From High-Throughput Screening to Target Validation: Benzo[ d]isothiazoles as Potent and Selective Agonists of Human Transient Receptor Potential Cation Channel Subfamily M Member 5 Possessing In Vivo Gastrointestinal Prokinetic Activity in Rodents

J Med Chem. 2021 May 13;64(9):5931-5955. doi: 10.1021/acs.jmedchem.1c00065. Epub 2021 Apr 23.

Abstract

Transient receptor potential cation channel subfamily M member 5 (TRPM5) is a nonselective monovalent cation channel activated by intracellular Ca2+ increase. Within the gastrointestinal system, TRPM5 is expressed in the stoma, small intestine, and colon. In the search for a selective agonist of TRPM5 possessing in vivo gastrointestinal prokinetic activity, a high-throughput screening was performed and compound 1 was identified as a promising hit. Hit validation and hit to lead activities led to the discovery of a series of benzo[d]isothiazole derivatives. Among these, compounds 61 and 64 showed nanomolar activity and excellent selectivity (>100-fold) versus related cation channels. The in vivo drug metabolism and pharmacokinetic profile of compound 64 was found to be ideal for a compound acting locally at the intestinal level, with minimal absorption into systemic circulation. Compound 64 was tested in vivo in a mouse motility assay at 100 mg/kg, and demonstrated increased prokinetic activity.

MeSH terms

  • Animals
  • Benzothiazoles / chemistry*
  • Benzothiazoles / metabolism
  • Benzothiazoles / pharmacokinetics
  • Benzothiazoles / pharmacology*
  • Drug Design
  • Gastrointestinal Tract / drug effects
  • Gastrointestinal Tract / metabolism*
  • High-Throughput Screening Assays*
  • Humans
  • Male
  • Mice
  • Molecular Targeted Therapy
  • Reproducibility of Results
  • TRPM Cation Channels / agonists*
  • Tissue Distribution

Substances

  • Benzothiazoles
  • TRPM Cation Channels
  • TRPM5 protein, human
  • benzothiazole