DEERFIELD, Ill., APRIL 17, 2015 - Baxter International Inc. (NYSE:BAX) today presented additional data from the Phase III clinical trial of BAX 111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of inherited bleeding disorder.1 The data were presented as an Abstract of Distinction during an oral session at the 2015 Scientific Symposium of the Hemostasis and Thrombosis Research Society (HTRS) in New Orleans, La. and expand on the topline data first disclosed in 2014. The trial met its primary efficacy endpoint defined by the number of patients who achieved treatment success for control of bleeding episodes.
"Today, people with von Willebrand disease have limited treatment options if they require replacement therapy with von Willebrand factor containing concentrate. This study has provided valuable evidence of the potential benefit of BAX 111 as a recombinant therapeutic option to resolve bleeding events," said Dr. Joan Gill, M.D., of the Blood Center and the Medical College of Wisconsin and lead study author.
All patients treated in the full analysis set (N=22) experienced a 100 percent treatment success rating based on a 4-point efficacy rating scale, comparing estimated number of infusions needed to treat the bleeding episodes to the actual number of infusions administered.
Efficacy for all treated bleeds (N=192) was rated excellent (96.9%) or good (3.1%), including major bleeds (6 excellent and 1 good). The median number of infusions required to treat bleeding events in the trial was 1 and the majority of events (81.8%) were resolved with a single infusion.
The multi-center, open-label clinical trial was designed to assess the safety, efficacy and pharmacokinetics of BAX 111 among patients with severe von Willebrand disease aged 18 to 65 years. Bleeding events that occurred during the study were treated with rVWF (40-60 IU/kg; up to 80 IU/kg for major bleeds) initially together with rFVIII and subsequently alone if hemostatic FVIII levels were maintained. rVWF pharmacokinetic (PK) parameters were determined with and without rFVIII and repeated after 6 months for rVWF; these analyses found that rVWF PK was not affected by administration together with rFVIII. The recombinant technology used to produce BAX 111 avoids certain protein maturation processes that typically occur with plasma fractionation. This preserves large functional units known as ultra large multimers, which have been shown to be efficient in clot formation and FVIII stabilization.
No patients developed inhibitors or binding antibodies to the treatment, and there were no reports of thrombotic events or severe allergic reactions. Eight adverse events (AEs) were considered causally related to BAX 111: six non-serious related AEs occurred in four patients, and two related serious AEs (chest discomfort and increased heart rate) occurred in one patient.
"These positive data, which formed the basis for our recent regulatory filing in the US, support the potential role for BAX 111 to become the first recombinant replacement treatment for managing bleeding episodes among these patients," said John Orloff, vice president and global head of research and development at Baxter BioScience. "With an expanding pipeline across multiple bleeding disorders, our goal is to advance programs that can effectively manage these challenging disorders and help improve patient lives as part of our pursuit of a Bleed-Free World."
The data support the application Baxter submitted to the United States Food and Drug Administration (FDA) in late 2014 for approval of BAX 111. Both the European Commission and the FDA have granted orphan-drug designation for BAX 111.
About von Willebrand Disease2,3
Von Willebrand Disease (VWD) is an autosomal genetic disorder related to quantitative deficits and/or qualitative defects of VWF, the result of which is impaired hemostasis. It is the most common hereditary coagulation disorder, occurring in approximately one to two percent of the general population. Many people who have VWD may experience mild symptoms, but some patients can experience severe bleeding events similar to bleeding experienced by patients with hemophilia.
About Baxter BioScience
Baxter BioScience is a leading provider of therapeutic treatments that save, sustain and improve the lives of people with rare conditions, chronic diseases or limited treatment options. Supported by advanced technical and manufacturing expertise, Baxter BioScience has a broad pipeline built on a legacy of innovation in bleeding disorders and immunology and is expanding to address emerging opportunities in niche areas of oncology as well as technology platforms such as biosimilars.
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, including expectations with regard to 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, manufacturing 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.
1Laffan MA, Lester W, O'Donnell JS, et al. The diagnosis and management of von Willebrand disease: a United Kingdom Haemophilia Centre Doctors Organization guideline approved by the British Committee for Standards in Haematology. Br J Haematol. 2014; 167: 453-465.
2Nichols WL, Hultin MB, James AH, et al. 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.
3Mannucci PM. Treatment of von Willebrand's disease. N Eng J Med. 2004; 351: 683-694.