Inhibition of complement pathway activation with Pozelimab, a fully human antibody to complement component C5

PLoS One. 2020 May 8;15(5):e0231892. doi: 10.1371/journal.pone.0231892. eCollection 2020.

Abstract

Complement is a key component of the innate immune system. Inappropriate complement activation underlies the pathophysiology of a variety of diseases. Complement component 5 (C5) is a validated therapeutic target for complement-mediated diseases, but the development of new therapeutics has been limited by a paucity of preclinical models to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) properties of candidate therapies. The present report describes a novel humanized C5 mouse and its utility in evaluating a panel of fully human anti-C5 antibodies. Surprisingly, humanized C5 mice revealed marked differences in clearance rates amongst a panel of anti-C5 antibodies. One antibody, pozelimab (REGN3918), bound C5 and C5 variants with high affinity and potently blocked complement-mediated hemolysis in vitro. In studies conducted in both humanized C5 mice and cynomolgus monkeys, pozelimab demonstrated prolonged PK and durable suppression of hemolytic activity ex vivo. In humanized C5 mice, a switch in dosing from in-house eculizumab to pozelimab was associated with normalization of serum C5 concentrations, sustained suppression of hemolytic activity ex vivo, and no overt toxicity. Our findings demonstrate the value of humanized C5 mice in identifying new therapeutic candidates and treatment options for complement-mediated diseases.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibodies, Monoclonal, Humanized / immunology*
  • Antibodies, Monoclonal, Humanized / pharmacokinetics
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Antigen-Antibody Reactions
  • Binding Sites
  • Complement Activation / drug effects
  • Complement C5 / chemistry
  • Complement C5 / genetics
  • Complement C5 / immunology*
  • Genetic Variation
  • Half-Life
  • Hemolysis / drug effects
  • Humans
  • Inhibitory Concentration 50
  • Macaca fascicularis
  • Mice
  • Protein Structure, Quaternary

Substances

  • Antibodies, Monoclonal, Humanized
  • Complement C5
  • eculizumab

Grants and funding

All authors have a commercial affiliation with Regeneron Pharmaceuticals, Inc., which fully supported all studies reported in this manuscript. As such, Regeneron employees played active roles in all aspects of the research including study design, data collection and analysis, and the preparation and publication of this manuscript.