First-in-Human Study of the Safety, Tolerability, Pharmacokinetics and - Preliminary Dynamics of Neuroprotectant 2-Iminobiotin in Healthy Subjects

Curr Clin Pharmacol. 2020;15(2):152-163. doi: 10.2174/1574884714666191017111109.

Abstract

Background: 2-iminobiotin (2-IB) is an investigational neuroprotective agent in development for the reduction of brain cell injury after cerebral hypoxia-ischemia.

Objective: The present first-in-human study evaluated the safety, tolerability, pharmacokinetics (PK) and -dynamics (PD) of 2-IB in healthy male subjects, intravenously infused with or without Captisol® as a solubilizing agent.

Methods: This randomized, double-blind, placebo-controlled, dose-escalation study was executed in 2 groups of 9 healthy male subjects. A single dose of 2-IB 0.6 mg/kg or placebo was infused over periods between 15 min and 4 h, and repeated doses escalating from 0.6 mg/kg to 12 mg/kg, or placebo were infused every 4 h for 6 administrations in total.

Results: Single and multiple doses of 2-IB up to 6 doses of 6 mg/kg with and without Captisol® were safe and well-tolerated in healthy male subjects. 2-IB proved to be a high-clearance drug with a volume of distribution slightly exceeding total body water volume, and with linear PK that appeared not to be affected by the presence of Captisol®.

Conclusion: Sulfobutyletherbeta-cyclodextrin (SBECD) in Captisol® had a low-clearance profile with a small volume of distribution, with time-independent PK. Preliminary PD characterization of repeated iv dosing of 2-IB in an acute peripheral hypoxic ischemia model in healthy subjects did not reveal any notable effects of 2-IB, noting that this model was not selected to guide efficacy in the currently pursued indication of cerebral hypoxia-ischemia.

Keywords: 2-iminobiotin; birth asphyxia; cardiac arrest; healthy subjects; neuroprotection; nitric oxide synthase; pharmacokinetics.

Publication types

  • Clinical Trial, Phase I
  • Comparative Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Biotin / administration & dosage
  • Biotin / adverse effects
  • Biotin / analogs & derivatives*
  • Biotin / pharmacokinetics
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Excipients / chemistry*
  • Humans
  • Hypoxia-Ischemia, Brain / drug therapy
  • Infusions, Intravenous
  • Male
  • Middle Aged
  • Neuroprotective Agents / administration & dosage*
  • Neuroprotective Agents / adverse effects
  • Neuroprotective Agents / pharmacokinetics
  • Time Factors
  • Tissue Distribution
  • Young Adult
  • beta-Cyclodextrins / chemistry*

Substances

  • Excipients
  • Neuroprotective Agents
  • beta-Cyclodextrins
  • SBE4-beta-cyclodextrin
  • Biotin
  • 2-iminobiotin