Longer-term data from the DERBY and OAKS phase 3 studies showed that pegcetacoplan, an intravitreal targeted C3 therapy, continued to reduce geographic atrophy (GA) lesion growth in patients with GA secondary to age-related macular degeneration (AMD), and demonstrated a favorable safety profile at month 18.

DERBY (N=621) and OAKS (N=637) are phase 3, multicenter, randomized, double-masked, sham-controlled studies evaluating the efficacy and safety of intravitreal pegcetacoplan vs sham injections. The primary endpoint for both studies is the change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 12 months. The trials also include an extension phase, where patients continue to receive masked treatment for up to 24 months.

Results from a longer-term analysis of the DERBY study showed that both monthly (13%; P =.0254) and every-other-month (12%; P =.0332) treatment with pegcetacoplan continued to reduce GA lesion growth compared with pooled sham at month 18. Additionally, marked improvements were observed in months 6 to12 for both monthly (17% reduction) and every-other-month (16% reduction) treatment compared with months 0 to 6. Treatment effects were sustained through month 18.

Similar results were reported in the OAKS study, which showed that both monthly (22%; P <.0001) and every-other-month (16%; P =.0018) treatments with pegcetacoplan continued to reduce GA lesion growth compared with pooled sham at month 18.

Pegcetacoplan continued to demonstrate a favorable safety profile consistent with long-term intravitreal injection exposure. The rates of infectious endophthalmitis and intraocular inflammation per injection were 0.044% and 0.23%, respectively.

The Company plans to include data from both studies for the New Drug Application (NDA) submission planned in the second quarter of 2022.

“It is exciting to see these positive data with pegcetacoplan, which showed continuous and potentially improving effects over time,” said Jeffrey S. Heier, MD, principal investigator of the DERBY study and Director of the Vitreoretinal Service and Director of Retina Research at Ophthalmic Consultants of Boston. “These 18-month results provide further evidence that pegcetacoplan meaningfully slows disease progression and has the potential to preserve vision longer.”


Apellis announces pegcetacoplan showed continuous and clinically meaningful effects at month 18 in phase 3 DERBY and OAKS studies for geographic atrophy (GA). News release. Accessed March 16, 2022. https://www.globenewswire.com/news-release/2022/03/16/2404200/0/en/Apellis-Announces-Pegcetacoplan-Showed-Continuous-and-Clinically-Meaningful-Effects-at-Month-18-in-Phase-3-DERBY-and-OAKS-Studies-for-Geographic-Atrophy-GA.html