{"id":"alk-001-oral-capsule","safety":{"commonSideEffects":[{"rate":null,"effect":"Nyctalopia (impaired night vision)"},{"rate":null,"effect":"Photopsia (flashing lights)"},{"rate":null,"effect":"Reduced visual acuity in low light"}]},"_chembl":{"chemblId":"CHEMBL2108778","moleculeType":"Vaccine component"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"ALK-001 works by slowing the regeneration of visual pigment in the retina, thereby reducing the formation and accumulation of lipofuscin and other bisretinoids that contribute to retinal degeneration. By modulating the visual cycle, it decreases the metabolic burden on retinal pigment epithelium cells, potentially slowing progression of inherited retinal dystrophies. This mechanism is particularly relevant in conditions where bisretinoid accumulation drives photoreceptor cell death.","oneSentence":"ALK-001 is a retinoid that reduces toxic bisretinoid accumulation in the retina by inhibiting the visual cycle.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T00:44:21.053Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Geographic atrophy secondary to age-related macular degeneration"},{"name":"Stargardt disease"},{"name":"Other inherited retinal dystrophies"}]},"trialDetails":[{"nctId":"NCT03845582","phase":"PHASE2, PHASE3","title":"Phase 2/3 Study of ALK-001 in Geographic Atrophy","status":"COMPLETED","sponsor":"Alkeus Pharmaceuticals, Inc.","startDate":"2019-05-07","conditions":"Geographic Atrophy, Age Related Macular Degeneration, AMD","enrollment":200},{"nctId":"NCT02402660","phase":"PHASE2","title":"Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease","status":"ENROLLING_BY_INVITATION","sponsor":"Alkeus Pharmaceuticals, Inc.","startDate":"2015-08","conditions":"Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy","enrollment":160},{"nctId":"NCT02230228","phase":"PHASE1","title":"Phase 1 Safety Study of ALK-001 in Healthy Volunteers","status":"COMPLETED","sponsor":"Alkeus Pharmaceuticals, Inc.","startDate":"2014-04","conditions":"Stargardt Disease, Age-related Macular Degeneration, Other Retinal Dystrophies","enrollment":40}],"_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":["C20-D3-Retinyl Acetate","C20 Deuterated vitamin A","Gildeuretinol acetate"],"phase":"phase_3","status":"active","brandName":"ALK-001 oral capsule","genericName":"ALK-001 oral capsule","companyName":"Alkeus Pharmaceuticals, Inc.","companyId":"alkeus-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"ALK-001 is a retinoid that reduces toxic bisretinoid accumulation in the retina by inhibiting the visual cycle. Used for Geographic atrophy secondary to age-related macular degeneration, Stargardt disease, Other inherited retinal dystrophies.","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}