Pegcetacoplan injection is effective for managing geographic atrophy

(Image Credit: AdobeStock/Marco)

Ophthalmology Times® Staff Report

Apellis Pharmaceuticals Inc has announced data from post hoc analyses of its 24-month, phase 3 OAKS and DERBY studies evaluating pegcetacoplan injection (Syfovre) for the treatment of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), according to a company news release.¹

The data were reported during The Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, which was held in April in New Orleans, Louisiana.²

The pegcetacoplan injection showed visual function and quality-of-life benefits in patients with extrafoveal lesions (≥ 0.25 mm from the foveal center), according to the news release.¹ The pegcetacoplan injection showed a meaningful reduction in the loss of photoreceptor and retinal pigmented epithelial (RPE) cells. The analyses used data from SPECTRALIS optical coherence tomography (OCT) images, which allowed for artificial intelligence–based automated segmentation of the photoreceptor and RPE layers as well as determination of the amount of the central foveal region covered by the GA lesion (foveal occupancy).

According to the news release, in the 24-month analysis, patients receiving the pegcetacoplan injection demonstrated¹:

• preservation of 5.6 letters, equivalent to more than 1 line of vision on an Early Treatment Diabetic Retinopathy Study chart, as measured by best corrected visual acuity; and
• a 4.1-point benefit in vision-related quality-of-life outcomes, as measured by the National Eye Institute Visual Function Questionnaire-25. For results of the questionnaire, which assesses outcomes such as social function, driving, and dependency on others, 4 points is considered clinically meaningful.²

“Vision loss caused by GA can profoundly impact a person’s independence and well-being, so it is vital that pegcetacoplan injection has shown slower vision loss and better quality of life compared [with] sham in this post hoc analysis. These data also support earlier treatment with pegcetacoplan injection,” Allen Chiang, MD, presenting author and associate professor of ophthalmology at Wills Eye Physicians-Mid Atlantic Retina and Thomas Jefferson University in Philadelphia, Pennsylvania, said in the news release.¹

Because of sample size considerations, every-other-month and monthly data from OAKS (NCT03525600) and DERBY (NCT03525613) were combined for the pegcetacoplan injection (n = 131) and sham (n = 61) groups, the news release stated.¹ These data are in addition to the functional benefit outcomes previously reported in the post hoc junctional zone microperimetry analysis. Pegcetacoplan injection also slowed photoreceptor and RPE cell loss compared with sham, according to the study results.²

According to the news release, in the 24-month analysis of OAKS (n = 456) and DERBY (n = 435), pegcetacoplan injection demonstrated a meaningful reduction in the loss of both photoreceptor and RPE cells compared with sham (all P values nominal)¹:

Photoreceptor cells

• Every other month: 46% (OAKS; P <.0001) and 46% (DERBY; P < .0001)
• Monthly: 53% (OAKS; P <.0001) and 47% (DERBY; P < .0001)

RPE cells

• Every other month: 20% (OAKS; P = .0002) and 21% (DERBY; P = .0005)
• Monthly: 22% (OAKS; P = .0002) and 27% (DERBY; P < .0001)

According to the news release, “RPE cells maintain the integrity of photoreceptor cells, and both types of cells are required for vision. Data were consistent when comparing pegcetacoplan injection-treated study eyes with the untreated fellow eyes.”¹

“We are proud to share these data as part of our 8 presentations at this year’s ARVO meeting, which showcase our leadership in GA and retina,” Caroline Baumal, MD, chief medical officer at Apellis, said in the news release.¹ “Syfovre is a gamechanger for GA as the first and only treatment for this relentless disease, and we look forward to exploring its potential to treat other complement-driven retina diseases with significant unmet needs.”

According to the news release, “Marketing applications are currently under review with 5 regulatory agencies worldwide. A decision in the [European Union] is expected in early 2024, and decisions in Canada, Australia, Switzerland, and the United Kingdom are expected in the first half of 2024.”¹

References:

1. Apellis presents phase 3 functional analyses of Syfovre (pegcetacoplan injection) for geographic atrophy. News release. Apellis. April 23, 2023. Accessed May 8, 2023. https://investors.apellis.com/news-releases/news-release-details/apellis-presents-phase-3-functional-analyses-syfovretm

2. Chiang A, Sarda S, Burch M, Tsuboi M, Jones D, Ribeiro R. Assessment of geographic atrophy progression in the phase 3 OAKS and DERBY trials. Presented at The Association for Research in Vision and Ophthalmology Annual Meeting; April 23-27, 2023; New Orleans, Louisiana. https://investors.apellis.com/static-files/ff6347c3-cfa3-4cd7-b7ec-547f7a9d3d6c