Neuroactive Steroids. 2. 3α-Hydroxy-3β-methyl-21-(4-cyano-1H-pyrazol-1'-yl)-19-nor-5β-pregnan-20-one (SAGE-217): A Clinical Next Generation Neuroactive Steroid Positive Allosteric Modulator of the (γ-Aminobutyric Acid)A Receptor

J Med Chem. 2017 Sep 28;60(18):7810-7819. doi: 10.1021/acs.jmedchem.7b00846. Epub 2017 Aug 17.

Abstract

Certain classes of neuroactive steroids (NASs) are positive allosteric modulators (PAM) of synaptic and extrasynaptic GABAA receptors. Herein, we report new SAR insights in a series of 5β-nor-19-pregnan-20-one analogues bearing substituted pyrazoles and triazoles at C-21, culminating in the discovery of 3α-hydroxy-3β-methyl-21-(4-cyano-1H-pyrazol-1'-yl)-19-nor-5β-pregnan-20-one (SAGE-217, 3), a potent GABAA receptor modulator at both synaptic and extrasynaptic receptor subtypes, with excellent oral DMPK properties. Compound 3 has completed a phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) clinical trial and is currently being studied in parallel phase 2 clinical trials for the treatment of postpartum depression (PPD), major depressive disorder (MDD), and essential tremor (ET).

MeSH terms

  • Allosteric Regulation / drug effects*
  • Animals
  • Depression, Postpartum / drug therapy
  • Depressive Disorder, Major / drug therapy
  • Female
  • GABA-A Receptor Agonists / chemistry*
  • GABA-A Receptor Agonists / pharmacokinetics
  • GABA-A Receptor Agonists / pharmacology*
  • Mice
  • Pregnanolone / analogs & derivatives*
  • Pregnanolone / chemistry
  • Pregnanolone / pharmacokinetics
  • Pregnanolone / pharmacology
  • Pyrazoles / chemistry
  • Pyrazoles / pharmacokinetics
  • Pyrazoles / pharmacology
  • Rats
  • Receptors, GABA-A / metabolism*

Substances

  • GABA-A Receptor Agonists
  • Pyrazoles
  • Receptors, GABA-A
  • pregnane-20-one
  • Pregnanolone