Therapy Detail

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Therapy Name ONC201
Synonyms
Therapy Description

ONC201 (TIC-10) binds to the G-protein coupled receptor, DRD2, leading to inactivation of AKT and ERK, which relocates Foxo3a to the nucleus and then leads to tumor cell apoptosis mediated by (TNF)-related apoptosis-inducing ligand (TRAIL) signaling (PMID: 23390247) and targets the mitochondrial protease, ClpP (PMID: 31021596).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
ONC201 TIC-10|TIC10 Akt Inhibitor (Pan) 20 ERK Inhibitor (pan) 16 ONC201 (TIC-10) binds to the G-protein coupled receptor, DRD2, leading to inactivation of AKT and ERK, which relocates Foxo3a to the nucleus and then leads to tumor cell apoptosis mediated by (TNF)-related apoptosis-inducing ligand (TRAIL) signaling (PMID: 23390247) and targets the mitochondrial protease, ClpP (PMID: 31021596, PMID: 31702782, PMID: 31456142).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown glioblastoma not applicable ONC201 Phase II Actionable In a Phase II trial, ONC201 treatment was well tolerated, however, did not meet the 6-month progression-free survival (PFS) endpoint at 5% in unselected glioblastoma multiforme patients (n=20), with a median PFS of 1.8 months and a median overall survival of 7.5 months, but led to complete tumor regression in one glioblastoma multiforme patient harboring H3 K28M (also referred to as K27M) with a durable response of over 1.5 years (PMID: 31702782; NCT02525692). 31702782
Unknown unknown prostate cancer not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate cancer patient demonstrated tumor regression in the primary tumor and metastatic lesions when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown thyroid gland medullary carcinoma not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with ONC201 (TIC-10) in medullary thyroid cancer cells resulted in inhibition of RET, VEGFR2, and IGFBP2 transcription, increased gene expression of ATF4 and ATF4 target genes, and induced apoptosis in culture, and in cell line xenograft models led to tumor growth inhibition and decreased cell proliferation and angiogenesis (PMID: 33536187). 33536187
Unknown unknown mantle cell lymphoma not applicable ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, ONC201 induced apoptosis and decreased viability of mantle cell lymphoma (MCL) cell lines in culture and demonstrated cytotoxicity in primary MCL samples in culture (PMID: 26884599). 26884599 detail...
Unknown unknown triple-receptor negative breast cancer not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several triple-negative breast cancer (TNBC) cell lines in culture, demonstrating variable pro-apototic and anti-proliferative activity, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown multiple myeloma not applicable ONC201 Preclinical - Cell culture Actionable In a preclinical study, a Velcade (bortezomib)-resistant multiple myeloma cell line demonstrated sensitivity to ONC201 in culture (PMID: 26884599). 26884599
Unknown unknown prostate adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate adenocarcinoma patient demonstrated extended stable disease for 27 weeks when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown endometrial cancer not applicable ONC201 Clinical Study Actionable In a clinical case study, a patient with endometrial cancer demonstrated stable disease for 42 weeks and continued regression of metastatic lesions in the lung when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown breast cancer not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several breast cancer cell lines in culture (including triple-negative breast cancer (TNBC) and non-TNBC cell lines), with some cell lines demonstrating increased apoptosis, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown Advanced Solid Tumor not applicable ONC201 Phase I Actionable In a Phase I trial, ONC201 treatment resulted in stable disease in 80% (8/10) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2514)). detail...
Unknown unknown Advanced Solid Tumor not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC-201 (TIC-10) induced apoptosis and tumor regression in a variety of cell line xenograft models, including colon, breast, and brain cancer (PMID: 23390247). 23390247
Unknown unknown acute myeloid leukemia not applicable ONC201 Preclinical - Pdx & cell culture Actionable In a preclinical study, ONC201 treatment induced ATF4 expression and apoptosis in acute myeloid leukemia (AML) cell lines in culture, independent of Tp53 status, and was toxic to AML primary samples in culture, resulting in decreased engraftment in xenograft models (PMID: 26884599). 26884599
Unknown unknown endometrial clear cell adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a patient with clear cell endometrial cancer treated with ONC201 (TIC-10) demonstrated some reduction in lesion size (>30%), but also developed new lesions (PMID: 28331050). 28331050

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT02863991 Phase II ONC201 Oral ONC201 in Relapsed/Refractory Multiple Myeloma Active, not recruiting USA 0
NCT03932643 Phase I ONC201 ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant Recruiting USA 0
NCT03416530 Phase I ONC201 ONC201 in Pediatric H3 K27M Gliomas Recruiting USA 0
NCT02392572 Phase Ib/II ONC201 ONC201 in Relapsed/Refractory Acute Leukemias and High-risk Myelodysplastic Syndromes (HR-MDS) Active, not recruiting USA 0
NCT03134131 Expanded access ONC201 Expanded Access Program for ONC201 to Treat Recurrent Histone H3 Mutant Glioma No longer available USA 0
NCT04617002 Expanded access ONC201 Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas Available USA 0
NCT02038699 Phase Ib/II ONC201 A First-in-man Phase I/II Study of Oral ONC201 in Patients With Advanced Cancer Withdrawn USA 0
NCT03394027 Phase II ONC201 ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma Active, not recruiting USA 0
NCT03485729 Phase II ONC201 ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer Recruiting USA 0
NCT03099499 Phase II ONC201 Single Agent ONC201 in Recurrent or Metastatic Endometrial Cancer Recruiting USA 0
NCT02250781 Phase I ONC201 Akt/ERK Inhibitor ONC201 in Treating Patients With Advanced Solid Tumors Completed USA 0
NCT04854044 Phase I ONC201 ONC201 and Radiation Therapy Before Surgery for the Treatment of Recurrent Glioblastoma Withdrawn USA 0
NCT03034200 Phase II ONC201 Phase 2 Study of ONC201 in Neuroendocrine Tumors Recruiting USA 0
NCT03733119 Phase II ONC201 ONC201 With and Without Methionine-Restricted Diet in Patients With Metastatic Triple Negative Breast Cancer Suspended USA 0
NCT03295396 Phase II ONC201 ONC201 in Adults With Recurrent H3 K27M-mutant Glioma Recruiting USA 0
NCT02525692 Phase II ONC201 Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma Recruiting USA 0
NCT02609230 Phase I ONC201 A Dose-Escalation Study of Onc201 Administered Every One or Three Weeks in Advanced Solid Tumors and Multiple Myeloma Active, not recruiting USA 0
NCT04629209 Phase II ONC201 A Phase II, Open Label Study of ONC201 in Adults With EGFR-low Glioblastoma Withdrawn USA 0
NCT02420795 Phase Ib/II ONC201 Study of Oral ONC201 in Patients With Relapsed/Refractory Non-Hodgkin's Lymphoma Terminated USA 0
NCT02324621 Phase I ONC201 Continuation of Oral ONC201 in Treating Patients With Advanced Solid Tumors Completed USA 0


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