Drugs / Dordaviprone
last change Jul 2026 re-read 2 minutes ago

Dordaviprone

Small molecule targets CLPP, DRD2 via inhibition

Developed for
diffuse midline glioma, H3 K27M-mutant · endometrial cancer · glioblastoma · glioma · plasma cell myeloma · triple-negative breast carcinoma · acute myeloid leukemia · colorectal cancer
+27 more · myelodysplastic syndrome · acute lymphoblastic leukemia · cancer · cancer of unknown primary site · carcinosarcoma · central nervous system non-hodgkin lymphoma · classic familial adenomatous polyposis · colorectal adenoma · colorectal carcinoma · diffuse intrinsic pontine glioma · endometrial clear cell adenocarcinoma · endometrial endometrioid adenocarcinoma · endometrial mixed adenocarcinoma · endometrial serous adenocarcinoma · fallopian tube carcinoma · gastric mantle cell lymphoma · gliosarcoma · grade III glioma · hormone receptor-positive breast cancer · meningioma · metastatic neoplasm · neuroendocrine neoplasm · non-small cell lung carcinoma · ovarian carcinoma · polyposis · primary peritoneal carcinoma · splenic mantle cell lymphoma
Investigated by
Jazz Pharmaceuticals · Oncoceutics, Inc. · National Cancer Institute (NCI) · Fox Chase Cancer Center · M.D. Anderson Cancer Center · Rutgers Cancer Institute of New Jersey
+29 more · Rutgers, The State University of New Jersey · University of Nebraska · Ajai Chari · Bristol-Myers Squibb · Brown University · CV Biomanufacturing · Chimerix, Inc. · Genentech, Inc. · Gustave Roussy, Cancer Campus, Grand Paris · Innovative Therapies For Children with Cancer Consortium · Ira Winer · Jonsson Comprehensive Cancer Center · Masonic Cancer Center, University of Minnesota · Ministry of Health, France · Mithil Prasad Foundation · National Institute of Neurological Disorders and Stroke (NINDS) · Neeve Kolte and Brave Ronil Foundation · Peter Anderson · Rhode Island Hospital · Storm the Heavens Fund · The ChadTough Defeat DIPG Foundation · The V Foundation for Cancer Research · Tough2gether Foundation · UNC Lineberger Comprehensive Cancer Center · University of California, Los Angeles · University of California, San Francisco · University of Minnesota · University of Wisconsin, Madison · Will Meeker Foundation

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) MODEYSO 2025-08-06 fda.gov

