First patient dosed in BAX 826 haemophilia A Phase I trial

Baxalta has dosed the first patient in its Phase I, first-in-human clinical trial of BAX 826, a recombinant Factor VIII (rFVIII) treatment for haemophilia A.

BAX 829 uses proprietary polysialic acid (PSA) technology to extend its circulating half-life. BAX 826 is under investigation as Baxalta’s second extended half-life treatment based on Advate [Antihaemophilic Factor (Recombinant)], the world’s most prescribed FVIII treatment.

Commenting on the news, John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta, said: “This is an important step in evolving extended half-life treatments as we aim to improve bleed protection for haemophilia 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.”

The open-label, dose-finding study evaluating the safety and pharmacokinetics of BAX 826 looks to enrol 30 patients in three dosing cohorts. Baxalta expects to complete enrolment by the end of 2016.

Preclinical studies indicated BAX 826 offered an extended circulating half-life

BAX 826 is a next-generation rFVIII treatment based on the full length Advate. The compound is modified using PSA technology licensed from Xenetic Biosciences to extend its circulating half-life. Baxalta has partnered with Xenetic to develop novel forms of polysialylated blood coagulation factors, including FVIII. Xenetic’s PolyXen technology utilises the biopolymer PSA in order to extend the circulating half-life and potentially improve the pharmacokinetic profile of therapeutic proteins, peptides, and small molecules. Preclinical studies indicated BAX 826 offered an extended circulating half-life compared to standard rFVIII.

Baxalta leads the industry with the broadest portfolio of haemophilia treatments. With BAX 826, the company says it strives to augment its growing portfolio of direct factor replacement treatments for haemophilia, including Advate and Adynovate [Antihaemophilic Factor (Recombinant), PEGylated], which was approved in the United States for on demand and prophylaxis treatment in adolescent and adult patients (12 years and older) with haemophilia A and is under regulatory review in Japan, Canada and Switzerland. The company also continues to drive haemophilia innovation with the development of new advances like gene therapy.