Pegcetacoplan for the treatment of geographic atrophy due to age-related macular degeneration: a plain language summary of OAKS and DERBY clinical studies
- PMID: 39819346
- PMCID: PMC12269671
- DOI: 10.1080/1750743X.2025.2449760
Pegcetacoplan for the treatment of geographic atrophy due to age-related macular degeneration: a plain language summary of OAKS and DERBY clinical studies
Plain language summary
What is this summary about?This is a summary of the results of 2 clinical studies , called OAKS and DERBY, that tested a medicine called pegcetacoplan. Pegcetacoplan is the first United States Food and Drug Administration (US FDA) approved treatment for geographic atrophy, commonly referred to as GA, a common eye disease in older people. Geographic atrophy is a form of late age-related macular degeneration, or AMD, that usually starts after age 50. The US FDA approved pegcetacoplan based on the results from these 2 clinical studies. Pegcetacoplan is covered by Medicare and most insurance companies in the United States.What are the key takeaways?The results of the OAKS and DERBY clinical studies show that treatment with pegcetacoplan led to slower growth of GA lesions in the retina. The retina is the light-sensitive tissue at the back of the eye. In other words, it slowed the growth of the areas where cells were dying. When compared to a sham procedure, pegcetacoplan injection slowed the growth of lesions by 19% to 22% (when given every 4 weeks) and 16% to 18% (when given every 8 weeks) in OAKS and DERBY, respectively.After 2 years of treatment, there were no differences in the average vision function between participants treated with pegcetacoplan and participants who received a sham procedure.The most common eye-related health issues reported were like those reported with all types of intravitreal injections and most were temporary.What were the main conclusions reported by the researchers?Based on the results of these 2 large clinical studies, pegcetacoplan slows GA disease progression with an incidence of eye-related health issues similar to other drugs given by intravitreal injection.[Box: see text].
Conflict of interest statement
Jeffrey S Heier (ORCID ID 0000-0003-4625-3145) has received grants paid to their institution from Annexon Biosciences, Apellis Pharmaceuticals, AsclepiX Therapeutics, Bayer, Genentech/Roche, Gyroscope Therapeutics, Iveric Bio, jCyte, Kodiak Sciences, NGM Biopharmaceuticals, Notal Vision, Regeneron Pharmaceuticals, and Regenxbio; and consultancy fees from 4DMT, Adverum Biotechnologies, AffaMed, Allegro Ophthalmics, Allergan, Annexon Biosciences,Apellis Pharmaceuticals, AsclepiX Therapeutics, Genentech/Roche, Gyroscope Therapeutics, Iveric, Janssen R & D, Chengdu Kanghong Pharmaceutical Group, NGM Biopharmaceuticals, Novartis, OcuTerra, Regenxbio, Regeneron Pharmaceuticals, RevOpsis, Stealth BioTherapeutics, and Thea Laboratories. Eleonora M Lad (ORCID ID 0000-0001-8435-521X) has received grants paid to their institution from Boehringer Ingelheim; research funding from Alexion Pharmaceuticals, Apellis Pharmaceuticals, Astellas Pharma, Boehringer Ingelheim, Gemini Therapeutics, Janssen, LumiThera, Neurotech, Novartis, and Roche; and consultancy fees from Alexion Pharmaceuticals, Allegro Ophthalmics, Annexon Biosciences, Apellis Pharmaceuticals, EdiGeneBio, Galimedix Therapeutics, Gemini Therapeutics, Iveric Bio,Janssen, LumiThera, Nanoscope Therapeutics, NGM Biopharmaceuticals, Novartis, Perceive Biotherapeutics, Retrotope, Roche, and Thea Laboratories; holds a provisional patent (A System and Method to Predict Progression of Age-Related Macular Degeneration; US patent application number 63162741); holds stock options with Retrotope; and is the recipient of medical writing services from Allegro Ophthalmics, Apellis Pharmaceuticals, and Boehringer Ingelheim. Frank G Holz (ORCID ID 0000-0001-8019-9272) has received grants paid to their institution and consultancy fees from Apellis Pharmaceuticals, Iveric Bio, Genentech/Roche, Pixium Vision, and Heidelberg Engineering; financial support from Apellis Pharmaceuticals, Genentech/Roche, and Heidelberg Engineering; is an executive board member of EURETINA and the German Ophthalmological Society; and is a scientific board member of Pro Retina. Philip J Rosenfeld (ORCID ID 0000-0002-4068-6671) has stock options in Apellis Pharmaceuticals. Robyn H Guymer (ORCID ID 0000-0002-9441-4356) has received consultancy fees from Apellis Pharmaceuticals, Bayer,Genentech/Roche, and Novartis; and financial support from Apellis Pharmaceuticals