PXL770

PXL770 is a novel, first-in-class direct activator of adenosine monophosphate-activated protein kinase (AMPK), developed as an orally active small molecule for the treatment of chronic and rare metabolic diseases. By directly activating AMPK through allosteric binding to the AdaM site, PXL770 modulates a master regulator of cellular energy homeostasis. This mechanism leads to inhibition of hepatic de novo lipogenesis, improved glucose tolerance and insulin sensitivity, and reduction in plasma di- and triglyceride levels. The drug has been investigated for non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), autosomal dominant polycystic kidney disease (ADPKD), X-linked adrenoleukodystrophy/adrenomyeloneuropathy (ALD/AMN), type 2 diabetes mellitus, and cardiomyopathies. It has received Orphan Drug Designation from both the FDA and European Commission for ALD/AMN and ADPKD[3][4][5][7][8].