Benzinga | Sep 23, 2020 09:27AM EDT

Capricor Therapeutics Reports Will Present Results From HOPE-2 Trial With CAP-1002 In Duchenne Muscular Dystrophy At International World Muscle Society Virtual Congress 2020 Oct. 1 At 12:30 p.m. EDT

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 Marb?n, 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.