IRON News & Events

Disc Medicine presented updated data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF at the ASCO Annual Meeting in Chicago. 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. 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. The trial was comprised of both patients receiving concomitant JAK inhibitor therapy and not receiving JAK inhibitor therapy. 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 greater than 75% reduction from baseline and corresponding increases in serum iron; 55% of baseline nTD patients achieved a hemoglobin increase of 1.5 g/dL for 12 weeks and 68% had an increase of =1 g/dL for =12 weeks; 64% of TD Low patients achieved transfusion independence over a 16-week period and 73% achieved a 50% reduction in transfusions; 50% of TD High patients achieved transfusion independence over a 12-week period and 88% achieved a 50% reduction in transfusion requirement; 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 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, 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.

"We continue to make strong progress across the portfolio, completing enrollment in our Phase 3 trial of bitopertin in EPP and remaining on track to report topline data in the fourth quarter of 2026. We were also pleased to have our Phase 2 data for DISC-0974 in MF anemia selected for oral presentation at ASCO, while continuing to advance multiple additional clinical trials across our pipeline," said John Quisel, Chief Executive Officer and President of Disc. "Together, these milestones reflect the strength of our execution and position us to deliver multiple important catalysts in the second half of this year."