Abstracts include pivotal efficacy and safety data for CSL Behring’s long-acting recombinant albumin fusion protein for hemophilia B; CSL Behring’s recombinant coagulation factor development program reinforces the company’s promise to develop specialty biotherapies that help people with serious medical conditions live full lives.
CSL Behring announced today that it will present several abstracts from its recombinant coagulation factor development programs at the American Society of Hematology’s (ASH) 57th ASH Annual Meeting in Orlando, December 5-8. An oral presentation will focus on the pivotal trial for CSL Behring’s novel, long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the treatment of hemophilia B.
Hemophilia is an inherited bleeding disorder caused by deficient or defective proteins that prevent the blood from clotting properly. According to the National Hemophilia Foundation, the condition affects approximately 400,000 people worldwide, the majority of whom have hemophilia A.
“The clinical findings that CSL Behring is presenting at ASH reinforce our promise to advance hemophilia care by developing innovative specialty biotherapies that have the potential to improve patients’ lives,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D, CSL Limited. “For nearly a century, CSL has developed and delivered innovations that have helped people with serious medical conditions live full lives. Our recombinant coagulation factor development pipeline is another example of our R&D focus to deliver treatment advancements that patients and providers want.”
CSL Behring medical representatives will be available at booth number 228 throughout the ASH Annual Meeting. Key abstracts from CSL Behring at ASH include:
Monday, December 7, 10:30 a.m. – 12:00 p.m. ET
- Efficacy and safety results of PROLONG-9FP clinical program of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients with hemophilia B. Session Name: 322. Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia.Oral Presentation Abstract #548
Saturday, December 5, 5:30 p.m. – 7:30 p.m. ET
- Long-Term Use of coagulation factor IX (Recombinant) albumin fusion protein (rIX-FP) in previously treated patients with hemophilia B in the PROLONG-9FP program. Poster Presentation Abstract # 1096
Sunday, December 6, 6:00 p.m. – 8:00 p.m. ET
- Efficacy and safety of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients with hemophilia B undergoing major orthopedic surgeries. Poster Presentation Abstract # 2298
Saturday, December 5, 5:30 p.m. - 7:30 p.m. ET
- Pharmacokinetic properties of different rFVIII products in rabbits, rats and FVIII ko mice. Poster Presentation Abstract #1078
Monday, December 7, 6:00-9:00 p.m. ET
- rVIII-SingleChain in surgical prophylaxis: Efficacy and safety of continuous and bolus infusion. Poster Presentation Abstract # 3525
Monday, December 7, 6:00-8:00 p.m. ET
- rVIII-SingleChain pharmacokinetics and safety in children less than 12 years of age. Poster Presentation Abstract #3524
CSL Behring engineered rIX-FP to extend the half-life of recombinant factor IX through genetic fusion with recombinant albumin. CSL Behring selected albumin as its recombinant genetic fusion partner due to its long physiological half-life. In addition, albumin has been shown to have a favorable tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance. The cleavable linker connecting recombinant factor IX and recombinant albumin has been specifically designed to preserve the native function of the coagulation factor in the fusion protein, while benefiting from recombinant albumin’s long physiological half-life.
The U.S. Food and Drug Administration, European Medicines Agency and Swissmedic are currently reviewing CSL Behring’s license applications for rIX-FP. Upon regulatory approvals, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up to 14 days.
The PROLONG-9FP clinical development program for rIX-FP covers patients from the age of 1 to 61 years. Studies in the program were conducted as open-label, multicenter, safety and efficacy studies of rIX-FP in previously treated patients with hemophilia B (factor IX level ≤ 2%). Study design details for rIX-FP (CSL654) are available at clinicaltrials.gov (NCT01496274).
Specifically designed for greater molecular stability, rVIII-SingleChain is the first and only recombinant single-chain factor VIII (FVIII) product in late-stage development for the treatment of hemophilia A. rVIII-SingleChain has a strong affinity for von Willebrand factor, leading to greater stability and integrity of factor VIII in circulation. The stability of rVIII-SingleChain leads to a longer-lasting therapeutic effect with reduced dosing frequency.
In July, the U.S. Food and Drug Administration accepted for review CSL Behring’s BLA for rVIII-SingleChain. The BLA submission is based on the AFFINITY clinical development program, which includes a phase I/III open-label, multi-center trial examining safety and efficacy. The pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alfa) were also studied. Study design details for rVIII-SingleChain (CSL627) are available at clinicaltrials.gov (NCT01486927).
For more information about CSL Behring's recombinant coagulation factor products in development to treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novel-recombinant-hemophilia-treatments.
CSL (ASX:CSL) is a leading global biotherapeutics company with a dynamic portfolio of life-saving innovations, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL — including our two businesses CSL Behring and Seqirus — operates in over 30 countries with more than 14,000 employees. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.
For more information about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.
Email: Greg. Healy@cslbehring.com