Capricor Therapeutics Advances Treatment for Duchenne Muscular Dystrophy

Shares in biotechnology company Capricor Therapeutics (Nasdaq: CAPR), rose on Tuesday after the company announced that it had successfully completed a Type-B meeting with the FDA regarding a Biologics License Application (BLA) submission for its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD).

Capricor also announced that the World Health Organization has selected Deramiocel as a proposed International Nonproprietary Name (INN) for CAP-1002.

Linda Marbán, PhD, Capricor’s Chief Executive Officer, commented, “The last year has been transformative for Capricor as we have moved rapidly towards potential approval for CAP-1002 for the treatment of DMD. The most recent meeting had several very important outcomes; the first of which was that the FDA has agreed to a pre-BLA meeting based on their review of our clinical data from HOPE-2, HOPE-2 open label extension (OLE) and available data from HOPE-3. This key agreement will allow us to begin our BLA filing with a rolling submission planned to initiate in the third quarter of 2024.”

Deramiocel (CAP-1002) for the treatment of DMD has received FDA Orphan Drug Designation, and may be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.

DMD is a devastating disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with a life expectancy of approximately 30 years. The patient population is estimated to be approximately 15,000-20,000 in the US. Treatment options are limited and there is no cure.

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, Deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown Deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel 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.