Conjugation site modulates the in vivo stability and therapeutic activity of antibody-drug conjugates

Nat Biotechnol. 2012 Jan 22;30(2):184-9. doi: 10.1038/nbt.2108.

Abstract

The reactive thiol in cysteine is used for coupling maleimide linkers in the generation of antibody conjugates. To assess the impact of the conjugation site, we engineered cysteines into a therapeutic HER2/neu antibody at three sites differing in solvent accessibility and local charge. The highly solvent-accessible site rapidly lost conjugated thiol-reactive linkers in plasma owing to maleimide exchange with reactive thiols in albumin, free cysteine or glutathione. In contrast, a partially accessible site with a positively charged environment promoted hydrolysis of the succinimide ring in the linker, thereby preventing this exchange reaction. The site with partial solvent-accessibility and neutral charge displayed both properties. In a mouse mammary tumor model, the stability and therapeutic activity of the antibody conjugate were affected positively by succinimide ring hydrolysis and negatively by maleimide exchange with thiol-reactive constituents in plasma. Thus, the chemical and structural dynamics of the conjugation site can influence antibody conjugate performance by modulating the stability of the antibody-linker interface.

MeSH terms

  • Aminobenzoates / chemistry
  • Aminobenzoates / immunology
  • Animals
  • Antibodies / blood*
  • Antibodies / chemistry
  • Antibodies / immunology*
  • Antibodies, Monoclonal, Humanized / chemistry
  • Antibodies, Monoclonal, Humanized / immunology
  • Binding Sites, Antibody / immunology*
  • Cell Line, Tumor
  • Cell Survival
  • Cysteine / chemistry
  • Humans
  • Immunoconjugates / administration & dosage
  • Immunoconjugates / chemistry*
  • Immunoconjugates / immunology*
  • Immunoglobulin G / chemistry*
  • Immunoglobulin G / immunology
  • Macaca fascicularis
  • Maleimides / chemistry
  • Mammary Neoplasms, Experimental / drug therapy
  • Mammary Neoplasms, Experimental / immunology
  • Maytansine / chemistry
  • Maytansine / immunology
  • Mice
  • Mice, Nude
  • Models, Molecular
  • Oligopeptides / chemistry
  • Oligopeptides / immunology
  • Protein Conformation
  • Protein Engineering*
  • Rats
  • Structure-Activity Relationship
  • Sulfhydryl Compounds / chemistry
  • Trastuzumab

Substances

  • Aminobenzoates
  • Antibodies
  • Antibodies, Monoclonal, Humanized
  • Immunoconjugates
  • Immunoglobulin G
  • Maleimides
  • Oligopeptides
  • Sulfhydryl Compounds
  • auristatin
  • Maytansine
  • Cysteine
  • Trastuzumab