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DISC-1459
DISC-1459 is a hepcidin mimetic that increases hepcidin levels to reduce iron absorption and circulating iron, thereby treating iron overload disorders.
DISC-1459 is a hepcidin mimetic that increases hepcidin levels to reduce iron absorption and circulating iron, thereby treating iron overload disorders. Used for Hereditary hemochromatosis, Transfusion-dependent anemias with iron overload.
At a glance
| Generic name | DISC-1459 |
|---|---|
| Also known as | Bitopertin, RO4917838 |
| Sponsor | Disc Medicine, Inc |
| Drug class | Hepcidin mimetic |
| Target | Ferroportin (iron exporter); hepcidin pathway agonist |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
Hepcidin is the master regulator of iron homeostasis that controls ferroportin-mediated iron export from cells. By mimicking hepcidin's function, DISC-1459 enhances iron sequestration and reduces systemic iron levels. This mechanism is designed to treat conditions characterized by pathological iron accumulation, such as hereditary hemochromatosis and transfusion-dependent anemias.
Approved indications
- Hereditary hemochromatosis
- Transfusion-dependent anemias with iron overload
Common side effects
- Anemia
- Iron deficiency
- Gastrointestinal disorders
Key clinical trials
- Study of Bitopertin in Participants With EPP or XLP (APOLLO) (PHASE3)
- Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline (PHASE1)
- Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP (PHASE2)
- HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- DISC-1459 CI brief — competitive landscape report
- DISC-1459 updates RSS · CI watch RSS
- Disc Medicine, Inc portfolio CI