ASRS 2026: AI-matched analysis finds pegcetacoplan slows visual acuity decline in GA

A retrospective analysis using the Vestrum database found that patients with geographic atrophy (GA) treated with pegcetacoplan (Syfovre; Apellis Pharmaceuticals) were more likely to maintain visual acuity or show slower visual acuity decline than untreated patients, according to Nimesh Patel, MD, of Mass Eye and Ear and Boston Children's Hospital, Boston.

Study design

Patel and colleagues used the Vestrum database, a real-world data set drawn from multiple retina practices, applying AI-based analysis of optical coherence tomography (OCT) images to match patients on GA lesion size, distance to the fovea, and subfoveal involvement. The AI-assisted matching created 2 comparison cohorts: patients with GA alone who received pegcetacoplan versus untreated patients and patients with GA and concurrent neovascular age-related macular degeneration (AMD) who received pegcetacoplan plus anti-VEGF therapy versus anti-VEGF therapy alone. Patel said the AI-based approach made it feasible to balance large real-world cohorts for confounding factors in a fraction of the time manual analysis would require.

Key findings

A greater proportion of patients in the pegcetacoplan groups had better visual acuity or slower visual acuity decline compared with the untreated or anti-VEGF-alone groups, an effect most pronounced in the combination-therapy cohort. When the analysis focused on patients who lost 15 letters or more, the difference reached statistical significance at 6, 12, and 18 months in both the GA-alone and GA-with-neovascular-AMD groups. Patel described the findings as the first to demonstrate a visual acuity benefit with treatment in this population.

Clinical implications

Patel noted that pegcetacoplan's initial clinical trials focused on GA without neovascular disease, but a companion practice-pattern analysis presented at last year's meeting found that roughly 25% of real-world pegcetacoplan use occurred in patients with concurrent neovascular AMD—use outside the drug's original trial population. According to Patel, the new visual acuity data suggest the treatment may benefit this combination population as much as, or more than, patients with GA alone. Patel also said patients requiring both pegcetacoplan and anti-VEGF therapy typically need less frequent anti-VEGF dosing, since these eyes have often transitioned to a more atrophic, less active disease phase—limiting the added injection burden of combination treatment.

Patel said earlier identification and treatment of GA, even in patients without central involvement or noticeable vision loss, is likely to become increasingly important, and encouraged comprehensive ophthalmologists and optometrists to refer patients with GA for evaluation.