LJPC-401: synthetic hepcidin for iron overload
Endogenous hepcidin regulates dietary iron absorption and tissue distribution, and in animal models, increasing hepcidin levels by synthetic hepcidin injection or genetic induction has been shown to improve iron overload. This suggests that increasing hepcidin may help treat the abnormal iron absorption in beta-thalassemia and related disorders.
For that purpose, the synthetic human hepcidin LJPC-401 has been tested in several trials. Dr. Vip Viprakasit (Mahihol University, Bangkok, Thailand) presented data from an international phase 1 open-label, dose-escalation study in 18 patients at risk for iron overload (TPP-1-trial).
“LJPC-401 was well tolerated at doses between 1 mg and 30 mg, with iron-lowering observed at 20 mg. Significant decreases in serum iron were sustained in most patients for up to 8 days.” A new LJPC-40-formulation increased subcutaneous (sc) bioavailability up to 3-fold.