• Alias: KPT-330
    • A small-molecule inhibitor of CRM1 (chromosome region maintenance 1 protein, exportin 1 or XPO1), with potential antineoplastic activity
    • Modifies the essential CRM1 cargo–binding residue cysteine-528, thereby irreversibly inactivating CRM1-mediated nuclear export of cargo proteins such as tumor suppressor proteins, including p53, p21, BRCA1/2, pRB, FOXO, and other growth regulatory proteins
    • Restores endogenous tumor suppressing processes to selectively eliminate tumor cells, while sparing normal cells
    • Currently under investigation in phase 1 clinical trials with solid tumors and hematologic cancers
    • Proposed expansion dose: 30 to 35 mg/m2 orally for 10 in 28-day cycle with food
    Other topics in Targeted and Immunotherapy Agents