Caprock Mining Corp

Fitusiran Shows Promise in Reducing Bleeding Episodes in Hemophilia A and B Patients

4 months ago

• 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

4 months ago

• 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

5 months ago

• 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

8 months ago

• 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.