LOS ANGELES, April 22, 2015 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that it has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its cell therapeutic product candidate, CAP-1002, for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
Cardiosphere-derived cells (CDCs), also known as CAP-1002, are a potential therapeutic approach for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy. The cells have been shown to promote cardiomyogenesis and angiogenesis, while inhibiting oxidative stress, inflammation and fibrosis in preclinical studies. Pre-clinical data presented at the American Heart Association's annual meeting in November 2014 demonstrated that cardiac function and exercise capacity improved in CDC-treated mdx mice, accompanied by attenuation of inflammation, improved mitochondrial function and a reduction in collagen content and fibrosis in CDC-treated mdx hearts. The findings raise the possibility that CDCs may be useful therapeutically for heart failure in patients with Duchenne muscular dystrophy.
"We are pleased to have received orphan drug designation for CAP-1002 for the treatment of DMD," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "Duchenne cardiomyopathy is a devastating complication of the disease that impacts virtually all those affected by the disease and is the leading cause of death in patients. We believe CDCs are uniquely suited to treat the cardiac component of DMD and could potentially be used in conjunction with any of the therapeutics currently in use or under investigation for the skeletal muscle pathology. Capricor is addressing the devastating cardiac complications for which there is no specific treatment. We anticipate filing an IND with the FDA in the near future and hope to be treating patients with Duchenne later this year subject to regulatory approval. We look forward to reporting additional milestones as they are achieved."
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. This designation confers special incentives to the drug developer, including tax credits on the clinical development costs, prescription drug user fee waivers and may allow for a seven year period of market exclusivity in the U.S. upon FDA approval.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (Nasdaq:CAPR) is a clinical-stage biotechnology company with expertise in the field of cardiovascular disease aiming to develop novel therapies for diseases with tremendous unmet medical needs. Our lead programs target post myocardial infarction (heart attack), heart failure and Duchenne Muscular Dystrophy. The Company has two leading product candidates under investigation: Cenderitide, a natriuretic peptide receptor agonist, and CAP-1002, a cardiac cell therapy. Cenderitide is in development for the outpatient treatment of heart failure as well as potential other indications. CAP-1002 is in development for the treatment of post myocardial infarction (heart attack), advanced heart failure and Duchenne muscular dystrophy cardiomyopathy. In addition, the Company 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; scope, duration, validity and enforceability of intellectual property rights; plans regarding regulatory filings, future research and clinical trials; plans regarding current and future collaborative activities and the ownership of commercial 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 our business are set forth in our 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, and in our Registration Statement on Form S-1, as filed with the Securities and Exchange Commission on March 6, 2015. All forward-looking statements in this press release are based on information available to us as of the date hereof, and we assume no obligation to update these forward-looking statements.
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