Press Release: Fitusiran prophylaxis reduced bleeds by 61% in people with hemophilia A or B, with or without inhibitors, compared to prior factor or bypassing agent prophylaxis

F itusiran prophylaxis reduced bleeds by 61% in people with hemophilia A or B, with or without inhibitors, compared to prior factor or bypassing agent prophylaxis A median annualized bleeding rate (ABR) of 0.0 was reported in the overall study population during fitusiran prophylaxis (80 mg monthly) Fitusiran is a novel, investigational subcutaneously administered small interference RNA therapy, in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitor Paris – July 10 , 2022 – Positive data from the Phase 3 ATLAS-PPX study evaluating the efficacy and safety of once-monthly fitusiran (80 mg) in adults and adolescents with severe hemophilia A or B who were previously treated with prior factor or bypassing agent (BPA) prophylaxis were presented today in a late-breaking session at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress. The study met the primary endpoint and demonstrated fitusiran prophylaxis significantly reduced bleeding episodes compared to prior factor or BPA prophylaxis. Gili Kenet , MD Investigator , professor of Hematology, Director of the Israeli National Hemophilia Center at Sheba Medical Center and head of the Amalia Biron Thrombosis Research Institute of Tel Aviv University , Tel Aviv, Israel " There is a continued need for transformative therapies that offer people with hemophilia consistent protection while also reducing treatment burden. Th ese phase 3 results are encouraging and support fitusiran's potential to provide people with hemophilia A or B, regardless of inhibitor status, with a meaningful reduction in bleeding episodes ." Key findings in the Phase 3 ATLAS-PPX study include the following: The overall median annualized bleeding rate (ABR) was 0.0 for fitusiran prophylaxis, compared to a median ABR of 4.4 with prior prophylaxis. Fitusiran prophylaxis resulted in a statistically significant reduction in estimated ABR of 61.1% (p= 0.0008) versus factor or BPA prophylaxis. 63.1% (n=41) of adults and adolescents treated with fitusiran experienced zero treated bleeds compared to 16.9% (n=11) with prior factor or BPA prophylaxis. Median ABR for treated bleeds was 0.0 with fitusiran prophylaxis for both participants with and without inhibitors compared to 6.5 and 4.4 for participants with and without inhibitors, respectively, on prior prophylaxis. Of the 67 participants exposed to a least one dose of fitusiran, the most common adverse events (≥6 participants) were increased alanine aminotransferase, nasopharyngitis, and upper respiratory tract infection. Consistent with the previously identified risk of fitusiran, suspected or confirmed thromboembolic events were reported in 2 participants (3.0%). Dietmar Berger MD, PhD Global Head of Development and Chief Medical Officer "T hese po sitive data support fitusiran's potential to transform prophylaxis treatment for people with hemophilia A or B , with or without inhibitors , with a median annua l bleed rate of zero acr o ss all patient populations. Moreover , we are excited to continue to explore fitusiran under an amended protocol that focuses on dose optimization , incl u ding lower doses and less frequent dosing regimen s, with the potential for as few as six injections per year ." Additional data from the fitusiran ... Full story available on Benzinga.com