and Genentech/Roche. David Boyer (ORCID ID 0000-0001-9219-1614) has received consultancy fees from Alexion Pharmaceuticals, Annexon Biosciences, Apellis Pharmaceuticals, Boehringer Ingelheim, Cell Cure Neurosciences, Gemini Therapeutics, Genentech, Gyroscope Therapeutics, Ionis Pharmaceuticals, Iveric Bio, and Stealth BioTherapeutics; holds stock in Digisight and Ora; and is an equity owner in Apellis Pharmaceuticals. Federico Grossi (ORCID ID 0000-0002-7290-9008) is a patent inventor (US patent application number 15/766,795 [Dosing Regimens]and Patent Cooperation Treaty [PCT] application number PCT/US2022/043127 [Treatment of Geographic Atrophy]; the patent rights are assigned to Apellis Pharmaceuticals); is an equity owner in Apellis Pharmaceuticals; and is an employee of Apellis Pharmaceuticals. Caroline R Baumal (ORCID ID 0000-0002-3651-8210)has received financial support from Apellis Pharmaceuticals; and is the Chief Medical Officer of Apellis Pharmaceuticals. Jean-Francois Korobelnik (ORCID ID 0000-0002-4438-9535) has received consultancy fees from Apellis Pharmaceuticals, Bayer, Janssen, Nano Retina, Roche, Thea Laboratories, and Carl Zeiss Meditec; and is a member of a data safety monitoring board for Alexion Pharmaceuticals and Novo Nordisk. Jason S Slakter (ORCID ID 0000-0003-4636-2851) has received a grant paid to their institution, consultancy fees, and financial support from Apellis Pharmaceuticals; and has stock in Apellis Pharmaceuticals. Nadia K Waheed (ORCID ID 0000-0002-8229-7519) has received a grant paid to their institution from Nidek, Topcon, and Zeiss; consulting fees from Complement Therapeutics, Hubble Therapeutics, Iolyx Therapeutics, OliX Pharmaceuticals, SalioGen Therapeutics, Syncona, and Topcon; financial support from Nidek; is an officeholder at AGTC; and has an equity interest in Gyroscope Therapeutics and OcuDyne. Ravi Metlapally (ORCID ID 0000-0002-5544-1984) is an employee of Apellis Pharmaceuticals. Ian Pearce (ORCID ID 0000-0003-3115-1859) has received consultancy fees from Apellis Pharmaceuticals, Bayer, Novartis, and Roche; and financial support from Apellis Pharmaceuticals, Bayer, and Novartis. Nathan Steinle (ORCID ID 0000-0002-9323-6594) has received consultancy fees from Apellis Pharmaceuticals, has received speaking honoraria from Apellis Pharmaceuticals, and has participated on a data safety monitoring board and advisory board for Apellis Pharmaceuticals. Anibal A Francone (ORCID ID 0000-0001-5727-7383) has received a grant paid to their institution from Novartis and Roche; financial support from Allergan, Novartis, and Roche; holds a patent for Probe for Identification of Ocular Tissues During Surgery (University of California Los Angeles reference number 2020-851-3 PCT); and holds stock in Apellis Pharmaceuticals and Iveric Bio. Allen Hu (ORCID ID 0009-0005-1304-9975) has received a grant paid to their institution and consultancy fees from Apellis Pharmaceuticals; and has stock in Apellis Pharmaceuticals. David R Lally (ORCID ID 0000-0002-4479-8216) has received a grant paid to their institution from Annexon Biosciences, Apellis Pharmaceuticals, Iveric Bio, and Stealth BioTherapeutics; consultancy fees from Annexon Biosciences, Apellis Pharmaceuticals, Applied Genetic Technologies Corporation, Iveric Bio, and Stealth BioTherapeutics; financial support from Annexon Biosciences, Apellis Pharmaceuticals, and Iveric Bio; is a steering committee member of Annexon Biosciences and Iveric Bio; and is a medical advisory board member of Applied Genetic Technologies Corporation. Pascal Deschatelets (ORCID ID 0009-0001-0123-5528) is an employee of Apellis Pharmaceuticals. Pascal Deschatelets and Cedric Francois are inventors of several patents and authors of several patent applications covering pegcetacoplan or relating to methods of treating ophthalmologic indications (US patent numbers 8,168,584 [Methods of Treating Age-related Macular Degeneration by Compstatin and Analogs Thereof], 10,125,171 [Cell-reactive, Long-acting, or Targeted Compstatin Analogs and Uses Thereof], 10,035,822 [Cell-reactive, Long-acting, or Targeted Compstatin Analogs and Related Compositions and Methods]; US patent application number 15/766,795 [Dosing Regimens]; and PCT application number PCT/US2022/043127 [Treatment of Geographic Atrophy]; the patent rights are assigned to Apellis Pharmaceuticals). Cedric Francois (ORCID ID 0009-0005-4302-5739) is an equity owner in Apellis Pharmaceuticals, and is an employee of Apellis Pharmaceuticals.Caleb Bliss (ORCID ID 0009-0005-5516-7937) has