DEERFIELD, Ill., May 12, 2014 - Baxter International Inc. (NYSE: BAX) showcased its commitment to innovation in hemophilia today with the presentation of new data from 60 oral and poster presentations during the 2014 World Federation of Hemophilia (WFH) World Congress in Melbourne, Australia (May 11-15). The data include safety, efficacy and immunogenicity findings on Baxter's global leading brands and new treatments for hemophilia A, von Willebrand disease and other coagulation disorders.
"Baxter's investigational pipeline spans multiple programs, underscoring Baxter's scientific commitment to advancing care for patients with rare and chronic conditions," said Ludwig Hantson, Ph.D., president of Baxter BioScience. "Baxter remains committed to the research and development of new treatments and product enhancements that improve the safety, efficacy and cost-effectiveness of treatments, as well as broadening access to quality care for hemophilia patients worldwide."
PRODUCT AND PIPELINE DATA HIGHLIGHTS
Data presented during WFH include several studies of Baxter's late-stage R&D pipeline, including BAX 855, Baxter's extended half-life investigational recombinant factor VIII (rFVIII) treatment for hemophilia A. BAX 855 is based on the full-length ADVATE [Antihemophilic Factor (Recombinant)] molecule, and is modified with PEGylation technology to extend its duration of activity in the body. As Baxter continues to evaluate the efficacy and safety of this extended half-life treatment in clinical studies, the data from these studies demonstrate the investigational treatment's structural and functional integrity.
Multiple studies will be presented on ADVATE, including a meta-analysis of ADVATE-PASS (Post-Authorization Safety Studies), a prospective, uncontrolled, open-label study, documenting the real-world clinical experience with ADVATE. Conducted in multiple countries between July 2003 and January 2014, the results presented at WFH are the first to illustrate the safety and efficacy of routine use of a rFVIII concentrate in patients with hemophilia A and a low titer inhibitor or inhibitor history. Earlier analysis of PASS data also showed a favorable overall safety and efficacy profile for ADVATE.1
Several presentations also support OBI-1, Baxter's investigational recombinant porcine sequence factor VIII treatment. The investigational treatment has completed a phase II/III study and is now under regulatory review in the United States for the treatment of acquired hemophilia A, a rare, potentially life-threatening bleeding disorder that typically affects older adults and occurs equally in males and females. These data offer insights into the safety, efficacy, and immunogenicity of OBI-1 in patients with acquired hemophilia A.
Additionally, data will highlight the scientific concepts behind BAX 111, Baxter's investigational recombinant von Willebrand factor – the first recombinant factor replacement treatment in clinical development for von Willebrand disease. Positive topline clinical results from the study were announced earlier this year.
Baxter will also share results related to products in earlier stages of its pipeline, including BAX 930, a recombinant ADAMTS13 compound in early stage development for hereditary thrombotic thrombocytopenic purpura (TTP), a condition marked by the formation of platelet-rich blood clots in the body's blood vessels.
"The data presented at WFH demonstrate the breadth of our pipeline and Baxter's dedication to delivering innovative treatment solutions that may help lessen the burden on patients around the world," said Bruce Ewenstein, M.D., Ph.D., vice president of clinical affairs, Baxter BioScience. "We are focused on driving scientific advancements that can make meaningful improvements in patient care across a range of bleeding and other coagulation disorders while contributing to the scientific understanding of these diseases."
PHYSICAL ACTIVITY AND INDIVIDUALIZED TREATMENT A KEY FOCUS
One of the company's priority focus areas is to better understand the unique needs of people with hemophilia who are physically active. In a presentation during the meeting, Baxter researchers will review existing literature associating participation in physical activity and bleeding outcomes to outline recommendations for future research. The company is sponsoring a symposium on Monday, May 12 (12:15 - 1:45 pm AEST) to address this topic in more detail and explore how hemophilia treatments can be tailored based on each person's individual activity profile.
For more information on Baxter's efforts to develop treatments for bleeding disorders, please visit www.baxterhemophilia.com.
About ADVATE [Antihemophilic Factor (Recombinant)]
ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency or classic hemophilia) for:
- Control and prevention of bleeding episodes.
- Perioperative management.
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
ADVATE is not indicated for the treatment of von Willebrand disease.
ADVATE has a demonstrated efficacy and safety profile. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.
ADVATE is approved in 61 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, China, Colombia, Croatia, Ecuador, Hong Kong, Iceland, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay and Venezuela.
DETAILED IMPORTANT RISK INFORMATION
ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, and pruritus.
Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
The serious adverse reactions seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
The most common adverse reactions observed in clinical trials (frequency ≥10% of subjects) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.
Please see full prescribing information for ADVATE at: http://www.baxter.com/downloads/healthcare_professionals/
Hemophilia is a rare genetic blood clotting disorder that primarily affects males. People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood. Two of the most common forms of hemophilia are A and B. In people with hemophilia A, clotting factor VIII (FVIII) is not present in sufficient amounts or is absent. Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal. People with hemophilia B (also called Christmas disease) do not have sufficient amounts of clotting factor IX. In about 30% of cases, there is no family history of hemophilia and the condition is the result of a spontaneous gene mutation. According to the World Federation of Hemophilia, it is estimated that more than 400,000 people in the world have hemophilia. All races and economic groups are affected equally. It is estimated that approximately 18,000 people in the U.S. have been diagnosed with hemophilia A or B.
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.
1Marcucci M, et al. Meta-analysis of Post Authorization Safety Studies (PASS): Worldwide postmarketing surveillance of hemophilia A patients treated with antihemophilic factor recombinant plasma/albumin-free method. Presented at ISTH 2013.
2About Bleeding Disorders: Treatment. World Federation of Hemophilia. Accessed: April 18, 2014. Available at: http://www.wfh.org/en/page.aspx?pid=646
3Frequently Asked Questions about Hemophilia. World Federation of Hemophilia. Accessed: April 18, 2014. Available at: http://www.wfh.org/en/page.aspx?pid=637
4How do you get hemophilia? World Federation of Hemophilia. Accessed: April 18, 2014. Available at: http://www.wfh.org/en/page.aspx?pid=644
5Types of Bleeding Disorders: Hemophilia B. National Hemophilia Foundation. Accessed: April 18, 2014. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=181&contentid=46&rptname=bleeding
6World Federation of Hemophilia Report on the Annual Global Survey 2012. World Federation of Hemophilia. Accessed: April 18, 2014. Available at: http://www1.wfh.org/publications/files/pdf-1574.pdf