SAN DIEGO–(BUSINESS WIRE)–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 proprietary PLR™ delivery platform and SPE™ technology into the clinic,” said Zhen Li, President and Chief Executive Officer. “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,” said 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.
More information can be found here:
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients – Full Text View – ClinicalTrials.gov
ADARx Pharmaceuticals, Inc., located in San Diego, is a clinical stage biotechnology company committed to turning cutting-edge science into life-saving therapeutics. ADARx is developing a proprietary RNA targeting platform, including oligonucleotides for inhibition, degradation, and editing, together with novel oligonucleotide delivery technologies including its PLR™ (“Preeminent Liver RNA”) platform and SPE™ (“Sequence Specific Platform Enhancement”) technology. ADARx has a growing pipeline of RNA targeting therapeutics for treating diseases across a range of therapeutic areas including genetic, cardiometabolic, complement-mediated and central nervous system diseases.