Discovery and Characterization of BAY 1214784, an Orally Available Spiroindoline Derivative Acting as a Potent and Selective Antagonist of the Human Gonadotropin-Releasing Hormone Receptor as Proven in a First-In-Human Study in Postmenopausal Women

J Med Chem. 2020 Oct 22;63(20):11854-11881. doi: 10.1021/acs.jmedchem.0c01076. Epub 2020 Oct 6.

Abstract

The growth of uterine fibroids is sex hormone-dependent and commonly associated with highly incapacitating symptoms. Most treatment options consist of the control of these hormonal effects, ultimately blocking proliferative estrogen signaling (i.e., oral contraceptives/antagonization of human gonadotropin-releasing hormone receptor [hGnRH-R] activity). Full hGnRH-R blockade, however, results in menopausal symptoms and affects bone mineralization, thus limiting treatment duration or demanding estrogen add-back approaches. To overcome such issues, we aimed to identify novel, small-molecule hGnRH-R antagonists. This led to the discovery of compound BAY 1214784, an orally available, potent, and selective hGnRH-R antagonist. Altering the geminal dimethylindoline core of the initial hit compound to a spiroindoline system significantly improved GnRH-R antagonist potencies across several species, mandatory for a successful compound optimization in vivo. In a first-in-human study in postmenopausal women, once daily treatment with BAY 1214784 effectively lowered plasma luteinizing hormone levels by up to 49%, at the same time being associated with low pharmacokinetic variability and good tolerability.

MeSH terms

  • Administration, Oral
  • Animals
  • Caco-2 Cells
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Female
  • Hepatocytes / chemistry
  • Hepatocytes / metabolism
  • Humans
  • Indoles / administration & dosage
  • Indoles / chemistry
  • Indoles / pharmacology*
  • Microsomes, Liver / chemistry
  • Microsomes, Liver / metabolism
  • Molecular Structure
  • Postmenopause*
  • Rats
  • Rats, Wistar
  • Receptors, LHRH / antagonists & inhibitors*
  • Receptors, LHRH / metabolism
  • Spiro Compounds / administration & dosage
  • Spiro Compounds / chemistry
  • Spiro Compounds / pharmacology*
  • Structure-Activity Relationship

Substances

  • GNRHR protein, human
  • Indoles
  • Receptors, LHRH
  • Spiro Compounds
  • indoline