Baxter International Inc. has announced that it has initiated pre-clinical programs to develop recombinant Factor IX proteins to treat hemophilia B.
Baxter will develop both an unmodified recombinant Factor IX therapy for treatment of acute bleeding episodes and a chemically modified, long-acting version of recombinant Factor IX therapy for prophylaxis of bleeding in hemophilia B patients. Hemophilia B is the second most common type of hemophilia, occurring in one in 25,000 male births and with an estimated 133,000 people living with hemophilia B worldwide.
"Extending our recombinant portfolio beyond ADVATE, which is indicated for the treatment of hemophilia A, to target hemophilia B is a natural progression of our leadership in hemophilia and in providing innovative therapies for rare blood disorders," said Hartmut J. Ehrlich, MD, vice president of global research and development for Baxter's BioScience business. "Our goal is to develop an unmodified version of recombinant Factor IX intended for acute treatment of bleeding episodes in hemophilia B patients as well as to innovate a longer-acting Factor IX therapy for prevention of bleeding episodes. A longer-acting Factor IX may translate into few required infusions, and can help people living with hemophilia B lead a more normal, healthy life."
Pre-clinical work on a longer-acting version of recombinant Factor IX includes an important new partnership with Nektar Therapeutics to use the company's leading PEGylation technology in combination with Baxter's recombinant Factor IX in order to increase the length of time that Factor levels are maintained in the body. Nektar and Baxter have an existing program aimed toward the development of longer acting Factor VIII therapies.
In accordance with generally accepted accounting principles, Baxter expects to record a special pre-tax charge of approximately $5 million for in- process research and development associated with the transaction in the fourth quarter of 2007. In addition, Baxter may make future development and sales milestone-related payments to Nektar.
Baxter Hemophilia Research and Development
Baxter is a global leader in hemophilia therapy, with innovations that span 40 years. Through its own scientific expertise and strategic partnerships, Baxter is applying novel technologies that will pioneer the next generation of hemophilia therapies. Baxter's goal is to greatly enhance treatment convenience through therapies that require fewer injections and offer non-intravenous dosing alternatives.
About Hemophilia and Hemophilia B
People with hemophilia do not produce adequate amounts of Factor VIII or Factor IX protein, which are necessary for blood to effectively clot. If untreated, patients with severe hemophilia have a greatly reduced life expectancy. According to the World Health Organization, more than 400,000 people in the world have hemophilia, corresponding to an incidence of 15 to 20 in every 100,000 males born worldwide. Hemophilia B is largely an inherited disorder in which the Factor IX protein needed to form blood clots is missing or reduced. In approximately 30 percent of cases, there is no family history of the disorder and the condition is the result of a spontaneous gene mutation. The first diagnosis of hemophilia B occurred in 1952.