Anyone heard of BAX 111, a potential new treatment for VWD?

Baxter Meets Primary Efficacy Endpoint in Phase 3 Trial of BAX 111, Investigational Stand-Alone Recombinant Treatment for Von Willebrand Disease

DEERFIELD, Ill., April 16, 2014 - Baxter International Inc. (NYSE:BAX) today announced topline results from a Phase 3 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).

"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," said Bruce Ewenstein, M.D., Ph.D., vice president of clinical affairs, in Baxter BioScience. "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 Phase 3 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 anemia (iron deficiency anemia), 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.

Full data from the trial, including efficacy and safety outcomes, will be presented later in 2014. Both the European Commission and the U.S. Food and Drug Administration granted orphan-drug designation for BAX 111 in November 2010. Baxter intends to file for approval in the United States before the end of 2014 and, based on these results, intends to pursue a study of
BAX 111 in a prophylaxis treatment setting before the end of the year.

This is very interesting, but it appears to be another medication that would be given via IV (they mention infusion). I sure wish they would come up with a medication that could just be taken orally to control VWD.

You're right, GG, infusions are definitely inconvenient, so perhaps this treatment would be reserved for more severe cases.

It sounds like a step in the right direction!

I am always hopeful that this can be reasonably managed, and soon cured!

They can provide meds orally for thinning your blood, cholesteral, etc. Still I wonder why they just don't make something to control our clotting that we could just take orally!!