Backbone Modification Provides a Long-Acting Inverse Agonist of Pathogenic, Constitutively Active PTH1R Variants

J Am Chem Soc. 2024 Mar 13;146(10):6522-6529. doi: 10.1021/jacs.3c09694. Epub 2024 Feb 28.

Abstract

Parathyroid hormone 1 receptor (PTH1R) plays a key role in mediating calcium homeostasis and bone development, and aberrant PTH1R activity underlies several human diseases. Peptidic PTH1R antagonists and inverse agonists have therapeutic potential in treating these diseases, but their poor pharmacokinetics and pharmacodynamics undermine their in vivo efficacy. Herein, we report the use of a backbone-modification strategy to design a peptidic PTH1R inhibitor that displays prolonged activity as an antagonist of wild-type PTH1R and an inverse agonist of the constitutively active PTH1R-H223R mutant both in vitro and in vivo. This peptide may be of interest for the future development of therapeutic agents that ameliorate PTH1R malfunction.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Drug Inverse Agonism*
  • Humans
  • Parathyroid Hormone / pharmacology
  • Peptides
  • Receptor, Parathyroid Hormone, Type 1*

Substances

  • Receptor, Parathyroid Hormone, Type 1
  • Peptides
  • Parathyroid Hormone
  • PTH1R protein, human