LOS ANGELES, March 17, 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 the Board of the California Institute for Regenerative Medicine (CIRM) has approved a grant award to Capricor for approximately $3.38 million to support the Company's Phase I/II HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial investigating CAP-1002, a cardiac cell therapy, in patients with Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The award was granted under the CIRM 2.0 program, a comprehensive collaborative initiative designed to accelerate the development of stem cell-based treatments for people with unmet medical needs. Capricor also reported today the completion of enrollment of the pre-specified first patient cohort in the HOPE-Duchenne trial.
"This grant award from CIRM provides significant validation of the therapeutic potential of CAP-1002 for patients with DMD-associated cardiomyopathy, a debilitating condition for which there is currently no approved treatment," said Linda Marbán, Ph.D., president and chief executive officer of Capricor. "These non-dilutive funds will be invaluable as we execute the HOPE-Duchenne clinical trial, for which we expect to report topline results during the first quarter of 2017."
"There can be nothing worse than for a parent to watch their child slowly lose a fight against a deadly disease," said C. Randal Mills, Ph.D., president and chief executive officer of CIRM. "Because diseases like Duchenne muscular dystrophy are rare, there are limited treatment options for them, which makes it all the more important for CIRM to focus on targeting these unmet medical needs."
CAP-1002, Capricor's lead product candidate, is an allogeneic, intracoronary-delivered, cardiosphere-derived cell treatment. This investigational therapy is an "off-the-shelf" cell therapy which comes from donor heart tissue and is infused directly into a patient's coronary artery during a catheterization procedure. CAP-1002 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of DMD.
In February 2016, Capricor announced treatment of the first patient in its Phase I/II HOPE-Duchenne clinical trial. The HOPE-Duchenne trial is a multi-center randomized open-label usual care-controlled study evaluating the safety and preliminary efficacy of CAP-1002 in 24 male patients with DMD who have significant cardiac involvement.
About Duchenne Muscular Dystrophy (DMD)
DMD afflicts approximately 20,000 boys and young men in the USA. The central cause is a genetic abnormality in the dystrophin complex, leading to membrane fragility with secondary damage to skeletal and cardiac muscle. No treatment has been proven effective for DMD, and patients usually die in young adulthood. Various clinical trials are ongoing, but almost all target the skeletal myopathy. Much of the death and disability in the later years of DMD is due to heart disease (cardiomyopathy) rather than to skeletal muscle disease. Virtually all DMD patients aged >15 years develop heart failure, and mortality is high despite optimal medical therapy. Heart transplantation is not typically an option for DMD patients.
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $3 billion in funding and approximately 300 active stem cell programs in our portfolio, CIRM is the world's largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information, please visit: https://www.cirm.ca.gov/.
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's lead programs target post myocardial infarction (heart attack), heart failure and Duchenne muscular dystrophy. Capricor has two lead product candidates under investigation: CAP-1002, a cardiac cell therapy, and Cenderitide, a 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 potential other indications. In addition, Capricor is conducting research and development on its exosomes platform technology for cardiac diseases and other potential indications. 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; 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 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, 2014, as filed with the Securities and Exchange Commission on March 16, 2015, 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 September 30, 2015, as filed with the Securities and Exchange Commission on November 13, 2015. 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.
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Capricor Therapeutics, Inc.
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