--Final 12-Month Results from Phase II Trial to be Presented on October 1--
LOS ANGELES, Sept. 23, 2020 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class cell and exosome-based therapeutics for the treatment and prevention of diseases, announced today that the Company will present novel data from its randomized, double-blind, Phase II HOPE-2 clinical trial with its lead investigational product, CAP-1002, in boys and young men with Duchenne muscular dystrophy (DMD).
Dr. Craig McDonald, Study Chair of the CINRG Duchenne Natural History Study, as well as the National Principal Investigator for Capricor’s HOPE-2 study, along with Company management, will be available to discuss the e-poster at this year’s International World Muscle Society (WMS) Virtual Congress on October 1 from 12:30 to 2:30 p.m. ET.
DMD is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30.
“As one of the most common rare diseases, there are currently thousands of patients across the country suffering from the immobilizing effects of DMD,” said Dr. Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “With our lead asset, CAP-1002, which has the potential to exert immunomodulatory activity, Capricor is steadfast in its mission to establish a treatment path and alleviate these symptoms for an overall better quality of life for all of the boys and young adults battling DMD. We continue to discuss this program with the FDA to determine next steps and a pathway forward towards potential approval. We are proud to present this data at a prestigious industry event like WMS 2020 to further raise awareness of this debilitating disease."
In May 2020, Capricor announced positive topline 12-month results of the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of DMD. The data showed improvements in upper limb, cardiac and respiratory function, with p-values less than p=0.05 in multiple measures. The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002-treated patients (p=0.05) with a mean change of 2.4 points over placebo patients. With the exception of steroids, preservation of function in DMD is uncommon. The placebo patients declined consistent with natural history, but in the treated group, most patients were stable or showed improvement in function throughout the one-year treatment period.
The International Virtual Congress of the World Muscle Society will take place from September 28 through October 2, 2020. Capricor’s e-poster will be made available to attendees of the WMS 2020 Conference and on the Company’s website after the presentation.
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 cell and exosome-based therapeutics for the treatment and prevention of diseases. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also investigating the field of extracellular vesicles and exploring the potential of exosome-based candidates to treat or prevent a variety of disorders. We are now developing two potential vaccines for COVID-19 as part of our exosome platform. For more information, visit www.capricor.com and follow the Company on Facebook, Instagram and Twitter.
CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac cell therapy that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity. It is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The cells function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair. CDCs have been the subject of over 100 peer-reviewed scientific publications and administered to approximately 200 human subjects across several clinical trials.
HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the company’s lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.
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.
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, revenue projections; 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, 2019 as filed with the Securities and Exchange Commission on March 27, 2020 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as filed with the Securities and Exchange Commission on August 10, 2020. 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. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
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