{"id":"amx0035","safety":{"commonSideEffects":[{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Vomiting"},{"rate":null,"effect":"Diarrhea"},{"rate":null,"effect":"Abdominal pain"},{"rate":null,"effect":"Headache"}]},"_chembl":{"chemblId":"CHEMBL1469","moleculeType":"Small molecule","molecularWeight":"164.20"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The combination works through dual mechanisms: sodium phenylbutyrate acts as a nitrogen scavenger and chemical chaperone to reduce protein misfolding and ER stress, while taurursodiol is a bile acid derivative that stabilizes mitochondrial membranes and reduces apoptosis. Together, these agents target the neurodegeneration cascade in motor neuron diseases by reducing oxidative stress, neuroinflammation, and cell death pathways.","oneSentence":"AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol that reduces neuroinflammation and protects motor neurons by stabilizing mitochondrial function and reducing ER stress.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:01:31.112Z","_scrapedBy":"cloudflare-swarm","_wikipedia":{"url":"https://en.wikipedia.org/wiki/Sodium_phenylbutyrate%2Fursodoxicoltaurine","title":"Sodium phenylbutyrate/ursodoxicoltaurine","extract":"Sodium phenylbutyrate/ursodoxicoltaurine, also known as sodium phenylbutyrate/taurursodiol and sold under the brand names Albrioza and Relyvrio, is a fixed-dose combination medication used for the treatment of amyotrophic lateral sclerosis (ALS). It contains sodium phenylbutyrate and ursodoxicoltaurine (taurursodiol)."},"indications":{"approved":[{"name":"Amyotrophic lateral sclerosis (ALS)"}]},"trialDetails":[{"nctId":"NCT03488524","phase":"PHASE2","title":"Open Label Extension Study of AMX0035 in Patients With ALS","status":"COMPLETED","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2018-03-29","conditions":"Amyotrophic Lateral Sclerosis, ALS","enrollment":90},{"nctId":"NCT05676034","phase":"PHASE2","title":"AMX0035 in Adult Patients With Wolfram Syndrome","status":"ACTIVE_NOT_RECRUITING","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2023-03-03","conditions":"Wolfram Syndrome","enrollment":12},{"nctId":"NCT03533257","phase":"PHASE2","title":"Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease","status":"COMPLETED","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2018-09-14","conditions":"Alzheimer Disease","enrollment":95},{"nctId":"NCT05619783","phase":"PHASE3","title":"Extension Study Evaluating The Safety And Tolerability of AMX0035","status":"COMPLETED","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2022-12-29","conditions":"Amyotrophic Lateral Sclerosis","enrollment":352},{"nctId":"NCT06122662","phase":"PHASE2, PHASE3","title":"AMX0035 and Progressive Supranuclear Palsy","status":"ACTIVE_NOT_RECRUITING","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2023-12-21","conditions":"Progressive Supranuclear Palsy, PSP, Neurodegenerative Diseases","enrollment":110},{"nctId":"NCT05021536","phase":"PHASE3","title":"Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment","status":"ACTIVE_NOT_RECRUITING","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2021-10-28","conditions":"Amyotrophic Lateral Sclerosis","enrollment":664},{"nctId":"NCT03127514","phase":"PHASE2","title":"AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)","status":"COMPLETED","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2017-06-22","conditions":"Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases","enrollment":137},{"nctId":"NCT04987671","phase":"PHASE1, PHASE2","title":"Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS","status":"UNKNOWN","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2021-08-05","conditions":"ALS","enrollment":14},{"nctId":"NCT04516096","phase":"PHASE2, PHASE3","title":"A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)","status":"COMPLETED","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"2020-11-22","conditions":"Amyotrophic Lateral Sclerosis","enrollment":28},{"nctId":"NCT05286372","phase":"","title":"An Intermediate Size Expanded Access Protocol of AMX0035 for ALS","status":"APPROVED_FOR_MARKETING","sponsor":"Amylyx Pharmaceuticals Inc.","startDate":"","conditions":"Amyotrophic Lateral Sclerosis","enrollment":""}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":17,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO; also known as Tauroursodeoxycholic Acid [TUDCA])","Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine or Tauroursodeoxycholic Acid [TUDCA])","Proprietary formulation of taurursodiol and sodium phenylbutyrate","RELYVRIO"],"phase":"phase_3","status":"active","brandName":"AMX0035","genericName":"AMX0035","companyName":"Amylyx Pharmaceuticals Inc.","companyId":"amylyx-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol that reduces neuroinflammation and protects motor neurons by stabilizing mitochondrial function and reducing ER stress. Used for Amyotrophic lateral sclerosis (ALS).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":6,"withResults":3},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}