Discovery of GSK3527497: A Candidate for the Inhibition of Transient Receptor Potential Vanilloid-4 (TRPV4)

J Med Chem. 2019 Oct 24;62(20):9270-9280. doi: 10.1021/acs.jmedchem.9b01247. Epub 2019 Oct 9.

Abstract

GSK3527497, a preclinical candidate for the inhibition of TRPV4, was identified starting from the previously reported pyrrolidine sulfonamide TRPV4 inhibitors 1 and 2. Optimization of projected human dose was accomplished by specifically focusing on in vivo pharmacokinetic parameters CLu, Vdssu, and MRT. We highlight the use of conformational changes as a novel approach to modulate Vdssu and present results that suggest that molecular-shape-dependent binding to tissue components governs Vdssu in addition to bulk physicochemical properties. Optimization of CLu within the series was guided by in vitro metabolite identification, and the poor FaSSIF solubility imparted by the crystalline properties of the pyrrolidine diol scaffold was improved by the introduction of a charged moiety to enable excellent exposure from high crystalline doses. GSK3527497 is a preclinical candidate suitable for oral and iv administration that is projected to inhibit TRPV4 effectively in patients from a low daily clinical dose.

MeSH terms

  • Administration, Oral
  • Animals
  • Drug Evaluation, Preclinical
  • Half-Life
  • Humans
  • Inhibitory Concentration 50
  • Pyrrolidines / chemistry*
  • Pyrrolidines / metabolism
  • Rats
  • Rats, Sprague-Dawley
  • Solubility
  • Structure-Activity Relationship
  • Sulfonamides / chemistry*
  • Sulfonamides / metabolism
  • TRPV Cation Channels / antagonists & inhibitors*
  • TRPV Cation Channels / metabolism

Substances

  • Pyrrolidines
  • Sulfonamides
  • TRPV Cation Channels
  • TRPV4 protein, human
  • pyrrolidine