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

Sunil S. Patel, David R. Lally, Jason Hsu, Charles C. Wykoff, David Eichenbaum, Jeffrey S. Heier, Glenn J. Jaffe, Keith Westby, Dhaval Desai, Liansheng Zhu, Arshad M. Khanani

Research output: Contribution to journalArticlepeer-review

36 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)3551-3557
Number of pages7
JournalEye (Basingstoke)
Issue number17
StatePublished - Dec 2023


  • Humans
  • Geographic Atrophy/drug therapy
  • Prospective Studies
  • Visual Acuity
  • Macular Degeneration/complications
  • Intravitreal Injections
  • Fluorescein Angiography

ASJC Scopus subject areas

  • Sensory Systems
  • Ophthalmology


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