Business
Capricor Therapeutics to Meet with FDA to Discuss CAP-1002 to Treat Duchenne Muscular Dystrophy
Company Exploring Potential for Accelerated Approval under RMAT Designation, an Expedited Program for Regenerative Therapies LOS ANGELES, Sept. 24, 2019
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\nLOS ANGELES, Sept. 24, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and other rare disorders, announced today that it has been granted a Type B End-of-Phase 2 meeting with the FDA to discuss prespecified interim analysis of the Company’s HOPE 2 clinical trial, which was reported in July of this year. The trial is a double-blind, placebo-controlled study of the company’s therapy, CAP-1002, in steroid-treated boys and young men in the later stages of DMD, a fatal genetic disease with few treatment options. The pre-specified interim analysis was performed on the six-month follow-up.\n Per FDA’s interpretation of Section 506(g) of the federal Food, Drug and Cosmetic Act, which was added by the 21st Century Cures Act enacted in 2016, therapies with an RMAT designation may be eligible for accelerated approval based on previously agreed-upon intermediate endpoints that are reasonably likely to predict long-term clinical benefit. “The data showed positive results across several independent measures, suggesting functional improvement in treated patients and providing the best evidence we’ve seen of clinical improvement in later stage Duchenne patients, with the exception of the use of steroids,” said Linda Marbán, CEO of Capricor. “We will be discussing the significance of this data and the next steps in our CAP-1002 program with the FDA. Our goal is to identify pathways to get this important therapy to DMD patients as soon as we can and we believe the FDA’s written guidance regarding accelerated approvals for therapies, like CAP-1002, that have the Regenerative Medicine Advanced Therapy (RMAT) designation provides the framework for these discussions.” The interim analysis of the six-month follow-up in HOPE-2 are consistent with the positive results seen in Capricor’s HOPE-Duchenne Phase I/II clinical trial which was published in Neurology, the medical journal of the American Academy of Neurology, earlier this year. The FDA has granted Capricor’s CAP-1002 RMAT and Orphan Drug Designation, for which the FDA has also granted a Rare Pediat...