Engineered antibody-drug conjugates with defined sites and stoichiometries of drug attachment

Protein Eng Des Sel. 2006 Jul;19(7):299-307. doi: 10.1093/protein/gzl013. Epub 2006 Apr 27.

Abstract

The chimeric anti-CD30 IgG1, cAC10, conjugated to eight equivalents of monomethyl auristatin E (MMAE) was previously shown to have potent antitumor activity against CD30-expressing tumors xenografts in mice. Moreover, the therapeutic index was increased by lowering the stoichiometry from 8 drugs/antibody down to 2 or 4. Limitations of such 'partially-loaded' conjugates are low yield (10-30%) as they are purified from mixtures with variable stoichiometry (0-8 drugs/antibody), and heterogeneity as the 2 or 4 drugs are distributed over eight possible cysteine conjugation sites. Here, the solvent-accessible cysteines that form the interchain disulfide bonds in cAC10 were replaced with serine, to reduce the eight potential conjugation sites down to 4 or 2. These Cys-->Ser antibody variants were conjugated to MMAE in near quantitative yield (89-96%) with defined stoichiometries (2 or 4 drugs/antibody) and sites of drug attachment. The engineered antibody-drug conjugates have comparable antigen-binding affinities and in vitro cytotoxic activities with corresponding purified parental antibody-drug conjugates. Additionally, the engineered and parental antibody-drug conjugates have similar in vivo properties including antitumor activity, pharmacokinetics and maximum tolerated dose. Our strategy for generating antibody-drug conjugates with defined sites and stoichiometries of drug loading is potentially broadly applicable to other antibodies as it involves engineering of constant domains.

MeSH terms

  • Animals
  • Antibodies, Monoclonal / chemistry
  • Antibodies, Monoclonal / immunology*
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacokinetics*
  • Base Sequence
  • Binding Sites
  • Cysteine / chemistry
  • Disulfides / chemistry
  • Disulfides / metabolism
  • Immunoconjugates / immunology
  • Immunoconjugates / pharmacokinetics*
  • Ki-1 Antigen / immunology
  • Ligands
  • Maximum Tolerated Dose
  • Mice
  • Oligopeptides / chemistry
  • Oligopeptides / pharmacokinetics*
  • Protein Engineering
  • Serine / chemistry
  • Solvents / chemistry
  • Transplantation, Heterologous
  • Tumor Cells, Cultured

Substances

  • Antibodies, Monoclonal
  • Antineoplastic Agents
  • Disulfides
  • Immunoconjugates
  • Ki-1 Antigen
  • Ligands
  • Oligopeptides
  • Solvents
  • Serine
  • Cysteine
  • monomethyl auristatin E