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Profile Name RET V804L
Gene Variant Detail

RET V804L (gain of function)

Relevant Treatment Approaches RET Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Amuvatinib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I clinical trial, Amuvatinib (MP470) in combination with chemotherapeutic agents, demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24849582). 24849582
Unknown unknown ovarian cancer not applicable RET Inhibitor ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown acute myeloid leukemia not applicable RET Inhibitor ENMD-2076 Phase I Actionable In a Phase I trial, treatment with ENMD-2076 resulted in a 25% (4/16) overall response rate in patients with acute myeloid leukemia as well as three patients achieving a complete response (with incomplete count recovery) and one patient with a morphological leukemia free state (PMID: 27406088). 27406088
Unknown unknown fallopian tube cancer not applicable RET Inhibitor ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown peritoneum cancer not applicable RET Inhibitor ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown ovarian clear cell carcinoma no benefit RET Inhibitor ENMD-2076 Phase II Actionable In a Phase II trial, ENMD-2076 did not meet efficacy standard, resulted in partial response in 7.9% (3/38) and stable disease in 68.4% (26/38) of patients with recurrent ovarian clear cell carcinoma, with an overall 6-month progression-free survival rate of 22% (PMID: 30108107). 30108107
Unknown unknown lung non-squamous non-small cell carcinoma not applicable RET Inhibitor Carboplatin + Motesanib Diphosphate Phase III Actionable In a Phase III study, Motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown lung non-squamous non-small cell carcinoma no benefit RET Inhibitor Carboplatin + Motesanib Diphosphate + Paclitaxel Phase III Actionable In a Phase III trial, Motesanib (AMG 706) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) did not improve progression-free survival significantly compared to placebo (6.1 vs 5.6 months) in patient with non-squamous non-small cell lung cancer (PMID: 28902534). 28902534
Unknown unknown lung non-squamous non-small cell carcinoma not applicable RET Inhibitor Motesanib Diphosphate + Paclitaxel Phase III Actionable In a Phase III study, Motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Carboplatin + Lenvatinib + Paclitaxel Phase I Actionable In a Phase I trial of patients with advanced or metastatic NSCLC, treatment with Lenvima (lenvatinib) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) was tolerated and demonstrated anti-tumor activity (PMID: 23860537). 23860537
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Everolimus + Lenvatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). 26482279 detail...
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Everolimus + Lenvatinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Lenvima (lenvatinib) and Afinitor (everolimus) demonstrated safety, and resulted in partial response in 30% (6/20) of patients with metastatic renal cell carcinoma (PMID: 24190702). 24190702
Unknown unknown thyroid gland papillary carcinoma not applicable RET Inhibitor Lenvatinib Clinical Study Actionable In a clinical study, Lenvima (lenvatinib) treatment resulted in prolonged response in a papillary thyroid carcinoma patient whose disease progressed despite 3 prior therapies including Nexavar (sorafenib), Sutent (sunitinib), and Votrient (pazopanib) (PMID: 29167691). 29167691
Unknown unknown thyroid gland cancer not applicable RET Inhibitor Lenvatinib Phase II Actionable In a Phase II clinical trial, Lenvima (lenvatinib) demonstrated partial response in 36% (21/59) of patients, partial response or stable disease in 80% (47/59), and median progression free survival of 9 months in patients with advanced medullary thyroid cancer (PMID: 26311725). 26311725
Unknown unknown thyroid gland cancer not applicable RET Inhibitor Lenvatinib FDA approved Actionable In a Phase III trial (SELECT) that supported FDA approval, treatment with Lenvima (lenvatinib) improved progression free survival (18.3 vs 3.6 months, HR=0.21, p<0.001) and response rates (64.8% vs 1.5%, p<0.001) compared to placebo in patients with radioiodine-refractory differentiated thyroid cancer (PMID: 25671254; NCT01321554). 25671254 detail...
Unknown unknown melanoma not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Lenvatinib FDA approved Actionable In a Phase III trial (REFLECT) that supported FDA approval, Lenvima (lenvatinib) demonstrated activity comparable to Nexavar (sorafenib), resulted in a median survival time of 13.6 months in patients with unresectable hepatocellular carcinoma (PMID: 29433850; NCT01761266). detail... 29433850
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Lenvatinib Phase II Actionable In a Phase II trial, Lenvima (lenvatinib) treatment resulted in partial response in 37% (17/46) and stable disease in 41% (19/46) of patients with advanced hepatocellular carcinoma, and a median overall survival of 18.7 months (PMID: 27704266). 27704266
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) resulted in safety and preliminary efficacy in patients with hepatocellular carcinoma, demonstrating a partial response in 15% (3/20) of patients and tumor regression in 70% (14/20) (PMID: 26500236). 26500236
Unknown unknown colon cancer not applicable RET Inhibitor Lenvatinib Preclinical Actionable In a preclinical study, Lenvima (lenvatinib) induced apoptosis and inhibited proliferation of colorectal cancer cells in culture (PMID: 24255582). 24255582
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 22516948). 22516948
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) treatment resulted in partial response in 11.7% (9/77) and stable disease in 51.9% (40/77) of patients with advanced solid tumors (PMID: 26169970). 26169970
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). 26169970
Unknown unknown thyroid gland medullary carcinoma not applicable RET Inhibitor Lenvatinib Phase II Actionable In a Phase II trial, advanced medullary thyroid cancer patients experienced an objective response rate of 36% (21/59, all partial responses) and median progression free survival was 9 months when treated with Lenvima (lenvatinib) (PMID: 26311725). 26311725
Unknown unknown sarcoma not applicable RET Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and anti-tumor activity in patients with advanced solid tumors, including partial responses in patients with soft-tissue sarcoma (PMID: 22516948). 22516948
Unknown unknown stomach cancer not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase II Actionable In a Phase II trial (EPOC1706), the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) treatment in patients with either stomach cancer or gastroesophageal junction cancer resulted in a 69% (20/29) objective response rate, a disease control rate of 100% (29/29), a median progression-free survival of 7.1 months, and a median overall survival that had not yet been reached, and 8 patients were still experiencing an ongoing response at data cut-off (PMID: 32589866; NCT03609359). 32589866
Unknown unknown transitional cell carcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 25% (5/20) in patients with metastatic urothelial cancer, with a median duration of response not evaluable, and a median progression-free survival of 5.4 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown melanoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 48% (10/21) in patients with metastatic melanoma, with a median duration of response of 12.5 months, and a median progression-free survival of 5.5 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 70% (21/30) in patients with metastatic renal cell carcinoma, with a median duration of response of 20.0 months, and a median progression-free survival of 19.8 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown head and neck squamous cell carcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 46% (10/22) in patients with metastatic head and neck squamous cell carcinoma, with a median duration of response of 8.2 months, and a median progression-free survival of 4.7 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown endometrial cancer not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 52% (12/23) in patients with metastatic endometrial cancer, with a median duration of response not evaluable, and a median progression-free survival of 9.7 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase Ib/II Actionable In a Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 33% (7/21) in patients with metastatic non-small cell lung cancer, with a median duration of response of 10.9 months, and a median progression-free survival of 5.9 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown endometrial carcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab FDA approved Actionable In a Phase II trial (Study 111/KEYNOTE-146) that supported FDA approval, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment resulted in an objective response rate of 35.6% (16/45) in patients with endometrial carcinoma that was not MSI-H or dMMR (PMID: 30922731; NCT02501096). detail... detail... 30922731
Unknown unknown gastroesophageal junction adenocarcinoma not applicable RET Inhibitor Lenvatinib + Pembrolizumab Phase II Actionable In a Phase II trial (EPOC1706), the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) treatment in patients with either stomach cancer or gastroesophageal junction cancer resulted in a 69% (20/29) objective response rate, a disease control rate of 100% (29/29), a median progression-free survival of 7.1 months, and a median overall survival that had not yet been reached, and 8 patients were still experiencing an ongoing response at data cut-off (PMID: 32589866; NCT03609359). 32589866
Unknown unknown myelofibrosis not applicable RET Inhibitor Fedratinib FDA approved Actionable In a Phase III trial (JAKARTA) that supported FDA approval, Inrebic (fedratinib) demonstrated both clinical benefits and toxicity in myelofibrosis patients, resulting in symptom response rates at week 24 of 36% (33/91), 34% (31/91), and 7% (6/85) in the Fedratinib (SAR302503) 400 mg, 500 mg, and placebo groups, respectively (P<.001) (PMID: 26181658; NCT01437787). 26181658 detail...
