The activity of DISC-0974 — an investigational monoclonal antibody targeting hemojuvelin (HJV) — in treating anemia in patients with myelofibrosis is demonstrated in an open label Phase II study.
The investigational drug blocks the expression of HJV in order to reduce hepcidin. It is in mid-stage development by Disc Medicine. Updated data from the phase 2 open label RALLY-MF study were presented at the EHA 2026 and ASCO 2026 meetings in June.
The study tested the drug in adults with primary or secondary MF with anemia serious enough to result in persistently low hemoglobin or the need for red blood cell transfusions. Participants were permitted to be on a Janus kinase (JAK) inhibitor, such as momelotinib (Ojjaara) or ruxolitinib (Jakafi)
As of January 2026, there were 54 participants — of which 31 completed treatment, 23 entered the continuation part of the study, and 6 withdrew. Withdrawals were not due to adverse events, Mayo Clinic’s Naseema Gangat, MBBS, and colleagues reported.
The overall response rate was 70% and was consistent regardless of whether participants were also on a JAK inhibitor.
Researchers evaluated participants divided in three categories, based on anemia severity, and found the following response rates:
- Non-transfusion dependent: 15/28 (54%)
- Low transfusion requirement: 6/9 (67%)
- High transfusion requirement: 3/6 (50%)
As for safety, researchers reported 48 had at least one adverse event, of which 11 were determined to be related to the study drug. No serious adverse events were reported.
The drug’s manufacturer plans to share additional data later this year and to have a meeting with the U.S. Food and Drug Administration by end-2026.
Reference
Gangat N, Tefferi A, Hexner E, et al. RALLY-MF: Initial efficacy of a phase 2 Study of DISCO-0974, an anti-hemojuvelin antibody, to treat anemia in myelofibrosis. Abstract# EHA-2314. Presented at the European Hematology Association annual meeting. June 11-14, 2026; Stockholm.
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