Structure-based design of non-hypertrophic apelin receptor modulator

Cell. 2024 Mar 14;187(6):1460-1475.e20. doi: 10.1016/j.cell.2024.02.004. Epub 2024 Feb 29.

Abstract

Apelin is a key hormone in cardiovascular homeostasis that activates the apelin receptor (APLNR), which is regarded as a promising therapeutic target for cardiovascular disease. However, adverse effects through the β-arrestin pathway limit its pharmacological use. Here, we report cryoelectron microscopy (cryo-EM) structures of APLNR-Gi1 complexes bound to three agonists with divergent signaling profiles. Combined with functional assays, we have identified "twin hotspots" in APLNR as key determinants for signaling bias, guiding the rational design of two exclusive G-protein-biased agonists WN353 and WN561. Cryo-EM structures of WN353- and WN561-stimulated APLNR-G protein complexes further confirm that the designed ligands adopt the desired poses. Pathophysiological experiments have provided evidence that WN561 demonstrates superior therapeutic effects against cardiac hypertrophy and reduced adverse effects compared with the established APLNR agonists. In summary, our designed APLNR modulator may facilitate the development of next-generation cardiovascular medications.

Keywords: apelin receptor; biased signaling transduction; cryo-EM structure; drug design; myocardial hypertrophy; side effects.

MeSH terms

  • Apelin Receptors* / agonists
  • Apelin Receptors* / chemistry
  • Apelin Receptors* / ultrastructure
  • Cardiovascular Agents* / chemistry
  • Cryoelectron Microscopy
  • Drug Design*
  • GTP-Binding Proteins / metabolism
  • Humans
  • Receptors, G-Protein-Coupled / metabolism
  • Signal Transduction

Substances

  • Apelin Receptors
  • GTP-Binding Proteins
  • Receptors, G-Protein-Coupled
  • Cardiovascular Agents