NIPPON SHINYAKU CO

Capricor Therapeutics initiates rolling BLA submission to FDA for deramiocel to treat DMD cardiomyopathy, aiming to complete by end of 2024, potentially eligible for priority review.

Capricor Therapeutics initiated a rolling BLA submission with the FDA for deramiocel, aiming for full approval to treat DMD cardiomyopathy by end of 2024. Deramiocel, with Orphan Drug and RMAT designations, may qualify for priority review, offering a novel treatment for DMD, a condition with no current approved therapies.

Capricor Therapeutics has started a rolling BLA submission with the FDA for deramiocel, aiming to treat Duchenne muscular dystrophy cardiomyopathy. The submission, potentially eligible for priority review, is expected to be completed by end of 2024. Deramiocel, a novel treatment, has shown promise in preclinical and clinical studies for its immunomodulatory and regenerative effects.

Six clinical-stage biotech companies are advancing Duchenne muscular dystrophy treatments: Wave Life Sciences, Sarepta Therapeutics, Capricor Therapeutics, Edgewise Therapeutics, Italfarmaco, and Avidity Biosciences. These companies focus on various therapeutic approaches, including RNA medicines, gene therapies, myosin inhibitors, and HDAC inhibitors. The global Duchenne treatment market is expected to grow significantly, driven by regulatory approvals and ongoing research.

Capricor Therapeutics signed a deal with Nippon Shinyaku for European commercialization of deramiocel, a DMD treatment, receiving $20 million upfront and potential $715 million in milestones. The partnership aims to bring deramiocel to market, addressing a disease with no cure, and extends Capricor's cash runway into 2026.

Capricor Therapeutics plans to file a Biologics License Application (BLA) in October 2024 for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The initial label will include all DMD cardiomyopathy patients, with potential label expansion for skeletal muscle myopathy post-approval.

Capricor Therapeutics plans to file a Biologics License Application (BLA) for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The BLA submission is expected by end of 2024, with a rolling submission starting October 2024. Deramiocel has shown potential in clinical trials to attenuate DMD's cardiac implications.

Capricor Therapeutics announced an investor webcast to discuss FDA interactions and updates on deramiocel (CAP-1002), a Phase 3 candidate for Duchenne muscular dystrophy. The company also explores exosome-based therapies via its StealthX™ platform. Capricor has a commercialization agreement with Nippon Shinyaku for deramiocel in the U.S. and Japan, pending approval.

Immunotherapy less risky for non-small cell lung cancer patients with BMI under 28, while chemotherapy is optimal for those with BMI 28 or higher, according to Osaka Metropolitan University study.