Long-Acting Tumor-Activated Prodrug of a TGFβR Inhibitor

J Med Chem. 2021 Nov 11;64(21):15787-15798. doi: 10.1021/acs.jmedchem.0c02043. Epub 2021 Oct 27.

Abstract

Inhibition of TGFβ signaling in concert with a checkpoint blockade has been shown to provide improved and durable antitumor immune response in mouse models. However, on-target adverse cardiovascular effects have limited the clinical use of TGFβ receptor (TGFβR) inhibitors in cancer therapy. To restrict the activity of TGFβR inhibitors to tumor tissues and thereby widen the therapeutic index, a series of tumor-activated prodrugs of a selective small molecule TGFβR1 inhibitor 1 were prepared by appending 1 to a serine protease substrate and a half-life extension fatty acid carbon chain. The prodrugs were shown to be selectively metabolized in tumor tissues relative to the heart and blood and demonstrated a prolonged favorable increase in the tumor-to-heart ratio of the active drug in tissue distribution studies. Once-weekly administration of the most tissue-selective compound 10 provided anti-tumor efficacy comparable to the parent compound and reduced systemic exposure of the active drug.

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / therapeutic use*
  • Area Under Curve
  • Drug Stability
  • Female
  • Half-Life
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred C57BL
  • Molecular Structure
  • Myocardium / metabolism
  • Neoplasms / drug therapy*
  • Neoplasms / metabolism
  • Prodrugs / chemistry
  • Prodrugs / pharmacokinetics
  • Prodrugs / therapeutic use*
  • Receptor, Transforming Growth Factor-beta Type I / antagonists & inhibitors*
  • Small Molecule Libraries / pharmacology
  • Tissue Distribution
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Prodrugs
  • Small Molecule Libraries
  • Receptor, Transforming Growth Factor-beta Type I
  • TGFBR1 protein, human