Further data for BAX 111 (highly-purified recombinant von Willebrand Factor) from Phase III study for von Willebrand disease-Baxter

Baxter International Inc. presented additional data from the Phase III clinical trial of BAX 111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of inherited bleeding disorder. The data were presented as an Abstract of Distinction during an oral session at the 2015 Scientific Symposium of the Hemostasis and Thrombosis Research Society (HTRS) in New Orleans, La. and expand on the topline data first disclosed in 2014.

The trial met its primary efficacy endpoint defined by the number of patients who achieved treatment success for control of bleeding episodes. All patients treated in the full analysis set (N=22) experienced a 100 percent treatment success rating based on a 4-point efficacy rating scale, comparing estimated number of infusions needed to treat the bleeding episodes to the actual number of infusions administered. Efficacy for all treated bleeds (N=192) was rated excellent (96.9%) or good (3.1%), including major bleeds (6 excellent and 1 good). The median number of infusions required to treat bleeding events in the trial was 1 and the majority of events (81.8%) were resolved with a single infusion.

The multi-center, open-label clinical trial was designed to assess the safety, efficacy and pharmacokinetics of BAX 111 among patients with severe von Willebrand disease aged 18 to 65 years. No patients developed inhibitors or binding antibodies to the treatment, and there were no reports of thrombotic events or severe allergic reactions. Eight adverse events (AEs) were considered causally related to BAX 111: six non-serious related AEs occurred in four patients, and two related serious AEs (chest discomfort and increased heart rate) occurred in one patient.

Comment: Both the FDA and European Commission granted Orphan Drug status to BAX 111 which was filed at the FDA in December 2014. BAX 111 is the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease. Baxter is preparing to split its biopharma and medical products businesses into separate companies later this year.The biopharma business will be known as Baxalta.