Melphalan Flufenamide CAS# 380449-51-4 美法仑氟灭酰胺

Melphalan flufenamide (melflufen, J1, or L-PAM-2) is a lipophilic peptide–drug conjugate that delivers melphalan intracellularly via aminopeptidase activity. In preclinical studies, it demonstrated potent cytotoxicity against multiple myeloma and other hematologic malignancy cell lines, with IC50 values in the low nanomolar range, significantly greater than melphalan. Melflufen rapidly accumulated in tumor cells, releasing active alkylating metabolites that induced DNA damage and apoptosis. In xenograft mouse models of multiple myeloma, treatment produced marked tumor growth inhibition and survival benefit. Pharmacokinetic studies revealed rapid plasma clearance but high tumor penetration, supporting its design as a targeted cytotoxic therapy for cancer.