Biogen Idec, Biovitrum Plan To Advance rFIXFc Into Registrational Trial

Biogen Idec and Biovitrum are planning to advance their long-acting, fully-recombinant Factor IX Fc fusion protein (rFIXFc) into a registrational clinical trial in hemophilia B patients. Reportedly, the decision is based on 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.

The results suggested that 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. The company has said that rFIXFc 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 is expected to 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 EMEA and FDA authorities.

Martin Nicklasson, CEO of Biovitrum, said: “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.”