百特（Baxter）4月16日公布了实验性药物BAX111一项III期临床试验的积极数据。该项研究是一项多中心、开放标签临床试验，在37例重症血管性血友病（von Willebrand disease）患者中开展，调查了BAX111联合Advate或作为一种单一制剂用于出血发作按需治疗（on-demand treatment）的安全性、疗效和药代动力学。该项研究的主要终点是经历成功治疗出血发作的患者数目。次要终点包括额外的疗效和安全性措施、药代动力学和健康相关生活质量（HRQoL）。
BAX111是一种重组血管性血友病因子（rVWF），是处于临床开发用于治疗血管性血友病的首个重组药物，目前正调查用于血管性血友病患者出血发作的治疗。在2010年11月，FDA和欧盟委员会（EC）均授予BAX111孤儿药地位。
该项研究中，在临床试验中经历过出血发作的所有患者（100%，n=22）在出血事件按需治疗方面均达到了预先定义的成功，达到了研究的主要疗效终点。
该项研究的完整数据将在2014年晚些时候公布。百特计划在今年年底向FDA提交监管文件，同时打算在年底开展一项研究，调查BAX111用于出血事件的预防性治疗
April 16, 2014 08:48 AM Eastern Daylight Time
DEERFIELD, Ill.--(BUSINESS WIRE)--Baxter International Inc. (NYSE:BAX) today announced topline results from a Phase 3 clinical trial eva1uating 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.
ab0ut von Willebrand Disease1,2
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.
ab0ut 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.
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