Mitigation of T-cell dependent immunogenicity by reengineering factor VIIa analogue

Blood Adv. 2019 Sep 10;3(17):2668-2678. doi: 10.1182/bloodadvances.2019000338.

Abstract

Vatreptacog alfa (VA), a recombinant activated human factor VII (rFVIIa) variant with 3 amino acid substitutions, was developed to provide increased procoagulant activity in hemophilia patients with inhibitors to factor VIII or factor IX. In phase 3 clinical trials, changes introduced during the bioengineering of VA resulted in the development of undesired anti-drug antibodies in some patients, leading to the termination of a potentially promising therapeutic protein product. Here, we use preclinical biomarkers associated with clinical immunogenicity to validate our deimmunization strategy applied to this bioengineered rFVIIa analog. The reengineered rFVIIa analog variants retained increased intrinsic thrombin generation activity but did not elicit T-cell responses in peripheral blood mononuclear cells isolated from 50 HLA typed subjects representing the human population. Our algorithm, rational immunogenicity determination, offers a broadly applicable deimmunizing strategy for bioengineered proteins.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Blood Coagulation Tests
  • Cells, Cultured
  • Factor VIIa / genetics*
  • Factor VIIa / pharmacology
  • Hemophilia A / drug therapy
  • Humans
  • Immunogenetic Phenomena / drug effects
  • Protein Engineering / methods*
  • Recombinant Proteins / genetics
  • Recombinant Proteins / pharmacology
  • T-Lymphocytes / drug effects
  • T-Lymphocytes / immunology*
  • Thrombin / biosynthesis

Substances

  • Recombinant Proteins
  • vatreptacog alfa
  • recombinant FVIIa
  • Factor VIIa
  • Thrombin