Intravitreal Pegcetaoplan May Control Lesion Growth in Geographic Atrophy

Amd screening
Vision, grille d’Amsler.
An AAO 2021 presentation reviewed clinical trial data of intravitreal pegcetacoplan, a targeted C3 inhibitor, in controlling lesion growth in patients with geographic atrophy.

This article is part of Ophthalmology Advisor’s Focus on Retina coverage from the 2021 meeting of the American Academy of Ophthalmology, held in New Orleans from November 12 to 15, 2021. The team at Ophthalmology Advisor will be reporting on a variety of the research presented by the retinal experts at the AAO. Check back for more from the AAO 2021 Meeting.


Patients with geographic atrophy secondary to age-related macular degeneration (AMD) may potentially benefit from monthly or every-other-month intravitreal pegcetacoplan, an investigational targeted C3 inhibitor, to control lesion growth, according to a paper presented at the American Academy of Ophthalmology 2021 Annual Meeting, held November 12-15, 2021 in New Orleans.

Lesion growth is irreversible in geographic atrophy. Researchers believe pegcetacoplan, an investigational therapy designed to target the complement overactivation that generates geographic atrophy progression, can prevent lesion growth and reduce the likelihood of severe disease.

The objective of the research was to evaluate the efficacy and safety of intravitreal pegacetacoplan in geographic atrophy from the DERBY ( Identifier: NCT03525613) and OAKS ( Identifier: NCT03525600) 24-month phase 3 clinical trials.

DERBY and OAKS were 2 randomized, double-masked, sham-controlled clinical trials that compared the efficacy and safety of monthly or every-other-month intravitreal pegacetacoplan to sham in this patient population. Enrollment was completed for DERBY in June 2020 and in July 2020 for OAKS. The trials included patients (DERBY, N=621; OAKS, N=638) who were at least 60 years old, had best corrected visual acuity 24 letters or better, and geographic atrophy area between 2.5 and 17.5 mm² or 1 focal point lesion 1.25 mm² if multifocal geographic atrophy at baseline. 

A change in lesion size in geographic atrophy through fundus autofluorescence from baseline to month 12 was the primary endpoint for both trials. Secondary endpoints included change in visual function from baseline. Safety was measured based on the number of incidences of ocular and systemic adverse events.

The researchers shared their findings of 12-month efficacy and safety data in both Phase 3 studies at the American Academy of Ophthalmology 2021 Annual Meeting on Monday, November 15. 

“Pegcetacoplan is the only targeted C3 inhibitor being evaluated in patients to control lesion growth in GA [geographic trophy],” presenters reported. 

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Singh RP, Steinle NC, Boyer DS, et al. Efficacy and safety of intravitreal pegcetacoplan in GA: results from the phase 3 DERBY and OAKS trials. Paper presented at: The American Academy of Ophthalmology 2021 Annual Meeting; November 12-15, 2021; New Orleans. Abstract PA064.