Baxter to Initiate Late-Stage Clinical Trial of BAX 855 for the Treatment of
Company Announces Top-line Phase I Clinical Data
DEERFIELD, Ill. -- January 7, 2013
Baxter International Inc. (NYSE:BAX) today announced it has submitted an
Investigational New Drug (IND) application for its investigational hemophilia
A treatment BAX 855 with the U.S. Food and Drug Administration, following
positive results from a Phase I trial. BAX 855 is a full-length longer-acting
recombinant factor VIII (rFVIII) that was developed to increase the half-life
of ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] –
the most widely chosen rFVIII in the world. Baxter expects to start enrollment
of adult patients in its Phase II/III study in the first quarter of 2013.
''With our focus on the needs of the hemophilia patient community, Baxter
screened more than 100 molecules over several years and has selected one that
has the potential to maintain the safety profile and meaningfully extend the
half-life of ADVATE to allow for less frequent dosing. Importantly, BAX 855 is
the only longer-acting rFVIII in development that is based on a treatment with
nearly a decade of patient experience,'' said Prof. Hartmut J. Ehrlich, M.D.,
vice president of global research and development in Baxter’s BioScience
A Phase I prospective, open-label trial assessing the safety, tolerability and
pharmacokinetics of BAX 855 was conducted in 19 previously treated patients
age 18 years or older with severe hemophilia A who completed the trial. The
half-life (measuring the duration of activity of the drug in the body) of BAX
855 was approximately 1.5-fold higher compared to ADVATE. A longer half-life
was achieved in all patients in the study using BAX 855, no patients developed
inhibitors to either base molecule, BAX 855 or PEG, and no patients had
allergic reactions. Eleven adverse events were reported in eight patients
across both treatment arms, but none was serious, treatment-related or
resulted in withdrawal from the study.
''Efficacy and safety remain the primary goals for choosing a therapy,'' said
David Bevan, M.D., St. Thomas Hospital, London, UK. ''This Phase I study
suggests that BAX 855 retains the underlying attributes of ADVATE and has the
potential for less frequent dosing, creating a potential new option for
hemophilia A patients.''
Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR), BAX 855
leverages proprietary PEGylation technology designed to extend the duration of
activity of proteins. The technology has been shown to be safe and tolerable,
and is used in various approved treatments.
A Phase II/III multi-center, open-label study called PROLONG-ATE will be
initiated in the United States during first quarter of 2013 with more than 100
previously treated adult patients with severe hemophilia A to assess the
efficacy, safety and pharmacokinetics of BAX 855 for prophylaxis and on-demand
treatment of bleeding. Information and enrollment details for the trial are
available at www.clinicaltrials.gov or by emailing email@example.com.
ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] was
initially approved by the FDA in July 2003 for control and prevention of
bleeding episodes in adults and children (0-16 years) with hemophilia A.
ADVATE is a full-length (derived from the complete FVIII gene) recombinant
FVIII product that is processed without any blood-based additives. Because no
blood-derived components are added at any stage of the manufacturing process,
the potential risk of transmitting pathogens that may be carried in
blood-based additives is eliminated. There have been no confirmed reports of
transmission of HIV, HBV or HCV with rFVIII therapies.
ADVATE is approved in 58 countries worldwide including the United States,
Canada, 27 countries in the European Union, Argentina, Australia, Brazil,
Chile, China, Colombia, Croatia, Ecuador, Hong Kong, Iceland, Iraq, Japan,
Kuwait, Macau, Malaysia, Mexico, New Zealand, Norway, Panama, Puerto Rico,
Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Ukraine,
Uruguay, and Venezuela.
In the United States, ADVATE [Antihemophilic Factor (Recombinant)
Plasma/AlbuminFree Method] is also indicated for routine prophylaxis to
prevent or reduce the frequency of bleeding episodes in adults and children
(0-16 years) with hemophilia A. ADVATE is the only antihemophilic recombinant
FVIII treatment approved in the United States for prophylactic use in both
adults and children. ADVATE is not indicated for the treatment of von
Detailed Important Risk Information for ADVATE
ADVATE is contraindicated in patients with known anaphylaxis to mouse or
hamster protein or other constituents of the product.
Allergic-type hypersensitivity reactions, including anaphylaxis, are possible
and have been reported with ADVATE. Symptoms have manifested as dizziness,
paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus.
Discontinue use if hypersensitivity symptoms occur and administer appropriate
Carefully monitor patients treated with AHF products for the development of
FVIII inhibitors by appropriate clinical observations and laboratory tests.
Inhibitors have been reported following administration of ADVATE predominantly
in previously untreated patients (PUPs) and previously minimally treated
If expected plasma FVIII levels are not attained, or if bleeding is not
controlled with an expected dose, perform an assay that measures FVIII
The serious adverse reactions seen with ADVATE are hypersensitivity reactions
and the development of high-titer inhibitors necessitating alternative
treatments to FVIII.
The most common adverse reactions observed in clinical trials (frequency
greater than or equal to 10 percent of patients) were pyrexia, headache,
cough, nasopharyngitis, vomiting, arthralgia, and limb injury.
Please see full prescribing information for ADVATE at:
About Hemophilia A
Hemophilia is a rare genetic blood clotting disorder that primarily affects
males.^1 People living with hemophilia do not have enough of, or are missing,
one of the blood clotting proteins naturally found in blood.^1 Two of the most
common forms of hemophilia are A and B.^2 In people with hemophilia A,
clotting factor VIII is not present in sufficient amounts or is absent.^2
Without enough FVIII, people with hemophilia can experience spontaneous,
uncontrolled internal bleeding that is painful, debilitating, damaging to
joints and potentially fatal.^2 According to the World Federation of
Hemophilia, more than 400,000 people in the world have hemophilia.^3,4 All
races and economic groups are affected equally.^3,4
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 855 and
related clinical studies.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 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. What is Hemophilia? World Federation of Hemophilia. Accessed on: May 24,
2012. Available at: http://www.wfh.org/en/page.aspx?pid=646
2.Frequently Asked Questions About Hemophilia. World Federation of
Hemophilia. Accessed on: May 24, 2012. Available at:
3. Stonebreaker et al “A study of variations in the reported haemophilia A
prevalence around the world” Haemophilia 2010. Jan;16(1):20-32
4. Guidelines for the Management of Hemophilia at WFH website Last Accessed
on: December 17,2012 Available at: http://www.wfh.org/en/page.aspx?pid=1270
Baxter International Inc.
Brian Kyhos, (224) 948-4210
Deborah Spak, (224) 948-2349
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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