Disc Medicine (IRON) Status Update summary

Strategic focus and pipeline overview

• Advancing NDA filing for bitopertin in EPP, with phase II completed and APOLLO trial underway.

• DISC-0974, a monoclonal antibody targeting hemojuvelin to suppress hepcidin, is in phase II for myelofibrosis anemia and phase I-B for CKD anemia.

• DISC-3405, another monoclonal antibody, is being developed for polycythemia vera and iron overload diseases.

Disease background and unmet need

• Myelofibrosis is a rare blood cancer with a high prevalence of anemia, impacting prognosis and quality of life.

• Anemia in myelofibrosis is multifactorial, involving bone marrow fibrosis, splenomegaly, JAK inhibitor effects, and elevated hepcidin.

• Anemia is prognostic, with severity correlating with reduced survival and increased healthcare burden.

• Current treatments for anemia are limited, with no approved agents specifically for myelofibrosis-associated anemia.

Current and emerging treatment landscape

• Four JAK inhibitors are approved for myelofibrosis, mainly addressing spleen size and symptoms, but often worsening anemia.

• Momelotinib and pacritinib offer some anemia benefit, but many patients still require additional anemia therapies.

• Off-label options include ESAs, danazol, steroids, and luspatercept, each with limitations in efficacy or tolerability.

• Investigational agents like DISC-0974 and activin receptor ligand traps (luspatercept, elritercept) are in advanced clinical development.