Repositioning of a GABA-B receptor agonist, AZD3355 (Lesogaberan), for the treatment of Non-Alcoholic Steatohepatitis

Non-alcoholic steatohepatitis (NASH) is a rising health challenge, with no approved drugs. We used a computational drug repositioning strategy to uncover a novel therapy for NASH, identifying a GABA-B receptor agonist, AZD3355 (lesogaberan) previously evaluated as a therapy for esophageal reflux. AZD3355's potential efficacy in NASH was tested in human stellate cells. Transcriptomic analysis of these responses identified key regulatory nodes impacted by AZD3355. Overall design: To investigate the effects of AZD3355 we treated primary human hepatic stellate cells with 30 nM of AZD3355 for 72 hours and then performed transcriptomic analysis.