LOS ANGELES, Sept. 7, 2016 /PRNewswire/ -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing biological therapies for cardiac and other serious medical conditions, today announced that the randomized Phase I/II HOPE-Duchenne clinical trial has completed its enrollment with 25 subjects. HOPE (Halt cardiomyOPathy progrEssion in Duchenne) is evaluating CAP-1002, Capricor's lead investigational cardiac cell therapy, in Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. Capricor expects to report top-line six-month data from this trial in the first quarter of 2017.
John Jefferies, M.D., Director of the Advanced Heart Failure and Cardiomyopathy Programs and Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at Cincinnati Children's Hospital, and Principal Investigator of the HOPE-Duchenne clinical trial, stated, "It is accepted that the most common cause of death from DMD relates to the impact of this disease on heart muscle. The enthusiasm for HOPE-Duchenne has been remarkable, and we are excited to see the upcoming results of this innovative study."
"The rate of patient enrollment into HOPE-Duchenne far surpassed our expectations, signifying the need for therapeutic options as well as the desire of the DMD community to address the heart disease that is highly prevalent in this population," said Linda Marbán, Ph.D., president and chief executive officer of Capricor. "We look forward to announcing top-line six-month results from HOPE-Duchenne in the first quarter of next year, in which we will report on the safety as well as the potential efficacy of CAP-1002. In DMD, scar tissue progressively aggregates in the heart, leading to a deterioration of cardiac function for which treatment options are limited. We believe CAP-1002 is the only therapeutic candidate in development for the treatment of DMD that has been clinically shown to reduce scar tissue in the damaged heart."
The HOPE trial enrolled 25 boys with DMD who were at least 12 years of age at the time of screening and who have DMD-associated cardiomyopathy, defined as the presence of scar tissue in at least four left ventricular segments as determined by magnetic resonance imaging (MRI). The 13 subjects randomized to the active treatment arm received CAP-1002 via intracoronary infusion into each of the three main coronary arteries during a single procedure. The 12 subjects randomized to the control arm received usual care and did not receive an infusion.
The primary outcome measures of the trial will consist of a broad assessment of safety and tolerability of CAP-1002. Efficacy will be evaluated according to pre-specified secondary outcome measures, including absolute and relative changes in cardiac scar tissue and cardiac function as measured by MRI, performance on the Six-Minute Walk Test (6MWT) and the Performance of the Upper Limb (PUL), and scoring on the Pediatric Quality of Life Inventory (PedsQL).
The HOPE-Duchenne trial is being conducted at Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio, Cedars-Sinai Heart Institute in Los Angeles, California, and the 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. It is caused by a genetic abnormality in the dystrophin complex, a structural element which plays a critical role in muscle fiber integrity, and leads to chronic skeletal and cardiac muscle damage. 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. The U.S. Food and Drug Administration (FDA) has not approved any treatment for DMD, and heart transplantation is not typically an option.
Capricor's lead product candidate, CAP-1002, is an investigational allogeneic cardiac cell therapy for the treatment of heart disease. CAP-1002 is manufactured from unrelated donor heart tissue through a proprietary process in which cells are expanded from cardiospheres, self-assembling, multicellular clusters of progenitor heart cells. The product is stored frozen, and stability data support "off the shelf" use for at least three years. The FDA has granted orphan drug designation to CAP-1002 for the treatment of DMD.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of biological therapies for the treatment of cardiac and other serious medical conditions. Capricor's lead candidate, CAP-1002, is a cardiac cell therapy that is currently being evaluated for the treatment of heart disease associated with Duchenne muscular dystrophy and myocardial infarction (heart attack). Capricor is advancing its proprietary exosome product candidate, CAP-2003, for the treatment of ophthalmic disorders and is exploring other therapeutic areas. Capricor's portfolio also features Cenderitide, a dual natriuretic peptide receptor agonist, which may have application for the outpatient treatment of advanced heart failure and other potential indications. For additional information, visit 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 initiation, 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 June 30, 2016, as filed with the Securities and Exchange Commission on August 15, 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.
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Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
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