ADARx Pharmaceuticals is recruiting for the first-in-human clinical study assessing the pharmacokinetics and pharmacodynamics of ADX-324, as well as the safety and tolerability of the experimental treatment, in healthy volunteers and patients with hereditary angioedema (HAE). 

ADX-324 is a short-interfering RNA (siRNA) duplex oligonucleotide. Participants will be randomized to receive either ADX-324 or the placebo in the first phase of the study. 

The research team aims to enroll 53 participants for the 2-part study. The first part consists of randomized, double-blind, placebo-controlled, parallel groups with single ascending doses among healthy individuals with 6 dose cohorts. The second part will be open-label and consist of participants with HAE administered a selected dose from the first part of the study.

Continue Reading

Read more about hereditary angioedema etiology

The primary outcome measure is the safety of ADX-324 for use in healthy volunteers and patients with HAE over 183 days. The second outcome measure is the pharmacokinetics and pharmacodynamics of this drug in both healthy volunteers and patients with this condition. 

The trial has an estimated primary completion date of January 2, 2024, and an estimated study completion date of November 26, 2024. 


Safety, tolerability, PK, PD of ADX-324 in healthy volunteers and hereditary angioedema patients. Published January 20, 2023. Accessed January 20, 2023.