Phase II Dose Selection for Alpha Synuclein-Targeting Antibody Cinpanemab (BIIB054) Based on Target Protein Binding Levels in the Brain

CPT Pharmacometrics Syst Pharmacol. 2020 Sep;9(9):515-522. doi: 10.1002/psp4.12538. Epub 2020 Aug 19.

Abstract

This modeling and simulation analysis was aimed at selecting doses of cinpanemab (BIIB054), a monoclonal antibody targeting aggregated α-synuclein, for a phase II study in Parkinson's disease (PD). Doses and regimens were proposed based on anticipated target concentration in brain interstitial fluid (ISF); in vitro/in vivo data on the affinity of monoclonal antibodies to the target protein; and safety, tolerability, and pharmacokinetic data (1-135 mg/kg intravenous administration) from a phase I single ascending dose (SAD) study. A population pharmacokinetic modeling approach was used to select intravenous doses of 250, 1,250, and 3,500 mg every 4 weeks, to maintain 50%, 90%, and > 90% of target binding in ISF of PD participants. A favorable safety profile from the SAD study-which showed that cinpanemab was generally well-tolerated at doses up to 90 mg/kg, supported by modeling and simulations of the anticipated safety margins-allowed implementation of a fixed-dose approach.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Intravenous
  • Adult
  • Aged
  • Antibodies, Monoclonal / administration & dosage
  • Antibodies, Monoclonal / pharmacokinetics*
  • Brain / metabolism*
  • Computer Simulation
  • Dose-Response Relationship, Immunologic
  • Drug Development / methods
  • Drug Tolerance
  • Extracellular Fluid / drug effects
  • Extracellular Fluid / metabolism
  • Female
  • Humans
  • Male
  • Middle Aged
  • Parkinson Disease / drug therapy*
  • Protein Binding
  • Safety
  • alpha-Synuclein / antagonists & inhibitors*
  • alpha-Synuclein / metabolism

Substances

  • Antibodies, Monoclonal
  • alpha-Synuclein