Disc Medicine reports Q2 2026 results, aligns with FDA on bitopertin path in EPP

Disc Medicine, Inc. reported second quarter 2026 results on July 30, 2026, along with a series of pipeline updates. Disc completed a Type A meeting with the FDA to discuss the complete response letter for bitopertin in erythropoietic protoporphyria and aligned with the agency that the Phase 3 APOLLO study, if successful, could serve as the basis for a CRL response and could potentially support a traditional approval.¹ The company said it remains on track to report APOLLO topline results in the fourth quarter of 2026 and expects to submit its CRL response and receive an FDA decision by mid-2027, with APOLLO having completed enrollment in March 2026 at a final N of 183.¹

Disc also launched an Expanded Access Program for bitopertin in the US and select other geographies, providing eligible patients with EPP and XLP access to treatment ahead of a regulatory decision.¹ Separately, the company presented updated HELIOS open-label extension data on bitopertin in EPP at the European Hematology Association meeting, and shared updated RALLY-MF data on selcodebart (DISC-0974) in myelofibrosis anemia, showing a major anemia response rate of 56% and an overall anemia response rate of 72% across evaluable patients.¹ Additional RALLY-MF data and an end-of-Phase 2 FDA meeting for selcodebart are expected by year-end 2026, and selcodebart also received EU Orphan Drug Designation for myelofibrosis, and its IBD Phase 2 study is progressing with initial data expected in 2027.¹

For DISC-3405, the company said it completed enrollment for the RESTORE-PV Phase 2 study in polycythemia vera, with initial data expected in the third quarter of 2026, while a Phase 1b sickle cell disease study is progressing with data expected in the fourth quarter of 2026.¹

On finances, Disc reported approximately $718 million in cash, cash equivalents, and marketable securities at quarter end, which the company said provides runway into 2029.¹