Biogen Idec and Sobi Announce Positive Top-Line Results from Phase 3 Study
Investigating Long-Lasting Recombinant Factor VIII Fc Fusion Protein in
– Individualized and weekly prophylactic regimens resulted in low single-digit
median annualized bleeding rates –
– 98% of bleeding episodes were controlled with one or two injections of
– No patients developed inhibitors to rFVIIIFc –
– The primary efficacy and safety objectives were met and Biogen Idec plans to
submit an application to US FDA in first half 2013 –
WESTON, Mass. & STOCKHOLM, Sweden -- October 31, 2012
Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum (Sobi) (STO: SOBI)
today announced positive results from A-LONG, a clinical study that evaluated
a new long-lasting clotting factor candidate in people with hemophilia A.
Hemophilia A is a rare inherited disorder that impairs blood coagulation.
Top-line results from A-LONG, a global, multi-center, Phase 3 clinical study
of the companies’ long-lasting recombinant Factor VIII Fc fusion protein
(rFVIIIFc), showed that rFVIIIFc was effective in the control and prevention
of bleeding, routine prophylaxis and perioperative management. Recombinant
FVIIIFc was generally well-tolerated. Additional analyses of the A-LONG study
are ongoing, and the companies anticipate presenting detailed results at a
future scientific meeting.
Biogen Idec plans to submit a Biologics License Application (BLA) to the U.S.
Food and Drug Administration (FDA) in the first half of 2013. Consistent with
guidelines published by the European Medicines Agency (EMA) that require a
study in children less than 12 years of age prior to filing, Biogen Idec and
Sobi expect to file a Marketing Authorization Application with the EMA upon
completion of the ongoing Kids A-LONG study.
“These top-line results demonstrated that rFVIIIFc has the potential to
enhance the care of people living with hemophilia A by offering protection
from bleeding with reduced treatment burden,” 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. “We share the enthusiasm of the
hemophilia community including the study participants and clinical
investigators who supported the rapid enrollment of the A-LONG study. We are
diligently working to prepare our regulatory submission with the goal of
providing rFVIIIFc to people with hemophilia A as soon as possible.”
“We are very encouraged by the positive A-LONG study results, which support
the application of Fc fusion technology in hemophilia A to prolong factor
activity and potentially offer extended protection from bleeding,” said
Geoffrey McDonough, M.D., Chief Executive Officer of Sobi. “The A-LONG
findings, coupled with recently announced results from the B-LONG study of our
companies’ long-lasting recombinant Factor IX Fc fusion product candidate for
hemophilia B, represent a major step forward for the hemophilia community.”
Summary of Key Data from A-LONG
In the A-LONG study, 165 male patients aged 12 years and older were enrolled.
The A-LONG study had three treatment arms: individualized prophylaxis, weekly
prophylaxis and episodic (on-demand) treatment (Arms 1, 2 and 3,
respectively). In a subgroup of patients across treatment arms, rFVIIIFc was
evaluated in the perioperative management of patients who required a major
surgical procedure during the study.
Overall, 93 percent of patients completed the study. Recombinant FVIIIFc was
generally well-tolerated. No inhibitors to rFVIIIFc were detected and no cases
of anaphylaxis were reported in any patients, all of whom switched from
commercially-available Factor VIII products. No serious adverse events were
assessed to be related to drug by the investigator. 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.
The median annualized bleeding rates (ABR), including spontaneous and
traumatic bleeds, were 1.6 in the individualized prophylaxis arm, 3.6 in the
weekly prophylaxis arm and 33.6 in the episodic treatment arm. In the
individualized prophylaxis arm, the median dosing interval was 3.5 days.
During the last three months on study, 30 percent of patients in the
individualized prophylaxis arm achieved a mean dosing interval of five days.
Control of bleeding was assessed in all patients who experienced a bleeding
episode during the study. Overall, 98 percent of bleeding episodes were
controlled by one or two injections of rFVIIIFc.
In addition, rFVIIIFc was assessed in the perioperative management of nine
patients undergoing nine major surgical procedures. The treating physicians
rated the hemostatic efficacy of rFVIIIFc as excellent or good in 100 percent
of these surgeries.
