Therapy Detail
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| Therapy Name | Ponatinib |
| Synonyms | |
| Therapy Description |
Iclusig (ponatinib) inhibits the Bcr-Abl fusion, and other tyrosine kinases, such as RET, DDR, VEGFR, FGFR, KIT, and FLT3 (PMID: 23526464, PMID: 25284748, PMID: 19878872). Iclusig (ponatinib) is FDA-approved for chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL) as well as ABL1 T315I CML and ABL1 T315I Ph-positive ALL, and in combination with chemotherapy for newly diagnosed Ph-positive ALL (FDA.gov). |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Ponatinib | Iclusig | AP24534 | ABL Inhibitor (pan) 9 BCR-ABL Inhibitor 32 DDR1 Inhibitor 10 DDR2 inhibitor 7 FGFR Inhibitor (Pan) 26 FLT3 Inhibitor 69 KIT Inhibitor 57 PDGFR-alpha Inhibitor 9 RET Inhibitor 53 SRC Inhibitor 31 VEGFR Inhibitor (Pan) 36 | Iclusig (ponatinib) inhibits the Bcr-Abl fusion, and other tyrosine kinases, such as RET, DDR, VEGFR, FGFR, KIT, and FLT3 (PMID: 23526464, PMID: 25284748, PMID: 19878872). Iclusig (ponatinib) is FDA-approved for chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL) as well as ABL1 T315I CML and ABL1 T315I Ph-positive ALL, and in combination with chemotherapy for newly diagnosed Ph-positive ALL (FDA.gov). |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| KIT D816V | Advanced Solid Tumor | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) did not inhibit growth of transformed cells expressing KIT D816V in culture (PMID: 22301675). | 22301675 |
| RET E768D | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET E768D in culture (PMID: 23526464). | 23526464 |
| FLT3 Q575del | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) decreased proliferation in transformed cells expressing FLT3 Q575del in culture (PMID: 33914060). | 33914060 |
| FGFR2 N550S | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 N550S (PMID: 23908597). | 23908597 |
| FGFR1 act mut | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited receptor phosphorylation and cell growth in transformed cells expressing constitutively active FGFR1 in culture (PMID: 22238366). | 22238366 |
| RET C634W | thyroid gland carcinoma | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited RET signaling and decreased growth of thyroid carcinoma cells harboring a RET C634W mutation in culture and in cell line xenograft models (PMID: 23526464). | 23526464 |
| FGFR3 N540K | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 N540K in culture (PMID: 28034880). | 28034880 |
| KIT exon9 | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing a KIT exon 9 mutation demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). | 25239608 |
| FLT3 D835K FLT3 D835L | hematologic cancer | no benefit | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) did not inhibit viability of transformed cells expressing FLT3 D835L and D835K in culture (PMID: 28077790). | 28077790 |
| RET act mut | lung non-small cell carcinoma | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited RET phosphorylation and reduced viability of non-small cell lung cancer cells with RET activating mutations (Cancer Res April 15, 2013 73; 2084). | detail... |
| EML4 - RET | lung adenocarcinoma | predicted - sensitive | Ponatinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) treatment induced apoptosis in a patient-derived lung adenocarcinoma cell line harboring EML4-RET in culture (PMID: 33795352). | 33795352 |
| KIT exon11 | gastrointestinal stromal tumor | predicted - sensitive | Ponatinib | Phase II | Actionable | In a Phase II trial, Iclusig (ponatinib) treatment in gastrointestinal stromal tumor patients with KIT exon 11 mutations who failed prior therapy versus those without KIT exon 11 mutations led to a 16-week clinical benefit rate of 36% (10/28; 1 partial response (PR), 9 stable disease (SD)) vs 20% (3/15; 3 SD), an objective response rate of 7% (2/28; 2 PR) vs 0% (0/15), and median progression-free survival of 4.0 vs 2.0 mo and overall survival of 14.7 vs 14.3 mo, respectively (PMID: 35091442; NCT01874665). | 35091442 |
| FLT3 exon 14 ins | acute myeloid leukemia | sensitive | Ponatinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited viability of patient derived acute myeloid leukemia cells harboring FLT3 ITD mutations in culture, and inhibited growth of tumors in cell line xenograft models (PMID: 21482694). | 21482694 |
