Capricor Therapeutics Reports Positive FDA Meeting for CAP-1002 Duchenne Muscular Dystrophy Program

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology firm specialized in innovative cell and exosome-based therapies for rare diseases, announced a crucial development regarding its lead product, CAP-1002, intended for treating Duchenne muscular dystrophy (DMD). The company recently concluded a Type-B meeting with the U.S. Food and Drug Administration (FDA) to discuss the next steps towards submitting a Biologics License Application (BLA) for CAP-1002. The World Health Organization has also proposed the name Deramiocel as the International Nonproprietary Name (INN) for CAP-1002.

Capricor’s CEO, Linda Marbán, Ph.D., highlighted the significance of the past year, noting the rapid advancements towards potentially securing approval for CAP-1002. The recent FDA meeting yielded several pivotal outcomes, including the FDA’s agreement to a pre-BLA meeting after reviewing clinical data from the HOPE-2, HOPE-2 open label extension (OLE), and available data from the HOPE-3 studies. This agreement facilitates the initiation of the BLA filing process with a rolling submission set to commence in the third quarter of 2024.

The rolling submission process allows for components of the BLA to be submitted as they are completed, which can expedite the regulatory review process and mitigate risks associated with the BLA. Capricor plans to further define the submission schedule with the FDA in the upcoming months.

Dr. Marbán expressed satisfaction with the progress made in the program and the anticipation for final data from the HOPE-3 pivotal trial, expected in the fourth quarter of 2024. Preparations for potential commercialization are also underway, which include finalizing the commercial manufacturing facility and getting ready for the pre-BLA meeting and rolling BLA submission. Additionally, Capricor looks forward to presenting further 3-year data from the HOPE-2 OLE study at the upcoming Parent Project Muscular Dystrophy (PPMD) conference, which is expected to reinforce the efficacy and safety of CAP-1002.

Deramiocel (CAP-1002) has been granted Orphan Drug Designation for the treatment of DMD. The regulatory pathway for this drug is bolstered by the Regenerative Medicine Advanced Therapy (RMAT) Designation. If the FDA grants marketing approval for Deramiocel for DMD, Capricor may be eligible to receive a Priority Review Voucher (PRV), following its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if awarded.

Duchenne muscular dystrophy (DMD) is a severe genetic disorder marked by progressive muscle weakness and chronic inflammation affecting the skeletal, heart, and respiratory muscles, with a median life expectancy of around 30 years. The disorder is estimated to affect approximately 15,000-20,000 individuals in the United States, occurring in roughly one in every 3,500 male births. The condition stems from the deficient production of functional dystrophin, a structural protein in muscle, leading to significant cellular damage, muscle cell death, and fibrotic replacement. Current treatment options are limited, and there is no cure.

Capricor Therapeutics is at the forefront of developing transformative therapies, with Deramiocel (CAP-1002) in Phase 3 clinical development for DMD. The company is also leveraging its exosome technology through its proprietary StealthX™ platform, focusing on vaccinology and targeted delivery of oligonucleotides, proteins, and small molecule therapeutics to address a variety of diseases. Capricor remains committed to pushing the boundaries of medical innovation and providing transformative treatments for those in need.