Therapy Detail

Contact

Missing content? – Request curation!

Request curation for specific Genes, variants, or PubMed publications.

Have questions, comments or suggestions? - Let us know!

Email us at : ckbsupport@jax.org

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 (FDA.gov).

Filtering

  • Case insensitive filtering will display rows where any text in any cell matches the filter term
  • Simple literal full or partial string matches
  • Separate multiple filter terms with a spaces, order doesn't matter (a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page, filtering has no impact on query parameters
  • Use quotes to match a longer phrase which contains spaces "mtor c1483f"

Sorting

  • Generally, the default sort order for tables is set to be first column ascending, however, specific tables may set a different default sort order.
  • Click on any column header arrows to sort by that column
  • Hold down the Shift key and click multiple columns to sort by more than one column, be sure to set ascending or descending order for a given column, before moving on to the next column.

Drug Name Trade Name Synonyms Drug Classes Drug Description
Ponatinib Iclusig AP24534 BCR-ABL Inhibitor 24 DDR1 Inhibitor 8 DDR2 inhibitor 7 FGFR Inhibitor (Pan) 18 FLT3 Inhibitor 53 KIT Inhibitor 50 PDGFR-alpha Inhibitor 9 RET Inhibitor 39 SRC Inhibitor 28 VEGFR Inhibitor (Pan) 32 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 (FDA.gov).

Filtering

  • Case insensitive filtering will display rows where any text in any cell matches the filter term
  • Simple literal full or partial string matches
  • Separate multiple filter terms with a spaces, order doesn't matter (a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page, filtering has no impact on query parameters
  • Use quotes to match a longer phrase which contains spaces "mtor c1483f"

Sorting

  • Generally, the default sort order for tables is set to be first column ascending, however, specific tables may set a different default sort order.
  • Click on any column header arrows to sort by that column
  • Hold down the Shift key and click multiple columns to sort by more than one column, be sure to set ascending or descending order for a given column, before moving on to the next column.

Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
RET rearrange thyroid gland papillary 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
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 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
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
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 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 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
FGFR2 - SHTN1 FGFR2 L617M cholangiocarcinoma sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, cholangiocarcinoma cells expressing both FGFR2-SHTN1 and FGFR2 E565A were sensitive to treatment with Iclusig (ponatinib) in culture, demonstrating reduced cell viability (PMID: 31911531). 31911531
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
KIT K550_W557del Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, transformed cells expressing KIT K550_W557del demonstrated sensitivity to treatment with Iclusig (ponatinib) in culture, resulting in reduced cell viability (PMID: 25239608). 25239608
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
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
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
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
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
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 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
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 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
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...
DDR2 L239R Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 L239R in culture (PMID: 22328973). 22328973
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
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 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
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
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
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
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 - TACC3 lung adenocarcinoma sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatanib) inhibited growth and FGFR3 phosphorylation in cells expressing FGFR3-TACC3 fusion in culture (PMID: 25294908). 25294908
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
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
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
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
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
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
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 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
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
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
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
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 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
Unknown unknown glioblastoma multiforme not applicable Ponatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Iclusig (ponatinib) induced apoptosis and inhibited growth in glioblastoma cells in culture and in cell line xenograft models (PMID: 25378936). 25378936
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
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
DDR2 G774V Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 G774V in culture (PMID: 22328973). 22328973
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
DDR2 I638F lung squamous cell carcinoma sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited proliferation of lung squamous cell carcinoma cells harboring a DDR2 I638F mutation in cell culture (PMID: 22328973). 22328973
FGFR2 - SHTN1 Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing FGFR2-SHTN1 were sensitive to treatment with Iclusig (ponatinib), demonstrating decreased cell viability in culture (PMID: 31911531). 31911531
DDR2 L63V Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 L63V in culture (PMID: 22328973). 22328973
FGFR2 - SHTN1 FGFR2 E565A cholangiocarcinoma sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, cholangiocarcinoma cells expressing both FGFR2-SHTN1 and FGFR2 E565A were sensitive to treatment with Iclusig (ponatinib) in culture, demonstrating reduced cell viability (PMID: 31911531). 31911531
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
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 exon11 gastrointestinal stromal tumor predicted - sensitive Ponatinib Phase II Actionable In a Phase II trial, Iclusig (ponatinib) treatment resulted in partial response in 7% (2/27), stable disease in 59% (16/27), a median progression free survival of 4.3 months and a median overall survival of 15.0 months in gastrointestinal stromal tumor patients harboring KIT exon 11 mutations after prior tyrosine kinase inhibitor treatment failure (J Clin Oncol 33, 2015 (suppl; abstr 10535)). detail...
FGFR2 - CLIP1 Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) treatment inhibited viability of a transformed cell line expressing FGFR2-CLIP1 in culture (PMID: 31371345). 31371345
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
FGFR2 - SHTN1 FGFR2 E565A Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing both FGFR2-SHTN1 and FGFR2 E565A were sensitive to treatment with Iclusig (ponatinib) in culture, demonstrating reduced cell viability (PMID: 31911531). 31911531
Unknown unknown chronic myeloid leukemia not applicable Ponatinib Clinical Study Actionable In a meta-analysis, Iclusig (ponatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 4.95 [0.97-25.19]), but not improved overall survival (OR: 2.00 [0.21-19.33]), and was associated with increased risk of vascular occlusive events (OR: 3.47 [1.23-9.78]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown chronic myeloid leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). 23935038 detail...
PDGFRA amp lung small cell carcinoma sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of lung squamous cell cancer cells with PDGFRA amplification in culture (PMID: 22328973). 22328973
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
Unknown unknown gastrointestinal stromal tumor not applicable Ponatinib Phase II Actionable In a Phase II trial, Iclusig (ponatinib) demonstrated preliminary activity in patients with advanced gastrointestinal stromal tumor (J Clin Oncol (Meeting Abstracts) 2014 32: 10506). detail...
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
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
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
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
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
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 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 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
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 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 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
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
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
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
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
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
RET Y791F Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited phosphorylation of Ret in transformed human cells expressing RET Y971F in culture (PMID: 23526464). 23526464
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
ERBB2 over exp PIK3CA H1047R SRC over exp urinary bladder cancer no benefit Ponatinib Preclinical - Pdx Actionable In a preclinical study, treatment with Iclusig (ponatinib) was not effective in inhibiting tumor growth in a patient-derived xenograft (PDX) model of bladder cancer with SRC over expression, which also over expressed ERBB2 (HER2) and harbored PIK3CA H1047R (PMID: 26270481). 26270481
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
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
DDR2 G253C Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 G253C in culture (PMID: 22328973). 22328973
DDR2 G505S Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 G505S in culture (PMID: 22328973). 22328973
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
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
FGFR2 - SHTN1 FGFR2 L617M Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing both FGFR2-SHTN1 and FGFR2 L617M were sensitive to treatment with Iclusig (ponatinib) in culture, demonstrating reduced cell viability (PMID: 31911531). 31911531
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
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
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
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 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
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 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
Unknown unknown lymphoid leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). 23935038 detail...
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...
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
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
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
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
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 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
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
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
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
FGFR2 - TACC3 Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited downstream phosphorylation of Akt and Erk1/2 in cells expressing the fusion, FGFR2 - TACC3 (PMID: 26048680). 26048680
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
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
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 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
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
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
DDR2 I638F Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing DDR2 I638F in culture (PMID: 22328973). 22328973
FGFR2 - CLIP1 FGFR2 N549H Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) treatment inhibited viability of a transformed cell line expressing both FGFR2-CLIP1 and FGFR2 N549H in culture (PMID: 31371345). 31371345
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
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
FGFR2 - SHTN1 cholangiocarcinoma sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, cholangiocarcinoma cells expressing FGFR2-SHTN1 were sensitive to treatment with Iclusig (ponatinib), demonstrating decreased cell viability in culture (PMID: 31911531). 31911531
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
DDR2 L239R lung squamous cell carcinoma sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of lung squamous cell cancer cells harboring DDR2 L239R in culture (PMID: 22328973). 22328973

