STOCKHOLM, SWEDEN--(MARKET WIRE)--Mar 4, 2008 -- Biovitrum AB (publ), Sweden, and Syntonix Pharmaceuticals, Inc., USA, have obtained approval from the US Food and Drug Administration to commence a clinical phase I/IIa study of a long-acting, recombinant Factor IXFc for the treatment of Hemophilia B.
The US Food and Drug Administration (FDA) has approved the initiation of a phase I/IIa safety and pharmacokinetic study of intravenous, long-acting, recombinant Factor IX (FIXFc) in previously treated hemophilia B patients. This first in human study aims to evaluate the safety and tolerability of FIXFc together with its pharmacokinetic profile (primarily its plasma half-life) after a single injection of six different doses. The study will be performed at two clinics in the United States.
There is an increasing trend toward use of Factor IX for prophylaxis treatment of Hemophilia B, which requires several infusions per week with the currently available drugs. The extended half-life of this new FIXFc product could enable effective treatment for both prophylaxis and on-demand therapy with less frequent intravenous injections and thereby provide a new, improved therapy for the patients. The total market potential for Factor IX products is estimated to be in excess of USD 600 million worldwide, per year.
"Hemophilia is a prioritized therapeutic and business area within Biovitrum. We are excited to develop FIXFc with Syntonix because we believe that the company's' SynFusionTM technology has resulted in a very promising, long-acting recombinant Factor IX product opportunity that has the potential to reduce the frequency of infusions required for Hemophilia B patients to manage their disease. The program fits well with Biovitrum's long experience and broad knowledge within the area of protein therapeutics and matches perfectly our focus on specialist indications." said Martin Nicklasson, CEO of Biovitrum.
About Biovitrum
Biovitrum is a pharmaceutical company with operations in Sweden and in the UK. Biovitrum has currently a research portfolio with several projects in clinical and preclinical phases for a number of well defined specialist indications as well as for common diseases within obesity, diabetes, inflammation and eye diseases. Biovitrum develops and produces protein-based drugs on a contractual basis and markets a range of specialist pharmaceuticals primarily in the Nordic countries. Biovitrum has revenues of approximately SEK 1.2 billion and around 500 employees. Biovitrum's share is listed on the OMX Nordic Exchange in Stockholm since September 15, 2006. For more information see 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 http://www.syntnx.com/. <<
BIIB, Biovitrum Advance Factor IX Program to Phase-3
[Factor IX is the clotting-cascade protein whose absence causes hemophilia B. (The more common hemophilia ‘A’ is caused by a lack of Factor VIII.) The drug being developed by BIIB and Biovitrum is a recombinant form of Factor IX fused to a proprietary constant (Fc) portion of a antibody; the point is to increase the half-life compared to ordinary Factor IX and thereby reduce the required injection frequency for hemophiliacs. (Enbrel, the blockbuster RA drug from PFE/AMGN, is based on a similar technology.)
BIIB acquired this program via the 2007 buyout of Syntonix (#msg-15983905). The drug has orphan status for hemophilia B in the US and EU.]
›Biogen Idec and Biovitrum Announce Decision to Advance Long-Acting Hemophilia B Therapy into a Registrational Trial
2:30 am EDT, Monday October 19, 2009
CAMBRIDGE, Mass. & STOCKHOLM--(BUSINESS WIRE)--Biogen Idec (NASDAQ: BIIB) and Biovitrum AB (STO: BVT) today announced that they plan to advance the companies’ long-acting, fully-recombinant Factor IX Fc fusion protein (rFIXFc) into a registrational clinical trial in hemophilia B patients. The decision to advance the program is based on promising data from a Phase I/IIa open-label, multi-center, safety dose-escalation and pharmacokinetic study of intravenous rFIXFc in severe, previously-treated hemophilia B patients. rFIXFc was well tolerated in the study. In addition, rFIXFc demonstrated a prolonged half-life compared to historical data for existing therapies, supporting advancement of the program.
Hemophilia B requires frequent injections, creating a significant burden for the majority of individuals with the disorder. The potential of rFIXFc, which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion technology, to prolong protection from bleeding and reduce the frequency of injections for both prophylaxis and on-demand therapy will be evaluated in the registrational trial.
The global trial is being designed to assess the safety, pharmacokinetics and efficacy of rFIXFc in the prevention and treatment of bleeding in hemophilia B patients. The trial will commence following communications with regulatory authorities. rFIXFc has received orphan medicinal product designation for the treatment of hemophilia B from both the European (EMEA) and US (FDA) authorities.
“rFIXFc is an example of Biogen Idec’s commitment to developing innovative therapies to address significant unmet medical needs. The rFIXFc program has the potential to improve the lives of individuals with hemophilia B and we are excited about advancing the program,” said Glenn Pierce, Vice President and Chief Medical Officer of Biogen Idec’s hemophilia therapeutic area.
"The Phase I/II results are very encouraging. The decision to initiate our first registrational program represents true progress in our efforts to offer hemophilia B patients treatment that makes a significant difference and is also an important milestone in the ongoing development of Biovitrum,” said Martin Nicklasson, CEO of Biovitrum.
About Hemophilia
Hemophilia is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia B occurs in about 1 in 25,000 male births annually and is caused by having substantially reduced or no factor IX protein, which is needed for normal blood clotting. People with hemophilia B therefore need injections of factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. Prophylaxis treatment with infusions two or three times per week to maintain a sufficient circulating level of coagulation factor is being increasingly used, and long-term studies demonstrate that such regimens increase the patient’s life expectancy and greatly reduce if not eliminate progressive joint deterioration.
About Biogen Idec
Biogen Idec creates new standards of care in therapeutic areas with high unmet medical needs. Biogen Idec is a global leader in the discovery, development, manufacturing, and commercialization of innovative therapies. Patients in more than 90 countries benefit from Biogen Idec's significant products that address diseases such as lymphoma, multiple sclerosis, and rheumatoid arthritis. For product labeling, press releases and additional information about the company, please visit www.biogenidec.com.
About Biovitrum
Biovitrum is an international pharmaceutical company that markets specialist pharmaceuticals in several regions globally. The company head office is located in Sweden. Using its expertise and experience Biovitrum takes scientific innovation to patients with significant medical need. Research expertise and capabilities are focused on development and production of biotechnology therapeutics within our prioritized areas of hemophilia, inflammation/autoimmune diseases and malabsorption. The company has revenues of approximately SEK 1.2 billion and around 400 employees. It is listed on the OMX Nordic Exchange in Stockholm. For more information go to www.biovitrum.com.‹
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