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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 BRAF Inhibitor ASN003 Phase I Actionable In a Phase I trial, ASN003 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 371PD; NCT02961283). detail...
Unknown unknown triple-receptor negative breast cancer not applicable BRAF Inhibitor SKLB646 Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB646 inhibited proliferation and migration and induced apoptosis of triple-negative breast cancer (TNBC) cell lines in culture, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 26721945). 26721945
Unknown unknown Advanced Solid Tumor not applicable BRAF 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 BRAF 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 leiomyosarcoma not applicable BRAF 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 stomach cancer not applicable BRAF 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 sarcoma not applicable BRAF 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 synovial sarcoma not applicable BRAF 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 BRAF 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 lung non-small cell carcinoma not applicable BRAF 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 BRAF 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 Advanced Solid Tumor not applicable BRAF 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 gastric adenocarcinoma not applicable BRAF 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 esophageal cancer not applicable BRAF 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 colorectal cancer not applicable BRAF 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 colorectal cancer not applicable BRAF 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 gastrointestinal stromal tumor not applicable BRAF 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 liposarcoma no benefit BRAF 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 BRAF 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 hepatocellular carcinoma not applicable BRAF Inhibitor Regorafenib Case Reports/Case Series Actionable In a Phase I trial, two hepatocellular carcinoma patients treated with Stivarga (regorafenib) following the discontinuation of CEBPA-51 (MTL-CEBPA) treatment achieved stable disease 3 months following treatment, but the treatment was discontinued due to toxicity (PMID: 32357963; NCT02716012). 32357963
Unknown unknown colorectal cancer no benefit BRAF 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 lung non-small cell carcinoma not applicable BRAF 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 BRAF 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 Advanced Solid Tumor not applicable BRAF Inhibitor Belvarafenib Phase I Actionable In a Phase I trial, Belvarafenib (HM95573) treatment demonstrated safety and preliminary efficacy, resulted in unconfirmed partial response in 3% (1/31) and stable disease in 29% (9/31) of patients with advanced solid tumors harboring BRAF (45%), KRAS (45%) or NRAS (10%) mutations (May 20 2016) 2570-2570; NCT02405065). detail...
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor WX-554 Phase I Actionable In a Phase I trial, WX-554 was well-tolerated and resulted in stable disease in 59% (20/34) and prolonged stable disease for greater than 6 months in 6% (2/34) of patients with advanced solid tumors (PMID: 27693888). 27693888
Unknown unknown Advanced Solid Tumor no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Copanlisib + Refametinib Phase I Actionable In a Phase I trial, Aliqopa (copanlisib) and Refametinib (BAY86-9766) combination treatment resulted in stable disease as best response in 33% (21/64) of patients with advanced solid tumors, however, the study failed to establish a tolerable and efficacious dose and schedule of this combination (PMID: 32314268; NCT01392521). 32314268
Unknown unknown pancreatic cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Refametinib Phase Ib/II Actionable In a Phase I/II trial, Refametinib (BAY86-9766) and Gemzar (gemcitabine) combination treatment resulted in an objective response rate of 23% and a disease control rate of 73% in patients with advanced pancreatic cancer (PMID: 27975152). 27975152
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Refametinib Phase I Actionable In a Phase I trial, Refametinib (BAY 86-9766) was well-tolerated and displayed some evidence of clinical benefit across several tumor types (PMID: 23434733). 23434733
Unknown unknown colorectal cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) 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 MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) 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 MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) 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 Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor BI-847325 Phase I Actionable In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). 26650227
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor BI-847325 Phase I Actionable In a Phase I trial, BI-847325 demonstrated safety and some efficacy in a variety of advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):B281). detail...
