{"id":"ag-348","safety":{"commonSideEffects":[{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Diarrhea"},{"rate":null,"effect":"Abdominal pain"}]},"_chembl":{"chemblId":"CHEMBL4299940","moleculeType":"Small molecule","molecularWeight":"450.56"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"AG-348 is a direct activator of pyruvate kinase R (PKR), the predominant form of pyruvate kinase in red blood cells. By enhancing PKR activity, the drug increases glycolytic ATP production in erythrocytes, which stabilizes the red blood cell membrane and reduces hemolysis. This mechanism is particularly beneficial in hemolytic anemias where red blood cell survival is compromised.","oneSentence":"AG-348 activates pyruvate kinase R (PKR), an enzyme that increases ATP production in red blood cells to improve their survival and reduce hemolysis.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:00:10.600Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Pyruvate kinase deficiency"},{"name":"Sickle cell disease"}]},"trialDetails":[{"nctId":"NCT05031780","phase":"PHASE2, PHASE3","title":"A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Agios Pharmaceuticals, Inc.","startDate":"2022-02-11","conditions":"Sickle Cell Disease","enrollment":286},{"nctId":"NCT06286046","phase":"PHASE2","title":"A Study of Mitapivat in Participants With Sickle Cell Disease and 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Erythrocyte Membranopathies","status":"NOT_YET_RECRUITING","sponsor":"EuroBloodNet Association","startDate":"2023-12-21","conditions":"Hereditary Red Blood Cell Disorder (Disorder)","enrollment":25},{"nctId":"NCT04995315","phase":"","title":"Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008","status":"COMPLETED","sponsor":"Agios Pharmaceuticals, Inc.","startDate":"2021-07-02","conditions":"Pyruvate Kinase Deficiency","enrollment":13},{"nctId":"NCT05610657","phase":"PHASE1","title":"A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function","status":"COMPLETED","sponsor":"Agios Pharmaceuticals, Inc.","startDate":"2023-01-10","conditions":"Moderate Hepatic Impairment","enrollment":20},{"nctId":"NCT04964323","phase":"","title":"Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes 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Used for Pyruvate kinase deficiency, Sickle cell disease.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":7,"withResults":5},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}