Biogen
Idec (NASDAQ:BIIB) and Swedish Orphan Biovitrum (STO:SOBI) today
announced the initiation of two global pediatric clinical trials of the
companies’ long-lasting recombinant Factor VIII and Factor IX Fc fusion
proteins (rFVIIIFc and rFIXFc) in hemophilia A and B.
rFVIIIFc and rFIXFc are fully-recombinant clotting factors developed
using Biogen Idec's novel and proprietary monomeric Fc-fusion
technology, which makes use of a natural mechanism to recycle rFVIIIFc
and rFIXFc in the circulation in the body. Biogen Idec and Sobi are
studying this technology to see whether it extends half-life and enables
the proteins to last longer in the body than commercially-available
factor products.
“Children with hemophilia share a need for long-lasting factor products
that may prolong protection from bleeding and reduce the frequency of
intravenous infusions,” said Glenn Pierce, M.D., Ph.D., senior vice
president of Global Medical Affairs and chief medical officer of Biogen
Idec’s hemophilia therapeutic area. “The pediatric clinical trials are
an important complement to the ongoing adult studies and further
demonstrate our commitment to develop new treatments for the hemophilia
community.”
“With these studies, we extend the clinical development of rFVIIIFc and
rFIXFc to address a broader patient population including children with
hemophilia,” said An van Es-Johansson, M.D., head of clinical
development at Sobi. “The initiation of these studies represents an
important milestone for our clinical development program and supports
our global registration strategy for the products.”
The clinical trials are open-label, multicenter studies designed to
evaluate the safety, pharmacokinetics and efficacy of rFVIIIFc and
rFIXFc in previously-treated children with severe hemophilia A or B
under the age of 12 years. The first patient has been dosed in the
rFIXFc pediatric study, and the rFVIIIFc pediatric study is now actively
recruiting patients. Global registration studies of rFVIIIFc and rFIXFc
are ongoing in previously-treated patients with severe hemophilia A or B
aged 12 years and over, with data readouts expected in the second half
of the year.
About the Kids A-LONG study
The Kids A-LONG study is an open-label, multicenter study designed to
evaluate the safety, pharmacokinetics and efficacy of rFVIIIFc in the
prevention and treatment of bleeding episodes in previously-treated
pediatric patients with hemophilia A. The study will include
approximately 50 male patients under the age of 12 who have a diagnosis
of severe hemophilia A and a history of at least 50 documented prior
exposure days with any currently-marketed Factor VIII product. All
patients will be treated with a twice weekly prophylactic dosing regimen.
The primary outcome measure is the frequency of inhibitor development
over an approximately 26-week treatment period and at least 50 exposure
days to rFVIIIFc. Secondary outcome measures include the number of
annualized bleeding episodes and assessments of response to treatment
over the same time period. For more information on the study, visit www.biogenidechemophilia.com
or www.clinicaltrials.gov
(NCT01458106).
About the Kids B-LONG study
The Kids B-LONG study is an open-label, multicenter study designed to
evaluate the safety, pharmacokinetics and efficacy of rFIXFc in the
prevention and treatment of bleeding episodes in previously-treated
pediatric patients with hemophilia B. The study will include
approximately 26 male patients under the age of 12 who have a diagnosis
of severe hemophilia B and a history of at least 50 documented prior
exposure days with any currently-marketed Factor IX product. All
patients will be treated with a weekly prophylactic dosing regimen.
The primary outcome measure is the frequency of inhibitor development
over an approximately 50-week treatment period and at least 50 exposure
days to rFIXFc. Secondary outcome measures include the number of
annualized bleeding episodes and assessments of response to treatment
over the same time period. For more information on the study, visit www.biogenidechemophilia.com
or www.clinicaltrials.gov
(NCT01440946).
About Hemophilia
Hemophilia is a rare, inherited disorder in which the ability of a
person's blood to clot is impaired. Hemophilia A occurs in about 1 in
5,000 male births annually and is caused by having substantially reduced
or no Factor VIII protein, which is needed for normal blood clotting.
Hemophilia B occurs in about 1 in 25,000 male births annually and is
caused by having substantially reduced or no Factor IX protein. People
with hemophilia A and B therefore need injections of Factor VIII and
Factor IX, respectively, to restore the coagulation process and prevent
frequent bleeds that could otherwise lead to pain, irreversible joint
damage and life-threatening hemorrhages. Prophylaxis treatment for
hemophilia A and B requires infusions three to four times a week or two
times a week, respectively, to maintain a sufficient circulating level
of coagulation factor. Long-term studies demonstrate that such regimens
increase the patient's life expectancy and greatly reduce if not
eliminate progressive joint deterioration.
About Biogen Idec
Through cutting-edge science and medicine, Biogen Idec discovers,
develops and delivers to patients worldwide innovative therapies for the
treatment of neurodegenerative diseases, hemophilia and autoimmune
disorders. Founded in 1978, Biogen Idec is the world's oldest
independent biotechnology company. Patients worldwide benefit from its
leading multiple sclerosis therapies, and the company generates more
than $5 billion in annual revenues. For product labeling, press releases
and additional information about the company, please visit www.biogenidec.com.
About Swedish Orphan Biovitrum (Sobi)
Sobi is a leading integrated biopharmaceutical company dedicated to
bringing innovative therapies and services to improve the health of rare
disease patients and their families. The product portfolio comprises
about 45 marketed products as well as projects in the late clinical
phase. Key therapeutic areas are Inflammation and Genetics & Metabolism.
In 2011 Sobi had revenues of SEK 1.9 billion and around 500 employees.
The share (STO: SOBI) is listed on OMX NASDAQ Stockholm. More
information is available at www.sobi.com.