| FLT3 exon 14 ins | acute myeloid leukemia | sensitive | Ponatinib | Phase I | Actionable | In a Phase I trial, Iclusig (ponatinib) resulted in a 25% (3/12) overall response rate, indicated as partial remission or better, in acute myeloid leukemia patients harboring FLT3-ITD (PMID: 23691988). | 23691988 |
| ABL1 rearrange | myeloid neoplasm | sensitive | Ponatinib | Guideline | Actionable | Iclusig (ponatinib) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an ABL1 rearrangement (NCCN.org). | detail... |
| FGFR2 amp | stomach cancer | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of FGFR2-amplified gastric cancer cells in culture, and inhibited tumor growth in FGFR2-amplified gastric cancer cell line xenograft models (PMID: 22238366). | 22238366 |
| KIT W557_K558del KIT D820G | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT D820G were sensitive to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| KIT W557C KIT K558_V560del | gastrointestinal stromal tumor | predicted - sensitive | Ponatinib | Case Reports/Case Series | Actionable | In a clinical study, a GIST patient harboring KIT K558_V560del and KIT W557C, resistant to Gleevec (imatinib), Sutent (sunitinib), and Stivarga (regorafenib), demonstrated sensitivity when treated with Iclusig (ponatinib), which resulted in a radiologic response and stabilization for six months (PMID: 25239608). | 25239608 |
| RET rearrange | lung non-small cell carcinoma | no benefit | Ponatinib | Case Reports/Case Series | Actionable | In a retrospective analysis, multikinase inhibitor treatment demonstrated suboptimal clinical efficacy in RET-rearranged non-small cell lung cancer patients with measurable baseline brain metastasis, with a confirmed intracranial response rate of 18% (2/11); Iclusig (ponatinib) treatment resulted in no intracranial response in 4 patients with evaluable brain metastasis (PMID: 30017832). | 30017832 |
| KIT V560D | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing KIT V560D demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). | 25239608 |
| KIT W557_K558del KIT D816G | Advanced Solid Tumor | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT D816G were sensitive to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FGFR2 V565I | Advanced Solid Tumor | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FGFR2 V565I were resistant to treatment with Iclusig (ponatinib) (PMID: 23908597). | 23908597 |
| KIT W557_K558del KIT D820A | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT D820A were sensitive to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| SMARCA4 mutant | small-cell carcinoma of the ovary of hypercalcemic type | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) treatment of small cell carcinoma of the ovary, hypercalcemic type (SCCOHT) cell lines and xenografts with mutant SMARCA4 resulted in decreased cell viability and tumor volume (PMID: 29440177). | 29440177 |
| FGFR2 N549K | endometrial cancer | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of endometrial cancer cells harboring FGFR2 N549K in culture and in cell line xenograft models (PMID: 22238366). | 22238366 |
| FGFR2 N550K | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 N550K (PMID: 23908597). | 23908597 |
| RET L790F | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET L790F in culture (PMID: 23526464). | 23526464 |
| FGFR2 K660E | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing FGFR2 K660E in culture (PMID: 23908597). | 23908597 |
| FLT3 I836T | hematologic cancer | no benefit | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) did not inhibit viability of transformed cells expressing FLT3 I836T in culture (PMID: 28077790). | 28077790 |
| RET wild-type | lung small cell carcinoma | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of small cell lung carcinoma lines expressing wild-type RET (PMID: 25122427). | 25122427 |
| FGFR3 K650E | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). | 28034880 |
| FGFR2 amp | colon cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr phosphorylation and cell proliferation in colon cancer cells harboring FGFR2 amplification in culture (PMID: 22238366). | 22238366 |
| KIT W557_K558del KIT N822K | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT N822K were sensitive to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FLT3 S574del | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) decreased proliferation in transformed cells expressing FLT3 S574del in culture (PMID: 33914060). | 33914060 |
| FLT3 Y572del | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) decreased proliferation in transformed cells expressing FLT3 Y572del in culture (PMID: 33914060). | 33914060 |
