CRESTWOOD, KY, and WALTHAM MA, USA I September 18, 2019 IApellis Pharmaceuticals Inc., (NASDAQ:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced details about its Phase 2 FILLY study investigating intravitreal (IVT) APL-2 (pegcetacoplan) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) have been published in Ophthalmology, the journal of the American Academy of Ophthalmology. Publication of the full study report follows the release of topline study results earlier this year and shows that treatment with APL-2 resulted in statistically significant reductions in the growth of GA lesion area compared to sham at month 12.

“There is a significant need to develop treatments for people living with GA, a progressive, chronic disease that often results in permanent loss of vision,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “The FILLY study results show preliminary positive benefits of targeting the complement cascade at C3, which is implicated in the destruction of eyesight-protecting retinal pigment epithelium cells, and the potential for APL-2 to be an effective treatment option for people living with GA.”

The FILLY study was a Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating APL-2 in 246 patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. APL-2 was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to month 12 compared to sham.

At 12 months, patients treated with APL-2 showed a 29% reduction in the growth of GA lesion area in the monthly treatment group (p=0.008) and a 20% reduction in the EOM treatment group (p=0.067) compared to the pooled sham group. Statistical significance was defined as p<0.1 for this study. Post-hoc analysis showed that the effect was more pronounced in the last six months of treatment, with observed reductions of 45% (p=0.0004) and 33% (p=0.009) for APL-2 monthly and EOM, respectively, compared to sham.

The administration of APL-2 was generally well tolerated. There was an increased incidence of exudation in APL-2 treated eyes (20.9% in the monthly group and 8.9% in the EOM group) compared to sham-treated eyes (1.2%), which was manageable with the administration of standard-of-care treatment.

“There are currently no approved treatments for GA, which means that the approximately five million GA patients globally live knowing that they will lose vision over time,” said David S. Liao, M.D., lead author and retina specialist at the Retina-Vitreous Associates Medical Group. “I’m very encouraged by the FILLY data and look forward to seeing the upcoming results from Apellis’ Phase 3 studies of APL-2 in GA.”

The U.S. Food and Drug Administration (FDA) granted APL-2 Fast Track Designation for the treatment of GA, which facilitates the development and expedites the review of investigational therapies to treat serious conditions and fill an unmet medical need.

Apellis is currently enrolling two global confirmatory Phase 3 studies (DERBY and OAKS) for patients with GA. These identical, prospective, multicenter, randomized, double-masked, sham-injection controlled studies are designed to assess the efficacy and safety of multiple IVT injections of APL-2 in patients with GA secondary to AMD.

More information regarding DERBY and OAKS can be found at

About APL-2 (pegcetacoplan)
APL-2, an investigational drug, is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with downstream inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). Apellis is currently evaluating APL-2 in clinical studies in patients with geographic atrophy (GA), in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with cold agglutinin disease (CAD) and warm autoimmune hemolytic anemia (wAIHA), and in patients with C3 glomerulopathy (C3G) and other glomerular diseases. For additional information regarding our clinical trials, visit

About Geographic Atrophy (GA)
GA is an advanced form of age-related macular degeneration (AMD), a disorder of the central portion of the retina, known as the macula, which is responsible for central vision and color perception. The disease is chronic and progressive, leading to central blind spots and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally. There are currently no FDA-approved treatments for GA.

About Apellis
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit

SOURCE: Apellis