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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 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 PIK3CA 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 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 Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 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 MEK inhibitor (Pan) MEK1 Inhibitor MEK2 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 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 ovarian cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PI3K Inhibitor (Pan) 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 PI3K Inhibitor (Pan) 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 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 PI3K Inhibitor (Pan) 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 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 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 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 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 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 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 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 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 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 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 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 efficacy in patients with advanced solid tumors (PMID: 22767668). 22767668
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 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 PI3K Inhibitor (Pan) 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 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 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 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 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 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 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 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 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 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 hepatocellular carcinoma not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 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 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 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 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 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 PI3K Inhibitor (Pan) 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 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 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 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 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 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 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 + 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 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 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 urinary bladder cancer not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor PI3K Inhibitor (Pan) 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 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 Advanced Solid Tumor not applicable MEK inhibitor (Pan) MEK1 Inhibitor MEK2 Inhibitor 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 colorectal cancer not applicable PI3K Inhibitor (Pan) R11.1.6 + ZSTK474 Preclinical - Cell culture Actionable In a preclinical study, R11.1.6 and ZSTK474 combination therapy did not inhibit cell proliferation in a colorectal cancer cell line in culture (PMID: 29720559). 29720559
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) ZSTK474 Preclinical - Cell line xenograft Actionable In a preclinical study, ZSTK474 inhibited cell proliferation of a variety of human advanced solid tumor cell lines in culture and in xenograft models (PMID: 25483727). 25483727
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 was well-tolerated and resulted in some efficacy in patients with breast cancer, including stable disease in 31% (7/22) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown renal cell carcinoma not applicable PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in renal cell carcinoma patients including stable disease in 13% (3/24), one patient with a complete response, and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown endometrial cancer not applicable PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in endometrial cancer patients including stable disease in 27% (4/15) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3018). detail...
Unknown unknown urinary bladder cancer not applicable PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In Phase I trial, GSK2126458 treatment was well-tolerated and resulted in a partial response and stable disease in two patients and one patient with bladder cancer, respectively (PMID: 26603258). 26603258
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Seribantumab + XL147 Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Pilaralisib (SAR245408, XL147) and Seribantumab (SAR256212) resulted in stable disease as best response in 52.2% (12/23) patients with advanced solid tumors, with no difference in response between patients harboring PIK3CA mutations and those with wild-type PIK3CA (PMID: 28031425). 28031425
Unknown unknown endometrial carcinoma not applicable PI3K Inhibitor (Pan) XL147 Phase II Actionable In a Phase II trial, Pilaralisib (XL147) treatment resulted in an objective response rate of 6% (4/67) in patients with endometrial carcinoma, although anti-tumor activity is not associated with molecular alterations in PTEN and PIK3R1 (PMID: 25528496). 25528496
Unknown unknown colon adenocarcinoma not applicable PI3K Inhibitor (Pan) XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 (SAR245408) demonstrated modest efficacy as a single agent in human cancer cell line xenograft models (PMID: 23002019). 23002019
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) XL147 Phase I Actionable In a Phase I trial, 43.9% (25/56) of patients with advanced solid tumors had stable disease as a best response to treatment with Pilaralisib (XL147), and one patient with NSCLC showed a partial response, and in a separate therapy arm, Pilaralisib (XL147) in tablet formulation demonstrated safety and some antitumor activity in advanced solid tumor patients, including 11.1% (2/18) of patients with a partial response (PMID: 29593099, PMID: 24166903; NCT00486135). 29593099 24166903
Unknown unknown lung non-small cell carcinoma not applicable PI3K Inhibitor (Pan) XL147 Phase I Actionable In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). 24166903
Unknown unknown melanoma not applicable PI3K Inhibitor (Pan) GNE-317 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line xenograft model demonstrated cell death of metastasized tumor cells within a small region of the brain when treated with GNE-317 (PMID: 27521448). 27521448
Unknown unknown stomach cancer not applicable PI3K Inhibitor (Pan) Cisplatin + Deguelin Preclinical Actionable In a preclinical study, the combination of Deguelin and Platinol (cisplatin) worked synergistically to inhibit growth of gastric cancer cells in culture (PMID: 25202376). 25202376
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited growth of several solid tumor cell lines in culture and in cell line xenograft models (PMID: 25700704). 25700704
Unknown unknown malignant glioma not applicable PI3K Inhibitor (Pan) GDC-0084 Phase I Actionable In a Phase I trial, GDC-0084 treatment demonstrated expected toxicity and blood-brain barrier penetration, resulted in stable disease as best response in 40% (19/47) of patients with recurrent high-grade glioma (J Clin Oncol (PMID: 31937616; NCT01547546). 31937616
Unknown unknown ovarian clear cell adenocarcinoma not applicable PI3K Inhibitor (Pan) DS-7423 Preclinical - Cell line xenograft Actionable In a preclinical study, DS-7423 inhibited proliferation of ovarian clear cell adenocarcinoma cell lines in culture and suppressed tumor growth in cell line xenograft animal models (PMID: 24504419). 24504419
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) DS-7423 Phase I Actionable In a Phase I trial, DS-7423 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors (Ann Oncol (2014) 25 (suppl 4): iv153). detail...
