BAY94-9027: Phase II/III data

The open-label, international Phase II/III PROTECT VIII trial in 134 patients aged 12-65 years who were previously treated with Factor VIII showed that all dose regimens of on-demand and prophylactic treatment with IV BAY 94-9027 met the primary endpoint of reducing annualized bleeding rate from baseline at week 36. Upon enrollment, patients had the option of selecting on-demand or prophylactic treatment. For the 3 prophylactic treatment groups, all patients initially received twice-weekly BAY 94-9027 for 10 weeks. Patients who experienced high bleeding rate, defined as >=1 bleed during the 10-week assessment period, then received higher dose of twice-weekly BAY 94-9027, while all other patients were randomized to receive BAY 94-9027 every 5 or 7 days for 6 months.

In patients receiving BAY 94-9027 every 5 days (n=43), median annualized bleeding rate was 1.9, with 44% of patients experiencing no bleeds at week 36. In patients receiving BAY 94-9027 every 7 days (n=43), median annualized bleeding rate was 3.9, with 37% of patients experiencing no bleeds at week 36. In patients receiving BAY 94-9027 twice weekly (n=13), median annualized bleeding rate was reduced from 17.4 to 4.1. ...