ADARx Pharmaceuticals, Inc. announces the dosing of the first cohort in a Phase 1 clinical study of ADX-324 for the treatment of HAE.

“We are very excited to be advancing our first candidate that utilizes our proprietary PLR™ delivery platform and SPE™ technology into the clinic,” says Zhen Li, President and CEO. “ADX-324 represents an innovative and differentiated approach for the treatment of HAE.”

“Dosing of our first participants in this trial is a major milestone for ADARx; in addition to its potential best-in-class efficacy, our pre-clinical studies in non-human primates have confirmed the likelihood for a bi-annual, and possibly annual, low volume subcutaneous dose regimen for ADX-324,” says Feriandas Greblikas, Vice President
of Clinical Development.

ADX-324 is a short-interfering RNA (siRNA) designed to reduce the production of PKK, a protein critical to the etiology of HAE. The study is being conducted in Australia as a randomized, placebo-controlled, double-blind, single ascending dose trial 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 when administered by subcutaneous injection.
(Source: ADARx)