Unknown unknown lymphoid leukemia not applicable RET Inhibitor 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...
Unknown unknown chronic myeloid leukemia not applicable RET Inhibitor 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...
Unknown unknown chronic myeloid leukemia not applicable RET Inhibitor 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 glioblastoma multiforme not applicable RET Inhibitor 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
Unknown unknown gastrointestinal stromal tumor not applicable RET Inhibitor 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...
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor RXDX-105 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) was tolerable and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2574)). detail...
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor RXDX-105 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including 2 patients with heavily pretreated non-small cell lung cancer that achieved stable disease for greater that 6 months (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C188). detail...
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Cetuximab + Regorafenib Phase I Actionable In a Phase I trial, the combination of Stivarga (regorafenib) and Erbitux (cetuximab) resulted in a clinical benefit of either stable disease or partial response in 46% (11/24) of advanced solid tumor patients, including eight patients with colorectal cancer, and one patient with head and neck cancer, one with carcinoma of unknown primary, and one with glioblastoma (PMID: 28422758; NCT02095054). 28422758
Unknown unknown kidney cancer not applicable RET Inhibitor DHA + Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, DHA and Stivarga (regorafenib) synergistically inhibited survival of kidney cancer cells in culture and reduced tumor growth in kidney cancer cell line xenograft models (PMID: 26921392). 26921392
Unknown unknown sarcoma not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (2.9 vs 1.0 months), but no difference in overall survival (HR=0.75, p=0.37) compared to placebo in patients with soft tissue sarcoma excluding liposarcoma, leiomyosarcoma, and synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) treatment in patients with non-small cell lung cancer resulted in stable disease (SD) in 76% (13/17) of patients, and one patient with SD experienced a progression free survival of 279 days and tumor reduction greater than 30% (J Clin Oncol 28:15s, 2010 (suppl; abstr 7585)). detail...
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga (regorafenib) showed antitumor activity in multiple murine xenograft models derived from melanoma, renal cell carcinoma, colorectal, breast, lung, pancreatic and ovarian tumor cell lines (PMID: 21170960). 21170960
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22421192). 22421192
Unknown unknown stomach cancer not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown gastric adenocarcinoma not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) resulted in a PFS of 2.6 months compared to .9 months with placebo in gastric adenocarcinoma patients (PMID: 27325864). 27325864
Unknown unknown leiomyosarcoma not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (3.7 vs 1.8 months), but no difference in overall survival (HR=0.50, p=0.056) compared to placebo in patients with leiomyosarcoma (PMID: 27751846). 27751846
Unknown unknown synovial sarcoma not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (5.6 vs 1.0 months), but no difference in overall survival (HR=0.87, p=0.79) compared to placebo in patients with synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown glioblastoma multiforme not applicable RET Inhibitor Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga, (regorafenib), inhibited tumor growth in cell line xenograft models of glioblastoma multiforme (PMID: 21170960). 21170960
Unknown unknown colorectal cancer not applicable RET Inhibitor Regorafenib FDA approved Actionable In a Phase III clinical trial (CORRECT) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved overall survival compared to placebo (6.4 vs 5.0 months, HR=0.77, p=0.0052) in patients with refractory metastatic colorectal cancer (PMID: 23177514; NCT01103323). 23177514 detail...
Unknown unknown colorectal cancer not applicable RET Inhibitor Regorafenib Phase III Actionable In a Phase III trial (CONSIGN), Stivarga (regorafenib) treatment demonstrated safety profile and efficacy consistent with previous studies, median progression-free survival (PFS) was 2.7 months overall, 2.8 months in KRAS wild-type, and 2.5 months in KRAS mutant colorectal cancer patients, and with no difference in KRAS status between long and short PFS groups (PMID: 30190299; NCT01538680). 30190299
Unknown unknown gastrointestinal stromal tumor not applicable RET Inhibitor Regorafenib FDA approved Actionable In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). detail... 23177515
Unknown unknown esophageal cancer not applicable RET Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown liposarcoma no benefit RET Inhibitor Regorafenib Phase I Actionable In a Phase II trial, Stivarga (regorafenib) treatment did not result in significant difference in median progression-free survival (1.1 vs 1.7 months) or overall survival (HR=1.57, p=0.21) compared to placebo in patients with liposarcoma (PMID: 27751846). 27751846
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Regorafenib FDA approved Actionable In a Phase III trial (RESORCE) that supported FDA approval, treatment with Stivarga (regorafenib) following Nexavar (sorafenib) treatment resulted in improved overall survival (10.6 vs 7.8 months, HR=0.63) compared to Nexavar (sorafenib) followed by placebo in patients with hepatocellular carcinoma (PMID: 27932229; NCT01774344). detail... 27932229
Unknown unknown colorectal cancer no benefit RET Inhibitor Regorafenib + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, Stivarga (regorafenib) treatment in combination with SBI-0206965 did not enhance apoptosis in a colorectal cancer cell line in culture (PMID: 30026382). 30026382
Unknown unknown thyroid gland cancer not applicable RET Inhibitor CLM3 Preclinical - Cell line xenograft Actionable In a preclinical study, CLM3 induced apoptosis and inhibited EGFR phosphorylation and cell proliferation in human anaplastic thyroid cancer cell lines in culture and inhibited tumor growth in xenograft models (PMID: 24423321). 24423321
Unknown unknown colorectal cancer not applicable RET Inhibitor Capecitabine + Oxaliplatin + Vandetanib Phase I Actionable In a Phase I clinical trial, the combination of Caprelsa (vandetanib), Xeloda (capecitabine), and Eloxatin (oxaliplatin) was well tolerated and demonstrated some efficacy in patients with colorectal cancer (PMID: 21404105). 21404105
Unknown unknown breast cancer not applicable RET Inhibitor Cyclophosphamide + Methotrexate + Vandetanib Phase I Actionable In a Phase I study, Caprelsa (vandetanib), in combination with Cytoxan (cyclophosphamide) and Abitrexate (methotrexate), demonstrated safety and some efficacy in breast cancer patients (PMID: 23001754). 23001754
