Therapy Detail

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Therapy Name Avapritinib
Synonyms
Therapy Description

Avapritinib (BLU-285) is a selective inhibitor of PDGFRA D842V and KIT exon 17 mutants, including KIT D816V, potentially resulting in decreased tumor growth (AACR; 2015. Abstract nr 791).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Avapritinib Ayvakit BLU-285 KIT Inhibitor 50 PDGFR-alpha Inhibitor 9 Ayvakit (avapritinib) is a selective inhibitor of PDGFRA D842V and KIT exon 17 mutants, including KIT D816V, potentially resulting in decreased tumor growth (PMID: 30274985). Ayvakit (avapritinib) is FDA approved for use in patients with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including D842V (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FLT3 D835Y hematologic cancer resistant Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed hematologic cells expressing FLT3 D835Y were resistant to Ayvakit (avapritinib)-induced growth inhibition in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia resistant Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) did not inhibit Flt3 phosphorylation or growth of acute myeloid leukemia cell lines harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
KIT N822K acute myeloid leukemia sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of acute myeloid leukemia cells harboring KIT N822K in culture (PMID: 29093181). 29093181
KIT T670V KIT D816V Advanced Solid Tumor predicted - resistant Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670V demonstrated some resistance to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
KIT V560G KIT D816V mast cell neoplasm sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited proliferation of mast cells harboring KIT D816V and KIT V560G in culture (PMID: 31085175). 31085175
KIT V560G mast-cell leukemia sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of mast cell leukemia cells harboring KIT V560G in culture (PMID: 29093181). 29093181
RUNX1 - RUNX1T1 KIT N822K acute myeloid leukemia sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and growth of acute myeloid leukemia cell lines harboring KIT N822K and RUNX1-RUNX1T1 in culture (PMID: 31309543). 31309543
PDGFRA exon18 gastrointestinal stromal tumor sensitive Avapritinib Guideline Actionable Ayvakit (avapritinib) is included in the guidelines for patients with unresectable/metastatic gastrointestinal stromal tumor (GIST) with PDGFRA exon 18 mutations, including PDGFRA D842V (NCCN.org). detail...
PDGFRA exon18 gastrointestinal stromal tumor sensitive Avapritinib FDA approved Actionable In a Phase I trial (NAVIGATOR) that supported FDA approval, Ayvakit (avapritinib) treatment resulted in an objective response in 86% (37/43, 3 complete response, 34 partial response) and stable disease in 12% (5/43) of patients with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including D842V, with median duration of response not reached (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11022-11022; NCT02508532). detail... detail...
KIT wild-type megakaryocytic leukemia decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, KIT wild-type megakaryocytic leukemia cells were less sensitive to Ayvakit (avapritinib) compared to cells harboring KIT mutations in culture (PMID: 29093181). 29093181
KIT mutant gastrointestinal stromal tumor predicted - sensitive Avapritinib Phase I Actionable In a Phase I (NAVIGATOR) trial, Ayvakit (avapritinib) treatment resulted in an objective response rate of 13% (7/52, 7 partial responses) and a disease control rate of 63% (33/52) in patients with KIT-mutant gastrointestinal stromal tumor (The CTOS 2018 Annual Meeting, Nov 14-17, Rome Italy, Paper 012 3027631; NCT02508532). detail...
KIT N655K KIT D816V Advanced Solid Tumor predicted - sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT N655K demonstrated some sensitivity to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
FLT3 exon 14 ins FLT3 F691L hematologic cancer decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed hematologic cells expressing both FLT3 exon 14 insertions (ITD) and FLT3 F691L were less sensitive to Ayvakit (avapritinib)-induced growth inhibition compared to cells expressing FLT3 ITD in culture (PMID: 31309543). 31309543
KIT V560D KIT V654A Advanced Solid Tumor decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT V560D and KIT V654A demonstrated a decreased response to treatment with Ayvakit (avapritinib) compared to cells expressing KIT V560D alone in culture (PMID: 31270078). 31270078
PDGFRA D842V gastrointestinal stromal tumor sensitive Avapritinib Case Reports/Case Series Actionable In a Phase I trial, Ayvakit (avapritinib) treatment resulted in partial response lasted for more than 15 cycles of treatment, and sustained reduction of plasma PDGFRA D842V in a patient with gastrointestinal stromal tumor harboring PDGFRA D842V, whose disease had progressed on previous Gleevec (imatinib mesylate), Sprycel (dasatinib), and Crenolanib treatment (PMID: 29093181; NCT02508532). 29093181
PDGFRA D842V gastrointestinal stromal tumor sensitive Avapritinib Guideline Actionable Ayvakit (avapritinib) is included in the guidelines for patients with unresectable/metastatic gastrointestinal stromal tumor (GIST) with PDGFRA exon 18 mutations, including PDGFRA D842V (NCCN.org). detail...
