Caroline Baumal, MD: Marking a New Treatment Era for GA with Pegcetacoplan


At ARVO 2023, Caroline Baumal, MD, chief medical officer of Apellis Pharmaceuticals, discussed new phase 3 functional analyses of pegcetacoplan injection, the future of the GA treatment landscape, and the next unmet need in ophthalmology.

The 2023 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans serves as a landmark meeting for ophthalmology research and an opportunity to hear from experts on the importance of new data.

In an interview with HCPLive at ARVO 2023, Caroline Baumal, MD, chief medical officer, Apellis Pharmaceuticals, discussed some of the biggest research news surrounding pegcetacoplan injection (SYFOVRE™). Approved by the US Food and Drug Administration (FDA) in February 2023, pegcetacoplan injection is the first and only approved therapy for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

The presented research included post-hoc analyses from the 24-month phase 3 OAKS AND DERBY studies. According to the analyses, pegcetacoplan showed visual function and quality-of-life benefits in patients with extrafoveal lesions (≥0.25 mm from the foveal enter), as well as a meaningful reduction in the loss of photoreceptor and retinal pigmented epithelial (RPE) cells.

Each analysis utilized data from optical coherence tomography (OCT) images, which allowed for artificial intelligence (AI)-based automated segmentation of the photoreceptor and RPE layers, as well as determination of the foveal occupancy.

Baumal dove into the presented research and shared these key highlights, including a preservation of 5.6 letters, equivalent to ≥1 line of vision on an ETDRS chart, as measured with best-corrected visual acuity (BCVA) with pegcetacoplan, as well as a 4.1-point benefit in vision-related quality-of-life outcomes measured by the NEI-VFQ-25.

As well, she shared data showed a 53% preservation of photoreceptors, and up to 27% preservation of RPE cells in pegcetacoplan-treated eyes compared to sham. Baumal noted that this is the first step of advancement, similar to the ones taken with anti-vascular endothelial growth factor (anti-VEGF) therapy, where a single step could further innovation.

"The next step that I see for pegcetacoplan is finding the maximal way for us to optimize this for our patient and further deep diving into the data to find the patients that will respond the best from therapy and really hone the patient experience to find ways to explain this to the patient so they understand the benefits of reducing lesion growth over time."

Baumal also spoke about finding the next steps forward with imaging and artificial intelligence, to better evaluate patients with early or intermediate AMD to understand who is at the greatest risk for developing GA progression. Lastly, she discussed innovative drug delivery systems that may reduce treatment burden, while also continuing excellent efficacy for patients with various retinal diseases.

Watch the above video for more perspective from Baumal and check out the rest of our ARVO 2023 coverage here.

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