Disc Medicine (IRON) investor relations material

Bitopertin Regulatory and Commercial Update

• NDA for bitopertin in EPP accepted for review under the Commissioner's National Priority Voucher Program, with potential FDA action and launch by end of January 2026 if approved.

• Bitopertin selected for FDA's CNPV program, enabling a 1-2 month review period and potential accelerated approval.

• Accelerated commercialization efforts include hiring a 24-person sales force and ensuring drug supply readiness.

• Launch strategy details to be presented at the JPMorgan Healthcare Conference in January 2026.

• Ongoing collaboration with FDA for bitopertin review and APOLLO confirmatory trial enrollment, focusing on ex-U.S. sites.

DISC-0974 Clinical and Market Update

• Interim Phase 2 RALLY-MF data show significant and durable improvements in anemia, including hemoglobin, transfusion burden, and fatigue, across all patient cohorts, with over 75% hepcidin reduction and increased serum iron.

• 63% of non-transfusion dependent patients achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks, and 50% had an increase of ≥1.5 g/dL for ≥12 weeks.

• 71% of low transfusion burden patients achieved transfusion independence, and 67% of high transfusion burden patients saw a ≥50% reduction in transfusion needs.

• Consistent efficacy observed regardless of background JAK inhibitor therapy, with overall response rates above 60% and major response rates ≥50% in key cohorts.

• Safety profile remains favorable, with most adverse events non-serious and no early withdrawals due to adverse events.

DISC-3405 and Pipeline Expansion

• DISC-3405 is in phase II for polycythemia vera and phase I-B for sickle cell disease, with rapid enrollment and initial data expected by end of 2026.

• Strong enrollment interest in the polycythemia vera trial led to an expanded sample size.

• DISC-3405 targets high unmet need markets: ~150,000 US PV patients and ~120,000 US SCD patients.

• Additional pipeline efforts include a planned phase II study in anemia of IBD and development of a long-acting antibody, DISC-0998.

• Company is funded into 2029, excluding any bitopertin revenue, supporting multiple key milestones.