Capricor Therapeutics Begins Rolling BLA Submission to FDA for Duchenne Muscular Dystrophy Treatment

Capricor Therapeutics, a biotechnology firm listed on NASDAQ as CAPR, has announced the start of its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA). This application seeks full approval for deramiocel, a treatment for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).

Linda Marbán, Ph.D., the Chief Executive Officer of Capricor, highlighted the significance of this announcement. She noted that obtaining FDA approval for deramiocel would allow the company to deliver this innovative treatment to patients more swiftly. Marbán emphasized the importance of collaborating with the FDA throughout this process.

Capricor aims to complete the BLA submission by the end of 2024. Deramiocel's application may qualify for priority review due to its potential to significantly improve the safety and effectiveness of treatment for DMD cardiomyopathy—a serious condition with no currently approved treatments. Following the completion of the rolling submission, the FDA will inform the company when the application is formally accepted for review.

Deramiocel, also known as CAP-1002, is composed of allogeneic cardiosphere-derived cells (CDCs). These cells have demonstrated potent immunomodulatory, antifibrotic, and regenerative effects in both preclinical and clinical studies, particularly for dystrophinopathy and heart failure. CDCs secrete extracellular vesicles called exosomes, which target macrophages and modify their expression to promote healing rather than inflammation. More than 100 peer-reviewed scientific publications have discussed CDCs, and over 200 human subjects have received CDC treatments in various clinical trials.

Deramiocel has been granted Orphan Drug Designation for the treatment of DMD. The regulatory pathway for deramiocel is further supported by the Regenerative Medicine Advanced Therapy (RMAT) Designation. If Capricor receives FDA marketing approval for deramiocel, the company could also obtain a Priority Review Voucher (PRV) owing to its previous receipt of a rare pediatric disease designation.

Duchenne muscular dystrophy is a severe genetic disorder marked by progressive muscle weakness and chronic inflammation affecting the skeletal, heart, and respiratory muscles. The median age of mortality for DMD patients is approximately 30 years. The disorder is estimated to occur in about one in every 3,500 male births, with an estimated patient population of 15,000 to 20,000 in the United States. DMD is caused by a lack of functional dystrophin, a protein essential for muscle integrity. The absence of dystrophin leads to significant muscle cell damage and ultimately results in muscle cell death and fibrotic replacement. Currently, there are limited treatment options and no cure for DMD.

Capricor Therapeutics is a pioneering biotechnology company focused on developing transformative cell and exosome-based therapies for rare diseases. Their leading product candidate, deramiocel, is an allogeneic cardiac-derived cell therapy that has shown immunomodulatory, antifibrotic, and regenerative properties in extensive preclinical and clinical research. Deramiocel is currently in Phase 3 clinical development for DMD treatment. Capricor is also leveraging its exosome technology through its proprietary StealthX™ platform, which is in preclinical development for various applications, including vaccinology, and targeted delivery of oligonucleotides, proteins, and small molecule therapeutics to treat a wide range of diseases. Capricor remains dedicated to advancing innovative treatments for those in need.