Biovitrum's and Syntonix's Novel Factor IXFc for Hemophilia B has
Received Orphan Drug Designation From the FDA
Stockholm, Sweden and Waltham, MA - November 24, 2008. Biovitrum AB
(publ) (STO:BVT) and Syntonix Pharmaceuticals, Inc., a subsidiary of
Biogen Idec (NASDAQ: BIIB) today announced that the company's FIXFc
compound for the control and prevention of hemorrhagic episodes in
patients with hemophilia B has received an orphan-drug designation
from the US Food and Drug Administration (FDA). The FIXFc compound is
a recombinant protein in early-stage development for the treatment of
hemophilia B, a hereditary bleeding disorder characterized by
impaired production of factor IX and the blood's inability to
coagulate. The Fc fusion with factor IX is intended to prolong the
effect of the compound, the goal being that patients will need less
frequent prophylactic treatments than currently existing therapies,
which require two to four administrations per week.
The companies' FIXFc has previously received an EMEA (COMP) orphan
medicinal product designation for the treatment of hemophilia B.
Pre-clinical studies have shown that this FIXFc compound may have an
extended half-life, and a study is currently ongoing to determine the
compound's safety and pharmacokinetics in hemophilia B patients. The
current global market for Factor IX products is estimated at $600 MM
USD annually. According to a recent analysis, the market is expected
to grow to $1.5 BN USD annually by 2015."This orphan drug designation is very good news for our existing
project; Beyond securing market exclusivity for the drug once it is
approved, it will allow for certain benefits, including reduced
marketing application and license fees, the ability to work with the
Office of Orphan Drugs for protocol assistance, and for appropriate
meetings with the Division of Blood Applications. This will help us
in our efforts to hopefully bring this improved recombinant Factor
IXFc hemophilia compound to those patients who have significant
unment medical needs." said Martin Nicklasson, CEO of Biovitrum."We are pleased to have achieved this important goal for the FIXFc
program, which we are developing in a 50/50 partnership with
Biovitrum. The purpose of both the FIXFc and Factor VIIIFc programs
is to improve the lives of hemophilia patients and their families,"
said Matt Ottmer, Vice President of Syntonix.
About Hemophilia
Hemophilia is a rare hereditary disorder in which the ability of
patients' blood to clot is impaired. As a result, the patient suffers
from excessive bleeding and uncontrolled internal bleeding, leading
to pain and eventual permanent damage to joints and muscles. One
form, Hemophilia B results from mutations that impair the production
of Factor IX. It has been reported that even with "proper treatment"
the life expectancy of hemophilia patients is about 10 years less
than for individuals without hemophilia. Increasingly, the normal
mode of treatment for younger patients is a prophylaxis regimen where
patients are infused two or three times per week to maintain a better
circulating level of coagulation factor. Long term studies
demonstrate that such regimens greatly reduce if not eliminate
progressive joint deterioration.
About SynFusionTM Technology
The SynFusion technology is based on Syntonix's proprietary Fc-fusion
technologies to create longer-acting biopharmaceuticals. Well-known
and validated traditional Fc-fusion drugs, such as Enbrel®
(etanercept) for the treatment of rheumatoid arthritis, consist of
two copies of a biopharmaceutical linked to the Fc region of an
antibody to improve pharmacokinetics, solubility, and production
efficiency. SynFusion drugs consist of a novel Fc-fusion construct,
called a monomer that links only a single copy of the drug to the Fc
region on an antibody to optimize the pharmacokinetic and
pharmacodynamic properties of the biopharmaceutical when compared to
traditional Fc-fusion constructs.
About Biovitrum
Biovitrum is a pharmaceutical company with operations in Sweden and
in the UK. The company markets a range of specialist pharmaceuticals
primarily in the Nordic countries. Using its expertise and experience
Biovitrum takes scientific innovation all the way to the market and
to specialist indication patients with significant medical need.
Research expertise and capabilities include development and
production of biotechnology therapeutics, as well as small molecule
discovery and development. With revenues of approximately SEK 1.3
billion and around 500 employees, Biovitrum is a significant European
specialty pharmaceutical player. Biovitrum's share is listed on the
OMX Nordic Exchange in Stockholm. For more information go to
www.biovitrum.com.
About Syntonix
Syntonix Pharmaceuticals, Inc. (Waltham, MA, USA) is a wholly-owned
subsidiary of Biogen Idec. Syntonix is developing next generation
biopharmaceuticals that enable better treatment options for patients
with devastating chronic diseases such as hemophilia and autoimmune
disorders. The company applies its core technologies to develop
long-acting biopharmaceuticals that may be injected less frequently,
and to discover novel drugs to treat antibody-mediated autoimmune and
inflammatory disorders. The resulting proteins, peptides and
antibodies are being commercialized through internal development
programs and collaborations with biotechnology and pharmaceutical
partners. More information is available at www.syntnx.com.
Safe Harbor/Forward-looking Statements
This press release contains forward-looking statements regarding the
market for Factor IX products and the impact of orphan drug
designation. These statements are based on current beliefs and
expectations. The development and commercial potential of the FIXFc
compound is subject to a number of risks and uncertainties, including
the potential safety and efficacy profile of the FIXFc compound and
the projected timeline and cost for completing clinical trials of the
FIXFc compound. Drug development and commercialization involves a
high degree of risk.
For more information please contact:
Biovitrum AB (publ)
Erik Kinnman, Vice President Investor Relations
Phone: +46 73 422 15 40
erik.kinnman@biovitrum.com
Martin Nicklasson, CEO
Phone. +46 8 697 20 00
martin.nicklasson@biovitrum.com
Biovitrum AB (publ) may be required to disclose the information
provided herein pursuant to the Swedish Securities Markets Act. The
information was provided for public release on November 24, 2008 at
02:00 p.m. CET.