Roche reports interim data from Phase III HAVEN 2 trial of emicizumab for hemophilia A

Roche (SIX:ROG; OTCQX:RHHBY) and its Genentech Inc. unit reported interim data from 19 evaluable patients ages <12 with hemophilia A with Factor VIII inhibitors who were previously treated with episodic or prophylactic bypassing agents in the Phase III HAVEN 2 trial showing that once-weekly subcutaneous emicizumab (ACE910, RG6013, RO5534262) as prophylaxis led 1 reported treated bleed at a median observation time of 12 weeks. No joint or muscle bleeds were reported. In 8 evaluable patients previously enrolled in a non-interventional study, emicizumab reduced treated bleeds by 100%. Data will be presented at International Society on Thrombosis and Haemostasis meeting in Berlin this month.

The 52-week, open-label, international trial is enrolling 60 patients ages ≤17. The primary endpoint is number of treated bleeds over time. Secondary endpoints include all bleed rate, joint bleed rate, target joint bleed rate, quality of life (QOL) scores, antibodies, pharmacokinetics and safety...