LOS ANGELES, June 23, 2016 /PRNewswire/ -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), 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.
About Duchenne Muscular Dystrophy (DMD)
DMD afflicts approximately 20,000 boys and young men in the U.S. DMD is caused by a genetic abnormality in the dystrophin complex, leading to membrane fragility with secondary damage to skeletal and cardiac muscle. Following years of progressive weakness, patients often die in their twenties. Heart disease is currently the most common cause of death among those with DMD. No medicine has been approved for the treatment of DMD in the United States, and heart transplantation is not typically an option.
CAP-1002, Capricor's lead product candidate, is a proprietary allogeneic adult stem cell therapy for the treatment of heart disease. Through a specialized process, cells obtained from donor heart tissue are expanded in the laboratory and then introduced directly into a patient's heart via infusion into a coronary artery using standard cardiac catheterization techniques.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics. Capricor has two product candidates under clinical investigation: CAP-1002, a cardiac cell therapy, and Cenderitide, a dual natriuretic peptide receptor agonist. CAP-1002 is in development for the treatment of post myocardial infarction, advanced heart failure and Duchenne muscular dystrophy-associated cardiomyopathy. Cenderitide is in development for the outpatient treatment of heart failure as well as other potential indications. In addition, Capricor is evaluating its exosomes platform technology for cardiac diseases and other therapeutic areas. For additional information, visit www.capricor.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, expectations with respect to the expected use of proceeds from the recently completed offering and the anticipated effects of the offering, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business are set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2015, as filed with the Securities and Exchange Commission on March 30, 2016, in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, and in its Quarterly Report on Form 10-Q for the quarter ended March 31, 2016, as filed with the Securities and Exchange Commission on May 13, 2016. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
CAP-1002 and Cenderitide are Investigational New Drugs and are not approved for any indications. Capricor's exosomes technology has not yet been investigated in any clinical trial.
For more information, please contact:
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
SOURCE Capricor Therapeutics, Inc.