Capricor Therapeutics Announces Issuance of Key U.S. Patent on Exosome Technology

LOS ANGELES, Dec. 13, 2017 /PRNewswire/ -- Capricor Therapeutics (NASDAQ: CAPR) today announced that the U.S. Patent and Trademark Office has issued U.S. Patent  9,828,603, which includes composition of matter claims covering cardiosphere-derived cell exosomes. This patent, entitled "Exosomes and Micro-ribonucleic Acids for Tissue Regeneration," is expected to run until at least 2033.

Exosomes are nano-sized, membrane-enclosed vesicles that are secreted by cells and contain bioactive molecules, including proteins, RNAs and microRNAs. They act as messengers to regulate cellular function. Their size, ease of crossing cell membranes and ability to communicate in native cellular language makes them an exciting class of potential therapeutic agents. CAP-2003 comprises exosomes isolated from the company's proprietary cardiosphere-derived cells and is being developed as a potential next-generation therapeutic for diseases of fibrosis and inflammation.

"The issuance of this patent is an important milestone as Capricor plans to extend the scope of its clinical programs to the development of CAP-2003, which has demonstrated the ability to profoundly modulate the immune response, decrease inflammation, and stimulate cell growth in a variety of disease models," said Linda Marbán, Ph.D., Capricor's president and CEO. "Capricor plans to initially develop CAP-2003 for hypoplastic left heart syndrome, a congenital heart defect that affects about 960 newborns in the U.S. each year and is associated with high rates of mortality and heart failure secondary to the condition. Pending regulatory clearance, we expect to commence a clinical trial of CAP-2003 in patients with hypoplastic left heart syndrome in 2018."

The therapeutic prospect of CAP-2003 in hypoplastic left heart syndrome (HLHS) is supported by preclinical studies in which this candidate was evaluated in a model of pressure-induced right ventricular dysfunction, representative of the disease present in many single ventricle congenital heart conditions such as HLHS. These studies, conducted by Sunjay Kaushal, M.D., associate professor of surgery and director of pediatric and adult congenital surgery at the University of Maryland, showed that, while untreated study animals developed right ventricular dilation and systolic dysfunction, intramyocardial injection of CAP-2003 significantly preserved right ventricular fractional area change (p=0.02 at one month of study follow-up).

U.S. Patent 9,828,603 is assigned to Cedars-Sinai Medical Center, which has granted Capricor worldwide rights to the technology under an exclusive license agreement.

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 biological therapeutics for the treatment of rare disorders. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.

The above-referenced preclinical studies in HLHS were supported in part with funding from the National Heart, Lung and Blood Institute of the National Institutes of Health under Award No. UT2HL131226.

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; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; 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 offerings 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 is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2016 as filed with the Securities and Exchange Commission on March 16, 2017, in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, together with the prospectus included therein and prospectus supplements thereto, and in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, as filed with the Securities and Exchange Commission on November 14, 2017. 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 is an Investigational New Drug and is not approved for any indications. CAP-2003 has not yet been approved for clinical investigation.

For more information, please contact:
AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com

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SOURCE Capricor Therapeutics, Inc.