Capricor to Present HOPE-2 Long-Term Data at 2024 World Muscle Society Congress
Capricor Therapeutics, a San Diego-based biotechnology company listed on NASDAQ under the symbol CAPR, has revealed promising three-year safety and efficacy data from its HOPE-2 open-label extension (OLE) study. This study investigates the therapeutic potential of its lead product candidate, deramiocel (CAP-1002), for treating Duchenne muscular dystrophy (DMD). These results will be showcased in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS 2024), scheduled for October 8-12, 2024, in Prague, Czechia.
Deramiocel has shown notable long-term, multi-modal benefits for patients with DMD, particularly targeting skeletal and cardiac muscle pathogenesis. The poster presentation, titled "Multi-modal benefits of deramiocel (CAP-1002) in late-stage patients with DMD: a new treatment approach to target skeletal and cardiac muscle pathogenesis (HOPE 2-OLE trial: 36-month data)," will be led by Dr. Craig McDonald from UC Davis. The presentation is set for October 11, 2024, from 9:45-10:45 a.m. EDT (15:45-16:45 CEST), under the session code 721LBP, Session 4.
Linda Marbán, Ph.D., CEO of Capricor, emphasized the significance of the HOPE-2 OLE trial results, stating that the study's findings reinforce the potential of deramiocel to treat DMD-cardiomyopathy. The company is moving forward with filing a Biologics License Application (BLA) to seek potential approval for deramiocel, highlighting its commitment to advancing therapies for rare diseases.
Capricor Therapeutics focuses on developing transformative treatments for rare diseases using cell and exosome-based therapeutics. Deramiocel, their flagship product, is an allogeneic cardiac-derived cell therapy that has demonstrated immunomodulatory, antifibrotic, and regenerative properties in clinical and preclinical studies. These properties make deramiocel particularly suitable for addressing dystrophinopathies and heart disease, including DMD. Currently, deramiocel is in Phase 3 clinical development for DMD treatment.
Capricor is also leveraging its proprietary StealthX™ platform, which focuses on exosome technology for various applications. The platform is in preclinical development for use in vaccinology, targeted delivery of oligonucleotides, proteins, and small molecule therapeutics. This technology aims to offer innovative solutions for a broad range of diseases, underscoring Capricor's dedication to exploring new therapeutic possibilities.
In addition to its research and development initiatives, Capricor has secured an exclusive commercialization and distribution agreement for deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., through its U.S. subsidiary NS Pharma, Inc., contingent on regulatory approval. It's important to note that deramiocel is currently an Investigational New Drug and has not yet received approval for any specific indications. Similarly, none of Capricor's exosome-based candidates are approved for clinical investigation at this stage.
Capricor continues to push the boundaries of medical science, striving to deliver transformative treatments for patients with rare diseases. With promising clinical data and strategic partnerships, the company remains at the forefront of developing innovative therapies that have the potential to significantly impact patients' lives.
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