Genentech: Emicizumab Phase III Studies Show Substantial Reduction In Bleeds

Genentech, a member of the Roche Group ( RHHBY ), reported positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII. The Phase III HAVEN 1 study compared emicizumab prophylaxis with on-demand and prophylactic use of bypassing agents in adults and adolescents with hemophilia A with inhibitors. The primary endpoint was treated bleeds, and results showed a statistically significant and clinically meaningful reduction in bleed rate of 87 percent with emicizumab prophylaxis compared to on-demand treatment with bypassing agents.

The company stated that adverse events occurring in five percent or more of patients treated with emicizumab were injection site reactions, headache, fatigue, upper respiratory tract infection and arthralgia. Serious adverse events of thromboembolic events and thrombotic microangiopathy occurred in two patients and three patients, respectively, while receiving emicizumab prophylaxis.