SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotechnology firm specializing in cell and exosome-based treatments for rare diseases, is set to discuss its manufacturing and commercial launch plans for CAP-1002, a therapy for Duchenne muscular dystrophy (DMD), with the U.S. Food and Drug Administration (FDA) in late March. The company aims to expedite the submission of a Biologics License Application (BLA) through this in-person Type-B meeting.
Capricor has already received Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for CAP-1002, which allow for closer collaboration with the FDA and guidance on accelerated approval pathways. With the pivotal Phase 3 HOPE-3 trial for CAP-1002 now fully enrolled in Cohort A, top-line data is expected in the fourth quarter of this year.
Linda Marbán, Ph.D., CEO of Capricor, expressed the company's commitment to fast-tracking the approval process. "At this time, with our pivotal Phase 3, HOPE-3 trial fully enrolled in Cohort A and top-line data expected in the fourth quarter of this year, we believe that discussing our CMC plans for potential commercialization with the FDA will allow us the opportunity to bring CAP-1002 to patients in the most expeditious manner possible for patients in need," said Marbán.
CAP-1002 is an allogeneic cardiac-derived cell therapy that has demonstrated immunomodulatory, antifibrotic, and regenerative effects in preclinical and clinical studies, specifically for dystrophinopathies and heart disease. The therapy is currently in Phase 3 clinical development for DMD treatment.
In addition to CAP-1002, Capricor is exploring the potential of its proprietary exosome technology platform, StealthX™, in various therapeutic areas, including vaccinology and targeted delivery of oligonucleotides, proteins, and small molecules.
This news is based on a press release statement and includes forward-looking statements subject to risks and uncertainties that could cause actual results to differ materially from those projected. Capricor's partnership with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of CAP-1002 in the United States and Japan is contingent upon regulatory approval. CAP-1002 is currently an Investigational New Drug and has not been approved for any indications.
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