BMN 270: Preliminary Phase I/II data

Preliminary data from the first 8 patients with severe hemophilia A in an open-label, dose-escalation, U.K. Phase I/II trial showed that a single IV infusion of BMN 270 improved disease severity per World Federation of Hemophilia (WFH) criteria from baseline to weeks 5-16 to either mild hemophilia or normal in 5 of 6 patients who received the highest dose of BMN 270. The 6th patient improved to moderate disease. A patient receiving an intermediate dose of BMN 270 had moderate disease after 16 weeks and 1 patient receiving a low dose of BMN 270 had severe disease after 20 weeks. Additionally, the only high-dose patient who had 16 weeks of observation had a Factor VIII activity level of 57% of normal. According to WFH, patients with severe hemophilia have Factor VIII activity of <1% of normal. Patients with 1-5% have moderate disease and patients with 5-40% have mild disease. ...