US-based biopharmaceutical firm Baxalta has begun its Phase I clinical trial evaluating BAX 826, a recombinant Factor VIII (rFVIII) treatment for haemophilia A.
Using proprietary polysialic acid (PSA) technology, BAX 826 is the company’s second extended half-life treatment based on Advate, which is claimed to be the world’s most prescribed FVIII treatment.
Advate is a full-length recombinant FVIII product that is processed without any blood-based additives, and which demonstrated efficacy and safety in treating haemophilia A,
Around 30 patients will be enrolled in three dosing groups in the open-label trial. Enrolment is expected to be completed by the end of the year.
Baxalta Research & Development head and chief scientific officer John Orloff said: "This is an important step in evolving extended half-life treatments as we aim to improve bleed protection for hemophilia A patients while potentially offering once-weekly dosing.
"Baxalta is dedicated to advancing innovative research on the principle of direct factor replacement, a proven treatment model, to support as many patients as possible."
BAX 826 is modified using PSA technology licensed from Xenetic Biosciences to extend its circulating half-life.
Baxalta has collaborated with Xenetic to develop new forms of polysialylated blood coagulation factors, including FVIII.
Xenetic’s PolyXen technology uses the biopolymer PSA to enhance the circulating half-life and pharmacokinetic profile of therapeutic proteins, peptides, and small molecules.
Preclinical trials have showed that BAX 826 offered an extended circulating half-life compared to standard rFVIII.
Baxalta’s haemophilia treatments include Adynovate, which was approved in the US for on-demand and prophylaxis treatment in adolescent and adult patients with hemophilia A, and is under regulatory review in Japan, Canada and Switzerland.