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