Baxter International Inc. (NYSE:BAX) today announced topline results from a Phase 3 clinical trial evaluating the safety, efficacy and pharmacokinetics (PK) of BAX 111. BAX 111 is a recombinant von Willebrand factor (rVWF) under investigation for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder. The study of BAX 111, the first recombinant treatment in clinical development for this condition, met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (100%, 22 of 22 patients who experienced bleeds in the trial). ''As the first recombinant, stand-alone treatment in development, BAX 111 has the potential to offer people with von Willebrand disease a new therapeutic option that may allow for greater precision and flexibility in managing the disease,'' said Bruce Ewenstein, M.D., Ph.D., vice president of clinical affairs, in Baxter BioScience. ''With these findings, we have taken another significant step forward as we continue to expand on our increasingly broad pipeline of potential treatments to improve outcomes for patients with a range of bleeding disorders.'' The Phase 3 multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 administered together with ADVATE or as a stand-alone therapeutic agent in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the United States, Europe, Australia, Japan, Russia and India. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy and safety measures, pharmacokinetics and health-related quality of life (HRQoL). There were no reports of inhibitor development or thrombotic events in the study participants. The most common adverse events in the study were headache, vomiting/nausea and anemia (iron deficiency anemia), which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication. The investigational treatment was developed using a plasma- and albumin-free manufacturing method.
Full data from the trial, including efficacy and safety outcomes, will be presented later in 2014. Both the European Commission and the U.S. Food and Drug Administration granted orphan-drug designation for BAX 111 in November 2010. Baxter intends to file for approval in the United States before the end of 2014 and, based on these results, intends to pursue a study of BAX 111 in a prophylaxis treatment setting before the end of the year.About von Willebrand Disease 1,2 Von Willebrand disease is the most common inherited bleeding disorder and affects both men and women. Patients with von Willebrand disease either produce insufficient von Willebrand factor, or carry defective von Willebrand factor that result in problems with forming clots to stop bleeding, particularly in mucous membranes such as in the gastrointestinal (GI) or the urogenital tract. It is estimated that up to one percent of the world's population suffers from von Willebrand disease, but many people have only mild symptoms and research has suggested that as many as nine out of 10 people with von Willebrand disease have not been diagnosed. About Baxter in Hemophilia Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, 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.
This release includes forward-looking statements concerning BAX 111 and related clinical studies, including expectations with regard to future regulatory filings and the potential impact of BAX 111 to patients. 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; actions of regulatory bodies and other governmental authorities; additional clinical results; changes in laws and regulations; product quality or supply or patient safety issues; 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 statements.1 Berntorp, E. Review. Von Willebrand Disease. Pediatr Blood Cancer 2013;60:S34–S36. 2 Nichols, WL. Guidelines. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia (2008), 14, 171–232.