Identification of Novel Series of Potent and Selective Relaxin Family Peptide Receptor 1 (RXFP1) Agonists

J Med Chem. 2024 Mar 28;67(6):4442-4462. doi: 10.1021/acs.jmedchem.3c02183. Epub 2024 Mar 19.

Abstract

Relaxin H2 is a clinically relevant peptide agonist for relaxin family peptide receptor 1 (RXFP1), but a combination of this hormone's short plasma half-life and the need for injectable delivery limits its therapeutic potential. We sought to overcome these limitations through the development of a potent small molecule (SM) RXFP1 agonist. Although two large SM HTS campaigns failed in identifying suitable hit series, we uncovered novel chemical space starting from the only known SM RXFP1 agonist series, represented by ML290. Following a design-make-test-analyze strategy based on improving early dose to man ranking, we discovered compound 42 (AZ7976), a highly selective RXFP1 agonist with sub-nanomolar potency. We used AZ7976, its 10 000-fold less potent enantiomer 43 and recombinant relaxin H2 to evaluate in vivo pharmacology and demonstrate that AZ7976-mediated heart rate increase in rats was a result of RXFP1 agonism. As a result, AZ7976 was selected as lead for continued optimization.

MeSH terms

  • Animals
  • Humans
  • Male
  • Rats
  • Receptors, G-Protein-Coupled / agonists
  • Receptors, Peptide / agonists
  • Relaxin* / pharmacology

Substances

  • Relaxin
  • Receptors, G-Protein-Coupled
  • Receptors, Peptide
  • RXFP1 protein, human