CSL Behring enrolls first patient in global pediatric Phase III pivotal study of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) to treat hemophilia B

KING OF PRUSSIA, Pa., Jan. 21, 2013 /PRNewswire/ -- CSL Behring has announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children (up to age 11 years). The study site for this first enrollment is the Czech Republic.

"CSL continues to advance at a very encouraging rate with our recombinant coagulation factor development program," said Dr. Andrew Cutherbertson, Chief Scientist, CSL Limited. "The long history of profound expertise and commitment CSL brings to developing safe and effective therapies to improve the lives of those affected by rare and serious bleeding disorders remains the key to our ongoing success in this therapeutic area. With each clinical milestone we meet, we draw closer to our goal of bringing another truly innovative new treatment option to patients."

CSL Behring, in collaboration with its parent company, CSL Limited (ASX:CSL), is developing rIX-FP through the PROLONG-9FP clinical trial program for the prophylaxis and treatment of bleeding episodes, including control and prevention of bleeding in surgical settings in patients with factor IX deficiency.

Results of a Phase I study evaluating recombinant fusion protein linking coagulation factor IX  with albumin (rIX-FP) in patients with severe hemophilia B were publicly presented earlier this year and published in BLOOD 2012 showing that rIX-FP achieved a 91.57 hours terminal half-life, incremental recovery of 1.376 (IU/dL) / (IU/kg), and clearance of 0.75 mL/h/kg.  This was an extension in half-life of 5.3 times that of the current recombinant FIX therapy. 

Innovative Look at Tissue Distribution of rIX-FP Produce Early Results Presented at ASH

CSL Behring presented data at the American Society of Hematology on the tissue distribution of rIX-FP that demonstrated that rIX-FP was detectable five times longer in the tissues, including the bone. The extent and speed of onset of tissue penetration were similar for both rIX-FP and the study comparator product.

"These results demonstrate that rIX-FP was detectable significantly longer than the study comparator product, though they both behave in essentially the same way in terms of speed of onset and tissue penetration," said Dr. Stefan Schulte, Vice President of Research and Development at CSL Behring. "These results provide further evidence that rIX-FP is an extended half-life recombinant factor IX with the potential to reduce the number of injections needed in patients receiving prophylaxis from two or three injections per week with the study comparator product, to once weekly or significantly less frequently with rIX-FP. Further, they show that rIX-FP may have the potential to improve compliance and ease prophylaxis. This may, in turn, improve the quality of life for people with hemophilia B."

CSL Behring is currently recruiting patients for its adult phase II/III and pediatric phase III trials, the final phase of the PROLONG-9FP clinical trial program. More information can be found at www.clinicaltrials.gov.

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 is a subsidiary of CSL Limited (ASX:CSL), a biopharmaceutical company with headquarters in Melbourne, Australia. For more information visit http://www.cslbehring.com. CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma.

Media Contact:
Sheila A. Burke

Director, Worldwide Commercial Operations Communications & Public Relations
CSL Behring
610-878-4209

SOURCE CSL Behring