Study Finds Capricor’s Cell Therapy Slows Arm Function Loss, Protects Heart

Rare Daily Staff

Capricor Therapeutics said its experimental cell therapy for the rare neuromuscular condition Duchenne muscular dystrophy slowed loss of arm function and helped protect heart function in a pivotal late-stage study.

In the phase 3 HOPE-3 trial, Capricor’s Deramiocel met its primary endpoing, showing a statistically significant benefit on upper limb function. It also achieved a key heart-related endpoint, left ventricular ejection fraction.

All secondary endpoints also reached statistical significance. Capricor said the findings translate into clinically meaningful benefits for both skeletal muscle and the heart, supporting Deramiocel as a potential first-in-class therapy for Duchenne-related cardiomyopathy, the leading cause of death in this disease.

Duchenne muscular dystrophy is an inherited muscle-wasting disorder that primarily affects boys and causes progressive weakness of the skeletal, breathing, and heart muscles. As the disease advances, damage to the heart muscle leads to cardiomyopathy and heart failure, which are the main causes of mortality. Current treatment options remain limited and do not cure the disease.

Deramiocel, also known as CAP-1002, is an investigational cell therapy made from cardiosphere-derived cells, a specialized population of heart cells taken from healthy donors. These cells are believed to release tiny vesicles called exosomes that can dampen harmful inflammation and scarring and promote tissue repair in both skeletal and cardiac muscle.

Deramiocel has received Orphan Drug and Regenerative Medicine Advanced Therapy designations in the United States. The company has an exclusive commercialization agreement with Japan’s Nippon Shinyaku and its U.S. subsidiary, NS Pharma, covering the United States and Japan.
Capricor reported that Deramiocel maintained a favorable safety and tolerability profile in the trial, consistent with findings from earlier studies.

Earlier this year, the U.S. Food and Drug Administration declined to approve Deramiocel and requested more information, including additional clinical data. The company said it now plans to submit a formal response incorporating the new HOPE-3 results, following what it described as prior alignment with the FDA that successful phase 3 data could be sufficient to support approval.

“These results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for more than 48 months, and highlight the strength, consistency, and reproducibility of Deramiocel’s clinical profile after more than a decade of rigorous clinical development,” said Linda Marbán, CEO of Capricor. “We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval.”

Photo: Linda Marbán, CEO of Capricor