Synthesis and biological evaluation of novel selective androgen receptor modulators (SARMs) Part III: Discovery of 4-(5-oxopyrrolidine-1-yl)benzonitrile derivative 2f as a clinical candidate

Bioorg Med Chem. 2017 Jul 1;25(13):3330-3349. doi: 10.1016/j.bmc.2017.04.018. Epub 2017 Apr 13.

Abstract

We previously reported that 4-(pyrrolidin-1-yl)benzonitrile derivative 1b was a selective androgen receptor modulator (SARM) that exhibited anabolic effects on organs such as muscles and the central nervous system (CNS), but neutral effects on the prostate. From further modification, we identified that 4-(5-oxopyrrolidine-1-yl)benzonitrile derivative 2a showed strong AR binding affinity with improved metabolic stabilities. Based on these results, we tried to enhance the AR agonistic activities by modifying the substituents of the 5-oxopyrrolidine ring. As a consequence, we found that 4-[(2S,3S)-2-ethyl-3-hydroxy-5-oxopyrrolidin-1-yl]-2-(trifluoromethyl)benzonitrile (2f) had ideal SARM profiles in Hershberger assay and sexual behavior induction assay. Furthermore, 2f showed good pharmacokinetic profiles in rats, dogs, monkeys, excellent nuclear selectivity and acceptable toxicological profiles. We also determined its binding mode by obtaining the co-crystal structures with AR.

Keywords: AR; Androgen receptor; Hershberger assay; Selective androgen receptor modulators (SARMs); Sexual behavior; Testosterone.

MeSH terms

  • Androgens / chemical synthesis
  • Androgens / chemistry
  • Androgens / pharmacology*
  • Animals
  • COS Cells
  • Chlorocebus aethiops
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Haplorhini
  • Humans
  • Male
  • Mice
  • Microsomes, Liver / chemistry
  • Microsomes, Liver / metabolism
  • Molecular Structure
  • Nitriles / chemical synthesis
  • Nitriles / chemistry
  • Nitriles / pharmacology*
  • Rats
  • Rats, Sprague-Dawley
  • Receptors, Androgen / metabolism*
  • Structure-Activity Relationship

Substances

  • Androgens
  • Nitriles
  • Receptors, Androgen
  • benzonitrile