{"id":"disc-1459","safety":{"commonSideEffects":[{"rate":null,"effect":"Anemia"},{"rate":null,"effect":"Iron deficiency"},{"rate":null,"effect":"Gastrointestinal disorders"}]},"_chembl":{"chemblId":"CHEMBL5877209","moleculeType":null,"molecularWeight":"447.94"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"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.","oneSentence":"DISC-1459 is a hepcidin mimetic that increases hepcidin levels to reduce iron absorption and circulating iron, thereby treating iron overload disorders.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:09:33.562Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Hereditary hemochromatosis"},{"name":"Transfusion-dependent anemias with iron overload"}]},"trialDetails":[{"nctId":"NCT06910358","phase":"PHASE3","title":"Study of Bitopertin in Participants With EPP or XLP (APOLLO)","status":"RECRUITING","sponsor":"Disc Medicine, Inc","startDate":"2025-04-04","conditions":"Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)","enrollment":150},{"nctId":"NCT07123363","phase":"PHASE1","title":"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Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline","status":"WITHDRAWN","sponsor":"Wake Forest University Health Sciences","startDate":"2026-01","conditions":"Protoporphyria","enrollment":""},{"nctId":"NCT05308472","phase":"PHASE2","title":"Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP","status":"COMPLETED","sponsor":"Disc Medicine, Inc","startDate":"2022-10-31","conditions":"Erythropoietic Protoporphyria","enrollment":75},{"nctId":"NCT05883748","phase":"PHASE2, PHASE3","title":"HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP","status":"ENROLLING_BY_INVITATION","sponsor":"Disc Medicine, Inc","startDate":"2023-08-31","conditions":"Erythropoietic Protoporphyria","enrollment":230}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Bitopertin","RO4917838"],"phase":"phase_3","status":"active","brandName":"DISC-1459","genericName":"DISC-1459","companyName":"Disc Medicine, Inc","companyId":"disc-medicine-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"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.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}