Unknown unknown esophageal cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor BI-847325 Phase I Actionable In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). 26650227
Unknown unknown Hodgkin's lymphoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Darinaparsin + U0126 Preclinical - Cell culture Actionable In a preclinical study, Darinaparsin treatment in combination with U0126 inhibited Erk1/2 phosphorylation and led to enhanced apoptosis in Hodgkin's lymphoma cells compared to Darinaparsin treatment alone in culture (PMID: 25316819). 25316819
Unknown unknown ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor ABT-737 + AZD8055 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of AZD8055 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in greater apoptotic activity and cell cycle arrest when compared to Mekinist (trametinib) alone (PMID: 27980105). 27980105
Unknown unknown ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor ABT-737 + Dactolisib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of BEZ235 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). 27980105
Unknown unknown ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor ABT-737 + MK2206 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of MK2206 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). 27980105
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Afuresertib + Trametinib Phase I Actionable In a Phase I trial, the combination of Mekinist (trametinib) and Afuresertib (GSK2110183) was not well-tolerated in patients with advanced solid tumors (PMID: 25417902). 25417902
Unknown unknown lung cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) AZ628 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and AZ628 resulted in greater inhibition of Mek and apoptosis in a non-BRAF V600 mutant lung cancer cell line in culture compared to the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (J Clin Oncol 35, 2017 (suppl; abstr e23154)). detail...
Unknown unknown ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Buparlisib + Trametinib Phase Ib/II Actionable In a Phase Ib trial, ovarian cancer patients demonstrated an objective response rate of 21% (6/21), including 1 complete response and 5 partial responses, to treatment with Buparlisib (BKM120) in combination with Mekinist (trametinib) (PMID: 25500057) 25500057
Unknown unknown lung non-small cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Docetaxel + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in an overall response rate (ORR) of 21% (10/47, all partial responses) and stable disease in 43% (20/47) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
Unknown unknown lung squamous cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Docetaxel + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in partial response in 1 patient and stable disease in 3 patients within the subset of 5 evaluable patients with squamous non-small cell lung cancer patients (PMID: 27876675). 27876675
Unknown unknown colorectal cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Fluorouracil + Trametinib Preclinical Actionable In preclinical studies, Mekinist (trametinib) enhanced the efficacy of Adrucil (fluorouracil) in colorectal cancer cells in culture (PMID: 23438367). 23438367
Unknown unknown pancreatic cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib trial, a partial response was seen in 30% (3/11) of patients with pancreatic cancer after treatment with Mekinist (trametinib) in combination with Gemzar (gemcitabine) (PMID: 23583440). 23583440
Unknown unknown breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, including one complete response in a patient with breast cancer (PMID: 23583440). 23583440
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 23583440). 23583440
Unknown unknown melanoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor KRT-232 + Trametinib Phase I Actionable In a Phase I trial, the combination therapy of KRT-232 (AMG 232) and Mekinist (trametinib) in 15 patients with metastatic cutaneous melanoma without a BRAF V600 mutation resulted in a partial response in 2 patients, stable disease in 11 patients, and progressive disease in 2 patients, and of the 15 patients, 11 experienced tumor reduction (J Clin Oncol 35, 2017 (suppl; abstr 2575)). detail...