Filtering

  • Case insensitive filtering will display rows where any text in any cell matches the filter term
  • Simple literal full or partial string matches
  • Separate multiple filter terms with a spaces, order doesn't matter (a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page, filtering has no impact on query parameters
  • Use quotes to match a longer phrase which contains spaces "mtor c1483f"

Sorting

  • Generally, the default sort order for tables is set to be first column ascending, however, specific tables may set a different default sort order.
  • Click on any column header arrows to sort by that column
  • Hold down the Shift key and click multiple columns to sort by more than one column, be sure to set ascending or descending order for a given column, before moving on to the next column.

Clinical Trial Phase Therapies Title Recruitment Status
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 Recruiting
NCT02478164 Phase II Ponatinib Trial of Ponatinib in Patients With Bevacizumab-Refractory Glioblastoma Completed
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. Recruiting
NCT01838642 Phase II Ponatinib Ponatinib for Advanced Medullary Thyroid Cancer Terminated
NCT03147612 Phase II Mesna Vincristine Dexamethasone Rituximab Filgrastim Ponatinib Cyclophosphamide Methotrexate Cytarabine Study of the Combination of Low-Intensity Chemotherapy and Ponatinib in Patients With Philadelphia Chromosome (Ph)-Positive and/or BCR-ABL Positive Acute Lymphoblastic Leukemia (ALL) Recruiting
NCT01620216 Phase II Sunitinib Dasatinib Nilotinib Ponatinib Sorafenib Validation of an in Vitro Assay to Predict Targeted Therapies for Acute Leukemia Patients Unknown status
NCT01570868 Phase II Ponatinib Ponatinib - Frontline for Chronic Myeloid Leukemia (CML) in Accelerated Phase (AP) Terminated
NCT01874665 Phase II Ponatinib A Phase 2 Trial of Ponatinib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor Completed
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
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) Recruiting
NCT01888562 Phase I Ponatinib Ponatinib in the Treatment of FGFR Mutation Positive Recurrent or Persistent Endometrial Carcinoma Withdrawn
NCT02265341 Phase II Ponatinib Ponatinib Hydrochloride in Treating Patients With Advanced Biliary Cancer With FGFR2 Fusions Completed
NCT01935336 Phase II Ponatinib Study of Ponatinib in Patients With Lung Cancer Preselected Using Different Candidate Predictive Biomarkers Active, not recruiting
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
NCT01813734 Phase II Ponatinib Ponatinib in Advanced NSCLC w/ RET Translocations Completed


Additional content available in CKB BOOST