Phase III HAVEN 1 study evaluating emicizumab prophylaxis reports progress in treating patients with hemophilia A and inhibitors to Factor VIII.- Genentech + Roche

Genentech, a member of the Roche Group announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia A and inhibitors to factor VIII. The study showed a statistically significant reduction in the number of bleeds over time in people treated with emicizumab prophylaxis compared to those receiving no prophylactic treatment. The study also met all secondary endpoints, including a statistically significant reduction in the number of bleeds over time with emicizumab prophylaxis treatment in an intra-patient comparison in people who had received prior bypassing agent prophylaxis treatment.

The most common adverse events with emicizumab were injection site reactions, consistent with prior studies.As previously reported, two patients had thromboembolic events and two patients developed thrombotic microangiopathy (TMA). The common aspect between all cases of thromboembolic events and TMA is that they occurred in patients who were on emicizumab prophylaxis and in addition received activated prothrombin complex concentrate to treat breakthrough bleeds. Neither thromboembolic event required anti-coagulation therapy and one patient restarted emicizumab. Both cases of TMA have completely resolved, and one patient restarted emicizumab.

HAVEN 1 is the first Phase III study in the emicizumab clinical development program to report results. Data from the study will be presented at an upcoming medical meeting and submitted to health authorities around the world for approval consideration.