DEERFIELD, Ill., and SAN DIEGO, Calif., JULY 8, 2011 - Baxter International Inc. and Halozyme Therapeutics, Inc. today announced top-line results of a phase III study of HyQ, an investigational facilitated subcutaneous immune globulin (IG) product for use in patients with primary immunodeficiency (PI). The subcutaneous administration is facilitated by recombinant human hyaluronidase, an enzyme that increases dispersion and absorption of the IG. The data confirm the interim results presented late in 2010 and support the recent submission of a biologics license application to the United States Food and Drug Administration.
"As a long-standing leader in the IG market, Baxter is committed to continually developing and delivering therapies that make a meaningful difference in the lives of patients," said Ludwig Hantson, President of Baxter's BioScience business. "Completion of this important trial brings us closer to providing an option that can address the needs of patients with immune deficiency disorders."
The phase III prospective, open-label, study enrolled 89 patients with PI in 15 centers in the United States and Canada, and evaluated the effectiveness of HyQ in the prevention of infections and measured other secondary endpoints including tolerability.
The objective of the study was to infuse a 3 week or 4 week dose of 10% HyQ in a single infusion site. In the study, the acute serious bacterial infection rate was 0.025 per patient per year, which is below the required efficacy threshold of 1.0. In the tolerability assessment of HyQ, the most frequently reported adverse reactions were infusion site reactions (20% of infusions), headache (3% of infusions), fatigue (1% of infusions) and pyrexia (fever) (1% of infusions).
"Patients with primary immune deficiencies have a chronic, life-long condition that requires diligent, ongoing treatment," said Richard L. Wasserman, MD, PhD, Clinical Professor of Pediatrics at University of Texas Southwestern Medical School, and an investigator in the HyQ phase III trial. "Physicians who are treating these patients will welcome this new option."
The completion of the phase III trial established a foundation for the HyQ extension study that will further evaluate HyQ administration to patients through March 2012. In addition to the recent regulatory submission in the United States, Baxter expects to file in Europe and Canada in the coming months, and will present results from the phase III study by the end of 2011.
"Submission of this BLA for HyQ represents a significant achievement for Halozyme and Baxter," said Gregory I. Frost, Ph.D., Halozyme's President and CEO. "Most importantly, these efforts represent the commitment of our scientists and clinicians to developing new treatment options for patients with primary immunodeficiency."
HyQ is a combination product that includes IG and recombinant human hyaluronidase. IG provides the therapeutic effect of HyQ and the recombinant human hyaluronidase facilitates the dispersion and absorption of the IG.
The IG is a 10% solution that is prepared from large pools of human plasma and contains a broad spectrum of antibodies. The recombinant human hyaluronidase facilitates the increased bioavailability of IG by temporarily increasing the permeability of the subcutaneous tissue.
HyQ is being developed by Baxter using a recombinant human hyaluronidase technology platform licensed from Halozyme Therapeutics, Inc. Submission of the BLA is a financial milestone in the development collaboration between Baxter and Halozyme.
Primary immunodeficiency (PI) comprises more than 150 diseases of the immune system. According to the Immune Deficiency Foundation, at least 250,000 persons in the United States have one of the primary immunodeficiency disorders. IG supplies a broad spectrum of IG antibodies against a wide variety of bacterial and viral agents, thereby preventing infections or fighting them when they occur.
Baxter International Inc. (NYSE: BAX), through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, 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.
Halozyme Therapeutics (Nasdaq: HALO) is a biopharmaceutical company developing and commercializing products targeting the extracellular matrix for the insulin, cancer, dermatology and drug delivery markets.
The company's product portfolio is based primarily on intellectual property covering the family of human enzymes known as hyaluronidases and additional enzymes that affect the extracellular matrix. Halozyme's Enhanze technology is a novel drug delivery platform designed to increase the absorption and dispersion of biologics. The company has key partnerships with Baxter, Roche, ViroPharma and Intrexon to apply Enhanze technology to therapeutic biologics including immune globulin, Herceptin®, MabThera®, Cinryze®and alpha 1-antitrypsin. Halozyme's Ultrafast Insulin program combines its rHuPH20 enzyme with mealtime insulins, which may produce more rapid absorption, faster action, and improved glycemic control. The product candidates in Halozyme's pipeline target multiple areas of significant unmet medical need. For more information visit www.halozyme.com
This release includes forward-looking statements concerning HyQ, including expectations with respect to related regulatory filings. The 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: actions of regulatory bodies and other governmental authorities; satisfaction of regulatory and other requirements; and other risks identified in each company's most recent filing on Form 10-K and other SEC filings, all of which are available on each company's website. Neither Baxter nor Halozyme undertake to update its forward-looking statements.