Release Details

• New analysis of age-related macular degeneration (AMD) progression in Phase 2 FILLY study accepted as late-breaking oral presentation at European Society of Retina Specialists Congress (EURETINA)
• Pegcetacoplan targets C3, and has the potential to control the irreversible lesion growth in GA, an advanced form of AMD and leading cause of blindness

WALTHAM, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) has been accepted as a late-breaking oral presentation by EURETINA 2020 Virtual.

This new analysis of the FILLY study demonstrates that pegcetacoplan impacts the progression of nascent GA,^1,2 the earlier stage of disease that precedes atrophy, in areas of the retina outside of GA lesions. The data are from a collaboration with the Doheny Image Reading Research Lab, and will be presented by SriniVas Sadda, M.D. at the “Late Breaking & Reviews” oral session.

“We are excited that this post hoc analysis of the FILLY study was selected by EURETINA as a late-breaking presentation. These new data highlight the potential of targeting C3 with pegcetacoplan to transform the treatment of GA, a disease that leads to irreversible vision loss and has no approved treatments,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis.

Featured Presentation
Impact of Pegcetacoplan on Progression of Nascent Atrophy in AMD, October 3, 5:05 p.m. CET.

About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

About Geographic Atrophy (GA) 
GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA³, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.⁴

GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment 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.^5.6  There are currently no approved treatments for GA.

About FILLY
The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan 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 in pegcetacoplan-treated patients compared to sham.

About Apellis 
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

Apellis Forward-Looking Statement 
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the company’s clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company’s clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact:
Mark Dole
media@apellis.com 
617.997.3484

Investor Contact: 
Sam Martin / Maghan Meyers
Argot Partners
sam@argotpartners.com / maghan@argotpartners.com
212.600.1902

1 Schaal, KB, Gregori, G., Rosenfeld, PJ. En face optical coherence tomography imaging for the detection of nascent geographic atrophy. Am J Ophthalmol 2017;174: 145–154.

2 Guymer, RH, et al. Incomplete retinal pigment epithelial and outer retinal atrophy in age-related macular degeneration. Ophthalmology 2020;127:394-409.

3 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 

4 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007;357.

5 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 133–8. 

6 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18.