Discovery of a Highly Potent and Selective BRD9 PROTAC Degrader Based on E3 Binder Investigation for the Treatment of Hematological Tumors

J Med Chem. 2024 Jul 11;67(13):11326-11353. doi: 10.1021/acs.jmedchem.4c00883. Epub 2024 Jun 24.

Abstract

BRD9 is a pivotal epigenetic factor involved in cancers and inflammatory diseases. Still, the limited selectivity and poor phenotypic activity of targeted agents make it an atypically undruggable target. PROTAC offers an alternative strategy for overcoming the issue. In this study, we explored diverse E3 ligase ligands for the contribution of BRD9 PROTAC degradation. Through molecular docking, binding affinity analysis, and structure-activity relationship study, we identified a highly potent PROTAC E5, with excellent BRD9 degradation (DC50 = 16 pM) and antiproliferation in MV4-11 cells (IC50 = 0.27 nM) and OCI-LY10 cells (IC50 = 1.04 nM). E5 can selectively degrade BRD9 and induce cell cycle arrest and apoptosis. Moreover, the therapeutic efficacy of E5 was confirmed in xenograft tumor models, accompanied by further RNA-seq analysis. Therefore, these results may pave the way and provide the reference for the discovery and investigation of highly effective PROTAC degraders.

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacology
  • Apoptosis / drug effects
  • Bromodomain Containing Proteins
  • Cell Line, Tumor
  • Cell Proliferation* / drug effects
  • Drug Discovery
  • Drug Screening Assays, Antitumor
  • Hematologic Neoplasms / drug therapy
  • Hematologic Neoplasms / metabolism
  • Hematologic Neoplasms / pathology
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Mice, Nude
  • Molecular Docking Simulation*
  • Proteolysis / drug effects
  • Structure-Activity Relationship
  • Transcription Factors / antagonists & inhibitors
  • Transcription Factors / metabolism
  • Ubiquitin-Protein Ligases* / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • BRD9 protein, human
  • Ubiquitin-Protein Ligases
  • Transcription Factors
  • Bromodomain Containing Proteins