An A+ partnership on hemophilia B

DEERFIELD, Ill.—Baxter International Inc. and ChathamTherapeutics LLC, an affiliate of Asklepios BioPharmaceutical Inc. (AskBio),have announced the signing of an exclusive global agreement for the developmentand commercialization of potential hemophilia B treatments.

Per the terms of the agreement, the companies willinvestigate Chatham's Biological Nano Particles (BNP), an advanced recombinantadeno-associated virus (rAAV)-based gene therapy technology. Baxter paidChatham $25 million in cash up front for the development and advancement of theprogram through early clinical trials, and as the collaboration progresses overthe next several years, Chatham stands to receive additional payments ifcertain development and commercial milestones are met. Additional detailsregarding each company's role in the agreement were not disclosed.

"This agreement initiates a clinical developmentcollaboration dedicated to advancing a potential long-term treatment paradigmfor hemophilia patients. We look forward to working with Baxter and view thistransaction as the optimal path toward providing a sustainable therapeutic to aworldwide patient population," Jade Samulski, vice president at AskBio andco-founder of Chatham Therapeutics, said in a press release.

The compound the companies are working on, AskBio009, iscurrently listed as being in Phase I development. AskBio's platform is relatedto AAV vectors, and the company notes on its website that using viral vectors"takes advantage of a virus' natural ability to introduce DNA to cells."AskBio's rAAV Biological Nano Particles offer premium transduction efficiency,"ramp-up" expression capabilities, as well as novel tissue targeting and detargetingproperties, the company adds. The technology has already shown potentialtherapeutic benefit in early clinical studies, and the agreement betweenChatham and Baxter will cover the next generation of the gene-therapytechnology.

Hemophilia B, the second-most common type of the disease,results from insufficient amounts of clotting Factor IX, a naturally occurringprotein found in the blood responsible for controlling bleeding. The diseaseoccurs in roughly one in 25,000 males, and there are approximately 4,000 peoplein the United States diagnosed with the disease. Hemophilia B, also known asChristmas disease, is often debilitating, a chronic disease that presentscomplications such as bleeding episodes, hemophilic arthropathy (bleeding intothe joints) and hospitalization.

Brian Kyhos, global communications director at Baxter'sBioScience business, says that currently, treatment for hemophilia B patientsconsists of plasma-derived or recombinant Factor IX, products that are"effective at managing bleeds, but due to the short half-life, they are usuallyadministered twice a week." The collaboration with Chatham, he adds, allowsBaxter to continue its focus on developing hemophilia treatments in hopes ofimproving patients' lives worldwide.

"Gene therapy could provide a significant change inhemophilia B treatment, away from routine administration of replacement factor(currently twice weekly for prophylaxis or 'on demand' for bleeds) to providean underlying level of very long-term protection (multiple years)," says Kyhos."The hope is that patients with hemophilia B treated with gene therapy willexperience reduced morbidity of dysfunctional joints, less time spent intreatments/infusions, improved functionality and improved quality of life."

The agreement is one of several hemophilia-focused endeavorsBaxter currently has underway. The company is also conducting a Phase I/IIIclinical trial of BAX326, its own recombinant Factor IX currently beingevaluated as a treatment for patients with hemophilia B. Baxter plans to filefor U.S. approval for the drug by the end of this year.

"This collaboration demonstrates Baxter's ongoing commitmentto scientific innovation in advancing treatment options for patients livingwith hemophilia. This initiative complements Baxter's extensive hemophiliaportfolio and helps to address unmet needs of hemophilia patients," Dr. LudwigHantson, president of Baxter's BioScience business, said in a press release.