Apellis to Showcase Leadership in Retina at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting


  • 10 accepted abstracts include five oral presentations from pioneering artificial intelligence (AI) collaboration
  • Presentations support the potential of intravitreal pegcetacoplan, an investigational targeted C3 therapy, as the first treatment for geographic atrophy (GA)

WALTHAM, Mass., April 16, 2021 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that 10 abstracts were accepted for presentation at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 1-7, 2021. These abstracts feature a breadth of data, from presentations that demonstrate the potential of AI to analyze the growth of GA lesions, to new safety and efficacy data for pegcetacoplan, an investigational targeted C3 therapy, in GA.

“New imaging approaches, AI-based technologies, and ultimately the development of new medicines will transform the treatment of geographic atrophy,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “Our data across these disciplines at ARVO exemplify our leadership in the retina and underscore the potential of pegcetacoplan to become the first medicine for people living with GA, a relentless disabling disease.”

The five oral presentations include studies that evaluated the use of AI to identify, monitor, and predict the growth of GA lesions. The analyses were performed on retinal scans from the positive Phase 2 FILLY study, which showed that pegcetacoplan reduced the growth rate of GA lesions, and were conducted in collaboration with the Ophthalmic Image Analysis (OPTIMA) group at the Medical University of Vienna, one of the world’s leading data analysis laboratories for retinal diseases.

“Our collaboration showed that AI can distinctly and reliably measure GA disease progression,” said Ursula Schmidt-Erfurth, M.D., professor and chair of the Department of Ophthalmology at the University Eye Hospital, Vienna, Austria. “The results support the use of AI analytics as an ideal tool to evaluate disease activity as well as therapeutic efficacy in GA.”

The oral presentations from the AI collaboration include:

  • AI-Based Quantification of Photoreceptor Maintenance in the Treatment of Geographic Atrophy Secondary to AMD in the FILLY Trial – May 2, 11:15 a.m. – 12:45 p.m. ET
  • SD-OCT Based Analysis of Treatment Effects on Geographic Atrophy Secondary to AMD in the FILLY Trial of Pegcetacoplan – May 2, 11:15 a.m. – 12:45 p.m. ET
  • Monitoring GA Lesion Size on Optical Coherence Tomography (OCT) Using Automated Deep Learning-Based Image Segmentation in the FILLY Phase 2 Clinical Trial – May 2, 11:15 a.m. – 12:45 p.m. ET
  • Topographic Effects on AI Quantified Regional Progression in the FILLY Trial of Pegcetacoplan for Treatment of Geographic Atrophy Secondary to AMD – May 3, 4:30 p.m. – 6:00 p.m. ET
  • Predictive Identification of the Fastest Progressing Geographic Atrophy Lesions Based on Deep Learning in the Phase 2 FILLY Clinical Trial of Pegcetacoplan – May 3, 4:30 p.m – 6:00 p.m. ET

Apellis abstracts regarding the safety and efficacy of pegcetacoplan and unmet need in GA include:

  • A Phase 1b Multi-Center, Open Label, Single-Arm Safety Study of Intravitreal Pegcetacoplan Supporting the Phase 3 DERBY and OAKS Studies for Geographic Atrophy: A Solution to “Tarmac Delay” – Poster Abstract – May 1, 10:15 a.m. – 12:00 p.m. ET
  • Safety of Intravitreal Pegcetacoplan in Patients with Neovascular Age-Related Macular Degeneration Receiving Anti-VEGF Therapy – Poster Abstract – May 3, 4:30 p.m. – 6:15 p.m. ET
  • Evaluation of Geographic Atrophy Secondary to Age-Related Macular Degeneration in Clinical Practice: Analysis of the AAO IRIS® Registry – Poster Abstract – May 4, 2:15 p.m. – 4:00 p.m. ET
  • Ocular Distribution of Pegcetacoplan in Rabbits Following a Single Intravitreal Injection. – Poster Abstract – May 5, 9:00 – 10:45 a.m. ET
  • Impact of Pegcetacoplan on Progression of Nascent Atrophy in Age-Related Macular Degeneration (AMD) – Paper Abstract – May 7, 4:15 p.m. – 5:45 p.m. ET

The Ophthalmic Image Analysis (OPTIMA) group at the Medical University of Vienna is a leading data analysis laboratory for retinal diseases. OPTIMA is a multidisciplinary group in retinology, computer science, and medical physics that develops computerized analysis methods for ophthalmic images and aims to individualize and lower treatment needs for patients affected by retinal diseases.

About Pegcetacoplan
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, 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. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan 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. GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. Excessive complement activation drives irreversible lesion growth in GA1, and C3 is the only target to precisely control complement overactivation. 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.2.3 There are currently no approved treatments for GA.

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 transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. 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, IC-MPGN, ALS 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’ Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2021 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.


Mark Dole

Argot Partners


1 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.

2 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571–580.

3 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.