CSL Behring has announced that the first patient has been screened for an open-label, multicenter Phase I/III clinical study into a single chain recombinant factor VIII (rFVIII) product called “rFVIII-SingleChain.” This developmental therapy uses a pair of linked protein chains–a heavy one and a light one–that are bonded together. The resultant single rFVIII exhibits stronger molecular integrity, which in turn yields a more stable product. The new trial is part of CSL’s AFFINITY clinical trial program. The therapy is being designed for patients with hemophilia A.
rVIII-SingleChain has also shown a strong affinity for von Willebrand factor (VWF), resulting in faster, more efficient binding to VWF, which is a carrier molecule for FVIII . The FVIII/VWF complex plays an important role in presenting FVIII to the immune system. The trial will measure the safety, efficacy and pharmacokinetics of rFVIII-SingleChain.
“CSL is advancing at a very encouraging rate with our recombinant coagulation factor development program,” said Andrew Cuthbertson, Chief Scientist of CSL Behring’s parent company, CSL Limited. “Our ongoing commitment to providing safe and effective therapies to help improve the lives of those affected by hemophilia A and other rare bleeding disorders is being fulfilled with each milestone we reach. Our recombinant coagulation single-chain factor VIII molecule is showing strong promise. We look forward with excitement to seeing results of the phase I/III study in coming months.”
Source: CSL Behring news release dated February 24, 2012