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Ref Type
Authors M. Heinrich, R. Jones, P. Schoffski, S. Bauer, M. von Mehren, F. Eskens, P. Cassier, O. MIr, H. Shi, T. Alvarez-Diez, M.E. Healy, B. Wolf, S. George
Title Preliminary safety and activity in a first-in-human phase 1 study of BLU-285, a potent, highly-selective inhibitor of KIT and PDGFRa activation loop mutants in advanced gastrointestinal stromal tumor (GIST)
Abstract Text


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
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...