% surge in the company's share price.

DMD is a severe, progressive genetic disorder caused by a lack of the dystrophin protein, leading to degeneration of skeletal, respiratory and cardiac muscle, with cardiomyopathy being the leading cause of death. Deramiocel consists of allogeneic cardiosphere-derived cells, which are believed to exert immunomodulatory, antifibrotic and regenerative effects via secreted exosomes.

The HOPE-3 study enrolled 106 non-ambulatory or late-stage ambulatory boys and young men with DMD. After 12 months of treatment, the deramiocel group showed a 54% slowing in the decline of upper limb function (the primary endpoint) and a 91% slowing in the decline of cardiac function (a key secondary endpoint) compared to the placebo group, with both results achieving statistical significance. Deramiocel demonstrated a favourable safety profile consistent with prior studies. If approved, it would be the first therapy specifically indicated for DMD-associated cardiomyopathy.

PharmCube's NextBiopharm® database shows that deramiocel is under development in eight more indications. Click here to request a free trial for NextBiopharm®.