Melflufen (Melphalan flufenamide) hydrochloride, an alkylating agent and dipeptide prodrug of Melphalan, exhibits antitumor activity by inducing irreversible DNA damage and cytotoxicity in multiple myeloma (MM) cells, while also inhibiting angiogenesis.

Melflufen (Melphalan flufenamide) hydrochloride (0.5-10 μM; 24 hours) decreases viability of MM.1S, INA-6, RPMI-8226, MM.1R, Dox-40, ARP-1, and ANBL-6 cells in a concentration-dependent manner[1]. Melflufen hydrochloride induces apoptosis in MM.1S cells[1]. Melflufen hydrochloride also is a potent activator of exosome secretion[3]. Cell Viability Assay[1]Cell Line: Multiple myeloma cells: MM.1S, INA-6, RPMI-8226, MM.1R, Dox-40, ARP-1, ANBL-6 cells Concentration: 0.5, 1, 3, 5, 10 μM Incubation Time: 24 hours Result: A significant concentration-dependent decrease in viability of all cell lines was observed.

Melflufen (Melphalan flufenamide) hydrochloride (3 mg/kg; i.v.; twice-weekly for two weeks) shows anti-MM activity in xenograft mouse model[1]. Animal Model: CB-17 SCID mice (human plasmacytoma MM.1S xenograft mouse model)[1]Dosage: 3 mg/kg Administration: 1.v.; twice-weekly for two weeks Result: Significantly inhibited MM tumor growth and prolonged survival of mice.