Baxalta to Initiate a First-in-Human Clinical Trial of BAX 826, an Investigational, Extended Half-Life FVIII Treatment Targeting Weekly Dosing for Hemophilia A

Baxalta Incorporated

, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced the submission of a Clinical Trial Application (CTA) to the UK Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a first-in-human clinical trial to evaluate the safety and efficacy of BAX 826, an investigational, extended half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A. "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," said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. "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." The open-label, dose-finding study of BAX 826 aims to enroll 30 patients; Baxalta expects to begin treating participants in the study by early 2016. BAX 826 is a next-generation rFVIII treatment based on the full length ADVATE [Antihemophilic Factor (Recombinant)] molecule. The compound is modified using proprietary polysialic acid (PSA) technology licensed from Xenetic Biosciences, Inc.

to extend its circulating half-life. Baxalta has partnered with Xenetic to develop novel forms of polysialylated blood coagulation factors, including factor VIII. Xenetic's PolyXen™ technology utilizes the biopolymer PSA in order to extend the circulating half-life and improve the pharmacokinetic profile of therapeutic proteins, peptides, and small molecules. Preclinical studies indicated BAX 826 offered an extended half-life compared to standard rFVIII. With the BAX 826 program, Baxalta aims to augment its growing portfolio of direct factor replacement treatments for hemophilia, including ADVATE as well as ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], which was recently approved in the United States. In addition, the company continues to advance hemophilia innovation with the development of new advances like gene therapy, which has the potential to transform the treatment of hemophilia.