Disc Medicine (IRON) Status Update summary

Bitopertin regulatory and commercial update

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

• 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, ensuring drug supply readiness, and ongoing commercial readiness activities.

• Launch preparations include reimbursement strategies, potential startup programs, and further launch details to be shared at the JP Morgan Healthcare Conference in January 2026.

• Ongoing collaboration with FDA for bitopertin review and APOLLO confirmatory trial enrollment.

DISC-0974 RALLY-MF phase II interim data

• Interim data from the RALLY-MF study show significant and durable improvements in anemia, including hemoglobin levels, transfusion burden, and fatigue, across all patient cohorts.

• Treatment with DISC-0974 led to over 75% reduction in hepcidin and increased serum iron, resulting in improved anemia measures.

• Overall response rates exceed 60%, with major response rates notably higher than historical benchmarks; 63% of non-transfusion dependent patients achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks.

• Safety profile remains favorable, with most adverse events non-serious and no treatment-related serious adverse events or early withdrawals.

• Phase III pivotal trial is planned, with an end-of-phase II FDA meeting anticipated and additional data expected in H2 2026.

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.

• The mechanism of DISC-3405 is differentiated, aiming to induce endogenous hepcidin and address diseases with iron overload or erythrocytosis.

• Targeting 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 inflammatory bowel disease and development of a long-acting antibody, DISC-0998.

• Continuing first-in-patient studies for DISC-3405 and exploring additional indications.