Disc Medicine (IRON) Barclays 27th Annual Global Healthcare Conference summary

Strategic overview and pipeline progress

• Focused on developing therapies for severe hematologic disorders by targeting red blood cell biology and iron metabolism pathways, with three clinical programs in mid to late-stage development.

• Lead program, bitopertin, targets erythropoietic protoporphyria (EPP) and has shown strong phase two results, leading to FDA alignment on an accelerated approval path; NDA filing planned for the second half of the year.

• Pipeline includes programs to both downregulate and upregulate hepcidin, addressing a range of anemias and iron overload disorders, with promising early data in myelofibrosis (MF) and chronic kidney disease (CKD).

• Phase 1b data in MF showed unprecedented hematologic activity, with phase two expansion ongoing and updates expected by year-end.

• DISC-3405, targeting polycythemia vera, leverages a novel approach to induce hepcidin and restrict iron, with phase two studies starting in the first half of the year.

Market opportunity and commercialization plans

• EPP patient population in the U.S. is estimated at 14,000 based on ICD-10 claims analysis, higher than previous literature estimates.

• Current treatment options for EPP are limited, with only one approved non-disease-modifying agent and another oral agent in phase three; significant unmet need remains.

• Commercial readiness efforts include building relationships with centers of excellence, key opinion leaders, and patient advocacy groups to support launch.

• Company is preparing for commercial launch and expects to be commercial within its current financial runway.

Financial position and outlook

• Ended the year with $490 million in cash and raised an additional $259 million in January, plus access to a $200 million debt facility with only $30 million drawn.

• Financial runway is expected to support the company through U.S. launch and commercialization, with proceeds from launch not yet included in projections.

• Confident in ability to deliver on phase two proof-of-concept for key pipeline programs and transition to a commercial-stage company.