Biogen Idec and Biovitrum AB have announced plans to advance the long-acting, fully recombinant Factor IX Fc fusion protein (rFIXFc) towards registrational clinical trials in haemophilia B patients.
Haemophilia B requires frequent injections, creating a significant burden for the majority of individuals with the disorder. This registration trial will evaluate the potential of the treatment to prolong protection from bleeding and reduce the frequency of injections.
The decision to advance the programme is based on promising data from a Phase I/IIa safety dose-escalation and pharmacokinetic study of intravenous rFIXFc in severe, previously-treated haemophilia B patients.
During the study rFIXFc was well tolerated and demonstrated a prolonged half-life compared to historical data for existing therapies, supporting advancement of the programme.
Biovitrum CEO Martin Nicklasson said that the Phase I/II results are very encouraging.
"The decision to initiate our first registrational programme represents true progress in our efforts to offer haemophilia B patients treatment that makes a significant difference," Nicklasson said.
The trial will begin following approval from relevant regulatory authorities. rFIXFc has already received orphan medicinal product designation for the treatment of haemophilia B from the European and US authorities.