The New England Journal of Medicine publishes pivotal data demonstrating prophylactic infusions of Alprolix(TM) effectively controlled bleeding in haemophilia B patients
- B-LONG Study Showed Investigational Therapy Administered Once Every One to Two
Weeks Prevented or Reduced Bleeding Episodes
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 ALPROLIX(TM), the companies' investigational long-lasting recombinant
factor IX Fc fusion protein candidate for haemophilia 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 haemophilia 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
haemophilia B to complete a Phase 3 study, ALPROLIX has the potential to be the
first important advance in haemophilia B treatment in more than 16 years.
The study, called B-LONG, is the largest Phase 3 clinical study in haemophilia 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 haemophilia 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 haemophilia, 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 haemophilia B, and encourage adoption."
Haemophilia 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
haemophilia and may reduce adoption to this type of treatment regimen.[i]
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 haemophilia B to receive fewer prophylactic
infusions than currently available therapy.
"Many of us at Biogen Idec have personal connections to the haemophilia
community and know first-hand 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 haemophilia therapeutic area. "If approved,
long-lasting ALPROLIX therapy will have the potential to change the way
haemophilia B is managed and address a critical need for patients."
B-LONG Study Results
B-LONG was a global, open-label, multi-centre 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 haemophilia. The study involved 50 haemophilia treatment centres 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 haemophilia 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 haematuria (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 haemophilia 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 haemophilia 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 haemophilia 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
haemophilia.
About Haemophilia B
Haemophilia B is a rare, inherited disorder in which the ability of a person's
blood to clot is impaired. Haemophilia 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 Haemophilia global survey conducted in
2011 estimates that more than 25,000 people are currently diagnosed with
haemophilia B worldwide. It is caused by having substantially reduced or no
factor IX activity, which is needed for normal blood clotting. People with
haemophilia B experience prolonged bleeding episodes that can cause pain,
irreversible joint damage and life-threatening haemorrhages. 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 Haemophilia Foundation
recommends prophylaxis as the optimal therapy for people with severe haemophilia
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 haemophilia 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 haemophilia 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 Harbour
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.
For more information - not for publication
Sobi:
Media relations Investor relations
Oskar Bosson, Head of Jörgen Winroth, Vice President, Head of Investor
Communications Relations
T: +46 70 410 71 80 T: +1 347-224-0819, +1 212-579-0506, +46 8 697 2135
E: oskar.bosson@sobi.com E: jorgen.winroth@sobi.com
Biogen Idec:
Media contact Investor relations
Todd Cooper Ben Strain
T: +1-781-464-3260 T: +1-781-464-2442
E: public.affairs@biogenidec.com E: IR@biogenidec.com
[i] 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.