Unknown unknown differentiated thyroid gland carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pazopanib + Trametinib Phase I Actionable In a Phase I trial, treatment with the combination of Votrient (pazopanib) and Mekinist (trametinib) was tolerable and resulted in an objective response rate of 33% (4/12, all partial responses), stable disease in 50% (5/12), a median progression-free survival (PFS) of 10.7 months, a 2-year PFS of 25%, and a median overall survival of 29.3 months in patients with differentiated thyroid cancer in the dose expansion cohort (PMID: 31186313; NCT01438554). 31186313
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pazopanib + Trametinib Phase I Actionable In a Phase I trial, combination treatment with Votrient (pazopanib) and Mekinist (trametinib) was tolerable and resulted in partial response in 12% (3/25), and stable disease in 72% (18/25) of patients with advanced solid tumors, and 9 patients remained on study for more than 6 cycles, including patients with differentiated thyroid cancer (n=3), colorectal cancer (n=2), melanoma (n=1), cholangiocarcinoma (n=1), ovarian cancer (n=1), and synovial cell sarcoma (n=1) (PMID: 31186313; NCT01438554). 31186313
Unknown unknown renal cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pazopanib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination of Mekinist (trametinib) and Votrient (pazopanib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of renal cell carcinoma (PMID: 26487278). 26487278
Unknown unknown sarcoma no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pazopanib + Trametinib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Votrient (pazopanib) and Mekinist (trametinib) demonstrated safety and resulted in partial response in 8% (2/25) and stable disease in 48% (12/25) of patients with advanced soft tissue sarcomas, but did not improve the 4 month progression-free survival rate over Votrient (pazopanib) alone (PMID: 28377484). 28377484
Unknown unknown lung non-small cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pemetrexed Disodium + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Alimta (pemetrexed) resulted in an overall response rate of 14% (6/42, all partial responses) and stable disease in 55% (23/42) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
Unknown unknown renal cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 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
Unknown unknown pancreatic cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Trametinib Phase I Actionable In a Phase I trial, 42% (11/26) of pancreatic cancer patients demonstrated a decrease in tumor formation when treated with Mekinist (trametinib) (PMID: 22805291). 22805291
Unknown unknown oral squamous cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Trametinib Phase II Actionable In a Phase II trial, Mekinist (trametinib) treatment in patients with oral cavity squamous cell carcinoma was associated with decreased phosphorylation of Erk1/2 and reduced CD44 expression, and resulted in a partial response in 65% (11/17), stable disease in 29% (5/17), and progressive disease in 1 patient (6%) (PMID: 28151720). 28151720
Unknown unknown uveal melanoma no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Trametinib Phase I Actionable In a Phase I trial, Mekinist (trametinib) treatment resulted in stable disease as best response in 50% (8/16) of patients with uveal melanoma (PMID: 22805292; NCT00687622). 22805292
Unknown unknown endometrial cancer no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Trametinib + Uprosertib Phase I Actionable In a Phase I trial, Mekinist (trametinib) and Uprosertib (GSK2141795) combination treatment demonstrated increased toxicity and limited efficacy, resulted in no response (0/14) at RP2D dose and 1 response (8.3%, 1/12) at reduced dose in patients with recurrent endometrial cancer, with progression-free survival at 6 months in 14% and 25% of the patients, respectively (PMID: 31623857). 31623857
Unknown unknown Advanced Solid Tumor no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Trametinib + Uprosertib Phase I Actionable In a Phase I trial, the combination of Trametinib (GSK1120212) and Uprosertib (GSK2141795) was not well-tolerated and resulted in minimal efficacy in patients with advanced solid tumors (n=126), demonstrating an objective response rate of less than 5%, with one complete response and five partial responses (PMID: 32062691). 32062691
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RO4987655 Phase I Actionable In a Phase I trial, RO4987655 demonstrated safety and preliminary clinical activity in Japanese patients with advanced solid tumors, including 1 partial response in a patient with esophageal cancer and 7 patients achieving progression-free survival for greater than 16 weeks (PMID: 25809858). 25809858
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RO4987655 Phase I Actionable In a Phase I trial, RO4987655 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22767668). 22767668
Unknown unknown biliary tract cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Binimetinib Phase I Actionable In a Phase I trial, Binimetinib (MEK162) treatment resulted in complete response in 3.3% (1/30) and partial response in 6.7% (2/30) of patients with biliary tract cancer, with estimated progression free survival of 2.1 months and overall survival of 4.8 months (PMID: 28152546). 28152546
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Binimetinib Phase I Actionable In a Phase I trial, Binimetinib (MEK162) treatment resulted in complete response in 1% (1/91), partial response in 2% (2/91), and stable disease with median duration of 3.94 months in 36% (33/91) of patients with advanced solid tumors (PMID: 28152546). 28152546
Unknown unknown colorectal cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Binimetinib + Fluorouracil + Leucovorin + Oxaliplatin Phase I Actionable In a Phase I clinical trial, the combination of Binimetinib (MEK162) and FOLFOX chemotherapy demonstrated manageable toxicity and preliminary efficacy in metastatic colorectal cancer patients with chemotherapy resistance (J Clin Oncol 34, 2016 (suppl; abstr 2544)). detail...