PDGFRA D842V gastrointestinal stromal tumor sensitive Avapritinib Phase I Actionable In a Phase I trial (NAVIGATOR), Ayvakit (avapritinib) treatment in patients with a gastrointestinal stromal tumor harboring PDGFRA D842V resulted in an overall response in 88% (49/56), with 5 patients experiencing a complete response (9%) and 44 patients with a partial response (79%) (PMID: 32615108; NCT02508532). 32615108
PDGFRA D842V gastrointestinal stromal tumor sensitive Avapritinib FDA approved Actionable In a Phase I trial (NAVIGATOR) that supported FDA approval, Ayvakit (avapritinib) treatment resulted in an objective response in 86% (37/43, 3 complete response, 34 partial response) and stable disease in 12% (5/43) of patients with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including D842V, with median duration of response not reached (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11022-11022; NCT02508532). detail... detail...
PDGFRA D842X gastrointestinal stromal tumor sensitive Avapritinib Phase I Actionable In a Phase I (NAVIGATOR) trial, Ayvakit (avapritinib) treatment resulted in an objective response rate of 70% (26/37, 2 complete responses, 24 partial responses) and a disease control rate of 97% (36/37) in patients with gastrointestinal stromal tumor harboring PDGFRA D842 mutations (The CTOS 2018 Annual Meeting, Nov 14-17, Rome Italy, Paper 012 3027631; NCT02508532). detail...
PDGFRA D842X gastrointestinal stromal tumor sensitive Avapritinib Phase I Actionable In a Phase I trial, Ayvakit (avapritinib) demonstrated preliminary antitumor activity in gastrointestinal stromal tumor patients harboring PDGFRA D842 mutations, with partial responses in 50% (6/12) and stable disease in 50% (6/12) of patients (EORTC-NCI-AACR 2016, Abs 6LBA). detail...
KIT W557_K558del KIT Y823D gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Pdx Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and resulted in tumor regression in patient-derived xenograft models of Gleevec (imatinib mesylate)-resistant gastrointestinal stromal tumor harboring KIT W557_K558del and Y823D (PMID: 29093181). 29093181
KIT V654A KIT D816V Advanced Solid Tumor predicted - sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT V654A demonstrated some sensitivity to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
KIT exon17 gastrointestinal stromal tumor sensitive Avapritinib Phase I Actionable In a Phase I trial, Ayvakit (avapritinib) demonstrated preliminary antitumor activity in gastrointestinal stromal tumor patients harboring KIT mutations, with reduced tumor burden in 38% (5/13) of patients, including 1 partial response and 4 stable diseases, and 3 of the 5 responding patients harbored KIT exon 17 mutations (EORTC-NCI-AACR 2016, Abs 6LBA). detail...
KIT W557_V559delinsF gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Pdx Actionable In a preclinical study, Ayvakit (avapritinib) treatment resulted in tumor regression in patient-derived xenograft models of gastrointestinal stromal tumor harboring KIT W557_V559delinsF (PMID: 29093181). 29093181
KIT W557_K558del KIT V654A gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Pdx Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and resulted in tumor regression in patient-derived xenograft models of Gleevec (imatinib)-resistant gastrointestinal stromal tumor harboring KIT W557_K558del and V654A (PMID: 29093181). 29093181
KIT T670I KIT D816V Advanced Solid Tumor resistant Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670I demonstrated resistance to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
KIT V654A gastrointestinal stromal tumor predicted - resistant Avapritinib Phase I Actionable In a Phase I trial, patients with gastrointestinal stromal tumors harboring either KIT V654A or KIT T670I demonstrated decreased overall response rate (0%, 0/25 vs. 26%, 22/84) and increased progressive disease rate (72% vs 23%) compared to those without either KIT mutation when treated with Ayvakit (avapritinib) (PMID: 31270078; NCT02508532). 31270078
KIT D677N KIT D816V Advanced Solid Tumor predicted - sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT D677N demonstrated some sensitivity to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
KIT D816V mast cell neoplasm predicted - sensitive Avapritinib Phase I Actionable In a Phase I trial, patients with either aggressive systemic mastocytosis, mast cell leukemia, or systemic mastocytosis with an associated hematologic neoplasm harboring KIT D816V demonstrated a reduction in mast cell burden and a decrease in KIT D816V allelic fraction when treated with Ayvakit (avapritinib) (ASH Annual Meeting, Dec 2017, Plenary 2). detail...
