Therapy Detail
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| Therapy Name | Pexidartinib |
| Synonyms | |
| Therapy Description |
Turalio (pexidartinib) inhibits multiple receptor tyrosine kinases, including KIT, CSF1R, FLT3, and FLT3/ITD, which may inhibit growth in cancer cells (PMID: 22294205, PMID: 25993548, PMID: 25847190). Turalio (pexidartinib) is FDA approved for use in patients with symptomatic tenosynovial giant cell tumor (FDA.gov). |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Pexidartinib | Turalio | PLX3397|CML-261 | CSF1R Inhibitor 28 FLT3 Inhibitor 69 KIT Inhibitor 57 | Turalio (pexidartinib) inhibits multiple receptor tyrosine kinases, including KIT, CSF1R, FLT3, and FLT3/ITD, which may inhibit growth in cancer cells (PMID: 22294205, PMID: 25993548, PMID: 25847190). Turalio (pexidartinib) is FDA approved for use in patients with symptomatic tenosynovial giant cell tumor (FDA.gov). |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| FLT3 exon 14 ins FLT3 D835V | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D835V were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 act mut | leukemia | sensitive | Pexidartinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PLX3397 inhibited FLT3 autophosphorylation in leukemia cells overexpressing FLT3 or harboring FLT3 activating mutations, and inhibited growth of leukemia cells harboring FLT3-ITD mutations in culture and in cell line xenograft models (ASH Annual Meeting Abstracts 2011 118: 3632). | detail... |
| FLT3 exon 14 ins FLT3 D839N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D839N were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 N676K | acute myeloid leukemia | predicted - resistant | Pexidartinib | Case Reports/Case Series | Actionable | In a Phase I/II trial, an acute myeloid leukemia patient harboring a FLT3-ITD was found to have acquired FLT3 N676K following relapse on Turalio (pexidartinib) treatment (PMID: 34103301; NCT01349049). | 34103301 |
| FLT3 exon 14 ins FLT3 F691L FLT3 G846R | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/G846R mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 R845G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 R845G were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D835N were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D835G mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D839H | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D839H were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 Y842C | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/Y842C mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 Y842H | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/Y842H mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L | Advanced Solid Tumor | sensitive | Pexidartinib | Preclinical | Actionable | In a preclinical study, PLX3397 inhibited growth of transformed cells expressing a compound FLT3 F691L/FLT3-ITD mutation in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins | leukemia | sensitive | Pexidartinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PLX3397 inhibited proliferation of human leukemia cells harboring FLT3-ITD in culture and in cell line xenograft models (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 M837_D839delinsGRH | leukemia | predicted - resistant | Pexidartinib | Case Reports/Case Series | Actionable | In a clinical case study, a leukemia patient with FLT3-ITD positive leukemia initially responded to Pexidartinib (PLX3397), but experienced relapse after emergence of a compound FLT3 M837_D839delinsGRH mutation on the FLT3-ITD allele (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 M664I | Advanced Solid Tumor | decreased response | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 M664I displayed reduced sensitivity to PLX3397 relative to cells expressing FLT3-ITD in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 M664I FLT3 F691L | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/M664I mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins | Advanced Solid Tumor | sensitive | Pexidartinib | Preclinical | Actionable | In a preclinical study, PLX3397 inhibited proliferation of transformed cells expressing FLT3-ITD in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 N676S FLT3 F691L | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/N676S mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| KIT act mut | gastrointestinal stromal tumor | sensitive | Pexidartinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PLX3397 inhibited growth of gastrointestinal stromal tumor cell lines harboring KIT activating mutations in culture and in cell line xenograft models (PMID: 24583793). | 24583793 |
| FLT3 exon 14 ins FLT3 D698N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D698N were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835Y | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D835Y mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 S652G | leukemia | predicted - resistant | Pexidartinib | Case Reports/Case Series | Actionable | In a clinical case study, a patient with FLT3-ITD positive leukemia initially responded to Pexidartinib (PLX3397), but experienced relapse after emergence of a FLT3 S652G mutation on the same allele as FLT3-ITD (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835E | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a FLT3 ITD with D835E and F691L were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins | acute myeloid leukemia | predicted - sensitive | Pexidartinib | Phase Ib/II | Actionable | In a Phase I/II trial, treatment with Turalio (pexidartinib) was well-tolerated and resulted in an overall response rate of 21% (19/90) and a composite complete remission (CRc) rate of 11% (10/90) in patients with relapsed/refractory acute myeloid leukemia harboring a FLT3-ITD mutation (PMID: 32330242; NCT01349049). | 32330242 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D835N mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835H | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, FLT3 D835H conferred resistance to PLX3397 in transformed cells when expressed as a compound mutation with FLT3-ITD in culture (PMID: 25847190). | 25847190 |
