Deramiocel
- Generic Name
- Deramiocel
Satellos Reports Promising Efficacy Signals for Novel DMD Treatment in Adult Patients
• Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.
• The development comes amid setbacks in the DMD field, including Pfizer's withdrawal of its gene therapy fordadistrogene movaparvovec following a Phase III failure and a Phase II patient fatality.
• The global DMD treatment market is projected to grow substantially from $2.3 billion in 2023 to $5.2 billion by 2033 across major markets, primarily driven by Elevidys and Santhera Pharmaceuticals' Agamree.
Fitusiran Shows Promise in Reducing Bleeding Episodes in Hemophilia A and B Patients
• Fitusiran, an RNA interference therapy, is highlighted as a potential breakthrough for hemophilia A and B treatment, regardless of inhibitor status.
• Phase 3 trials (NCT03417245) demonstrated that fitusiran significantly reduced annualized bleeding rates in hemophilia A and B patients compared to on-demand factor concentrates.
• Another study (NCT03417102) showed a 90.8% reduction in annualized bleeding rate in patients with inhibitors treated with fitusiran compared to on-demand treatment.
• Fitusiran's efficacy and convenient subcutaneous administration position it as a drug to watch in hematology, offering a more personalized approach to hemophilia management.
Spinogenix's SPG601 Shows Promise in Phase 2 Trial for Fragile X Syndrome
• Spinogenix's SPG601 demonstrated a significant reduction in high-frequency gamma band activity, an EEG abnormality associated with Fragile X syndrome (FXS).
• The Phase 2 trial met its primary endpoint, suggesting SPG601's potential to improve brain activity related to learning and memory in FXS patients.
• SPG601 has received Fast Track designation from the FDA, which could expedite its development and regulatory review for FXS treatment.
• SPG601 targets large-conductance, calcium-activated potassium channels to correct synaptic dysfunctions, addressing core symptoms of FXS.
Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy
• Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.
• The BLA is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, showing attenuation of cardiac implications of DMD.
• The FDA has been requested to grant priority review, potentially reducing the review period to six months from the standard ten months.
• The BLA submission triggers a $10 million milestone payment to Capricor from its distribution partner, Nippon Shinyaku.
Capricor Therapeutics Advances Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy
• Capricor Therapeutics plans to file a Biologics License Application (BLA) with the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.
• The BLA will be supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 OLE trials, compared with natural history data.
• Capricor has initiated its rolling submission process with the FDA for deramiocel, with completion expected by the end of 2024.
• Deramiocel has shown immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure in clinical studies.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath