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. 2018 Mar 9;131(16):1790–1794. doi: 10.1182/blood-2017-11-737411

Table 1.

Classification of hepcidin agonists

Hepcidin agonists Company Drug Target Clinical trials Reference
Class 1: hepcidin mimetics University of California, Los Angeles MHs (PR65, PR73, M009, M012) Ferroportin Validated in preclinical studies See articles cited in the review
La Jolla Pharmaceutical Company LJPC-401 (hepcidin formulation) Ferroportin Phase 1: no toxicity reported; expected hypoferremia observed 45
Protagonist Therapeutics PTG-300 Ferroportin Phase 1: no serious adverse events reported; expected hypoferremia observed 58
Class 2: stimulators of hepcidin production Ionis Pharmaceuticals Tmprss6-ASO Tmprss6 Phase 1 ongoing 59
Alnylam Pharmaceuticals Tmprss6-siRNA Tmprss6 Validated in preclinical studies See articles cited in the review
Class 3: ferroportin inhibitors Vifor Pharma VIT-2763 Ferroportin Phase 1 planned in 2018 60

ASO, antisense oligonucleotide; siRNA, small-interfering RNA.