Apellis Pharmaceuticals Announces APL-2 Met Primary Endpoint in Phase II Geographic Atrophy Story

Apellis Pharmaceuticals, a clinical-stage biopharmaceutical company developing a platform of novel therapeutic compounds for the treatment of autoimmune diseases, announced that its complement C3 inhibitor, APL-2, met its primary endpoint in a Phase 2 clinical trial (FILLY) in patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD). At 12 months, APL-2, administered monthly via intravitreal injection, showed a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham. With every other month administration, a 20% (p=0.067) reduction was observed. Additionally, in a post hoc analysis, a greater effect was observed during the second six months of the study: a reduction in growth rate of 47% (p<0.001) with monthly administration, and a reduction of 33% (p=0.01) with every other month administration.

The most frequently reported adverse events in the study eye were associated with the injection procedure. A higher incidence of exudative AMD was observed in the treatment groups, predominantly in subjects with a history of exudative AMD in the fellow eye, and was managed with the administration of standard-of-care therapies.

“We are very excited about the results of this study,” said Cedric Francois, MD, PhD, founder and chief executive officer of Apellis. “In addition to demonstrating a statistically significant slowing of disease over 12 months, APL-2’s effect appears to increase in the second six months of the study, slowing down the rate of degeneration by almost half. We plan to move forward with Phase 3 studies as soon as possible.”

David Boyer, MD, of Retina-Vitreous Associates Medical Group, said, “These results are very exciting for all people afflicted with dry AMD with geographic atrophy. It is currently an untreatable condition, and the reduction of the progression of atrophy in this trial offers new hope for vision maintenance for our patients.”

Results, including an analysis of genetic markers, will be presented at an upcoming major medical meeting.