Disc Medicine (IRON) Corporate presentation summary

Strategic focus and pipeline overview

• Portfolio targets heme and iron pathways to address a spectrum of hematologic diseases, from rare to widely prevalent conditions.

• Lead programs include bitopertin (GlyT1 inhibitor), DISC-0974 (anti-HJV mAb), and DISC-3405 (anti-TMPRSS6 mAb), each with distinct mechanisms and clinical targets.

• Programs are advancing through various clinical stages, with bitopertin in Phase 3, DISC-0974 in Phase 2, and DISC-3405 entering Phase 2.

Bitopertin clinical development and results

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

• Long-term data show sustained efficacy and safety, with over 80% of patients rolling over to extension studies.

• APOLLO Phase 3 trial is closing enrollment in March 2026, with topline data expected in Q4 2026.

• Planning for FDA resubmission following a complete response letter, with a potential decision by mid-2027.

DISC-0974 clinical progress and opportunity

• DISC-0974 targets hepcidin suppression to increase iron availability, addressing anemia in myelofibrosis (MF) and inflammatory diseases.

• Phase 2 RALLY-MF data show positive, durable benefits on hemoglobin and transfusion burden, with response rates above 60% across cohorts.

• Efficacy observed regardless of JAK inhibitor use, supporting broad applicability in MF.

• Safety profile is consistent with selective target biology, with no early withdrawals due to adverse events.

• Phase 2 trial in IBD anemia initiated in Q1 2026; topline MF data expected H2 2026.