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Ref Type
PMID
Authors WA Harb, CS Diefenbach, N Lakhani, SC Rutherford, MT Schreeder, SM Ansell, T Sher, DM Aboulafia, JB Cohen, D Nix, S Landrette, K Flanders, LL Miller, H Lichenstein, JS. Abramson
Title Phase 1 Clinical Safety, Pharmacokinetics (PK), and Activity of Apilimod Dimesylate (LAM-002A), a First-in-Class Inhibitor of Phosphatidylinositol-3-Phosphate 5-Kinase (PIKfyve), in Patients with Relapsed or Refractory B-Cell Malignancies
Journal Blood
Vol
Issue
Date
URL http://www.bloodjournal.org/content/130/Suppl_1/4119
Abstract Text Blood 2017 130 (Suppl 1):4119

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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
LAM-002A LAM-002A 3 1
Drug Name Trade Name Synonyms Drug Classes Drug Description
LAM-002A apilimod dimesylate LAM-002A inhibits phosphatidylinositol-3-phosphate 5-kinase (PIKfyve), leads to inhibition of autophagy and subsequent tumor cell death (PMID: 28104689, Blood 2017 130 (Suppl 1):4119).
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
Unknown unknown marginal zone B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 3 patients with marginal zone lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown chronic lymphocytic leukemia not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 4 patients with chronic lymphocytic leukemia (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in systemic partial metabolic responses and decreased tumor size in 3 of 11 patients with diffuse large B-cell lymphoma (Blood 2017 130 (Suppl 1):4119). detail...