Oxetane Promise Delivered: Discovery of Long-Acting IDO1 Inhibitors Suitable for Q3W Oral or Parenteral Dosing

J Med Chem. 2022 Apr 28;65(8):6001-6016. doi: 10.1021/acs.jmedchem.1c01670. Epub 2022 Mar 3.

Abstract

3,3-Disubstituted oxetanes have been utilized as bioisosteres for gem-dimethyl and cyclobutane functionalities. We report the discovery of a novel class of oxetane indole-amine 2,3-dioxygenase (IDO1) inhibitors suitable for Q3W (once every 3 weeks) oral and parenteral dosing. A diamide class of IDO inhibitors was discovered through an automated ligand identification system (ALIS). Installation of an oxetane and fluorophenyl dramatically improved the potency. Identification of a biaryl moiety as an unconventional amide isostere addressed the metabolic liability of amide hydrolysis. Metabolism identification (Met-ID)-guided target design and the introduction of polarity resulted in the discovery of potent IDO inhibitors with excellent pharmacokinetic (PK) profiles in multiple species. To enable rapid synthesis of the key oxetane intermediate, a novel oxetane ring cyclization was also developed, as well as optimization of a literature route on kg scale. These IDO inhibitors may enable unambiguous proof-of-concept testing for the IDO1 inhibition mechanism for oncology.

MeSH terms

  • Amides
  • Cyclization
  • Enzyme Inhibitors* / pharmacology
  • Ethers, Cyclic*
  • Indoleamine-Pyrrole 2,3,-Dioxygenase / metabolism

Substances

  • Amides
  • Enzyme Inhibitors
  • Ethers, Cyclic
  • Indoleamine-Pyrrole 2,3,-Dioxygenase
  • oxetane