Disc Medicine (IRON) Jefferies 2024 Global Healthcare Conference summary

Strategic focus and portfolio overview

• Focus on manipulating red blood cell biology by targeting heme and iron metabolism, with three clinical-stage molecules addressing both rare and common hematologic disorders.

• Lead program bitopertin targets erythropoietic protoporphyria (EPP), with phase II trials completed and plans to progress to pivotal trials.

• DISC-0974, an antibody for anemias of inflammation, is in phase 1b/2 studies for myelofibrosis and chronic kidney disease, with ongoing data collection.

• DISC-3405, targeting TMPRSS6, aims to restrict iron for diseases like polycythemia vera, with first clinical data expected soon.

• All programs leverage fundamental mechanisms of red blood cell formation and iron regulation.

Recent clinical results and upcoming data

• Bitopertin phase II trials (AURORA and BEACON) showed strong reduction in toxic protoporphyrin IX, with a 40-50% decrease from baseline and placebo.

• AURORA trial met its primary endpoint but had mixed secondary results due to an unexpected placebo effect; robust reduction in phototoxic reactions and improved quality of life were observed.

• DISC-0974 showed promising hepcidin suppression and iron mobilization in myelofibrosis patients, with a 57% hemoglobin response rate at ASH and transfusion independence in some cases.

• DISC-3405 preclinical data supports its use in iron overload disorders, with healthy volunteer data to be presented at EHA.

• Upcoming EHA meeting will feature updated and expanded datasets for all three programs, including new analyses and strategies for future pivotal trials.

Forward-looking plans and financial position

• Plans to present new analyses at EHA to address placebo effects and define robust endpoints for pivotal bitopertin trials, with a focus on the 60 mg dose.

• End-of-phase II meeting with FDA expected in the second half of the year, aiming to start pivotal bitopertin trials next year.

• DISC-0974 data in chronic kidney disease to be presented at a kidney conference later in the year.

• DISC-3405 to move into polycythemia vera studies next year, following healthy volunteer data releases.

• Financially strong with $340 million in cash, providing runway into 2026 to support ongoing and planned clinical development.