BAX 111 completes successful Phase III trial for von Willebrand Disease - Baxter

Baxter International Inc. announced topline results from a Phase III clinical trial evaluating the safety, efficacy and pharmacokinetics (PK) of BAX 111. BAX 111 is a recombinant von Willebrand factor (rVWF) under investigation for the treatment of bleeding episodes in patients with von Willebrand Disease, the most common type of inherited bleeding disorder.

The study of BAX 111, the first recombinant treatment in clinical development for this condition, met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (100%, 22 of 22 patients who experienced bleeds in the trial).

The Phase III multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 administered together with Advate or as a stand-alone therapeutic agent in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the United States, Europe, Australia, Japan, Russia and India. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy and safety measures, pharmacokinetics and health-related quality of life (HRQoL). There were no reports of inhibitor development or thrombotic events in the study participants. The most common adverse events in the study were headache, vomiting/nausea and anaemia (iron deficiency anaemia), which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication. The investigational treatment was developed using a plasma- and albumin-free manufacturing method.