Apellis Announces Late-Breaking Presentation of Largest Retrospective Database Study in Geographic Atrophy (GA) at AAO 2020 Virtual

Rhea-AI Summary

Apellis Pharmaceuticals (APLS) announced findings from a major retrospective study on geographic atrophy (GA) secondary to age-related macular degeneration (AMD), presented at the AAO 2020. Involving over 69,000 patients, the data reveal significant disease progression, with up to 21.6% of bilateral GA patients developing new-onset wet AMD in 24 months. The study emphasizes the pressing need for effective treatment for GA, which affects five million people globally. Currently, there are no approved therapies for GA, although Apellis is advancing pegcetacoplan in Phase 3 studies.

Positive

• Presented findings from a large retrospective study involving over 69,000 patients with GA.
• Highlighted significant disease progression, reflecting the urgent need for GA treatment.
• Pegcetacoplan is in ongoing Phase 3 studies, aiming to address the unmet need for GA therapies.

Negative

• No approved therapies for GA currently available.
• Only 40% of eligible GA patients returned for follow-up, potentially skewing data.

• Verana Health analysis of American Academy of Ophthalmology IRIS^® Registry presented in a late-breaking oral session
  
  
• Real-world clinical data show significant disease progression over a two-year period in more than 69,000 patients with GA, highlighting the urgent need for treatment
  
  
• Patients were nearly three times more likely to develop new onset wet age-related macular degeneration (AMD) in an eye with GA when wet AMD had already been detected in the contralateral eye
  

WALTHAM, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced findings from the largest retrospective database study in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The analysis of the American Academy of Ophthalmology (AAO) IRIS^® (Intelligent Research in Sight) Registry, the nation's first comprehensive clinical registry for eye disease, was conducted in partnership with Verana Health, the world’s leading data analysis group in retinal diseases. The study highlights the significant impact of GA progression on vision, underscoring the high unmet need for GA treatment in clinical practice. The data were presented today in a late-breaking oral session as part of the Retina Subspecialty Day at AAO 2020 Virtual.

The retrospective study included more than 69,000 patients diagnosed with GA and analyzed changes in visual acuity and disease progression for over two years, as well as the occurrence of concurrent wet AMD. GA is a complement-driven eye disease^1,2 that can lead to significant vision loss and affects approximately five million people around the world.^3,4  

“There is no approved therapy for GA and with new agents under development, it is essential to have a detailed understanding of disease progression in real-world clinical practice,” said Ehsan Rahimy, M.D., lead study author and surgical and medical vitreoretinal specialist at the Palo Alto Medical Foundation. “The data show that GA patients at their first encounter have useful vision that may be preserved if an effective treatment were available. The progressive loss of visual acuity observed in this study over a two-year period underscores the urgent need for a therapy to slow disease progression.” 

Key findings from the real-world clinical data show: 

• Progression from GA to new onset wet AMD was observed in 4.7% of patients with bilateral GA (GA in both eyes) and 13.3% of patients with wet AMD in the contralateral eye during the first 12 months. The rate at 24 months was 8.2% and 21.6% in bilateral GA and wet AMD in the contralateral eye, respectively.
  
  
• At the first study visit, patients presented with relatively preserved vision, especially in eyes with extrafoveal GA lesions (lesions outside the fovea, which is the central portion of the retina). However, patients with extrafoveal and foveal GA lesions progressively lost vision over time at a rate of approximately five letters per year. 
  
  
• A large proportion of GA patients did not return for a follow-up visit after two years. Of the GA patients potentially eligible for inclusion in the analysis, only 40% had a follow-up visit after two years and were ultimately included in the study.
  

“As we work to develop the first potential medicine for people with GA, we are committed to improving understanding of the disease. Our collaboration with Verana Health and the AAO IRIS^® Registry shows that wet AMD is not a rare occurrence in GA patients, and there is an opportunity to preserve vision if new treatments become available,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “These results highlight the significant unmet need in GA, and we are working urgently to advance pegcetacoplan, a targeted C3 therapy, in two ongoing Phase 3 GA studies.” 

About the IRIS^® Registry
The American Academy of Ophthalmology IRIS^® (Intelligent Research in Sight) Registry is the nation's first electronic health record-based comprehensive eye disease and condition registry. As of September 2020, the registry features over 59.99 million unique patients and includes 16,030 clinicians. It is a centralized data repository and reporting tool that can analyze patient data to produce easy-to-interpret national and inter-practice benchmark reports and provide scientific information to improve public health. The reports can validate the quality of care ophthalmologists provide and pinpoint opportunities for improvement.

About Verana Health
Verana Health, Inc. partners with leading medical associations to transform clinical data into actionable real-world evidence. These partnerships enable Verana Health to harness the comprehensive data found in qualified clinical data registries and other specialty data sources to accelerate medical research and enhance patient care. Learn more at veranahealth.com.

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 GA,⁵ and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.⁶ 

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 five million people have GA globally.^2,7 There are currently no approved treatments for GA. 

About Pegcetacoplan (APL-2)  
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. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy (C3G) by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials. 

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’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 2, 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 
+1 617 997 3484 

Investor Contact: 
Argot Partners 
apellis@argotpartners.com
+1 212 600 1902 

¹ 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.  
² Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18.  
³ 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. 
⁴ 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. 
⁵ 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.  
⁶ Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007; 357. 
⁷ 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. 

FAQ

What did the recent APLS press release reveal about GA?

The press release highlighted findings from a study indicating significant disease progression in GA patients, emphasizing the urgent need for treatment.

How many patients were involved in the APLS GA study?

The study involved over 69,000 patients diagnosed with geographic atrophy.

What is the percentage of GA patients developing wet AMD according to APLS findings?

The findings showed that up to 21.6% of patients with bilateral GA developed new-onset wet AMD within 24 months.

What is pegcetacoplan and its relevance to APLS?

Pegcetacoplan is an investigational C3 therapy being evaluated by APLS in Phase 3 studies for the treatment of GA.

Is there currently an approved treatment for geographic atrophy?

No, there are currently no approved therapies for geographic atrophy.