Disc Medicine unveils positive multi-program clinical trial data at EHA 2026
Disc Medicine (NASDAQ:IRON) disclosed positive, updated clinical data across its core hematology pipeline during the 2026 European Hematology Association (EHA) Annual Meeting in Stockholm, Sweden.
The presentations highlighted robust efficacy readouts for its lead programs in myelofibrosis-associated anemia and erythropoietic protoporphyria (EPP), establishing a series of catalyst events for the second half of 2026.
In an oral presentation tracking its Phase 2 RALLY-MF study, the biopharmaceutical firm reported detailed results for DISC-0974, an anti-hemojuvelin monoclonal antibody designed to suppress overproduced hepcidin and restore iron homeostasis.
Among non-transfusion-dependent patients presenting with severe anemia, 55% achieved a meaningful hemoglobin increase of at least 1.5 grams per deciliter sustained for 12 weeks or longer.
The trial also demonstrated high clinical benefit among heavily transfused sub-populations: 64% of patients classified with a low transfusion burden and 50% of those with a high baseline transfusion burden achieved total transfusion independence.
The overall anemia responses remained durable regardless of whether patients were taking concurrent Janus kinase (JAK) inhibitor background therapies.
From a safety standpoint, DISC-0974 was generally well-tolerated, with mild diarrhea representing the only drug-related adverse event observed in more than two participants.
Concurrently, Disc shared clinical extension results from its Phase 2 HELIOS open-label extension trial evaluating bitopertin, an oral, small-molecule glycine transporter 1 (GlyT1) inhibitor targeting EPP.
Long-term treatment exposure up to 2.5 years generated sustained reductions in toxic protoporphyrin IX (PPIX) levels alongside corresponding improvements in patients' light tolerance and pain-free sun exposure metrics.
The longitudinal data confirmed a stable safety and tolerability profile across both adult and adolescent patient groups.
