Boehringer Ingelheim initiates Phase IIa study of liver disease drug

Pharmaceutical company Boehringer Ingelheim has initiated a European and North American Phase IIa trial in non-alcoholic steatohepatitis (NASH) of investigational drug candidate BI 1467335 (formerly known as PXS-4728A), acquired from fellow pharma company Pharmaxis in May 2015.

Non-alcoholic fatty liver disease (NAFLD) is the most common liver disorder in Western industrialised nations; its more serious form, NASH, is particularly prevalent amongst patients with type 2 diabetes. NASH is a major cause of liver fibrosis and cirrhosis and is an area of high unmet medical need, with no treatments currently available.

In 2016 Boehringer Ingelheim obtained Fast Track designation from the US FDA for the development of BI 1467335 in NASH. The compound is an oral inhibitor of amine oxidase, copper containing 3 (AOC3), and works by blocking leucocyte adhesion and tissue infiltration in inflammatory processes underlying NASH.

The new Phase IIa trial is a multicentre, double-blind design in 150 patients with clinical evidence of NASH. The primary objectives are to establish proof of clinical principle, investigate suitable dosing and evaluate the safety of BI 1467335. Patients will be randomised to either one of four dosages of BI 1467335 or to placebo for a 12-week treatment period. A subsequent Phase IIb study will seek to confirm and extend these findings.