DEERFIELD, Ill., Feb. 11, 2015 /PRNewswire/ -- Nektar Therapeutics
(NASDAQ:NKTR) reported that partner Baxter International Inc. today
presented additional efficacy and safety data from the Phase III
pivotal study of BAX 855, an investigational, extended half-life
recombinant factor VIII (rFVIII) treatment for hemophilia A based
on ADVATE [Antihemophilic Factor (Recombinant)] at the
8th Annual Congress of the European Association for
Haemophilia and Allied Disorders (EAHAD) in Helsinki, Finland.
The new data expand on the previously disclosed topline results
from the pivotal trial, which found that BAX 855 met the study's
primary endpoint in the control and prevention of bleeding episodes
and routine prophylaxis. Patients in the twice-weekly prophylaxis
arm of the trial experienced a 95 percent reduction in median
annualized bleed rate (ABR) as compared to those in the on-demand
arm (1.9 vs. 41.5, respectively). The study findings supported
Baxter's December 2014 submission for
approval of BAX 855 to the United States Food and Drug
Administration (FDA).
"These pivotal trial results provide evidence to support the
efficacy profile of BAX 855 in controlling, preventing or reducing
the frequency of bleeding episodes when administered
prophylactically twice weekly. Our goal with BAX 855 is to extend
the interval between infusions while maintaining a similar efficacy
profile to ADVATE," said John
Orloff, M.D., vice president and global head of research and
development for Baxter BioScience.
The prospective, global, multi-center, open-label, two-arm Phase
III study evaluated BAX 855 among 137 previously treated hemophilia
A patients (PTP) who were 12 years or older. Patients were assigned
either to twice weekly prophylaxis (40-50 IU/kg, n=120) or
on-demand treatment (10-50 IU/kg, n=17). In addition to a reduced
ABR, BAX 855 was also effective in treating bleeding episodes, 96
percent of which were controlled with one or two infusions at a
median dose of 29.0 IU/kg per infusion. Treatment was rated
excellent or good for nearly all episodes (96.2%). In the
prophylactic group (n=101), 40 percent of patients experienced no
bleeds. The study also showed that BAX 855 pharmacokinetics offered
a 1.4-1.5-fold extended half-life compared to ADVATE with a median
infusion interval of 3.6 days, supporting the findings from the
Phase I trial.
No patients developed inhibitors to BAX 855 and no
treatment-related serious adverse events, including
hypersensitivity, were reported. Seven adverse reactions in six
patients, including headache, diarrhea, nausea, and flushing were
reported.
Baxter's continuation study for patients who completed the
pivotal trial and the Phase 3 study among previously treated
patients under the age of 12 with severe hemophilia A remain
ongoing. Upon completion of the pediatric study, Baxter expects to
file for marketing authorization with the European Medicines Agency
in 2016.
BAX 855 is based on ADVATE, a full-length FVIII molecule with
more than 11 years of real-world patient experience. Through a
collaboration with Nektar Therapeutics (NASDAQ: NKTR), BAX 855
leverages proprietary PEGylation technology designed to prolong the
amount of factor VIII available for use in the body. This
proprietary technology has been used for 15 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 the world's most prescribed FVIII treatment. It is
currently approved in 66 countries worldwide, including
the United States, Canada, 28 countries in the European Union,
Algeria, Argentina, Australia, Brazil, Chile, China,
Colombia, Ecuador, Hong
Kong, Iceland, Iraq, Israel,
Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New
Zealand, Norway,
Panama, Puerto Rico, Qatar, Russia, Saudi
Arabia, Serbia, Singapore,
South Korea, Suriname,
Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Detailed Important Risk Information for ADVATE
[Antihemophilic Factor (Recombinant)]
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 BioScience
Baxter BioScience is a
leading provider of therapeutic treatments that save, sustain and
improve the lives of people with rare conditions, chronic diseases
or limited treatment options. Supported by advanced technical and
manufacturing expertise, Baxter BioScience has a broad pipeline
built on a legacy of innovation in bleeding disorders and
immunology and is expanding to address emerging opportunities in
niche areas of oncology as well as technology platforms such as
biosimilars.
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, including expectations with regard to clinical studies and
related data, regulatory filings, and its potential impact 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, manufacturing or supply issues, 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.
Media Contact
Brian
Kyhos
(224) 948-5353, media@baxter.com
Investor Contact
Mary Kay
Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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SOURCE Nektar Therapeutics