Baxalta applies for clinical trial of BAX 826 to treat haemophilia A

BAX 826 is an investigational, extended half-life recombinant Factor VIII (rFVIII) candidate based on the full length ADVATE [Antihemophilic Factor (Recombinant)] molecule.

Baxalta plans to enroll 30 patients and start treatment by early 2016 through weekly dosing of BAX 826. In preclinical trials, BAX 826 offered an extended half-life compared to standard rFVIII.

BAX 826 uses polysialic acid (PSA) technology licensed from Xenetic Biosciences to extend dosing intervals.

Baxalta and Xenetic are jointly developing novel forms of polysialylated blood coagulation factors, including factor VIII.

Xenetic’s PolyXen technology uses the biopolymer PSA to extend the circulating half-life and enhance the pharmacokinetic profile of therapeutic proteins, peptides, and small molecules.

Baxalta head of research & development and chief scientific officer John Orloff said: "We are advancing a number of approaches, including BAX 826 as well as our gene therapy program, to evaluate potential new options for hemophilia patients that can offer efficacy while also easing the treatment burden with a goal of once-weekly or even less frequent infusions.

"The CTA submission represents an important advancement in our efforts to expand our portfolio of extended half-life treatments and further reinforces our leadership in hemophilia innovation."

With the BAX 826 program, Baxalta intends to strengthen its portfolio of direct factor replacement treatments for hemophilia, including ADVATE as well as ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated].

Image: Deficiency in coagulation factor VIII is the cause of haemophilia A. Photo: courtesy of ProteinBoxBot.