| FGFR3 S249C | urinary bladder cancer | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of bladder cancer cells harboring FGFR3 S249C in culture and in cell line xenograft models (PMID: 22238366). | 22238366 |
| FGFR1 amp | breast cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr phosphorylation and cell proliferation in ER-positive breast cancer cells harboring FGFR1 amplification in culture (PMID: 22238366). | 22238366 |
| FGFR2 W290C | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing FGFR2 W290C in culture (PMID: 23786770). | 23786770 |
| FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Ponatinib | Guideline | Actionable | Iclusig (ponatinib) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an FGFR1 rearrangement (NCCN.org). | detail... |
| RET V804L | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed human cell lines expressing RET V804L demonstrated sensitivity to Iclusig (ponatinib) (PMID: 23811235). | 23811235 |
| KIT A829P | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT A829P demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| KIT W557_K558del KIT T670I | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT T670I demonstrated sensitivity to treatment with Iclusig (ponatinib) in both culture and cell line xenograft models, resulting in reduced cell viability and decreased tumor volume (PMID: 25239608). | 25239608 |
| FGFR3 S249C | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing FGFR3 S249C in culture (PMID: 23786770). | 23786770 |
| FGFR2 E566G | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 E566G (PMID: 23908597). | 23908597 |
| FLT3 E573del | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) decreased proliferation in transformed cells expressing FLT3 E573del in culture (PMID: 33914060). | 33914060 |
| FGFR2 L618M | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FGFR2 L618M were sensitive to treatment with Iclusig (ponatinib) (PMID: 23908597). | 23908597 |
| KIT D820E | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT D820E demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FGFR1 amp | Ewing sarcoma | predicted - sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of Ewing’s sarcoma cell lines with FGFR1 copy number gain in culture (PMID: 26179511). | 26179511 |
| FGFR1 V561M | Advanced Solid Tumor | predicted - resistant | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, cells expressing FGFR1 V561M demonstrated insensitivity to Iclusig (ponatinib) in culture (PMID: 34114373). | 34114373 |
| KIT D816H | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT D816H demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FLT3 D835G | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835G in culture (PMID: 28077790). | 28077790 |
| FGFR2 M536I | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 M536I (PMID: 23908597). | 23908597 |
| KIT T670I | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT T670I demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FLT3 I836D FLT3 I836L | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 I836D and I836L in culture (PMID: 28077790). | 28077790 |
| FGFR2 I548V | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 I548V (PMID: 23908597). | 23908597 |
| FGFR3 Y375C | urinary bladder cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr phosphorylation and cell proliferation in bladder cancer cells harboring FGFR3 Y375C in culture (PMID: 22238366). | 22238366 |
| KIT W557_K558del KIT V654A | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT V654A demonstrated sensitivity to treatment with Iclusig (ponatinib) in both culture and cell line xenograft models, resulting in reduced cell viability and decreased tumor volume (PMID: 25239608). | 25239608 |
| RET M918T | thyroid gland carcinoma | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited RET signaling and decreased proliferation of thyroid carcinoma cells harboring a RET M918T mutation in culture (PMID: 23526464). | 23526464 |
| FGFR1 wild-type | breast cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, ER-positive breast cancer cells with wild-type FGFR1 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| FGFR2 amp | breast cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr phosphorylation and cell proliferation in ER-negative breast cancer cells harboring FGFR2 amplification in culture (PMID: 22238366). | 22238366 |