Unknown unknown brain glioma not applicable RET Inhibitor Dasatinib + Vandetanib Phase I Actionable In a Phase I trial, Caprelsa (vandetanib), in combination with dasatinib, demonstrated safety in pediatric patients with intrinsic pontine glioma (PMID: 23536435). 23536435
Unknown unknown ovarian cancer no benefit RET Inhibitor Docetaxel + Vandetanib Phase II Actionable In a Phase II trial, the combination of Caprelsa (vandetanib) plus Taxotere (docetaxel) did not result in a greater PFS when compared to Taxotere (docetaxel) alone (PMID: 24709487). 24709487
Unknown unknown colon adenocarcinoma not applicable RET Inhibitor Fluorouracil + Leucovorin + Oxaliplatin + Vandetanib Phase I Actionable In a Phase I study, Caprelsa (vandetanib) in combination with mFOLFOX6 was well tolerated in advanced colorectal cancer patients (PMID: 19002384). 19002384
Unknown unknown pancreatic adenocarcinoma not applicable RET Inhibitor Gemcitabine + Vandetanib Phase I Actionable In a Phase I trial, Caprelsa (vandetanib), in combination with Gemzar (gemcitabine), demonstrated safety and resulted in stable disease in metastatic pancreatic adenocarcinoma patients (PMID: 21921646). 21921646
Unknown unknown pancreatic cancer no benefit RET Inhibitor Gemcitabine + Vandetanib Phase II Actionable In a Phase II trial, addition of Caprelsa (vandetanib) to Gemzar (gemcitabine) did not improve median overall survival (8.83 vs 8.95 months) compared to Gemzar (gemcitabine) monotherapy in patients with advanced pancreatic cancer (PMID: 28259610). 28259610
Unknown unknown glioblastoma multiforme not applicable RET Inhibitor Temozolomide + Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetinib), in combination with radiation therapy and Temodar (temozolomide), demonstrated no difference in PFS and OS when compared to radiation therapy and Temodar (temozolomide) alone in glioblastoma patients (PMID: 25910950). 25910950
Unknown unknown thyroid gland carcinoma not applicable RET Inhibitor Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) demonstrated efficacy in patients with advanced differentiated thyroid carcinoma (PMID: 22898678). 22898678
Unknown unknown melanoma not applicable RET Inhibitor Vandetanib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Caprelsa (vandetanib) resulted in a significant reduction in tumor outgrowth in mouse xenograft models of melanoma (PMID: 22075979). 22075979
Unknown unknown colon cancer not applicable RET Inhibitor Vandetanib Preclinical - Cell line xenograft Actionable In a preclinical study, Caprelsa (vandetanib) reduced human colon cancer cell line-induced angiogenesis and delayed tumor growth in colon cancer cell line xenograft models, with the highest delay corresponding to tumors with the highest VEGF-expression levels (PMID: 19528456). 19528456
Unknown unknown duodenum adenocarcinoma not applicable RET Inhibitor Vandetanib Preclinical Actionable In preclinical studies, Caprelsa (vandetinib) reduced the number and size of polyps in mouse models of intestinal cancer and may be a beneficial strategy in early intestinal cancer (PMID: 18347145). 18347145
Unknown unknown ovarian cancer no benefit RET Inhibitor Vandetanib Phase II Actionable In a Phase II trial, treatment with Caprelsa (vandetanib) in ovarian cancer patients resulted in no benefit and led to early termination of the trial (PMID: 20068097). 20068097
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) alone, or in combination with Taxotere (docetaxel), improved progression-free survival of NSCLC patients (PMID: 17243944). 17243944
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Vandetanib Clinical Study Actionable In a meta-analysis of 2,284 NSCLC patients, Caprelsa (vandetanib) in combination with chemotherapy, increased progression-free survival (PFS) and overall response rate (ORR) but not overall survival (PMID: 23861835). 23861835
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Vandetanib Phase II Actionable In a Phase II trial, treatment with Caprelsa (vandetanib) increased progression-free survival in NSCLC patients, compared to Iressa (gefitinib) (PMID: 19332730). 19332730
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Vandetanib Phase I Actionable In a Phase I trial, treatment with Caprelsa (vandetanib) resulted in stable disease in 40% (31/77) of patients with advanced solid tumors (PMID: 15905307). 15905307
Unknown unknown lung small cell carcinoma no benefit RET Inhibitor Vandetanib Phase II Actionable In a Phase II trial, addition of Caprelsa (vandetanib) to platinum (cisplatin or carboplatin) and etoposide did not improve time to progression (5.62 vs 5.68 months) or overall survival (12.24 vs 9.23 months) compared to placebo in untreated extensive-stage small cell lung cancer (PMID: 27583688). 27583688
Unknown unknown thyroid gland medullary carcinoma not applicable RET Inhibitor Vandetanib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Caprelsa (vandetanib) resulted in improved progression-free survival, an objective response rate of 45%, and a disease control rate of 87% in patients with medullary thyroid carcinoma (PMID: 22025146, PMID: 22723734). 22723734 detail... 22025146
Unknown unknown prostate cancer not applicable RET Inhibitor Danusertib Phase II Actionable In a Phase II clinical trial, Danusertib (PHA-739358) monotherapy was well tolerated, but showed minimal efficacy in patients with castration-resistant prostate cancer (PMID: 22928785). 22928785
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Danusertib Phase I Actionable In a phase I trial, Danusertib (PHA-739358) demonstrated safety and minimal efficacy in a patients with advanced solid tumors (PMID: 19770380, PMID: 22242557). 22242557 19770380
Unknown unknown colorectal cancer not applicable RET Inhibitor Danusertib Phase II Actionable In a Phase II clinical trial, Danusertib (PHA-739358) showed limited efficacy in patients with metastatic colorectal cancer (J Clin Oncol 28, 2010 (suppl; abstr e13558)). detail...
Unknown unknown breast cancer not applicable RET Inhibitor Danusertib Preclinical - Cell culture Actionable In a preclinical study, Danusertib (PHA-739358) disrupted cell cycle progression and inhibited growth of breast cancer cell lines, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). 25667100
Unknown unknown breast cancer not applicable RET Inhibitor Danusertib + Fulvestrant Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Danusertib (PHA-739358) and Faslodex (fulvestrant) resulted in increased growth inhibition in aromatase inhibitor-resistant breast cancer cell lines compared to either agent alone (PMID: 25667100). 25667100
Unknown unknown breast cancer not applicable RET Inhibitor SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of breast cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown acute myeloid leukemia not applicable RET Inhibitor SKI-G-801 Preclinical - Cell line xenograft Actionable In a preclinical study, SKI-G-801 treatment induced tumor regression in a cell line xenograft model of acute myeloid leukemia (Mol Cancer Ther 2011;10(11 Suppl):Abstract nr C196). detail...