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) SRX3207 Preclinical - Cell culture Actionable In a preclinical study, SRX3207 treatment induced cytotoxicity in Lewis lung carcinoma cells in culture, and inhibited tumor growth, and increased survival in a syngeneic mouse model (PMID: 31974273). 31974273
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Gedatolisib Phase I Actionable In a Phase I trial, Gedatolisib (PF-05212384) demonstrated safety and efficacy, which resulted in antitumor activity and stable disease greater than six months in 10.4% (8/27) of patients with solid malignant tumors (PMID: 25652454). 25652454
Unknown unknown head and neck squamous cell carcinoma not applicable PI3K Inhibitor (Pan) Gedatolisib Preclinical - Cell line xenograft Actionable In a preclinical study, Gedatolisib (PF-05212384) increased the the sensitivity of head and neck squamous cancer cells to radiation as indicated by decreased downstream signaling of the PI3K/mTOR pathway and reduced growth both in culture and in cell line xenograft models (PMID: 25724523). 25724523
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) Gedatolisib Preclinical - Cell line xenograft Actionable In a preclinical study, Gedatolisib (PF-05212384) suppressed phosphorylation of PI3K/mTOR effectors and induced apoptosis in human breast cancer cell lines with elevated PI3K/mTOR signaling in culture and in cell line xenograft models (PMID: 21325073). 21325073
Unknown unknown colorectal cancer not applicable PI3K Inhibitor (Pan) Gedatolisib + Irinotecan Case Reports/Case Series Actionable In a Phase I trial, treatment with the combination of Gedatolisib (PF-05212384) and Camptosar (irinotecan) resulted in an overall response rate of 4.7%, with 2 colorectal cancer patients achieving a partial response, and clinical benefit rate of 16.3% in an advanced solid tumor patient cohort comprising 93% colorectal cancer patients (PMID: 29067643; NCT01347866)). 29067643
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) PF-04691502 Phase I Actionable In a Phase I trial, PF-04691502 demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 24395457). 24395457
Unknown unknown myelodysplastic syndrome no benefit PI3K Inhibitor (Pan) Rigosertib Sodium Phase III Actionable In a Phase III trial (ONTIME), Rigosertib (ON 01910.Na) did not improve median overall survival compared to best supportive care (8.2 vs 5.9 months, HR=0.87, p=0.33) in patients with myelodysplastic syndrome (PMID: 26968357; NCT01241500). 26968357
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Rigosertib Sodium Phase I Actionable In a Phase I study, Rigosertib (ON 01910.Na) demonstrated both safety and preliminary efficacy, resulted in a best overall response of stable disease in 41% (9/22) of patients with advanced solid tumors (PMID: 23841031; NCT01538537). 23841031
Unknown unknown colorectal cancer not applicable PI3K Inhibitor (Pan) R11.1.6 + Wortmannin Preclinical - Cell culture Actionable In a preclinical study, R11.1.6 and Wortmannin combination therapy did not inhibit cell proliferation in a colorectal cancer cell line in culture (PMID: 29720559). 29720559
Unknown unknown T-cell acute lymphoblastic leukemia not applicable PI3K Inhibitor (Pan) BGT226 Preclinical - Cell culture Actionable In a preclinical study, BGT226 treatment inhibited viability of NUP214-ABL1 fusion-positive T-cell acute lymphoblastic leukemia cell lines in culture (PMID: 27821800). 27821800
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) BGT226 Phase I Actionable In a Phase I trial of BGT226 in patients with advanced solid tumors, limited preliminary antitumor activity and inconsistent target inhibition were observed (PMID: 22357447). 22357447
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) BGT226 Phase I Actionable In a Phase I trial, BGT226 treatment was tolerated, and resulted in stable disease as best response in 28% (5/18) of patients with advanced solid tumors (PMID: 31192075). 31192075
Unknown unknown Ewing sarcoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with Ewing sarcoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown hematologic cancer not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment was well-tolerated, demonstrated safety, and resulted in stable disease in 57% (21/37) of patients with a hematological cancer (PMID: 27049457). 27049457
