From chemical tools to clinical medicines: nonimmunosuppressive cyclophilin inhibitors derived from the cyclosporin and sanglifehrin scaffolds

J Med Chem. 2014 Sep 11;57(17):7145-59. doi: 10.1021/jm500223x. Epub 2014 Jun 3.

Abstract

The cyclophilins are widely expressed enzymes that catalyze the interconversion of the cis and trans peptide bonds of prolines. The immunosuppressive natural products cyclosporine A and sanglifehrin A inhibit the enzymatic activity of the cyclophilins. Chemical modification of both the cyclosporine and sanglifehrin scaffolds has produced many analogues that inhibit cyclophilins in vitro but have reduced immunosuppressive properties. Three nonimmunosuppressive cyclophilin inhibitors (alisporivir, SCY-635, and NIM811) have demonstrated clinical efficacy for the treatment of hepatitis C infection. Additional candidates are in various stages of preclinical development for the treatment of hepatitis C or myocardial reperfusion injury. Recent publications suggest that cyclophilin inhibitors may have utility for the treatment of diverse viral infections, inflammatory indications, and cancer. In this review, we document the structure-activity relationships of the nonimmunosuppressive cyclosporins and sanglifehrins in clinical and preclinical development. Aspects of the pharmacokinetic behavior and chemical biology of these drug candidates are also described.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antiviral Agents / chemistry*
  • Antiviral Agents / metabolism
  • Antiviral Agents / therapeutic use
  • Chemistry, Pharmaceutical / methods
  • Chemistry, Pharmaceutical / trends
  • Cyclophilins / antagonists & inhibitors
  • Cyclophilins / chemistry*
  • Cyclophilins / metabolism
  • Cyclosporine / chemistry*
  • Cyclosporine / metabolism
  • Cyclosporine / therapeutic use
  • Cyclosporins / chemistry*
  • Cyclosporins / metabolism
  • Cyclosporins / therapeutic use
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / metabolism
  • Enzyme Inhibitors / therapeutic use
  • Hepacivirus / drug effects
  • Hepacivirus / physiology
  • Hepatitis C / drug therapy
  • Hepatitis C / virology
  • Host-Pathogen Interactions / drug effects
  • Humans
  • Lactones / chemistry
  • Models, Molecular
  • Molecular Structure
  • Protein Binding
  • Protein Structure, Tertiary
  • Spiro Compounds / chemistry
  • Structure-Activity Relationship

Substances

  • Antiviral Agents
  • Cyclosporins
  • Enzyme Inhibitors
  • Lactones
  • Spiro Compounds
  • sanglifehrin A
  • Cyclosporine
  • (melle-4)cyclosporin
  • Cyclophilins
  • alisporivir
  • SCY-635