Last reviewed 2026-05-28 · How we verify

ADX-324 Dose Level 1

ADARx Pharmaceuticals, Inc. · Phase 3 active Small molecule Under review

ADX-324 Dose Level 1 is a ADAR inhibitor Small molecule drug developed by ADARx Pharmaceuticals, Inc.. It is currently in Phase 3 development for Autoimmune or inflammatory disease (specific indication not publicly detailed for Phase 3).

ADX-324 is an ADAR inhibitor that reduces adenosine-to-inosine RNA editing to modulate immune and inflammatory responses.

ADX-324 Dose Level 1 is being studied in a Phase 3 extension study for the treatment of Hereditary Angioedema (HAE). The study is targeting prekallikrein with ADX-324.

Likelihood of approval

59.3% vs 58.3% industry baseline

If approved by FDA: likely 2028–2030

Steps remaining: NDA/BLA submission

Confidence: High

Why this estimate

Baseline phase 3 → approval rate +58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
Immunology slight uplift +1.0pp
Mature endpoint landscape (ACR, DAS28, PASI) makes immunology approvals slightly more predictable.

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2028–2030	—
EMA	EU	2029–2031	+0.7 yr
MHRA	GB	2029–2031	+0.7 yr
Health Canada	CA	2029–2032	+0.9 yr
TGA	AU	2029–2032	+1.2 yr
PMDA	JP	2029–2032	+1.5 yr
NMPA	CN	2030–2033	+2.3 yr
MFDS	KR	2029–2032	+1.4 yr
CDSCO	IN	2029–2033	+1.8 yr
ANVISA	BR	2030–2033	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

At a glance

Generic name	ADX-324 Dose Level 1
Sponsor	ADARx Pharmaceuticals, Inc.
Drug class	ADAR inhibitor
Target	ADAR1 and/or ADAR2
Modality	Small molecule
Therapeutic area	Immunology / Autoimmune
Phase	Phase 3

Mechanism of action

ADAR (adenosine deaminase acting on RNA) enzymes catalyze A-to-I editing of RNA transcripts, which affects protein diversity and immune signaling. By inhibiting ADAR activity, ADX-324 alters the RNA editing landscape in immune cells and other tissues, potentially reducing pathogenic immune activation or inflammatory responses. This mechanism is being explored in autoimmune and inflammatory conditions.

Approved indications

Autoimmune or inflammatory disease (specific indication not publicly detailed for Phase 3)

Common side effects

No common side effects on file.

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

ADX-324 Dose Level 1 CI brief — competitive landscape report
ADX-324 Dose Level 1 updates RSS · CI watch RSS
ADARx Pharmaceuticals, Inc. portfolio CI

Frequently asked questions about ADX-324 Dose Level 1

What is ADX-324 Dose Level 1?

ADX-324 Dose Level 1 is a ADAR inhibitor drug developed by ADARx Pharmaceuticals, Inc., indicated for Autoimmune or inflammatory disease (specific indication not publicly detailed for Phase 3).

How does ADX-324 Dose Level 1 work?

ADX-324 is an ADAR inhibitor that reduces adenosine-to-inosine RNA editing to modulate immune and inflammatory responses.

What is ADX-324 Dose Level 1 used for?

ADX-324 Dose Level 1 is indicated for Autoimmune or inflammatory disease (specific indication not publicly detailed for Phase 3).

Who makes ADX-324 Dose Level 1?

ADX-324 Dose Level 1 is developed by ADARx Pharmaceuticals, Inc. (see full ADARx Pharmaceuticals, Inc. pipeline at /company/adarx-pharmaceuticals-inc).

What drug class is ADX-324 Dose Level 1 in?

ADX-324 Dose Level 1 belongs to the ADAR inhibitor class. See all ADAR inhibitor drugs at /class/adar-inhibitor.

What development phase is ADX-324 Dose Level 1 in?

ADX-324 Dose Level 1 is in Phase 3.

What does ADX-324 Dose Level 1 target?

ADX-324 Dose Level 1 targets ADAR1 and/or ADAR2 and is a ADAR inhibitor.

Drug class: All ADAR inhibitor drugs
Target: All drugs targeting ADAR1 and/or ADAR2
Manufacturer: ADARx Pharmaceuticals, Inc. — full pipeline
Therapeutic area: All drugs in Immunology / Autoimmune
Indication: Drugs for Autoimmune or inflammatory disease (specific indication not publicly detailed for Phase 3)

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing