DISC-3405 - (RESTORE-PV)

July 30, 2026: Disc Medicine, Inc. (NASDAQ: IRON) reported Q2 2026 results and provided pipeline/regulatory updates across bitopertin, selcodebart (DISC-0974), and DISC-3405.

Bitopertin (GlyT1 inhibitor) in erythropoietic protoporphyria (EPP): The company completed a Type A FDA meeting regarding a prior CRL and aligned that the Phase 3 APOLLO study, if successful, can support the CRL response and could potentially support a traditional approval. APOLLO completed enrollment in March 2026 (N=183); topline results are on track for Q4 2026. Disc expects to submit a CRL response and receive an FDA decision by mid-2027. Disc also launched an Expanded Access Program for eligible EPP and XLP patients and presented HELIOS open-label extension updates at EHA showing sustained PPIX reductions, improved light tolerance measures, and favorable longer-term safety.

Selcodebart (anti-hemojuvelin) in myelofibrosis (MF) anemia: Updated RALLY-MF data presented at ASCO and EHA showed a 56% major anemia response rate and 72% overall anemia response rate, with similar responses across transfusion cohorts and regardless of background JAK inhibitor use. Additional RALLY-MF data are anticipated Q4 2026; an end-of-Phase 2 FDA meeting is expected by year-end. Received EU Orphan Drug Designation for MF; Phase 2 in IBD has initial data expected in 2027.

DISC-3405 (anti-TMPRSS6): RESTORE-PV Phase 2 in polycythemia vera fully enrolled; initial data expected Q3 2026. Phase 1b in sickle cell disease: initial data expected Q4 2026.

Financials: Cash/cash equivalents/marketable securities of $717.7M at June 30, 2026; runway into 2029. Net loss was $59.5M for Q2 2026.