Biotie Therapies Corp announces the start of patient enrolment into the Phase 2a clinical study evaluating BTT1023, Biotie's fully human monoclonal antibody targeting Vascular Adhesion Protein-1, in primary sclerosing cholangitis (PSC). PSC is a progressive immune mediated biliary disease characterised by bile duct inflammation and fibrosis, and accompanying hepatic fibrosis, that frequently results in the need for liver transplantation. The study is being funded through the Efficacy and Mechanism Evaluation (EME) Programme, an MRC and NIHR partnership*.
The BUTEO study (BTT1023, a human monoclonal antibody targeting vascular adhesion protein (VAP-1), in the treatment of patients with primary sclerosing cholangitis) is an investigator-sponsored open label, single arm, multi-centre study that will evaluate efficacy, safety and pharmacokinetic properties of BTT1023 in 41 patients with PSC. Patients will receive BTT1023 via intravenous infusion every two weeks over an 11 week treatment period. The primary efficacy endpoint is a reduction of elevated levels of alkaline phosphatase, a blood biomarker of bile duct inflammation; secondary endpoints include various measures of liver injury and fibrosis.
The grant holder and Co-Investigator for the study, Professor David Adams, Director of the National Institute for Health Research (NIHR) Biomedical Research Unit in Liver Disease and Centre for Liver Research at the University of Birmingham, UK, said "We have demonstrated that PSC is driven by aberrant lymphocyte homing and were the first to report a role for VAP-1 in mediating liver inflammation and fibrosis. We are excited to be working with Biotie to investigate whether blocking VAP-1 with BTT1023 can offer the first effective therapeutic option for this life-changing disease."
The two-stage study design includes a pre-planned futility analysis. Based on current estimates, it is expected that the requisite number of patients will have been treated by the end of 2016 to enable the futility analysis to be completed.
Clinicaltrials.gov identifier: NCT02239211.