Optimizing the Benefit/Risk of Acetyl-CoA Carboxylase Inhibitors through Liver Targeting

J Med Chem. 2020 Oct 8;63(19):10879-10896. doi: 10.1021/acs.jmedchem.0c00640. Epub 2020 Sep 18.

Abstract

Preclinical and clinical data suggest that acetyl-CoA carboxylase (ACC) inhibitors have the potential to rebalance disordered lipid metabolism, leading to improvements in nonalcoholic steatohepatitis (NASH). Consistent with these observations, first-in-human clinical trials with our ACC inhibitor PF-05175157 led to robust reduction of de novo lipogenesis (DNL), albeit with concomitant reductions in platelet count, which were attributed to the inhibition of fatty acid synthesis within bone marrow. Herein, we describe the design, synthesis, and evaluation of carboxylic acid-based ACC inhibitors with organic anion transporting polypeptide (OATP) substrate properties, which facilitated selective distribution of the compounds at the therapeutic site of action (liver) relative to the periphery. These efforts led to the discovery of clinical candidate PF-05221304 (12), which selectively inhibits liver DNL in animals, while demonstrating considerable safety margins against platelet reduction in a nonhuman primate model.

MeSH terms

  • Acetyl-CoA Carboxylase / antagonists & inhibitors*
  • Acetyl-CoA Carboxylase / metabolism
  • Animals
  • Drug Delivery Systems*
  • Enzyme Inhibitors / pharmacology*
  • Enzyme Inhibitors / therapeutic use
  • Humans
  • Lipogenesis
  • Liver / drug effects*
  • Non-alcoholic Fatty Liver Disease / drug therapy
  • Substrate Specificity

Substances

  • Enzyme Inhibitors
  • Acetyl-CoA Carboxylase