Hope for GA Treatment on HorizonApellis Pharmaceuticals has announced top-line data at 24 months showing increased effects over time with intravitreal pegcetacoplan, an investigational, targeted C3 therapy, in the Phase 3 DERBY and OAKS studies in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In a pre-specified analysis of GA lesion growth over 24 months, both monthly and everyother-month (EOM) pegcetacoplan showed a clinically meaningful reduction in GA lesion growth from baseline compared to sham (all p-values are nominal): • DERBY: 19% monthly, p=0.0004; 16% EOM, p=0.0030 • OAKS: 22% monthly, p<0.0001; 18% EOM, p=0.0002 Between months 18–24, the pegcetacoplan treatment effect accelerated compared to previous six-month periods, with robust reductions of GA lesion growth versus sham (all p-values are nominal). The increased effects were driven by a greater slowing of lesion growth by pegcetacoplan and not by an increase in the lesion growth rate in the sham group, which was highly consistent over each of the four six-month intervals (1.0+/-0.05 mm2). • DERBY: 36% monthly, p<0.0001; 29% EOM, p=0.0002 • OAKS: 24% monthly, p=0.0080; 25% EOM, p=0.0007 Additionally, the reduction of GA lesion growth in patients with extrafoveal lesions (28% monthly; 28% EOM) was comparable to the reduction in patients with foveal lesions (34% monthly; 28% EOM) in the combined studies between months 18-24. Consistent with expectations, no clinically meaningful difference was observed between pegcetacoplan and sham in the key secondary endpoints measuring visual function at 24 months. Studies show that GA lesion growth is correlated with loss of visual function over longer periods of time.1 The visual function outcomes at 24 months are believed to be due to the limitations of the endpoints when used for GA and the relatively early assessment timeframe. Patients will be treated with pegcetacoplan in the GALE extension study for an additional three years. Pegcetacoplan continued to demonstrate a favourable safety profile, consistent with safety data to date and longer-term exposure to intravitreal injections. No cases of endophthalmitis were reported between months 18 and 24. Over 24 months, the rate of infectious endophthalmitis was 0.034% per injection and the rate of intraocular inflammation was 0.24% per injection, which continue to be generally in line with reported rates in studies of other intravitreal therapies.2,3,4 No events of occlusive vasculitis or retinitis were observed over 24 months, and no serious adverse events of ischemic optic neuropathy were reported between months 18 and 24. The combined rate of new-onset exudations at month 24 was 11.9%, 6.7%, and 3.1% in the pegcetacoplan monthly, every-other-month, and sham groups, respectively. The company plans to submit to the European Medicines Agency by the end of this year. The U.S. marketing application is under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of Nov. 26, 2022. References 1. Sunness JS et al. Ophthalmology. 1999;106:1768–79. 2. Morioka et al. Incidence of endophthalmitis after intravitreal injection of an anti-VEGF agent with or without topical antibiotics. Scientific Reports 2020. 3. Kiss et al. Endophthalmitis rates among patients receiving intravitreal anti-VEGF injections: a USA claims analysis. Clin Ophthalmol 2018.4. Cox et al. Inflammatory complications of intravitreal anti-VEGF injections. Journal of Clinical Medicine 2021.

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