Publications and Supporting Literature
Disc Publications
EHA 2025
Single- and Multiple-Ascending Doses of DISC-3405, a Recombinant Humanized Antibody Targeting TMPRSS6, Increased Hepcidin and Reduced Iron and Hematocrit in Healthy Volunteers
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EHA 2025
Results from the HELIOS Study: A Phase 2, Open-Label, Long-Term ExtensionStudy of Bitopertin in Erythropoietic Protoporphyria
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EHA 2025
An Iron Pulse Study to Assess Oral Iron Absorption FollowingTreatment With DISC-3405 in Healthy Volunteers
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EHA 2025
A Phase 1b/2 Study of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Myelofibrosis and Anemia
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EHA 2025
Anti-Hemojuvelin Monoclonal Antibody Provided Further Improvement in Anemia in Combination with ESA and/or Luspatercept (RAP-536) in Mice
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ASH 2024
Anti-Hemojuvelin Monoclonal Antibody Reverses Anemia and Exerts Disease-Modifying Effects in a Mouse Model of Inflammatory Bowel Disease
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ASH 2024
Pharmacological Inhibition of TMPRSS6 Decreases Hemoglobin Concentration and Red Blood Cell Hemolysis in a Mouse Model of Sickle Cell Disease
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ASH 2024
A Phase 1b Study of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Myelofibrosis and Anemia
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ASH 2024
Results from the BEACON Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria
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ASH 2024
Inhibition of Glycine Transporter 1 Reduces EPP Phototoxicity in a Mouse Model of Erythropoietic Protoporphyria
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ASH 2024
The EPP Light Study: A Survey Describing the Burden of Erythropoietic Protoporphyria
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ASH 2024
Anti-Hemojuvelin Monoclonal Antibody Alleviated Anemia Induced by Ruxolitinib Treatment in Mice
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ASH 2024
Results from the AURORA Trial: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of Bitopertin in Erythropoietic Protoporphyria
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ASH 2024
Phase 1 Healthy Volunteer Study of DISC-3405, a Recombinant Humanized Antibody Targeting TMPRSS6
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ASN 2024
A Phase 1b Double-Blind, Placebo-Controlled Study Of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Non-Dialysis Dependent Chronic Kidney Disease and Anemia
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EHA 2024
Phase 1 Healthy Volunteer Study of DISC-3405, A Recombinant Humanized Antibody Targeting TMPRSS6
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EHA 2024
Topline Results from the AURORA Trial: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of Bitopertin in Erythropoietic Protoporphyria
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EHA 2024
Results from the BEACON Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria
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ASH 2023
Interim Analyses from the BEACON trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria
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ASH 2023
A Phase 1b Trial of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Myelofibrosis and Anemia
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EHA 2023
Initial Data from the BEACON Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria
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ASH 2022
Bitopertin, a Selective Glycine Transporter 1 Inhibitor, Reduced PPIX Level and Improved Liver Fibrosis in a Mouse Model of Erythropoietic Protoporphyria (EPP)
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ASH 2022
BEACON: A Phase 2, Randomized, Open Label Study of Bitoperin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX Concentrations in Participants…
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ASH 2022
DISC-0974, an Anti-Hemojuvelin Antibody, Reduces Hepcidin and Mobilizes Iron in Healthy Volunteers
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ASH 2022
DISC-0974, an Anti-Hemojuvelin (HJV) Monoclonal Antibody, Reduced Hepcidin and Improved Anemia in a Rat Model of Chronic Kidney Disease
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EHA 2022
DISC-0974, a First-in-Human Anti-Hemojuvelin Monoclonal Antibody, Increases Serum Iron and Transferrin Saturation in Healthy Participants
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ASH 2022
Preclinical Pharmacokinetics and Pharmacodynamics of DISC-0998, A Humanized Anti-Hemojuvelin (HJV) Monoclonal Antibody to Suppress the Production of Hepcidin
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Porphyria Symposium 2021
Proof of Mechanism Studies with Bitopertin, a Selective Glycine Transporter 1 Inhibitor Under Development for the Treatment of Erythropoietic Protoporphyria (EPP) and…
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EHA 2021
Disc-B, a selective matriptase-2 inhibitor, elicited robust increase in hepcidin-25 and reduction in serum iron in cynomolgus moneys
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ASH 2021
Treating iron overload disorders with a novel therapeutic antibody targeting TMPRSS6
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ASH 2021
Proof of Mechanism Studies with Bitopertin, a Selective Glycine Transporter 1 Inhibitor Under Development for the Treatment of Erythropoietic Protoporphyria (EPP) and…
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ASH 2020
DISC-0974, a novel, first-in-class, anti-hemojuvelin monoclonal antibody decreases hepcidin and increases transferrin saturation in a non-human primate model of cytokine (IL-6) induced…
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Supporting Literature
Bitopertin
Clinical, Biochemical, and Genetic Characterization of North American Patients With Erythropoietic Protoporphyria and X-linked Protoporphyria
Balwani, M., Naik, H., Anderson, K. E., Bissell, D. M., Bloomer, J., Bonkovsky, H. L., Phillips, J. D., Overbey, J. R., Wang, B., Singal, A. K., Liu, L. U., & Desnick, R. J. (2017). Clinical, biochemical, and genetic characterization of North American patients with erythropoietic protoporphyria and X-linked protoporphyria. JAMA Dermatology, 153(8), 789.
