Bispecific Estrogen Receptor α Degraders Incorporating Novel Binders Identified Using DNA-Encoded Chemical Library Screening

J Med Chem. 2021 Apr 22;64(8):5049-5066. doi: 10.1021/acs.jmedchem.1c00127. Epub 2021 Apr 12.

Abstract

Bispecific degraders (PROTACs) of ERα are expected to be advantageous over current inhibitors of ERα signaling (aromatase inhibitors/SERMs/SERDs) used to treat ER+ breast cancer. Information from DNA-encoded chemical library (DECL) screening provides a method to identify novel PROTAC binding features as the linker positioning, and binding elements are determined directly from the screen. After screening ∼120 billion DNA-encoded molecules with ERα WT and 3 gain-of-function (GOF) mutants, with and without estradiol to identify features that enrich ERα competitively, the off-DNA synthesized small molecule exemplar 7 exhibited nanomolar ERα binding, antagonism, and degradation. Click chemistry synthesis on an alkyne E3 ligase engagers panel and an azide variant of 7 rapidly generated bispecific nanomolar degraders of ERα, with PROTACs 18 and 21 inhibiting ER+ MCF7 tumor growth in a mouse xenograft model of breast cancer. This study validates this approach toward identifying novel bispecific degrader leads from DECL screening with minimal optimization.

MeSH terms

  • Animals
  • Breast Neoplasms / drug therapy
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Click Chemistry
  • DNA / chemistry*
  • DNA / metabolism
  • Estrogen Antagonists / chemistry
  • Estrogen Antagonists / metabolism
  • Estrogen Antagonists / pharmacology
  • Estrogen Antagonists / therapeutic use
  • Estrogen Receptor alpha / chemistry
  • Estrogen Receptor alpha / genetics
  • Estrogen Receptor alpha / metabolism*
  • Female
  • Half-Life
  • Humans
  • Indoles / chemistry
  • Indoles / metabolism
  • Kinetics
  • Mice
  • Small Molecule Libraries / chemistry*
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology
  • Small Molecule Libraries / therapeutic use
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Estrogen Antagonists
  • Estrogen Receptor alpha
  • Indoles
  • Small Molecule Libraries
  • DNA
  • bazedoxifene