Computational Design, Synthesis, and Biological Evaluation of Diimidazole Analogues Endowed with Dual PCSK9/HMG-CoAR-Inhibiting Activity

J Med Chem. 2023 Jun 22;66(12):7943-7958. doi: 10.1021/acs.jmedchem.3c00279. Epub 2023 Jun 1.

Abstract

Proprotein convertase subtilisin/kexin 9 (PCSK9) is responsible for the degradation of the hepatic low-density lipoprotein receptor (LDLR), which regulates circulating cholesterol levels. Consequently, the PCSK9 inhibition is a valuable therapeutic approach for the treatment of hypercholesterolemia and cardiovascular diseases. In our studies, we discovered Rim13, a polyimidazole derivative reducing the protein-protein interaction between PCSK9 and LDLR with an IC50 of 1.6 μM. The computational design led to the optimization of the shape of the PCSK9/ligand complementarity, enabling the discovery of potent diimidazole derivatives. In fact, carrying out biological assays to fully characterize the cholesterol-lowering activity of the new analogues and using both biochemical and cellular techniques, compound Dim16 displayed improved PCSK9 inhibitory activity (IC50 0.9 nM). Interestingly, similar to other lupin-derived peptides and their synthetic analogues, some compounds in this series showed dual hypocholesterolemic activity since some of them complementarily inhibited the 3-hydroxy-3-methylglutaryl coenzyme A reductase.

MeSH terms

  • Cholesterol
  • Proprotein Convertase 9* / metabolism
  • Proprotein Convertases / metabolism
  • Receptors, LDL / metabolism
  • Subtilisin*

Substances

  • PCSK9 protein, human
  • Proprotein Convertase 9
  • Subtilisin
  • Proprotein Convertases
  • Receptors, LDL
  • Cholesterol