Disc Medicine Posts $791 M Cash, FDA CRL for Bitopertin, Q4 2026 Readout

IRONIRON

Disc Medicine ended Q4 2025 with $791.2 million in cash, cash equivalents and marketable securities, extending funding into 2029 after raising $454.4 million through offerings. The FDA issued a Complete Response Letter for bitopertin in erythropoietic protoporphyria, while Phase 3 APOLLO enrollment completes in March 2026 with topline data due Q4 2026.

1. Financial Position

Disc Medicine ended 2025 with $791.2 million in cash, cash equivalents and marketable securities, up from $489.9 million a year earlier, following net proceeds of $454.4 million from underwritten offerings in January and October. This strong balance sheet is expected to fund the company’s operational plans into 2029.

2. Bitopertin Program and FDA Response

In February 2026 the FDA issued a Complete Response Letter citing insufficient evidence of association between percent change in PPIX and sunlight exposure‐based endpoints for bitopertin. The Phase 3 APOLLO trial in adults and adolescents with erythropoietic protoporphyria is on track to complete enrollment in March 2026, with topline data due in Q4 2026 and a planned CRL response submission thereafter.

3. DISC-0974 Phase 2 Data

Initial results from the RALLY-MF trial presented at the ASH conference demonstrated meaningful, durable improvements in hemoglobin levels and reduced transfusion burden across all patient subgroups with anemia of myelofibrosis, irrespective of baseline transfusion status or concomitant JAK inhibitor use. A Phase 2 study of DISC-0974 in inflammatory bowel disease–related anemia was initiated in Q1 2026, with initial data expected in 2027.

4. DISC-3405 Development Progress

The Phase 2 study of DISC-3405 in polycythemia vera is ongoing, and a Phase 1b study in sickle cell disease was initiated in Q4 2025. Data from both studies are anticipated in the second half of 2026, advancing DISC-3405’s potential across multiple hematologic indications.

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