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Protagonist posts phase 2 hormone replacement data, pitching for lead role in genetic iron overload disease

November 15, 2021

Protagonist Therapeutics wants to take the lead in a genetic iron disease, delivering midphase data on a hormone replacement therapy it envisages offering an alternative to blood draws.

Rusfertide is Protagonist’s attempt to improve on the druglike properties of the hormone hepcidin to alter the regulation of iron homeostasis. In people with hereditary hemochromatosis, who absorb too much iron from food, the mechanism of action could enable physicians to stop excessive iron accumulation—and the negative outcomes it causes—without relying solely on blood draws.

The 16 subjects in the phase 2 clinical trial self-administered a subcutaneous dose of rusfertide once or twice a week, with physicians adjusting the dose based on disease biomarkers over the course of the six-month study.

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