Baxter International Inc. 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.
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