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Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated with Lead Candidate CAP-1002
-One-Year Results from Randomized, Double-Blind, Placebo-Controlled Study Demonstrate Improved Performance of Upper Limb (PUL) 2.0 (p=0.05)- -First Ever Study
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-One-Year Results from Randomized, Double-Blind, Placebo-Controlled Study Demonstrate Improved Performance of Upper Limb (PUL) 2.0 (p=0.05)-\n -First Ever Study in DMD that Correlates Stabilization in Cardiac Function with a Reduction in a Biomarker of Cell Damage- -Company Planning to Meet with FDA to Discuss Pathway to Approval- -To Host Conference Call and Webcast Today at 8AM ET- LOS ANGELES, May 13, 2020 (GLOBE NEWSWIRE) -- Capricor Therapeutics (“Capricor”) (NASDAQ: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment and prevention of diseases, announced today positive top-line 12-month results of the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Boys and young men typically lose their ability to walk in their teens and generally die of cardiac or respiratory complications by the 3rd decade of life. 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 improved throughout the one-year treatment period. The performance of the upper limb (PUL) is a clinically validated measure that evaluates upper limb (shoulder, arm, hand) strength in patients who are generally non-ambulant. Retention of upper limb function is important for self-care and preservation of human dignity and has become a focus for physicians and advocates to find treatments to help these later stage patients. The FDA has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA). Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation commented, “I...