Discovery of a Potent and Orally Bioavailable Zwitterionic Series of Selective Estrogen Receptor Degrader-Antagonists

J Med Chem. 2023 Feb 23;66(4):2918-2945. doi: 10.1021/acs.jmedchem.2c01964. Epub 2023 Feb 1.

Abstract

Herein, we report the optimization of a meta-substituted series of selective estrogen receptor degrader (SERD) antagonists for the treatment of ER+ breast cancer. Structure-based design together with the use of modeling and NMR to favor the bioactive conformation led to a highly potent series of basic SERDs with promising physicochemical properties. Issues with hERG activity resulted in a strategy of zwitterion formation and ultimately in the identification of 38. This compound was shown to be a highly potent SERD capable of effectively degrading ERα in both MCF-7 and CAMA-1 cell lines. The low lipophilicity and zwitterionic nature led to a SERD with a clean secondary pharmacology profile and no hERG activity. Favorable physicochemical properties resulted in good oral bioavailability in preclinical species and potent in vivo activity in a mouse xenograft model.

MeSH terms

  • Animals
  • Breast Neoplasms* / drug therapy
  • Cell Line
  • Estrogen Antagonists / therapeutic use
  • Estrogen Receptor alpha / metabolism
  • Female
  • Humans
  • Mice
  • Receptors, Estrogen* / metabolism
  • Selective Estrogen Receptor Modulators / pharmacology

Substances

  • Receptors, Estrogen
  • Selective Estrogen Receptor Modulators
  • Estrogen Antagonists
  • Estrogen Receptor alpha