Design of a Potent TLX Agonist by Rational Fragment Fusion

J Med Chem. 2022 Feb 10;65(3):2288-2296. doi: 10.1021/acs.jmedchem.1c01757. Epub 2022 Jan 6.

Abstract

As a master regulator of neurogenesis, the orphan nuclear receptor tailless homologue (TLX, NR2E1) maintains neuronal stem cell homeostasis by acting as a transcriptional repressor of tumor suppressor genes. It is hence considered as an appealing target for the treatment of neurodegenerative diseases, but a lack of potent TLX modulators as tools to probe pharmacological TLX control hinders further validation of its promising potential. Here, we report the development of a potent TLX agonist based on fragment screening, pharmacophore modeling, and fragment fusion. Pharmacophore similarity of a fragment screening hit and the TLX ligand ccrp2 provided a rational basis for fragment linkage, which resulted in several TLX activator scaffolds. Among them, the fused compound 10 evolved as a valuable TLX agonist tool with submicromolar potency and high selectivity over related nuclear receptors, rendering it suitable for functional studies on TLX.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Survival / drug effects
  • Drug Design*
  • Drug Stability
  • HEK293 Cells
  • Humans
  • Ligands
  • Microsomes, Liver / metabolism
  • Orphan Nuclear Receptors / agonists*
  • Orphan Nuclear Receptors / metabolism
  • Piperazine / chemistry
  • Piperazine / metabolism
  • Piperazine / pharmacology
  • Protein Binding
  • Rats
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship

Substances

  • Ligands
  • NR2E1 protein, human
  • Orphan Nuclear Receptors
  • Piperazine