Discovery of ARD-69 as a Highly Potent Proteolysis Targeting Chimera (PROTAC) Degrader of Androgen Receptor (AR) for the Treatment of Prostate Cancer

J Med Chem. 2019 Jan 24;62(2):941-964. doi: 10.1021/acs.jmedchem.8b01631. Epub 2019 Jan 10.

Abstract

We report herein the discovery of highly potent PROTAC degraders of androgen receptor (AR), as exemplified by compound 34 (ARD-69). ARD-69 induces degradation of AR protein in AR-positive prostate cancer cell lines in a dose- and time-dependent manner. ARD-69 achieves DC50 values of 0.86, 0.76, and 10.4 nM in LNCaP, VCaP, and 22Rv1 AR+ prostate cancer cell lines, respectively. ARD-69 is capable of reducing the AR protein level by >95% in these prostate cancer cell lines and effectively suppressing AR-regulated gene expression. ARD-69 potently inhibits cell growth in these AR-positive prostate cancer cell lines and is >100 times more potent than AR antagonists. A single dose of ARD-69 effectively reduces the level of AR protein in xenograft tumor tissue in mice. Further optimization of ARD-69 may ultimately lead to a new therapy for AR+, castration-resistant prostate cancer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Androgen Receptor Antagonists / chemistry*
  • Androgen Receptor Antagonists / pharmacology
  • Animals
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Design
  • Drug Evaluation, Preclinical
  • Gene Expression / drug effects
  • Humans
  • Ligands
  • Male
  • Mice
  • Mice, SCID
  • Prostatic Neoplasms / drug therapy
  • Prostatic Neoplasms / metabolism
  • Prostatic Neoplasms / pathology
  • Protein Binding
  • Proteolysis* / drug effects
  • Receptors, Androgen / genetics
  • Receptors, Androgen / metabolism*
  • Structure-Activity Relationship
  • Transplantation, Heterologous
  • Von Hippel-Lindau Tumor Suppressor Protein / chemistry
  • Von Hippel-Lindau Tumor Suppressor Protein / metabolism

Substances

  • Androgen Receptor Antagonists
  • Ligands
  • Receptors, Androgen
  • Von Hippel-Lindau Tumor Suppressor Protein
  • VHL protein, human