{"id":"apl-2-pegcetacoplan","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Meningococcal infection risk"},{"rate":null,"effect":"Nasopharyngitis"},{"rate":null,"effect":"Headache"}]},"_chembl":{"chemblId":"CHEMBL4298211","moleculeType":"Protein"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"APL-2 (pegcetacoplan) is a synthetic cyclic peptide that binds to complement component C3 and prevents its cleavage into C3a and C3b, thereby inhibiting both the classical and alternative complement pathways. By blocking C3 activation, the drug reduces downstream complement-mediated inflammation and tissue damage, which is implicated in several rare and systemic diseases.","oneSentence":"Pegcetacoplan is a complement C3 inhibitor that blocks the activation and amplification of the complement cascade by targeting C3.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:02:08.098Z","_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":"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":"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":271,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_3","status":"active","brandName":"APL-2, Pegcetacoplan","genericName":"APL-2, Pegcetacoplan","companyName":"Apellis Pharmaceuticals, Inc.","companyId":"apellis-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Pegcetacoplan is a complement C3 inhibitor that blocks the activation and amplification of the complement cascade by targeting C3. Used for Geographic atrophy (age-related macular degeneration), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}