Genentech announced that the Phase 3 HAVEN 1 study, which evaluated emicizumab (ACE910) as prophylaxis therapy for patients aged ≥12 years with hemophilia A and Factor VIII inhibitors, met its primary endpoint and all secondary endpoints.
HAVEN 1 was a randomized, multicenter, open-label study that evaluated the safety, efficacy, and pharmacokinetics of emicizumab prophylaxis for once-weekly subcutaneous (SC) injection (n=109). The study enrolled patients aged ≥12 years with Factor VIII inhibitors who were treated previously with episodic or prophylactic bypassing agents.
The primary endpoint was the number of bleeds over time among patients with emicizumab prophylaxis or no prophylaxis. Secondary endpoints included all bleed rate, joint bleed rate, spontaneous bleed rate, target joint bleed rate, health-related quality of life/health status, intra-patient comparison to bleed rate on their prior prophylaxis regimen with bypassing agents and safety.
In the study, there was a statistically significant reduction in the number of bleeds in patients treated with emicizumab prophylaxis vs. patients who did not receive prophylaxis. In addition, there was a statistically significant reduction in the number of bleeds over time with emicizumab prophylaxis treatment in an intra-patient comparison in patients who received prior bypassing agent prophylaxis.
Emicizumab, an investigational bispecific monoclonal antibody, was designed with the Company’s proprietary antibody engineering technologies. It binds to Factors IXa and X while promoting the interaction between these factors.
Emicizumab was granted Breakthrough Therapy Designation by the Food and Drug Administration (FDA) in 2015.
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