Randomized, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study of the Penetration of a Monoclonal Antibody Combination (ASN100) Targeting Staphylococcus aureus Cytotoxins in the Lung Epithelial Lining Fluid of Healthy Volunteers

Antimicrob Agents Chemother. 2019 Jul 25;63(8):e00350-19. doi: 10.1128/AAC.00350-19. Print 2019 Aug.

Abstract

ASN100 is a novel antibody combination of two fully human IgG1(κ) monoclonal antibodies (MAbs), ASN-1 and ASN-2, which neutralize six Staphylococcus aureus cytotoxins, alpha-hemolysin (Hla) and five bicomponent leukocidins. We assessed the safety, tolerability, and serum and lung pharmacokinetics of ASN100 in a randomized, double-blind, placebo-controlled single-dose-escalation first-in-human study. Fifty-two healthy volunteers were enrolled and randomized to receive either ASN-1, ASN-2, a combination of both MAbs (ASN100), or a corresponding placebo. Thirty-two subjects in the double-blind dose escalation portion of the study received ASN-1 or ASN-2 at a 200-, 600-, 1,800-, or 4,000-mg dose, or placebo. Eight subjects received both MAbs simultaneously in a 1:1 ratio (ASN100) at 3,600 or 8,000 mg, or they received placebos. Twelve additional subjects received open-label ASN100 at 3,600 or 8,000 mg to assess the pharmacokinetics of ASN-1 and ASN-2 in epithelial lining fluid (ELF) by bronchoalveolar lavage fluid sampling. Subjects were monitored for 98 days (double-blind cohorts) or 30 days (open-label cohorts) for safety assessment. No dose-limiting toxicities were observed, and all adverse events were mild and transient, with only two adverse events considered possibly related to the investigational product. ASN100 exhibited linear serum pharmacokinetics with a half-life of approximately 3 weeks and showed detectable penetration into the ELF. No treatment-emergent anti-drug antibody responses were detected. The toxin neutralizing potency of ASN100 in human serum was confirmed up to 58 days postdosing. The favorable safety profile, ELF penetration, and maintained functional activity in serum supported the further clinical development of ASN100.

Keywords: ASN100; Staphylococcus aureus cytotoxins; anti-infective monoclonal antibodies; epithelial lining fluid pharmacokinetics; first-in-human trial; phase 1.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Anti-Bacterial Agents / administration & dosage*
  • Anti-Bacterial Agents / pharmacokinetics
  • Antibodies, Monoclonal / administration & dosage*
  • Antibodies, Monoclonal / adverse effects
  • Antibodies, Monoclonal / pharmacokinetics*
  • Antibodies, Monoclonal / pharmacology
  • Antibodies, Monoclonal, Humanized / administration & dosage*
  • Antibodies, Monoclonal, Humanized / adverse effects
  • Antibodies, Monoclonal, Humanized / pharmacokinetics*
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Bacterial Proteins / antagonists & inhibitors
  • Bacterial Proteins / immunology
  • Bacterial Toxins / antagonists & inhibitors*
  • Bacterial Toxins / immunology
  • Bronchoalveolar Lavage Fluid
  • Cytotoxins / antagonists & inhibitors
  • Cytotoxins / immunology*
  • Cytotoxins / metabolism
  • Double-Blind Method
  • Female
  • Healthy Volunteers
  • Hemolysin Proteins / antagonists & inhibitors
  • Hemolysin Proteins / immunology
  • Humans
  • Leukocidins / antagonists & inhibitors
  • Leukocidins / immunology
  • Male
  • Placebos
  • Staphylococcal Infections
  • Staphylococcus aureus / immunology

Substances

  • ASN100
  • Anti-Bacterial Agents
  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Bacterial Proteins
  • Bacterial Toxins
  • Cytotoxins
  • Hemolysin Proteins
  • Leukocidins
  • Placebos
  • leukocidin AB, Staphylococcus aureus
  • leukocidin S-component protein, Staphylococcus
  • staphylococcal alpha-toxin
  • LukF protein, Staphylococcus aureus