| RET C634Y | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells harboring RET C634Y in culture (PMID: 23526464). | 23526464 |
| RET mutant | colorectal cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) demonstrated efficacy in RET mutant positive colorectal cancer cell lines (PMID: 23811235). | 23811235 |
| FGFR3 amp | Advanced Solid Tumor | predicted - sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of several tumor cell lines with FGFR alterations in culture (Cancer Res April 15, 2011 71:3560). | detail... |
| RET M918T | lung small cell carcinoma | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of small cell lung carcinoma lines expressing RET M918T (PMID: 25122427). | 25122427 |
| FGFR3 V555M | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 V555M in culture (PMID: 28034880). | 28034880 |
| KIT W557_K558del KIT D816H | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT D816H demonstrated sensitivity to treatment with Iclusig (ponatinib) in both culture and cell line xenograft models, resulting in reduced cell viability and tumor regression (PMID: 25239608). | 25239608 |
| RET Y806C | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET Y806C in culture (PMID: 23526464). | 23526464 |
| RET S891A | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET S891A in culture (PMID: 23526464). | 23526464 |
| KIT W557_K558del | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, transformed cells expressing KIT W557_K558del demonstrated sensitivity to treatment with Iclusig (ponatinib) in both culture and cell line xenograft models, resulting in reduced cell viability and near complete tumor regression (PMID: 25239608). | 25239608 |
| RET C634R | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed human cell lines expressing RET C634R demonstrated sensitivity to Iclusig (ponatinib) (PMID: 23811235). | 23811235 |
| RET mutant | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of cancer cell lines harboring RET mutations in cultured and in cell line xenograft models (PMID: 23526464). | 23526464 |
| FGFR3 act mut | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited receptor phosphorylation and cell growth in transformed cells expressing constitutively active FGFR3 in culture (PMID: 22238366). | 22238366 |
| KIT V560_Y578del | gastrointestinal stromal tumor | sensitive | Ponatinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, a patient-derived gastrointestinal stromal tumor (GIST) cell line harboring KIT V560_Y578del demonstrated sensitivity to Iclusig (ponatinib) in culture (PMID: 25239608). | 25239608 |
| FGFR2 wild-type | colon cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, colon cancer cells with wild-type FGFR2 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| FGFR3 wild-type | urinary bladder cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, bladder cancer cells with wild-type FGFR3 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| KIT K558delinsNP | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing KIT K558delinsNP demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). | 25239608 |
| FGFR3 L608V | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 L608V in culture (PMID: 28034880). | 28034880 |
| KIT V559D | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT V559D demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| KIT W557_K558del KIT A829P | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells co-expressing KIT W557_K558del and KIT A829P were sensitive to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FGFR2 K660N | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing FGFR2 K660N in culture (PMID: 23786770). | 23786770 |
| KIT V560G KIT D816V | mast-cell leukemia | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of a human mast cell line harboring both KIT V560G and KIT D816V in culture (PMID: 24552773). | 24552773 |
| FGFR1 amp | lung squamous cell carcinoma | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited growth of squamous cell lung cancer cell lines harboring FGFR1 amplification (PMID: 22238366). | 22238366 |
| KIT P551_K558del | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing KIT P551_K558del (reported as K550_W557del) demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). | 25239608 |
| KIT P551_E554del | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing KIT P551_E554del demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). | 25239608 |
| FGFR2 W290_I291delinsC | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited downstream phosphorylation of Akt and Erk1/2 in cells expressing Fgfr2 W290_I291delinsC (PMID: 26048680). | 26048680 |
| FGFR2 wild-type | breast cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, ER-negative breast cancer cells with wild-type FGFR2 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| KIT V560_L576del | gastrointestinal stromal tumor | sensitive | Ponatinib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, a patient derived GIST cell line harboring KIT V560_L576del demonstrated sensitivity to Iclusig (ponatinib) in both culture and PDX models, resulting in phosphorylation inhibition of Akt, Kit, and Erk (PMID: 25239608). | 25239608 |
| RET E884K | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET E884K in culture (PMID: 23526464). | 23526464 |
| FGFR2 S252W | endometrial cancer | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and cell proliferation in endometrial cancer cells harboring FGFR2 S252W in culture (PMID: 22238366). | 22238366 |
| KIT M552_K558del | gastrointestinal stromal tumor | predicted - resistant | Ponatinib | Case Reports/Case Series | Actionable | In a clinical study, a GIST patient harboring KIT M552_K558del, resistant to multiple tyrosine kinase inhibitors, demonstrated resistance to Iclusig (ponatinib) treatment as indicated by disease progression after 4 weeks of treatment (PMID: 25239608). | 25239608 |
| FGFR2 act mut | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited receptor phosphorylation and cell growth in transformed cells expressing constitutively active FGFR2 in culture (PMID: 22238366). | 22238366 |
| FGFR2 S267_D273dup | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited downstream phosphorylation of Akt and Erk1/2 in cells expressing Fgfr2 S267_D273dup (PMID: 26048680). | 26048680 |
| KIT V654A | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT V654A demonstrated some sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| KIT N822K | leukemia | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Kit signaling and viability in leukemia cells harboring KIT N822K in culture (PMID: 21482694). | 21482694 |
| FGFR2 N550H | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Fgfr2 phosphorylation and proliferation in transformed cells expressing FGFR2 N550H (PMID: 23908597). | 23908597 |
| RET M918T | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET M918T in culture (PMID: 23526464). | 23526464 |
| KIT W557_K558del KIT Y823D | gastrointestinal stromal tumor | sensitive | Ponatinib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, a patient derived GIST cell line co-harboring KIT W557_K558del and KIT Y823D demonstrated sensitivity to Iclusig (ponatinib) in a PDX model, resulting in complete tumor regression (PMID: 25239608). | 25239608 |
| RET A883F | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET A883F in culture (PMID: 23526464). | 23526464 |
| FGFR2 wild-type | endometrial cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, endometrial cancer cells with wild-type FGFR2 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| RET V804M | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed human cell lines expressing RET V804M demonstrated sensitivity to Iclusig (ponatinib) (PMID: 23811235). | 23811235 |
| KIT V560G | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, cells expressing KIT V560G demonstrated sensitivity to Iclusig (ponatinib) in an in vitro kinase assay (PMID: 25239608). | 25239608 |
| FGFR2 M538I | Advanced Solid Tumor | decreased response | Ponatinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing FGFR2 M538I demonstrated a decreased response to treatment with Iclusig (ponatinib) (PMID: 23908597). | 23908597 |
| RET Y791F | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical - Biochemical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET Y791F in culture (PMID: 23526464). | 23526464 |
| RET D898V | Advanced Solid Tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET D898V in culture (PMID: 23526464). | 23526464 |
| KIT V560_Y578del KIT T670I | gastrointestinal stromal tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, a patient derived GIST cell line co-harboring KIT V560_Y578del and KIT T670I demonstrated sensitivity to treatment with Iclusig (ponatinib) (PMID: 25239608). | 25239608 |
| RET rearrange | papillary thyroid carcinoma | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, Iclusig (ponatinib) decreased proliferation of papillary thyroid carcinoma cells harboring the RET/PTC1 rearrangement in culture (PMID: 23526464). | 23526464 |
| KIT K642E | gastrointestinal stromal tumor | sensitive | Ponatinib | Preclinical | Actionable | In a preclinical study, a patient derived GIST cell line harboring KIT K642E demonstrated sensitivity to Iclusig (ponatinib) in culture (PMID: 25239608). | 25239608 |
| FLT3 D835N | hematologic cancer | sensitive | Ponatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Iclusig (ponatinib) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835N in culture (PMID: 28077790). | 28077790 |
| KIT P551_W557delinsL | gastrointestinal stromal tumor | predicted - sensitive | Ponatinib | Case Reports/Case Series | Actionable | In a clinical study, a GIST patient harboring KIT P551_W557delinsL, resistant to Gleevec (imatinib), Sutent (sunitinib), and Stivarga (regorafenib), demonstrated sensitivity to Iclusig (ponatinib) treatment, which resulted in tumor regression and stable disease (PMID: 25239608). | 25239608 |
| FGFR2 wild-type | stomach cancer | resistant | Ponatinib | Preclinical | Actionable | In a preclinical study, gastric cancer cells with wild-type FGFR2 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). | 22238366 |
| KIT exon9 | gastrointestinal stromal tumor | no benefit | Ponatinib | Phase II | Actionable | In a Phase II trial, Iclusig (ponatinib) treatment did not result in clinical benefit in gastrointestinal stromal tumor patients with KIT exon 9 mutations who had failed prior tyrosine kinase inhibitor treatment (PMID: 35091442; NCT01874665). | 35091442 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| NCT03690115 | Phase II | Ponatinib | Study of Ponatinib (Iclusig) for Prevention of Relapse After Allogeneic Stem Cell Transplantation (Allo-SCT) in FLT3-ITD AML Patients (PONALLO) | Active, not recruiting | FRA | 0 |
| NCT03838692 | Phase II | Ponatinib | Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer | Withdrawn | 0 | |
| NCT01935336 | Phase II | Ponatinib | Study of Ponatinib in Patients With Lung Cancer Preselected Using Different Candidate Predictive Biomarkers | Completed | USA | 0 |
| NCT01813734 | Phase II | Ponatinib | Ponatinib in Advanced NSCLC w/ RET Translocations | Completed | USA | 0 |
| NCT01838642 | Phase II | Ponatinib | Ponatinib for Advanced Medullary Thyroid Cancer | Terminated | USA | 0 |
| NCT02551718 | Phase I | Bosutinib Irinotecan Romidepsin Busulfan Melphalan Nilotinib Crizotinib Cytarabine Mitoxantrone Dasatinib Pazopanib Paclitaxel Clofarabine Hydroxyurea Tretinoin Carfilzomib Nelarabine Bexarotene Pentostatin Everolimus Cabozantinib Mercaptopurine Methotrexate Cladribine Thioguanine Daunorubicin Ponatinib Etoposide Afatinib Gefitinib Gemcitabine Regorafenib Arsenic trioxide Trametinib Imatinib Erlotinib Dabrafenib Decitabine Axitinib Azacitidine Ruxolitinib Fludarabine Lapatinib Ceritinib Sirolimus Sorafenib Lomustine Sunitinib Cabazitaxel Temsirolimus Topotecan Bortezomib Pralatrexate | High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia | Completed | USA | 0 |
| NCT05306301 | Phase II | Ponatinib | Ponatinib Plus Chemotherapy in Acute Lymphoblastic Leukemia Patients | Recruiting | ITA | 0 |
| NCT04591431 | Phase II | Everolimus Nivolumab Itacitinib Erlotinib Ponatinib Pemigatinib Ipatasertib Ipilimumab Cobimetinib Atezolizumab Palbociclib Entrectinib Vemurafenib Lapatinib Trastuzumab Brigatinib Alectinib Ado-trastuzumab emtansine Pertuzumab Vismodegib | The Rome Trial From Histology to Target: the Road to Personalize Target Therapy and Immunotherapy (ROME) | Active, not recruiting | ITA | 0 |
| NCT01888562 | Phase I | Ponatinib | Ponatinib in the Treatment of FGFR Mutation Positive Recurrent or Persistent Endometrial Carcinoma | Withdrawn | 0 | |
| NCT03147612 | Phase II | Pegfilgrastim Blinatumomab Vincristine Sulfate Rituximab Filgrastim Ponatinib Cyclophosphamide Methotrexate Cytarabine | Low-Intensity Chemotherapy, Ponatinib and Blinatumomab in Treating Patients With Philadelphia Chromosome-Positive and/or BCR-ABL Positive Acute Lymphoblastic Leukemia | Active, not recruiting | USA | 0 |
