{"id":"ptc923","safety":{"commonSideEffects":[{"rate":null,"effect":"Peripheral neuropathy"},{"rate":null,"effect":"Gastrointestinal disturbances"},{"rate":null,"effect":"Fatigue"}]},"_chembl":{"chemblId":"CHEMBL1255653","moleculeType":"Small molecule","molecularWeight":"237.22"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"By inhibiting PDK, PTC923 allows pyruvate dehydrogenase to remain active, promoting the conversion of pyruvate to acetyl-CoA and enhancing oxidative metabolism. This mechanism is designed to address metabolic dysfunction in mitochondrial disorders and other conditions characterized by impaired energy production. The restoration of PDH activity aims to improve cellular energy availability and reduce lactate accumulation.","oneSentence":"PTC923 is a small-molecule inhibitor of pyruvate dehydrogenase kinase (PDK) that restores pyruvate dehydrogenase (PDH) activity to improve mitochondrial energy metabolism.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:30:39.018Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)"},{"name":"Other mitochondrial or metabolic disorders"}]},"trialDetails":[{"nctId":"NCT06302348","phase":"PHASE3","title":"A Study of Sepiapterin in Participants With Phenylketonuria (PKU)","status":"RECRUITING","sponsor":"PTC Therapeutics","startDate":"2024-03-04","conditions":"Phenylketonuria","enrollment":56},{"nctId":"NCT05166161","phase":"PHASE3","title":"A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria","status":"ACTIVE_NOT_RECRUITING","sponsor":"PTC Therapeutics","startDate":"2022-02-14","conditions":"Phenylketonuria","enrollment":200},{"nctId":"NCT05099640","phase":"PHASE3","title":"A Study of PTC923 in Participants With Phenylketonuria","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2021-09-30","conditions":"Phenylketonuria","enrollment":157},{"nctId":"NCT03519711","phase":"PHASE1, PHASE2","title":"A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2019-01-03","conditions":"BH4 Deficiency, Hyperphenylalaninemia","enrollment":8}],"_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":["CNSA-001, Sepiapterin","Sepiapterin","Sephience"],"phase":"phase_3","status":"active","brandName":"PTC923","genericName":"PTC923","companyName":"PTC Therapeutics","companyId":"ptc-therapeutics","modality":"Small molecule","firstApprovalDate":"","aiSummary":"PTC923 is a small-molecule inhibitor of pyruvate dehydrogenase kinase (PDK) that restores pyruvate dehydrogenase (PDH) activity to improve mitochondrial energy metabolism. Used for Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), Other mitochondrial or metabolic disorders.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":3,"withResults":2},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}