Evaluation of Baseline Factors on Progression in a Large Phase-2 Clinical Trial for Geographic Atrophy (FILLY Study)

Abstract

Purpose : To evaluate the impact of select baseline factors on progression of geographic atrophy (GA) in eyes receiving treatment with APL-2 or sham.

Methods : The FILLY trial was a 246-patient Phase 2 multicenter, randomized, single-masked, sham-controlled clinical trial of APL-2 (a complement inhibitor) in patients with GA. Intravitreal injection of APL-2 was administered in the study eye monthly or every other month for 12 months. The primary efficacy endpoint was the change in GA lesion size from baseline to Month 12 compared to sham. The effect of select baseline factors (gender, baseline GA lesion size and type [unifocal or multifocal]) on mean change in GA lesion size at Month 12 from baseline was analyzed. Only patients with observed data at Month 12 were included.

Results : The study met its primary endpoint. The overall mean change in GA lesion size (mm²) was 1.46 (SD 0.98, 95% CI 1.21-1.70, p< 0.05), 1.63 (SD 1.61, 95% CI 1.20-2.05, p= 0.067), 2.19 (SD 1.50, 95% CI 1.82-2.55) in the monthly APL-2 (n=67), bi-monthly APL-2 (n=58) and sham (n=67), respectively. Change in lesion size by select baseline factors are summarized in Table 1.

Conclusions : Benefit of treatment with APL-2 was observed across the selected subgroups of baseline factors. Treatment with APL-2 trended to be more effective in controlling GA progression with increasing baseline lesion size in a non-linear manner. The impact of active treatment was observed irrespective of lesion type.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.