Hepcidin trials – TRANSCEND
Update: 01 February 2019
Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body. Thalassaemia patients have low hepcidin levels due to iron overload.
Abnormally low hepcidin levels caused by genetic mutations or secondary pathology can be replaced by a hepcidin mimetic to restore iron balance in the body. Increasing hepcidin levels can reduce iron overload and improve anaemia.
PTG-300 is an injectable hepcidin mimetic peptide, that has been granted Orphan Drug designation in the U.S. and EU and has received Fast Track designation by the FDA for development in the treatment of β-thalassaemia.
A Phase 1 study in healthy volunteers showed that administration of PTG-300 was well tolerated and demonstrated a dose-related and sustained reduction in serum iron levels.
As such a global Phase 2 study (TRANSCEND) with an open label and multiple-ascending dose design has commenced to evaluate the safety and preliminary efficacy of PTG-300 in approximately 84 adolescent and adult patients with non-transfusion-dependent or transfusion-dependent β-thalassemia.
Preliminary results are to be announced in the second half of 2019.