SAN FRANCISCO, July 16, 2015 /PRNewswire/ -- Nektar Therapeutics
(Nasdaq: NKTR) reported that partner Baxalta Incorporated, a global
biopharmaceutical leader dedicated to delivering transformative
therapies to patients with orphan diseases and underserved
conditions, today announced the publication of the complete data
from the Phase II/III pivotal study and Phase I trial of BAX 855 in
Blood, the journal of the American Society of Hematology.
BAX 855 is Baxalta's investigational, extended half-life
recombinant factor VIII (rFVIII) treatment for hemophilia A based
on ADVATE [Antihemophilic Factor (Recombinant)], a leading
treatment for hemophilia A with more than 11 years of real-world
patient experience.
Following on initial presentations of the data in 2014, the
publication provides a comprehensive overview of the clinical trial
results of BAX 855, which will be marketed in the United States under the brand name
ADYNOVATE [Antihemophilic Factor (Recombinant), Pegylated] upon
approval. The trial assessed the treatment's safety and efficacy
profiles for bleed prevention with a twice-weekly dosing schedule,
showing a mean half-life extension of 1.4- to 1.5-fold compared
with ADVATE. The positive study results were originally reported in
August of 2014 and supported the company's December 2014 submission for approval of BAX 855
to the United States Food and Drug Administration (FDA).
"ADYNOVATE has the potential to offer an important new option
for patients, providing the combination of recognized ADVATE bleed
prevention with a simple, twice-weekly dosing schedule," said
Leonard Valentino, M.D., global
head, Hematology Medical Affairs, Baxalta. "The results of this
pivotal trial demonstrate the ability of ADYNOVATE to help further
personalize treatment regimens to minimize the impact that
hemophilia has on patients' lives."
The prospective, global, multi-center, open-label, two-arm Phase
II/III study evaluated BAX 855 among 137 previously treated
patients (PTP) with hemophilia A who were aged 12 to 65. Patients
were assigned to either twice weekly prophylaxis (40-50 IU/kg,
n=120) or on-demand treatment (10-60 IU/kg, n=17). As previously
disclosed, BAX 855 met the study's primary endpoint for the
prevention of bleeding episodes and the treatment with prophylaxis
compared to on-demand treatment. Patients in the twice-weekly
prophylaxis arm of the trial experienced a 95 percent reduction in
median annualized bleed rate (ABR) as compared with those in the
on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also
effective in treating all bleeding episodes, 95.9 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 bleeding
episodes (96.1 percent). In the prophylactic group (n=101), 39.6
percent of compliant patients experienced no bleeds. The study also
showed that BAX 855 pharmacokinetics offered a 1.4 to 1.5-fold mean
extended half-life compared with 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 reactions, were reported. Seven adverse reactions
in six patients, including headache, diarrhea, nausea, and flushing
were reported.
"Upon approval, ADYNOVATE will be one of the first new
treatments that we bring to market as Baxalta, representing a major
milestone toward our plan to bring 20 new products to market by
2020," said Ludwig Hantson, chief
executive officer and president, Baxalta. "With a decades-long
heritage in hematology and commitment to innovation, we continue to
build a patient-centric portfolio that provides new solutions for
evolving patient needs."
Baxalta's Continuation Study is ongoing for patients who
completed the pivotal trial and the pediatric Phase III study among
previously treated patients under the age of 12 with severe
hemophilia A. This continuation study is also available for
patients who have not participated in previous BAX 855 studies.
Upon completion of the pediatric study, Baxalta 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 (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 over 15 years in a number of approved
medicines that treat chronic or serious conditions.
Baxalta leads the industry with the broadest portfolio of
hemophilia treatments, and meets individual therapy choices with a
range of options at each treatment stage. As an estimated 70
percent of people with hemophilia globally remain undiagnosed and
untreated or undertreated, Baxalta is focusing on continuing to
innovate to address some of the greatest challenges associated with
hematologic disorders including hemophilia.
About ADVATE
ADVATE [Antihemophilic Factor (Recombinant)] 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 episode
- 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 for the
treatment of 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 treatments.
ADVATE is the world's most prescribed FVIII treatment. It is
currently approved in 64 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, 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:
www.advate.com/assets/pdf/advate_iri_pi.pdf.
About Baxalta
Baxalta Incorporated is a $6
billion global biopharmaceutical leader developing,
manufacturing and commercializing therapies for orphan diseases and
underserved conditions in hematology, oncology and immunology.
Driven by passion to make a meaningful impact on patients' lives,
Baxalta's broad and diverse pipeline includes biologics with novel
mechanisms and advanced technology platforms such as gene therapy.
The Baxalta Global Innovation and R&D Center is located in
Cambridge, Massachusetts. Launched
in 2015 following separation from Baxter International, Baxalta's
heritage in biopharmaceuticals spans decades. Baxalta's therapies
are available in more than 100 countries and it has advanced
biological manufacturing operations across 12 facilities, including
state-of-the-art recombinant production and plasma fractionation.
Headquartered in Northern
Illinois, Baxalta employs 16,000 employees worldwide.
This release includes forward-looking statements concerning
ADYNOVATE and Baxalta's R&D pipeline, including expectations
with regard to regulatory actions and ADYNOVATE's potential impact
on patients. Such statements are made of the date that they were
first issued and are based on current expectations, beliefs and
assumptions of management. Forward-looking statements are
subject to a number of risks and uncertainties, many of which
involve factors or circumstances that are beyond Baxalta's control
and which could cause actual results to differ materially from
those in the forward-looking statements, including the following:
clinical trial results; satisfaction of regulatory and other
requirements; actions of regulatory bodies and other governmental
authorities; changes in laws and regulations; product quality,
manufacturing or supply issues; patient safety issues; and other
risks identified in Baxalta's Registration Statement on Form 10 and
other Securities and Exchange Commission filings, all of which are
available on Baxalta's website. Baxalta expressly disclaims
any intent or obligation to update these forward-looking statements
except as required by law.
Baxalta, Advate and Adynovate are trademarks of Baxalta
Incorporated
Contact:
Baxalta Media Relations
Kellie Hotz
Central media line: 224-940-2202
Email: media@baxalta.com
Baxalta Investor Relations
Mary Kay Ladone
Central phone: 224-948-3371
mary.kay.ladone@baxalta.com
Lorna Williams
Central phone: 224-948-3511
lorna.williams@baxalta.com
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SOURCE Nektar Therapeutics