{"id":"at-007","safety":{"commonSideEffects":[{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Diarrhea"}]},"_chembl":{"chemblId":"CHEMBL4303347","moleculeType":"Small molecule","molecularWeight":"455.60"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"AT-007 blocks aldose reductase, which catalyzes the conversion of glucose to sorbitol in the polyol pathway. By inhibiting this enzyme, the drug reduces intracellular sorbitol accumulation in tissues affected by diabetes, thereby mitigating osmotic stress and oxidative damage that contribute to diabetic complications such as peripheral neuropathy and other microvascular disease.","oneSentence":"AT-007 is a small molecule that inhibits aldose reductase, the first enzyme in the polyol pathway, to reduce sorbitol accumulation and prevent diabetic complications.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:02:45.068Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Diabetic peripheral neuropathy"},{"name":"Galactokinase deficiency"}]},"trialDetails":[{"nctId":"NCT04902781","phase":"PHASE2, PHASE3","title":"Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia","status":"COMPLETED","sponsor":"Applied Therapeutics, Inc.","startDate":"2021-03-20","conditions":"Classic Galactosemia","enrollment":47},{"nctId":"NCT07191912","phase":"PHASE3","title":"Confirmatory Study of Govorestat in CMT-SORD","status":"NOT_YET_RECRUITING","sponsor":"Applied Therapeutics, Inc.","startDate":"2025-10","conditions":"Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)","enrollment":155},{"nctId":"NCT05397665","phase":"PHASE2, PHASE3","title":"Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency","status":"ACTIVE_NOT_RECRUITING","sponsor":"Applied Therapeutics, Inc.","startDate":"2022-01-01","conditions":"Hereditary Neuropathy Caused by SORD Deficiency","enrollment":56},{"nctId":"NCT04117711","phase":"PHASE1, PHASE2","title":"Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia","status":"COMPLETED","sponsor":"Applied Therapeutics, Inc.","startDate":"2019-06-21","conditions":"Classic Galactosemia","enrollment":114},{"nctId":"NCT05418829","phase":"PHASE3","title":"AT-007 in Adult Subjects With Classic Galactosemia (CG)","status":"UNKNOWN","sponsor":"Applied Therapeutics, Inc.","startDate":"2021-10-01","conditions":"Classic Galactosemia","enrollment":7}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":9,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["aldose reductase inhibitor","Govorestat"],"phase":"phase_3","status":"active","brandName":"AT-007","genericName":"AT-007","companyName":"Applied Therapeutics, Inc.","companyId":"applied-therapeutics-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"AT-007 is a small molecule that inhibits aldose reductase, the first enzyme in the polyol pathway, to reduce sorbitol accumulation and prevent diabetic complications. Used for Diabetic peripheral neuropathy, Galactokinase deficiency.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":2,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}