Capricor Reaches 50% Enrollment for Duchenne MD HOPE Clinical Trial

Capricor Therapeutics has reached half its target enrollment for its ongoing HOPE-Duchenne clinical trial, the company announced. Capricor discovers, develops, and commercializes first-in-class therapeutics such as the cardiac cell therapy (CAP-1002) for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy.

The company expects to complete enrollment by the end of September and report top-line results in the first quarter of 2017. The HOPE-Duchenne trial will evaluate the investigational allogeneic cardiosphere-derived cell (CDC) CAP-1002. The trial is partly funded by the California Institute for Regenerative Medicine (CIRM).

“The pace of enrollment in HOPE-Duchenne has exceeded our expectations, reflecting the large unmet need among boys suffering from Duchenne cardiomyopathy, a progressive disease with limited therapeutic options, and the excellent work by our clinical investigators,” John Jefferies, MD, the principal investigator of the HOPE-Duchenne clinical trial, said in a press release.

Jefferies is the director of Advanced Heart Failure and Cardiomyopathy and associate professor of pediatric cardiology and adult cardiovascular diseases at Cincinnati Children’s Hospital. “We believe that CAP-1002 is the only clinical-stage therapy intended to treat the heart disease that results from DMD,” he said.

CAP-1002 is a proprietary allogeneic adult stem cell therapy designed to treat heart disease, where cells obtained from donor heart tissue are expanded in the laboratory and later introduced directly into the patient’s heart via infusion into a coronary artery using regular cardiac catheterization methods.

The randomized, multi-center trial will evaluate the safety and efficacy of CAP-1002 in 24 boys with DMD-associated cardiomyopathy. Patients who fall in the active treatment arm will receive CAP-1002 in the three main coronary arteries to enable broad delivery across the myocardium.

HOPE-Duchenne is now enrolling patients at Cincinnati Children’s Hospital Medical Center in Cincinnati; Cedars-Sinai Heart Institute in Los Angeles; and at the University of Florida in Gainesville.

“The underlying cause of DMD is a mutation in the gene that codes for dystrophin, resulting in breakdown of the muscle cell membrane with tissue inflammation, necrosis, and subsequent fibrosis. As CDCs have been demonstrated to be both anti-inflammatory and anti-fibrotic, they appear to be mechanistically well-positioned to favorably modulate this process,” said Capricor President and CEO Linda Marbán, PhD. “In pre-clinical studies, CDCs have been demonstrated to improve cardiac structure and function, exercise capacity, protein expression, and mitochondrial energetics.”