The Case For A New Paradigm In Geographic Atrophy Clinical Trials: Leveraging Genetics, Smarter Endpoints, And Artificial Intelligence

By Marcia Swank, VP, Head of Ophthalmology

Geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD), affects nearly five million people worldwide and remains a leading cause of irreversible vision loss. Recent approvals of the complement inhibitors pegcetacoplan and avacincaptad pegol have marked major therapeutic breakthroughs for GA. However, challenges persist in optimizing patient selection, endpoint sensitivity, and trial efficiency.

Emerging evidence highlights the influence of genetic variants — particularly those within the alternative complement pathway — in driving disease progression and therapeutic response. At the same time, advances in imaging biomarkers, artificial intelligence (AI)–based disease modeling, and patient-reported outcome measures are transforming how GA is understood and measured.

This white paper introduces a new framework for GA clinical development that integrates genetic profiling, novel biomarkers, and predictive analytics to improve precision, streamline study design, and accelerate therapy development. It also outlines practical strategies for principal investigators and clinical sites to strengthen recruitment, enhance retention, and enable more personalized, data-driven treatment pathways for patients with GA.