Disc Medicine presents clinical data updates across portfolio at EHA 2025

Disc Medicine (IRON) spotlights 5 posters presented at the EHA 2025 annual meeting in Milan, Italy. This year’s presentations included data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX reductions, improvement in quality of life, and improved liver biomarkers. Disc is advancing development and registrational activities for bitopertin in EPP, with plans to submit an NDA in H2 2025. The company has initiated APOLLO, a confirmatory clinical trial of bitopertin in adults and adolescents with EPP. Disc recently launched a campaign to raise awareness of EPP among physicians, patients, and caregivers with emphasis on the causative role of PPIX accumulation in the disease. Learn more at PPIXisWhy.com. Disc also presented longer term data from the continuation phase of its Phase 1b trial in MF anemia, showing sustained activity on key biomarkers and durable anemia response among major responders. Enrollment for the Phase 2 RALLY-MF trial of DISC-0974 is ongoing with the exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled. The company expects to present initial RALLY-MF data in H2 2025. Additionally, Disc shared an update of the Phase 1 healthy volunteer trial of DISC-3405, initially presented at ASH 2024, which demonstrated deep, sustained reductions in serum iron and meaningful changes in hematologic parameters, establishing proof of mechanism. Based on these data, the company has initiated a Phase 2 trial of DISC-3405 in polycythemia vera. Disc also presented an iron pulse study of DISC-3405 in healthy volunteers which showed inhibition of dietary iron uptake, further confirming the mechanism of action and supporting DISC-3405’s potential in diseases of iron overload. The collection of data presented at EHA adds to the evidence base supporting Disc’s continued advancement of all three clinical candidates and provides support for expansion opportunities in new indications. Management will host a call to review these updates on June 16 at 8:00 am ET.