First patient dosed in CSL Behring global Phase II/III pivotal study of recombinant fusion protein linking coagulation factor IX with recombinant albumin
Study follows a Phase I PK trial that showed "very promising" half-life extension results
Hannover, Germany — 20 March 2012
CSL Behring has announced that the first patient has been dosed in a phase II/III, prospective, open-label, multi-center study designed to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). The study will enroll adolescents and adults (12 – 65 years) who have hemophilia B. The patient was dosed at Werlhof Institute for Hemostasis and Thrombosis in Hannover, Germany.
CSL Behring, in collaboration with its parent company,
CSL Limited (ASX:CSL), is developing rIX-FP for the prophylaxis and treatment of bleeding episodes in patients with congenital Factor IX (FIX) deficiency as part of the PROLONG-9FP clinical study program.
To date, the PROLONG-9FP program has established study sites in Austria, Bulgaria, France, Germany, Italy, Spain, Japan and Israel. CSL Behring plans to establish additional trial sites in the United States and Russia in coming months.
CSL Behring has engineered rIX-FP to extend the half-life of Factor IX through genetic fusion with recombinant albumin. Albumin was chosen as an ideal recombinant fusion partner due to its inherently long half-life, high potential for tolerability, known mechanism of clearance and low potential for immunogenic reactions. The specifically designed linker between the recombinant factor IX and recombinant albumin enables the fusion protein to optimally benefit from recombinant albumin’s inherently long half-life while preserving the native function of the coagulation factor. The Phase II/III study consists of a screening period, a pharmacokinetic (PK) evaluation period, followed by an approximate 12-month safety and efficacy evaluation period with rIX-FP. A surgical prophylaxis sub-trial is included. More information can be found at
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 patients who are factor deficient 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 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 diseases in newborns. 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.
Sheila A. Burke