PTG-300: hepcidin mimetic
Dr. Richard Shames (Protagonist Therapeutics Inc, Newark, USA) showed that PTG-300, an injectable peptide hepcidin mimetic, was well-tolerated and demonstrated marked and sustained dose-related effects on iron distribution in a first-in-human study.
Sixty-two healthy male volunteers received either PTG-300 as a single sc dose over a 1-80 mg dose range, or a placebo, and were followed for one week. PTG-300 induced a dose-related reduction in serum iron with a maximum mean reduction of ca. 60% from baseline.
The effect plateaued at a dose of 20 mg and persisted for at least 72 h at higher doses. PTG-300 was generally well tolerated without dose-limiting toxicities or serious adverse events. “This study provides a range of doses that could be evaluated in treating iron-loading anemias, such as beta-thalassemia and MDS.”