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. 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.