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Clinical Trial
. 2025 Jul;56(7):398-406.
doi: 10.3928/23258160-20250217-01. Epub 2025 May 15.

Pegcetacoplan for Geographic Atrophy Over 30 Months: Data From OAKS, DERBY, and the GALE Long-Term Extension Study

Roger A Goldberg, David S Boyer, Frank G Holz, Mathew W MacCumber, Sunir J Garg, David M Brown, Eleonora M Lad, Nathan Steinle, P Singh Rishi, Ian Pearce, Thomas Ach, Ramiro Ribeiro, Chao Li, Daniel Jones, Min Tsuboi, Philip J Ferrone, Caroline R Baumal, Charles C Wykoff
Free article
Clinical Trial

Pegcetacoplan for Geographic Atrophy Over 30 Months: Data From OAKS, DERBY, and the GALE Long-Term Extension Study

Roger A Goldberg et al. Ophthalmic Surg Lasers Imaging Retina. 2025 Jul.
Free article

Abstract

Background and objective: This study will report safety and efficacy of pegcetacoplan for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Patients and methods: GALE is a phase 3, open-label, multicenter, 36-month extension of the OAKS and DERBY studies. Patients who received pegcetacoplan monthly (PM) or every other month (PEOM) in OAKS or DERBY continued the same regimen in GALE (PM-PM and PEOM-PEOM); sham-observed patients initiated pegcetacoplan, maintaining the same interval.

Results: In the first 6 months of GALE, 3.0% of study eyes developed exudative AMD, 1.3% intraocular inflammation, 0.1% ischemic optic neuropathy, and none endophthalmitis. Pegcetacoplan reduced GA growth rate by 39% (PM-PM) and 32% (PEOM-PEOM), with increasing efficacy over time across GA subtypes. In eyes with nonsubfoveal GA, pegcetacoplan reduced GA growth rate by 45% (PM-PM) and 33% (PEOM-PEOM).

Conclusion: Pegcetacoplan reduced GA growth rate up to 45% with increasing efficacy over 30 months and demonstrated a favorable safety profile. [Ophthalmic Surg Lasers Imaging Retina 2025;56:398-406.].

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Conflict of interest statement

