Overview
At ADARx, we are committed to better outcomes for patients. We leverage our innovative RNA therapeutic approach with a goal of providing highly effective and convenient treatments addressing unmet needs across a broad range of diseases, including genetic, complement-mediated, cardiometabolic and central nervous system diseases.
Patients are at the center of everything we do, and we work with urgency knowing that we have the potential to improve the lives of patients.
Clinical Trials
Advancements in RNA medicines would not be possible without participation from patients in clinical trials. Learn more about our ongoing clinical trials below:
STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
If you are interested, visit this website:
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study in Healthy Volunteers and a Phase 2a, Open-Label Study in Patients with Hereditary Angioedema to Evaluate the Safety, Tolerability, PK and PD of ADX-324.
ADX-324 is novel siRNA drug that provides deep and long-lasting suppression of prekallikrein levels for HAE (American Academy of Allergy, Asthma & Immunology Annual Meeting 2026)
Phase 2 Study of ADX-038 in Complement-Mediated Kidney Disease
This Phase 2 study is designed to assess the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in adults with complement-mediated kidney diseases.
Phase 2 Study of ADX-038 in Participants With Geographic Atrophy
Phase 2 study is designed to assess the efficacy of ADX-038 compared with placebo in participants with GA secondary to AMD. Safety, pharmacokinetics (PK), and pharmacodynamics (PD) will also be assessed.
Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
The first-in-human Phase 1/Phase 2a study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Interim Phase 1 Study Results of ADX-038: A Novel siRNA Against Complement Factor B, and Next Steps in IgAN and C3 Glomerulopathy (C3G) (American Society of Nephrology Kidney Week 2025)
Phase 1 Study of ADX-626 in Healthy Participants
This first-in-human study will evaluate the safety and tolerability of ADX-626 in healthy participants. The study will also look at how ADX-626 interacts with the human body (the pharmacokinetics and pharmacodynamics of ADX-626).
For Patients
ADARx invites patients to consider the possibility of participating in our clinical studies. Your doctor may contact ADARx at info@adarx.com to learn more.
Expanded Access Policy
ADARx is committed to advancing RNAi therapeutics for patients with serious and rare diseases, and we deeply understand the urgency felt by individuals and families seeking every available treatment option. At this time, our development resources are focused on conducting rigorous clinical studies to establish the safety and efficacy of our investigational medicines and to obtain regulatory approval that will enable broader, equitable patient access. Because all available resources, including limited supplies of our investigational products, are dedicated to our ongoing clinical trials, ADARx does not have an Expanded Access Program at this time and is not able to consider requests for expanded access or compassionate use. Participation in our clinical trials currently represents the only pathway to access ADARx investigational medicines. As our programs advance and we gather additional safety and efficacy data, we will continue to evaluate the feasibility of establishing an Expanded Access Program, and we will update this policy should circumstances change.
We encourage patients to speak with their healthcare provider about clinical trial eligibility. If there is interest in learning more about clinical trials, please have your healthcare provided contact info@adarx.com for more information.
