Tumor-specific activation of an EGFR-targeting probody enhances therapeutic index

Sci Transl Med. 2013 Oct 16;5(207):207ra144. doi: 10.1126/scitranslmed.3006682.

Abstract

Target-mediated toxicity constitutes a major limitation for the development of therapeutic antibodies. To redirect the activity of antibodies recognizing widely distributed targets to the site of disease, we have applied a prodrug strategy to create an epidermal growth factor receptor (EGFR)-directed Probody therapeutic-an antibody that remains masked against antigen binding until activated locally by proteases commonly active in the tumor microenvironment. In vitro, the masked Probody showed diminished antigen binding and cell-based activities, but when activated by appropriate proteases, it regained full activity compared to the parental anti-EGFR antibody cetuximab. In vivo, the Probody was largely inert in the systemic circulation of mice, but was activated within tumor tissue and showed antitumor efficacy that was similar to that of cetuximab. The Probody demonstrated markedly improved safety and increased half-life in nonhuman primates, enabling it to be dosed safely at much higher levels than cetuximab. In addition, we found that both Probody-responsive xenograft tumors and primary tumor samples from patients were capable of activating the Probody ex vivo. Probodies may therefore improve the safety profile of therapeutic antibodies without compromising efficacy of the parental antibody and may enable the wider use of empowered antibody formats such as antibody-drug conjugates and bispecifics.

Publication types

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

MeSH terms

  • Animals
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Antibodies, Neoplasm / pharmacology
  • Antibodies, Neoplasm / therapeutic use*
  • Cell Proliferation / drug effects
  • Cetuximab
  • ErbB Receptors / metabolism*
  • Humans
  • Immunohistochemistry
  • Macaca fascicularis
  • Mice
  • Mice, Nude
  • Neoplasms / drug therapy*
  • Neoplasms / pathology*
  • Prodrugs / therapeutic use*
  • Prodrugs / toxicity
  • Skin / drug effects
  • Skin / pathology
  • Xenograft Model Antitumor Assays

Substances

  • Antibodies, Monoclonal, Humanized
  • Antibodies, Neoplasm
  • Prodrugs
  • ErbB Receptors
  • Cetuximab