NEW PHASE 3 DATA REINFORCE LONG-LASTING PROTECTION FROM BLEEDING FOR PATIENTS WITH HEMOPHILIA A AND B
BIOGEN IDEC CONTACT:
Media Contact: Jeff Boyle
+1-781-464-3260
Investor Relations Contact: Kia Khaleghpour
Ph: +1-781-464-2442
SWEDISH ORPHAN BIOVITRUM CONTACT:
Media Contact: Birgitte Volck
Ph: +45 4 012 2310/+46 8 697 2188
Analyst/Investor Contact: Jorgen Winroth
Ph: +46 8 697 2000/+1-212-579-0506
- Recombinant Fc fusion proteins show potential to transform care by providing
long-lasting protection from bleeding with fewer injections than the current
standard of care -
WARSAW, Poland - February 8, 2013 - Biogen Idec (NASDAQ: BIIB) and Swedish
Orphan Biovitrum (Sobi) (STO: SOBI) released data that confirmed the ability of
investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc
fusion protein (rFIXFc) to provide long-lasting protection from bleeding with
fewer injections than are required with the current standard of care for people
with hemophilia. The data, from the largest phase 3 registrational studies
conducted in hemophilia to date, were presented this week at the 6th Annual
Congress of the European Association for Haemophilia and Allied Disorders
(EAHAD).
The studies compared the pharmacokinetic activity of rFVIIIFc for hemophilia A
and rFIXFc for hemophilia B to currently available treatments. In the studies,
the long-lasting candidates stayed active in the body longer, enabling study
participants to prevent bleeding with less frequent injections than are required
with the current standard of care. In the A-LONG study, patients with hemophilia
A were able to use once to twice weekly prophylactic (preventative dosing)
injections of rFVIIIFc while maintaining low bleeding rates. In the B-LONG
study, rFIXFc allowed patients with hemophilia B to use prophylactic injections
every one to two weeks with low bleeding rates.
"Data from these phase 3 trials demonstrate a potential to transform the
treatment of hemophilia by offering long-lasting protection from bleeding while
meaningfully reducing treatment burden associated with this rare disease," 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. "Less
frequent injections may help more people with hemophilia adhere to a
preventative treatment schedule, which can help prevent the long-term health
consequences associated with treating a bleed after it occurs."
The current standard of care for hemophilia A and B requires frequent
injections, which are a burden for patients. Prophylactic treatment for
hemophilia A typically requires injections three times per week or every other
day, and injections 2-3 times per week for the treatment of hemophilia B,
according to the National Hemophilia Foundation's Medical and Scientific
Advisory Council guidelines. People with severe hemophilia who do not follow a
prophylactic injection schedule remain vulnerable to bleeding that can cause
irreversible joint damage and life-threatening hemorrhages.
Recombinant FVIIIFc and recombinant FIXFc were developed using Fc fusion
technology, which has safely been used in FDA-approved medicines for more than a
decade. Biogen Idec and Swedish Orphan Biovitrum applied Fc fusion technology in
hemophilia for the first time with the goal of making clotting factors last
longer and reduce the burden of injections for patients and their families.
About the A-LONG (rFVIIIFc) Data at EAHAD
The A-LONG results confirm the long-lasting characteristics of rFVIIIFc;
specifically, the data show that rFVIIIFc stays in the body for 50 percent
longer than Advate(®) [antihemophilic factor (recombinant), plasma/albumin-free
method], the most frequently used factor VIII therapy. In the trial, the
terminal half-life for rFVIIIFc was 19 hours compared to 12 hours for Advate.
Other measures of rFVIIIFc's activity in the body reinforce its long-lasting
characteristics: the mean time for maintaining a clotting factor activity level
associated with less bleeding (time to 1 percent) was approximately 5 days for
rFVIIIFc compared to 3.5 days for Advate and the average rate at which rFVIIIFc
was cleared from the body was 2.0 mL/hr/kg compared with 3.0 mL/hr/kg for
Advate. In the study's individualized prophylaxis arm, patients received
rFVIIIFc at a median dosing interval of 3.5 days and a median weekly dose of 78
IU per kg to prevent bleeding, which compares favorably to the recommended dose
for the standard of care. Nearly one-third of patients were able to achieve
every 5 day dosing in this arm. Overall, the A-LONG data indicate that rFVIIIFc
has the potential to become the first product to offer hemophilia A patients
long-lasting protection from bleeding with less frequent dosing than the current
standard of care.
