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ADX-324 Dose Level 2
ADX-324 Dose Level 2 is a ADAR1 inhibitor Small molecule drug developed by ADARx Pharmaceuticals, Inc.. It is currently in Phase 3 development for Advanced solid tumors (in combination with checkpoint inhibitors).
ADX-324 is an ADAR1 inhibitor that modulates RNA editing to enhance innate immune responses against cancer.
ADX-324 is an ADAR1 inhibitor that modulates RNA editing to enhance innate immune responses against cancer. Used for Advanced solid tumors (in combination with checkpoint inhibitors).
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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). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| 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 2 |
|---|---|
| Sponsor | ADARx Pharmaceuticals, Inc. |
| Drug class | ADAR1 inhibitor |
| Target | ADAR1 (Adenosine Deaminase Acting on RNA 1) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
ADX-324 selectively inhibits adenosine deaminase acting on RNA 1 (ADAR1), an enzyme that suppresses interferon signaling and antiviral immunity. By blocking ADAR1 activity, the drug restores interferon-beta production and activates pattern recognition receptors, thereby enhancing anti-tumor immune responses. This mechanism is designed to work synergistically with checkpoint inhibitors and other immunotherapies.
Approved indications
- Advanced solid tumors (in combination with checkpoint inhibitors)
Common side effects
- Interferon-related symptoms (fatigue, fever, myalgia)
- Transaminitis
- Cytokine release-related events
Key clinical trials
- Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (PHASE1, PHASE2)
- Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) (PHASE3)
- STOP-HAE: A Phase 3 Study of ADX-324 in HAE (PHASE3)
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 2 CI brief — competitive landscape report
- ADX-324 Dose Level 2 updates RSS · CI watch RSS
- ADARx Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about ADX-324 Dose Level 2
What is ADX-324 Dose Level 2?
How does ADX-324 Dose Level 2 work?
What is ADX-324 Dose Level 2 used for?
Who makes ADX-324 Dose Level 2?
What drug class is ADX-324 Dose Level 2 in?
What development phase is ADX-324 Dose Level 2 in?
What are the side effects of ADX-324 Dose Level 2?
What does ADX-324 Dose Level 2 target?
Related
- Drug class: All ADAR1 inhibitor drugs
- Target: All drugs targeting ADAR1 (Adenosine Deaminase Acting on RNA 1)
- Manufacturer: ADARx Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Advanced solid tumors (in combination with checkpoint inhibitors)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing