CSL(US:CSLLY) Prnewswire·2025-12-07 17:01Core Insights - The five-year results from the Phase 3 HOPE-B study confirm the long-term durability and safety of HEMGENIX, a gene therapy for adults with hemophilia B, demonstrating sustained therapeutic benefits and a favorable safety profile [2][3][4] Efficacy and Safety - 94% of patients remained free from continuous prophylaxis treatment five years after a single infusion of HEMGENIX, indicating its effectiveness in providing sustained bleed protection [1][5] - Mean factor IX activity levels were maintained at 36.1% at year five, showcasing the therapy's durable efficacy [1][5] - The mean adjusted annualized bleeding rate (ABR) for all bleeds decreased by approximately 90% from the lead-in period (4.16) to year five (0.40) [5] Real-World Adoption - More than 75 individuals across eight countries have received HEMGENIX in real-world settings, reflecting its growing global adoption [1][7] Clinical Study Details - The pivotal Phase 3 HOPE-B trial involved 54 adult male participants with severe or moderately severe hemophilia B, with 50 completing five years of follow-up [3][9] - The study's primary endpoint was the ABR measured from month seven to month 18 post-infusion, ensuring a steady-state factor IX expression [9] Regulatory Status - HEMGENIX has received regulatory approval in multiple countries, including the United States, Canada, and the European Union, indicating its acceptance in various markets [7][21]