Deerfield, Ill - June 19, 2015
45 oral and poster presentations highlight data across leading brands and investigational molecules
Baxalta Incorporated, a wholly-owned subsidiary of Baxter International Inc. (NYSE:BAX), will highlight the breadth and depth of its hematology portfolio and commitment to innovation in hemophilia and blood disorders with the presentation of new data at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress taking place June 20-25 in Toronto, Canada. Researchers and investigators will discuss the safety and effectiveness of the company's leading brands ADVATE and FEIBA, and will showcase strong clinical data supporting investigational products, including BAX 855 intended for the treatment of hemophilia A, BAX 111 for von Willebrand disease, and potential new treatments for other coagulation disorders.
"The data we are presenting at ISTH include 10 oral presentations and several symposia featuring seven programs across an array of bleeding disorders, showcasing the strength of our differentiated portfolio and our continued leadership across the hematology community," said Brian Goff, head of Hematology for Baxalta. "Our patient-centric innovation reflects our commitment to improving lives as part of our pursuit of a bleed-free world, where patients can access personalized and effective treatment."
Data Highlight the Promise of BAX 855
Data to be presented at ISTH include studies of Baxalta's late-stage R&D pipeline, including BAX 855, to be marketed in the United States under the brand name ADYNOVATE [Antihemophilic Factor (Recombinant), Pegylated], Baxalta's investigational extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A. BAX 855 is based on the ADVATE [Antihemophilic Factor (Recombinant)] full-length rFVIII protein, that is pegylated to extend its duration of activity in the body, and allow for greater personalization of care with a twice-weekly infusion regimen. BAX 855 is currently under regulatory review by the U.S. Food and Drug Administration (FDA), and approval is expected later this year.
Of particular note, a bleeding risk model examines the relative risk of bleeding from different prophylaxis dosing schedules for rFVIII and BAX 855 among patients with various lifestyles and exercise routines. The study results will be shared in the oral presentation "Personalization of Treatment Regimens for Active Patients: A Comparison of Factor VIII and Extended Half-life Treatment Regimens" (Tuesday, June 23, from 2:00 - 2:15 p.m. ET.)
"Our goal is to better understand how a personalized treatment approach based on patient activity levels and treatment schedules may help to reduce relative bleeding risk," said John Orloff, MD, vice president and global head of research and development for Baxalta. "With the inclusion of BAX 855 in our portfolio, we will provide an increasingly diverse portfolio of therapeutic options to meet individual patient needs."
Additional Product and Pipeline Data Highlights
Baxalta will also share clinical updates on its existing portfolio of leading products as well as other investigational therapies for a variety of blood disorders. Key highlights are as follows:
- Multiple studies on ADVATE, including interim results of the AHEAD (ADVATE HaEmophilia A Database) study, a four-year outcomes registry of hemophilia A patients treated with ADVATE (PO243; Tuesday, June 23)
- New data related to OBIZUR [Antihemophilic FactorVIII (Recombinant), Porcine Sequence] for the perioperative management of bleeds in adults with acquired hemophilia A (AHA) (OR028; Monday, June 22, 8:30 - 8:45 a.m. ET) and a presentation by a winner of a Young Investigator Award on his findings into prognostic biomarkers for treatment outcomes in AHA (AS221; Wednesday, June 24, 10:50 - 11:04 a.m. ET)
- Studies supporting the real-world experience with FEIBA [Anti-Inhibitor Coagulant Complex], for use in hemophilia A and hemophilia B patients with inhibitors (OR029; Monday, June 22, 8:45 - 9:00 a.m.)
- Data highlighting the scientific concepts behind BAX 111 (to be marketed under the trade name VONVENDI [von Willebrand Factor (Recombinant)] in the United States), Baxalta's investigational recombinant von Willebrand factor - the first recombinant factor replacement treatment in clinical development for von Willebrand disease (OR088; Monday, June 22, 2:30 - 2:45 p.m. ET)
- An update on an ongoing Phase 1/2 clinical trial for BAX 335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for the treatment of hemophilia B (LB010; Wednesday, June 24, 9:00 - 9:15 a.m. ET)
- 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) (OR151; Tuesday, June 23 8:00 - 8:15 a.m.)
ADVATE [Antihemophilic Factor (Recombinant)] 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 episode
- 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 for the treatment of hemophilia A.
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 the world's most prescribed FVIII treatment. It is currently approved in 64 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Detailed Important Risk Information for ADVATE [Antihemophilic Factor (Recombinant)]
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.
