Disc Medicine (IRON) Study update summary

Bitopertin Program Updates

• FDA aligned that the Phase III APOLLO study can support CRL resubmission and potential traditional approval, with submission expected by year-end or end of 2026.

• APOLLO Phase III study completed enrollment (183 patients) in March 2026, with top-line data expected in Q4 2026; strong adolescent representation and global diversity.

• HELIOS open-label extension showed sustained PPIX reduction, significant, durable improvements in light tolerance for over one year, and a favorable safety profile up to 2.5+ years.

• Expanded Access Program launched for eligible EPP and XLP patients in the US and select countries, maintaining clinical trial criteria.

• Commercial preparations underway, focusing on disease education, patient identification, and a potential launch mid-next-year.

Selcodebart (DISC-0974) in Myelofibrosis Anemia

• Phase II RALLY-MF study showed meaningful, durable anemia responses across all patient subgroups, regardless of transfusion status or JAK inhibitor use.

• Major response rates: 55% in non-transfused, 64% in TD low, and 50% in TD high cohorts; overall response rates up to 88%.

• Clinically significant improvements in fatigue and symptom scores, with 50% of major responders achieving MPN-SAF TSS50.

• Safety profile remains favorable, with no serious treatment-related adverse events; most common TEAEs were muscle spasms, diarrhea, and fatigue.

• Broad commercial potential estimated at over $4 billion in the U.S. alone, addressing both JAK inhibitor-treated and untreated MF patients.

Selcodebart in Other Anemias and Pipeline Expansion

• Phase 2 RALLY-IBD trial initiated in Q1 2026 for anemia of inflammatory bowel disease; preclinical data show hepcidin suppression and hematologic improvement.

• Exploratory work ongoing in additional anemia indications; IND-enabling activities for long-acting anti-HJV (DISC-0998) continue.