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Journal Articles

A phase 2, open-label study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405 in participants with polycythemia vera (PV)

Free
Naseema Gangat, Ayalew Tefferi, Aaron Gerds, Michael Grunwald, Anna Halpern, Laura Michaelis, Stephen Oh, Caitlin O'Neill, Jeanne Palmer, Kristen Pettit, Lindsay Rein, Candido Rivera, Gary Schiller, Joseph Shatzel, Lisa Guiducci, Jennifer Vaughn, Anne Wofford, Marcus Carden, Will Savage, Prithviraj Bose, Naveen Pemmaraju
Journal: Blood
Blood (2025) 146 (Supplement 1): 2053.
https://doi.org/10.1182/blood-2025-2053
Published: 2025
Journal Articles

RALLY MF: A phase 2 Study of DISC-0974, an anti-hemojuvelin antibody, in patients with myelofibrosis and anemia

Free
Naseema Gangat, Ayalew Tefferi, Prithviraj Bose, Elizabeth Hexner, Laura Michaelis, Aaron Gerds, Akriti Jain, Prioty Islam, Raajit Rampal, Ronan Swords, Moshe Talpaz, Kristen Pettit, Sima Bhatt, Akshay Buch, Olivia Pelletier, Will Savage, Anna Halpern
Journal: Blood
Blood (2025) 146 (Supplement 1): 2042.
https://doi.org/10.1182/blood-2025-2042
Published: 2025
Journal Articles

Results from the first clinical trial of the selective GlyT1 inhibitor bitopertin for steroid-refractory Diamond-Blackfan Anemia (NCT05828108)

Free
David Young, Tania Machado, Bretagne Cowling, Raymond Doty, Colin Wu, Janis Abkowitz, Will Savage, Cynthia Dunbar
Journal: Blood
Blood (2025) 146 (Supplement 1): 4992.
https://doi.org/10.1182/blood-2025-4992
Published: 2025
Journal Articles

Results from the Beacon Trial: A Phase 2, Randomized, Open-Label Study of Bitopertin in Erythropoietic Protoporphyria

Free
Gayle Ross, Peter Stewart, George Mensing, Melanie Chin, Haley Howell, Heidi Mangus, Will Savage
Journal: Blood
Blood (2024) 144 (Supplement 1): 1093.
https://doi.org/10.1182/blood-2024-203387
Published: 2024
Journal Articles

Phase 1 Healthy Volunteer Study of DISC-3405, a Recombinant Humanized Antibody Targeting TMPRSS6

Free
Guowen Liu, Haley Howell, Dan Rudin, Nanthini Arumugam, Janel Jadia, Hua Yang, Will Savage
Journal: Blood
Blood (2024) 144 (Supplement 1): 1092.
https://doi.org/10.1182/blood-2024-203255
Published: 2024
Journal Articles

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

Free
Naseema Gangat, James M. Foran, Anna B. Halpern, Raajit Rampal, Prithviraj Bose, Elizabeth O. Hexner, Moshe Talpaz, Laura C. Michaelis, Prioty Islam, Ronan T. Swords, Sima Bhatt, Akshay Buch, Olivia Pelletier, Will Savage, Ayalew Tefferi
Journal: Blood
Blood (2024) 144 (Supplement 1): 657.
https://doi.org/10.1182/blood-2024-203719
Published: 2024
Journal Articles

Results from the Aurora Study: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin in Erythropoietic Protoporphyria

Free
Amy Dickey, Sioban Keel, Herbert L Bonkovsky, Karl Anderson, Manisha Balwani, Cynthia Levy, Manish Thapar, Bruce Wang, Brendan McGuire, Will Savage
Journal: Blood
Blood (2024) 144 (Supplement 1): 1089.
https://doi.org/10.1182/blood-2024-203503
Published: 2024
Journal Articles

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

Free
Yue Chen, Will Savage, Rajiv Panwar, Richard Rodriguez, Min Wu, John Quisel, Hua Yang
Journal: Blood
Blood (2022) 140 (Supplement 1): 8184–8185.
https://doi.org/10.1182/blood-2022-168985
Published: 2022
Journal Articles

Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria: Beacon Trial Design

Free
George Mensing, Haley Howell, Katherine Chan, Brian MacDonald, Will Savage
Journal: Blood
Blood (2022) 140 (Supplement 1): 5352.
https://doi.org/10.1182/blood-2022-157810
Published: 2022
Journal Articles

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

Free
Natasha Novikov, Akshay Buch, Hua Yang, Sarah Tuller, Michelle Andruk, Katherine Chan, Min Wu, Richard Rodriguez, Rajiv Panwar, Haley Howell, Brian MacDonald, Will Savage
Journal: Blood
Blood (2022) 140 (Supplement 1): 5339–5340.
https://doi.org/10.1182/blood-2022-167870
Published: 2022
Journal Articles

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 Hypoferremia

Free
Brian MacDonald, PhDMBBChir,MRCP, Cecile Blaustein, PhD, Sophia Nguyen, MS, Christopher King, PhD MBA, Vu Hong, PhD, Will Savage, MDPhD, Srikanth Venkatraman, PhD, Maria G. Beconi, PhD MS
Journal: Blood
Blood (2020) 136 (Supplement 1): 2.
https://doi.org/10.1182/blood-2020-138720
Published: 2020
Journal Articles

DISC-0974, a Novel, First-in-Class, Anti-Hemojuvelin Monoclonal Antibody Is Predicted to be Efficacious at Low Doses in Humans As Determined By Pharmacokinetic/Pharmacodynamic (PK/PD) Modeling in Non-Clinical Studies

Free
Mark Hixon, PhD, Cecile Blaustein, PhD, Christopher King, PhD MBA, Sophia Nguyen, MS, Will Savage, MDPhD, Maria G. Beconi, PhD MS
Journal: Blood
Blood (2020) 136 (Supplement 1): 14–15.
https://doi.org/10.1182/blood-2020-138547
Published: 2020
Journal Articles

DISC-a, the First in a Novel Class of Potent and Selective Matriptase-2 Inhibitors for the Treatment of Hematologic Disorders Characterized By Low Hepcidin

Free
Vu Hong, PhD, Ravi Krishna Babu, PhD, Cecile Blaustein, PhD, Sophia Nguyen, MS, Venkateshwar Rao, PhD, Will Savage, MDPhD, Brian MacDonald, PhDMBBChir,MRCP, Maria G. Beconi, PhD MS, Srikanth Venkatraman, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 41–42.
https://doi.org/10.1182/blood-2020-138509
Published: 2020