Capricor Therapeutics Advances Duchenne Muscular Dystrophy Candidate
`Capricor Therapeutics (Nasdaq: CAPR) has successfully completed a Type-B Chemistry, Manufacturing and Controls (CMC) meeting with the FDA to discuss next steps for the company’s Biologics License Application (BLA) submission for its lead asset CAP-1002 in the treatment of Duchenne muscular dystrophy (DMD). Capricor’s share price settled up 2.18% on Thursday, rising an additional 2.91% in after market trading.
Capricor has been granted a subsequent Type-B meeting which it expects to take place in Q2 2024 to discuss its HOPE-2 open label extension 3-year safety and efficacy data. Data from Cohort B of the HOPE-3 clinical trial will not be necessary for FDA approval of the product. The company believes the upcoming discussions will potentially lead to an accelerated BLA filing.
“I am extremely pleased with our recent FDA interactions as we continue to work collaboratively with the agency to align on the most expeditious path towards registration of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Capricor has generated extensive safety and efficacy data in multiple clinical trials and we are very encouraged by the FDA’s agreement that we have successfully demonstrated product comparability which allows for a seamless transition to our San Diego manufacturing facility without the need for additional clinical data.”
CAP-1002 has been granted Orphan Drug Designation by the FDA for the treatment of DMD.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins, and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
