Long Residence Time at the Vasopressin V2 Receptor Translates into Superior Inhibitory Effects in Ex Vivo and In Vivo Models of Autosomal Dominant Polycystic Kidney Disease

J Med Chem. 2022 Jun 9;65(11):7717-7728. doi: 10.1021/acs.jmedchem.2c00011. Epub 2022 Apr 1.

Abstract

Prevailing strategies directing early-phase drug discovery heavily rely on equilibrium-based metrics such as affinity, which overlooks the kinetic process of a drug molecule interacting with its target. Herein, we developed a number of vasopressin V2 receptor (V2R) antagonists with divergent binding affinities and kinetics for autosomal dominant polycystic kidney disease (ADPKD). Surprisingly, the residence time of the V2R antagonists, but not their affinity, was correlated with the efficacy in both ex vivo and in vivo models of ADPKD. We envision that the kinetics-directed drug candidate selection and development may have general applicability for ADPKD and other therapeutic areas as well.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antidiuretic Hormone Receptor Antagonists* / chemistry
  • Antidiuretic Hormone Receptor Antagonists* / pharmacology
  • Drug Design
  • Humans
  • Polycystic Kidney, Autosomal Dominant* / drug therapy
  • Polycystic Kidney, Autosomal Dominant* / metabolism
  • Receptors, Vasopressin* / metabolism

Substances

  • AVPR2 protein, human
  • Antidiuretic Hormone Receptor Antagonists
  • Receptors, Vasopressin