WESTON, Mass. & STOCKHOLM--(BUSINESS WIRE)--Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum (STO: SOBI) today announced that they plan to advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial in people with hemophilia A. The decision to advance the program is based on promising data from a Phase 1/2a open-label, cross-over, multi-center, dose-escalation study that evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe hemophilia A. In the study, rFVIIIFc was well tolerated and demonstrated a prolonged half-life compared to Advate® (antihemophilic factor recombinant, plasma/albumin-free method, rFVIII), supporting advancement of the program.
The primary objective of the Phase 1/2a study was to assess the safety of rFVIIIFc at different doses; the secondary objective was to estimate the pharmacokinetic parameters of rFVIIIFc at doses ranging from 25 to 65 IU/kg. Preliminary results demonstrated that rFVIIIFc’s prolonged half-life compared to Advate was seen consistently across all patients and dose levels, and other measures of pharmacokinetics including mean residence time and area under the curve were also increased. There were no signs of injection site reactions, inhibitor development or anti-rFVIIIFc drug antibodies in the single-dose study, and there have been no reports of drug-related serious adverse events.
The treatment of severe hemophilia A requires frequent injections, creating a significant burden for individuals with the disorder. The potential of rFVIIIFc, which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion technology, to prolong protection from bleeding and to reduce the frequency of injections needed for treatment will be evaluated in the registrational trial. The global trial, which will commence following communications with regulatory authorities, is being designed to assess the safety, pharmacokinetics and efficacy of rFVIIIFc in both the prevention and treatment of bleeding in hemophilia A patients.
"Biogen Idec is committed to transforming the lives of people with hemophilia by developing a portfolio of long-lasting therapies,” said Glenn Pierce, M.D., Ph.D., Vice President and Chief Medical Officer of Biogen Idec’s hemophilia therapeutic area. “We are excited to announce the advancement of rFVIIIFc into a registrational study. Like our long-lasting hemophilia B program, which entered a registrational trial earlier this year, rFVIIIFc offers the potential to make a significant difference in the lives of people with hemophilia.”
"The Phase 1/2a results are very encouraging, and the decision to initiate our second registrational program in hemophilia represents strong progress in our efforts to improve the lives of individuals with hemophilia,” said Peter Edman, Ph.D., Chief Scientific Officer of Swedish Orphan Biovitrum. “This is also an important milestone in the ongoing development of Swedish Orphan Biovitrum."
In June, the European Medicines Agency’s Committee for Orphan Medicinal Products issued a positive opinion regarding the orphan drug application for rFVIIIFc. A final decision is expected in the coming weeks.
About Hemophilia A
Hemophilia A is a rare, inherited disorder in which the ability of a person's blood to clot is impaired. Hemophilia A occurs in about one in 5,000 male births annually and is caused by having substantially reduced or no factor VIII protein, which is needed for normal blood clotting. People with hemophilia A therefore need injections of factor VIII 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 three times per week or every second day 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. Founded in 1978, Biogen Idec is a global leader in the discovery, development, manufacturing, and commercialization of innovative therapies. Patients worldwide 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 Swedish Orphan Biovitrum
Swedish Orphan Biovitrum is a Swedish-based niche specialty pharmaceutical company with an international market presence. The company is focused on providing and developing specialist pharmaceuticals for rare disease patients with high medical needs. The portfolio consists of about 60 marketed products and an emerging late-stage clinical development pipeline. Our focus areas are: hemophilia, autoimmune diseases, metabolic diseases and cancer supportive care. Swedish Orphan Biovitrum had pro-forma revenues 2009e of about 2 BSEK and approximately 500 employees. The head office is located in Sweden and the share (STO:SOBI ) is listed on NASDAQ OMX Stockholm. For more information please visit www.sobi.com.
This press release contains forward-looking statements regarding the development of long-lasting hemophilia therapies, which may be identified by words such as “believe,” “expect,” “may,” “plan,” “will” and similar expressions. These statements are based on the companies’ current beliefs and expectation. Drug development involves a high degree of risk. Factors which could cause actual results to differ materially from the companies’ current expectations include the risk that we may not fully enroll our planned clinical trials, unexpected concerns may arise from additional data or analysis, regulatory authorities may require additional information, further studies, or may fail to approve the drug, or the companies may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with Biogen Idec's drug development and other activities, see the periodic reports of Biogen Idec filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and the companies assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Swedish Orphan Biovitrum may be required to disclose the information provided herein pursuant to the Swedish Securities Markets Act. The information was provided for public release on July 9, 2010 at 3:30 p.m. CET.
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