Unknown unknown diffuse large B-cell lymphoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 was well-tolerated, demonstrated safety, and resulted in a 56% (5/9) objective response rate, including two complete responses and three partial responses, in patients with diffuse large B-cell lymphoma (PMID: 27049457). 27049457
Unknown unknown pediatric ependymoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with ependymoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown non-Hodgkin lymphoma not applicable PI3K Inhibitor (Pan) CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited cell growth in a human non-Hodgkin lymphoma cell line in culture, and stabilized tumor growth in xenograft models (PMID: 22693356). 22693356
Unknown unknown renal cell carcinoma not applicable PI3K Inhibitor (Pan) GDC-0980 Phase II Actionable In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) (median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively) and overall survival trended lower in the Apitolisib (GDC-0980) arm in patients with metastatic renal cell carcinoma (PMID: 26951309). 26951309
Unknown unknown colorectal cancer not applicable PI3K Inhibitor (Pan) GDC-0980 Preclinical - Cell line xenograft Actionable In a preclinical study, the dual PI3K/mTOR inhibitor Apitolisib (GDC-0980) reduced vascularization in cell line xenograft models of colorectal cancer (PMID: 23814482). 23814482
Unknown unknown malignant pleural mesothelioma not applicable PI3K Inhibitor (Pan) GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in a partial response in 7.4% (2/27) and stable disease in 74.1% (20/27) of malignant pleural mesothelioma patients, including one with PTEN loss and another with PIK3CA E545K (PMID: 26787751). 26787751
Unknown unknown peritoneal mesothelioma not applicable PI3K Inhibitor (Pan) GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression in two patients with peritoneal mesothelioma (PMID: 26787751). 26787751
Unknown unknown ovarian clear cell carcinoma not applicable PI3K Inhibitor (Pan) GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression by 48.3% in a patient with ovarian clear cell carcinoma (PMID: 26787751). 26787751
Unknown unknown prostate cancer no benefit PI3K Inhibitor (Pan) Abiraterone + Dactolisib Phase I Actionable In a Phase Ib trial, the combination of Zytiga (abiraterone) and Dactolisib (BEZ235) did not demonstrate positive safety and tolerability, and further study of this combination is not planned (PMID: 28282611; NCT01634061). 28282611
Unknown unknown leiomyosarcoma not applicable PI3K Inhibitor (Pan) Aldoxorubicin + Dactolisib Preclinical Actionable In a preclinical study, the combination of doxorubicin and BEZ235 produced a synergistic effect in leiomyosarcoma cells both in culture and in mouse models, resulting in apoptotic induction and a 68% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown endometrial cancer not applicable PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
Unknown unknown leiomyosarcoma not applicable PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, leiomyosarcoma cells treated with BEZ235 in culture and in mouse models demonstrated increased levels of apoptosis and a 42% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown peritoneal mesothelioma not applicable PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, BEZ235 treatment resulted in suppression of PI3K and mTOR signaling and growth inhibition in patient-derived malignant peritoneal mesothelioma cell lines in culture (PMID: 20839315). 20839315
Unknown unknown prostate carcinoma not applicable PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, treatment with BEZ235 resulted in a decrease in prostate cancer progenitor cells in culture and reduced tumor growth in prostate carcinoma xenograft models (PMID: 21138868). 21138868
Unknown unknown pancreatic endocrine carcinoma no benefit PI3K Inhibitor (Pan) Dactolisib Phase II Actionable In a Phase II clinical trial, BEZ235 was not well-tolerated and demonstrated modest anti-tumor activity in pancreatic neuroendocrine tumor patients who had progressed on Afinitor (everolimus), with a 51.6% (16/31) progression-free survival rate at 16 weeks (PMID: 26851029). 26851029
Unknown unknown Advanced Solid Tumor no benefit PI3K Inhibitor (Pan) Dactolisib + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in limited efficacy in patients with advanced solid tumors and was discontinued due to multiple intolerable side effects (PMID: 28357727). 28357727
Unknown unknown astrocytoma not applicable PI3K Inhibitor (Pan) Dactolisib + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in stable disease in a patient with astrocytoma (PMID: 28357727). 28357727
