ADARx Pharmaceuticals, Inc., a clinical stage biotechnology company developing RNA-targeting therapeutics, today announced the dosing of the first cohort in a Phase 1 clinical study of ADX-324 for the treatment of hereditary angioedema (HAE). HAE is a rare genetic disease that causes painful,rapid, and potentially life-threatening swelling attacks.
“We are very excited to be advancing our first candidate that utilizes our proprietaryPLR™ delivery platform and SPE™ technology into the clinic,” said Zhen Li, President and Chief Executive Officer. “ADX-324 represents an innovative anddifferentiated approach for the treatment of HAE.”“Dosing of our first participants in this trial is a major milestone for ADARx; in additionto its potential best-in-class efficacy, our pre-clinical studies in non-human primateshave confirmed the likelihood for a bi-annual, and possibly annual, low volumesubcutaneous dose regimen for ADX-324,” said Feriandas Greblikas, Vice Presidentof Clinical Development.

ADX-324 is a short-interfering RNA (siRNA) designed to reduce the production ofPKK, a protein critical to the etiology of HAE. The study is being conducted in Australia as a randomized, placebo-controlled, double-blind, single ascending dosetrial in healthy volunteers with an expansion cohort in patients with HAE. The primary objective of the study is to evaluate the safety and tolerability of ADX-324 whenadministered by subcutaneous injection.

Source: https://www.adarx.com/news/