Disc Medicine Achieves >75% Hepcidin Reduction and 55% Major Hemoglobin Response
IRON•DISC-0974 treatment in 50 evaluable myelofibrosis patients achieved >75% hepcidin reduction, with 55% experiencing ≥1.5 g/dL hemoglobin gain for ≥12 weeks and 73% cutting transfusions by ≥50%. In 86 erythropoietic protoporphyria patients, bitopertin 60 mg daily delivered sustained PPIX reduction, light tolerance gains and favorable safety through 2.5+ years.
1. RALLY-MF Phase 2 Updates
RALLY-MF enrolled 61 myelofibrosis patients, 50 of whom were included in the responder analysis. DISC-0974 at 50 mg every four weeks produced over 75% hepcidin reduction, with 55% of patients achieving a ≥1.5 g/dL hemoglobin rise for at least 12 weeks and 73% cutting transfusion needs by ≥50%, alongside improvements in fatigue scores and minimal drug-related adverse events.
2. HELIOS Extension Results
HELIOS open-label extension enrolled 86 erythropoietic protoporphyria patients, all transitioning to 60 mg daily bitopertin. Treatment sustained significant reductions in protoporphyrin IX levels, improved average light tolerance and time to prodrome measures, and maintained a favorable safety profile over more than 2.5 years of follow-up across adult and adolescent cohorts.
3. Upcoming Catalysts
Disc Medicine will host a corporate update call on June 15 to review the EHA data and strategic priorities. The company aims to complete End of Phase 2 discussions with the FDA for DISC-0974 by year-end, expects the Phase 3 APOLLO readout in Q4 for bitopertin, and plans initial RESTORE-PV results in Q4 2026, setting up a potential third pivotal program in 2027.
