Disc Medicine Presents Updated Data From Myelofibrosis And Anemia Treatment RALLY-MF Phase 2 Trial Showing Reductions In Hepcidin And Higher Iron Levels, A Positive Clinical Impact On Anemia Measures Across A Broad Range Of Patient Types
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.
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