Discovery of Novel, Orally Bioavailable Pyrimidine Ether-Based Inhibitors of ELOVL1

J Med Chem. 2021 Dec 23;64(24):17777-17794. doi: 10.1021/acs.jmedchem.1c00948. Epub 2021 Dec 6.

Abstract

In our efforts to identify novel small molecule inhibitors for the treatment of adrenoleukodystrophy (ALD), we conducted a high-throughput radiometric screen for inhibitors of elongation of very long chain fatty acid 1 (ELOVL1) enzyme. We developed a series of highly potent, central nervous system (CNS)-penetrant pyrimidine ether-based compounds with favorable pharmacokinetics culminating in compound 22. Compound 22 is a selective inhibitor of ELOVL1, reducing C26:0 VLCFA synthesis in ALD patient fibroblasts and lymphocytes in vitro. Compound 22 reduced C26:0 lysophosphatidyl choline (LPC), a subtype of VLCFA, in the blood of ATP binding cassette transporter D1 (ABCD1) KO mice, a murine model of ALD to near wild-type levels. Compound 22 is a low-molecular-weight, potent ELOVL1 inhibitor that may serve as a useful tool for exploring therapeutic approaches to the treatment of ALD.

MeSH terms

  • Administration, Oral
  • Adrenoleukodystrophy / drug therapy
  • Animals
  • Biological Availability
  • Dogs
  • Drug Discovery*
  • Enzyme Inhibitors / administration & dosage
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology*
  • Ethers / chemistry
  • Fatty Acid Elongases / antagonists & inhibitors*
  • HEK293 Cells
  • Humans
  • Macaca fascicularis
  • Mice
  • Pyrimidines / administration & dosage
  • Pyrimidines / pharmacokinetics
  • Pyrimidines / pharmacology*
  • Rats

Substances

  • ELOVL1 protein, human
  • Enzyme Inhibitors
  • Ethers
  • Pyrimidines
  • Fatty Acid Elongases