Core Insights - CSL Limited and uniQure Inc announced the four-year results from the pivotal HOPE-B study, confirming the long-term durability and safety of Hemgenix for adults with hemophilia B [1][2] Group 1: Study Results - Hemgenix demonstrated sustained factor IX activity levels over four years, providing long-term bleed protection compared to prophylactic treatment [2][4] - The mean factor IX levels were recorded at 41.5 IU/dL in year one, 36.7 IU/dL in year two, 38.6 IU/dL in year three, and 37.4 IU/dL in year four [4] - The adjusted annualized bleeding rate (ABR) for all bleeds decreased by approximately 90% from lead-in to year four, with joint bleeds reducing from a mean ABR of 2.34 to 0.09 [5] Group 2: Patient Outcomes - In year four, 94% of patients remained free of continuous prophylaxis treatment, and no patients returned to continuous prophylaxis between year three and year four [5] - There were no serious adverse events related to the treatment throughout the study [5] Group 3: Product Information - Hemgenix, approved by the FDA in 2022, is the first gene therapy for adults with hemophilia B, applicable to patients with or without AAV5-neutralizing antibodies [3]
uniQure/ CSL Behring's Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years