Biogen Idec and Swedish Orphan Biovitrum Announce Decision to Advance Long-Lasting Hemophilia A Therapy into a Registrational Trial
Weston, Mass and Stockholm, Sweden - July 9, 2010 - Biogen Idec (NASDAQ: BIIB)
and Swedish Orphan Biovitrum (STO: SOBI) today announced that they plan to
advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion
protein (rFVIIIFc) into a registrational clinical trial in people with
hemophilia A. The decision to advance the program is based on promising data
from a Phase 1/2a open-label, cross-over, multi-center, dose-escalation study
that evaluated the safety and pharmacokinetics of an intravenous injection of
rFVIIIFc in 16 previously-treated patients with severe hemophilia A. In the
study, rFVIIIFc was well tolerated and demonstrated a prolonged half-life
compared to Advate(® )(antihemophilic factor recombinant, plasma/albumin-free
method, rFVIII), supporting advancement of the program.
The primary objective of the Phase 1/2a study was to assess the safety of
rFVIIIFc at different doses; the secondary objective was to estimate the
pharmacokinetic (PK) parameters of rFVIIIFc at doses ranging from 25 to 65
IU/kg. Preliminary results demonstrated that rFVIIIFc's prolonged half-life
compared to Advate(®) was seen consistently across all patients and dose levels,
and other measures of pharmacokinetics including mean residence time and area
under the curve were also increased. There were no signs of injection site
reactions, inhibitor development or anti-rFVIIIFc drug antibodies in the
single-dose study, and there have been no reports of drug-related serious
The treatment of severe hemophilia A requires frequent injections, creating a
significant burden for individuals with the disorder. The potential of rFVIIIFc,
which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion
technology, to prolong protection from bleeding and to reduce the frequency of
injections needed for treatment will be evaluated in the registrational trial.
The global trial, which will commence following communications with regulatory
authorities, is being designed to assess the safety, pharmacokinetics and
efficacy of rFVIIIFc in both the prevention and treatment of bleeding in
hemophilia A patients.
"Biogen Idec is committed to transforming the lives of people with hemophilia by
developing a portfolio of long-lasting therapies," said Glenn Pierce, Vice
President and Chief Medical Officer of Biogen Idec's hemophilia therapeutic
area. "We are excited to announce the advancement of rFVIIIFc into a
registrational study. Like our long-lasting hemophilia B program, which entered
a registrational trial earlier this year, rFVIIIFc offers the potential to make
a significant difference in the lives of people with hemophilia."
"The Phase 1/2a results are very encouraging, and the decision to initiate our
second registrational program in hemophilia represents strong progress in our
efforts to improve the lives of individuals with hemophilia," said Peter Edman,
Ph.D., Chief Scientific Officer of Swedish Orphan Biovitrum. "This is also an
important milestone in the ongoing development of Swedish Orphan Biovitrum."
In June, the European Medicines Agency's Committee for Orphan Medicinal Products
issued a positive opinion regarding the orphan drug application for rFVIIIFc. A
final decision is expected in the coming weeks.
About Hemophilia A
Hemophilia A is a rare, inherited disorder in which the ability of a person's
blood to clot is impaired. Hemophilia A occurs in about one 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. People with hemophilia A
therefore need injections of factor VIII to restore the coagulation process and
prevent frequent bleeds that could otherwise lead to pain, irreversible joint
damage and life-threatening hemorrhages. Prophylaxis treatment with infusions
three times per week or every second day to maintain a sufficient circulating
level of coagulation factor is being increasingly used, and 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
Biogen Idec creates new standards of care in therapeutic areas with high unmet
medical needs. Founded in 1978, Biogen Idec is a global leader in the discovery,
development, manufacturing, and commercialization of innovative therapies.
Patients worldwide benefit from Biogen Idec's significant products that address
diseases such as lymphoma, multiple sclerosis, and rheumatoid arthritis. For
product labeling, press releases and additional information about the company,
About Swedish Orphan Biovitrum
Swedish Orphan Biovitrum is a Swedish-based niche specialty pharmaceutical
company with an international market presence. The company is focused on
providing and developing specialist pharmaceuticals for rare disease patients
with high medical needs. The portfolio consists of about 60 marketed products
and an emerging late-stage clinical development pipeline. Our focus areas are:
hemophilia, inflammation/autoimmune diseases, fat malabsorption, cancer
supportive care and inherited metabolic disorders.
Swedish Orphan Biovitrum had pro-forma revenues 2009e of about 2 BSEK and
approximately 500 employees. The head office is located in Sweden and the share
(STO: SOBI ) is listed on NASDAQ OMX Stockholm. For more information please
For more information contact
Biogen Idec Media Contact: Swedish Orphan Biovitrum
Tracy Vineis, Senior Manager, Public Affairs Peter Edman, CSO
Phone: +1 (781) 464-3260 Phone: +46 8 629 21 77
Biogen Idec Investor Relations Contact: Swedish Orphan Biovitrum
Kia Khaleghpour Erik Kinnman, VP Investor Relations
and Public Affairs
Associate Director, Investor Relations
Phone: +46 73 422 15 40
Phone: +1 (781) 464-2442
This press release contains forward-looking statements regarding the development
of long-lasting hemophilia therapies, which may be identified by words such as
"believe," "expect," "may," "plan," "will" and similar expressions. These
statements are based on the companies' current beliefs and expectation. Drug
development involves a high degree of risk. Factors which could cause actual
results to differ materially from the companies' current expectations include
the risk that we may not fully enroll our planned clinical trials, unexpected
concerns may arise from additional data or analysis, regulatory authorities may
require additional information, further studies, or may fail to approve the
drug, or the companies may encounter other unexpected hurdles. For more detailed
information on the risks and uncertainties associated with Biogen Idec's drug
development and other activities, see the periodic reports of Biogen Idec filed
with the Securities and Exchange Commission. Any forward-looking statements
speak only as of the date of this press release and the companies assume no
obligation to update any forward-looking statements, whether as a result of new
information, future events or otherwise.
Swedish Orphan Biovitrum may be required to disclose the information provided
herein pursuant to the Swedish Securities Markets Act. The information was
provided for public release on July 9, 2010 at 3:30 p.m. CET.
Press Release July 9, 2010: http://hugin.info/134557/R/1430680/377376.pdf
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Source: Swedish Orphan Biovitrum AB (publ) via Thomson Reuters ONE