A-LONG included pharmacokinetic (PK) analysis of rFVIIIFc in all patients in
the study. In a protocol-defined subset of patients with extensive PK
sampling, the approximate terminal half-life of rFVIIIFc was 19.0 hours
compared to 12.4 hours for Advate® [antihemophilic factor (recombinant),
plasma/albumin-free method], consistent with the results obtained in the Phase
1/2a study of rFVIIIFc.
Biogen Idec and Sobi senior management will host a webcast to discuss the
A-LONG study results today, October 31, 2012, at 9:00 a.m. Eastern Time.
Previously announced results from the B-LONG Phase 3 study in hemophilia B
will also be discussed. Participants may access the webcast through the
Investors section of Biogen Idec's homepage, www.biogenidec.com.
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. The
study was designed to evaluate rFVIIIFc in the control and prevention of
bleeding, routine prophylaxis and perioperative management in patients with
hemophilia A. A-LONG involved 60 hemophilia treatment centers in 19 countries
on six continents.
The A-LONG study had three treatment arms. In Arm 1 (individualized
prophylaxis; n=118), patients were treated with 25-65 IU/kg of rFVIIIFc, at an
interval of every three to five days, which was individualized to maintain
factor trough levels sufficient to prevent bleeding. In Arm 2 (weekly
prophylaxis; n=24), patients were treated with a weekly dose of 65 IU/kg. In
Arm 3 (episodic treatment; n=23), patients received rFVIIIFc as needed for
bleeding. In a subgroup of patients across treatment arms, rFVIIIFc was
evaluated in the surgical setting.
The primary efficacy and safety measures were the annualized bleeding rate and
the incidence of adverse events including inhibitor development in patients
studied for up to approximately 52 weeks. Secondary endpoints included
response to treatment of bleeding episodes and the pharmacokinetics of
rFVIIIFc versus Advate.
Ongoing clinical studies of rFVIIIFc include the Kids A-LONG and ASPIRE
studies. Kids A-LONG is a Phase 3, open-label study in previously-treated
children with hemophilia A under age 12, which is actively recruiting
patients. ASPIRE is a long-term open-label study for patients who completed
the A-LONG study or who complete the Kids A-LONG study.
About the Fc Fusion Technology Platform
Recombinant FVIIIFc is a clotting factor developed using Biogen Idec's novel
and proprietary monomeric Fc fusion technology, which makes use of a natural
pathway to recycle rFVIIIFc in circulation and enable it to remain in the body
longer. With this technology, rFVIIIFc is designed to provide long-lasting
protection from bleeding and reduce the treatment burden associated with
hemophilia A, which currently requires up to 180 injections annually for
prophylaxis with commercially-available Factor VIII products. Fc fusion
technology is used in seven FDA-approved products for the long-term treatment
of chronic diseases including rheumatoid arthritis, psoriasis and platelet
Using the same Fc fusion technology, Biogen Idec and Sobi are also developing
a long-lasting recombinant Factor IX Fc fusion protein (rFIXFc) for the
control and prevention of bleeding episodes and routine prophylaxis in
hemophilia B. On September 26, the companies announced top-line results from
B-LONG, a global pivotal Phase 3 clinical study of rFIXFc in patients with
hemophilia B. For more information on Biogen Idec’s hemophilia research
programs, visit www.biogenidechemophilia.com or www.biogenidec.com.
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. The Medical
and Scientific Advisory Council of the National Hemophilia Foundation
recommends prophylaxis as the optimal therapy for people with severe
hemophilia A. 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.
About the Biogen Idec and Sobi Collaboration
Biogen Idec and Sobi are partners in the development and commercialization of
rFIXFc and rFVIIIFc. 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
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
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.
This press release contains forward-looking statements, including statements
about the development and commercialization of long-lasting hemophilia
therapies and regulatory filings. These statements may be identified by words
such as "believe," "expect," "may," "plan," "potential," "will" and similar
expressions, and are based on the companies' current beliefs and expectations.
Drug development and commercialization involve a high degree of risk. Factors
which could cause actual results to differ materially from the companies'
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 the companies 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 the companies 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 October 31, 2012 at 1:00 p.m. CET.
Biogen Idec Media Contact:
Jim Baker, +1-781-464-3260
Senior Manager, Public Affairs
Biogen Idec Investor Relations Contact:
Kia Khaleghpour, +1-781-464-2442
Director, Investor Relations
Swedish Orphan Biovitrum Contact:
Åsa Stenqvist, +46 8 697 21 88
Vice President, Head of Communications and Investor Relations (Interim)
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