Business
Capricor Presents Additional Positive Data from Ongoing HOPE-2 Study of CAP-1002 in Duchenne Muscular Dystrophy at World Muscle Society: Data Demonstrates Improved PUL 2.0 Performance at 6 Months
--Company Exploring Potential for Accelerated Approval under RMAT Designation, an Expedited Program for Regenerative Therapies-- --Company to Host Conference
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"--Company Exploring Potential for Accelerated Approval under RMAT Designation, an Expedited Program for Regenerative Therapies--\n --Company to Host Conference Call Today at 5:30 AM PT / 8:30 AM ET-- LOS ANGELES, Oct. 07, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company, in its presentation at the 24th Annual International Congress of the World Muscle Society, presented additional six-month interim data from HOPE-2, its ongoing Phase 2 clinical trial of its lead investigational product, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory, anti-fibrotic and has been shown to generate new muscle cells. Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients. As previously reported, additional independent tests assessing grip strength and tip to tip pinch strength also showed positive results. The U.S. Food and Drug Administration (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). The PUL scale was designed to measure upper limb motor performance across the spectrum of severity of DMD and is specifically focused on patients that are unable to perform the six-minute walk test. “We are very pleased that the interim analysis from this double-blind placebo-controlled study, has demonstrated meaningful improvements across three clinically relevant endpoints in older patients with limited remaining treatment options,” said Linda Marban, PhD, Chief Executive Officer of Capricor. “We continue to explore ways to utilize our FDA-granted Regenerative Medicine Advanced Therapy (RMAT) designation to pursue accelerated approval of CAP-1002, and we believe the agency’s written guidance on the matter supports our efforts to i...