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. Author manuscript; available in PMC: 2021 Oct 26.
Published in final edited form as: Trends Pharmacol Sci. 2021 Jun 2;42(8):640–656. doi: 10.1016/j.tips.2021.05.001

Table 1. Pharmacological agents targeting iron metabolism.

Name Type Mechanism Indication Clinical status Trial Number Ref.
Therapeutics for iron deficiency conditions
Iron supplement for oral administration
Ferrous sulfate Iron salt Refills depleted iron stores Iron deficiency anemia Marketed NCT01904864 [2832]
Ferrous gluconate Iron salt Refills depleted iron stores Iron deficiency anemia Marketed NCT01528644 [2832]
Iron supplement for intravenous administration
Ferumoxytol Iron oxide nanoparticle Refills depleted iron stores CKD Marketed NCT01227616 [104]
Ferric carboxymaltose Iron complex Refills depleted iron stores Iron deficiency anemia Marketed NCT03523117 [2832]
Sodium ferric gluconate Macromolecule Refills depleted iron stores Iron deficiency anemia Marketed NCT00223964 [2832]
Hepcidin downregulation
LY3113593 Ant¡-BMPΘ antibodies Binds/inhibits BMP6 CKD Phase 1 NCT02144285 [47]
Hepcidin capture
Anticalin Binds hepcidin Binds/inhibits hepcidin CKD Phase 1/II NCT02754167 (Ph. 1)
NCT03325621 (Ph. II)
[49]
LY2787106 Anti-hepcidin antibody Binds/inhibits hepcidin Anemia of chronic disease Phase 1 NCT01340976 [48]
Stimulation of erythropoiesis
Luspatercept Recombinant fusion protein Ligand trap for TGF-beta superfamily β-Thalassemia Phase III NCT02604433 [37]
Sotatercept Recombinant fusion protein Ligand trap for TGF-beta superfamily β-Thalassemia Phase II NCT01S71635 [38]
Gene therapy
OTL-300 Cells/Lentiviral vector Cells genetically modified with a vector encoding human β-globin β-Thalassemia Marketed NCT03275051 [116]
LentiGlobinR BB305 Cells/Lentiviral vector Engineered hematopoietic stem cells with a functional version of human β-globin gene β-Thalassemia Phase III NCT03207009 [114]
ST-400, CTX001 Cells/Vector Modified patients’ blood stem cells with disrupted BCL11A gene, induction of fetal hemoglobin synthesis β-Thalassemia Phase I/ll CTX001: NCT03655678
ST-400: NCT03432364
[111], [112]
Therapeutics for iron overload conditions
Iron absorption
Esomeprazole Small molecule Proton pump inhibitor β-Thalassemia Phase III NL6659 (Netherlands trial Register) [76]
PT-2385 Small molecule Inhibits HIF2α-dependent genes Hemochromatosis Preclinîcal NCT03108066 [77]
Ebselen Small molecule DMT1 inhibitor acting as a mimic of glutathione peroxidase Alzheimer’s disease Preclinical Not listed yet [91],[92]
Iron binding
Deferoxamine (DFO), Small molecule Iron chelation Secondary Marketed NCT00001203 [69]
Deferasirox (DFX), Small molecule Iron chelation Hemochromatosis NCT00171171 [35]
Deferiprone (DFP) Small molecule Iron chelation NCT02189941 [69]
Triapine Small molecule Iron chelation, RR inhibitor Cancer Phase II NCT00293345 [63]
Cefiderocol Small molecule Antibiotic-siderophore conjugate Urinary tract infection Phase II NCT02321800 [96]
Dpc Small molecule Iron chelation Cancer Phase 1 NCT02688101 [63]
Polymeric DFO Nanopartîcle Iron chelation Hemochromatosis Preclinical [69]
Hepcidin upreguiotion
TMPRSS6-LRX Antisense DNA oligonucleotide Inhibition of TMPRSS6 expression β-Thalassemia Phase 1 NCT03165864 [70]
SLN124 GalNAcdouble-stranded siRNA conjugate Inhibition of TMPRSS6 expression Hemochromatosis Phase 1 NCT04176653 [71], [72]
Antî-ERFE 15.1 Anti-ERFE antibody Binds/inhibits ERFE β-Thalassemia Preclinical No listed yet [73]
Ferroportin downregulotion
VIT-2736 Anti-Fpn antibody Fpn inhibitor β-Thalassemia Phase 1 NCT04364269 [75]
Ferroptosis inducer
Sorafenib Small molecule Tyrosine kinase inhibitor, Inhibition of Xc-induced glutathione depletion and lipid peroxidation Cancer Marketed [106]
Erastîn Small molecule Inhibition of Xc-induced glutathione depletion and lipid peroxidation Cancer Preclinical No listed yet [106]
RSL3 Small molecule GPX4 inhibition, ROS production Cancer Preclinical No listed yet [104]
Ferumoxytol Iron oxide nanoparticle ROS production AML Preclinical No listed yet [105]