Biogen Idec (NASDAQ: BIIB) and Biovitrum AB (STO: BVT) today announced that they plan to advance the companies’ long-acting, fully-recombinant Factor IX Fc fusion protein (rFIXFc) into a registrational clinical trial in hemophilia B patients.
The decision to advance the program is based on promising 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. 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.
Hemophilia B requires frequent injections, creating a significant burden for the majority of individuals with the disorder. The potential of rFIXFc, which 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 will 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 European (EMEA) and US (FDA) authorities.
“rFIXFc is an example of Biogen Idec’s commitment to developing innovative therapies to address significant unmet medical needs. The rFIXFc program has the potential to improve the lives of individuals with hemophilia B and we are excited about advancing the program,” said Glenn Pierce, Vice President and Chief Medical Officer of Biogen Idec’s hemophilia therapeutic area.
"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,” said Martin Nicklasson, CEO of Biovitrum.