ORLANDO, Fla., Dec. 11 /PRNewswire-FirstCall/ -- Baxter Healthcare Corporation today announced the presentation of preclinical data from ongoing research studies aimed at developing a novel, longer-acting form of factor VIII, a protein essential for the normal clotting of blood. Preclinical studies suggest that Baxter's proprietary blood-free factor VIII formulated with chemically modified recombinant von Willebrand factor (rVWF) may extend the half-life of the circulating factor VIII two- to three-fold in mouse models of hemophilia A. Results of these preliminary studies were presented at the American Society of Hematology (ASH) 48th annual meeting held December 9-12 (Abstracts 1001, 1002 & 1021).
"These experiments offer proof-of-concept of our ability to improve the pharmacology of recombinant factor VIII (rFVIII) activity and provide early evidence for the potential for these long acting proteins in the treatment of hemophilia A," said Peter Turecek, Ph.D., senior director of preclinical research and development for Baxter's BioScience business. "We continue to build upon our proven foundation of blood-free recombinant protein processing with the goal of developing recombinant proteins with longer half-life, which if supported by future clinical trial results, potentially allow for less frequent injections and therefore increase convenience and compliance for patients."
Baxter scientists presented preclinical results utilizing a novel approach to extend the half-life of factor VIII, based on modifying a rVWF using pegylation (conjugation with synthetic polymers of polyethylene glycol, PEG) or glycosylation technologies. The results suggest that these approaches may make FVIII less susceptible to the body's breakdown process. Baxter will continue to conduct extensive preclinical research before the modified protein will be available for clinical evaluation.
Pegylation of VWF protein (Abstracts 1002, 1021)
Baxter scientists co-administered pegylated rVWF with rFVIII to mouse models of hemophilia A to evaluate whether the pegylated form of the protein could improve the half-life of administered rFVIII. In the presence of pegylated rVWF, the estimated half-life of rFVIII was 4.4 hours compared to a half-life of 1.2 hours in the presence of unmodified rVWF. In addition, FVIII levels at all time points were substantially higher in the groups co-administered with pegylated VWF.
Glycosylation Modification of VWF (Abstract 1001)
Similar to pegylation, modification of rVWF using glycosylation technology (polysialylation) resulted in a two-fold increase in the lifetime of the polysialylated rVWF as compared to non-modified rVWF when administered to knock-out mice with von Willebrand disease. These early stage data support the potential of glycosylation technology to extend the circulation time of
recombinant proteins, and may be used by Baxter to develop a FVIII with an extended half-life.
Baxter Research and Development
Baxter BioScience focuses its own scientific resources and leverages strategic collaborations to advance the treatment of hemophilia and blood clotting disorders. Building on its history of innovation, the company is developing therapies that will provide less invasive dosing techniques, require less frequent infusions, and/or reduce the potential for inhibitor formation to improve the lives of people with hemophilia.
About Hemophilia A
People with hemophilia A do not produce adequate amounts of factor VIII, which is necessary to effectively clot blood. Without enough factor VIII, patients can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating and damaging to joints. If untreated, patients with severe hemophilia A have a greatly reduced life expectancy. According to the World Health Organization, more than 400,000 people in the world may have hemophilia, corresponding to a prevalence of 15 to 20 in every 100,000 males born worldwide. People with hemophilia A infuse themselves with clotting factors three times a month, on average, unless they are on a preventive regimen, in which case their physician may prescribe infusing a therapy as often as three or four times per week.
About Baxter
Baxter Healthcare Corporation is the principal domestic operating subsidiary of Baxter International Inc. . Baxter International Inc., through its subsidiaries, assists healthcare professionals and their patients with the treatment of complex medical conditions, including cancer, hemophilia, immune disorders, kidney disease and trauma. The company applies its expertise in medical devices, pharmaceuticals and biotechnology to make a meaningful difference in patients' lives.
This release includes forward-looking statements concerning the potential extension of the half-life of rFVIII. 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: satisfaction of regulatory and other requirements for timely commencement of clinical trials; additional results, including results in humans, demonstrating the safety and efficacy of the product; market acceptance of such products; and other risks identified in the company's most recent filing on Form 10-Q and other SEC filings, all of which are available on the company's web site. The company does not undertake to update its forward-looking statements.
Baxter Healthcare CorporationCONTACT: Media, Chris Bona, +1-847-948-2815 (U.S.), or Lorraine Young, 441635 206324 (UK), or Investors, Mary Kay Ladone, +1-847-948-3371, or ClareTrachtman, +1-847-948-3085, all of Baxter
Web site: http://www.baxter.com/
