AL-amyloidosis

AL- amyloidosis is a rare disease that occurs in 30,000 to 45,000 patients in the U.S. and Europe. The patients have a plasma-cell disease including a heterogenous collection of diseases involving protein deposition in one or several organs. The median overall survival is 3.5 years.

Heterogenous diseases with protein deposition

Amyloidosis is a term use for used to describe a highly heterogenous collection of diseases that involve some form of protein deposition in one or several organs. Patients with light chain (AL) amyloidosis suffer from a clonal plasma-cell disease, usually a monoclonal gammopathy of unknown significance (MGUS) or more rarely, myeloma.

Limited treatment options

AL-amyloidosis is a rare disease that occurs in about 30,000 to 45,000 patients in the U.S. and Europe. Current treatment alternatives are limited, and the median overall survival is 3.5 years.

Investigational Drug

Melflufen (INN melphalan flufenamide) is an investigational first in class peptide-drug conjugate that targets aminopeptidases and releases alkylating agents into tumor cells.

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Mechanism of action

Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to release an entrapped hydrophilic alkylator payload.

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