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An A+ partnership on hemophilia B
July 2012
by Kelsey Kaustinen  |  Email the author


DEERFIELD, Ill.Baxter International Inc. and Chatham Therapeutics LLC, an affiliate of Asklepios BioPharmaceutical Inc. (AskBio), have announced the signing of an exclusive global agreement for the development and commercialization of potential hemophilia B treatments.  
Per the terms of the agreement, the companies will investigate Chatham's Biological Nano Particles (BNP), an advanced recombinant adeno-associated virus (rAAV)-based gene therapy technology. Baxter paid Chatham $25 million in cash up front for the development and advancement of the program through early clinical trials, and as the collaboration progresses over the next several years, Chatham stands to receive additional payments if certain development and commercial milestones are met. Additional details regarding each company's role in the agreement were not disclosed.
"This agreement initiates a clinical development collaboration dedicated to advancing a potential long-term treatment paradigm for hemophilia patients. We look forward to working with Baxter and view this transaction as the optimal path toward providing a sustainable therapeutic to a worldwide patient population," Jade Samulski, vice president at AskBio and co-founder of Chatham Therapeutics, said in a press release.
The compound the companies are working on, AskBio009, is currently listed as being in Phase I development. AskBio's platform is related to 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 detargeting properties, the company adds. The technology has already shown potential therapeutic benefit in early clinical studies, and the agreement between Chatham and Baxter will cover the next generation of the gene-therapy technology.  
Hemophilia B, the second-most common type of the disease, results from insufficient amounts of clotting Factor IX, a naturally occurring protein found in the blood responsible for controlling bleeding. The disease occurs in roughly one in 25,000 males, and there are approximately 4,000 people in the United States diagnosed with the disease. Hemophilia B, also known as Christmas disease, is often debilitating, a chronic disease that presents complications such as bleeding episodes, hemophilic arthropathy (bleeding into the joints) and hospitalization.  
Brian Kyhos, global communications director at Baxter's BioScience business, says that currently, treatment for hemophilia B patients consists of plasma-derived or recombinant Factor IX, products that are "effective at managing bleeds, but due to the short half-life, they are usually administered twice a week." The collaboration with Chatham, he adds, allows Baxter to continue its focus on developing hemophilia treatments in hopes of improving patients' lives worldwide.
"Gene therapy could provide a significant change in hemophilia B treatment, away from routine administration of replacement factor (currently twice weekly for prophylaxis or 'on demand' for bleeds) to provide an underlying level of very long-term protection (multiple years)," says Kyhos. "The hope is that patients with hemophilia B treated with gene therapy will experience reduced morbidity of dysfunctional joints, less time spent in treatments/infusions, improved functionality and improved quality of life."  
The agreement is one of several hemophilia-focused endeavors Baxter currently has underway. The company is also conducting a Phase I/III clinical trial of BAX326, its own recombinant Factor IX currently being evaluated as a treatment for patients with hemophilia B. Baxter plans to file for U.S. approval for the drug by the end of this year.
"This collaboration demonstrates Baxter's ongoing commitment to scientific innovation in advancing treatment options for patients living with hemophilia. This initiative complements Baxter's extensive hemophilia portfolio and helps to address unmet needs of hemophilia patients," Dr. Ludwig Hantson, president of Baxter's BioScience business, said in a press release.
Code: E071220



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