OBIZUR [Antihemophilic FactorVIII (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in adults with acquired hemophilia A.
Limitations of Use:
- Safety and Efficacy of OBIZUR has not been established in patients with baseline anti-porcine factor VIII inhibitor titer greater than 20 BU.
- OBIZUR is not indicated for the treatment of congenital hemophilia A or von Willebrand disease.
Detailed Important Risk Information for OBIZUR
OBIZUR is contraindicated in patients who have had life-threatening hypersensitivity reactions to OBIZUR or its components (including traces of hamster proteins).
WARNINGS & PRECAUTIONS
Hypersensitivity reactions can occur with OBIZUR. OBIZUR contains trace amounts of hamster proteins. Early signs of allergic reactions, which can progress to anaphylaxis, include angioedema, chest-tightness, dyspnea, hypotension, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if allergic or anaphylactic-type reactions occur.
Inhibitory antibodies to OBIZUR have occurred. Monitor patients for the development of antibodies to OBIZUR by appropriate assays. If the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled after OBIZUR administration, suspect the presence of an anti-porcine factor VIII antibody. If such inhibitory antibodies to anti-porcine factor VIII are suspected and there is a lack of clinical response, consider other therapeutic options.
Monitoring Laboratory Tests
- Perform one-stage clotting assay to confirm that adequate factor VIII levels have been achieved and maintained
- Monitor factor VIII activity 30 minutes and 3 hours after initial dose
- Monitor factor VIII activity 30 minutes after subsequent doses
- Monitor the development of inhibitory antibodies to OBIZUR. Perform a Nijmegen Bethesda inhibitor assay if expected plasma factor VIII activity levels are not attained or if bleeding is not controlled with the expected dose of OBIZUR. Use Bethesda Units (BU) to report inhibitor levels.
Common adverse reactions observed in greater than 5% of subjects in the clinical trial were development of inhibitors to porcine factor VIII.
FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:
- Control and prevention of bleeding episodes
- Perioperative management
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation Factor VIII or coagulation Factor IX.
Detailed Important Risk Information
WARNING: THROMBOEMBOLIC EVENTS
- Thromboembolic events have been reported during post-marketing surveillance, particularly following the administration of high doses and/or in patients with thrombotic risk factors.
- Monitor patients receiving FEIBA for signs and symptoms of thromboembolic events.
The use of FEIBA is contraindicated in patients with:
- Known anaphylactic or severe hypersensitivity reactions to FEIBA or any of its components, including factors of the kinin generating system
- Disseminated intravascular coagulation (DIC)
- Acute thrombosis or embolism (including myocardial infarction)
Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur with FEIBA, particularly following the administration of high doses (above 200 units per kg per day) and/or in patients with thrombotic risk factors.
Infusion of FEIBA should not exceed a dose of 100 units per kg body weight every 6 hours and daily doses of 200 units per kg body weight. Monitor patients receiving more than 100 units per kg of body weight of FEIBA for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue the infusion and initiate appropriate diagnostic and therapeutic measures.
Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur following the infusion of FEIBA. The symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. These reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of FEIBA and provide appropriate supportive care.
Because FEIBA is made from human plasma, it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.
The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.
Use of antifibrinolytics within approximately 6 to 12 hours after the administration of FEIBA is not recommended.
Baxalta Incorporated is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, oncology and immunology. Driven by passion to make a meaningful impact on patients' lives, Baxalta's broad and diverse pipeline includes biologics with novel mechanisms and advanced technology platforms such as gene therapy. The Baxalta Global Innovation and R&D Center is located in Cambridge, Massachusetts. Expected to be launched in 2015 following separation from Baxter International Inc., Baxalta's heritage in biopharmaceuticals spans decades. Baxalta's therapies are available in more than 100 countries and it has advanced biological manufacturing operations across 12 facilities, including state-of-the-art recombinant production and plasma fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000 employees worldwide. 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, 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 Baxter's hemophilia and blood disorder R&D and commercial programs, including expectations with regard to regulatory filings and the potential impact on patients, as well as plans to separate Baxter's biopharmaceutical and medical products businesses. These 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: clinical trial results; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality, manufacturing or supply issues; patient safety issues; the ability to successfully separate the businesses on the terms or timeline currently contemplated, if at all, and achieve the intended results; 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.
Baxter, ADVATE, ADYNOVATE, FEIBA, OBIZUR and VONVENDI are trademarks of Baxter International, Inc.