Unknown unknown lung cancer not applicable RET Inhibitor SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of lung cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown melanoma not applicable RET Inhibitor SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor burden in a metastatic mouse model of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown acute myeloid leukemia not applicable RET Inhibitor Cytarabine + Daunorubicin + Quizartinib Phase I Actionable In a Phase I trial (QuANTUM-First), the combination therapy, Quizartinib (AC220) with Cytosar-U (cytarabine) and Cerubidine (daunorubicin), resulted in a response in 84% (16/19) of patients with acute myeloid leukemia, including fourteen patients with a composite complete response and two patients achieving morphologic leukemia-free state (PMID: 29139135; NCT01390337). 29139135
Unknown unknown acute myeloid leukemia not applicable RET Inhibitor Quizartinib Phase II Actionable In a Phase II trial, Quizartinib (AC220) treatment resulted in a composite complete remission (CCR) in 36% (16/44; 1 complete remission (CR)) of FLT3-ITD-negative patients vs. 56% (63/112; 3 CR) of FLT3-ITD-positive patients with relapsed/refractory acute myeloid leukemia (AML) after first-line therapy, and CCR in 30% (12/40; 1 CR) of FLT3-ITD-negative vs. 46% (62/136; 5 CR) in FLT3-ITD-positive patients with relapsed/refractory AML after salvage chemotherapy or transplant (PMID: 29859851; NCT00989261). 29859851
Unknown unknown chronic myelomonocytic leukemia not applicable RET Inhibitor TG101209 Preclinical - Patient cell culture Actionable In a preclinical study, TG101209 inhibited colony formation of granulocytes and macrophages cultured from patients with chronic myelomonocytic leukemia (PMID: 27157043). 27157043
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Camrelizumab + Rivoceranib Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 50% (8/16), a disease control rate of 93.8% (15/16, stable disease or better), a median progression-free survival (PFS) of 5.8 months, and a 9-month PFS rate of 41.0% in evaluable patients with advanced hepatocellular carcinoma (PMID: 30348638; NCT02942329). 30348638
Unknown unknown gastroesophageal junction adenocarcinoma not applicable RET Inhibitor Camrelizumab + Rivoceranib Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). 30348638
Unknown unknown stomach cancer not applicable RET Inhibitor Camrelizumab + Rivoceranib Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). 30348638
Unknown unknown gastric adenocarcinoma not applicable RET Inhibitor Rivoceranib Phase III Actionable In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown gastroesophageal junction adenocarcinoma not applicable RET Inhibitor Rivoceranib Phase III Actionable In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown lung cancer not applicable RET Inhibitor Rivoceranib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in lung cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown gastrointestinal stromal tumor not applicable RET Inhibitor Rivoceranib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) produced a partial response in 18.9% (7/37) and stable disease in 64.9% (24/37) of patients with advanced solid tumors, including partial response in one patient with GIST (PMID: 20923544). 20923544
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Rivoceranib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) demonstrated safety and efficacy in patients with a variety of solid tumor types (PMID: 20923544). 20923544
Unknown unknown synovial sarcoma not applicable RET Inhibitor Rivoceranib Case Reports/Case Series Actionable In a retrospective analysis, Rivoceranib (apatinib) treatment demonstrated manageable safety profile, resulted in a partial response in 42.9% (9/21) and stable disease in 38.1% (8/21) of patients with advanced synovial sarcoma, with a median progression-free survival of 13.1 months and a median overall survival of 15.5 months (PMID: 32669874). 32669874
Unknown unknown gastrointestinal system cancer not applicable RET Inhibitor Rivoceranib Phase III Actionable In a Phase III trial, treatment with Apatinib (YN968D1) at 850mg resulted in a greater progression free survival (2.6 mo vs 1.8 mo) and overall survival (6.5 mo vs 4.7 mo) when compared to placebo in patients with either gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown gastrointestinal system cancer not applicable RET Inhibitor Rivoceranib Phase II Actionable In a Phase II trial, Apatinib (YN968D1) improved progression-free survival and overall survival in metastatic gastric cancer patients (PMID: 23918952). 23918952
Unknown unknown stomach cancer not applicable RET Inhibitor Rivoceranib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in gastric cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown triple-receptor negative breast cancer not applicable RET Inhibitor Rivoceranib Phase II Actionable In a Phase II trial, 500 mg of Apatinib (YN968D1) produced a median progression-free survival of 3.3 months in TNBC patients (PMID: 24604288). 24604288
Unknown unknown colon cancer not applicable RET Inhibitor Rivoceranib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in colon cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown ovarian cancer not applicable RET Inhibitor Carboplatin + Nintedanib + Paclitaxel Phase III Actionable In a Phase III clinical trial, patients with advanced ovarian cancer treated with Ofev (nintedanib), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel), experienced a progression free survival of 17.2 months versus 16.6 months in patients treated with a combination of placebo, carboplatin, and paclitaxel in first-line treatment (PMID: 26590673). 26590673
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Docetaxel + Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in improved progression-free survival and overall survival compared to placebo plus Taxotere (docetaxel) in non-small cell lung cancer patients (PMID: 24411639). 24411639
Unknown unknown lung adenocarcinoma not applicable RET Inhibitor Docetaxel + Nintedanib Phase III Actionable In a Phase III trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in an improved overall survival in lung adenocarcinoma patients, particularly those patients with shorter time to progression after first line chemotherapy, which included measures that were time from start or time from end of first line chemotherapy (PMID: 28702806). 28702806
Unknown unknown brain glioma not applicable RET Inhibitor Nintedanib Phase II Actionable In a Phase II clinical trial, Ofev (nintedanib) failed to show any efficacy in patients with recurrent high-grade glioma, regardless of prior bevacizumab therapy (PMID: 25338318). 25338318
Unknown unknown gastric adenocarcinoma not applicable RET Inhibitor Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown Indication other than cancer not applicable RET Inhibitor Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail... detail...
