Disc Medicine (IRON) investor relations material

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.

• Three lead investigational agents: bitopertin (GlyT1 inhibitor), DISC-0974 (anti-HJV mAb), and DISC-3405 (anti-TMPRSS6 mAb), each with distinct mechanisms and clinical targets.

• Programs span multiple development stages, with bitopertin in Phase 3 for rare blood disorders, DISC-0974 in Phase 2 for anemia of chronic diseases, and DISC-3405 in Phase 2 for polycythemia vera and sickle cell disease.

Bitopertin clinical progress and market opportunity

• Bitopertin demonstrated significant reductions in PPIX and improvements in sunlight tolerance and quality of life for EPP and XLP patients in Phase 2 and 3 trials.

• APOLLO Phase 3 trial fully enrolled, topline data expected Q4 2026, with submission for CRL response planned by end of 2026.

• 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.

DISC-0974: Hepcidin suppression for anemia

• DISC-0974 targets hepcidin to increase iron availability, showing proof-of-mechanism in healthy volunteers and proof-of-concept in myelofibrosis (MF) and IBD.

• RALLY-MF Phase 2 data show durable hemoglobin increases and transfusion reduction across anemia severity, with efficacy regardless of JAK inhibitor use.

• 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), with Phase 3 initiation expected in H1 2027.