CSL Behring has reported results from a pharmacokinetic study of novel investigational recombinant coagulation single-chain factor VIII (rVIII-SingleChain), which demonstrated advantages over multi-chain rFVIII for the treatment of Haemophilia A.
The study, part of the AFFINITY clinical trial programme, demonstrated an improved half-life of rVIII-SingleChain against the comparator, octocog alfa, in addition to a safety and efficacy profile that supports progress to late-stage clinical development.
The Medical University of Vienna, Austria, professor Dr Ingrid Pabinger-Fasching said that the data suggests that the recombinant single-chain design for Factor VIII may help address the need for haemophilia A treatment with a longer half-life.
"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," Pabinger-Fasching added.
The pharmacokinetic (PK) measurements were carried out for more than 72 hours for both octocog alfa and rVIII-SingleChain on a total of 27 adult haemophilia A patients, subsequent to a single infusion of 50 IU/kg body weight of each of the compounds, respectively.
The objectives were to characterise the PK profile of rVIII-SingleChain, the PK comparison of rVIII-SingleChain to octocog alfa on the basis of FVIII activity, and the characterisation of the safety profile of rVIII-SingleChain.
PK parameters were calculated for baseline-corrected FVIII activity using a non-compartmental model analysis with WinNonlin Phoenix under the plasma activity-time curve from time zero to the last quantifiable concentration.
Additional parameters include the area under the plasma activity-time curve from time zero to infinity, observed maximum plasma activity after drug administration, incremental recovery (IU/mL/IU/kg) defined as FVIII activity (IU/mL) obtained 30 minutes following infusion, clearance and terminal elimination half-life.
CSL global clinical research and development senior vice president Dr Russell Basser said: "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."