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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 |
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Date | |
URL | http://www.bloodjournal.org/content/130/Suppl_1/4119 |
Abstract Text | Blood 2017 130 (Suppl 1):4119 |
Molecular Profile | Treatment Approach |
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Gene Name | Source | Synonyms | Protein Domains | Gene Description | Gene Role |
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Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
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LAM-002A | Apilimod dimesylate|LAM 002A | LAM-002A (Apilimod dimesylate) 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 |
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Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
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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 | 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... |
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... |