Baxter Presents ADVATE Data on Prophylaxis Management and Efficacy for Hemophilia A Patients

Baxter Presents ADVATE Data on Prophylaxis Management and Efficacy for Hemophilia A Patients

ISTH Data Also Reinforce Efficacy as Patients' Primary Treatment Priority

AMSTERDAM--(BUSINESS WIRE)-- Baxter International Inc. (NYS: BAX) today announced data featuring ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method], a leading recombinant treatment available worldwide, during the 24^th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam, The Netherlands. The first study identifies a correlation between peak levels of factor VIII (FVIII) in the body and the efficacy of treatment, indicating that frequency of peaks may be important when choosing hemophilia treatments. A second presentation reinforces that for both hemophilia patients and parents, efficacy through reduction in bleeds considerably outweighs infusion convenience as the primary treatment priority.

Exploring the Importance of Managing Both Peaks and Troughs

New findings from a post-hoc analysis of the ADVATE prophylaxis study presented at ISTH (PB 4.39-1) indicate important insights on the efficacy of prophylaxis treatment by closely monitoring the levels of recombinant treatment in a patient's body.

The study examined ''peaks,'' the highest levels of factor in the body in the first hours after infusion. Prior studies focused on ''troughs,'' representing the lowest activity levels as the body metabolizes the treatment (typically the period just prior to the next dose), and targeted a trough level of >1% above baseline, but did not consider peak values.¹

The analysis identified a potentially important relationship between higher peak values, as well as time spent above 30 and 40 percent FVIII levels, (the "hemostatically effective" non-hemophilic FVIII range) and efficacy for prophylaxis in hemophilia A patients. This adds to the body of knowledge that by monitoring how long the factor remains active in the body, clinicians may be able to design optimal individualized dosing regimens to reduce the frequency of bleeds.¹

''Efficacy in preventing bleeding is the number one reason for choosing a hemophilia treatment. This pharmacokinetic research suggests that the dosing frequency to manage peaks in addition to troughs may aid in maximizing treatment effectiveness,'' said Leonard A. Valentino, M.D., director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. ''The finding may have implications when considering regimens with less frequent infusions that offer fewer FVIII peaks and less time within the hemostatically effective range.''

Both Patients and Parents Seek Efficacy as Primary Treatment Goal

Additional research presented at ISTH (#PB 3.36-4) discusses early findings that achieving low annual bleed rates is the most critical goal for people living with hemophilia. ² In the survey of treatment preferences among 76 adults living with hemophilia and 86 parents of children with hemophilia in the United States, the respondents agreed that achieving zero bleeds per year was more important than reducing dosing frequency from three times per week to twice weekly. Further, both groups preferred a "track record of ten years of product safety over a product new to market," even one with less frequent dosing.²

''Overall, these studies emphasize that with regard to hemophilia patient care regimens, treatment is simply not a 'one size fits all' approach, and individualized regimens can help optimize the effectiveness each patient achieves,'' said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter's BioScience business. ''Meeting the needs of individual patients and raising the standard of care are the primary focus of Baxter's long-term commitment in hemophilia and our pursuit of a future where one day, every patient can live a life without bleeds.''

About ADVATE

ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] is indicated for the control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A. ADVATE is also indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with hemophilia A. ADVATE is not indicated for the treatment of von Willebrand disease.

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 58 countries worldwide, including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, China, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau, Malaysia, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela.

Detailed Important Risk Information for ADVATE

ADVATE is contraindicated in patients with known anaphylaxis to mouse or hamster protein or other constituents of the product.

Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE. Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus. Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Carefully monitor patients treated with AHF products for the development of FVIII inhibitors by appropriate clinical observations and laboratory tests. Inhibitors have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs).

If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures FVIII inhibitor concentration.

The serious adverse reactions seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to FVIII.

The most common adverse reactions observed in clinical trials (frequency greater than or equal to 10 percent 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/products/ADVATE_PI.pdf

About Hemophilia A

Hemophilia is a rare genetic³ blood clotting disorder and the most severe forms of the disease primarily affect males.⁴ People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood.⁵ Two of the most common forms of hemophilia are A and B. In people with hemophilia A, clotting factor VIII is not present in sufficient amounts or is absent.⁵ Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.^4,6 According to the World Federation of Hemophilia, it is estimated that more than 400,000 people in the world have hemophilia. ⁷ All races and economic groups are affected equally.⁸

About Inhibitors

As many as one-third of patients with severe or moderately severe hemophilia A are at risk for developing inhibitors,⁹ which are antibodies produced by the body's immune system in response to factor replacement therapy.¹⁰ Inhibitors cause the body to work against the factor replacement therapy, neutralizing its effect and preventing an individual's blood from clotting.⁹ Individuals who have inhibitors have a form of hemophilia that is more difficult to control, with an increased risk of uncontrolled bleeding, compared to patients without inhibitors. Inhibitor development is considered one of the most serious complications associated with hemophilia treatment, and may include other associated complications such as impaired movement, increased need for surgery and greater complexity or risk associated with surgery.⁹

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 is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B 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, 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 studies related to the use of ADVATE and the treatment of hemophilia, including with regard to findings related to the efficacy of prophylaxis treatment and the potential impact on dosing regimens. 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: actions of regulatory bodies and other governmental authorities; additional analysis of clinical results or the development of other clinical evidence; product quality 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 2013 ISTH Congress. E-poster presentation. Peak FVIII levels and time spent in ''hemostatically effective'' FVIII range post-infusion correlates with improved efficacy for prophylaxis in hemophilia A: a closer look at the other end of the curve.

² 2013 ISTH Congress. E-poster presentation. Patient and parent preferences for efficacy, inhibitor risk, safety, and infusion frequency associated with prophylactic Factor VIII treatment.

³ How do you get hemophilia? World Federation of Hemophilia. Accessed on: June 3, 2013. Available at: http://www.wfh.org/en/page.aspx?pid=644

⁴ Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on: June 3, 2013. Available at: http://www.wfh.org/en/page.aspx?pid=637

⁵ What is Hemophilia? World Federation of Hemophilia. Accessed on: June 3, 2013. Available at: http://www.wfh.org/en/page.aspx?pid=646

⁶ Lee, C. A. Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), 2011.

⁷ Treatment. World Federation of Hemophilia. Accessed on June 3, 2013. Available at: http://www.wfh.org/en/page.aspx?pid=642

⁸ What is Hemophilia? Hemophilia Federation of America. Accessed on June 3, 2013. Available at: http://www.hemophiliafed.org/bleeding-disorders/hemophilia/

⁹ Leissinger, Cindy A. Prevention of Bleeds in Hemophilia Patients With Inhibitors: Emerging Data and Clinical Direction. American Journal of Hematology. 2004.

¹⁰ What are Inhibitors? World Federation of Hemophilia. Accessed on June 3, 2013. Available at: http://www.wfh.org/en/page.aspx?pid=651

Baxter International Inc.
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