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) Dactolisib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with BEZ235 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 prostate cancer not applicable PI3K Inhibitor (Pan) Abiraterone + PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) and Zytiga (abiraterone) combination treatment was tolerated, resulted in stable disease as best response in 54.5% (6/11) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer who demonstrated early Zytiga (abiraterone) resistance, no PSA response (0/25) was observed, and 24% (6/25) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) treatment was tolerated, resulted in a partial response in 11% (2/18) and stable disease in 56% (10/18) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer, PSA response was observed in 2% (1/43) of evaluable patients, and 32.6% (14/43) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown Advanced Solid Tumor not applicable PI3K 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 prostate cancer no benefit PI3K Inhibitor (Pan) Abiraterone + Buparlisib Phase I Actionable In a Phase Ib trial, the combination of Zytiga (abiraterone) and Buparlisib (BKM120) did not demonstrate significant clinical activity in patients with castration-resistant prostate cancer, resulting in a best overall response of stable disease in 15% (3/20) patients treated at the 100 mg QD Buparlisib (BKM120) dose level, and further study of this combination is not planned (PMID: 28282611; NCT01634061). 28282611
Unknown unknown leiomyosarcoma not applicable PI3K Inhibitor (Pan) Aldoxorubicin + Buparlisib Preclinical Actionable In a preclinical study, the combination of BKM120 and doxorubicin produced an additive effect when treating leiomyosarcoma cells (PMID: 26952093). 26952093
Unknown unknown glioblastoma multiforme not applicable PI3K Inhibitor (Pan) BLZ945 + Buparlisib Preclinical Actionable In a preclinical study, the combination of Buparlisib (BKM120) and BLZ945 compared to BLZ945 alone resulted in enhanced survival and tumor regression in transgenic mouse models of glioblastoma (PMID: 27199435). 27199435
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Buparlisib Phase I Actionable In a Phase I trial, Buparlisib (BKM120) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22162589). 22162589
Unknown unknown glioblastoma multiforme no benefit PI3K Inhibitor (Pan) Buparlisib Preclinical Actionable In a preclinical study, treatment with BKM120 demonstrated a survival similar to vehicle in transgenic mouse models of glioblastoma and therefore, resulted in no benefit (PMID: 27199435). 27199435
Unknown unknown transitional cell carcinoma not applicable PI3K Inhibitor (Pan) Buparlisib Phase II Actionable In a Phase II trial, Buparlisib (BKM120) demonstrated unfavorable toxicity profile and modest activity in patients with platinum-refractory metastatic urothelial carcinoma, resulting in a partial response in 7.7% (1/13) and stable disease in 46.2% (6/13) of the patients, which did not meet the cutoff to proceed the trial (PMID: 32767682; NCT01551030). 32767682
Unknown unknown head and neck squamous cell carcinoma not applicable PI3K Inhibitor (Pan) Buparlisib Preclinical Actionable In a preclinical study, Buparlisib (BKM-120) reduced viability of head and neck squamous cell carcinoma (HNSSC) cells in culture and demonstrated anti-tumor activity in HNSSC xenograft models (PMID: 22116303). 22116303
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) Buparlisib + Everolimus Preclinical Actionable In a preclinical study, Buparlisib (BKM120) in combination with Afinitor (everolimus) inhibited tumor growth in mouse lung cancer xenograft models (PMID: 22781393). 22781393
Unknown unknown follicular lymphoma not applicable PI3K Inhibitor (Pan) Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 20% (1/5, 1 complete response) in patients with relapsed/refractory follicular lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable PI3K Inhibitor (Pan) Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 31% (4/13, 3 complete response, 1 partial response) in patients with relapsed/refractory diffuse large B-cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown mantle cell lymphoma not applicable PI3K Inhibitor (Pan) Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 88% (15/17, 11 complete response, 4 partial response) in patients with relapsed/refractory mantle cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) PKI-402 Preclinical - Cell culture Actionable In a preclinical study, PKI-402 inhibited growth of several human solid tumor cell lines in culture (PMID: 20371716). 20371716