Unknown unknown ovary epithelial cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Binimetinib + Paclitaxel Phase Ib/II Actionable In a Phase Ib trial, combination therapy with Taxol (paclitaxel) and Mektovi (binimetinib) was tolerable and resulted in an objective response rate of 18% (5/28, 1 complete response, 4 partial responses) and a clinical benefit rate of 57% (16/28, best overall response of stable disease or better) in patients with platinum resistant/refractory epithelial ovarian cancer, and clinical benefit was observed in all patients with MAPK pathway alterations (n=4) (PMID: 29844129; NCT01649336). 29844129
Unknown unknown rhabdomyosarcoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor AZD8055 + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Koselugo (selumetinib) and AZD8055 worked synergistically to inhibit tumor growth in xenograft models of rhabdomyosarcoma (PMID: 23918606). 23918606
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Dacarbazine + Selumetinib Phase I Actionable In a Phase I trial, the combination of Koselugo (selumetinib) and Deticene (dacarbazine) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with partial responses in 17% (4/23) and stable disease for greater than 6 weeks in 65% (15/23) of patients (PMID: 28264648). 28264648
Unknown unknown Hodgkin's lymphoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Darinaparsin + Selumetinib Preclinical - Cell culture Actionable In a preclinical study, Darinaparsin treatment in combination with Koselugo (selumetinib) inhibited Erk1/2 phosphorylation and led to enhanced apoptosis in Hodgkin's lymphoma cells compared to Darinaparsin treatment alone in culture (PMID: 25316819). 25316819
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Docetaxel + Selumetinib Phase I Actionable In a Phase I trial, the combination of Koselugo (selumetinib) and Taxotere (docetaxel) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with partial responses in 22% (6/27) and stable disease for greater than 6 weeks in 52% (14/27) of patients (PMID: 28264648). 28264648
Unknown unknown triple-receptor negative breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor KW-2450 + Selumetinib Preclinical Actionable In a preclinical study, the combination of KW-2450 and Selumetinib worked synergistically to inhibit growth of triple-negative breast cancer cells in culture (PMID: 26443806). 26443806
Unknown unknown pancreatic adenocarcinoma no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor MK2206 + Selumetinib Phase II Actionable In a Phase II trial, the combination of Selumetinib (AZD6244) and MK2206 did not result in improved median overall survival compared to mFOLFOX therapy (3.9 mo vs. 6.7 mo) or improved median progression-free survival (1.9 mo vs 2.0 mo) in patients with pancreatic adenocarcinoma (PMID: 27978579). 27978579
Unknown unknown triple-receptor negative breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the Mek inhibitor Selumetinib (AZD6244) inhibited tumorigenicity and invasiveness of triple-receptor negative breast cancer cell lines in culture and in cell line xenograft models (PMID: 26384399). 26384399
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Clinical Study Actionable In a meta-analysis, Selumetinib (AZD6244) was shown to have modest clinical efficacy as a monotherapy in a variety of solid tumors (PMID: 24716986). 24716986
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Phase I Actionable In a Phase I trial, treatment with Selumetinib (AZD6244) demonstrated safety and preliminary efficacy patients with advanced solid tumors, with several patients achieving prolonged stable disease (PMID: 18390968). 18390968
Unknown unknown multiple myeloma no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Phase II Actionable In a Phase II clinical trial, Selumetinib (AZD6244) demonstrated safety and tolerability but had minimal activity with a complete response achieved in 5.6% (2/36) of refractory multiple myeloma patients (PMID: 26446942). 26446942
Unknown unknown breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Phase I Actionable In a Phase I trial, treatment with Selumetinib (AZD6244) demonstrated safety and preliminary efficacy patients with advanced solid tumors, including patients with breast cancer, with several patients achieving prolonged stable disease (PMID: 18390968). 18390968
Unknown unknown uveal melanoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib Phase II Actionable In a Phase II trial, Koselugo (selumetinib) improved median progression-free survival (15.9 vs 7 weeks, HR=0.46, p<0.001) but not overall survival (11.8 vs 9.1 months, HR=0.66, p=0.09) compared to chemotherapy in patients with uveal melanoma, but also caused high adverse event rate (PMID: 24938562; NCT01143402). 24938562
