Baxter to Initiate Late-Stage Clinical Trial of BAX 855 for the Treatment of Hemophilia A
Company Announces Top-line Phase I Clinical Data
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 business.
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 medinfo@baxter.com.
About ADVATE
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 Willebrand disease.
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 emergency treatment.
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 patients (MTPs).
If expected plasma FVIII levels are not attained, or if bleeding is not
controlled with an expected dose, perform an assay that measures FVIII
inhibitor concentration.
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: www.baxter.com/downloads/healthcare_professionals/products/ADVATE_PI.pdf
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: http://www.wfh.org/en/page.aspx?pid=637
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