RALLY-MF: Initial efficacy of a phase 2 study of DISC-0974, an anti-hemojuvelin antibody, to treat anemia in myelofibrosis.

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Authors: Naseema Gangat, Ayalew Tefferi, Prithviraj Bose, Elizabeth O. Hexner, Laura Christian Michaelis, Aaron Thomas Gerds, Akriti G. Jain, Prioty Islam, Raajit K. Rampal, Ronan T. Swords, Moshe Talpaz, Kristen Marie Pettit
+5 more Sima Bhatt, Akshay Buch, Olivia Pelletier, Will Savage, Anna B. Halpern
Cited by: 2

Abstract

6501 Background: Hepcidin, a central regulator of iron homeostasis, is pathologically elevated in patients with myelofibrosis (MF) and anemia. Chronic hepcidin elevation limits iron availability for red blood cell (RBC) production and contributes to anemia onset and severity. DISC-0974 is an investigational, first-in-class, monoclonal antibody that blocks hemojuvelin, a co-receptor in the bone morphogenetic protein-signaling pathway driving hepcidin expression. Methods: RALLY-MF (NCT05320198) is an ongoing Phase 2, open-label study conducted in the US. The primary aim is to evaluate DISC-0974 efficacy in participants with MF and anemia. Secondary aims include evaluating safety, pharmacokinetics (PK), and pharmacodynamics (PD). Eligible participants are ≥18 years of age with primary or secondary MF and hemoglobin (Hgb)

Fields of Study & Subjects

Myeloproliferative Neoplasms: Diagnosis and Treatment Iron Metabolism and Disorders Blood properties and coagulation

Publication details

Journal / source
Journal of Clinical Oncology
Publisher
American Society of Clinical Oncology (ASCO)
Volume
44
Issue
16_suppl
Pages
6501–6501
Publication date
27 May 2026
Language
EN