Disc Medicine (IRON) Corporate presentation summary

Strategic focus and pipeline overview

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

• Lead programs include bitopertin (heme synthesis modulation), DISC-0974 (hepcidin suppression), and DISC-3405 (hepcidin induction), each advancing through clinical stages.

• Programs are positioned for blockbuster markets: EPP, MF anemia, and PV, with expansion potential into additional indications.

Bitopertin clinical development and results

• Bitopertin, an oral GlyT1 inhibitor, demonstrated significant reductions in PPIX and improvements in sunlight tolerance for EPP/XLP patients in Phase 2/3 trials.

• Clinical trials (AURORA, BEACON, HELIOS) showed up to 60% PPIX reduction, 2x increase in pain-free sunlight exposure, and 75–92% reduction in phototoxic reactions.

• Long-term safety and efficacy confirmed in HELIOS extension, with sustained quality-of-life improvements.

• APOLLO Phase 3 trial fully enrolled; topline data expected Q4 2026, supporting resubmission for FDA approval.

DISC-0974 clinical progress and opportunity

• DISC-0974, an anti-HJV mAb, suppresses hepcidin to increase iron availability, targeting anemia of inflammation and MF.

• Phase 2 RALLY-MF data show durable hemoglobin increases and transfusion reduction across all anemia severities, with 50–73% major response rates.

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

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

• Phase 2 in IBD anemia initiated; additional MF data and pivotal trial plans expected by end of 2026.