Publications and Supporting Literature

Disc Publications

ASH 2023

Interim Analyses from the BEACON trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria

ASH 2023

A Phase 1b Trial of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Myelofibrosis and Anemia

EHA 2023

Initial Data from the BEACON Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria

ASH 2022

Bitopertin, a Selective Glycine Transporter 1 Inhibitor, Reduced PPIX Level and Improved Liver Fibrosis in a Mouse Model of Erythropoietic Protoporphyria (EPP)

ASH 2022

BEACON: A Phase 2, Randomized, Open Label Study of Bitoperin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX Concentrations in Participants…

ASH 2022

DISC-0974, an Anti-Hemojuvelin Antibody, Reduces Hepcidin and Mobilizes Iron in Healthy Volunteers

ASH 2022

DISC-0974, an Anti-Hemojuvelin (HJV) Monoclonal Antibody, Reduced Hepcidin and Improved Anemia in a Rat Model of Chronic Kidney Disease

EHA 2022

DISC-0974, a First-in-Human Anti-Hemojuvelin Monoclonal Antibody, Increases Serum Iron and Transferrin Saturation in Healthy Participants

ASH 2022

Preclinical Pharmacokinetics and Pharmacodynamics of DISC-0998, A Humanized Anti-Hemojuvelin (HJV) Monoclonal Antibody to Suppress the Production of Hepcidin

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…

EHA 2021

Disc-B, a selective matriptase-2 inhibitor, elicited robust increase in hepcidin-25 and reduction in serum iron in cynomolgus moneys

ASH 2021

Treating iron overload disorders with a novel therapeutic antibody targeting TMPRSS6

ASH 2021

Proof of Mechanism Studies with Bitopertin, a Selective Glycine Transporter 1 Inhibitor Under Development for the Treatment of Erythropoietic Protoporphyria (EPP) and…

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…

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.