| NCT04530565 | Phase III | Cyclophosphamide + Dasatinib + Dexamethasone + Doxorubicin + Vincristine Sulfate Blinatumomab + Dexamethasone + Ponatinib + Prednisone Blinatumomab + Dasatinib + Dexamethasone + Prednisone Dasatinib Ponatinib Blinatumomab + Dexamethasone + Methotrexate + Ponatinib + Prednisone Blinatumomab + Dasatinib + Dexamethasone + Methotrexate + Prednisone Ponatinib + Prednisone Cyclophosphamide + Cytarabine + Dexamethasone + Doxorubicin + Mesna + Methotrexate + Vincristine Sulfate Cytarabine + Methotrexate + Ponatinib Cytarabine + Dasatinib + Methotrexate Cyclophosphamide + Dexamethasone + Doxorubicin + Ponatinib + Vincristine Sulfate Cyclophosphamide + Cytarabine + Dasatinib + Dexamethasone + Doxorubicin + Methotrexate + Vincristine Sulfate Cyclophosphamide + Cytarabine + Dexamethasone + Doxorubicin + Methotrexate + Ponatinib + Vincristine Sulfate Dasatinib + Prednisone | Testing the Use of Steroids and Tyrosine Kinase Inhibitors With Blinatumomab or Chemotherapy for Newly Diagnosed BCR-ABL-Positive Acute Lymphoblastic Leukemia in Adults | Recruiting | USA | ISR | 1 |
| NCT02478164 | Phase II | Ponatinib | Trial of Ponatinib in Patients With Bevacizumab-Refractory Glioblastoma | Completed | USA | 0 |
| NCT06061094 | Phase II | Blinatumomab + Ponatinib Imatinib Blinatumomab + Imatinib Ponatinib | Randomized Trial in Adult de Novo Ph Positive ALL With Chemotherapy, Imatinib or Ponatinib, Blinatumomab and SCT (GMALL-EVOLVE) | Recruiting | DEU | 0 |
| NCT01874665 | Phase II | Ponatinib | A Phase 2 Trial of Ponatinib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor | Completed | USA | 0 |
| NCT04043676 | Phase II | Ponatinib | Consolidation Treatment With Ponatinib 15 mg on Treatment Free-Remission Rate in Patients With Chronic Myeloid Leukemia | Unknown status | ESP | 0 |
| NCT05268003 | Phase II | Ponatinib Venetoclax | A Phase II Study of the Combination of Ponatinib With Mini-hyper CVD Chemotherapy and Venetoclax in Patients With Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia | Active, not recruiting | USA | 0 |
| NCT02272998 | Phase II | Ponatinib | Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT. | Active, not recruiting | USA | 0 |
| NCT03878524 | Phase I | Oxaliplatin Palbociclib Vemurafenib Sirolimus Tretinoin Celecoxib Ipilimumab Ruxolitinib Dasatinib Abiraterone Idelalisib Trametinib Imatinib Erlotinib Carboplatin Olaparib Panobinostat Bortezomib Afatinib Fluorouracil Vorinostat Pembrolizumab Leucovorin Enzalutamide Ponatinib Nivolumab Everolimus Sunitinib Cabazitaxel Cabozantinib Lenvatinib Pertuzumab Sorafenib Venetoclax Bevacizumab | A Personalized Medicine Study for Patients With Advanced Cancer of the Breast, Prostate, Pancreas or Those With Refractory Acute Myelogenous Leukemia (SMMART) | Terminated | USA | 0 |
| NCT02779283 | Phase I | Dasatinib Nilotinib Ponatinib Sorafenib Cytarabine + Idarubicin Sunitinib | Personalized Kinase Inhibitor Therapy Combined With Chemotherapy in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia | Completed | USA | 0 |
| NCT04475731 | Phase II | Ponatinib | Ponatinib in Adult Ph+ ALL Patients With MRD Positivity or Hematological Relapse | Active, not recruiting | ITA | 0 |
| NCT01746836 | Phase II | Ponatinib | Ponatinib Hydrochloride as Second Line Therapy in Treating Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib Mesylate, Dasatinib, or Nilotinib | Recruiting | USA | 0 |
| NCT03934372 | Phase Ib/II | Ponatinib | Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors | Recruiting | SWE | NLD | ITA | GBR | FRA | ESP | DNK | DEU | BEL | 0 |
| NCT02467270 | Phase II | Ponatinib | Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia (CML) to Characterize the Efficacy and Safety of a Range of Doses | Active, not recruiting | USA | SWE | POL | ITA | GBR | FRA | ESP | DNK | DEU | CZE | CHE | CAN | AUS | ARG | 7 |
| NCT04501614 | Phase Ib/II | Ponatinib | A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia | Completed | USA | POL | NLD | ITA | GBR | FRA | ESP | CZE | BRA | AUS | ARG | 4 |
| NCT04709731 | Phase II | Ponatinib | Cardiovascular Assessment of Ponatinib as Third Line Treatment in Chronic Phase Chronic Myeloid Leukemia (CarPAs) | Not yet recruiting | ITA | 0 |
| NCT01570868 | Phase II | Ponatinib | Ponatinib - Frontline for Chronic Myeloid Leukemia (CML) in Accelerated Phase (AP) | Terminated | USA | 0 |
| NCT01620216 | Phase II | Sunitinib Dasatinib Nilotinib Ponatinib Sorafenib | Validation of an in Vitro Assay to Predict Targeted Therapies for Acute Leukemia Patients | Terminated | USA | 0 |
| NCT02265341 | Phase II | Ponatinib | Ponatinib Hydrochloride in Treating Patients With Advanced Biliary Cancer With FGFR2 Fusions | Completed | USA | 0 |
| NCT03807479 | Phase II | Ponatinib | Study in Patients With Chronic Leukemia, Where Previous Therapy Failed, and Who Will be Treated With Ponatinib as Second Line Therapy (PONS) | Terminated | DEU | 0 |
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