Drug intelligence / Profile preview

hepcidin (2c6922)

Development stage
Discontinued
Modality
Peptides
Administration
Subcutaneous
01

Overview

Hepcidin (specifically hepcidin-25) is a synthetic equivalent of the endogenous 25-amino acid peptide hormone hepcidin, which acts as the master regulator of systemic iron homeostasis. Developed by La Jolla Pharmaceutical Company (as LJPC-401), hepcidin binds directly to the cellular iron exporter ferroportin (SLC40A1), inducing its internalization and lysosomal degradation. This action limits iron efflux from macrophages and enterocytes into the circulation, thereby reducing serum iron levels. LJPC-401 was investigated in Phase 2 clinical trials for the treatment of iron overload disorders, including hereditary hemochromatosis and beta thalassemia. However, due to a lack of efficacy in a pivotal Phase 2 trial for myocardial iron overload in beta thalassemia, its development was discontinued in late 2019.

Other names
epcidina-25 (umana)HAMPhepcidin (human)hepcidin acetatehepcidin-25hepcidin25hepcidin 25LEAP-1LEAP1LEAP 1liver-expressed antimicrobial peptide 1synthetic human hepcidin
02

Targets

SLC40A1 (Ferroportin)

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