Successful Phase III trial for BAX 817 for haemophilia inhibitor treatment-Baxter

Baxter International Inc. announced positive results from its Phase III clinical trial evaluating the safety and efficacy of BAX 817, an investigational recombinant factor VIIa (rFVIIa) treatment for people with hemophilia A or B who develop inhibitors. The prospective, open-label, randomized, multicenter trial was designed to assess the safety and efficacy of BAX 817 in male patients ages 12 to 65 with hemophilia A or B with inhibitors over a 6-month period using on-demand therapy. The trial met its primary endpoint of successful resolution of acute bleeding episodes at 12 hours with both on-demand treatment regimens, dosing either 3x90 �g/kg or 1x270 �g/kg, with an overall success rate of 92 percent (98 percent and 85 percent in each dosing group, respectively). Further, 89 percent of patients in the trial achieved sustained bleeding control for all acute bleeding episodes 24 hours after infusion.

No patients developed inhibitors or binding antibodies to BAX 817, and none discontinued treatment due to an adverse event (AE). One patient was hospitalized following a traumatic muscle bleed that did not respond to BAX 817. Non-serious AEs observed in the trial were generally consistent with the underlying disease or other etiology, and were all deemed to be unrelated to treatment. Full data from the trial, including additional efficacy and safety outcomes, will be presented at a medical meeting later in 2015.

Comment: People with hemophilia use factor clotting concentrates. This treatment improves blood clotting and is used to stop or prevent a bleeding episode. Inhibitors develop when the body�s immune system stops accepting the factor (factor VIII for hemophilia A and factor IX for hemophilia B) as a normal part of blood. The body thinks the factor is a foreign substance and tries to destroy it using inhibitors. The inhibitors stop the factor from working. This makes it more difficult to stop a bleeding episode. People with hemophilia who develop an inhibitor do not respond as well to treatment. Inhibitors most often appear during the first year of treatment.