DEERFIELD, Ill., MARCH 28, 2013 - Baxter International Inc. (NYSE:BAX) and Chatham Therapeutics today announced that the first patient has been dosed in a Phase I/II open-label clinical trial to assess the safety and optimal dosing schedule of BAX 335, an investigational factor IX gene therapy treatment for hemophilia B, the second most common form of hemophilia.
Patients with hemophilia lack the ability to produce the clotting factor IX and are treated with plasma-derived or recombinant factor IX today. BAX 335 provides a mechanism for the patient's own liver to begin producing factor IX following a single dose of the genetically engineered treatment.
"Gene therapy has the potential to redefine the treatment paradigm for hemophilia B patients, helping to reduce the daily burden of their disease and restore more normalized levels of clotting factor," said Bruce Ewenstein, M.D., Ph.D., vice president of clinical strategy, Hemophilia, in Baxter's BioScience business. "This important research program is a representation of Baxter's aspiration to create a bleed-free world for hemophilia patients."
The trial is designed to assess the safety of ascending doses of BAX 335 to determine the optimal single dosing regimen in up to 16 adult patients with hemophilia B at treatment centers in the United States. Interim analyses will be conducted at six months and two years of follow-up. The study's primary endpoint is the safety of intravenous administration of a single dose of BAX 335. Secondary endpoints include evaluation of the optimal dose to achieve stable plasma factor IX activity, as well as kinetics and immune response to treatment. Additional information on the trial is available at www.clinicaltrials.gov, by using Identifier #01687608.
"Chatham's Biological Nano Particles (BNP) technology represents the culmination of decades of research towards the development of long-term sustainable treatments. The platform technology is being developed for a broad diversity of patients in need of dramatically improved treatment options. The successful safe initiation of the BAX 335 clinical trial is an important first step towards the realization of this goal," said Scott McPhee, Ph.D., M.P.H., vice president of research and development, Chatham Therapeutics.
In June 2012, Baxter announced that it entered into an exclusive global agreement with Chatham Therapeutics, LLC, an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio), for the development and commercialization of potential treatments for hemophilia B utilizing Chatham's Biological Nano Particles (BNP), an advanced recombinant adeno-associated virus- (rAAV-) based gene therapy technology that has shown potential therapeutic benefit in early studies. The patient dosing is the first clinical milestone from the collaboration.
About Hemophilia B
Hemophilia B, also known as Christmas disease, is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.1 Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B.2 Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.3
About Baxter in Hemophilia
Baxter has more than 60 years of scientific experience in supporting the treatment needs of patients with hemophilia and has introduced a number of therapeutic firsts. Baxter has the broadest portfolio of hemophilia treatments and is able to meet individualized patient therapeutic needs by providing a range of options at each treatment stage. The company's work focuses on optimizing hemophilia care and improving the lives of people worldwide living with bleeding disorders.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
About Chatham Therapeutics, LLC
Chatham Therapeutics, LLC is a private clinical development-stage biotechnology company engaged in the development of novel, gene therapy-mediated cures for hemophilia. The company is currently engaged in gene therapy-based therapeutic programs for both hemophilia A and B. Chatham Therapeutics, LLC is an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio). AskBio is currently developing proprietary gene-delivery technologies for therapeutics, targeting diseases in the heart, CNS, muscle, ocular and liver tissues. For additional information, visit AskBio's Web site at http://www.askbio.com.
This release includes forward-looking statements concerning a Phase I/II open-label clinical trial of BAX 335, an investigational factor IX gene therapy treatment for hemophilia B. These statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: clinical results demonstrating the safety and optimal dosing regimen of BAX 335; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; and other risks identified in Baxter's most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter's website. Baxter does not undertake to update its forward-looking statement.
1Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on April 20, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=637
2World Federation of Hemophilia Report on the Annual Global Survey 2010. World Federation of Hemophilia. Accessed on April 20, 2012. Available at: http://www1.wfh.org/publications/files/pdf-1427.pdf
3Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. Accessed on April 20, 2012. Screen shot of page available here