Disclosure: RAG reports consulting and/or speaking fees from AbbVie, Allergan, Annexon, Apellis, Biogen, Boehringer Ingelheim, Carl Zeiss Meditech, Genentech/Roche, and Regeneron; receiving research/grant support from Allergan/AbbVie, Aerie, Alexion, Annexon, Apellis, Boehringer Ingelheim, Carl Zeiss Meditec, Eyepoint, Genentech/Roche, Janssen, Novo Nordisk, Ocuphire, and Unity Bio; and holding equity in Emmetrope Ophthalmics. DSB reports consulting fees from Acuela, Adverum, Aerpio, Alcon, Alkahest, Allegro, Allergan, Amgen, Amydis, Annexon, Apellis, Asclepix, Aviceda, Bausch & Lomb, Bayer, BioMotiv, Chengdu Kanghong, Clearside Biomedical, EyePoint, Genentech/Roche, Glaukos, Iveric, Jcyte, Kala, Kodiak, Lumithera, Nanoscope, Novartis, Oculis, Ocugenix, ONL Therapeutics, Ocular Therapeutics, Oxurion, Perfuse, Perceive, Ray, Recens Medical, Regeneron, Regenxbio, Thea, Unity, Stealth, and 4-DMT; and research grants from Acuela, Adverum, Aerie Pharmaceuticals, Aerpio, Allergan, Annexon, Apellis, Asclepix, Aviceda, Boehringer Ingelheim, Chengdu Kanghong, Clearside Biomedical, EyePoint, Gemini Therapeutics, Genentech/Roche, Graybug Vision, Ionis Pharmaceuticals, Iveric, Jcyte, Kodiak, Lumithera, Nanoscope, Neurotech, Novartis, Oculis, Opthea, Ocular Therapeutics, Outlook Therapeutics, Ray, Recens Medical, Regeneron, Regenxbio, Unity, and Stealth. FGH reports receiving institution grants from Apellis Pharmaceuticals, Iveric Bio, Genentech/Roche, Pixium Vision, and Heidelberg Engineering; receiving consultancy fees from Apellis Pharmaceuticals, Iveric Bio, Genentech/Roche, Pixium Vision, and Heidelberg Engineering; receiving financial support from Apellis Pharmaceuticals, Genentech/Roche, and Heidelberg Engineering; and serving as an Executive Board Member of EURETINA and the German Ophthalmological Society and as a Scientific Board Member of Pro Retina. MWM reports receiving consulting fees from AbbVie, Alimera Sciences, Apellis Pharmaceuticals, Astellas, Bausch & Lomb, EyePoint Pharmaceuticals, Genentech, Novartis, Ocular Therapeutix, and Regeneron; and receiving research funds from Aviceda and Regenxbio. SJG reports receiving consulting fees from American Academy of Ophthalmology, Apellis, Bausch & Lomb, Boehringer Ingelheim, Merck Manual, and West Pharmaceuticals; receiving contracted research funding from American Academy of Ophthalmology, Apellis, Boehringer Ingelheim, Genentech, NGM Bio, Regeneron, Kodiak Biosciences; serving on an advisory board for Coherus; and receiving lecture fees from Canadian Ophthalmological Society, Cole Eye Summit, Physicians' Education Resource, and Retina Fellows Forum. DMB reports receiving consulting fees from AAVantgarde Bio S.r.l., AbbVie, Alexion Pharmaceuticals Inc., Apellis Pharmaceuticals Inc., Bayer, Biogen MA Inc., Boehringer Ingelheim, Celltrion Inc., Chengdu Kanghong Pharmaceutical, Clearside Biomedical Inc., Heidelberg Engineering Inc., Impact Education, LLC, Kowa, Medscape LLC, Novartis Pharma AG, Ocular Therapeutix, Ocuphire Pharma Inc., OPTOS PLC, Pontifax, Regeneron Pharmaceuticals Inc., RetinAI Medical AG, Ray Therapeutics Inc., RMEI, Senju Pharmaceuticals Co. Ltd., SIMR Biotech, Stealth BioTherapeutics Inc., THEA, Topcon, WebMD Global LLC, Xequel Bio, and Xfacto; receiving grant support from AffaMed Therapeutics (US) Inc., Alcon, Alexion Pharmaceuticals Inc., Allergan, Amgen Inc., Annexon Inc., Apellis Pharmaceuticals Inc., Astellas, Bayer, BIRC, Boehringer Ingelheim, Chengdu Kanghong Biotechnology Co. Ltd., Clearside, Eyebiotech Ltd., EyePoint Pharmaceuticals Inc., F. Hoffmann- La Roche Limited, Gemini Therapeutics Inc., Genentech Inc., Graybug Vision Inc., Gyroscope Therapeutics Ltd., Heidelberg Engineering Inc., i-Lumen Scientific Inc., IONIS Pharmaceuticals, Jaeb-/DRCR AA, Jeff Heier MD, Kodiak Sciences Inc., LumiThera, MeiraGTx, Mylan, NEI/NIH, NGM