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Swedish Orphan Biovitrum AB (publ): Biogen Idec and Sobi announce positive top-line efficacy and safety results from phase-3 paediatric study of investigationa

- Data showed twice-weekly prophylactic dosing with Eloctate maintained low
bleeding rates in children under age 12-

- Study meets primary objectives; Results enable regulatory submission in EU-

Biogen Idec(NASDAQ: BIIB) andSwedish Orphan BiovitrumAB (publ) (Sobi) (STO:
SOBI) today released positive top-line results of the Kids A-LONG Phase 3
clinical study that evaluated the safety and efficacy of Eloctate(TM), an
investigational recombinant factor VIII Fc fusion protein product candidate,
in children with severe haemophilia A. Eloctate was generally well-tolerated
and no inhibitors (neutralising antibodies that may interfere with the
activity of the therapy) were detected. Efficacy analyses showed twice-weekly
prophylactic dosing with Eloctate maintained low bleeding rates in children.

Kids A-LONG is the first study to evaluate a long-lasting, investigational
haemophilia therapy in children under 12 years old. Eloctate was developed
using a process called Fc fusion and is designed to prolong the circulation
of infused clotting factor in the body. The successful completion of this
study will support applications for paediatric indications globally and is an
important step to obtaining marketing authorisation in Europe. The European
Medicines Agency requires the inclusion of paediatric study data in a
marketing application for a new haemophilia therapy.

"Prophylactic treatment is recommended for children with severe haemophilia
due to its proven health benefits. However, current prophylactic injection
schedules are challenging for many children with haemophilia A and their
parents," said Glenn Pierce, M.D., Ph.D., senior vice president of
Hematology, Cell and Gene Therapies at Biogen Idec. "The results of this
study support the potential for Eloctate to address a significant need for
children with haemophilia A by providing prolonged intervals between
scheduled prophylactic injections to protect against bleeding episodes."

Kids A-LONG investigated the safety, efficacy and pharmacokinetics
(measurement of the presence of the drug in a person's body over time) of
Eloctate in previously treated children under 12 years old with severe
haemophilia A. The primary endpoint of the study was to evaluate the
frequency of inhibitor (neutralising antibody) development and none were
detected. Secondary endpoints included the overall and spontaneous annualised
bleeding rates (ABR), or projected number of yearly bleeding episodes, and
the number of injections used to prevent and treat bleeding episodes.

In the study, the relative increase in half-life in children with severe
haemophilia A was consistent with the 1.5-fold increase in half-life seen in
the A-LONG study of adults and adolescents. Children treated prophylactically
with Eloctate had an overall median ABR of 2.0 and a median ABR for
spontaneous bleeds of 0.0. Forty-six per cent of participants in the study
experienced zero bleeding episodes. Overall, ninety three per cent of
bleeding episodes were controlled by one to two injections of Eloctate.
Additional analyses of the Kids A-LONG study are ongoing, and the companies
plan to present detailed results at a future scientific meeting.

"Sobi and Biogen Idec recognise the importance of advancing treatment options
for adults and children with haemophilia," said Birgitte Volck, M.D., Ph.D.,
senior vice president development and chief medical officer of Sobi. "The
successful completion of the Kids A-LONG study in children under 12 years old
is an important step to bringing this potential new treatment option to
adults and children with haemophilia A and a milestone that will enable
regulatory submission in Europe."


About Kids A-LONG

Kids A-LONG was a global, open-label, multi-centre Phase 3 study involving 71
boys with severe haemophilia A (factor VIII activity less than 1 IU per dL,
or 1 per cent) with at least 50 prior exposure days to factor VIII therapies.
The study was conducted at 23 haemophilia treatment centres in eight
countries. Overall, 67 participants (94 per cent) completed the study (33
under six years old and 34 six to 11 years old). The average time
participants spent in the study was 25 weeks and 61 participants received
Eloctate injections on at least 50 separate days (exposure days) to assess
inhibitor development.

