Analysis Suggests Consistent Efficacy of Pegcetacoplan on GA Lesion Growth

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At ARVO 2023, 24-month data suggests the change from baseline in GA lesion growth consistently favored pegcetacoplan vs. sham across subgroups in DERBY and OAKS trials.

A 24-month post-hoc analysis of the phase 3 OAKS and DERBY trials demonstrated the consistent efficacy of pegcetacoplan (SYFOVRE) across baseline lesion subgroups in patients with geographic atrophy (GA).1

The analysis, presented at the 2023 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans, Louisiana, showed the slowest progressing GA quartile included a higher proportion of patients treated with pegcetacoplan than sham.

“Vision loss caused by GA can profoundly impact a person’s independence and well-being, so it is vital that SYFOVRE has shown slower vision loss and better quality of life compared to sham in this post-hoc analysis,” said Allen Chiang, MD, presenting author and associate professor of ophthalmology, Willis Eye Hospital Retina Service.

Chiang and colleagues assessed the efficacy of pegcetacoplan in patient subgroups based on baseline lesion characteristics and to characterize overall trial results based on treatment response quartiles. The randomized, double-masked, sham-controlled, phase 3 OAKS (n = 637) and DERBY (n = 621) enrolled patients with best-corrected visual acuity (BCVA) ≥21 letters and GA area of 2.5 - 17.5 mm2. Patients with both nonsubfoveal and subfoveal lesions were included in the analysis.

Primary endpoints were the change in total area of GA lesions measured by fundus autofluorescence and the efficacy of pegcetacoplan at 24 months was assessed in patient subgroups based on baseline demographics and GA lesion characteristics. Investigators pooled patients across trials and analyzed subgroups based on age, sex, GA lesion location, focality and laterality, and BCVA. A quartile analysis based on GA lesion growth (mm2) over 24 months allowed further characterization of the efficacy of pegcetacoplan.

Patients were pooled across both trial and trial arms, with Quartile 1 including slow progressors and Quartile 4 including fast progressors. Only those with 24-month growth measurements were included in the final analysis. The analysis revealed GA lesion growth across subgroups in the sham arm was consistent with natural history studies at 24 months.

Data showed the change from baseline in GA lesion growth at month 24 consistently favored pegcetacoplan across subgroups, including those with nonsubfoveal (monthly, 26% [P <.0001]; every-other-month, 22% [P <.0001]) and subfoveal lesions (monthly, 19% [P <.0001]; every-other-month, 16% [P =.0003).

Additionally, the change from baseline in GA lesion growth favored pegcetacoplan in those with unifocal (monthly, 26% [P <.0001]; every-other-month, 21% [P <.0007]) and multifocal lesions (monthly, 20% [P <.0001]; every-other-month, 17% [P <.0001]).

A quartile analysis revealed quartile 1 (slow progressors) had a higher proportion of patients from the pegcetacoplan monthly and every-other-months arms versus sham. In contrast, quartile 4 (fast progressors) had a higher proportion of sham patients than pegcetacoplan monthly or every other month dosing intervals.


  1. Chiang A. Bliss C. Ribeiro R. Assessment of geographic atrophy (GA) lesion progression in the phase 3 OAKS and DERBY trials. Presented at the 2023 Association for Research in Vision and Ophthalmology Annual Meeting; April 2023; New Orleans, LA.
  2. Apellis presents phase 3 functional analyses of SYFOVRE™ (Pegcetacoplan Injection) for geographic atrophy. Apellis Pharmaceuticals, Inc. Published April 23, 2023. Accessed April 28, 2023.