DEERFIELD, Ill., Aug. 21, 2014 /PRNewswire/ -- Nektar Therapeutics
(NASDAQ: NKTR) reports Baxter International Inc. today announced
positive results from its Phase 3 pivotal clinical trial of BAX
855, an investigational, extended half-life recombinant factor VIII
(rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic
Factor (Recombinant)], which met its primary endpoint in reducing
annualized bleed rates (ABR) in the prophylaxis arm compared to the
on-demand arm.
Top-line results from the prospective, global, multi-center
Phase 3 study demonstrated that BAX 855 met its primary endpoint in
the control and prevention of bleeding, routine prophylaxis and
perioperative management for patients who were 12 years or older.
Patients in a twice-weekly prophylaxis arm experienced a 95 percent
reduction in median ABR as compared to those in the on-demand arm
(1.9 vs. 41.5, respectively). BAX 855 was also effective in
treating bleeding episodes, 96 percent of which were controlled
with one or two infusions. The half-life of BAX 855 was 1.4 - 1.5
times that of ADVATE, consistent with the findings from the Phase 1
study. No patients developed inhibitors to BAX 855 and no
treatment-related serious adverse events, including
hypersensitivity, were reported. The most common (three patients)
product-related adverse event was headache.
"The positive results of the BAX 855 study reflect our ongoing,
long-term commitment to drive innovation and expand treatment
options for patients with hemophilia," said John Orloff, M.D., vice president and global
head of research and development for Baxter BioScience. "We look
forward to advancing the BAX 855 program to U.S. regulatory
submission by the end of this year."
The multi-center, open-label study evaluated BAX 855 among 138
adolescent and adult patients with previously-treated hemophilia A.
Patients received treatment twice weekly (45 IU/kg) or on-demand,
and were followed for six months. The primary objective of the
study was the reduction in ABR during the treatment period compared
to on-demand treatment. The study also evaluated the safety and
immunogenicity of the compound when administered on either
prophylaxis or on-demand treatment regimens.
Baxter expects to submit a Biologics License Application (BLA)
for BAX 855 to the U.S. Food and Drug Administration (FDA) before
the end of 2014 and will present additional data in the coming
months. In addition to an ongoing continuation study for patients
who have completed the pivotal trial, the company is initiating a
Phase 3, prospective, open-label, multi-center study to evaluate
the safety and efficacy of BAX 855 among 60 previously treated
patients under the age of 12 with severe hemophilia A. Consistent
with guidelines published by the European Medicines Agency (EMA)
that require a study in children less than 12 years of age prior to
filing, Baxter expects to file a Marketing Authorization
Application with the EMA upon the completion of the pediatric
study.
BAX 855 is based on ADVATE, a full-length FVIII molecule with
more than 10 years of real-world experience. Through a
collaboration with Nektar Therapeutics (NASDAQ: NKTR), BAX 855
leverages proprietary PEGylation technology designed to extend the
duration of activity of proteins in the body. This proprietary
technology has been used for over 10 years in a number of approved
medicines that treat chronic or serious conditions.
About ADVATE
ADVATE is a recombinant antihemophilic
factor indicated for use in children and adults with hemophilia A
(congenital factor VIII deficiency or classic hemophilia) for:
- Control and prevention of bleeding episodes.
- Perioperative management.
- Routine prophylaxis to prevent or reduce the frequency of
bleeding episodes.
ADVATE is not indicated for the treatment of von Willebrand
disease.
ADVATE has a demonstrated efficacy and safety profile. 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 treatments.
ADVATE is approved in 64 countries worldwide, including
the United States, Canada, 27 countries in the European Union,
Algeria, Argentina, Australia, Brazil, Chile, China,
Colombia, Croatia, Ecuador, Hong
Kong, Iceland, Iraq, Japan,
Kuwait, Macau, Malaysia, Mexico, Morocco, New
Zealand, Norway,
Panama, Puerto Rico, Russia, Serbia, Singapore, South
Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Detailed Important Risk Information for ADVATE
CONTRAINDICATIONS
ADVATE is contraindicated in patients who
have life-threatening hypersensitivity reactions, including
anaphylaxis, to mouse or hamster protein or other constituents of
the product.
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Allergic-type hypersensitivity
reactions, including anaphylaxis, have been reported with ADVATE.
Symptoms include dizziness, paresthesia, rash, flushing, facial
swelling, urticaria, dyspnea, and pruritus.
Discontinue ADVATE if hypersensitivity symptoms occur and
administer appropriate emergency treatment.
Neutralizing Antibodies
Neutralizing antibodies
(inhibitors) have been reported following administration of ADVATE
predominantly in previously untreated patients (PUPs) and
previously minimally treated patients (MTPs). Monitor all patients
for the development of factor VIII inhibitors by appropriate
clinical observation and laboratory testing. If expected plasma
factor VIII activity levels are not attained, or if bleeding is not
controlled with an expected dose, perform an assay that measures
factor VIII inhibitor concentration.
ADVERSE REACTIONS
The serious adverse reactions seen
with ADVATE are hypersensitivity reactions and the development of
high-titer inhibitors necessitating alternative treatments to
factor VIII.
The most common adverse reactions observed in clinical trials
(frequency >10% of subjects) were pyrexia, headache, cough,
nasopharyngitis, vomiting, arthralgia, and limb injury.
Please see full prescribing information for ADVATE at:
http://www.baxter.com/downloads/healthcare_professionals/
products/ADVATE_PI.pdf
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, including expectations with
regard to regulatory filings. 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.
SOURCE Nektar Therapeutics; Baxter International Inc.