Series of Novel and Highly Potent Cyclic Peptide PCSK9 Inhibitors Derived from an mRNA Display Screen and Optimized via Structure-Based Design

J Med Chem. 2020 Nov 25;63(22):13796-13824. doi: 10.1021/acs.jmedchem.0c01084. Epub 2020 Nov 10.

Abstract

Proprotein convertase subtilisin-like/kexin type 9 (PCSK9) is a key regulator of plasma LDL-cholesterol (LDL-C) and a clinically validated target for the treatment of hypercholesterolemia and coronary artery disease. In this paper, we describe a series of novel cyclic peptides derived from an mRNA display screen which inhibit the protein-protein interaction between PCSK9 and LDLR. Using a structure-based drug design approach, we were able to modify our original screening lead 2 to optimize the potency and metabolic stability and minimize the molecular weight to provide novel bicyclic next-generation PCSK9 inhibitor peptides such as 78. These next-generation peptides serve as a critical foundation for continued exploration of potential oral, once-a-day PCSK9 therapeutics for the treatment of cardiovascular disease.

MeSH terms

  • Animals
  • Cells, Cultured
  • Crystallography, X-Ray / methods
  • Drug Design*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / metabolism*
  • Female
  • Humans
  • Macaca fascicularis
  • Male
  • Mice
  • Mice, Inbred C57BL
  • PCSK9 Inhibitors*
  • Proprotein Convertase 9 / chemistry
  • Proprotein Convertase 9 / metabolism*
  • Protein Structure, Secondary
  • Protein Structure, Tertiary
  • RNA, Messenger / chemistry
  • RNA, Messenger / metabolism*
  • Rats
  • Rats, Wistar
  • Structure-Activity Relationship

Substances

  • Enzyme Inhibitors
  • PCSK9 Inhibitors
  • RNA, Messenger
  • PCSK9 protein, human
  • Proprotein Convertase 9