CAMBRIDGE, Mass. & STOCKHOLM--(BUSINESS WIRE)--Today Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) announced the publication of detailed results from the pivotal Phase 3 study of ALPROLIXTM, the companies’ investigational long-lasting recombinant factor IX Fc fusion protein candidate for hemophilia B. The study appears in the Online First edition and will appear in the December 12 print issue of The New England Journal of Medicine (NEJM).
The study of ALPROLIX showed that people with severe hemophilia B safely and effectively prevented or reduced bleeding episodes with prophylactic infusions every one to two weeks. As the first long-lasting investigational therapy for hemophilia B to complete a Phase 3 study, ALPROLIX has the potential to be the first important advance in hemophilia B treatment in more than 16 years.
The study, called B-LONG, is the largest Phase 3 clinical study in hemophilia B ever completed. It examined the effect of ALPROLIX therapy delivered with multiple dosing regimens, including prophylactic (weekly or longer), episodic (on-demand) and surgical (perioperative management). Results of B-LONG formed the basis of regulatory applications for ALPROLIX, which are currently under review in several countries including the United States, Canada, Australia and Japan.
“Today, many people with hemophilia B do not follow a prophylactic regimen, and the burdensome infusion schedules associated with currently available treatment may contribute to its limited adoption,” said Marilyn Manco-Johnson, M.D., professor of Pediatrics, University of Colorado and director, Hemophilia and Thrombosis Center, University of Colorado and Children’s Hospital, Colorado. “The National Hemophilia Foundation recommends a prophylactic regimen as optimal for people with severe hemophilia, and studies show this approach reduces bleeding episodes and associated risks. It is my hope that long-lasting therapies in development, such as ALPROLIX, may lessen the burden of prophylactic dosing for people with hemophilia B, and encourage adoption.”
Hemophilia B is a rare, chronic, inherited disorder in which the ability of a person’s blood to clot is impaired, which can lead to extended bleeding episodes. Currently available therapy requires prophylactic infusions two times a week or more. Frequent prophylactic infusions can be a burden to people with hemophilia and may reduce adoption to this type of treatment regimen.1 ALPROLIX has a prolonged circulation time in the blood, and utilizes a technology called Fc fusion. The B-LONG study showed that the interval between prophylactic infusions was extended with ALPROLIX, so that infusions were only needed once a week to once every two weeks in the study. If approved, ALPROLIX may enable people with severe hemophilia B to receive fewer prophylactic infusions than currently available therapy.
“Many of us at Biogen Idec have personal connections to the hemophilia community and know firsthand the burden of frequent infusions,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer at Biogen Idec’s hemophilia therapeutic area. “If approved, long-lasting ALPROLIX therapy will have the potential to change the way hemophilia B is managed and address a critical need for patients.”
B-LONG Study Results
B-LONG was a global, open-label,
multi-center Phase 3 study that evaluated the efficacy, safety and
pharmacokinetics (measurement of the presence of the drug in a patient’s
body over time) of ALPROLIX in 123 males aged 12 years and older with
hemophilia. The study involved 50 hemophilia treatment centers in 17
countries on six continents.
The B-LONG study evaluated ALPROLIX via four treatment regimens:
- Weekly prophylaxis (arm 1)
- Individualized-interval prophylaxis dosing – starting at every 10 days (arm 2)
- Episodic (on-demand) therapy as needed to manage bleeding episodes (arm 3)
- Perioperative (surgical) management (arm 4)
Dose (arm 1) and interval (arm 2) were adjusted during the study to maintain target factor IX levels intended to prevent bleeding.
The overall median annualized bleeding rates (ABR), or projected rate of bleeding episodes per year, were 3.0 for weekly prophylaxis arm and 1.4 for individualized-interval prophylactic regimens, compared to 17.7 for the episodic therapy group. The median dosing interval with individualized-interval prophylaxis (arm 2) was 12.5 days. Bleeding episodes for participants in arms 1-3 were documented and more than 90 percent of all bleeding episodes were controlled by a single infusion of ALPROLIX. ALPROLIX was assessed in the perioperative management of 12 study participants undergoing 14 major surgical procedures. The treating physicians rated response to surgery of ALPROLIX as “excellent” or “good” in 100 percent of these surgeries.
