Lead Optimization and Avoidance of Reactive Metabolite Leading to PCO371, a Potent, Selective, and Orally Available Human Parathyroid Hormone Receptor 1 (hPTHR1) Agonist

J Med Chem. 2020 May 28;63(10):5089-5099. doi: 10.1021/acs.jmedchem.9b01743. Epub 2020 Feb 17.

Abstract

We have previously shown that the oral administration of the small molecule hPTHR1 agonist PCO371 and its lead compound, 1 (CH5447240) results in PTH-like calcemic and hypophostemic activity in thyroparathyroidectomized rats. However, 1 was converted to a reactive metabolite in a human liver microsome assay. In this article, we report on the modification path that led to an enhancement of PTHR1 agonistic activity and reduction in the formation of a reactive metabolite to result in a potent, selective, and orally active PTHR1 agonist 1-(3,5-dimethyl-4-(2-((4-oxo-2-(4-(trifluoromethoxy)phenyl)-1,3,8-triazaspiro[4.5]dec-1-en-8-yl)sulfonyl)ethyl)phenyl)-5,5-dimethylimidazolidine-2,4-dione (PCO371, 16c). This compound is currently being evaluated in a phase 1 clinical study for the treatment of hypoparathyroidism.

MeSH terms

  • Administration, Oral
  • Animals
  • Female
  • Humans
  • Hypoparathyroidism / drug therapy
  • Hypoparathyroidism / metabolism
  • Imidazolidines / administration & dosage*
  • Imidazolidines / chemistry
  • Imidazolidines / metabolism*
  • LLC-PK1 Cells
  • Rats
  • Rats, Sprague-Dawley
  • Receptor, Parathyroid Hormone, Type 1 / agonists*
  • Receptor, Parathyroid Hormone, Type 1 / metabolism*
  • Spiro Compounds / administration & dosage*
  • Spiro Compounds / chemistry
  • Spiro Compounds / metabolism*
  • Swine

Substances

  • Imidazolidines
  • PCO371
  • PTH1R protein, human
  • Receptor, Parathyroid Hormone, Type 1
  • Spiro Compounds