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Voxtalisib Phase I Actionable In a Phase I trial, SAR245409 (XL765) demonstrated safety and efficacy in patients with solid tumors (PMID: 18959794). 18959794
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Voxtalisib Phase I Actionable In a Phase I trial, SAR245409 (XL765) reduced PI3K and mTORC1/mTORC2 pathway signaling and demonstrated safety and efficacy irrespective of molecular alterations in the PI3K pathway, in patients with advanced solid tumors (PMID: 24583798). 24583798
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 inhibited growth of a variety of human tumor cell lines in culture and inhibited tumor growth in cell line xenograft models (PMID: 23270925). 23270925
Unknown unknown ovarian cancer not applicable PI3K Inhibitor (Pan) VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 reduced the proportion of cancer stem cells in primary ovarian tumors in culture, and reduced tumorigenicity of dissociated human ovarian tumors in xenograft models (PMID: 25432176). 25432176
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) VS-5584 Preclinical - Patient cell culture Actionable In a preclinical study, treatment with VS-5584 resulted in decreased cancer stem cell (CSC) number in a triple-negative breast cancer cell line in culture and in xenograft models, and decreased CSCs in patient breast cancer tumor samples in culture (PMID: 25432176). 25432176
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) SF1126 Preclinical Actionable In a preclinical study, SF1126 decreased tumor immunosuppression and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown hepatocellular carcinoma not applicable PI3K Inhibitor (Pan) SF1126 Preclinical - Cell line xenograft Actionable In a preclinical study, SF1126 inhibited proliferation of hepatocellular carcinoma cell lines in culture, and inhibited tumor growth in hepatocellular carcinoma cell line xenograft models (PMID: 27496136). 27496136
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) SF1126 Phase I Actionable In a Phase I trial, SF1126 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 22921184). 22921184
Unknown unknown hepatocellular carcinoma not applicable PI3K 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 Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) AZD8055 Phase I Actionable In a Phase I trial, AZD8055 treatment demonstrated safety and tolerability, and resulted in stable disease for more than 4 months in 14% (7/49) of patients with advanced solid tumors or lymphoma (PMID: 22935583). 22935583
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) AZD8055 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA (H1047R or E545K) and PTEN mutations demonstrated sensitivity to AZD8055 treatment in culture (PMID: 31879363). 31879363
Unknown unknown lung non-small cell carcinoma not applicable PI3K Inhibitor (Pan) AZD8055 + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, the addition of SBI-0206965 to treatment with AZD8055 resulted in increased apoptosis in a non-small cell lung cancer cell line in culture (PMID: 26118643). 26118643
Unknown unknown diffuse large B-cell lymphoma not applicable PI3K Inhibitor (Pan) Ibrutinib + PQR309 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Imbruvica (ibrutinib) led to a synergistic effect in 6/7 diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and the effect was confirmed in a DLBCL cell line xenograft model (PMID: 29066507). 29066507
Unknown unknown lymphoma not applicable PI3K Inhibitor (Pan) Panobinostat + PQR309 Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Farydak (panobinostat) and PQR309 induced apoptosis and led to synergistic and additive effects in lymphoma cell lines in culture (PMID: 29066507). 29066507
Unknown unknown lymphoma not applicable PI3K Inhibitor (Pan) PQR309 Preclinical - Cell culture Actionable In a preclinical study, PQR309 inhibited proliferation of lymphoma cells in culture, which was found to be due to the induction cell cycle arrest (PMID: 29066507). 29066507
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) PQR309 Phase I Actionable In a Phase I trial, PQR309 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2560)). detail...
