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Updated: Jun 5, 2025

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Discovery of ART0380, a Potent and Selective ATR Kinase Inhibitor Undergoing Phase 2 Clinical Studies for the

Christopher L Carroll1, Michael G Johnson2, Yanbing Ding3

  • 1Institute for Applied Cancer Science (IACS), The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, United States.

Journal of Medicinal Chemistry
|December 4, 2024
PubMed

Insights

ART0380 is a new drug that targets ATR, a key protein involved in cancer DNA repair. This potent and selective inhibitor shows promise in clinical trials for advanced solid tumors.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • Cancer is characterized by DNA replication stress and defects in DNA damage response (DDR) pathways.
  • Ataxia telangiectasia and Rad3-related protein (ATR) is a crucial regulator of DDR and a therapeutic target.
  • Existing ATR inhibitors show promise in clinical studies.

Purpose of the Study:

  • To describe the discovery and characterization of ART0380, a novel ATR inhibitor.
  • To evaluate the in vitro and in vivo pharmacological profile of ART0380.
  • To present the clinical development status of ART0380.

Main Methods:

  • Drug discovery and chemical synthesis of ART0380.
  • In vitro and in vivo preclinical testing to assess potency and selectivity.
  • Pharmacokinetic (PK) studies in humans.

Main Results:

  • ART0380 was discovered as a potent and selective ATR inhibitor.
  • The compound demonstrated a compelling in vitro and in vivo pharmacological profile.
  • ART0380 exhibits a favorable human PK profile suitable for once-daily dosing with dose-proportional exposure and low variability.

Conclusions:

  • ART0380 is a promising ATR inhibitor with a strong preclinical profile.
  • The drug is currently in Phase 2 clinical studies for advanced solid tumors.
  • ART0380's favorable PK supports its clinical development as monotherapy and in combination regimens.

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