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Baxter Reports Progress on Hemophilia and Recombinant VWF Treatments

August 1, 2011

Last month, Baxter International Inc. announced results of early clinical trials on two of its products: BAX 499 for hemophilia A and B, and a recombinant von Willebrand factor therapy. The data were presented at the XXIIIrd Congress of the International Society on Thrombosis and Haemostasis, held in Kyoto, Japan, on July 23-29, 2011.


BAX 499

BAX 499, a fully synthetic compound, is being developed as a potential treatment for hemophilia A and B that could be administered via subcutaneous (under the skin) injection. The new drug blocks the activity of the tissue factor pathway inhibitor, helping patients with hemophilia achieve better clotting. In the studies, BAX 499 also increased the rate and amount of thrombin, a key clotting protein, that was generated. BAX 499 is in phase I clinical trials, expected to be completed in the second half of 2011.

“We are encouraged by these data that help deepen our understanding of potential new approaches to address complex blood disorders,” said Hartmut Ehrlich, MD, vice president, global research and development and medical affairs, Baxter’s BioScience business. “The studies presented on BAX 499 validate tissue factor pathway inhibitor as a viable target for subcutaneous hemophilia therapy and we look forward to sharing future data as they become available.”


rVWF Therapy

Baxter also reported a recombinant von Willebrand Factor (rVWF) therapy that, unlike current treatments made from plasma (pdVWF), contains no blood-based additives.

A Phase I clinical study was performed on 32 patients with different types of VWD, measuring the safety, tolerability and pharmacokinetics (processing and duration of a drug’s effect in the body) of rVWF versus pdVWF. Four concentrations of rVWF were administered in a dose-escalating manner. Investigators reported no serious and 12 “nonserious” adverse reactions in patients with type 3 and severe type 1 VWD. The clinical data also showed that the pharmacokinetics of rVWF and pdVWF were comparable. Baxter announced that it will recruit patients for a larger study to evaluate safety and efficacy of rVWF, which will begin in the latter part of 2011.    


Source: Baxter press release dated July 26, 2011