Disc Medicine, Inc. (NASDAQ:IRON) presented initial data from its RALLY‑MF Phase 2 trial of DISC‑0974 at the 67th American Society of Hematology (ASH) meeting in Orlando on December 6 2025. The open‑label study enrolled 47 patients with myelofibrosis‑associated anemia and used a data cutoff of October 16 2025, with 34 patients included in the responder analysis.
Subcutaneous DISC‑0974, administered at 50 mg every four weeks for up to six doses, lowered hepcidin levels and increased iron availability. In non‑transfusion‑dependent patients, 63 % achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks, and 50 % reached ≥1.5 g/dL for the same duration. Among transfusion‑dependent patients, 71 % of those with a low transfusion burden achieved transfusion independence over a 16‑week period, while 67 % of patients with a high transfusion burden experienced at least a 50 % reduction in transfusion requirements. Additionally, 50 % of patients receiving concomitant JAK‑inhibitor therapy achieved a major hematologic response.
Safety data were encouraging: diarrhea and urinary tract infections were reported in two or more subjects, and no serious adverse events were recorded. The safety profile aligns with the known tolerability of monoclonal antibodies targeting hemojuvelin.
CEO John Quisel emphasized that the hematologic responses were observed regardless of background JAK‑inhibitor therapy and that reductions in hepcidin translated into a lower transfusion burden and reduced fatigue—symptoms that significantly impact patient quality of life.
The results reinforce Disc Medicine’s strategy to address anemia in myelofibrosis, a condition with limited approved therapies. By targeting iron homeostasis through HJV blockade, DISC‑0974 offers a novel mechanism that could complement or provide an alternative to existing JAK‑inhibitor treatments. The company plans to advance the program into larger, controlled studies, with additional data expected in the second half of 2026 to support regulatory submissions.
The positive data strengthen the broader platform and may accelerate market approval for this indication. Disc Medicine’s pipeline also includes bitopertin for erythropoietic protoporphyria and DISC‑3405 for polycythemia vera, underscoring a diversified approach to blood disorders.
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