CSL Behring enrolls first patient in global pediatric Phase III pivotal study of recombinant factor VIII single chain (rVIII-Si

CSL Behring enrolls first patient in global pediatric Phase III pivotal study
 of recombinant factor VIII single chain (rVIII-SingleChain) to treat severe
                                 hemophilia A

PR Newswire

KING OF PRUSSIA, Pa., May 2, 2014

KING OF PRUSSIA, Pa., May 2, 2014 /PRNewswire/ --CSL Behring today announced
that the first patient has been enrolled in the pivotal pediatric phase III
study to evaluate the efficacy, safety and pharmacokinetics of its novel
investigational recombinant factor VIII single chain (rVIII-SingleChain) for
the treatment of previously treated children (up to age 11 years) with severe
hemophilia A. The study site for this first enrollment is Malaysia.

A minimum of 25 previously treated subjects from six to 11 years of age and at
least 25 subjects under six years of age who have undergone more than 50
exposure days with a previous factor VIII product are planned to be enrolled
in this international, multicenter, open-label study. Subjects will be
assigned to either an on-demand or prophylaxis treatment regimen for the
treatment of bleeding episodes and will receive rVIII-SingleChain at a dose to
be determined by the investigator. Hemostatic efficacy will be assessed by the
subject or caregiver and the investigator, who will assess overall efficacy by
a 4-point scale.

In an earlier study, rVIII-SingleChain showed improved pharmacokinetics over
octocog alfa, the comparator, and demonstrated a safety and efficacy profile
that supported advancement to late-stage clinical development. CSL Behring, in
collaboration with its parent company CSL Limited, is developing
rVIII-SingleChain for the treatment of hemophilia A as part of the AFFINITY
clinical trial program.

About rVIII-SingleChain
Recombinant FVIII molecules currently available consist of a heavy and a light
chain. Under certain conditions, these chains can dissociate, resulting in the
formation of separated, or "dissociated," rFVIII chains that are not
hemostatically active. The CSL Behring rVIII-SingleChain uses a strong,
covalent bond that connects the light and heavy chains, thereby creating a
stable single chain rFVIII.

In-house CSL Behring studies have shown that the molecular integrity of
rVIII-SingleChain is significantly increased using the single-chain design,
resulting in a homogenous product that is more stable than currently available
FVIII products. In addition, in-vitro studies have shown that
rVIII-SingleChain demonstrates a strong affinity for von Willebrand factor
(VWF), resulting in a faster and more efficient binding to VWF. The FVIII/VWF
complex plays an important role in the physiological activity and clearance of
FVIII and has been shown to have an influence on the presentation of FVIII to
the immune system.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or
spontaneous bleeding, especially into the muscles, joints, or internal organs.
In nearly all cases, it affects only males. The disease is caused by deficient
or defective blood coagulation proteins known as factor VIII or IX. The most
common form of the disease is hemophilia A, or classic hemophilia, in which
the clotting factor VIII is either deficient or defective. Hemophilia B is
characterized by deficient or defective factor IX. Hemophilia A affects
approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately
1 in 25,000 to 50,000 people. The recommended treatment for people with
hemophilia deficiency is to treat by replacement factor therapy.

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 used around the world to treat coagulation disorders
including hemophilia and von Willebrand disease, primary immune deficiencies,
hereditary angioedema and inherited respiratory disease, and neurological
disorders in certain markets. The company's products are also used in cardiac
surgery, organ transplantation, burn treatment and to prevent hemolytic
diseases in the newborn. CSL Behring 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.

Sheila A. Burke
Director, Communications & Public Relations
Worldwide Commercial Operations
CSL Behring
O: 610-878-4209
Sheila.Burke at cslbehring.com


Website: http://www.cslbehring.com
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