Drug intelligence / Profile preview

gandotinib

Development stage
Phase 2
Lead developer
Eli Lilly
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Gandotinib is an orally bioavailable small molecule inhibitor developed by Eli Lilly for the treatment of cancer, particularly myeloproliferative neoplasms. It selectively and competitively inhibits Janus kinase 2 (JAK2), with increased potency for the JAK2 V617F mutation—a key driver in diseases such as primary myelofibrosis, polycythemia vera, and essential thrombocythemia. By inhibiting JAK2V617F activation, gandotinib disrupts the JAK-STAT signaling pathway and induces apoptosis in affected tumor cells. Clinical trials have shown its potential antineoplastic activity and acceptable safety profile at a maximum-tolerated dose of 120 mg daily[1][3][5].

Other names
1229236-86-5
02

Targets

JAK2 (Janus kinase 2)STAT3 (Signal Transducer and Activator of Transcription 3)

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