Discovery of a Myeloid Cell Leukemia 1 (Mcl-1) Inhibitor That Demonstrates Potent In Vivo Activities in Mouse Models of Hematological and Solid Tumors

J Med Chem. 2024 Aug 22;67(16):14370-14393. doi: 10.1021/acs.jmedchem.4c01188. Epub 2024 Aug 5.

Abstract

Myeloid cell leukemia 1 (Mcl-1) is a key regulator of the intrinsic apoptosis pathway. Overexpression of Mcl-1 is correlated with high tumor grade, poor survival, and both intrinsic and acquired resistance to cancer therapies. Herein, we disclose the structure-guided design of a small molecule Mcl-1 inhibitor, compound 26, that binds to Mcl-1 with subnanomolar affinity, inhibits growth in cell culture assays, and possesses low clearance in mouse and dog pharmacokinetic (PK) experiments. Evaluation of 26 as a single agent in Mcl-1 sensitive hematological and solid tumor xenograft models resulted in regressions. Co-treatment of Mcl-1-sensitive and Mcl-1 insensitive lung cancer derived xenografts with 26 and docetaxel or topotecan, respectively, resulted in an enhanced tumor response. These findings support the premise that pro-apoptotic priming of tumor cells by other therapies in combination with Mcl-1 inhibition may significantly expand the subset of cancers in which Mcl-1 inhibitors may prove beneficial.

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacokinetics
  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Cell Line, Tumor
  • Docetaxel / chemistry
  • Docetaxel / pharmacokinetics
  • Docetaxel / pharmacology
  • Docetaxel / therapeutic use
  • Dogs
  • Drug Discovery
  • Female
  • Humans
  • Mice
  • Myeloid Cell Leukemia Sequence 1 Protein* / antagonists & inhibitors
  • Myeloid Cell Leukemia Sequence 1 Protein* / metabolism
  • Structure-Activity Relationship
  • Taxoids / chemistry
  • Taxoids / pharmacokinetics
  • Taxoids / pharmacology
  • Taxoids / therapeutic use
  • Xenograft Model Antitumor Assays*

Substances

  • Myeloid Cell Leukemia Sequence 1 Protein
  • Antineoplastic Agents
  • MCL1 protein, human
  • Taxoids
  • Docetaxel