Capricor Therapeutics Awarded Up to $4.2 Million from National Institutes of Health to Evaluate Exosomes for Hypoplastic Left Heart Syndrome

LOS ANGELES, Oct. 20, 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 it has been awarded a grant of up to $4.2 million from the National Institutes of Health to study CAP-2003 (cardiosphere-derived cell exosomes) for hypoplastic left heart syndrome (HLHS).

"Capricor's successful record of competing for non-dilutive sources of capital reflects our commitment to scientific excellence as well as the importance of pursuing the therapeutic potential of our technologies. To date, we have been awarded over $30 million from government agencies to support the preclinical and clinical advancement of our cell and exosome candidates," said Linda Marbán, Ph.D., president and chief executive officer of Capricor. "HLHS is a tragic congenital defect of the heart that often results in heart failure and premature death despite several complex surgeries. With this grant award together with a recently-awarded grant from the Department of Defense to fund the development of a commercial manufacturing process for CAP-2003, we now have two federally-funded applications that will enable us to broaden the scope of our exosome program."

"Exosomes offer a different approach to regenerative medicine as compared to cell-based strategies, and can be handled more like traditional pharmaceuticals. CAP-2003 is independent of its cells of origin, so we believe this product may offer opportunities for clinical use that may not be ideal targets for our cellular therapeutic product (CAP-1002, allogeneic cardiosphere-derived cells). We are currently pursuing indications in which CAP-2003 can be locally delivered so as to have the best opportunity to act upon the injured cells. Both our ophthalmology program and our new HLHS program were chosen based on the direct delivery that each affords. CAP-2003 has shown compelling data in models of eye injury, and we look forward to its entry into the clinic next year for ocular graft-versus-host disease, a condition involving severe dry eye, pain and light sensitivity that is a frequent complication of bone marrow transplant," added Dr. Marbán.

"We are excited to be part of this research effort to understand how CDC Exosomes, which are believed to be responsible for the regenerative effect of cardiosphere-derived cells, may be useful in the treatment of a serious congenital heart condition," said Sunjay Kaushal, M.D., Ph.D., associate professor of surgery at the University of Maryland School of Medicine and director of pediatric cardiac surgery at the University of Maryland Medical Center, in Baltimore. Dr. Kaushal is a collaborator of Capricor and will be the principal investigator of a future clinical trial in HLHS. "We look forward to seeing if CAP-2003 can improve heart function, initially in pre-clinical models, and eventually in HLHS patients."

CAP-2003 represents exosomes isolated from Capricor's allogeneic cardiosphere-derived cells, (CAP-1002), which are in clinical development for several cardiac conditions including that associated with Duchenne muscular dystrophy.

About Hypoplastic Left Heart Syndrome

HLHS is a condition that occurs when parts of the left side of the heart such as the mitral valve, left ventricle, aortic valve, and aorta do not develop normally. The causes of HLHS among most babies are unknown, and if left untreated, HLHS is fatal. According to the Centers for Disease Control and Prevention (CDC), an estimated 960 babies are born with HLHS in the U.S. each year.

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.

This award was supported by the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number UT2HL131226. The content of this press release is solely the responsibility of the Company and does not necessarily represent the official views of the National Institutes of Health.

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 offerings, 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, including CAP-2003, has not yet been investigated in any clinical trial.

For more information, please contact:

Corporate
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
+1-310-358-3200
[email protected]

Investor Relations
Argot Partners
Kimberly Minarovich
+1-212-600-1902
[email protected]

Media
Argot Partners
Eliza Schleifstein
+1-917-763-8106
[email protected]

SOURCE Capricor Therapeutics, Inc.

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