Research Update

Phase 3 Study Enrollment Completed to Investigate Geographic Atrophy Treatment

Enrollment to the DERBY and OAKS phase 3 studies which will examine the safety and efficacy of intravitreal pegcetacoplan to treat Geographic Atrophy secondary to Age-related Macular Degeneration (AMD) has been completed1. 1259 patients have been recruited for these studies which will assess the drug designed to target and inhibit the complement cascade, a component of the body’s immune system. When not tightly regulated, the complement cascade can lead to the progression of many serious diseases, and has been proposed as a contributing factor in the onset of AMD2.

The DERBY and OAKS phase 3 trials follow the completion of the phase 2 FILLY study. In this study, significant reductions in the growth of Geographic Atrophy lesion area; a pigmented region of the retina which can reduce the visual field, were recorded from baseline to 12 months in patients receiving injections monthly or every other month, compared to sham. The patient group receiving monthly treatment showed a 29% reduction (p<0.008) in GA lesion size compared to sham injections, while the patient group receiving injections every other month had a 20% reduction (p<0.067; statistical significance of p<0.1 in this study).

The primary outcome measure for the DERBY and OAKS studies will be the reduction in growth of Geographic Atrophy lesion size recorded at month 12 and compared to baseline, measured by fundus autofluorescence. Top-line results are expected from these studies in the third quarter of 2021.

According to Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis, “Completing enrollment in the Phase 3 DERBY and OAKS studies is a critical milestone that moves us one step closer to bringing the first treatment for Geographic Atrophy to patients.”

In July 2018, the U.S. Food and Drug Administration (FDA) granted pegcetacoplan Fast Track Designation for the treatment of Geographic Atrophy.


1. Apellis. Apellis Completes Enrollment in Two Phase 3 Studies of the Targeted C3 Therapy, Pegcetacoplan, in Patients with Geographic Atrophy (GA). Available Accessed July 2020.

2. Murphy, K. and Weaver, C., 2016. Janeway’s immunobiology. Garland science.

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