Results of the first-in-human, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose study of BIIB113 in healthy volunteers.

ABSTRACT:

Background

Preclinical studies have demonstrated that inhibition of the O-linked β-N-acetylglucosaminidase enzyme increases tau O-linked β-N-acetylglucosaminylation and may attenuate tau pathology in Alzheimer's disease.

Objectives

To examine the safety, tolerability, pharmacokinetics, and target occupancy of single- and multiple-ascending oral doses of the small-molecule O-linked β-N-acetylglucosaminidase inhibitor, BIIB113.

Design

Study 276HV101 was a first-in-human, multicenter, Phase 1, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose trial.

Setting

72 participants were enrolled from February 2022 through July 2023.

Participants

Adult healthy female and infertile/vasectomized male participants.

Intervention

In

SUBMITTER: Nery FC 

PROVIDER: S-EPMC12413702 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature