June 2022
Volume 63, Issue 7
Open Access
ARVO Annual Meeting Abstract  |   June 2022
Efficacy of intravitreal pegcetacoplan in patients with geographic atrophy (GA): 12-month results from the phase 3 OAKS and DERBY studies.
Author Affiliations & Notes
  • Roger Goldberg
    Bay Area Retina Associates, San Francisco, California, United States
  • Jeffrey S Heier
    OCB, Boston, Massachusetts, United States
  • Charles Clifton Wykoff
    Retina Consultants of Texas, Houston, Texas, United States
  • Giovanni Staurenghi
    Ospedale Luigi Sacco-Polo Universitario, Milano, Lombardia, Italy
  • Rishi P Singh
    Cleveland Clinic Cole Eye Institute, Cleveland, Ohio, United States
  • Nathan Steinle
    California Retina Consultants, California, United States
  • David S Boyer
    Retina-Vitreous Associates Medical Group, Los Angeles, California, United States
  • Jordi Mones
    Institut de la Màcula Centro Médico Teknon, Barcelona, Spain
  • Frank G Holz
    Rheinische Friedrich-Wilhelms-Universitat Bonn, Bonn, Nordrhein-Westfalen, Germany
  • Caleb Bliss
    Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
  • Federico Grossi
    Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
  • Ravi Metlapally
    Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
  • Ramiro Ribeiro
    Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Roger Goldberg Apellis, Code C (Consultant/Contractor); Jeffrey Heier 4DMT, Abpro, Adverum, Aerie, Affamed, Allegro, Allergan, Allgenesis, Annexon, Apellis, Aprea, Asclepix, Aviceda, BVT, DTx, Eloxx, Galimedix, Genentech/Roche, Graybug, Gyroscope, Horizon Therapeutics, Iveric, Kanghong, Lensgen, NGM, Novartis, Ocular Therapeutix, Oriole, Oxurion, Palatin, Regeneron, REGENXBIO, Roche, Santen, Scifluor, Stealth Biotherapeutics, Surrozen, Thea, Verseon, Vinci, Code C (Consultant/Contractor), Apellis, Code F (Financial Support); Charles Wykoff Alcon Laboratories, Allergan, Alimera Sciences, Alnylam Pharmaceuticals, Bayer, Clearside Biomedical, Dutch Ophthalmic Research Center International, Genentech, ONL Therapeutics; Regeneron, ThromboGenics, Valeant, Code C (Consultant/Contractor), Alcon Laboratories, Allegro Ophthalmics, Allergan, Apellis, Alimera Sciences, Alnylam Pharmaceuticals, Bayer, Clearside Biomedical, Diabetic Retinopathy Clinical Research Network, Dutch Ophthalmic Research Center International, Genentech, Iconic Therapeutics, ONL Therapeutics, Ophthotech Corporation, Regeneron, ThromboGenics, Tyrogenex, Valeant, Code F (Financial Support); Giovanni Staurenghi Allergan, Apellis, Bayer, Boehringer Ingelheim, Centervue, Genentech, Heidelberg Engineering, Iveric, Novartis, Roche, Code C (Consultant/Contractor), Alcon, Allergan, Apellis, Bayer, Boehringer Ingelheim, Centervue, Genentech, Heidelberg Engineering, Ocular Instruments, Optos, Optovue, Carl Zeiss Meditec, Novartis, Quantel Medical, Roche, Code F (Financial Support); Rishi Singh Aerie, Alcon, Apellis, Bausch and Lomb, Genentech, Graybug, Novartis, Regeneron Pharmaceuticals, Zeiss, Code C (Consultant/Contractor); Nathan Steinle Alimera Sciences, Apellis, Genentech, Notal Vision, Novartis, Regeneron, Regenerative Patch Technologies, REGENXBIO, Vortex Surgical, Zeiss., Code C (Consultant/Contractor), Vortex Surgical, Code O (Owner); David Boyer Acucela, Aerie Pharmaceuticals, Alcon, Alimera Sciences, Allergan, Apellis, Bausch and Lomb, Bayer, Biomotiv, BioTime, Boehringer Ingelheim, Cell Care Therapeutics, Chengdu Kanghong Biotechnology, Clearside, Coda Therapeutics, D4T, Foresight Biotherapeutics, Genentech, Gensight Biologics, Glaukos, Graybug, GSK, InFlammX, Ionis Pharmaceuticals, Isarna Therapeutics, IVERIC Bio Lumithera, Merck, Notal Vision, Novartis, Ocular Therapeutics, Ohr, Ophthotec, Optos, Optovue, Oxurion, Psivida Cor, Regeneron, REGENXBIO, Regulus Therapeutics, Roche, Santen, Scifluor Life Sciences, Shire, Stealth Biotherapeutics, Sun Pharmaceutical Industries, Taiwan Liposomal Company., Code C (Consultant/Contractor), InFLammX, Code E (Employment), Ohr, Allegro, Code O (Owner); Jordi Mones Allegro, Apellis, Bayer, Iveric Bio, Kodiak, Novartis, Reneuron, Roche, Thrombogenics; and holds stock in Iveric Bio, Notal Vision, Code C (Consultant/Contractor), Apellis, Bayer, Gyroscope, Iveric Bio, Novartis, Reneuron, Roche, Thrombogenics, Code F (Financial Support); Frank Holz Acucela, Allergan, Apellis, Bayer, Boehringer Ingelheim, Bioeq/Formycon, CenterVue, Ellex, Geuder, Grayburg Vision, Heidelberg Engineering, Kanghong, LinBioscience, NightStarX, Novartis, Optos, Pixium Vision, Oxurion, Roche/Genentech, Stealth BioTherapeutic, Zeiss., Code C (Consultant/Contractor), Acucela, Allergan, Apellis, Bayer, Boehringer Ingelheim, Bioeq/Formycon, CenterVue, Ellex, Geuder, Grayburg Vision, Heidelberg Engineering, Kanghong, LinBioscience, NightStarX, Novartis, Optos, Pixium Vision, Oxurion, Roche/Genentech, Stealth BioTherapeutic, Zeiss., Code F (Financial Support); Caleb Bliss Apellis , Code E (Employment); Federico Grossi Apellis, Code E (Employment), Apellis, Code O (Owner), Apellis 15/766,795., Code P (Patent); Ravi Metlapally Apellis, Code E (Employment); Ramiro Ribeiro Apellis, Code E (Employment), Apellis, Code O (Owner)
  • Footnotes
    Support  Apellis Pharmaceuticals
Investigative Ophthalmology & Visual Science June 2022, Vol.63, 1500. doi:
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      Roger Goldberg, Jeffrey S Heier, Charles Clifton Wykoff, Giovanni Staurenghi, Rishi P Singh, Nathan Steinle, David S Boyer, Jordi Mones, Frank G Holz, Caleb Bliss, Federico Grossi, Ravi Metlapally, Ramiro Ribeiro; Efficacy of intravitreal pegcetacoplan in patients with geographic atrophy (GA): 12-month results from the phase 3 OAKS and DERBY studies.. Invest. Ophthalmol. Vis. Sci. 2022;63(7):1500.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To investigate the efficacy of monthly or every-other-month (EOM) intravitreal pegcetacoplan compared with sham in patients with GA secondary to age-related macular degeneration.