Unknown unknown lymphoma not applicable PI3K Inhibitor (Pan) PQR309 + Rituximab Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of PQR309 and Rituxan (rituximab) led to a synergistic effect in 2/5 lymphoma cell lines in culture (PMID: 29066507). 29066507
Unknown unknown lymphoma not applicable PI3K Inhibitor (Pan) PQR309 + Venetoclax Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Venclexta (venetoclax) led to antitumor activity in lymphoma cells in culture and cell line xenograft models, demonstrating both synergistic and additive effects (PMID: 29066507). 29066507
Unknown unknown endometrial cancer not applicable PI3K Inhibitor (Pan) LY3023414 Phase II Actionable In a Phase II trial, patients with advanced endometrial cancer harboring a PI3K pathway mutation demonstrated a best overall response rate of 16% (4/25), a clinical benefit rate of 28% (7/25) at 12 weeks, a progression-free survival of 2.5 months, and overall survival of 9.2 months when treated with LY3023414 (PMID: 31880826; NCT01775072). 31880826
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) LY3023414 Phase I Actionable In a Phase I trial, LY3023414 demonstrated tolerability and inhibition of PI3K/mTOR signaling and resulted a disease control rate of 34.0% (16/47) in patients with advanced solid tumors, with one partial response and 15 patients achieving stable disease (PMID: 29636360; NCT01655225). detail... 29636360
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Erlotinib + Pictilisib Phase I Actionable In a Phase I trial, the combination treatment of Tarceva (erlotinib) and Pictilisib (GDC-0941) in patients with advanced solid tumors resulted in toxicity, requiring dose adjustments, and led to minimal antitumor activity including two partial responses and stable disease in nineteen patients (PMID: 28798270). 28798270
Unknown unknown lung small cell carcinoma not applicable PI3K Inhibitor (Pan) Navitoclax + Pictilisib Preclinical - Pdx Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and GDC-0941 delayed tumor growth in a circulating tumor cell (CTC)-derived xenograft model derived using CTCs from a small cell lung cancer patient (PMID: 27197306). 27197306
Unknown unknown islet cell tumor not applicable PI3K Inhibitor (Pan) Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) inhibited AKT activation, reduced tumor burden, and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CLR457 Phase I Actionable In a Phase I trial, CLR457 treatment demonstrated limited efficacy, resulting in no confirmed responses, stable disease in 25.8% (8/31) , and non-complete response/non-progressive disease in 6.5% (2/31) of patients with advanced solid tumors with PI3K pathway activation (PMID: 30073466; NCT02189174). 30073466
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CLR457 Preclinical - Pdx Actionable In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). 26479923
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, increased T-lymphocyte infiltration, and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown melanoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 reduced tumor growth and decreased lung metastasis in syngeneic mouse melanoma models (PMID: 31018997). 31018997
Unknown unknown colon adenocarcinoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 increased T-lymphocyte infiltration and reduced tumor growth in syngeneic mouse colon adenocarcinoma models (PMID: 31018997). 31018997
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, and inhibited tumor growth and progression in a mouse breast cancer model (PMID: 31018997). 31018997
Unknown unknown pancreatic cancer not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). 31018997
Unknown unknown head and neck squamous cell carcinoma not applicable PI3K Inhibitor (Pan) GSK1059615 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1059615 treatment in a head and neck squamous cell carcinoma cell line resulted in inhibition of both cell growth and cell proliferation and induced necrosis in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28187451). 28187451
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited PI3K and mTOR activation and inhibited growth of several human tumor cell lines in culture and in xenograft models (Mol Cancer Ther November 2013 12; C270). detail...
