Azepanone-based inhibitors of human and rat cathepsin K

J Med Chem. 2001 Apr 26;44(9):1380-95. doi: 10.1021/jm000481x.

Abstract

The synthesis, in vitro activities, and pharmacokinetics of a series of azepanone-based inhibitors of the cysteine protease cathepsin K (EC 3.4.22.38) are described. These compounds show improved configurational stability of the C-4 diastereomeric center relative to the previously published five- and six-membered ring ketone-based inhibitor series. Studies in this series have led to the identification of 20, a potent, selective inhibitor of human cathepsin K (K(i) = 0.16 nM) as well as 24, a potent inhibitor of both human (K(i) = 0.0048 nM) and rat (K(i,app) = 4.8 nM) cathepsin K. Small-molecule X-ray crystallographic analysis of 20 established the C-4 S stereochemistry as being critical for potent inhibition and that unbound 20 adopted the expected equatorial conformation for the C-4 substituent. Molecular modeling studies predicted the higher energy axial orientation at C-4 of 20 when bound within the active site of cathepsin K, a feature subsequently confirmed by X-ray crystallography. Pharmacokinetic studies in the rat show 20 to be 42% orally bioavailable. Comparison of the transport of the cyclic and acyclic analogues through CaCo-2 cells suggests that oral bioavailability of the acyclic derivatives is limited by a P-glycoprotein-mediated efflux mechanism. It is concluded that the introduction of a conformational constraint has served the dual purpose of increasing inhibitor potency by locking in a bioactive conformation as well as locking out available conformations which may serve as substrates for enzyme systems that limit oral bioavailability.

MeSH terms

  • Administration, Oral
  • Animals
  • Azepines / chemical synthesis*
  • Azepines / chemistry
  • Azepines / pharmacokinetics
  • Azepines / pharmacology
  • Biological Availability
  • Cathepsin K
  • Cathepsins / antagonists & inhibitors*
  • Chromatography, High Pressure Liquid
  • Crystallography, X-Ray
  • Enzyme Inhibitors / chemical synthesis*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology
  • Humans
  • In Vitro Techniques
  • Leucine / analogs & derivatives
  • Leucine / chemical synthesis*
  • Leucine / chemistry
  • Leucine / pharmacokinetics
  • Leucine / pharmacology
  • Mass Spectrometry
  • Models, Molecular
  • Molecular Structure
  • Osteoclasts / drug effects
  • Protein Binding
  • Rats
  • Stereoisomerism
  • Structure-Activity Relationship

Substances

  • 5-(2-morpholin-4-ylethoxy)benzofuran-2-carboxylic acid (3-methyl-1-(3-oxo-1-(2-(3-pyridin-2-ylphenyl)acetyl)azepan-4-ylcarbamoyl)butyl)amide
  • Azepines
  • Enzyme Inhibitors
  • Cathepsins
  • CTSK protein, human
  • Cathepsin K
  • Ctsk protein, rat
  • Leucine