On June 2, 2026 Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, reported updated data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF at the ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, IL. In this updated data set, treatment with DISC-0974 shows substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types.

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"We are excited to have solidified and strengthened the magnitude, durability, and consistency of responses as we have enrolled more patients and extended follow-up," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. "The activity observed both with and without background JAK inhibitor therapy, together with improvements in transfusion burden and fatigue, continues to support the potential for DISC-0974 to serve a broad population of patients living with MF-associated anemia, an area where substantial unmet need remains."

This ongoing Phase 2 open-label study had enrolled 61 adult patients with MF and anemia as of the data cutoff date of April 27, including 50 patients with sufficient follow up to be included in the responder analysis (non-transfusion dependent receiving no transfusions (nTD, n=31), transfusion dependent with low transfusion burden (TD Low, n=11) and transfusion dependent with high transfusion burden (TD High, n=8)). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=25) and not receiving JAK inhibitor therapy (n=25). DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments. The updated results demonstrated:

Consistent, substantial decreases in hepcidin reaching >75% reduction from baseline and corresponding increases in serum iron
55% (N=17 of 31) of baseline nTD patients achieved a hemoglobin increase of ≥1.5 g/dL for ≥12 weeks (major response) and 68% had an increase of ≥1 g/dL for ≥12 weeks (overall response)
64% (N=7 of 11) of TD Low patients achieved transfusion independence (TI, major response) over a 16-week period and 73% achieved a ≥50% reduction in transfusions (overall response)
50% (N=4 of 8) of TD High patients achieved transfusion independence (TI, major response) over a 12-week period and 88% achieved a ≥50% reduction in transfusion requirement (overall response)
56% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response across transfusion groups and 72% achieved an overall response, with similar response rates regardless of which specific JAK inhibitor the patient received
Dosing with DISC-0974 was associated with improvements in patient-reported outcomes:
Clinically significant improvements in FACIT-Fatigue scores in nTD and TD Low participants that were correlated with hemoglobin change
MPN-SAF TSS50 at EOS was achieved by 50% of nTD and TD low major responders
DISC-0974 was generally well-tolerated. Diarrhea, not considered serious, was the only adverse event (AE) that was reported as related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974.

Additional data from the RALLY-MF study is to be shared in Q4 2026, with an end of Phase 2 meeting with the FDA expected to occur by end of year.

DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide

(Press release, Disc Medicine, JUN 2, 2026, View Source [SID1234666398])