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown leukemia not applicable PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited tumor growth greater than 65% in a leukemia cell line xenograft model (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown lung non-small cell carcinoma not applicable PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, non-small cell lung carcinoma cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CH5132799 Phase I Actionable In a Phase I trial, CH5132799 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25231405). 25231405
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) X480 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with X480 in prostate cancer cell lines resulted in reduced cell proliferation and induced apoptotic activity in culture, and decreased tumor load and metastasis in cell line xenograft models (Eur J Cancer, 2012, 48, S5:235). detail...
Unknown unknown mantle cell lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 17% (1/6) and partial response in 67% (4/6) of patients with mantle cell lymphoma (PMID: 24852792). 24852792
Unknown unknown lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 20% (1/5) and stable disease in 40% (2/5) of patients with transformed lymphoma (PMID: 24852792). 24852792
Unknown unknown lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, treatment with Aliqopa (copanlisib) in patients with indolent lymphoma resulted in an objective response rate of 59% (84/142), including a complete response in 12%, and demonstrated a median duration of response of 22.6 months and a median progression-free survival of 11.2 months (PMID: 28976790, PMID: 28633365; NCT01660451). 28633365 28976790
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Copanlisib Phase I Actionable In a Phase I clinical trial, treatment with Aliqopa (copanlisib) was well-tolerated and demonstrated preliminary activity in patients with advanced solid tumors, with complete response in 2% (1/48), partial response in 4% (2/48), and stable disease in 31% (15/48) of patients (PMID: 27672108). 27672108
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Copanlisib Phase I Actionable In a Phase I trial, Aliqopa (copanlisib) treatment resulted in no complete or partial response (0/10) and a disese control rate of 40% in patients with advanced solid tumors (PMID: 27915408). 27915408
Unknown unknown peripheral T-cell lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 50% (2/4) of patients with peripheral T-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown follicular lymphoma not applicable PIK3CA inhibitor Copanlisib FDA approved Actionable In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). detail... detail...
Unknown unknown follicular lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). 24852792
Unknown unknown chronic lymphocytic leukemia not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 67% (6/9) and stable disease in 22% (2/9) of patients with chronic lymphocytic leukemia (PMID: 24852792). 24852792
Unknown unknown diffuse large B-cell lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 6% (1/17), partial response in 6% (1/17) and stable disease in 41% (7/17) of patients with diffuse large B-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown breast cancer not applicable PIK3CA inhibitor CYH33 Preclinical - Cell culture Actionable In a preclinical study, CYH33 inhibited proliferation of 56% (18/32) of breast cancer cell lines in culture and demonstrated increased activity compared to Piqray (Alpelisib) (PMID: 30003928). 30003928
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib Phase I Actionable In a Phase I clinical trial, Alpelisib (BYL719) demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24617631). 24617631
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib Phase I Actionable In a Phase I clinical trial, Alpelisib (BYL719) treatment was well tolerated in Japanese patients with advanced solid tumors and resulted in a 3% (1/33) objective response rate and a disease control rate of 57.6% (19/33, 1 partial response, 18 stable disease); in patients with PIK3CA mutations or amplification disease control rates of 75.0% (6/8) were observed vs. 78.6% (11/14) in all patients (PMID: 30588709; NCT01387321). 30588709
Unknown unknown head and neck squamous cell carcinoma not applicable PIK3CA inhibitor Alpelisib + Cetuximab + Radiotherapy Phase II Actionable In a Phase Ib trial, 11 of 11 patients with metastatic head and neck squamous cell carcinoma demonstrated no evidence of disease at 3 to 4 months following treatment with Piqray (alpelisb) in combination with Erbitux (cetuximab) and intensity modulated radiation therapy (IMRT), and 10 of 11 remained disease-free at a median follow up of 23.5 months( (range: 8.4-37.7 months) from completion of radiation (PMID: 31678634). 31678634
Unknown unknown head and neck squamous cell carcinoma not applicable PIK3CA inhibitor Alpelisib + Cisplatin + Radiotherapy Phase I Actionable In a Phase I trial, Piqray (Alpelisib) treatment in combination with Platinol (cisplatin) and radiotherapy demonstrated manageable safety, and resulted in an objective response rate of 66.7% (6/9) in head and neck squamous cell carcinoma patients, the 3-year progression-free survival (PFS) and overall survival (OS) were 77.8%, and the median progression-free survival and overall survival were not reached (PMID: 32464516; NCT02537223). 32464516
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib + Infigratinib Phase I Actionable In a Phase Ib trial, Infigratinib (BGJ398) and Alpelisib (BYL719) combination treatment resulted in partial response in 25% (8/32) of patients with advanced solid tumors, including urothelial, head and neck, melanoma, and anal cancer (J Clin Oncol 34, 2016 (suppl; abstr 2500)). detail...