Biopharmaceuticals Inc., Novartis, Ocular Therapeutix, Ocuphire Pharma Inc., OcuTerra Therapeutics Inc., Opthea, OPTOS, Outlook Therapeutics Inc., OXULAR, Pennsylvania State University, Regeneron Pharmaceuticals, RegenXBio, Rezolute Inc., SamChunDang Pharm. Co. Ltd., Samsung, Sandoz Biopharmaceuticals, Santen, SciFluor Life Sciences, Senju Pharmaceutical, Shanghai Henlius Biotech Inc., Stealth BioTherapeutics Inc., Taiwan Liposome Company Ltd., Thrombogenics, Xbrane, and Zeiss Meditec Inc.; holds a patent with OPTOS; and has stock options with Adverum, Clearside Biomedical Inc., and Ocuphire Pharma, Inc. EML reports receiving an institution grant from Annexon Biosciences, Apellis Pharmaceuticals, Iveric Bio, and Stealth Biotherapeutics; receiving consultancy fees from Annexon Biosciences, Apellis Pharmaceuticals, Applied Genetic Technologies Corporation, Iveric Bio, and Stealth Biotherapeutics; receiving financial support from Annexon Biosciences, Apellis Pharmaceuticals, and Iveric Bio; serving as a Steering Committee member of Annexon Biosciences and Iveric Bio; and serving as a Medical Advisory Board member of Applied Genetic Technologies Corporation. NS reports receiving consulting fees from AbbVie, Alimera, Allergan, Apellis, Bausch and Lomb, Biogen, Genentech, Novartis, Ocular Therapeutix, Opthea, Oyster Point Pharma, Regeneron, Regenxbio, Vortex Surgical, and Zeiss; receiving independent research support from Genentech, Novartis, Regeneron, Regenxbio, Zeiss, Ocular Therapeutix, and Opthea; serving on a speaker bureau for Alimera, Apellis, Bausch and Lomb, Biogen, Evolve Medical, Genentech, Novartis, Regeneron, and Vindico Medical; and holding stock and stock options in Vortex Surgery. RPS reports receiving personal fees from Apellis, Iveric Bio, Eye-point, Regenxbio, Genentech, Bausch and Lomb, Zeiss, Alcon, and Regeneron; and receiving research grants from Janssen. IP reports receiving consultancy fees from Apellis Pharmaceuticals, Bayer, Novartis, and Roche; and receiving financial support from Apellis Pharmaceuticals, Bayer, and Novartis. TA reports serving as a speaker for Apellis, Bayer, Heidelberg Engineering, Nidek, Novartis, and Roche; serving as an advisory board member for Apellis and Roche; and receiving a research grant from Nidek. RR reports being a former employee of Apellis Pharmaceuticals. CL reports being an employee of Apellis Pharmaceuticals, and holding stock or stock options in Apellis. DJ reports being an employee of Apellis Pharmaceuticals, and holding stock or stock options in Apellis. MT reports being an employee of Apellis Pharmaceuticals, and holding stock or stock options in Apellis. PJF reports being an employee of Apellis Pharmaceuticals. CRB reports being an employee of and Chief Medical Officer at Apellis Pharmaceuticals and owning stock in Apellis. CCW reports consulting fees from 4DMT, AbbVie, Adverum, Aerie, Alcon, Annexon, Apellis, Ascidian, Bausch + Lomb, Bayer, Boehringer Ingelheim, Cholgene, Clearside, Curacle, Eyebiotech, EyePoint, Genentech, Gyroscope, IVERIC Bio, Janssen, Kato, Kiora, Kodiak, Merck, Nanoscope, Neurotech, NGM, Novartis, Ocular Therapeutix, Ocuphire, ONL, Opthea, Oxular, Palatin, PerceiveBio, Ray, RecensMedical, Regeneron, RegenXBio, Roche, Sandoz, Sanofi, Stealth, THEA, and Valo; receiving research funds from 4DMT, AbbVie, Adverum, AffaMed, Alexion, Alimera, Annexin, Annexon, Apellis, Ascidian, Bayer, Boehringer Ingelheim, Chengdu Kanghong, Clearside, Curacle, Eyebiotech, EyePoint, Genentech, Gyroscope, IONIS, IVERIC Bio, Janssen, Kodiak, Nanoscope, Neurotech, NGM, Novartis, Ocular Therapeutix, Ocuphire, Opthea, Oxular, PerceiveBio, Regeneron, RegenXBio, Roche, Sandoz, Sanofi, Stealth, and Valo; and holding stock options in ONL, PolyPhotonix, RecensMedical, TissueGen, Visgenx, and Vitranu.

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