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Authors Daniel J. DeAngelo, MD, PhD, Albert Thomas Quiery, MD, Deepti Radia, MD, Mark W Drummond, MB ChB, PhD, FRCPath, Jason Gotlib, MD, William A Robinson, MD, PhD, Elizabeth Hexner, MD, MS, Srdan Verstovsek, MD, PhD, Hongliang Shi, et al.
Title 2 Clinical Activity in a Phase 1 Study of Blu-285, a Potent, Highly-Selective Inhibitor of KIT D816V in Advanced Systemic Mastocytosis (AdvSM)
Abstract Text ASH Annual Meeting, Dec 2017, Plenary 2


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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 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...