Phase III HOPE-B study of Hemgenix showing long term benefits in haemophilia B presented at the 64th ASH Annual Meeting.- CSL
CSL announced the presentation of data affirming the long-term durability and safety of single-infusion Hemgenix (etranacogene dezaparvovec-drlb) in the treatment of certain adults living with hemophilia B at the 64th American Society of Hematology (ASH) Annual Meeting. Results from the pivotal HOPE-B study, the largest gene therapy study in haemophilia B to date, demonstrated that after two years of follow-up, adults with severe or moderately severe hemophilia B treated with a one-time infusion of Hemgenix generated elevated and sustained mean factor IX activity levels and durable hemostatic protection.
In the open-label, single-arm HOPE-B study, 54 adult male participants with severe or moderately severe hemophilia B, with or without pre-existing AAV5 neutralizing antibodies were infused with a single dose of Hemgenix. Hemgenix produced mean factor IX activity of 39.0 IU/dL at six months and 36.9 IU/dL at 18 months post infusion, which was sustained at 36.7 IU/dL in the long-term follow-up data at two years. In addition, 94 percent (51 out of 54) of patients treated with Hemgenix discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy.
The data demonstrate the ABR (annual bleeding rate) for all bleeds was reduced by 64% during months 7-24 of the study (mean ABR 1.51 vs. 4.18 during the lead-in period; p=0.0002), sustaining the same bleed reduction that satisfied the study's primary endpoint. The FDA-approved prescribing information for Hemgenix shows that ABR for all bleeds was reduced by 54% during months 7-18 of the study. Treatment with Hemgenix also reduced mean unadjusted annualized factor IX consumption by 96% from the lead-in period (257,339 IU/year/participant) to months 19–24 (9751 IU/year/participant; p<0.0001). of the 557 treatment-emergent adverse events reported 24-months post-infusion, 424 (76%) were mild, 115 (21%) were moderate and 18 (3%) were severe.></0.0001).>
A total of 38 participants (70.4%) experienced 93 treatment-related adverse events, with only one occurring during months 18-24. There were no serious adverse effects related to treatment; a serious AE of hepatocellular carcinoma was determined by independent molecular genomic and integration analysis to be unrelated to treatment.
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