All study participants were to be initially treated with twice-weekly
prophylactic injections of Eloctate (25 IU/kg day 1, 50 IU/kg day 4). Study
investigators could adjust the dose or interval based on individual response.
Approximately ninety per cent of study participants were on twice-weekly
dosing at the end of the study.

Eloctate was generally well-tolerated. No inhibitors to Eloctate were detected
and no cases of serious allergic reactions were reported in any participants,
all of whom switched from commercially available factor VIII products. No
serious adverse events were assessed to be related to drug by the
investigator. Two non-serious events, rash and myalgia (muscle pain), were
considered related to Eloctate treatment and were reported in one participant
each. No participant discontinued the study due to an adverse event after
receiving Eloctate. The pattern of treatment-emergent adverse events reported
was typical of the population studied and generally consistent with results
seen in adolescents and adults in the A-LONG study.

About Eloctate

Eloctate is an investigational, recombinant clotting factor therapy developed
for haemophilia A by fusing factor VIII to the Fc portion of immunoglobulin G
subclass 1, or IgG1(a protein commonly found in the body). It is believed
that this enables Eloctate to use a naturally occurring pathway to prolong
the time therapy remains in the body. While Fc fusion has been used for more
than 15 years, Biogen Idec is the only company to apply it to the treatment
of haemophilia.

Regulatory applications for Eloctate approval are currently under review in
several countries including the United States, Australia, Canada and Japan.

About Haemophilia A

Haemophilia A is a rare, chronic, genetic disorder in which the ability of a
person's blood to clot is impaired, due to missing or reduced levels of a
protein known as factor VIII. People with haemophilia A experience recurrent
and extended bleeding episodes that cause pain and irreversible joint damage.
Some of these bleeding episodes can be life-threatening. According to the
World Federation of Hemophilia, an estimated 142,000 people worldwide are
identified living with haemophilia A.[1]Prophylactic injections of factor
VIII can temporarily replace the clotting factor necessary to control
bleeding and prevent new bleeding episodes.

Inhibitor development is a response of the body's immune system that
interferes with the activity of therapy. About 25 to 30 per cent of people
with severe haemophilia A develop inhibitors during their lifetime.
Inhibitors typically develop after a median of 8-10 exposure days (EDs),
though this number varies widely.[2]

About the Biogen Idec and Sobi Collaboration

Biogen Idec andSwedish Orphan Biovitrum (Sobi)are partners in the development
and commercialisation of Eloctate for haemophilia A. Biogen Idec leads
development, has manufacturing rights, and has commercialisation rights in
North America and all other regions in the world excluding the Sobi
territory. Sobi has the right to opt in to assume final development and
commercialisation 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, haemophilia 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. For
product labelling, press releases and additional information about the
company, please visitwww.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 2013, Sobi had total
revenues of SEK 2.2 billion (€253 M) and about 550 employees. The share (STO:
SOBI) is listed on NASDAQ OMX Stockholm. More information is available

For more information please contact


Media Contact: Investor Relations contact:
Oskar Bosson Jörgen Winroth
T: +46 70 410 71 80 T: +1 347-224-0819, +1
+46 8 697 2135
oskar.bosson@sobi.com jorgen.winroth@sobi.com

Biogen Idec

Media Contact: Investor Relations Contact:
Lee-Ann Murphy Ben Strain
+1-781-464-3260 +1-781-464-2442
public.affairs@biogenidec.com IR@biogenidec.com

---------------------------------------[1]World Federation of Hemophilia. Annual Global Survey
2012.http://www1.wfh.org/publications/files/pdf-1574.pdf. Accessed January
28, 2014.
[2]Mariani G, Konkle BA, Kessler CM. Inhibitors in Hemophilia A and B. In:
Hoffman R, ed.Hematology : basic principles and practice
. 6th ed. Philadelphia, PA: Saunders/Elsevier; 2013:1961-1970.

Kids A-LONG results


This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Swedish Orphan Biovitrum AB (publ) via Globenewswire


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