Baxter's BioScience Unit Announces Positive Phase III Results for BAX 817

Baxter International Inc.

today 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 µg/kg or 1x270 µg/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. ''The development of inhibitors remains one of the most significant challenges in treating hemophilia, as it may place patients at increased risk for life-threatening complications resulting from difficult-to-treat bleeding episodes,'' said John Orloff, M.D., vice president and global head of research and development at Baxter BioScience. ''These positive results reflect our commitment to addressing the complex treatment of hemophilia patients with inhibitors, and reinforce our legacy of advancing hemophilia care worldwide.'' 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. The company plans to initiate regulatory submissions aligned to manufacturing expansions currently underway. Baxter remains committed to advancing our portfolio in inhibitor management. If approved, BAX 817 would broaden the existing portfolio of hemophilia and inhibitor treatments within Baxter's biopharmaceutical business, including FEIBA [Anti-Inhibitor Coagulant Complex] and the recently approved OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] for acquired hemophilia A.