Capricor Therapeutics Provides Enrollment Update on HOPE Clinical Trial in Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc.

, a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced that patient enrollment in its ongoing randomized HOPE-Duchenne clinical trial (Halt cardiomyOPathy progrEssion in Duchenne) has exceeded 50% of its 24-patient target. Capricor expects the trial to complete its enrollment in the third quarter of 2016, and to report top line data from HOPE-Duchenne in the first quarter of 2017. HOPE-Duchenne is evaluating CAP-1002, Capricor's investigational allogeneic cardiosphere-derived cell (CDC) therapy, in patients with Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy. John Jefferies, M.D., Director of Advanced Heart Failure and Cardiomyopathy and Associate Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at Cincinnati Children's Hospital, and Principal Investigator of the HOPE-Duchenne clinical trial, stated, "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. We believe that CAP-1002 is the only clinical-stage therapy intended to treat the heart disease that results from DMD." Linda Marbán, Ph.D., President and Chief Executive Officer of Capricor commented, "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. In pre-clinical studies, CDCs have been demonstrated to improve cardiac structure and function, exercise capacity, protein expression, and mitochondrial energetics. We anticipate reporting top line results of HOPE-Duchenne in the first quarter of next year." Capricor recently reported 12-month preliminary data from its DYNAMIC trial in adults with advanced heart failure, which demonstrated consistent and sustained improvement in cardiac structure and function. The randomized, multi-center HOPE-Duchenne trial is designed to evaluate the safety and efficacy of CAP-1002 in approximately 24 boys with DMD-associated cardiomyopathy. Patients randomized to the active treatment arm will receive CAP-1002 in the three main coronary arteries, to enable broad delivery across the myocardium. The HOPE-Duchenne trial is currently enrolling patients at Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio, Cedars-Sinai Heart Institute in Los Angeles, California, and at University of Florida in Gainesville, Florida. HOPE-Duchenne is being funded in part through the support of the California Institute for Regenerative Medicine. For more information, please visit: http://capricor.com/hope/ or ClinicalTrials.gov (NCT02485938).