The A-LONG data were presented in the late-breaking oral abstract session and in
poster 104, "Phase 3 clinical study of recombinant FC fusion factor FVIII
(rFVIIIFc) demonstrated safety, efficacy, and improved pharmacokinetics (A-
LONG)."
About the B-LONG (rFIXFc) Data at EAHAD
The B-LONG results confirm the long-lasting characteristics of rFIXFc;
specifically, the data show that rFIXFc stays in the body for more than twice as
long as BeneFIX(®) [Coagulation Factor IX (Recombinant)], the only recombinant
factor IX therapy currently approved for prophylactic use. The terminal half-
life for rFIXFc was 82 hours compared to 34 hours for BeneFIX. Other measures of
rFIXFc's activity in the body reinforce its long-lasting characteristics: the
mean time for maintaining a normal clotting factor activity level (time to 1
percent) was 11 days for rFIXFc compared to 5 days for BeneFIX and the average
rate at which rFIXFc was cleared from the body was 3.2 mL/hr/kg compared with
6.3 mL/hr/kg for BeneFIX. All patients in the individualized interval
prophylaxis arm of the study were able to go at least one week between rFIXFc
injections and 50 percent were able to go 14 days or longer before needing
another dose to prevent bleeding. The median weekly dose was 45 IU per kg,
comparable to the recommended dose for the current standard of care. Overall,
the B-LONG data support the potential for rFIXFc to become the first product to
offer hemophilia B patients long-lasting protection from bleeding with a more
convenient injection schedule than the current standard of care.
The B-LONG data were presented in poster 115, "Safety, efficacy, and improved
pharmacokinetics (PK) demonstrated in a phase 3 clinical trial of extended half-
life recombinant FC fusion factor IX (B-LONG)."
Importantly, the difference in the duration of activity of rFVIIIFc and rFIXFc
was expected and consistent with the differences between the natural clotting
factors that these products augment. Fc Fusion technology extends FVIII and FIX
differently based on the biological differences in hemophilia A and B. The
length of time that FVIII stays active is dictated by its own duration as well
as that of the blood protein that it binds to, known as von Willebrand factor.
The activity of FIX is not restricted in this way.
"These new data support the application of Fc fusion technology in hemophilia,
using a naturally occurring pathway to delay the breakdown of factor in the body
and cycle it back into the bloodstream," said Birgitte Volck, M.D., Ph.D.,
senior vice president & chief medical officer of Sobi. "While Fc fusion has been
used in medicines for more than a decade, rFVIIIFc and rFIXFc are the first
investigational therapies to use the technology to successfully extend the half-
lives of clotting factors, which could offer protection from bleeding while
reducing the burden of injections for people with hemophilia."
About the A-LONG Study and the rFVIIIFc Program
A-LONG was a global, open-label, multi-center phase 3 study that evaluated the
efficacy, safety and pharmacokinetics of intravenously-injected rFVIIIFc in 165
male patients aged 12 years and older. The study results, first announced in
October 2012, showed that rFVIIIFc was effective in the control and prevention
of bleeding, routine prophylaxis and perioperative management, with low single-
digit median annualized bleeding rates using individualized and weekly
prophylactic regimens. Overall, 98 percent of bleeding episodes were controlled
by one or two injections of rFVIIIFc. Recombinant FVIIIFc was generally well-
tolerated and no inhibitors to rFVIIIFc were detected. The most common adverse
events (incidence of at least 5 percent) occurring outside of the perioperative
management period were nasopharyngitis, arthralgia, headache and upper
respiratory tract infection. No serious adverse events were assessed to be
related to the therapy by the investigators.
Ongoing clinical studies of rFVIIIFc include Kids A-LONG, for previously-treated
children with hemophilia A under age 12, and ASPIRE, for patients who completed
the A-LONG study or who complete the Kids A-LONG study.
About the B-LONG Study and the rFIXFc Program
B-LONG was a global, open-label, multi-center phase 3 study that evaluated the
efficacy, safety and pharmacokinetics of intravenously-injected rFIXFc in 123
male patients aged 12 years and older. The study results, first announced in
September 2012, showed that rFIXFc was effective in the control and prevention
of bleeding, routine prophylaxis, and perioperative management, with low single-
digit median annualized bleeding rates using individualized prophylactic
regimens at a median dosing interval of 14 days. More than 90 percent of
bleeding episodes were controlled by a single injection of rFIXFc. Recombinant
FIXFc was generally well-tolerated and no inhibitors to rFIXFc were detected.
The most common adverse events (incidence of at least 5 percent) occurring
outside of the perioperative management arm (i.e., Arms 1, 2 and 3, but not Arm
4) were nasopharyngitis, influenza, arthralgia (joint pain), upper respiratory
infection, hypertension and headache. One serious adverse event, obstructive
uropathy in the setting of hematuria, was assessed to be possibly related to
therapy by the investigator. The patient continued rFIXFc treatment and the
event resolved with medical management.
Ongoing clinical studies of rFIXFc include Kids B-LONG, for previously treated
children with hemophilia B under age 12, and B-YOND, for patients who completed
the B-LONG study or who complete the Kids B-LONG study.
About the Fc Fusion Technology Platform
Recombinant FVIIIFc and recombinant FIXFc are clotting factors developed using
Biogen Idec's novel and proprietary monomeric Fc fusion technology, which makes
use of a naturally occurring pathway that delays the breakdown of factor in the
body and cycles it back into the bloodstream, enabling it to remain in the body
longer following an injection. Fc fusion technology is used in seven FDA-
approved products for the long-term treatment of chronic diseases including
rheumatoid arthritis, psoriasis and platelet disorders.
About Hemophilia
Hemophilia is a rare, inherited disorder in which the ability of a person's
blood to clot is impaired. Hemophilia A is caused by reduced or no Factor VIII
protein, whereas hemophilia B is caused by reduced or no Factor IX protein. Both
proteins are needed for normal blood clotting. Hemophilia A and hemophilia B
occur in about one in 5,000 and one in 25,000 male births, respectively. People
with hemophilia need injections of clotting factors to restore the coagulation
process and prevent frequent bleeds that could otherwise lead to pain,
irreversible joint damage and life-threatening hemorrhages. The Medical and
Scientific Advisory Council of the National Hemophilia Foundation recommends
prophylaxis as the optimal therapy for people with severe hemophilia A and
severe hemophilia B. Currently, prophylaxis for hemophilia A typically requires
injections three times per week or every other day to maintain a sufficient
circulating level of clotting factor, while prophylaxis in hemophilia B
typically requires injections two to three times per week.
About the Biogen Idec and Sobi Collaboration
Biogen Idec and Sobi are partners in the development and commercialization of
rFVIIIFc and rFIXFc. Biogen Idec leads development, has manufacturing rights,
and has commercialization rights in North America and all other regions
excluding the Sobi territory. Sobi has the right to opt in to assume final
development and commercialization in Europe, Russia, the Middle East and
Northern Africa.
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 Sobi
Sobi is an international healthcare company dedicated to bringing innovative
therapies and services to improve the lives of rare disease patients. The
product portfolio is primarily focused on inflammation and genetic diseases,
with three late stage biological development projects within hemophilia and
neonatology. Sobi also markets more than 40 products for companies in the
specialty and rare disease space. In 2011, Sobi had revenues of SEK 1.9 billion
and around 500 employees. The share (STO: SOBI) is listed on NASDAQ OMX
Stockholm. More information is available at www.sobi.com.
Safe Harbor
This press release contains forward-looking statements, including statements
about the commercialization and impact of long-lasting hemophilia therapies.
These statements may be identified by words such as "believe," "expect," "may,"
"plan," "potential," "will" and similar expressions, and are based on our
current beliefs and expectations. Drug development and commercialization involve
a high degree of risk. Factors which could cause actual results to differ
materially from our current expectations include the risk that unexpected
concerns may arise from additional data or analysis, regulatory authorities may
require additional information or further studies, or may fail to approve or may
delay approval of our drug candidates, or we may encounter other unexpected
hurdles. For more detailed information on the risks and uncertainties associated
with Biogen Idec's drug development and commercialization activities, please
review the Risk Factors section of Biogen Idec's most recent annual or quarterly
report filed with the Securities and Exchange Commission. Any forward-looking
statements speak only as of the date of this press release and we assume no
obligation to update any forward-looking statements, whether as a result of new
information, future events or otherwise.
The information above has been published pursuant to the Swedish Securities
Market Act and/or the Financial Instruments Trading Act. The information was
released for public distribution on February 8, 2013 at 1:30 p.m. CET.
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