New Phase 3 Data Reinforce Long-Lasting Protection from Bleeding for Patients with Hemophilia A and B
– 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--(BUSINESS WIRE)--Feb 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 ‰¥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 ‰¥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.
Photos/Multimedia Gallery Available: http://www.businesswire.com/multimedia/home/20130208005166/en/
Contact: BIOGEN IDEC CONTACT:
Media Contact:
Jeff Boyle, +1-781-464-3260
or
Investor Relations Contact:
Kia Khaleghpour, +1-781-464-2442
or
SWEDISH ORPHAN BIOVITRUM CONTACT:
Media Contact:
Birgitte Volck, +45 4 012 2310/+46 8 697 2188
or
Analyst/Investor Contact:
Jorgen Winroth, +46 8 697 2000/+1-212-579-0506
Posted: February 2013
