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CSL Behring Initiates Study of Subcutaneous Administration of C1-esterase inhibitor in Patients with Hereditary Angioedema
CSL Behring announced today that it has initiated an international phase I/II study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor (C1-INH) concentrate in patients with hereditary angioedema (HAE). Part of the COMPACT (Clinical Studies for Optimal Management in Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement Therapy) program, the study will evaluate the pharmacokinetics, pharmacodynamics and safety of various doses of this presentation of C1-INH.

CSL Behring announced today that it has initiated an international phase I/II study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor (C1-INH) concentrate in patients with hereditary angioedema (HAE). Part of the COMPACT (Clinical Studies for Optimal Management in Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement Therapy) program, the study will evaluate the pharmacokinetics, pharmacodynamics and safety of various doses of this presentation of C1-INH.

"We're enormously pleased that a range of therapy options is now available to treat HAE," said Bruce Zuraw, MD, Professor of Medicine at the University of California, San Diego, USA, and Chairman of the Steering Committee for the COMPACT program. "Yet, we know that a significant number of patients would prefer a preventive therapy that does not require intravenous infusions and that might offer fewer side effects than currently available treatments. We are confident this study will guide us in the development of this novel method of using C1-esterase inhibitor replacement for the routine prevention of HAE attacks."

The open-label study will enroll adult patients with HAE type I or II. After an initial intravenous injection of C1-INH, each participant will be assigned to receive a single subcutaneous injection of the volume-reduced formulation of C1-INH twice a week for four weeks. Subjects will participate in two such periods with two different doses of volume-reduced C1-INH. Throughout the study, researchers will monitor C1-esterase inhibitor levels in the blood, as well as assess the safety and tolerability of the formulation at different doses.

"As a pioneer in the development of subcutaneous immunoglobulin therapy, we are excited to apply our expertise and experience in the treatment of hereditary angioedema," said Russell Basser, Senior Vice President of Clinical Research and Development at CSL Behring. "Subcutaneous administration of C1-esterase inhibitor will represent another important advance for patients suffering from the frequent and often debilitating attacks of HAE."

About Hereditary Angioedema
Hereditary angioedema due to decreased C1-esterase inhibitor (C1-INH) is caused by mutations in SERPING1, the gene coding for C1-INH. It is inherited in an autosomal dominant manner. Symptoms of HAE include recurring episodes of edema, or swelling, in the hands, feet, the face, the abdomen, and/or the larynx. Patients who have abdominal attacks of HAE can experience episodes of severe pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face and larynx can result in airway closure, asphyxiation, and, if untreated, death. Diagnosis of HAE requires a blood test to confirm low or abnormal levels of C1-INH. For more information about HAE, please visit www.haei.org.

CSL Behring has currently licensed treatments in Australia, Canada, Europe, Japan, the United States and several other countries in Asia and South America for treatment of acute attacks of HAE.

About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients’ lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company’s therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, haemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com.

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Media contact:
Sheila A. Burke
Director, Public Relations & Communications
CSL Behring
610-878-4209

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