Product Description
ONC201 is the founding member of the imipridone class of anti-cancer small molecules which selectively targets Dopamine Receptor D2 (DRD2) and ClpP. ONC201-mediated cell death occurs via induction of the integrated stress response and upregulation of apoptotic factors, such as tumor necrosis factor (TNF)-related apoptosis-inducing ligand (TRAIL). It is dosed orally and has been well-tolerated and shown clinical activity in Phase I and II trials for specific advanced cancers. (Sourced from: https://www.chimerix.com/our-pipeline/imipridones/onc201/)
Mechanisms of Action: D2 Antagonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Priority Review - Glioma *
Approval Status: Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Jazz
Company Location: Europe
Company CEO: Bruce C. Cozadd
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Australia, Austria, Brazil, Canada, China, Denmark, France, Germany, Hong Kong, Israel, Italy, Japan, Netherlands, Singapore, South Korea, Spain, Switzerland, United Kingdom, United States
Active Clinical Trial Count: 9
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Jazz presented P3 Glioma results on 2025-06-02 for Dordaviprone
- PDUFA summary: Accelerated approval decision for Dordaviprone in recurrent H3 K27M-mutant diffuse glioma scheduled by FDA for August 18, 2025.
- Clinical Outcomes Reported - Chimerix presented P2 Glioma results on 2024-11-22 for Dordaviprone
Highest Development Phases
Phase 3: Glioma
Phase 2: Meningioma
Phase 1: Carcinosarcoma|Clear Cell Adenocarcinoma|Clear Cell Sarcoma|Endometrial Cancer|Endometrioid Carcinoma|Healthy Volunteers|Obesity|Oncology Unspecified|Urogenital Cancer
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05542407 |
LCCC2036 | P1 |
Recruiting |
Endometrioid Carcinoma|Obesity|Clear Cell Sarcoma|Endometrial Cancer|Clear Cell Adenocarcinoma|Carcinosarcoma |
2026-02-21 |
2% |
2025-09-13 |
Primary Endpoints|Treatments |
ACTRN12622000405718 |
ACTRN12622000405718 | P1 |
Completed |
Urogenital Cancer |
2023-04-20 |
2024-08-29 |
Treatments |
|
ACTRN12622001020774 |
ACTRN12622001020774 | P1 |
Completed |
Oncology Unspecified|Healthy Volunteers |
2023-04-03 |
2024-08-29 |
Treatments |
|
NCT06012929 |
0137-24-FB | P2 |
Suspended |
Meningioma |
2026-04-01 |
12% |
2025-08-27 |
Primary Endpoints|Treatments |
NCT05580562 |
ACTION | P3 |
Recruiting |
Glioma |
2026-08-01 |
18% |
2025-08-27 |
|
jRCT2051240141 |
jRCT2051240141 | P3 |
Not yet recruiting |
Glioma |
2026-09-30 |
|||
NCT05476939 |
BIOMEDE 2 | P3 |
Recruiting |
Glioma |
2028-09-01 |
30% |
2024-03-02 |
Patient Enrollment|Primary Endpoints|Treatments |
CTR20233492 |
CTR20233492 | P1 |
Recruiting |
Glioma |
None |
2025-04-29 |
Patient Enrollment|Start Date|Treatments|Trial Status |
|
2022-502051-56-00 |
ONC201-108 | P3 |
Recruiting |
Glioma |
2026-12-15 |
2025-05-02 |
Treatments |