Discovery of GNE-502 as an orally bioavailable and potent degrader for estrogen receptor positive breast cancer

Bioorg Med Chem Lett. 2021 Oct 15:50:128335. doi: 10.1016/j.bmcl.2021.128335. Epub 2021 Aug 20.

Abstract

Fulvestrant is an FDA-approved drug with a dual mechanism of action (MOA), acting as a full antagonist and degrader of the estrogen receptor protein. A significant limitation of fulvestrant is the dosing regimen required for efficacy. Due to its high lipophilicity and poor pharmacokinetic profile, fulvestrant needs to be administered through intramuscular injections which leads to injection site soreness. This route of administration also limits the dose and target occupancy in patients. We envisioned a best-in-class molecule that would function with the same dual MOA as fulvestrant, but with improved physicochemical properties and would be orally bioavailable. Herein we report our progress toward that goal, resulting in a new lead GNE-502 which addressed some of the liabilities of our previously reported lead molecule GNE-149.

Keywords: Estrogen receptor; Fulvestrant; GNE-149; GNE-502.

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacokinetics*
  • Antineoplastic Agents / pharmacology*
  • Breast Neoplasms / drug therapy*
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Female
  • Humans
  • MCF-7 Cells
  • Mice
  • Molecular Structure
  • Protein Conformation
  • Receptors, Estrogen / metabolism*
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Receptors, Estrogen