Translating slow-binding inhibition kinetics into cellular and in vivo effects

Nat Chem Biol. 2015 Jun;11(6):416-23. doi: 10.1038/nchembio.1796. Epub 2015 Apr 20.

Abstract

Many drug candidates fail in clinical trials owing to a lack of efficacy from limited target engagement or an insufficient therapeutic index. Minimizing off-target effects while retaining the desired pharmacodynamic (PD) response can be achieved by reduced exposure for drugs that display kinetic selectivity in which the drug-target complex has a longer half-life than off-target-drug complexes. However, though slow-binding inhibition kinetics are a key feature of many marketed drugs, prospective tools that integrate drug-target residence time into predictions of drug efficacy are lacking, hindering the integration of drug-target kinetics into the drug discovery cascade. Here we describe a mechanistic PD model that includes drug-target kinetic parameters, including the on- and off-rates for the formation and breakdown of the drug-target complex. We demonstrate the utility of this model by using it to predict dose response curves for inhibitors of the LpxC enzyme from Pseudomonas aeruginosa in an animal model of infection.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amidohydrolases / antagonists & inhibitors*
  • Animals
  • Anti-Bacterial Agents / chemistry
  • Anti-Bacterial Agents / pharmacokinetics
  • Anti-Bacterial Agents / pharmacology*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology*
  • Hydroxamic Acids / chemistry
  • Hydroxamic Acids / pharmacokinetics
  • Hydroxamic Acids / pharmacology*
  • Kinetics
  • Mice, Inbred Strains
  • Microbial Sensitivity Tests
  • Models, Biological
  • Molecular Structure
  • Protein Binding
  • Pseudomonas aeruginosa / drug effects
  • Pseudomonas aeruginosa / enzymology
  • Threonine / analogs & derivatives*
  • Threonine / chemistry
  • Threonine / pharmacokinetics
  • Threonine / pharmacology
  • Time Factors

Substances

  • Anti-Bacterial Agents
  • CHIR 090
  • Enzyme Inhibitors
  • Hydroxamic Acids
  • Threonine
  • Amidohydrolases
  • LpxC deacetylase, Pseudomonas