Unknown unknown plexiform neurofibroma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib FDA approved Actionable In a Phase II trial (SPRINT) that supported FDA approval, Koselugo (selumetinib) treatment resulted in an objective response rate of 70% (35/50) in pediatric patients 2 years or older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas, with 28 of the responses lasted over 1 year (PMID: 32187457; NCT01362803). 32187457
Unknown unknown pancreatic ductal adenocarcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib + SHP099 Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of SHP099 and Selumetinib (AZD6244) resulted in greater sensitivity compared to either agent alone in pancreatic ductal adenocarcinoma cells, demonstrating decreased cell viability and reduced colony formation in culture and decreased tumor growth in patient-derived xenograft (PDX) models (PMID: 30045908). 30045908
Unknown unknown triple-receptor negative breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib + SHP099 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of SHP099 and Selumetinib (AZD6244) led to decreased cell viability and reduced colony formation in triple-receptor negative breast cancer cells in culture and tumor regression in xenograft models (PMID: 30045908). 30045908
Unknown unknown ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Selumetinib + SHP099 Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Selumetinib (AZD6244) and SHP099 resulted in decreased colony formation and reduced cell viability in ovarian cancer cells in culture and inhibition of tumor growth and reduced tumor angiogenesis in a patient-derived xenograft (PDX) model of ovarian cancer (PMID: 30045908). 30045908
Unknown unknown hepatocellular carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) 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 colorectal cancer not applicable MEK inhibitor (Pan) HL-085 + OKI-005 Preclinical - Cell culture Actionable In a preclinical study, HL085 and OKI-005 demonstrated synergistic activity in 3 of 6 colorectal cancer cell lines, and increased immunogenicity of tumor cells in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 4753). detail...
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor AZD8330 Phase I Actionable In a Phase I trial, AZD8330 demonstrated safety and some preliminary clinical activity in patients with advanced solid tumors (PMID: 23433846). 23433846
Unknown unknown pancreatic adenocarcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Pimasertib Phase I Actionable In a Phase I trial, Pimasertib in combination with Gemzar (gemcitabine) demonstrated safety and efficacy in metastatic pancreatic adenocarcinoma patients (PMID: 23846936). 23846936
Unknown unknown pancreatic cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Gemcitabine + Pimasertib Preclinical Actionable In a preclinical study, treatment with Pimasertib (MSC1936369B) followed by Gemzar (gemcitabine) resulted in enhanced inhibition of proliferation and induction of apoptosis in pancreatic cell lines in culture (PMID: 26228206). 26228206
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pimasertib Phase I Actionable In a Phase I trial, Pimasertib (MSC1936369B) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (J Clin Oncol 28:15s, 2010 (suppl; abstr 2504)). detail...
Unknown unknown ovarian cancer no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Pimasertib + Voxtalisib Phase II Actionable In a Phase II trial, the combination of Pimasertib (MSC1936369B) and XL765 (SAR245409) versus Pimasertib (MSC1936369B) alone resulted in an objective response rate (ORR) of 9.4% and 12.1%, and a median progression-free survival of 9.99 mo and 7.23 mo, respectively, in patients with ovarian carcinoma (n=65), and 18 pts treated with combination and 19 pts treated with single therapy discontinued the study, and thus, the study was terminated due to a low ORR and high rate of discontinuation (PMID: 31870556). 31870556
Unknown unknown thyroid gland cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 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 Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor TAK-733 Phase I Actionable In a Phase I clinical trial, TAK-733 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors with partial response in 5% (2/41) and stable disease in 37% (15/41) of patients (PMID: 27650277). 27650277 detail...
