{"id":"alxn2060","safety":{"commonSideEffects":[{"rate":null,"effect":"Meningococcal infection"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Fatigue"},{"rate":null,"effect":"Infusion-related reactions"}]},"_chembl":{"chemblId":"CHEMBL4650226","moleculeType":"Small molecule","molecularWeight":"328.77"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"ALXN2060 selectively inhibits complement component C3, preventing its cleavage and activation of both the classical and alternative complement pathways. By targeting C3, the drug reduces downstream complement-mediated inflammation, cell lysis, and tissue injury. This mechanism is intended to treat complement-driven diseases including paroxysmal nocturnal hemoglobinuria (PNH) and other rare blood disorders.","oneSentence":"ALXN2060 is a complement C3 inhibitor that blocks the central complement pathway to reduce inflammation and tissue damage.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:01:04.335Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Paroxysmal nocturnal hemoglobinuria (PNH)"},{"name":"C3 glomerulopathy"}]},"trialDetails":[{"nctId":"NCT04958135","phase":"PHASE1","title":"Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants","status":"COMPLETED","sponsor":"Alexion Pharmaceuticals, Inc.","startDate":"2021-05-30","conditions":"Healthy","enrollment":18},{"nctId":"NCT06563895","phase":"PHASE3","title":"Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant","status":"RECRUITING","sponsor":"Eidos Therapeutics, a BridgeBio company","startDate":"2025-05-12","conditions":"Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis","enrollment":582},{"nctId":"NCT04622046","phase":"PHASE3","title":"A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM","status":"COMPLETED","sponsor":"Alexion Pharmaceuticals, Inc.","startDate":"2020-11-13","conditions":"Symptomatic Transthyretin Amyloid Cardiomyopathy","enrollment":25},{"nctId":"NCT04988386","phase":"PHASE3","title":"Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants","status":"ACTIVE_NOT_RECRUITING","sponsor":"Eidos Therapeutics, a BridgeBio company","startDate":"2021-10-27","conditions":"Amyloid Cardiomyopathy, Transthyretin-Related","enrollment":389},{"nctId":"NCT07116473","phase":"PHASE3","title":"To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)","status":"NOT_YET_RECRUITING","sponsor":"Eidos Therapeutics, a BridgeBio company","startDate":"2025-11","conditions":"Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy","enrollment":250},{"nctId":"NCT03860935","phase":"PHASE3","title":"Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy","status":"COMPLETED","sponsor":"Eidos Therapeutics, a BridgeBio company","startDate":"2019-03-19","conditions":"Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis","enrollment":632}],"_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":["AG10","Acoramidis"],"phase":"phase_3","status":"active","brandName":"ALXN2060","genericName":"ALXN2060","companyName":"Alexion Pharmaceuticals, Inc.","companyId":"alexion-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"ALXN2060 is a complement C3 inhibitor that blocks the central complement pathway to reduce inflammation and tissue damage. Used for Paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}