Biotie’s BTT1023 drug recently received positive feedback regarding Orphan Drug Designation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). BTT1023 is an experimental therapy indicated for the treatment of primary sclerosing cholangitis (PSC), a rare and severe chronic condition that can progress to liver fibrosis, and for which no treatment has been found.
Biotie’s BTT1023 is a human monoclonal antibody that binds to the Vascular Adhesion Protein -1 (VAP-1). Previous studies have found that the drug is effective and safe in inflammatory and fibrotic conditions, such as rheumatoid arthritis and psoriasis, as well as in pre-clinical studies in models of COPD. Furthermore, because the Company has linked VAP-1 to fibrotic diseases, BTT1023 or a related compound may open doors to new ways to address pulmonary fibrosis as well.
The BUTEO Phase 2 clinical trial will evaluate the drug’s efficacy in an open label, multi center study. The study is single-arm and is going to be conducted in collaboration with the University of Birmingham in the United Kingdom, and funded by an external company.
The study will test BTT1023’s effectiveness, safety and pharmacokinetics in 41 patients with primary sclerosing cholangitis. Patients’ enrollment in the drug study is expected to last 11 weeks. BUTEO’s primary endpoint is aimed at the reduction of the high levels of alkaline phosphatase, a biomarker of bile duct inflammation, which is detected in the blood. Patients for the study will be recruited in the United Kingdom and the first patient is expected to enroll during the first months of 2015.
Biotie is focused on the development of drugs for neurodegenerative and psychiatric conditions, including Selincro (nalmefene) for alcohol dependence, which was approved in Europe in 2013 and is currently distributed in Europe by Lundbeck.
The company is currently working on tozadenant for Parkinson’s disease, in a transitioning into Phase 3 study. Furthermore, the company is testing three compounds, currently in Phase 2 studies, targeting Parkinson’s disease dementia and cocaine dependence. The Company is expected to explore BTT1023’s application in inflammatory and fibrotic pulmonary diseases upon its success in treating PSC.
In other developments related to respiratory health, Enterprise Therapeutics Ltd, a startup drug discovery company founded in May, 2014 with a central research and development focus on new respiratory disease therapeutics, has announced that it has secured $2.4 million in initial Series-A financing from healthcare oriented venture capital firm Epidarex Capital to address chronic respiratory ailments.