Unknown unknown esophagus adenocarcinoma not applicable RET Inhibitor Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown lung cancer not applicable RET Inhibitor Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors (PMID: 25795637). 25795637
Unknown unknown gastroesophageal junction adenocarcinoma not applicable RET Inhibitor Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown ovarian cancer not applicable RET Inhibitor Nintedanib Phase I Actionable In a Phase I clinical trial, Vargatef (nintedanib) demonstrated safety in patients with ovarian cancers, clinical trials to determine efficacy in these patients are ongoing (PMID: 19889612). 19889612
Unknown unknown lung adenocarcinoma not applicable RET Inhibitor Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) improved progression-free survival and overall survival in patients with lung adenocarcinoma compared to Taxotere (docetaxel) alone (PMID: 24411639). 24411639
Unknown unknown pancreatic endocrine carcinoma not applicable RET Inhibitor Nintedanib Preclinical Actionable In a preclinical study, Ofev (nintedanib) induced tumor cell apoptosis, decreased microvessel density, inhibited tumor growth, and improved survival in transgenic mouse models of pancreatic neuroendocrine carcinoma (PMID: 26206868). 26206868
Unknown unknown pancreatic cancer not applicable RET Inhibitor Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown colorectal cancer not applicable RET Inhibitor Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and efficacy in patients with advanced colorectal cancer (PMID: 25012508). 25012508
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Famitinib Phase I Actionable In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). 24238512
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Famitinib Phase I Actionable In a Phase I trial, patients with advanced solid tumors had antitumor activity in response to the receptor tyrosine kinase inhibitor, famitinib (PMID: 24043137). 24043137
Unknown unknown prostate cancer not applicable RET Inhibitor Atezolizumab + Cabozantinib Phase I Actionable In a Phase I trial (COSMIC-021), Tecentriq (atezolizumab) and Cometriq (Cabometyx, cabozantinib) combination therapy was tolerated, resulted in an objective response rate of 32% (14/44, 2 complete responses, 12 partial responses) and stable disease in 48% (21/44) of patients with metastatic castration-resistant prostate cancer whose disease progressed after Xtandi (enzalutamide) or Zytiga (abiraterone) treatment (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5564-5564; NCT03170960). detail...
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Cabozantinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Cabometyx (cabozantinib) resulted in a median progression-free survival of 7.4 months in patients with renal cell carcinoma, compared to 3.8 months with Afinitor (everolimus), and an objective response rate of 22% (17/76) versus 3% (2/77) with Afinitor (everolimus) (PMID: 26406150). detail... 26406150
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cabometyx (cabozantinib) treatment demonstrated improved median progression-free survival (8.2 vs 5.6 months) and overall response rate (46% vs 18%) over Sutent (sunitinib) in untreated patients with metastatic renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA30_PR). detail... detail...
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial, final results extending those that supported FDA approval demonstrated Cabometyx (cabozantinib) improved median overall survival compared to Afinitor (everolimus) (21.4 m vs. 16.5 m) and progression-free survival (7.4 m vs. 3.9 m), and led to a 17% (57/330) objective response rate vs. 3% (11/328) with Afinitor (everolimus) in renal cell carcinoma patients (PMID: 27279544). 27279544 detail...
Unknown unknown gastrointestinal system cancer not applicable RET Inhibitor Cabozantinib Preclinical - Pdx Actionable In a preclinical study, Cometriq (cabozantinib) demonstrated efficacy in colorectal cancer patient-derived tumor explant models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1). detail...
Unknown unknown uveal melanoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in stable disease in 61% (14/23) of patients with metastatic uveal melanoma, with a median progression free survival of 4.8 months and an overall survival of 12.6 months (PMID: 28103611; NCT00940225). 28103611
Unknown unknown uveal melanoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, treatment with Cometriq (Cabometyx, cabozantinib) did not result in an improved progression-free survival at 4 months (32.3% vs 26.7%, P=0.35) and median overall survival (6.4 mo vs 7.3 mo, P=0.58) when compared to treatment with Temodar (temozolomide) or Deticene (dacarbazine) in patients with uveal melanoma (PMID: 31558480; NCT01835145). 31558480
Unknown unknown renal carcinoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, renal cancer patients treated with Cabozantinib demonstrated a 28 % objective response rate, a 62 % stable disease rate, and a median progression free survival of 14.7 months (PMID: 23292795). 23292795
Unknown unknown pancreatic adenocarcinoma not applicable RET Inhibitor Cabozantinib Preclinical Actionable In a preclinical trial, Cometriq (cabozantinib) promoted apoptosis of pancreactic ductal adenocarcinoma cells (PMID: 23661005). 23661005
Unknown unknown uterine carcinosarcoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II (NCI9322/PHL86) trial, Cometriq (Cabometyx, cabozantinib) treatment resulted in a partial response in 5% (1/19) and progression-free survival (PFS) at 12 weeks in 42% (8/19) of patients with endometrial carcinosarcoma, with a median PFS of 3.0 months (PMID: 31992589; NCT01935934). 31992589
Unknown unknown endometrial cancer not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II (NCI9322/PHL86) trial, Cometriq (Cabometyx, cabozantinib) treatment resulted in a response rate (RR) of 14% (5/36), a 12-week progression-free survival (PFS) rate of 67%, and a mPFS of 4.8 mo in patients with recurrent/metastatic endometrial cancer of endometrioid histology, and a RR of 12% (4/34), a 12-week PFS of 56%, and a mPFS of 4.0 mo in patients of serous histology, and a RR of 6% (2/32) and a 12-week PFS of 47% in patients of uncommon histology (PMID: 31992589; NCT01935934). 31992589
Unknown unknown colorectal cancer not applicable RET Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) showed anti-tumor activity in human colorectal cancer explants (PMID: 25242168). 25242168
Unknown unknown colorectal cancer not applicable RET Inhibitor Cabozantinib Preclinical - Pdx Actionable In a preclinical study, Cometriq (Cabometyx, cabozantinib) treatment inhibited activation of PI3K/AKT/MTOR signaling pathway, reduced phosphorylation of Met, Ret, and Axl, and induced autophagy in colorectal cancer cells in culture, and decreased tumor vascularity, reduced tumor growth, and induced regression in patient-derived xenograft (PDX) models (PMID: 30026382). 30026382
Unknown unknown pancreatic endocrine carcinoma not applicable RET Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) inhibited pancreatic neuroendocrine tumor growth and invasion in transgenic mouse models (PMID: 22585997). 22585997
Unknown unknown transitional cell carcinoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in complete response in 2% (1/41), partial response in 17% (7/41), stable disease in 44% (18/41) of urothelial carcinoma patients, with a median progression-free survival of 3.7 months and median overall survival of 8.2 months (J Clin Oncol 34, 2016 (suppl; abstr 4534)). detail...
Unknown unknown breast cancer not applicable RET Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) suppressed metastasis, angiogenesis, and tumor growth in mouse models of breast cancer (PMID: 21926191). 21926191
Unknown unknown multiple myeloma no benefit RET Inhibitor Cabozantinib Phase I Actionable In a Phase Ib trial, Cometriq (Cabometyx, cabozantinib) treatment did not demonstrate significant efficacy, resulting in a minimal response in 9% (1/11), stable disease in 73% (8/11), and progression in 18% (2/11) of patients with relapsed and/or refractory multiple myeloma (PMID: 27020089; NCT01866293). 27020089
Unknown unknown brain glioma not applicable RET Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) decreased cell proliferation and induced apoptosis in mouse models of glioma (PMID: 21926191). 21926191
Unknown unknown cholangiocarcinoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in a median progression free survival of 1.8 months, and a median overall survival of 5.2 months in patients with advanced cholangiocarcinoma, but also induced grade 3/4 adverse events in 89% (17/19) of the patients (PMID: 28192597). 28192597
Unknown unknown clear cell renal cell carcinoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in improved progression free survival (8.2 v 5.6 months) and objective response rate (46% vs 18%) compared to Sutent (sunitinib) in patients with untreated clear cell metastatic renal cell carcinoma, with a 34% reduction in rate of progression or death (HR=0.66, p=0.012) (PMID: 28199818). 28199818
Unknown unknown ovarian cancer not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib ) demonstrated safety and efficacy in patients with ovarian cancers (J Clin Oncol 29: 2011 (suppl; abstr 5008)). detail...
