Benzinga | Oct 19, 2020 07:04AM EDT

Apellis Announces 18-Month Data from Phase 1b Study of Pegcetacoplan in Patients with Geographic Atrophy

* First data for 18 months of treatment with intravitreal pegcetacoplan demonstrates continued slowing of GA lesion growth beyond 12 months

* Post hoc analysis demonstrated a 52% decrease in mean lesion growth in seven patients with bilateral GA comparing treated eye vs. untreated fellow eye at 18 months (p=0.01)

* Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021



Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 18-month data from the Phase 1b APL2-103 study of pegcetacoplan (APL-2) in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a complement-driven eye disease1,2 that can cause blindness, affects approximately five million people globally3,4 and has no approved treatment. The study, which enrolled 12 patients with bilateral GA (disease in both eyes), was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL).

Patients were dosed monthly with pegcetacoplan in one eye using the fellow eye as an untreated control. Apellis previously reported results at Month 12 in nine patients demonstrating a trend in reduced GA lesion growth in treated eyes versus untreated fellow eyes. The current post hoc analysis reports on the seven Phase 1b study patients for whom data were available for at least 18 months. In this population, the growth rate of GA lesions in the treated eye was on average 52% (mean square root) slower than the opposite untreated eye (p=0.01). It has been shown that lesions in both eyes tend to grow at the same rate in patients with bilateral GA.5 Of the 12 enrolled patients, there were no reported cases of inflammation and, as previously reported, one patient (8%) developed new-onset exudation at Month 12.

"It is exciting to see data for the efficacy of intravitreal pegcetacoplan at 18 months among patients with GA," said Charles Wykoff, M.D., Ph.D., Director of Research, Retina Consultants of Houston, and Investigator of Apellis' Phase 1b and Phase 3 GA trials. "These data align with the Phase 2 FILLY results, where patients with bilateral GA in the monthly treated population had a significant reduction in growth relative to their untreated fellow eye in a post hoc analysis at 12 months. Finally, while this is a limited number of patients, I am encouraged to see that the benefit of pegcetacoplan in slowing GA growth seems to be maintained through Month 18. I believe that a 52% reduction in GA lesion growth at Month 18 is likely to be highly clinically meaningful."

Post hoc analysis at 18 months

Figure 1. Mean (? SE) change from baseline in square root GA lesion measured by fundus autofluorescence in the study eye (SE) and fellow eye (FE). Percentage difference and p value represents the comparison in GA growth between the study and fellow eye at each timepoint.





The ongoing pegcetacoplan development program in GA includes the Phase 1b APL2-103 study and the Phase 3 DERBY and OAKS studies. The patient population enrolled in the Phase 1b study is similar to DERBY and OAKS but allowed for more advanced disease with a wider range of baseline lesion size and lower baseline visual acuity.

The DERBY and OAKS studies were initiated in 2019 with the pegcetacoplan formulation tested in this Phase 1b study and top-line data are expected in the third quarter of 2021.