Discovery of a Series of Potent, Selective, and Orally Bioavailable Nucleoside Inhibitors of CD73 That Demonstrates In Vivo Antitumor Activity

J Med Chem. 2023 Jan 12;66(1):345-370. doi: 10.1021/acs.jmedchem.2c01287. Epub 2022 Dec 18.

Abstract

CD73 (ecto-5'-nucleotidase) has emerged as an attractive target for cancer immunotherapy of many cancers. CD73 catalyzes the hydrolysis of adenosine monophosphate (AMP) into highly immunosuppressive adenosine that plays a critical role in tumor progression. Herein, we report our efforts in developing orally bioavailable and highly potent small-molecule CD73 inhibitors from the reported hit molecule 2 to lead molecule 20 and then finally to compound 49. Compound 49 was able to reverse AMP-mediated suppression of CD8+ T cells and completely inhibited CD73 activity in serum samples from various cancer patients. In preclinical in vivo studies, orally administered 49 showed a robust dose-dependent pharmacokinetic/pharmacodynamic (PK/PD) relationship that correlated with efficacy. Compound 49 also demonstrated the expected immune-mediated antitumor mechanism of action and was efficacious upon oral administration not only as a single agent but also in combination with either chemotherapeutics or checkpoint inhibitor in the mouse tumor model.

MeSH terms

  • 5'-Nucleotidase
  • Adenosine Monophosphate
  • Animals
  • CD8-Positive T-Lymphocytes*
  • Disease Models, Animal
  • Mice
  • Neoplasms* / drug therapy
  • Nucleosides

Substances

  • Nucleosides
  • 5'-Nucleotidase
  • Adenosine Monophosphate