Randomised, phase 1/2a trial of ION-827359, an antisense oligonucleotide inhibitor of ENaC.

ABSTRACT:

Background

Hyperactivity of epithelial sodium channel (ENaC) with increased sodium absorption is a feature of cystic fibrosis (CF). ION-827359 is a 2.5-generation antisense oligonucleotide targeted to reduce ENaC protein. This study evaluated ION-827359 safety, pharmacokinetics and pharmacodynamics.

Methods

In this three-part phase 1/2a, double-blind, randomised study, healthy volunteers received single doses of placebo or ION-827359 (3, 10, 37.5 or 100 mg; Part 1) or multiple doses of placebo or ION-827359 (5×10 mg, 5×37.5 mg, 5×75 mg or 10×37.5 mg; Part 2). People with CF (pwCF) received multiple doses of placebo or ION-827359 (5×10 mg, 5×37.5 mg, 5×75 mg and 5×100 mg; Part 3). Treatments were administered via Pari eFlow^© mesh nebuliser. The primary outco

SUBMITTER: Sutharsan S 

PROVIDER: S-EPMC11403593 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature