Disc Medicine (IRON) Corporate presentation summary

Strategic focus and pipeline overview

• Portfolio targets fundamental pathways in red blood cell biology, focusing on heme and iron metabolism to address a spectrum of hematologic diseases from rare to widely prevalent conditions.

• Lead programs include bitopertin (GlyT1 inhibitor), selcodebart (anti-HJV mAb), and DISC-3405 (anti-TMPRSS6 mAb), each with distinct mechanisms and clinical targets.

• Programs are advancing through late-stage clinical trials, with bitopertin in Phase 3 for erythropoietic porphyrias, selcodebart in Phase 2 for myelofibrosis and IBD anemia, and DISC-3405 in Phase 2 for polycythemia vera and Phase 1b for sickle cell disease.

Bitopertin clinical progress and market opportunity

• Bitopertin demonstrated significant reductions in PPIX, improved sunlight tolerance, and reduced phototoxic reactions in EPP/XLP patients across multiple trials (AURORA, BEACON, HELIOS).

• APOLLO Phase 3 trial fully enrolled, with topline data expected Q4 2026; study design powered for robust efficacy endpoints.

• Potential to be the first approved disease-modifying therapy for EPP, addressing a $2B+ US market.

• Expanded Access Program launched for eligible patients in the US and select countries.

Selcodebart clinical progress and market opportunity

• Selcodebart targets hepcidin suppression to increase iron availability, with proof-of-concept established in healthy volunteers and myelofibrosis patients.

• RALLY-MF Phase 2 data show durable hemoglobin increases and transfusion reduction across all anemia severity cohorts, with clinically meaningful improvements in fatigue and quality of life.

• Safety profile consistent with target biology; most common adverse events were mild to moderate.

• Positioned to address the full MF anemia market (~$4B US opportunity) and expanding into other anemias of inflammation, including IBD.