Methods : OAKS (NCT03525613) and DERBY (NCT03525600) are phase 3, randomized, double-masked, sham-controlled studies. Enrolled patients were ≥60 years old, with best-corrected visual acuity ≥24 letters, and GA area between 2.5 and 17.5 mm2, including foveal and extrafoveal lesions. The primary endpoint for both studies was change in GA lesion size from baseline to Month 12 measured by fundus autofluorescence. Safety measures included incidences of ocular and systemic adverse events.

Results : OAKS and DERBY enrolled 637 and 621 patients, respectively. OAKS showed statistically significant reductions in GA lesion growth vs sham in the monthly and EOM arms by 22% (p=0.0003) and 16% (p=0.0052), respectively. The results of DERBY did not reach statistical significance: pegcetacoplan decreased GA lesion growth vs sham by 12% (p=0.0528) and 11% (p=0.0750) in the monthly and EOM arms, respectively. In a prespecified pooled analysis, reductions were 17% (p<0.0001; nominal) and 14% (p=0.0012; nominal) in monthly and EOM arms vs sham. In a prespecified analysis of extrafoveal GA lesions, growth was reduced by 35% (p<0.0001; nominal) and 21% (p=0.0159; nominal) in OAKS and by 16% (p=0.0712; nominal) and 25% (p=0.0028; nominal) in DERBY in the monthly and EOM arms, respectively, compared with sham. In a prespecified analysis of foveal GA lesions, growth was reduced by 16% (p=0.0324; nominal) and 16% (p=0.0179; nominal) in OAKS and by 7% (p=0.3611; nominal) and increased by 1% (p=0.8993; nominal) in DERBY in the monthly and EOM arms, respectively, compared with sham.

Conclusions : Monthly and EOM pegcetacoplan met the primary endpoint in OAKS, with positive trends also observed in DERBY. Pegcetacoplan demonstrated greater efficacy in patients with extrafoveal lesions at baseline. Taken together with the positive results from the well-controlled phase 2 FILLY study, these findings support the efficacy of pegcetacoplan in slowing the progression of GA lesions.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.

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