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Capricor to Meet with FDA under its RMAT Designation to Discuss HOPE-2 Clinical Trial
Focus will be on Milestones for Development and Commercialization CAP-1002 Therapy for Duchenne Muscular Dystrophy
LOS ANGELES, Nov. 12, 2018 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR) announced today that it will meet with the U.S. Food and Drug Administration (FDA) in December to discuss clinical trial design, surrogate or intermediate endpoints and manufacturing processes for CAP-1002, Capricor’s novel cell therapy. Currently, patients are being enrolled in the HOPE-2 clinical trial, which is investigating CAP-1002 as a therapy for the treatment of Duchenne muscular dystrophy.
The in-person meeting is part of the expedited review process afforded to Capricor for its CAP-1002 product candidate after being granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA in February 2018. The FDA grants the RMAT designation to regenerative medicine therapies intended to treat a serious condition and for which preliminary clinical evidence indicates a potential to address unmet medical needs for that condition.
“We are very pleased to have this opportunity to discuss CAP-1002 and the HOPE-2 clinical trial with the FDA, and we appreciate the key opinion leaders in the field of Duchenne muscular dystrophy who will be joining us for the meeting,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “HOPE-2 is a randomized, placebo-controlled clinical trial testing repeat intravenous doses of CAP-1002. The study is currently enrolling up to 84 boys and young men at approximately 10-15 centers across the U.S. The participants are in the advanced stages of Duchenne muscular dystrophy and are typically not eligible for many of the other clinical trials being conducted for Duchenne. We believe that CAP-1002 could fill a currently unmet medical need for these patients.”
Pre-clinical and clinical studies have shown that CAP-1002 is generally safe, well-tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options.
The RMAT designation allows eligible therapies similar access to expedited development and review of a marketing application that are available to drugs that receive breakthrough therapy designation – including increased opportunities to meet with the FDA, early interactions with the FDA to discuss any potential surrogate or intermediate endpoints and the potential to support accelerated approval of drugs receiving the RMAT designation.
CAP-1002 consists 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 alters the immune system's activity to encourage 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 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.
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.
CONTACT: For more information, please contact: AJ Bergmann, Chief Financial Officer +1-310-358-3200 firstname.lastname@example.org