{"id":"adx-324","safety":{"commonSideEffects":[]},"_chembl":{"chemblId":"CHEMBL3545036","moleculeType":"Small molecule","molecularWeight":"240.28"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"ADX-324 works by activating ADAR (adenosine deaminase acting on RNA) enzymes, which catalyze the conversion of adenosine to inosine in RNA molecules. This RNA editing process can alter the amino acid sequence of proteins or affect RNA stability and localization, potentially correcting disease-causing mutations or modulating pathogenic protein function. This represents a novel approach to treating genetic and acquired diseases through post-transcriptional RNA modification.","oneSentence":"ADX-324 is an ADAR enzyme activator that enhances adenosine-to-inosine RNA editing to modulate gene expression and protein function.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:00:01.723Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Duchenne muscular dystrophy (DMD)"}]},"trialDetails":[{"nctId":"NCT05691361","phase":"PHASE1, PHASE2","title":"Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients","status":"ACTIVE_NOT_RECRUITING","sponsor":"ADARx Pharmaceuticals, Inc.","startDate":"2022-12-14","conditions":"Hereditary Angioedema","enrollment":53},{"nctId":"NCT07428499","phase":"PHASE3","title":"Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)","status":"NOT_YET_RECRUITING","sponsor":"ADARx Pharmaceuticals, Inc.","startDate":"2026-04-01","conditions":"Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2","enrollment":90},{"nctId":"NCT06960213","phase":"PHASE3","title":"STOP-HAE: A Phase 3 Study of ADX-324 in HAE","status":"RECRUITING","sponsor":"ADARx Pharmaceuticals, Inc.","startDate":"2025-08-28","conditions":"Hereditary Angioedema, HAE, Hereditary Angioedema - Type 1","enrollment":90}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["siRNA"],"phase":"phase_3","status":"active","brandName":"ADX-324","genericName":"ADX-324","companyName":"ADARx Pharmaceuticals, Inc.","companyId":"adarx-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"ADX-324 is an ADAR enzyme activator that enhances adenosine-to-inosine RNA editing to modulate gene expression and protein function. Used for Duchenne muscular dystrophy (DMD).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":false,"trials":true,"score":3}}