Biotie’s BTT1023 receives positive opinion for Orphan Drug Designation from COMP

The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has in its February meeting issued a positive opinion recommending orphan drug designation for Biotie’s BTT1023 drug candidate for the treatment of primary sclerosing cholangitis (PSC). PSC is a chronic and progressive fibrotic liver disease for which there are currently no effective therapeutic treatments.

BTT1023 is a fully human monoclonal antibody targeting Vascular Adhesion Protein -1 (VAP-1) and is currently advancing into Phase 2 clinical development. The BUTEO study will be an open label, single arm, multi-centre investigator-sponsored study that will be conducted in partnership with the University of Birmingham, UK and supported through external grant funding. The study is expected to enroll 41 patients and will examine the efficacy, safety and pharmacokinetic properties of BTT1023 in PSC patients. The duration of drug treatment in the study is 11 weeks and the primary efficacy endpoint is reduction of elevated levels of alkaline phosphatase, a blood biomarker of bile duct inflammation. The BUTEO study will be conducted in the UK and is expected to start recruiting patients in Q1/2015.

About BTT1023

BTT1023 is a fully human monoclonal antibody that specifically binds to vascular adhesion protein-1 (VAP-1). BTT1023 has demonstrated encouraging efficacy and safety in early clinical studies in rheumatoid arthritis and psoriasis patients and in a range of preclinical models of inflammatory diseases, including COPD. More recently, an important role for VAP-1 has also been demonstrated in fibrotic diseases.

BTT1023 is transitioning into Phase 2 clinical development in primary sclerosing cholangitis (PSC), a chronic and progressive fibrotic liver disease for which there are currently no effective therapeutic treatments. BTT1023 has received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), recommending Orphan Drug Designation for BTT1023 for the treatment of PSC.