Conserved class B GPCR activation by a biased intracellular agonist

Nature. 2023 Sep;621(7979):635-641. doi: 10.1038/s41586-023-06467-w. Epub 2023 Jul 31.

Abstract

Class B G-protein-coupled receptors (GPCRs), including glucagon-like peptide 1 receptor (GLP1R) and parathyroid hormone 1 receptor (PTH1R), are important drug targets1-5. Injectable peptide drugs targeting these receptors have been developed, but orally available small-molecule drugs remain under development6,7. Here we report the high-resolution structure of human PTH1R in complex with the stimulatory G protein (Gs) and a small-molecule agonist, PCO371, which reveals an unexpected binding mode of PCO371 at the cytoplasmic interface of PTH1R with Gs. The PCO371-binding site is totally different from all binding sites previously reported for small molecules or peptide ligands in GPCRs. The residues that make up the PCO371-binding pocket are conserved in class B GPCRs, and a single alteration in PTH2R and two residue alterations in GLP1R convert these receptors to respond to PCO371. Functional assays reveal that PCO371 is a G-protein-biased agonist that is defective in promoting PTH1R-mediated arrestin signalling. Together, these results uncover a distinct binding site for designing small-molecule agonists for PTH1R and possibly other members of the class B GPCRs and define a receptor conformation that is specific only for G-protein activation but not arrestin signalling. These insights should facilitate the design of distinct types of class B GPCR small-molecule agonist for various therapeutic indications.

MeSH terms

  • Arrestin / metabolism
  • Binding Sites
  • Drug Design
  • GTP-Binding Protein alpha Subunits, Gs / metabolism
  • Humans
  • Imidazolidines* / pharmacology
  • Ligands
  • Peptides / pharmacology
  • Protein Conformation
  • Receptor, Parathyroid Hormone, Type 1 / agonists
  • Receptor, Parathyroid Hormone, Type 1 / classification
  • Receptor, Parathyroid Hormone, Type 1 / metabolism
  • Receptors, G-Protein-Coupled* / agonists
  • Receptors, G-Protein-Coupled* / classification
  • Receptors, G-Protein-Coupled* / metabolism
  • Signal Transduction / drug effects
  • Spiro Compounds* / pharmacology

Substances

  • Arrestin
  • GTP-Binding Protein alpha Subunits, Gs
  • Imidazolidines
  • Ligands
  • PCO371
  • Peptides
  • PTH1R protein, human
  • Receptor, Parathyroid Hormone, Type 1
  • Receptors, G-Protein-Coupled
  • Spiro Compounds