July 2, 2013
today announced that pharmacokinetic results for its novel investigational recombinant coagulation single-chain factor VIII (rVIII-SingleChain) showed improved half-life over octocog alfa (the comparator). It also demonstrated a safety and efficacy profile that supports advancement to late-stage clinical development. The data were presented at the International Society on Thrombosis and Haemostasis (ISTH) congress in
CSL Behring, in collaboration with its parent company,
(ASX:CSL), is developing rVIII-SingleChain for the treatment of hemophilia A as part of the AFFINITY clinical trial program.
"These data are promising and suggest that the recombinant single-chain design for Factor VIII may help address the need for a hemophilia A treatment with a longer half-life," said Professor
, M.D., of the Medical University of
. "A treatment with an improved half-life has the potential to increase the quality of life for those with severe hemophilia A by reducing the number of factor VIII protein infusions required to restore normal blood clotting."
The CSL Behring rVIII-SingleChain design uses a strong, covalent bond shown to improve the stability and half-life of factor VIII (FVIII). The investigational treatment is currently being studied in a Phase III trial.
"As part of our commitment to developing effective therapies to treat hemophilia, we sought to develop a novel recombinant single-chain Factor VIII design that improves the stability and half-life of factor VIII," said
, M.D., CSL Senior Vice President, Global Clinical Research & Development. "We are encouraged by these clinical results and very pleased that our investigation of rVIII-SingleChain molecule has progressed to Phase III."
About the Pharmacokinetic Study
The study enrolled 27 participants
18 years old with severe hemophilia A. Study participants had pharmacokinetic (PK) measurements performed over 72 hours for both octocog alfa and rVIII-SingleChain after they had received a single infusion of 50 IU/kg body weight of each of the compounds, respectively. In between study drug administration there was a 4-day minimum washout phase. Study objectives comprised the characterization of the PK profile of rVIII-SingleChain, the PK comparison of rVIII-SingleChain to octocog alfa and the characterization of the safety profile of rVIII-SingleChain.
The pharmacokinetics of rVIII-SingleChain and octocog alfa were assessed on the basis of FVIII activity. The following PK parameters were calculated for baseline-corrected FVIII activity using a non-compartmental model analysis with WinNonlin Phoenix (Version 6.3): The area under the plasma activity-time curve from time zero to the last quantifiable concentration (AUClast); the area under the plasma activity-time curve from time zero to infinity (AUCinf); the observed maximum plasma activity after drug administration (Cmax); incremental Recovery (IU/mL/IU/kg) defined as FVIII activity (IU/mL) obtained 30 minutes following infusion; clearance (CL), and terminal elimination half-life (t½).