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July 14

Biogen Idec and Swedish Orphan Biovitrum Announce Decision to Advance Long-Lasting Hemophilia A and B Therapies:

Biogen Idec and Swedish Orphan Biovitrum  today announced results of their program with their recombinant Fc-fusion hybrids of the coagulation Factors VIII and FIX.  The absence -or abnormally low levels- of these factors in blood cause hemophilia A and B, respectively.

The companies agreed to advance the recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial. 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 hemophilia A patients (>100 exposure days) with severe hemophilia A. In the study, rFVIIIFc was well tolerated and demonstrated a prolonged half-life compared to Advate® (Baxter's 3rd generation rFVIII).  The primary objective of the Phase 1/2a study was to assess the safety of rFVIIIFc at different doses; secondary objectives included the estimation of the pharmacokinetic (PK) 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 (in previously treated patients) or anti-rFVIIIFc drug antibodies in the single-dose study, and there have been no reports of drug-related serious adverse events.  The preliminary data appear to be in agreement with the half-life prolongation seen in a population of hemophilia A dogs presented in last year's ASH meeting.

In addition to the data with FVIIIFc, Biogen Idec  and Orphan Biovitrum AB announced results from a Phase 1/2a open-label, dose-escalation, safety and pharmacokinetic study of the companies' recombinant factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. The data, which were presented at the World Federation of Hemophilia Congress in Buenos Aires, Argentina, on July 11, 2010, showed that rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.  This was in line with previously published data in a number of animal models.

Increasing the terminal half-life of FVIII (approximately 10 - 12 hours) and FIX (approximately 18 hours)  has been an important goal in hemophilia treatment.  Over the last decade, long-term prophylaxis has become the state of the art in hemophilia treatment, limiting damage to joints and improving quality of life.  However, frequent infusions (2-3 times weekly) and problems/complications with venous access make long-term prophylaxis daunting for patients.  Improving the terminal half-lives of these clotting factors would facilitate adherence and have substantial impact on patients' quality of life and overall health maintenance. Although the results so far have been encouraging, pivotal studies of these two fusion proteins would investigate their efficacy and safety in long-term prophylaxis (> 50 - 75 exposure days).  Breakthrough bleeding rates and inhibitor development should be on par with current concentrates.  The development of inhibitors both in previously treated and untreated hemophilia patients is a serious complication, leading to difficult and expensive treatment considerations.