Unknown unknown melanoma not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in partial response in 5% (4/77) and sttable disease in 39% (30/77) of patients with metastatic melanoma, with a median overall progression free survival of 3.8 months (PMID: 28103611). 28103611
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial (CELESTIAL) that supported FDA approval, Cabometyx (cabozantinib) significantly improved overall survival (10.2 vs 8.0 months, HR=0.76, p=0.005) and progression-free survival (5.2 vs 1.9 months, HR=0.44, p<0.001) compared to placebo in patients with previously treated advanced hepatocellular carcinoma (PMID: 29972759; NCT01908426). 29972759 detail...
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Cabozantinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) treatment resulted in partial response in 6.7% (1/15) of patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib), with a median progression free survival of 1.9 months (PMID: 28352985). 28352985
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib) showed safety and anti-tumor activity in several advanced solid tumor types (J Clin Oncol 29: 2011 (suppl; abstr 3010)). detail...
Unknown unknown thyroid gland medullary carcinoma not applicable RET Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cometriq (cabozantinib) resulted in improved progression free survival in patients with metastatic medullary thyroid cancer (PMID: 23319867). detail... 23319867
Unknown unknown colorectal cancer not applicable RET Inhibitor Cabozantinib + Chloroquine Preclinical - Cell culture Actionable In a preclinical study, Cometriq (Cabometyx, cabozantinib) treatment in combination with Chloroquine enhanced apoptosis in a colorectal cancer cell line in culture (PMID: 30026382). 30026382
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Cabozantinib + CT-707 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). 27638856
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Cabozantinib + Erlotinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) and Tarceva (erlotinib) combination treatment resulted in no response (0/13) in patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib) in Phase II, despite an objective response rate of 8.2% (5/61) in Phase I (PMID: 28352985). 28352985
Unknown unknown triple-receptor negative breast cancer not applicable RET Inhibitor Cabozantinib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Cometriq (cabozantinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown colorectal cancer not applicable RET Inhibitor Cabozantinib + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, Cometriq (Cabometyx, cabozantinib) treatment in combination with SBI-0206965 enhanced apoptosis in colorectal cancer cells in culture (PMID: 30026382). 30026382
Unknown unknown prostate cancer not applicable RET Inhibitor Cabozantinib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with Cometriq (cabozantinib) and immune checkpoint blockade with anti-CTLA4 and anti-PD-1 antibodies resulted in synergistic inhibition of tumor growth and metastasis in transgenic mouse models of metastatic castration-resistant prostate cancer (PMID: 28321130). 28321130
Unknown unknown stomach cancer not applicable RET Inhibitor JNJ-26483327 Preclinical Actionable In a preclinical study, human gastric cancer cells demonstrated sensitivity to JNJ-26483327 (PMID: 19584230). 19584230
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor JNJ-26483327 Phase I Actionable In a Phase I clinical trial, JNJ-26483327 was well-tolerated in patients with a range of advanced solid tumors, and demonstrated preliminary anti-tumor activity with 32% (6/19) of patients achieving stable disease for greater than 2 cycles (PMID: 20823884). 20823884
Unknown unknown clear cell renal cell carcinoma no benefit RET Inhibitor Bevacizumab + Sorafenib Phase III Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Avastin (bevacizumab) did not result in a significant improvement in progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (9.2 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Bevacizumab + Sorafenib Phase I Actionable In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). 25363205
Unknown unknown invasive bladder transitional cell carcinoma not applicable RET Inhibitor Cisplatin + Gemcitabine + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) in combination with Platinol (cisplatin) and Gemzar (gemcitabine) resulted in pathologic complete response in 42.2% (19/45) of patients with muscle-invasive urothelial bladder cancer (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). detail...
Unknown unknown colon cancer not applicable RET Inhibitor CVX-060 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Nexavar (sorafenib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown hepatocellular carcinoma no benefit RET Inhibitor Dalantercept + Sorafenib Phase I Actionable In a Phase Ib trial, addition of Dalantercept (ACE-041) to Nexavar (sorafenib) was well tolerated, but did not improve the efficacy of Nexavar (sorafenib) in patients with advanced hepatocellular carcinoma, with overall survival ranged from 1.9 to 23.3 months and stable disease as best overall response in 53.3% (11/21) of the patients (PMID: 30352941; NCT02024087). 30352941
Unknown unknown hepatocellular carcinoma no benefit RET Inhibitor Erlotinib + Sorafenib Phase III Actionable In a Phase III trial, the combination treatment of Nexavar (sorafenib) with Tarceva (erlotinib) compared to Nexavar (sorafenib) plus placebo did not demonstrate an improved overall survival and time to progression in patients with hepatocellular carcinoma (PMID: 25547503). 25547503
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Fingolimod + Sorafenib Preclinical Actionable In a preclinical study, Gilenya (fingolimod) worked synergistically with Nexavar (sorafenib) to inhibit growth and induce apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26516583). 26516583
Unknown unknown colorectal cancer not applicable RET Inhibitor Fluorouracil + Quinacrine + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergistically enhanced the cytotoxicity of Nexavar (sorafenib) in human colorectal cancer cell lines in culture (PMID: 21725213). 21725213
Unknown unknown colon cancer not applicable RET Inhibitor Fluorouracil + Sorafenib Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Fluorouracil + Sorafenib Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor GW5074 + Sorafenib Preclinical Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) induced cell death in several tumor cell lines, and phosphorylation of DAPK at amino acid S308 correlated positively with response to therapy (PMID: 26100670). 26100670
Unknown unknown renal cell carcinoma not applicable RET Inhibitor GW5074 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) worked synergistically to induce cell death in renal cell carcinoma cells in culture and inhibited tumor growth in xenograft and spontaneous mouse models of renal cell carcinoma (PMID: 26100670). 26100670
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Intuvax + Sorafenib Case Reports/Case Series Actionable In a Phase I trial, Intuvax (Ilixadencel) alone or in combination with Nexavar (sorafenib) demonstrated safety in hepatocellular carcinoma patients, and resulted in one partial response (Intuvax monotherapy), stable disease in 5 patients, increased circulating tumor-specific CD8-positive T-cells in 82% (9/11) of patients receiving Intuvax alone and 50% (2/4) of patients also receiving Nexavar, median time to progression of 5.5 months, and median overall survival of 7.5 months (PMID: 30719425; NCT01974661). 30719425
