Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) presented new data that support the clinical and safety profile of their long-lasting recombinant factor VIII candidate ELOCTATE for hemophilia A. Five platform and oral presentations at the XXIV International Society on Thrombosis and Haemostasis (ISTH) Congress in Amsterdam, The Netherlands, highlight the new FVIII candidate's potential to reduce the number of intravenous injections people with hemophilia A require, its efficacy in controlling bleeding during and after surgery, and its efficacy in treating acute bleeding episodes.

“We are excited to share these data on ELOCTATE, which provide additional evidence of the potential of ELOCTATE to make a meaningful difference in the lives of patients with hemophilia A.”

"We are excited to share these new data, which include patients' and physicians' assessment of ELOCTATE's efficacy in treating bleeding episodes and in controlling bleeding during surgery," said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec's hemophilia therapeutic area. "The A-LONG data presented at ISTH support the potential of ELOCTATE to enable longer intervals between prophylactic (preventative) injections compared to the current standard of care."

Treatment of Bleeding

An evaluation of the treatment of acute bleeding episodes across the prophylaxis and episodic (on demand) treatment arms of the phase 3 A-LONG study showed that more than 87% of bleeds were controlled with a single injection of ELOCTATE and more than 97% of were controlled with two or fewer injections. These data were showcased in the e-poster presentation:

• Treatment of Bleeding Episodes in Subjects with Haemophilia A With Long-Lasting Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Phase 3 A-Long Study

Novel Assay Clinical Research

For the first time Biogen Idec shared results of evaluations of the performance of ELOCTATE when evaluated using two investigational hemostasis assays. Results from a large scale, global clinical evaluation of ELOCTATE by thrombin generation assay (TGA), using a standardized sample collection procedure and an optimized and validated assay performed at a central laboratory showed that, despite inherent patient-to-patient differences in thrombin generation activity, ELOCTATE and Advate showed equivalent thrombin generation potential post-infusion. ELOCTATE also showed prolonged thrombin generation activity relative to Advate, correlating with the pharmacokinetic data observed in these subjects.

Results from a rotation thromboelastometry (ROTEM^®) analysis conducted on 44 patients treated with ELOCTATE from 13 A-LONG study sites showed that ELOCTATE was fully active in patient samples. Additionally, the FVIII activity as measured by clotting time seen for ELOCTATE at 72 hours after dosing was comparable to the activity of Advate at 48 hours after dosing (mean clotting times of 1,238 seconds and 1,213 seconds, respectively).

The data presentations were consistent with conventional assay results from the A-LONG study. These data were showcased in two oral platform presentations:

• Evaluation of The Thrombin Generation Potential of a Recombinant Factor VIII Fc Fusion Protein in a Phase III Multi-National Clinical Trial
• Evaluation of Whole Blood Clotting Activity of Recombinant Factor VIII Fc Fusion Protein by ROTEM Analysis in a Multi-Center Phase 3 Clinical Trial

Surgery Analysis

Results from an analysis of the phase 3 A-LONG study showed that ELOCTATE consistently controlled bleeding during and after 9 major surgeries in 9 patients with hemophilia A. Physicians reported high efficacy levels of ELOCTATE during surgery, with hemostasis (the stoppage of bleeding) rated as "excellent" for 8/9 surgeries and "good" for 1/9 surgeries. According to the investigators' analysis, the results were comparable to that for similar surgeries in people without hemophilia. These data were showcased in the e-poster presentation:

• Long-Lasting Recombinant Factor VIII Fc Fusion (rFVIIIFc) for Perioperative Management of Subjects with Haemophilia A in the Phase 3 A-LONG Study

Population Pharmacokinetics (PK) Analysis

Analysis of a population pharmacokinetics (popPK) model developed for ELOCTATE demonstrate that the model accurately predicts peak and trough factor VIII activity levels achieved in the A-LONG clinical study at a variety of ELOCTATE doses. These data were showcased in the e-poster presentation:

• Population Pharmacokinetic Analysis of Long-Lasting Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Patients with Severe Haemophilia A

"These new data from the A-LONG study show potential for the application of Fc fusion technology in hemophilia," said Birgitte Volck, M.D., Ph.D., senior vice president development and chief medical officer of Sobi. "We are excited to share these data on ELOCTATE, which provide additional evidence of the potential of ELOCTATE to make a meaningful difference in the lives of patients with hemophilia A."

ELOCTATE Global Regulatory Status

A Biologics License Application (BLA) for Biogen Idec's long-lasting hemophilia product candidate ELOCTATE is currently under review with the FDA for the treatment of hemophilia A. If approved, ELOCTATE would be the first major treatment advance for the hemophilia A community in more than two decades.

A Marketing Application for ELOCTATE has been submitted in Australia for the treatment of hemophilia A. Additional regulatory filings are planned.