Capricor Therapeutics (CAPR) Study Result summary

Study background and design

• HOPE-3 is a phase III, randomized, double-blind, placebo-controlled trial evaluating Deramiocel for Duchenne muscular dystrophy (DMD).

• 106 patients were randomized; 105 received treatment, with balanced demographics and most being non-ambulatory.

• The primary endpoint was the Performance of the Upper Limb (PUL) 2.0; key secondary endpoints included cardiac MRI measures and patient-reported outcomes.

• The trial included both late ambulatory and non-ambulatory patients, targeting those at high risk for upper limb and cardiac function loss.

• Deramiocel is a cell therapy with anti-fibrotic, immunomodulatory, and anti-inflammatory mechanisms.

Efficacy results

• Deramiocel showed a 54% slowing of upper limb deterioration compared to placebo, achieving statistical significance (p=0.029).

• Cardiac function, measured by left ventricular ejection fraction (LVEF), showed a 91% slowing of disease progression in treated patients.

• Patients with pre-existing cardiomyopathy had even greater benefit, with over a 3 percentage point difference in LVEF compared to placebo.

• All Type I error-controlled secondary endpoints, including mid-level PUL, global statistical test, and late gadolinium enhancement (cardiac scar), reached statistical significance.

• Improvements were observed in both skeletal muscle and cardiac function, with Deramiocel potentially being first-in-class for DMD cardiomyopathy.

Safety and tolerability

• Deramiocel was generally well tolerated; most adverse events were mild or moderate and resolved within 24 hours.

• Only one serious adverse event occurred in the treatment arm, resolving without sequelae; the placebo arm had five serious events.

• A pretreatment protocol with steroids and antihistamines was implemented to prevent infusion reactions.

• Long-term safety data from the HOPE-2 open-label extension (over four years) showed no new safety concerns.

• Infusions are administered quarterly at medical centers, not suitable for home infusion.