Unknown unknown colorectal cancer no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Atezolizumab + Cobimetinib Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment resulted in partial response in 7% (7/84) of patients with metastatic colorectal cancer, with a median duration of response of 14.8 months, a disease control rate of 31% (26/84), a median progression-free survival of 1.9 months, and a median overall survival of 10.0 months (PMID: 30918950; NCT01988896). 30918950
Unknown unknown colorectal cancer no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Atezolizumab + Cobimetinib Phase III Actionable In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment did not improve median overall survival (8.87 vs 8.51 months, HR=1.00, p=0.99) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (PMID: 31003911; NCT02788279). 31003911
Unknown unknown melanoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Atezolizumab + Cobimetinib Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment demonstrated safety and preliminary clinical activity, resulted in a confirmed response in 41% (9/22) of patients with melanoma, regardless of KRAS/BRAF status (PMID: 30918950; NCT01988896). 30918950
Unknown unknown lung non-small cell carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Atezolizumab + Cobimetinib Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment demonstrated safety and preliminary clinical activity, resulted in a confirmed response in 18% (5/28) of patients with non-small cell lung cancer, regardless of KRAS/BRAF status (PMID: 30918950; NCT01988896). 30918950
Unknown unknown melanoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Cobimetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Cobimetinib (GDC-0973) induced cell death in several human melanoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22084396). 22084396
Unknown unknown lymphatic system cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Cobimetinib Phase II Actionable In a Phase II trial, treatment with Cotellic (cobimetinib) in patients with histiocytic neoplasms resulted in a PET overall response rate of 89% (16/18), with complete response in 72% (13/18) and partial response in 17% (3/18), and resulted in stable disease in 6% (1/18) of patients, and at 11.9 months the median duration of response and progression-free survival were not yet reached (PMID: 30867592; NCT01953926). 30867592
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Cobimetinib Phase I Actionable In a Phase I trial, Cotellic (cobimetinib) treatment resulted in stable disease for five months or more in five patients with advanced solid tumors (PMID: 27424159). 27424159 detail...
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Cobimetinib + Ipatasertib Phase I Actionable In a Phase I clinical trial Ipatasertib (GDC-0068), in combination with the Mek inhibitor Cobimetinib (GDC-0973) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res, October 1, 2014 74; CT328). detail...
Unknown unknown Advanced Solid Tumor no benefit MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor Cobimetinib + Ravoxertinib Phase I Actionable In a Phase Ib trial, treatment with the combination of Ravoxertinib (GDC-0994) and Cotellic (cobimetinib) in patients with advanced solid tumors resulted in stable disease in 29% (7/24) of patients and one unconfirmed partial response in a patient with pancreatic adenocarcinoma; however, the study was terminated due to intolerable toxicity of the treatment combination observed in patients (PMID: 32311798; NCT02457793). 32311798
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor CI-1040 Phase I Actionable In a Phase I trial, CI-1040 treatment resulted in median stable disease for 5.5 months in 28% (19/66) of patients with advanced solid tumors (including colorectal, non-small-cell lung, pancreatic, melanoma, and kidney) and partial response in 1 pancreatic cancer patient (PMID: 16009947). 16009947
Unknown unknown glioblastoma multiforme not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor BI2536 + PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, the combination of BI 2536 and PD-0325901 resulted in greater inhibition of cell proliferation in glioblastoma cells in culture compared to treatment with either agent alone (PMID: 26573800). 26573800
Unknown unknown breast cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy, however, the dose administered resulted in a significant degree of toxicity (PMID: 21516509). 21516509
Unknown unknown colorectal cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PD-0325901 Phase I Actionable In a Phase I study, PD-325901 demonstrated efficacy but toxicity at current dose was unacceptable (PMID: 21516509). 21516509
