{"id":"apl-2","safety":{"commonSideEffects":[{"rate":null,"effect":"Meningococcal infection risk"},{"rate":null,"effect":"Nasopharyngitis"},{"rate":null,"effect":"Headache"}]},"_chembl":{"chemblId":"CHEMBL4594355","moleculeType":"Unknown"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"APL-2 inhibits complement factor C3, a central hub protein in the complement cascade that is activated in all three pathways (classical, alternative, and lectin). By blocking C3 activation and cleavage, the drug prevents downstream complement-mediated inflammation, cell lysis, and tissue injury. This mechanism is intended to treat diseases driven by excessive or dysregulated complement activation.","oneSentence":"APL-2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:02:03.327Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Geographic atrophy (age-related macular degeneration)"},{"name":"C3 glomerulopathy"},{"name":"Post-transplant thrombotic microangiopathy"}]},"trialDetails":[{"nctId":"NCT07495722","phase":"PHASE1","title":"Safely Quenching Complement in Stroke Survivors","status":"NOT_YET_RECRUITING","sponsor":"Columbia University","startDate":"2026-04","conditions":"Acute Ischemic Stroke, Cerebral Infarction","enrollment":20},{"nctId":"NCT04919629","phase":"PHASE2","title":"APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion","status":"RECRUITING","sponsor":"Roswell Park Cancer Institute","startDate":"2023-04-27","conditions":"Fallopian Tube Carcinosarcoma, Fallopian Tube Clear Cell Adenocarcinoma, Fallopian Tube Endometrioid Adenocarcinoma","enrollment":60},{"nctId":"NCT07215390","phase":"PHASE2","title":"A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio","status":"RECRUITING","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2025-06-23","conditions":"Geographic Atrophy Secondary to Age-related Macular Degeneration","enrollment":240},{"nctId":"NCT07214298","phase":"PHASE1, PHASE2","title":"Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma","status":"RECRUITING","sponsor":"Roswell Park Cancer Institute","startDate":"2026-03-01","conditions":"Metastatic Pancreatic Ductal Adenocarcinoma, Stage IV Pancreatic Cancer AJCC v8","enrollment":35},{"nctId":"NCT07214740","phase":"PHASE3","title":"Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2025-10-24","conditions":"Geographic Atrophy Secondary to Age-related Macular Degeneration","enrollment":44},{"nctId":"NCT04770545","phase":"PHASE3","title":"An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2021-03-04","conditions":"Geographic Atrophy Secondary to Age-related Macular Degeneration","enrollment":792},{"nctId":"NCT07213960","phase":"PHASE2, PHASE3","title":"A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis","status":"NOT_YET_RECRUITING","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2025-12","conditions":"FSGS","enrollment":270},{"nctId":"NCT04901936","phase":"PHASE2","title":"A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","status":"RECRUITING","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2021-02-04","conditions":"Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria","enrollment":12},{"nctId":"NCT04579666","phase":"PHASE2","title":"MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)","status":"TERMINATED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2020-09-30","conditions":"Amyotrophic Lateral Sclerosis, Motor Neuron Disease","enrollment":249},{"nctId":"NCT05809531","phase":"PHASE3","title":"An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis","status":"ACTIVE_NOT_RECRUITING","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2023-05-29","conditions":"C3G, IC-MPGN, C3 Glomerulopathy","enrollment":100},{"nctId":"NCT03453619","phase":"PHASE2","title":"Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-02-26","conditions":"IgA Nephropathy, Lupus Nephritis, Membranous Nephropathy","enrollment":21},{"nctId":"NCT03226678","phase":"PHASE2","title":"Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2017-08-31","conditions":"Warm Autoimmune Hemolytic Anemia, Cold Agglutinin Disease","enrollment":24},{"nctId":"NCT03525613","phase":"PHASE3","title":"A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-08-31","conditions":"Geographic Atrophy","enrollment":637},{"nctId":"NCT03525600","phase":"PHASE3","title":"Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-08-31","conditions":"Geographic Atrophy","enrollment":621},{"nctId":"NCT04085601","phase":"PHASE3","title":"A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2019-08-27","conditions":"Paroxysmal Nocturnal Hemoglobinuria","enrollment":53},{"nctId":"NCT03500549","phase":"PHASE3","title":"Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-06-14","conditions":"Paroxysmal Nocturnal Hemoglobinuria","enrollment":80},{"nctId":"NCT03777332","phase":"PHASE1","title":"Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-11-05","conditions":"Geographic Atrophy","enrollment":19},{"nctId":"NCT02588833","phase":"PHASE1","title":"Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2015-12-01","conditions":"Paroxysmal Nocturnal Hemoglobinuria","enrollment":23},{"nctId":"NCT02264639","phase":"PHASE1","title":"A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2015-02-23","conditions":"Paroxysmal Nocturnal Hemoglobinuria (PNH)","enrollment":9},{"nctId":"NCT03593200","phase":"PHASE2","title":"A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-08-16","conditions":"PNH","enrollment":4},{"nctId":"NCT02503332","phase":"PHASE2","title":"Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2015-09-24","conditions":"Geographic Atrophy","enrollment":246},{"nctId":"NCT02461771","phase":"PHASE1","title":"Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD","status":"COMPLETED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2015-01-28","conditions":"Neovascular Age-Related Macular Degeneration","enrollment":13},{"nctId":"NCT03465709","phase":"PHASE1, PHASE2","title":"Pegcetacoplan (APL-2) in Neovascular AMD","status":"TERMINATED","sponsor":"Apellis Pharmaceuticals, Inc.","startDate":"2018-02-14","conditions":"Neovascular Age-related Macular Degeneration","enrollment":17}],"_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":["Pegcetacoplan"],"phase":"phase_3","status":"active","brandName":"APL-2","genericName":"APL-2","companyName":"Apellis Pharmaceuticals, Inc.","companyId":"apellis-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"APL-2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage. Used for Geographic atrophy (age-related macular degeneration), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":5,"withResults":5},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}