Dual A1/A3 Adenosine Receptor Antagonists: Binding Kinetics and Structure-Activity Relationship Studies Using Mutagenesis and Alchemical Binding Free Energy Calculations

J Med Chem. 2022 Oct 13;65(19):13305-13327. doi: 10.1021/acs.jmedchem.2c01123. Epub 2022 Sep 29.

Abstract

Drugs targeting adenosine receptors (AR) can provide treatment for diseases. We report the identification of 7-(phenylamino)-pyrazolo[3,4-c]pyridines L2-L10, A15, and A17 as low-micromolar to low-nanomolar A1R/A3R dual antagonists, with 3-phenyl-5-cyano-7-(trimethoxyphenylamino)-pyrazolo[3,4-c]pyridine (A17) displaying the highest affinity at both receptors with a long residence time of binding, as determined using a NanoBRET-based assay. Two binding orientations of A17 produce stable complexes inside the orthosteric binding area of A1R in molecular dynamics (MD) simulations, and we selected the most plausible orientation based on the agreement with alanine mutagenesis supported by affinity experiments. Interestingly, for drug design purposes, the mutation of L2506.51 to alanine increased the binding affinity of A17 at A1R. We explored the structure-activity relationships against A1R using alchemical binding free energy calculations with the thermodynamic integration coupled with the MD simulation (TI/MD) method, applied on the whole G-protein-coupled receptor-membrane system, which showed a good agreement (r = 0.73) between calculated and experimental relative binding free energies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine A3 Receptor Antagonists* / chemistry
  • Alanine
  • Mutagenesis
  • Purinergic P1 Receptor Antagonists / chemistry
  • Pyridines / chemistry
  • Receptor, Adenosine A1 / genetics
  • Receptor, Adenosine A1 / metabolism
  • Receptor, Adenosine A2A / genetics
  • Receptor, Adenosine A3* / metabolism
  • Structure-Activity Relationship

Substances

  • Adenosine A3 Receptor Antagonists
  • Purinergic P1 Receptor Antagonists
  • Pyridines
  • Receptor, Adenosine A1
  • Receptor, Adenosine A2A
  • Receptor, Adenosine A3
  • Alanine