Unknown unknown hepatocellular carcinoma no benefit RET Inhibitor Lenalidomide + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Revlimid (lenalidomide) and Nexavar (sorafenib) was not well-tolerated and did not demonstrate clinical activity in patients with hepatocellular carcinoma, and the study was terminated early due to toxicity (PMID: 27256874). 27256874
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Metformin + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, addition of Glucophage (metformin) sensitized hepatocellular carcinoma cells to Nexavar (sorafenib) induced apoptosis in culture, resulted in suppression of postoperative intrahepatic recurrence and lung metastasis in cell line xenograft models (PMID: 26957312). 26957312
Unknown unknown breast cancer not applicable RET Inhibitor Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with breast cancer, with 58% (7/12) of breast cancer patients achieving objective response or stable disease (PMID: 27213589). 27213589
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 15% (5/33) and stable disease in 45% (15/33) of patients (PMID: 27213589). 27213589
Unknown unknown triple-receptor negative breast cancer not applicable RET Inhibitor Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with triple-receptor breast cancer (TNBC), with 60% (3/5) of TNBC patients demonstrating an objective response and 100% (5/5) of patients achieving stable disease or better (PMID: 27213589). 27213589
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor PX-866 + Sorafenib Preclinical Actionable In a preclinical study, treatment with the combination of Stivarga (regorafenib) PK-866 resulted in increased cell death in a variety of solid tumor cell lines in culture (PMID: 23877009). 23877009
Unknown unknown colorectal cancer not applicable RET Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, a patient with colorectal cancer demonstrated a durable partial response for 358 days when treated with the combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) (PMID: 26644411). 26644411
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, 43.8% (7/16) of hepatocellular carcinoma patients treated with a combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, the combination treatment of Refametinib (BAY86-9766) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in a disease control rate of 65.8% (25/38), specifically, 2.6% (1/38) experienced a partial response and 63.2% (24/38) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Resminostat + Sorafenib Phase Ib/II Actionable In a Phase I/II trial, the combination of Resminostat (4SC-201) and Nexavar (sorafenib) demonstrated increased efficacy compared to Resminostat (4SC-201) alone in advanced hepatocellular carcinoma patients, resulting in an improved progression-free survival rate of 62.5% vs. 12.5%, a median time to progression of 4.1 vs. 1.8 months, and an overall survival of 8.0 vs. 6.5 months (PMID: 26952006). 26952006
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Selumetinib + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) and Selumetinib (AZD6244) combination treatment resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of hepatocellular carcinoma patients (PMID: 27681866). 27681866
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor SF1126 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SF1126 and Nexavar (sorafenib) was synergistic or additive in inhibiting proliferation of hepatocellular carcinoma cell lines in culture, and demonstrated increased efficacy in hepatocellular carcinoma cell line xenograft models compared to SF1126 alone (PMID: 23355037). 23355037
Unknown unknown thyroid gland medullary carcinoma not applicable RET Inhibitor Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 6.3% (1/16) and stable disease in 87.5% (14/16) of patients with sporadic medullary thyroid carcinoma, with a median progression free survival of 17.9 months (PMID: 20368568). 20368568
Unknown unknown lung non-small cell carcinoma no benefit RET Inhibitor Sorafenib Phase III Actionable In a Phase III trial (MISSION), Nexavar (sorafenib) treatment in non-small cell lung carcinoma patients did not reach its primary endpoint, resulting in an overall survival similar to that when treated with placebo (8.2 vs 8.3 mo, HR=0.99, p=0.47), however, did meet its secondary endpoint, demonstrating a greater progression free survival (2.8 vs 1.4 mo, HR=0.61, p<0.0001) and time to progression (2.9 vs 1.4 mo, HR=0.54, p<0.0001) when compared to placebo (PMID: 26743856; NCT00863746). 26743856
Unknown unknown Her2-receptor negative breast cancer not applicable RET Inhibitor Sorafenib Clinical Study Actionable In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). 24940450
Unknown unknown gastrointestinal stromal tumor not applicable RET Inhibitor Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 13% (4/31), stable disease in 52% (16/31), a median progression-free survival of 4.9 months and an overall survivals of 9.7 months in gastrointestinal stromal tumor patients who failed prior tyrosine kinase inhibitors (PMID: 22270258). 22270258
Unknown unknown thyroid gland carcinoma not applicable RET Inhibitor Sorafenib Clinical Study Actionable In a clinical case report, Nexavar (sorafenib) therapy based on in vitro efficacy testing using patient-derived tumor cells resulted in 43 months of disease-free in a patient with anaplastic thyroid carcinoma (PMID: 27379749). 27379749
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) resulted in an improved median progression-free survival of 167 days in patients with renal cell carcinoma (PMID: 17189398). detail... 17189398
Unknown unknown hepatocellular carcinoma not applicable RET Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). 19144678 detail...
Unknown unknown endometrial carcinoma not applicable RET Inhibitor Sorafenib Preclinical Actionable In preclinical studies, Nexavar (sorafenib) promoted apoptosis of endometrial carcinoma cells (PMID: 23463670). 23463670
Unknown unknown thyroid gland cancer not applicable RET Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). detail... 24768112
Unknown unknown desmoid tumor not applicable RET Inhibitor Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment resulted in an increased 2-year progression-free survival compared to placebo (81% vs 36%), an objective response rate of 33% (16/49; 1 complete response and 15 partial responses (PR)) compared in 20% (7/25; all PR) with placebo, and a median time to objective response of 9.6 months, compared to 13.3 months with placebo, in patients with refractory desmoid tumors (PMID: 30575484; NCT02066181). 30575484
Unknown unknown clear cell renal cell carcinoma no benefit RET Inhibitor Sorafenib + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor XL999 Phase II Actionable In six Phase II clinical trials, XL999 demonstrated anti-tumor activity in patients with advanced solid tumors, but also resulted in significant cardiotoxicity, which improved after discontinuation of the drug (J Clin Oncol, 25:18s, 2007 (Suppl; abstr 3591)). detail...
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor MGCD516 Phase I Actionable In a Phase I trial, MGCD516 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2575)). detail...
