DEERFIELD, Ill. -- (Business Wire)
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.
About von Willebrand Disease1,2
Von Willebrand disease is the most common inherited bleeding disorder
and affects both men and women. Patients with von Willebrand disease
either produce insufficient von Willebrand factor, or carry defective
von Willebrand factor that result in problems with forming clots to stop
bleeding, particularly in mucous membranes such as in the
gastrointestinal (GI) or the urogenital tract. It is estimated that up
to one percent of the world's population suffers from von Willebrand
disease, but many people have only mild symptoms and research has
suggested that as many as nine out of 10 people with von Willebrand
disease have not been diagnosed.
About Baxter in Hemophilia
Baxter has more than 60 years experience in hemophilia and has
introduced a number of therapeutic firsts for hemophilia patients.
Baxter has the broadest portfolio of hemophilia treatments in the
industry and is able to meet individual therapy choices, providing a
range of options at each treatment stage. The company’s work focuses on
optimizing hemophilia care and improving the lives of people worldwide
living with bleeding disorders.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops,
manufactures and markets products that save and sustain the lives of
people with hemophilia, immune disorders, cancer, infectious diseases,
kidney disease, trauma and other chronic and acute medical conditions.
As a global, diversified healthcare company, Baxter applies a unique
combination of expertise in medical devices, pharmaceuticals and
biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning BAX 111
and related clinical studies, including expectations with regard to
future regulatory filings and the potential impact of BAX 111 to
patients. The statements are based on assumptions about many important
factors, including the following, which could cause actual results to
differ materially from those in the forward-looking statements:
satisfaction of regulatory and other requirements; actions of regulatory
bodies and other governmental authorities; additional clinical results;
changes in laws and regulations; product quality or supply or patient
safety issues; and other risks identified in Baxter's most recent filing
on Form 10-K and other SEC filings, all of which are available on
Baxter's website. Baxter does not undertake to update its
forward-looking statements.
1 Berntorp, E. Review. Von Willebrand Disease. Pediatr Blood
Cancer 2013;60:S34–S36.
2 Nichols, WL. Guidelines. von Willebrand disease (VWD):
evidence-based diagnosis and management guidelines, the National Heart,
Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia
(2008), 14, 171–232.
Contacts:
Baxter International Inc.
Media Contact
Brian Kyhos,
(224) 948-5353
media@baxter.com
or
Investor
Contacts
Mary Kay Ladone, (224) 948-3371
Clare Trachtman,
(224) 948-3085
Source: Baxter International Inc.
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