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Source: Capricor Therapeutics, Inc.

Capricor Therapeutics Presents Results of Studies of CAP-2003 at Gordon Research Conference on Extracellular Vesicles

Research Demonstrates the Investigational Therapy’s Mechanism of Action and Immunomodulatory Capacities

LOS ANGELES, Aug. 21, 2018 (GLOBE NEWSWIRE) -- At the Gordon Research Conference on Extracellular Vesicles in Newry, Maine, Capricor Therapeutics (NASDAQ: CAPR) presented research findings on the mechanism of action and the immunomodulatory capacities of CAP-2003, the company’s investigational therapy comprised of proprietary extracellular vesicles, including exosomes, which are derived from cardiosphere-derived cells (CDC-EVs). Capricor is developing CAP-2003 as a therapeutic platform for treating diseases involving inflammation and fibrosis.

The Gordon Research Conference on Extracellular Vesicles is focused on cutting-edge research on the biogenesis, molecular composition, functions, physio-pathological roles and potential clinical applications of extracellular vesicles. Gordon Research Conferences are a group of prestigious international scientific conferences that are at the forefront of research in the biological, chemical and physical sciences and their related technologies.

“The pre-clinical studies presented at the Gordon Research Conference further elucidate  Capricor’s progress in developing this exciting new class of therapeutics, the exosomes which comprise our investigational therapy, CAP-2003,” said Linda Marbán, Ph.D., Capricor chief executive officer. “The studies further demonstrate that exosomes may be the active pharmaceutical ingredient (API) in CAP-1002, our cell therapy product, because these extracellular vesicles serve as cellular-messengers, altering function and physiology to balance inflammation so that cellular repair can be facilitated.”

In the first study, Capricor compared CAP-2003 with exosomes made from mesenchymal stem cells (MSCs). The research found that CAP-2003 contains a unique cocktail of microRNAs, non-coding RNAs and proteins that drive greater immunomodulation, suggesting that CAP-2003 may be a more efficacious product candidate in diseases of inflammation and fibrosis.

This study also showed that CAP-2003 polarizes macrophages – a type of white blood cells involved in removing cellular debris and in tissue repair – to elicit anti-inflammatory and pro-regenerative responses. In addition, the study found that CAP-2003 has an immunomodulatory effect on T-cells, modifying the immune response of this critical component of the immune system. Researchers concluded that this study supports the use of CAP-2003 for the potential treatment of inflammatory indications.

An abstract from the second study reported that in a mouse model of Duchenne muscular dystrophy, CAP-2003 produced improvements that are similar to those attributed to CAP-1002, Capricor’s cell therapy product. The findings suggest that the exosomes that make up CAP-2003 mediate the CDCs’ mechanism of action. Capricor researchers also tested in vitro uptake of CDC-EVs by different cell types and found that CDC-EVs are taken up by immune cells with little uptake by fibroblasts and epithelial cells. This finding sheds further light on the mechanism of action of CDC-EVs in modulating inflammation and stimulating tissue repair.

Capricor is now conducting HOPE-2, a Phase II, randomized, double-blind, placebo-controlled study of repeat doses of CAP-1002 delivered intravenously. The study is enrolled in approximately 84 boys and young men in advanced stages of Duchenne muscular dystrophy. For more information on HOPE-2, please visit www.HOPE2Trial.com.

The posters presented at the Gordon Conference will be available on the Events & Presentations section of Capricor's website.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002

CAP-1002 is comprised of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

About CAP-2003

CAP-2003 is being developed as a next-generation therapeutic platform in regenerative medicine. CAP-2003 is comprised of nano-sized extracellular vesicles, including exosomes and microvesicles, derived from human CDCs, which exert anti-inflammatory, pro-angiogenic, anti-apoptotic, and anti-fibrotic effects. CAP-2003 contains several characteristic lipids, proteins, and RNA molecules (e.g., microRNAs). They act as messengers to regulate the functions of neighboring cells. Pre-clinical research has shown that exogenously-administered extracellular vesicles can direct or, in some cases, re-direct cellular activity, supporting their therapeutic potential. Their size, ease of crossing cell membranes and ability to communicate in native cellular language make them an exciting class of potential therapeutic agents.

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, please visit www.capricor.com.

Keep up with Capricor on social media: www.facebook.com/capricortherapeutics, www.instagram.com/capricortherapeutics/ and https://twitter.com/capricor.

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, 2017 as filed with the Securities and Exchange Commission on March 22, 2018, 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 June 30, 2018, as filed with the Securities and Exchange Commission on August 13, 2018. 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.