No participants in the study developed inhibitors to ALPROLIX (antibodies that may interfere with the activity of the therapy). There were no reports of vascular clots or serious allergic reactions. Overall, safety events were consistent with those expected in the general hemophilia population. The most common adverse events (incidence of ≥5 percent) occurring outside of the perioperative period were nasopharyngitis (common cold), influenza (flu), arthralgia (joint pain), upper respiratory infection, hypertension (high blood pressure) and headache. One participant had a single serious adverse event that was considered to be possibly related to treatment with ALPROLIX. The participant, who had a history of hematuria (presence of blood in the urine), developed an obstructive clot in the urinary collecting system; he continued ALPROLIX treatment and the event resolved with medical management.
“The publication of the B-LONG pivotal study in The New England Journal of Medicine is a significant milestone that will contribute to the advancement of medical science in hemophilia care,” said Birgitte Volck, M.D., Ph.D., senior vice president development and chief medical officer of Sobi. “These data support the potential of ALPROLIX to provide a meaningful new option to people with hemophilia B by addressing the need for long-lasting therapies for this population.”
About ALPROLIX
ALPROLIX is an investigational fully
recombinant, long-lasting clotting factor therapy being developed for
hemophilia B. It uses Fc fusion technology, which takes advantage of a
naturally occurring pathway that delays the breakdown of Immunoglobulin
G Subclass 1, or IgG1 (protein commonly found in the body), and cycles
it back into the bloodstream. The Fc portion of IgG1 is fused to factor
IX in ALPROLIX and is thought to be responsible for the prolonged time
ALPROLIX circulates in the body. While Fc fusion is an established
technology that has been used for more than 15 years, Biogen Idec is the
only company to apply it in hemophilia.
About Hemophilia B
Hemophilia B is a rare, inherited
disorder in which the ability of a person’s blood to clot is impaired.
Hemophilia B occurs in about one in 25,000 male births annually, and
more rarely in females, affecting about 3,300 people in the United
States. The World Federation of Hemophilia global survey conducted in
2011 estimates that more than 25,000 people are currently diagnosed with
hemophilia B worldwide. It is caused by having substantially reduced or
no factor IX activity, which is needed for normal blood clotting. People
with hemophilia B experience prolonged bleeding episodes that can cause
pain, irreversible joint damage and life-threatening hemorrhages.
Prophylactic infusions of factor IX can temporarily replace the missing
clotting factors that are needed to control bleeding and prevent new
bleeding episodes. The Medical and Scientific Advisory Council of the
National Hemophilia Foundation recommends prophylaxis as the optimal
therapy for people with severe hemophilia B.
About the Biogen Idec and Sobi Collaboration
Biogen Idec and
Swedish Orphan Biovitrum (Sobi) are partners in the development and
commercialization of ALPROLIX for hemophilia B. 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 (including 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 specialty healthcare
company dedicated to rare diseases. Our mission is to develop and
deliver innovative therapies and services to improve the lives of
patients. The product portfolio is primarily focused on inflammation and
genetic diseases, with three late stage biological development projects
within hemophilia and neonatology. We also market a portfolio of
specialty and rare disease products for partner companies. Sobi is a
pioneer in biotechnology with world-class capabilities in protein
biochemistry and biologics manufacturing. In 2012, Sobi had total
revenues of SEK 1.9 billion (€ 215 M) and about 500 employees. The share
(STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is
available at www.sobi.com.
Biogen Idec Safe Harbor
This press release contains
forward-looking statements, including statements about the potential
advances, impact and therapeutic effect of ALPROLIX, our investigational
long-lasting recombinant factor IX candidate. 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 data or 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 our
drug development and commercialization activities, please review the
Risk Factors section of our 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.
1 Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia : the official journal of the World Federation of Hemophilia 2001;7:392-6.