Bitopertin
Evidence in the UK Biobank for the underdiagnosis of erythropoietic protoporphyria
Dickey, A. K., Quick, C., Ducamp, S., Zhu, Z., Feng, Y.-C. A., Naik, H., Balwani, M., Anderson, K. E., Lin, X., Phillips, J. E., Rebeiz, L., Bonkovsky, H. L., McGuire, B. M., Wang, B., Chasman, D. I., Smoller, J. W., Fleming, M. D., & Christiani, D. C. (2021). Evidence in the UK Biobank for the…
Bitopertin
Extracellular glycine is necessary for optimal hemoglobinization of erythroid cells
Garcia-Santos, D., Schranzhofer, M., Bergeron, R., Sheftel, A. D., & Ponka, P. (2017). Extracellular glycine is necessary for optimal hemoglobinization of erythroid cells. Haematologica, 102(8), 1314–1323.
Bitopertin
Glycine Transporter Type I (GlyT1) Inhibitor, Bitopertin: A Journey from Lab to Patient
Pinard, E., Borroni, E., Koerner, A., Umbricht, D., & Alberati, D. (2018). Glycine transporter type I (glyt1) inhibitor, Bitopertin: A journey from lab to patient. CHIMIA, 72(7-8), 477.
Bitopertin
Effects of GlyT1 inhibition on erythropoiesis and iron homeostasis in rats
Winter, M., Funk, J., Körner, A., Alberati, D., Christen, F., Schmitt, G., Altmann, B., Pospischil, A., & Singer, T. (2016). Effects of GLYT1 inhibition on erythropoiesis and iron homeostasis in rats. Experimental Hematology, 44(10).
Bitopertin
Updates on the diagnosis and management of the most common hereditary porphyrias: AIP and EPP
Linenberger, M., & Fertrin, K. Y. Hematology. American Society of Hematology. Education Program, 2020(1), 400–410.
Bitopertin
Protoporphyrin IX in the skin measured noninvasively predicts photosensitivity in patients with erythropoietic protoporphyria
Heerfordt, I. M., & Wulf, H. C. (2016). The British journal of dermatology, 175(6), 1284–1289.
Bitopertin
Inactivation of protoporphyrin IX in erythrocytes in patients with erythropoietic protoporphyria: A new treatment modality
Wulf, H. C., Nissen, C. V., & Philipsen, P. A. (2020). Photodiagnosis and photodynamic therapy, 29, 101582.
DISC-0974
Myelofibrosis-Related Anemia: Current and Emerging Therapeutic Strategies
Naymagon, L., & Mascarenhas, J. (2017). Myelofibrosis-Related Anemia. HemaSphere, 1(1), e1.
DISC-0974
Anemia of inflammation
Weiss, G., Ganz, T., & Goodnough, L. T. (2019). Anemia of inflammation. Blood, 133(1), 40–50.
DISC-0974
Hepcidin-mediated hypoferremic response to acute inflammation requires a threshold of Bmp6/Hjv/Smadsignaling
Fillebeen, C., Wilkinson, N., Charlebois, E., Katsarou, A., Wagner, J., & Pantopoulos, K. (2018). Hepcidin-mediated hypoferremic response to acute inflammation requires a threshold of Bmp6/Hjv/Smad signaling. Blood, 132(17), 1829–1841.
DISC-0974
Iron Balance and the Role of Hepcidin in Chronic Kidney Disease
Ganz, T., & Nemeth, E. (2016). Iron Balance and the Role of Hepcidin in Chronic Kidney Disease. Seminars in Nephrology, 36(2), 87–93.
DISC-0974
The Relation of Hepcidin to Iron Disorders, Inflammation and Hemoglobin in Chronic Kidney Disease
Mercadel, L., Metzger, M., Haymann, J. P., Thervet, E., Boffa, J.-J., Flamant, M., Vrtovsnik, F., Houillier, P., Froissart, M., & Stengel, B. (2014). The Relation of Hepcidin to Iron Disorders, Inflammation and Hemoglobin in Chronic Kidney Disease. PLoS ONE, 9(6), e99781.
DISC-0974
Associations and prognostic interactions between circulating levels of hepcidin, ferritin and inflammatory cytokines in primary myelofibrosis
Pardanani, A., Mengistu, B., Finke, C., Lasho, T. L., & Tefferi, A. (2012). Associations and Prognostic Interactions Between Circulating Levels of Hepcidin, Ferritin, and Inflammatory Cytokines in Primary Myelofibrosis. Blood, 120(21), 2831–2831.
DISC-0974
Anti-repulsive Guidance Molecule C (RGMc) Antibodies Increases Serum Iron in Rats and Cynomolgus Monkeys by Hepcidin Downregulation
Böser, P., Seemann, D., Liguori, M. J., Fan, L., Huang, L., Hafner, M., Popp, A., & Mueller, B. K. (2015). Anti-repulsive Guidance Molecule C (RGMc) Antibodies Increases Serum Iron in Rats and Cynomolgus Monkeys by Hepcidin Downregulation. The AAPS Journal, 17(4), 930–938.
DISC-0974
Anti-hemojuvelin antibody corrects anemia caused by inappropriately high hepcidin levels
Citation: Kovac, S., Boser, P., Cui, Y., Ferring-Appel, D., Casarrubea, D., Huang, L., Fung, E., Popp, A., Mueller, B. K., & Hentze, M. W. (2016). Anti-hemojuvelin antibody corrects anemia caused by inappropriately high hepcidin levels. Haematologica, 101(5), e173–e176.
Mat2
Hepcidin agonists as therapeutic tools
Casu, C., Nemeth, E., & Rivella, S. (2018). Hepcidin agonists as therapeutic tools. Blood, 131(16), 1790–1794.