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PD-0325901 Phase I Actionable In a Phase I clinical trial, PD-0325901 demonstrated preliminary clinical activity in patients with advanced solid tumors, however late-onset dose-limiting toxicities were encountered (PMID: 20215549). 20215549
Unknown unknown melanoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy in previously treated melanoma patients, however, there was significant toxicity above 10 mg BID (PMID: 21516509). 21516509
Unknown unknown urinary bladder cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PD-0325901 + PF-04691502 Preclinical - Pdx Actionable In a preclinical study, PD-0325901, in combination with PF-04691502, delayed tumor growth in patient-derived xenograft models of bladder cancer (PMID: 24442130). 24442130
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) RO5126766 Phase I Actionable In a Phase I trial, RO5126766 demonstrated safety and preliminary efficacy in patients with a variety of solid tumors (PMID: 22761467). 22761467
Unknown unknown Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor RAF Inhibitor (Pan) RO5126766 Phase I Actionable In a Phase I trial, RO5126766 demonstrated safety and some preliminary activity in Japanese patients with advanced solid tumors, with 42% (5/12) of patients achieving stable disease (PMID: 23860959). 23860959
Unknown unknown colon cancer not applicable RAF Inhibitor (Pan) AZ628 + CPI-455 Preclinical - Cell culture Actionable In a preclinical study, a colon cancer cell line treated with AZ628 demonstrated increased sensitivity when co-treated with CPI-455 in culture, resulting in decreased survival of cells, in particular the cells that eventually develop drug resistance (PMID: 27214401). 27214401
Unknown unknown Advanced Solid Tumor no benefit RAF Inhibitor (Pan) LY3009120 Phase I Actionable In a Phase I trial, LY3009120 did not achieve expected pharmacodynamic effects, resulted in stable disease as best overall response in 15.7% (8/51) of patients with advanced or metastatic cancer, and demonstrated no target inhibition (PMID: 31645440; NCT02014116). 31645440
Unknown unknown clear cell renal cell carcinoma no benefit RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 renal cell carcinoma not applicable RAF Inhibitor (Pan) 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 Advanced Solid Tumor not applicable RAF Inhibitor (Pan) 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 hepatocellular carcinoma not applicable RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 triple-receptor negative breast cancer not applicable RAF Inhibitor (Pan) 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 breast cancer not applicable RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 Advanced Solid Tumor not applicable RAF Inhibitor (Pan) 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 hepatocellular carcinoma not applicable RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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 cancer not applicable RAF Inhibitor (Pan) 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 thyroid gland medullary carcinoma not applicable RAF Inhibitor (Pan) 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 Her2-receptor negative breast cancer not applicable RAF Inhibitor (Pan) 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 renal cell carcinoma not applicable RAF Inhibitor (Pan) 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 thyroid gland carcinoma not applicable RAF Inhibitor (Pan) 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 lung non-small cell carcinoma no benefit RAF Inhibitor (Pan) 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 hepatocellular carcinoma not applicable RAF Inhibitor (Pan) Sorafenib Case Reports/Case Series Actionable In a Phase I trial, three hepatocellular carcinoma patients treated with Nexavar (sorafenib) following CEBPA-51 (MTL-CEBPA) treatment achieved complete radiologic response (PMID: 32357963; NCT02716012). 32357963
Unknown unknown hepatocellular carcinoma not applicable RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) Sorafenib Preclinical Actionable In preclinical studies, Nexavar (sorafenib) promoted apoptosis of endometrial carcinoma cells (PMID: 23463670). 23463670
Unknown unknown gastrointestinal stromal tumor not applicable RAF Inhibitor (Pan) 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 desmoid tumor not applicable RAF Inhibitor (Pan) 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 RAF Inhibitor (Pan) 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
Clinical Trial Phase Therapies Title Recruitment Status