Unknown unknown ovary epithelial cancer not applicable PIK3CA inhibitor Alpelisib + Olaparib Phase I Actionable In a Phase Ib trial, Alpelisib (BYL719) and Lynparza (olaparib) combination therapy demonstrated safety and preliminary efficacy, resulted in partial response in 36% (10/28) and stable disease in 50% (14/28) of patients with epithelial ovarian cancer, overall response rate was similar for germline BRCA mutated and wild-type patients (30%, 3/10 vs 35%, 6/17, p=0.42) (PMID: 30880072; NCT01623349). 30880072
Unknown unknown breast cancer not applicable PIK3CA inhibitor MLN1117 Phase I Actionable In a Phase I trial, treatment with MLN1117 resulted in antitumor activity, demonstrating partial responses in patients with advanced solid tumors including breast and gastric cancer (J Clin Oncol, May 2015 vol. 33 no. 15_suppl 2501). detail...
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor MLN1117 + Sapanisertib Preclinical Actionable In a preclinical study, the combination of Sapanisertib (MLN0128) and MLN1117 demonstrated synergistic anti-tumor activity in a variety of solid tumor xenograft models (Mol Cancer Ther 2013;12(11 Suppl):C176)). detail...
Unknown unknown islet cell tumor not applicable PIK3CA inhibitor GDC-0326 Preclinical Actionable In a preclinical study, GDC-0326 inhibited AKT activation, decreased tumor growth, metastasis, and angiogenesis and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown Advanced Solid Tumor not applicable PIK3CA 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 PIK3CA inhibitor Enzalutamide + Taselisib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Taselisib (GDC-0032) and Xtandi (enzalutamide) resulted in a clinical benefit rate of 35.7% (5/14) in patients with triple-negative breast cancer, with partial response in one patient and stable disease in 4 patients, compared to no clinical benefit with Xtandi (enzalutamide) alone, and PIK3CA mutations and AR expression did not correlate with response (PMID: 31822498; NCT02457910). 31822498
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Taselisib Phase I Actionable In a Phase I trial, Taselisib (GDC-0032) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res Feb 2016 (76) (4 Supplement) P3-14-10). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable RAS Inhibitor (Pan) Gemcitabine + Pelareorep Phase II Actionable In a Phase II trial, Reolysin (pelareorep) in combination with Gemzar (gemcitabine) resulted in partial response in 3% (1/34), stable disease in 68% (23/34) of patients with advanced pancreatic ductal adenocarcinoma, with a median overall survival of 10.2 months, and a 1- and 2-year survival rate of 45% and 24% respectively (PMID: 29799479). 29799479
Unknown unknown Advanced Solid Tumor not applicable RAS Inhibitor (Pan) Pelareorep Phase I Actionable In a Phase I study, Reolysin (pelareorep) demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 22886613). 22886613
Unknown unknown Advanced Solid Tumor not applicable RAS Inhibitor (Pan) Salirasib Phase I Actionable In a Phase I clinical trial, Salirasib treatment was well tolerated in Japanese patients with advanced solid tumors (n=21) and resulted in a median progression-free survival of 53 days (PMID: 29992354). 29992354
Clinical Trial Phase Therapies Title Recruitment Status