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Clinical Trial
. 2023 Dec;37(17):3551-3557.
doi: 10.1038/s41433-023-02497-w. Epub 2023 Mar 24.

Avacincaptad pegol for geographic atrophy secondary to age-related macular degeneration: 18-month findings from the GATHER1 trial

Sunil S Patel  1 , David R Lally  2 , Jason Hsu  3 , Charles C Wykoff  4 , David Eichenbaum  5   6 , Jeffrey S Heier  7 , Glenn J Jaffe  8 , Keith Westby  9 , Dhaval Desai  9 , Liansheng Zhu  9 , Arshad M Khanani  10
Affiliations
Clinical Trial

Avacincaptad pegol for geographic atrophy secondary to age-related macular degeneration: 18-month findings from the GATHER1 trial

Sunil S Patel et al. Eye (Lond). 2023 Dec.

Erratum in

Abstract

Background/objectives: To assess the safety and efficacy of avacincaptad pegol (ACP), a C5 inhibitor, for geographic atrophy (GA) secondary to age-related macular degeneration (AMD) over an 18-month treatment course.

Subjects/methods: This study was an international, prospective, randomized, double-masked, sham-controlled, phase 2/3 clinical trial that consisted of 2 parts. In part 1, 77 participants were randomized 1:1:1 to receive monthly intravitreal injections of ACP 1 mg, ACP 2 mg, or sham. In part 2, 209 participants were randomized 1:2:2 to receive monthly ACP 2 mg, ACP 4 mg, or sham. The mean rate of change of GA over 18 months was measured by fundus autofluorescence.

Results: Compared with their respective sham cohorts, monthly ACP treatment reduced the mean GA growth (square root transformation) over 18 months by 28.1% (0.168 mm, 95% CI [0.066, 0.271]) for the 2 mg cohort and 30.0% (0.167 mm, 95% CI [0.062, 0.273]) for the 4 mg cohort. ACP treatment was generally well tolerated over 18 months, with most ocular adverse events (AEs) related to the injection procedure. Macular neovascularization (MNV) was more frequent in both 2 mg (11.9%) and 4 mg (15.7%) cohorts than their respective sham control groups (2.7% and 2.4%).

Conclusions: Over this 18-month study, ACP 2 mg and 4 mg showed continued reductions in the progression of GA growth compared to sham and continued to be generally well tolerated. A pivotal phase 3 GATHER2 trial is currently underway to support the efficacy and safety of ACP as a potential treatment for GA.

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Conflict of interest statement

The authors disclose the following conflicts of interest related to this manuscript: SSP has received grants/contracts from Apellis, Genentech-Roche, Ionis, IVERIC bio, and Stealth, consulting fees from IVERIC bio and Genentech-Roche, and has participated on a data safety monitoring board or advisory board for IVERIC bio. DRL has received investigator grants from Annexon, Apellis, IVERIC bio, and Stealth, consultant fees from Annexon, Apellis, IVERIC bio, and Stealth, honoraria from Annexon, Apellis, IVERIC bio, and Stealth, travel support from Annexon, Apellis, IVERIC bio, and Stealth, and served on steering committees for Annexon and IVERIC bio. JH has received institutional research grants from Genetech-Roche and IVERIC bio, and consulting fees from Gyroscope and IVERIC bio. CCW’s research has been funded in part through grants or contracts from Annexon, Apellis, Genentech-Roche, Gyroscope, Ionis, IVERIC bio, and NGM, and has received consulting fees from Annexon, Apellis, Genentech-Roche, Gyroscope, IVERIC Bio, NGM, and Stealth. DE has received contracts or grants from Annexon, Genentech-Roche, Gyroscope, Ionis, IVERIC bio, and NGM, consulting fees from Apellis, Genentech-Roche, Gyroscope, and IVERIC bio, payment or honoraria from Apellis, Genentech-Roche, and has served on advisory boards for Genentech-Roche, IVERIC bio. JSH has received consulting fees from Allegro, Annexon, Apellis, Genentech-Roche, Gyroscope, IVERIC bio, NGM, Stealth, has participated on advisory boards for Annexon, Apellis, Genentech-Roche, Gyroscope, IVERIC bio, NGM, and holds stock/stock options in Allegro. GJJ has received institutional research grants or contracts from Annexon, Ionis, and Genentech-Roche, consulting fees from Genentech-Roche, and has participated on an advisory board for Annexon. KW, DD, and LZ are employees of IVERIC bio. AMK has received contracts or grants from Annexon, Apellis, Gyroscope, IVERIC bio, NGM, Genentech-Roche, consulting fees from Apellis, Genentech-Roche, Gyroscope, IVERIC bio, and Stealth, and honoraria from Apellis and Genentech-Roche.

Figures

Fig. 1
Fig. 1. Mean change of baseline GA lesion size over 18 months (least squares mean).
A 2 mg avacincaptad pegol compared to sham (B) 4 mg avacincaptad pegol compared to sham.
See this image and copyright information in PMC

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