Trials 29 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 18 trials
Phase 1 NCT05630794 Oct 2025 → May 2027 expected classic familial adenomatous polyposis, colorectal adenoma, colorectal carcinoma, polyposis National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 1 NCT05542407 Oct 2023 → Feb 2028 expected carcinosarcoma, endometrial clear cell adenocarcinoma, endometrial endometrioid adenocarcinoma, endometrial mixed adenocarcinoma +2 UNC Lineberger Comprehensive Cancer Center Recruiting No outcome recorded
Phase 1 NCT04854044 May 2021 → Jul 2025 gliosarcoma Jonsson Comprehensive Cancer Center Withdrawn No outcome recorded
Phase 1 NCT03932643 Jul 2019 → Mar 2025 acute myeloid leukemia, myelodysplastic syndrome University of Nebraska Completed No outcome recorded
Phase 1 NCT03416530 Jan 2018 → Dec 2022 diffuse intrinsic pontine glioma Jazz Pharmaceuticals Terminated No outcome recorded
Phase 1 NCT02609230 Nov 2015 → Mar 2020 plasma cell myeloma Fox Chase Cancer Center Completed No outcome recorded
Phase 1 NCT02324621 Feb 2015 → Oct 2018 cancer of unknown primary site Rutgers, The State University of New Jersey Completed No outcome recorded
Phase 1 NCT02250781 Jan 2015 → Oct 2018 cancer Rutgers, The State University of New Jersey Completed No outcome recorded
Phase 1/26 trials
Phase 1/2 NCT03791398 Nov 2019 → Jun 2021 colorectal cancer Brown University Terminated No outcome recorded
Phase 1/2 NCT03492138 Mar 2018 → Jan 2020 plasma cell myeloma Ajai Chari Terminated No outcome recorded
Phase 1/2 NCT02863991 Apr 2017 → Sep 2019 plasma cell myeloma Jazz Pharmaceuticals Terminated No outcome recorded
Phase 1/2 NCT02392572 Nov 2015 → Nov 2026 expected acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 1/2 NCT02420795 Nov 2015 → Nov 2020 central nervous system non-hodgkin lymphoma, gastric mantle cell lymphoma, splenic mantle cell lymphoma M.D. Anderson Cancer Center Terminated No outcome recorded
Phase 1/2 NCT02038699 Jan 2014 → Dec 2015 colorectal cancer, glioblastoma, non-small cell lung carcinoma, triple-negative breast carcinoma Jazz Pharmaceuticals Withdrawn No outcome recorded
Phase 211 trials
Phase 2 NCT06012929 Dec 2026 → Apr 2027 meningioma University of Nebraska Withdrawn No outcome recorded
Phase 2 NCT04629209 Jun → Dec 2024 glioblastoma Masonic Cancer Center, University of Minnesota Withdrawn No outcome recorded
Phase 2 NCT05009992 Oct 2021 → Dec 2027 expected diffuse midline glioma, H3 K27M-mutant, grade III glioma University of California, San Francisco Recruiting No outcome recorded
Phase 2 NCT04055649 Jan 2020 → May 2027 expected fallopian tube carcinoma, ovarian carcinoma, primary peritoneal carcinoma Ira Winer Recruiting No outcome recorded
Phase 2 NCT03733119 Nov 2018 → Feb 2021 triple-negative breast carcinoma University of Wisconsin, Madison Terminated No outcome recorded
Phase 2 NCT03485729 Mar 2018 → Jan 2023 endometrial cancer Jazz Pharmaceuticals Terminated No outcome recorded
Phase 2 NCT03394027 Jan 2018 → Mar 2021 endometrial cancer, hormone receptor-positive breast cancer, triple-negative breast carcinoma National Cancer Institute (NCI) Completed No outcome recorded
Phase 2 NCT03295396 Oct 2017 → Dec 2022 glioma Jazz Pharmaceuticals Terminated No outcome recorded
Phase 2 NCT03034200 Aug 2017 → May 2023 neuroendocrine neoplasm Peter Anderson Completed No outcome recorded
Phase 2 NCT03099499 Jun 2017 → Sep 2020 endometrial cancer Fox Chase Cancer Center Terminated No outcome recorded
Phase 32 trials
Phase 3 NCT05580562 Jan 2023 → Jun 2028 expected diffuse midline glioma, H3 K27M-mutant Jazz Pharmaceuticals Recruiting No outcome recorded
Phase 3 NCT05476939 Sep 2022 → Sep 2028 expected diffuse midline glioma, H3 K27M-mutant Gustave Roussy, Cancer Campus, Grand Paris Recruiting No outcome recorded
Phase not stated2 trials
NCT05392374 glioma Jazz Pharmaceuticals No longer available No outcome recorded
NCT03134131 glioma Jazz Pharmaceuticals No longer available No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
NCT04617002 Background diffuse midline glioma, H3 K27M-mutant Jazz Pharmaceuticals Approved for marketing No outcome recorded

Evidence & citations 14 cited values

Every value below carries the sentence it was read from. 33 sources stand behind the page.

FieldValueCited text
Known as dordaviprone “ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma.” PMID 38335473 Feb 2024
8

“ONC201 (dordaviprone) is a ClpP agonist and DRD2 antagonist.” NCT04617002

Dordaviprone (ONC201)” NCT05476939

NCT02525692

NCT03416530

NCT05630794

NCT05580562

NCT03485729

NCT03295396

Known as Dordaviprona ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as Dordaviprone dihydrochloride ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as DORDAVIPRONE HYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as NSC-350625 ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as ONC-201 DIHYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as ONC201 “The purpose of this phase I trial is to test the safety and cancer preventive effects of different doses of ONC201 in people with familial adenomatous polyposis (FAP) or a...” NCT05630794
29

“Dordaviprone (ONC201) is a brain-penetrant, small-molecule imipridone that acts as a mitochondrial caseinolytic protease P (ClpP) agonist and a dopamine receptor D2 (DRD2) antagonist.” NCT02525692

“This integrated analysis evaluated single-agent ONC201 (dordaviprone), a first-in-class imipridone, in recurrent H3 K27M-mutant DMG.” PMID 38335473 Feb 2024

“Dordaviprone (ONC201)” NCT05580562

“Dordaviprone (ONC201)” NCT03485729

“Dordaviprone (ONC201)” NCT03416530

NCT04629209

NCT05542407

NCT02609230

NCT03099499

NCT02420795

NCT05009992

NCT02863991

NCT02250781

NCT05392374

NCT03932643

NCT03733119

NCT04854044

NCT03492138

NCT03394027

NCT05476939

NCT03134131

NCT02392572

NCT04617002

NCT03034200

NCT02324621

NCT04055649

NCT03791398

NCT06012929

NCT02038699

Known as ONC201 hydrochloride ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Known as TIC10 “ONC201 (TIC10) is a first-in-class small molecule” NCT02038699
Known as TIC10 hydrochloride ChEMBL registry synonym — accepted as the source's own label CHEMBL4297310
Action Inhibit “small molecule antagonist of DRD2” PMID 31118108 May 2019
Modality Small molecule “ONC201 (TIC10) is a first-in-class small moleculeNCT02038699
Route Oral “ONC201 capsules will be administered orally” NCT02038699
Target DRD2 “ONC201 is a small molecule antagonist of DRD2PMID 31118108 May 2019