Mitigation of Pre-existing Antibodies to a Biotherapeutic in Non-clinical Species When Establishing Anti-drug Antibody Assay Cutpoint

AAPS J. 2017 Jan;19(1):313-319. doi: 10.1208/s12248-016-0011-2. Epub 2016 Nov 21.

Abstract

Biotherapeutics are known for their potential to induce drug specific immune responses, which are commonly evaluated by the detection of anti-drug antibodies (ADAs). For some biotherapeutics, pre-existing ADAs against drug have been observed in drug-naïve matrix. The presence of pre-existing drug specific antibodies may significantly complicate assessment of the screening ADA assay cutpoint value, which is usually established based on the statistical analysis of signal distribution from the drug-naïve individuals. A Gaussian mixture model-based approach is presented herein to address high prevalence of pre-existing ADAs to a modified monoclonal antibody-based biotherapeutic (m-mAb). A high prevalence of pre-existing anti-m-mAb antibodies was observed in drug-naïve individual cynomolgus monkey serum samples with signal ranging from 100 to 7000 relative light units (RLU, as determined in an electrochemiluminescence readout-based assay). Application of the industry standard statistical algorithm resulted in a relatively high floating screening assay cutpoint factor (CPF) of 9.80, which potentially would have reported a high percent of false negative samples. An alternative, Gaussian mixture model-based approach was applied to identify the least reactive individual samples in the tested population, which resulted in a floating screening assay CPF of 2.35. The low CPF value significantly reduced the risk of reporting false negative results. The proposed Gaussian mixture model-based approach described herein provides an alternate method for the calculation of biologically relevant screening assay CPF when high prevalence of pre-existing drug specific antibodies is observed.

Keywords: Gaussian mixture model; anti-drug antibody (ADA); assay cutpoint; floating cutpoint factor (CPF); negative control (NC); positive control (PC); pre-existing antibodies.

MeSH terms

  • Animals
  • Antibodies, Monoclonal / blood*
  • Antibodies, Monoclonal / immunology
  • Antibodies, Monoclonal / therapeutic use
  • Biological Products / blood*
  • Biological Products / immunology
  • Biological Therapy / methods
  • False Positive Reactions
  • In Vitro Techniques
  • Luminescent Measurements / methods*
  • Luminescent Measurements / statistics & numerical data
  • Macaca fascicularis
  • Models, Biological*
  • Models, Statistical*
  • Rats, Inbred Strains

Substances

  • Antibodies, Monoclonal
  • Biological Products