Discovery of CZL-046 with an (S)-3-Fluoropyrrolidin-2-one Scaffold as a p300 Bromodomain Inhibitor for the Treatment of Multiple Myeloma

J Med Chem. 2024 Oct 24;67(20):18606-18628. doi: 10.1021/acs.jmedchem.4c01984. Epub 2024 Oct 2.

Abstract

E1A binding protein (p300) is a promising therapeutic target for the treatment of cancer. Herein, we report the discovery of a series of novel inhibitors with an (S)-3-fluoropyrrolidin-2-one scaffold targeting p300 bromodomain. The best compound 29 (CZL-046) shows potent inhibitory activity of p300 bromodomain (IC50 = 3.3 nM) and antiproliferative activity in the multiple myeloma (MM) cell line (OPM-2 IC50 = 51.5 nM). 29 suppressed the mRNA levels of c-Myc and IRF4 and downregulated the expression of c-Myc and H3K27Ac. Compared to the lead compound 5, 29 exhibits significantly improved in vitro and in vivo metabolic properties. Oral administration of 29 with 30 mg/kg achieved a TGI value of 44% in the OPM-2 xenograft model, accompanied by good tolerability. The cocrystal structure of CREB binding protein bromodomain with 29 provides an insight into the precise binding mode. The results demonstrate that 29 is a promising p300 bromodomain inhibitor for the treatment of MM.

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Discovery
  • E1A-Associated p300 Protein* / antagonists & inhibitors
  • E1A-Associated p300 Protein* / metabolism
  • Humans
  • Male
  • Mice
  • Multiple Myeloma* / drug therapy
  • Multiple Myeloma* / metabolism
  • Multiple Myeloma* / pathology
  • Pyrrolidinones / chemical synthesis
  • Pyrrolidinones / chemistry
  • Pyrrolidinones / pharmacology
  • Pyrrolidinones / therapeutic use
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • E1A-Associated p300 Protein
  • Antineoplastic Agents
  • EP300 protein, human
  • Pyrrolidinones