Neuroactive Steroid N-Methyl-d-aspartate Receptor Positive Allosteric Modulators: Synthesis, SAR, and Pharmacological Activity

J Med Chem. 2019 Aug 22;62(16):7526-7542. doi: 10.1021/acs.jmedchem.9b00591. Epub 2019 Aug 13.

Abstract

Neuroactive steroids (NASs) play a pivotal role in maintaining homeostasis is the CNS. We have discovered that one NAS in particular, 24(S)-hydroxycholesterol (24(S)-HC), is a positive allosteric modulator (PAM) of NMDA receptors. Using 24(S)-HC as a chemical starting point, we have identified other NASs that have good in vitro potency and efficacy. Herein, we describe the structure activity relationship and pharmacokinetic optimization of this series that ultimately led to SGE-301 (42). We demonstrate that SGE-301 enhances long-term potentiation (LTP) in rat hippocampal slices and, in a dose-dependent manner, improves cognition in a rat social recognition study.

MeSH terms

  • Age Factors
  • Allosteric Regulation*
  • Animals
  • Cognition / drug effects
  • Hippocampus / drug effects
  • Hippocampus / physiology
  • Humans
  • Long-Term Potentiation / drug effects
  • Male
  • Methylation
  • Molecular Structure
  • Neurosteroids / chemistry
  • Neurosteroids / pharmacokinetics
  • Neurosteroids / pharmacology*
  • Rats, Wistar
  • Receptors, N-Methyl-D-Aspartate / metabolism*
  • Structure-Activity Relationship

Substances

  • Neurosteroids
  • Receptors, N-Methyl-D-Aspartate