melphalan flufenamide

Melphalan flufenamide is a first-in-class peptide-drug conjugate and a lipophilic prodrug of the alkylating agent melphalan. It is designed for targeted delivery within tumor cells by exploiting elevated aminopeptidase activity in malignant cells. Once inside cancer cells, it is rapidly hydrolyzed by peptidases (notably aminopeptidase N), releasing hydrophilic alkylator payloads such as melphalan. This leads to high intracellular concentrations of the active drug, resulting in rapid and irreversible DNA damage and apoptosis. Melphalan flufenamide has demonstrated cytotoxic activity against myeloma cell lines resistant to other treatments and also inhibits angiogenesis and metastatic processes in preclinical studies. It was developed primarily for use with dexamethasone in adults with relapsed or refractory multiple myeloma who have received at least three or four prior lines of therapy[1][2][4][5][6][8].

Brand names

PepaxtoPepaxti

Other names

melphalan flufenamidemelflufen