First patient enrolled in phase III of COMPACT, a study of volume-reduced subcutaneous C1-INH for prevention of Hereditary

  First patient enrolled in phase III of COMPACT, a study of volume-reduced
  subcutaneous C1-INH for prevention of Hereditary Angioedema (HAE) attacks

International study by CSL Behring is world's first placebo-controlled phase
III trial to examine subcutaneous administration of C1-INH for use in treating
rare, sometimes life-threatening, condition

PR Newswire

KING OF PRUSSIA, Pa., Feb. 20, 2014

KING OF PRUSSIA, Pa., Feb. 20, 2014 /PRNewswire/ -- CSL Behring today
announced it has enrolled the first patient in COMPACT, an international phase
III study of a volume-reduced, subcutaneous formulation of C1-esterase
inhibitor (C1-INH) concentrate in patients with frequent hereditary angioedema
(HAE) attacks (NCT01912456). This phase of the COMPACT program will assess the
efficacy and safety of a new formulation of the CSL Behring C1-INH concentrate
in preventing hereditary angioedema attacks when the therapy is administered
twice weekly under the skin (i.e., subcutaneously) of patients diagnosed with

COMPACT is an acronym for Clinical Studies for Optimal Management in
Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement

"To date, COMPACT has shown that various doses of this volume-reduced
formulation of C1-INH concentrate are well tolerated when administered at a
single infusion site twice weekly," 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. "We also observed a
dose-dependent, physiologically relevant increase in functional C1-INH plasma
levels. From a clinical perspective, these results are intriguing and could
lead to a more convenient option for people with HAE."

The COMPACT phase III, double-blind, randomized, placebo-controlled,
cross-over study enrolls adolescent and adult patients with HAE types I or II
who have frequent attacks. The study will measure the number of hereditary
angioedema attacks that subjects experience while receiving each treatment.
Subjects will be able to take on-demand medication for acute attacks at any
time during the study.

"The COMPACT study is an important demonstration of the commitment CSL Behring
has to the HAE community," said Russell Basser, Senior Vice President of
Clinical Research and Development at CSL Behring. "CSL Behring has been a
leader in this area for decades, so we are confident that our current efforts
to develop a safe, effective and convenient new treatment option for HAE
patients will be successful."

Additional information about the COMPACT trial and participating centers can
be found here:

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 or

CSL Behring  currently has licensed a C1-inhibitor product 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 leader in the plasma protein therapeutics industry. Committed
to saving lives and improving the quality of life for people with rare and
serious diseases, the company manufactures and markets a range of
plasma-derived and recombinant therapies worldwide. CSL Behring therapies are
indicated for the treatment of coagulation disorders including hemophilia and
von Willebrand disease, primary immune deficiencies, hereditary angioedema and
inherited respiratory disease. The company's products are also used in cardiac
surgery, organ transplantation, burn treatment and to prevent hemolytic
disease of the newborn. CSL Behring operates one of the world's largest plasma
collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited
(ASX:CSL), a biopharmaceutical company headquartered in Melbourne, Australia.
For information:

Media contact: Sheila A. Burke, CSL Behring, 610-878-4209 (US)


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