Discovery of a Novel Bifunctional Steroid Analog, YXG-158, as an Androgen Receptor Degrader and CYP17A1 Inhibitor for the Treatment of Enzalutamide-Resistant Prostate Cancer

J Med Chem. 2023 Jul 27;66(14):9972-9991. doi: 10.1021/acs.jmedchem.3c00880. Epub 2023 Jul 17.

Abstract

The androgen/androgen receptor (AR) signaling pathway plays an important role in castration-resistant prostate cancer (CRPC). Bifunctional agents that simultaneously degrade AR and inhibit androgen synthesis are expected to block the androgen/AR signaling pathway more thoroughly, demonstrating the promising therapeutic potential for CRPC, even enzalutamide-resistant CRPC. Herein, a series of steroid analogs were designed, synthesized, and identified as selective AR degraders, among which YXG-158 (23-h) was the most potent antitumor compound with dual functions of AR degradation and CYP17A1 inhibition. In addition, 23-h abrogated the hERG inhibition and exhibited excellent PK profiles. In vivo, 23-h effectively inhibited the growth of hormone-sensitive organs in the Hershberger assay and exhibited robust antitumor efficacy both in enzalutamide-sensitive (LNCaP/AR) and enzalutamide-resistant (C4-2b-ENZ) xenograft models. Thus, 23-h was chosen as a preclinical candidate for the treatment of enzalutamide-resistant prostate cancer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Androgen Antagonists / pharmacology
  • Androgens
  • Cell Line, Tumor
  • Cell Proliferation
  • Drug Resistance, Neoplasm
  • Enzyme Inhibitors / pharmacology
  • Humans
  • Male
  • Nitriles / pharmacology
  • Prostatic Neoplasms, Castration-Resistant* / metabolism
  • Receptors, Androgen* / metabolism
  • Steroid 17-alpha-Hydroxylase
  • Steroids / pharmacology

Substances

  • Receptors, Androgen
  • enzalutamide
  • Androgens
  • Androgen Antagonists
  • Nitriles
  • Enzyme Inhibitors
  • Steroids
  • CYP17A1 protein, human
  • Steroid 17-alpha-Hydroxylase