Pegcetacoplan: 5-Year Data Shows GA Progression Slowing in AMD

Early and consistent treatment with pegcetacoplan appears to yield better long-term outcomes for individuals with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), according to data presented at the Macula Society meeting this week. The findings, discussed by Theodore Leng, MD, FACS, underscore the potential benefits of initiating and maintaining treatment as early as possible to preserve retinal tissue and slow the progression of this vision-threatening condition.

Geographic atrophy is an advanced form of dry AMD, characterized by the gradual loss of cells in the macula, the central part of the retina responsible for sharp, central vision. As GA progresses, it leads to irreversible vision loss. Currently, there are limited treatment options available for GA, making the recent data on pegcetacoplan particularly significant.

Understanding Pegcetacoplan and its Mechanism

Pegcetacoplan, developed by Apellis Pharmaceuticals, is a C3 complement inhibitor. The complement system is a part of the immune system that, when overactive, can contribute to inflammation and cell damage in the retina. By blocking the C3 protein, pegcetacoplan aims to reduce this harmful activity and slow the progression of GA. Previous research has demonstrated that pegcetacoplan can slow GA progression and the latest 5-year data builds upon these findings.

Five Years of Data: What the Findings Demonstrate

Dr. Leng, professor of ophthalmology and director of clinical and translational research at Byers Eye Institute, Stanford University, highlighted the 5-year results presented at the Macula Society meeting. The data suggest that earlier and continuous treatment with pegcetacoplan is associated with improved preservation of retinal tissue and a delayed progression of GA over the long term. The study, led by Dhoot D, et al., was presented February 25-28, 2026, in Coronado, California. Further details about the study are available through Healio.

Who is Affected by Geographic Atrophy?

Age-related macular degeneration is a leading cause of vision loss in people aged 50 and older. Although the “dry” form of AMD, which can lead to GA, is more common than the “wet” form, it has historically had fewer treatment options. The prevalence of AMD increases with age, and genetic predisposition as well plays a role. Individuals with a family history of AMD are at higher risk. The impact of GA is significant, as it can severely impair a person’s ability to read, drive, and recognize faces.

Study Details and Limitations

The data presented at the Macula Society meeting stemmed from an ongoing clinical trial evaluating pegcetacoplan in individuals with GA. While the specific details of the trial design, including sample size and endpoints, were not fully detailed in the initial report, the 5-year follow-up provides valuable long-term insights. It’s important to note that clinical trials have inherent limitations. Factors such as patient selection, adherence to treatment protocols, and the potential for confounding variables can influence the results. The study’s findings need to be interpreted in the context of these limitations.

What Does This Mean for Patients?

The findings reinforce the importance of early detection and intervention in AMD. Individuals at risk for AMD, particularly those with a family history or early signs of the disease, should undergo regular eye exams. If diagnosed with GA, discussing treatment options, including pegcetacoplan, with a qualified ophthalmologist is crucial. It’s important to understand that pegcetacoplan is not a cure for GA, but it may support to slow its progression and preserve vision for a longer period. The decision to initiate treatment should be made on an individual basis, considering the patient’s overall health, disease severity, and personal preferences.

The Role of the Complement System in AMD

The complement system, the target of pegcetacoplan, is a complex network of proteins that plays a critical role in the immune response. In AMD, dysregulation of the complement system can lead to chronic inflammation and damage to the retinal cells. Research has shown that genetic variations in complement-related genes are associated with an increased risk of AMD. This has led to the development of complement inhibitors, such as pegcetacoplan, as a potential therapeutic strategy.

What Comes Next: Ongoing Research and Future Directions

Further research is ongoing to evaluate the long-term efficacy and safety of pegcetacoplan in larger and more diverse populations. Researchers are also exploring other complement inhibitors and combination therapies to improve treatment outcomes for GA. The FDA is currently reviewing pegcetacoplan for approval, and a decision is expected in the coming months. Continued surveillance of patients treated with pegcetacoplan will be essential to monitor for any potential adverse effects and to assess the durability of the treatment response. The National Eye Institute (NEI) continues to fund research into the underlying causes of AMD and the development of new therapies. Learn more about AMD research at the NEI website.

The evolving understanding of AMD and the development of innovative treatments like pegcetacoplan offer hope for individuals at risk of vision loss. Early detection, consistent treatment, and ongoing research are key to preserving vision and improving the quality of life for those affected by this debilitating condition.

Sources/Disclosures: Healio Interviews. Dhoot D, et al. Earlier treatment yields better outcomes: 5 years of pegcetacoplan treatment for geographic atrophy secondary to AMD. Presented at: Macula Society meeting; Feb. 25-28, 2026; Coronado, California. Leng reports receiving grants from Astellas and Luxa Biotechnology and consulting for Apellis Pharmaceuticals, Astellas, Boehringer Ingelheim, Roche/Genentech, Toku, Topcon, Verana Health and Virtual Field.