KIT D816V acute myeloid leukemia sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and growth of erythroleukemia cells expressing KIT D816V in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins hematologic cancer decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture with reduced potency compared to other kinase inhibitors (PMID: 31309543). 31309543
KIT T670I gastrointestinal stromal tumor predicted - resistant Avapritinib Phase I Actionable In a Phase I trial, patients with gastrointestinal stromal tumors harboring either KIT V654A or KIT T670I demonstrated decreased overall response rate (0%, 0/25 vs. 26%, 22/84) and increased progressive disease rate (72% vs 23%) compared to those without either KIT mutation when treated with Ayvakit (avapritinib) (PMID: 31270078; NCT02508532). 31270078
KIT A502_Y503dup gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model with a gastrointestinal stromal tumor harboring KIT A502_Y503dup was sensitive to treatment with Ayvakit (avapritinib), demonstrating a 90% stabilized tumor volume at a dose of 30mg/kg and tumor regression at a dose of 60mg/kg, and inhibition of cell proliferation (PMID: 30274985). 30274985
KIT exon17 Advanced Solid Tumor sensitive Avapritinib Preclinical Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of various tumor cell lines harboring KIT exon 17 mutations in culture, and induced tumor regression in an allograft animal model (PMID: 29093181). 29093181
KIT Y672C KIT D816V Advanced Solid Tumor predicted - sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT Y672C demonstrated some sensitivity to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
KIT D816V hematologic cancer sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and growth of transformed hematologic cells expressing KIT D816V in culture (PMID: 31309543). 31309543
KIT D820V gastrointestinal stromal tumor sensitive Avapritinib Phase I Actionable In a Phase I trial, Ayvakit (avapritinib) treatment resulted in tumor reduction lasting through 15 cycles of treatment in a patient with gastrointestinal stromal tumor harboring KIT D820V, whose disease had progressed on previous Gleevec (imatinib), Sutent (sunitinib), and Stivarga (regorafenib) treatment (PMID: 29093181; NCT02508532). 29093181
CBL Y371H FLT3 wild-type hematologic cancer decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited growth of transformed hematologic cells expressing CBL Y371H and wild-type FLT3 in culture with reduced potency compared to other kinase inhibitors (PMID: 31309543). 31309543
KIT T670A KIT D816V Advanced Solid Tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670A demonstrated sensitivity to treatment with Ayvakit (avapritinib) in culture (PMID: 31270078). 31270078
PDGFRA V561D Advanced Solid Tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation in transformed cells overexpressing PDGFRA V561D in culture (PMID: 29093181). 29093181
KIT exon 11 del KIT T670I gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited proliferation of gastrointestinal stromal tumor cells harboring both KIT exon 11 deletion and KIT T670I in culture (PMID: 31085175). 31085175
Unknown unknown indolent systemic mastocytosis not applicable Avapritinib Phase I Actionable In a Phase I trial, BLU-285 treatment reduced bone marrow mast cell infiltration in a patient with systemic mastocytosis (PMID: 29093181; NCT02561988). 29093181
KIT V560G KIT D816V mast-cell leukemia sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of a mast cell leukemia cell line harboring KIT D816V and V560G in culture (PMID: 29093181). 29093181
KIT exon 11 del KIT D816E gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited proliferation of gastrointestinal stromal tumor cells harboring both KIT exon 11 deletion and KIT D816E in culture (PMID: 31085175). 31085175
KIT D816Y mast cell neoplasm predicted - sensitive Avapritinib Preclinical Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of murine mastocytoma cells harboring KIT D814Y (corresponding to human D816Y) in culture, and resulted in tumor regression in allograft animal models (PMID: 29093181). 29093181
KIT D816V Advanced Solid Tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing KIT D816V were sensitive to treatment with Ayvakit (avapritinib) in culture, demonstrating decreased cell proliferation (PMID: 31270078). 31270078
KIT V560D gastrointestinal stromal tumor predicted - sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing KIT V560D were sensitive to treatment with Ayvakit (avapritinib) in culture, demonstrating decreased cell growth (PMID: 31270078). 31270078
KIT exon 11 del gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation and proliferation of gastrointestinal stromal tumor cells harboring KIT exon 11 deletion in culture (PMID: 31085175). 31085175
CBL Q365_E366insSK FLT3 wild-type hematologic cancer decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited growth of transformed hematologic cells expressing CBL Q365_E366insSK and wild-type FLT3 in culture with reduced potency compared to other kinase inhibitors (PMID: 31309543). 31309543
PDGFRA D842V Advanced Solid Tumor sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited Kit phosphorylation in transformed cells overexpressing PDGFRA D842V in culture (PMID: 29093181). 29093181
KIT W557Lfs*5 KIT P577del KIT D820G gastrointestinal stromal tumor sensitive Avapritinib Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model with a gastrointestinal stromal tumor harboring KIT W557Lfs*5, KIT P577del, and KIT D820G was sensitive to treatment with Ayvakit (avapritinib), demonstrating a stabilized tumor volume at a dose of 10mg/kg and 27% tumor regression at a dose of 30mg/kg, a histologic response, and apoptotic activity (PMID: 30274985). 30274985
KIT V560G mast cell neoplasm sensitive Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited proliferation of mast cells harboring KIT V560G in culture (PMID: 31085175). 31085175

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Clinical Trial Phase Therapies Title Recruitment Status
NCT03731260 Phase II Avapritinib (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent and Smoldering Systemic Mastocytosis Recruiting
NCT03580655 Phase II Avapritinib (PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis Recruiting
NCT02508532 Phase I Avapritinib Phase 1 Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors Active, not recruiting
NCT03465722 Phase III Regorafenib Avapritinib (VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST Active, not recruiting
NCT02561988 Phase I Avapritinib (EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies Active, not recruiting


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