News Feature | April 17, 2014

Baxter's Rare Disease Drug Meets Phase 3 Endpoint

By Estel Grace Masangkay

Baxter International reported top line results from the Phase III clinical trial evaluating the safety and efficacy of its orphan drug candidate BAX 111.

BAX 111 is a recombinant von Willebrand factor (rVWF) being evaluated for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder. The investigational compound was developed using a plasma- and albumin-free manufacturing method.           

The open-label, multi-center, Phase III trial met its primary efficacy endpoint, with all patients achieving pre-specified success in the on-demand treatment of bleeding events. Secondary endpoints included added efficacy and safety measures, health-related quality of life (HRQoL), and pharmacokinetics. Thirty-seven patients with severe von Willebrand disease were administered BAX111 together with ADVATE or as a stand-alone therapeutic agent. The study was conducted in trial sites in the U.S., EU, Japan, Russia, Australia, and India.

Bruce Ewenstein, VP of clinical affairs in Baxter BioScience, said, “As the first recombinant, stand-alone treatment in development, BAX 111 has the potential to offer people with von Willebrand disease a new therapeutic option that may allow for greater precision and flexibility in managing the disease. With these findings, we have taken another significant step forward as we continue to expand on our increasingly broad pipeline of potential treatments to improve outcomes for patients with a range of bleeding disorders.”

The U.S. Food and Drug Administration (FDA) and the European Commission both granted Orphan Drug designation to BAX111 in November 2010. The company said it plans to file for approval in the U.S. based on the results of the trial before the end of the year. Baxter also announced its intent to pursue a study of the investigational compound in a prophylaxis treatment setting.

The company said it will present full data from the trial including safety and efficacy outcomes later in 2014.