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in a variety of human advanced solid tumor cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown lung carcinoma not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown glioblastoma multiforme not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human glioblastoma multiforme cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown colorectal cancer not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human colorectal carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown breast carcinoma not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human breast carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown liposarcoma not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). 26675259
Unknown unknown stomach carcinoma not applicable RET Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human stomach carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown renal cell carcinoma no benefit RET Inhibitor AGS-003 + Sunitinib Phase III Actionable In a Phase III trial (ADAPT), the combination of AGS-003 (Rocapuldencel-T) and Sutent (sunitinib) did not demonstrate improved efficacy compared to Sutent alone in metastatic renal cell carcinoma patients, resulting in median overall survival of 27.7 v 32.4 months, progression-free survival of 6.0 v 7.83 months, and objective response rate of 42.7% (131/307, 9 complete, 122 partial) v 39.4% (61/155, 3 complete, 58 partial) in the combination or monotherapy groups, respectively (PMID: 32034074; NCT01582672). 32034074
Unknown unknown renal cell carcinoma no benefit RET Inhibitor AGS-003 + Sunitinib Phase II Actionable In a Phase II clinical trial, treatment with the combination of AGS-003 and Sutent (sunitinib) resulted in clinical benefit in 62% (13/21) of patients with advanced renal cell carcinoma, with 9 partial responses and 4 patients achieving stable disease, a median overall survival of 30.2 months, and median progression-free survival of 11.2 months (PMID: 25901286). 25901286
Unknown unknown colon cancer not applicable RET Inhibitor CVX-060 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown kidney cancer not applicable RET Inhibitor CVX-060 + Sunitinib Preclinical Actionable In a preclinical study, the combination of CVX-060 and Sutent (sunitinib) demonstrated a trend improved overall survival compared to single agent Sutent (sunitinib) in mouse models of unresected and resected renal cancer, however, also demonstrated increased toxicity (PMID: 27651308). 27651308
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Everolimus + Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) as first line therapy followed by second line therapy, Afinitor (everolimus), resulted in a greater overall survival (29.5 mo vs 22.4 mo) compared to the reverse treatment of the two therapies in patients with metastatic renal cell carcinoma (PMID: 28327953). 28327953
Unknown unknown brain glioblastoma multiforme not applicable RET Inhibitor Irinotecan + Sirolimus + Sunitinib + Temozolomide Clinical Study Actionable In two clinical case studies, RIST (rapamycin, irinotecan, sunitinib, temozolomide) resulted in anti-tumor activity in patients with glioblastoma (PMID: 25123598). 25123598
Unknown unknown Advanced Solid Tumor not applicable RET Inhibitor Ixabepilone + Sunitinib Phase I Actionable In a Phase I trial, Ixabepilone and Sutent (sunitinib) combination therapy resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of patients with advanced solid tumors (PMID: 26864210). 26864210
Unknown unknown glioblastoma multiforme not applicable RET Inhibitor PRX177561 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PRX177561 and Sutent (sunitinib) decreased tumor growth and improved time-to-progression, disease-free survival, and overall survival over either agent alone in glioblastoma cell line xenograft models (PMID: 28057017). 28057017
Unknown unknown thyroid gland cancer not applicable RET Inhibitor SL327 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SL327 and Sutent (sunitinib) worked additively to decrease viability, induce apoptosis, and decrease migration of Taxotere (docetaxel)-resistant anaplastic thyroid cancer cell lines in culture, and to inhibit tumor growth in xenograft models (PMID: 28178630) 28178630
Unknown unknown paraganglioma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II trial (SNIPP), Sutent (sunitinib) treatment in patients with paraganglioma (PGL, n=11) or pheochromocytoma (PCC, n=14) resulted in an overall response rate of 13% (3/23), including partial responses in one PGL patient and two PCC patients, a disease control rate of 83% (19/23), and median progression-free survival of 13.4 months (PMID: 31105270). 31105270
Unknown unknown pheochromocytoma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II trial (SNIPP), Sutent (sunitinib) treatment in patients with pheochromocytoma (PCC, n=14) or paraganglioma (PGL, n=11) resulted in an overall response rate of 13% (3/23), including partial responses in two PCC patients and one PGL patient, a disease control rate of 83% (19/23), and median progression-free survival of 13.4 months (PMID: 31105270). 31105270
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in non-small cell lung cancer patients resulted in an objective response rate of 11.1% (7/63), stable disease in 28.6% (18/63), and a PFS of 12 weeks and OS of 23.4 weeks (PMID: 18235126). 18235126
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Sunitinib Phase III Actionable In a Phase III trial, Sutent (sunitinib) as maintenance therapy resulted in improved progression free survival (4.3 vs 2.6 months) but not overall survival (11.7 vs 12.1 months) compared to placebo in patients with stage IIIB/IV non-small cell lung cancer (PMID: 28161554). 28161554
Unknown unknown lung non-small cell carcinoma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment resulted in an objective response rate of 8% (1/13, partial response) and stable disease as the best response in 69% (9/13) of non-small cell lung carcinoma patients (PMID: 32312893; NCT01829217). 32312893
Unknown unknown ovarian cancer not applicable RET Inhibitor Sunitinib Phase Ib/II Actionable In a Phase II trial, Sutent (sunitinib) treatment in ovarian cancer patients resulted in an increased PFS and a response rate of 16.7% (6/36) in those that received Sutent (sunitinib) continuously and a a response rate of 5.4% (2/37) in those that received the drug non-continuously (PMID: 22377563, PMID: 24070205). 22377563 24070205
Unknown unknown glioblastoma multiforme no benefit RET Inhibitor Sunitinib Phase II Actionable In multiple Phase II clinical trials, Sutent (sunitinib) failed to demonstrate any benefit in patients with glioblastoma with or without concurrent bevacizumab treatment (PMID: 24424564, PMID: 23086433). 23086433 24424564
Unknown unknown gastrointestinal stromal tumor not applicable RET Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) improved median progression free survival to 27.3 weeks in GIST patients (PMID: 17332278). 17332278 detail...
Unknown unknown malignant glioma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with high grade glioma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown malignant glioma not applicable RET Inhibitor Sunitinib Preclinical Actionable In a preclinical study, Sutent (sunitinb) induced cell death and decreased proliferation of glioma cells in culture (PMID: 25458015). 25458015
Unknown unknown endometrial cancer not applicable RET Inhibitor Sunitinib Phase II Actionable In Phase II clinical trials, Sutent (sunitinib) demonstrated efficacy in patients with metastatic or recurrent endometrial carcinoma (PMID: 24882554). 24882554
Unknown unknown pancreatic endocrine carcinoma not applicable RET Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, Sutent (sunitinib) demonstrated safety and improved progression free survival in patients with pancreatic neuroendocrine tumors (PMID: 21306237). 21306237 detail...
Unknown unknown lung small cell carcinoma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in small cell lung cancer patients resulted in a partial response of 11% (1/9) and stable disease in 30% (3/9) (PMID: 26716400). 26716400
Unknown unknown colon cancer not applicable RET Inhibitor Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sutent (sunitinib) induced apoptosis in colon cancer cells in culture and in cell line xenograft models (PMID: 22912816). 22912816
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) resulted in improved median progression free survival (47.3 weeks) and objective response rate (27.5%) in patients with renal cell carcinoma (PMID: 19707433). 19707433 detail...
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Sunitinib Phase III Actionable In a Phase III clinical trial, treatment with Sutent (sunitinib) resulted in prolonged disease free survival (HR = 0.761) compared to placebo in post-nephrectomy patients with renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA11_PR). detail...
Unknown unknown malignant ependymoma not applicable RET Inhibitor Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with ependymoma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown renal cell carcinoma not applicable RET Inhibitor Sunitinib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination of Mekinist (trametinib) and Sutent (sunitinib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of Sutent (sunitinib)-refractory renal cell carcinoma (PMID: 26487278). 26487278
Clinical Trial Phase Therapies Title Recruitment Status