Targeted Delivery of LXR Agonist Using a Site-Specific Antibody-Drug Conjugate

Bioconjug Chem. 2015 Nov 18;26(11):2216-22. doi: 10.1021/acs.bioconjchem.5b00203. Epub 2015 May 20.

Abstract

Liver X receptor (LXR) agonists have been explored as potential treatments for atherosclerosis and other diseases based on their ability to induce reverse cholesterol transport and suppress inflammation. However, this therapeutic potential has been hindered by on-target adverse effects in the liver mediated by excessive lipogenesis. Herein, we report a novel site-specific antibody-drug conjugate (ADC) that selectively delivers a LXR agonist to monocytes/macrophages while sparing hepatocytes. The unnatural amino acid para-acetylphenylalanine (pAcF) was site-specifically incorporated into anti-CD11a IgG, which binds the α-chain component of the lymphocyte function-associated antigen 1 (LFA-1) expressed on nearly all monocytes and macrophages. An aminooxy-modified LXR agonist was conjugated to anti-CD11a IgG through a stable, cathepsin B cleavable oxime linkage to afford a chemically defined ADC. The anti-CD11a IgG-LXR agonist ADC induced LXR activation specifically in human THP-1 monocyte/macrophage cells in vitro (EC50-27 nM), but had no significant effect in hepatocytes, indicating that payload delivery is CD11a-mediated. Moreover, the ADC exhibited higher-fold activation compared to a conventional synthetic LXR agonist T0901317 (Tularik) (3-fold). This novel ADC represents a fundamentally different strategy that uses tissue targeting to overcome the limitations of LXR agonists for potential use in treating atherosclerosis.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Benzoates / administration & dosage*
  • Benzoates / immunology
  • Benzoates / pharmacokinetics
  • Benzylamines / administration & dosage*
  • Benzylamines / immunology
  • Benzylamines / pharmacokinetics
  • CD11a Antigen / immunology*
  • Cell Line
  • Drug Delivery Systems*
  • Hep G2 Cells
  • Hepatocytes / drug effects
  • Hepatocytes / immunology
  • Humans
  • Hydrocarbons, Fluorinated / administration & dosage*
  • Hydrocarbons, Fluorinated / immunology
  • Hydrocarbons, Fluorinated / pharmacokinetics
  • Immunoconjugates / administration & dosage*
  • Immunoconjugates / immunology
  • Immunoconjugates / pharmacokinetics
  • Immunoglobulin G / immunology
  • Liver X Receptors
  • Macrophages / drug effects
  • Macrophages / immunology
  • Monocytes / drug effects
  • Monocytes / immunology
  • Orphan Nuclear Receptors / agonists*
  • Sulfonamides / administration & dosage*
  • Sulfonamides / immunology
  • Sulfonamides / pharmacokinetics

Substances

  • Benzoates
  • Benzylamines
  • CD11a Antigen
  • GW 3965
  • Hydrocarbons, Fluorinated
  • Immunoconjugates
  • Immunoglobulin G
  • Liver X Receptors
  • Orphan Nuclear Receptors
  • Sulfonamides
  • T0901317