| FLT3 D835V | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3 D835V were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835H | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D835H mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 R834Q | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 R834Q were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 N841K | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/N841K mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 R845G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/R845G mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D698N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D698N mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L | leukemia | sensitive | Pexidartinib | Preclinical | Actionable | In a preclinical study, PLX3397 inhibited proliferation of human leukemia cells harboring both FLT3-ITD and FLT3 F691L in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835Y | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, FLT3 D835Y conferred resistance to PLX3397 when expressed in a compound mutation with FLT3-ITD in cell culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 Y842S | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 Y842S were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835E | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D835E were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 D835Y | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3 D835Y were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835Y | acute myeloid leukemia | predicted - resistant | Pexidartinib | Case Reports/Case Series | Actionable | In a Phase I/II trial, an acute myeloid leukemia patient harboring a FLT3-ITD was found to have acquired FLT3 D835Y following relapse on Turalio (pexidartinib) treatment (PMID: 34103301; NCT01349049). | 34103301 |
| FLT3 exon 14 ins FLT3 F691L FLT3 Y842S | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/Y842S mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 G846R | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 G846R were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D839G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, FLT3 D839G conferred resistance to PLX3397 when expressed in a compound mutation with FLT3-ITD in cell culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835F | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D835F were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D839G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD /F691L/D839G mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D839A | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D839A were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835Y | leukemia | predicted - resistant | Pexidartinib | Case Reports/Case Series | Actionable | In a clinical case study, a patient with FLT3-ITD positive leukemia initially responded to Pexidartinib (PLX3397) therapy, but experienced relapse after emergence of FLT3 D835Y on the same allele as the FLT3-ITD mutation (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 Y842H | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 Y842H were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 R834Q | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/R834Q mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835H | leukemia | resistant | Pexidartinib | Clinical Study | Actionable | In a clinical study, a patient with FLT3-ITD positive leukemia initially responded to PLX3397, but experienced relapse after emergence of a FLT3 D835H mutation on the same allele as the FLT3-ITD mutation (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 D835G | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 D835G were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D839A | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D839A mutation were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 N676S | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD with FLT3 N676S were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D839N | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a compound FLT3-ITD/F691L/D839N were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 N841K | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD along with FLT3 N841K were resistant to PLX3397 in culture (PMID: 25847190). | 25847190 |
| FLT3 exon 14 ins FLT3 Y842C | Advanced Solid Tumor | resistant | Pexidartinib | Preclinical | Actionable | In a preclinical study, FLT3 Y842C conferred resistance to PLX3397 when expressed in a compound mutation with FLT3-ITD in cell culture (PMID: 25847190). | 25847190 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| NCT01596751 | Phase Ib/II | Eribulin Pexidartinib | Phase Ib/II Study of PLX 3397 and Eribulin in Patients With Metastatic Breast Cancer | Completed | USA | 0 |
| NCT02472275 | Phase I | Leuprolide Pexidartinib Goserelin Degarelix | PLX3397, Radiation Therapy, and Antihormone Therapy in Treating Patients With Intermediate- or High-Risk Prostate Cancer | Completed | USA | 0 |
| NCT02390752 | Phase Ib/II | Pexidartinib | PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) | Recruiting | USA | 0 |
| NCT02371369 | Phase III | Pexidartinib | PLX3397 Phase 3 Study for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS) | Completed | USA | POL | NLD | ITA | HUN | GBR | FRA | ESP | DNK | DEU | CAN | AUS | 0 |
| NCT01004861 | Phase I | Pexidartinib | Safety Study of PLX108-01 in Patients With Solid Tumors | Completed | USA | 0 |
| NCT01790503 | Phase Ib/II | Pexidartinib Temozolomide | A Phase 1b/2 Study of PLX3397 + Radiation Therapy + Temozolomide in Patients With Newly Diagnosed Glioblastoma | Completed | USA | 0 |
| NCT04526704 | FDA approved | Pexidartinib | Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib (PLX3397) | Completed | USA | NLD | ITA | HUN | ESP | AUS | 1 |
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