Discovery of Tetrahydropyrazolopyrazine Derivatives as Potent and Selective MYT1 Inhibitors for the Treatment of Cancer

J Med Chem. 2024 Jan 11;67(1):420-432. doi: 10.1021/acs.jmedchem.3c01476. Epub 2023 Dec 26.

Abstract

Breast and gynecological cancers are among the leading causes of death in women worldwide, illustrating the urgent need for innovative treatment options. We identified MYT1 as a promising new therapeutic target for breast and gynecological cancer using PandaOmics, an AI-driven target discovery platform. The synthetic lethal relationship of MYT1 in tumor cell lines with CCNE1 amplification enhanced this rationale. Through structure-based drug design, we developed a series of novel, potent, and highly selective inhibitors specifically targeting MYT1. Importantly, our lead compound, featuring a tetrahydropyrazolopyrazine ring, exhibits remarkable selectivity over WEE1, a related kinase associated with bone marrow suppression upon inhibition. Optimization of potency and physical properties resulted in the discovery of compound 21, a novel MYT1 inhibitor, exhibiting optimal pharmacokinetic properties and promising in vivo antitumor efficacy.

MeSH terms

  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Breast
  • Cell Line, Tumor
  • Cell Proliferation
  • DNA-Binding Proteins / metabolism
  • Drug Design
  • Female
  • Humans
  • Neoplasms* / drug therapy
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use
  • Transcription Factors / metabolism

Substances

  • Protein Kinase Inhibitors
  • Antineoplastic Agents
  • MYT1 protein, human
  • DNA-Binding Proteins
  • Transcription Factors