Biogen Idec and Swedish Orphan Biovitrum AB (Sobi) have presented new findings for their long-lasting recombinant factor IX candidate Alprolix for haemophilia B at the XXIV International Society on Thrombosis and Haemostasis (ISTH) Congress in Amsterdam, The Netherlands. Three oral presentations were showcase new data that reinforce the potential safety, efficacy and pharmacokinetic profile of Alprolix. The data highlight the consistency of results with Alprolix across patient types and favourable physician ratings of its efficacy in treating acute bleeding episodes and controlling bleeding during and after major surgery.

“Alprolix is the first product candidate in a new class of long-lasting clotting factor therapies, and the data presented today support the potential of the therapy to reduce the frequency of prophylactic infusions for patients with haemophilia B,” said Glenn Pierce, senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s haemophilia therapeutic area. “These new data help to build the clinical profile of Alprolix by increasing our understanding of its efficacy.”

An evaluation of the treatment of acute bleeding episodes across the prophylaxis and episodic (on-demand) treatment arms of the phase III B-LONG study showed that more than 90 per cent of bleeds were controlled with a single injection of Alprolix and more than 97 per cent were controlled with two or fewer injections.

These data were showcased in the e-poster presentation: treatment of bleeding episodes in subjects with haemophilia B with the long-lasting recombinant Factor IX Fc Fusion Protein (rFIXFc) in the phase III B-LONG Study; surgery analysis; results from an analysis of the phase III B-LONG study showed that Alprolix consistently controlled bleeding during and after 14 major surgeries in 12 patients with haemophilia B. Physicians reported high efficacy levels of Alprolix during surgery, with haemostasis (the stoppage of bleeding) rated as “excellent” for 13/14 surgeries and “good” for 1/14 surgeries.

According to investigator analyses, the results were comparable to that for similar surgeries in people without haemophilia. These data were showcased in the e-poster presentation: long-lasting Recombinant Factor IX Fc Fusion (rFIXFc) for Perioperative Management of Subjects with Haemophilia B in the phase III B-LONG Study; analysis of a population pharmacokinetics (popPK) model developed for Alprolix demonstrated that the model accurately predicts peak and trough factor IX activity levels achieved in the B-LONG clinical study at a variety of Alprolix doses.

These data were showcased in the e-poster presentation: clinical implications of Population Pharmacokinetics of rFIXFc in Routine Prophylaxis, Control of Bleeding and Perioperative Management for Haemophilia B Patients.

“These new data from the B-LONG study support the potential application of Fc fusion technology in haemophilia,” said Birgitte Volck, MD, Ph.D., senior vice president development and chief medical officer of Sobi. “The results add to the growing body of evidence supporting the potential efficacy and safety of this long-lasting clotting factor candidate for the treatment of haemophilia B.”

A Biologics License Application (BLA) for Biogen Idec’s long-lasting haemophilia product candidate Alprolix is currently under review with the US Food and Drug Administration (FDA) for the treatment of haemophilia B.

Marketing Applications for Alprolix have been submitted in Canada and Australia for the treatment of haemophilia B. Additional regulatory filings are planned.

Alprolix is a clotting factor under development using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the breakdown of factor in the body and cycles it back into the bloodstream, resulting in a longer circulating half-life. Fc fusion technology is used in seven FDA-approved products for the treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders. Biogen Idec is the first and only to apply this proprietary technology to haemophilia.

According to the World Federation of Haemophilia, prophylaxis in haemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.

Biogen Idec and Sobi are partners in the development and commercialization of ELOCTATE in haemophilia A and Alprolix in haemophilia B. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North America and all other regions excluding the Sobi territory. Sobi has the right to opt in to assume final development and commercialization in Europe (including Russia), the Middle East and Northern Africa.

Biogen Idec discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, haemophilia and autoimmune disorders.

Sobi is an international specialty healthcare company dedicated to rare diseases. The mission is to develop and deliver innovative therapies and services to improve the lives of patients.