stock in Apellis Pharmaceuticals, and is an employee of Apellis Pharmaceuticals. Giovanni Staurenghi (ORCID ID 0000-0002-2299-5251) has received grants for clinical trials from Apellis Pharmaceuticals, Genentech, and Iveric Bio; financial support from Apellis Pharmaceuticals;and has participated on a data safety monitoring board and advisory board for Apellis Pharmaceuticals, Bayer, Boehringer Ingelheim, Genentech, Iveric Bio, Novartis,and Roche. Jordi Monés (ORCID ID 0000-0003-3685-2160) has been an investigator for Apellis Pharmaceuticals, Genentech, Ionis Pharmaceuticals, Iveric Bio, Kodiak Sciences, Novartis, ReNeuron, and Roche; has received consultancy fees from Annexon Biosciences, Apellis Pharmaceuticals, Cell Cure Neurosciences, EyeBio,Genentech, Iveric Bio, Lineage Cell Therapeutics, Maculogix, Novartis, and ReNeuron; financial support from Apellis Pharmaceuticals, Cell Cure Neurosciences,Genentech, Iveric Bio, Lineage Cell Therapeutics, Novartis, ReNeuron, and Roche; has participated on an advisory board for Apellis Pharmaceuticals, Cell Cure Neurosciences, Genentech, Iveric Bio, Kodiak Sciences, Lineage Cell Therapeutics, Novartis, ReNeuron, and Roche; and holds stock in Notal Vision. Rishi P Singh (ORCID ID 0000-0001-5859-8162) has received consultancy fees from Alcon, Apellis Pharmaceuticals, AsclepiX Therapeutics, Bausch and Lomb, Genentech, Gyroscope Therapeutics, Novartis, and Regeneron Pharmaceuticals; financial support from Genentech, Novartis, and Regeneron Pharmaceuticals; is a member of the board of directors for the American Society of Retina Specialists; and is an executive board member of the Retina World Congress. Ramiro Ribeiro (ORCID ID 0000-0003-1429-7354) is an equity owner in Apellis Pharmaceuticals, and an employee of Apellis Pharmaceuticals. Charles C Wykoff (ORCID ID 0000-0001-7756-5091)has received a grant paid to their institution from 4DMT, Adverum Biotechnologies, Aerie Pharmaceuticals, AffaMed, Aldeyra Therapeutics, Alexion Pharmaceuticals,Alimera Sciences, Alkahest, Allergan, Allgenesis, Amgen, Annexon Biosciences, Apellis Pharmaceuticals, AsclepiX Therapeutics, Bayer, Boehringer Ingelheim,Chengdu Kanghong Pharmaceutical Group, Clearside Biomedical, Curacle, EyePoint Pharmaceuticals, Gemini Therapeutics, Genentech, Graybug Vision, Gyroscope Therapeutics, Ionis Pharmaceuticals, iRenix, Iveric Bio, Kodiak Sciences, The Lowy Medical Research Institute, Nanoscope Therapeutics, Neurotech, NGM Biopharmaceuticals, Novartis, Ocular Therapeutix, Ocuphire Pharma, OcuTerra, Opthea, Oxular, Oxurion, Oyster Point Pharma, Recens Medical, Regeneron Pharmaceuticals, Regenxbio, Roche, SamChunDang Pharm, Sandoz, Taiwan Liposome, Unity Biotechnology, Verily, and Xbrane Biopharma; consultancy fees from 4DMT, AbbVie, Adverum Biotechnologies, Aerie Pharmaceuticals, AGTC, Alcon, Alimera Sciences, Allergan, Allgenesis, Alnylam Pharmaceuticals, Annexon Biosciences, Apellis Pharmaceuticals, Arrowhead Pharmaceuticals, Bausch and Lomb, Bayer, Bionic Vision Technologies, Boehringer Ingelheim, Chengdu Kanghong Pharmaceutical Group, Cholgene Therapeutics, Clearside Biomedical, Curacle, EyePoint Pharmaceuticals, Foresite, Frontera Therapeutics, Genentech, Gyroscope Therapeutics, IACTA Pharmaceuticals, Iveric Bio, Janssen, Kato Pharmaceuticals, Kiora Pharmaceuticals, Kodiak Sciences, Kriya Therapeutics, Merck, Nanoscope Therapeutics, NGM Biopharmaceuticals, Notal Vision, Novartis, OccuRx, Ocular Therapeutix, OcuTerra, OliX Pharmaceuticals, ONL Therapeutics, Opthea, Palatin Technologies, Perceive Biotherapeutics, Perfuse Therapeutics, PolyPhotonix, Ray Therapeutics, Recens Medical, Regeneron Pharmaceuticals, Regenxbio, Resonance Pharma, Roche, SciNeuro Pharmaceuticals, Stealth BioTherapeutics, Surrozen, Thea Laboratories, TissueGen, Valo Therapeutics, Verana Health, and Vitranu; has participated on a data safety monitoring board and advisory board for Aerie Pharmaceuticals and Kato Pharmaceuticals; is a board member of the American Society of Retina Specialists and the Vit-Buckle Society; and holds stock options in ONL Therapeutics, PolyPhotonix, Recens Medical, TissueGen, Visgenx, and Vitranu.The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Professional medical writing and editorial assistance in the development of this summary were provided by OPEN Health and were funded by Apellis Pharmaceuticals. Research and manuscript support were provided by Apellis Pharmaceuticals.
Patient reviewers on this PLSP have received honorarium from
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