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

- Verana Health analysis of American Academy of Ophthalmology IRISĀ® 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 onsetĀ 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) IRISĀ® (Intelligent Research in Sight) Registry, the nation’s first comprehensive clinical registry for eye disease, was conducted in partnership with Verana Health, the worldās 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 retrospectiveĀ studyĀ includedĀ more than 69,000Ā patients diagnosed with GA and analyzedĀ changes inĀ visual acuity and disease progression for over two years, as well as the occurrence of concurrent wet AMD.Ā GA is a complement-driven eye disease1,2āÆthat can lead to significant vision loss andĀ affects approximately five million peopleĀ around the world.3,4āÆĀ
āThere 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,ā said EhsanĀ Rahimy, M.D., lead study author and surgicalĀ andĀ medical vitreoretinal specialist at the Palo Alto Medical Foundation. āThe 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.āĀ
Key findings from the real-world clinical dataĀ show:Ā
- 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 firstĀ study visit, patientsĀ presented with relatively preserved vision,Ā especially in eyes with extrafoveal GA lesions (lesions outside the fovea, which is the central portion of the retina). However,Ā patients with extrafovealĀ and fovealĀ GAĀ lesionsĀ progressively lost visionĀ over time at a rate of approximately five letters per year.Ā
- A large proportion of GA patientsĀ didĀ not return for a follow-up visit after two years.Ā Of the GA patients potentially eligible for inclusion in the analysis, only 40% had a follow-up visitĀ afterĀ twoĀ years and were ultimately included in the study.
āAs we work to develop the first potentialĀ medicine for people withĀ GA, we are committed toĀ improvingĀ understandingĀ ofĀ theĀ disease. Our collaboration with Verana Health and the AAO IRISĀ®Ā Registry showsĀ that wet AMD is not a rare occurrence in GA patients, and there is an opportunity to preserve vision if new treatments become available,āĀ said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis.Ā āThese resultsĀ highlightĀ theĀ significantĀ unmet need in GA, and we are working urgently to advanceĀ pegcetacoplan, a targeted C3 therapy, in twoĀ ongoingĀ Phase 3Ā GAĀ studies.āĀ
About the IRISĀ®Ā Registry
The American Academy of Ophthalmology IRISĀ®Ā (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)āÆĀ
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,5āÆand C3 is the only target to precisely control complement overactivation.Ā 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.6Ā
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 ināÆthe United States and five million people have GA globally.2,7āÆThere are currently noāÆapproved treatments for GA.Ā
AboutĀ PegcetacoplanĀ (APL-2)āÆĀ
PegcetacoplanāÆis an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the bodyās immune system, which can lead to the onset and progression of many serious diseases.āÆPegcetacoplanāÆis a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b.āÆApellis is evaluatingāÆpegcetacoplanāÆin several clinical studies across hematology, ophthalmology, nephrology, and neurology.āÆPegcetacoplanāÆwas granted Fast Track designation by the U.S. Food and Drug Administration (FDA) forāÆthe treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographicĀ atrophy, andĀ 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,āÆvisitāÆhttps://apellis.com/our-science/clinical-trials.Ā
About ApellisāÆĀ
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, pleaseāÆvisitāÆhttp://apellis.com.Ā
Apellis Forward-Looking StatementĀ
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 āforward-looking statementsā 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 āanticipate,ā ābelieve,ā ācontinue,ā ācould,ā āestimate,ā āexpect,ā āintend,ā āmay,ā āplan,ā āpotential,ā āpredict,ā āproject,ā āshould,ā ātarget,ā āwill,ā āwouldā 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 companyās 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; whetherāÆpegcetacoplanāÆwill successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the companyās clinical trials will warrant regulatory submissions and whetherāÆpegcetacoplanāÆwill 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 Apellisā products receive approval, they will be successfully distributed and marketed; and other factors discussed in the āRisk Factorsā section of Apellisā Quarterly Report on Form 10-Q filed with theāÆSecurities and Exchange CommissionāÆon November 2, 2020āÆand the risks described in other filings that Apellis may make with theāÆSecurities 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.āÆĀ
MediaāÆContact:Ā
Mark DoleĀ
media@apellis.comĀ
+1āÆ617āÆ997 3484Ā
InvestorāÆContact:Ā
Argot PartnersĀ
apellis@argotpartners.com
+1āÆ212āÆ600 1902Ā
1Ā Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration.Ā Ā
DtschĀ ArzteblĀ Int 2014; 111(8): 133ā8.Ā Ā
2Ā Heesterbeek, TJ,Ā LechanteurĀ YTE, et al. Complement activation levels are related to disease stage in AMD. InvestĀ OphthalmolĀ Vis Sci. 2020;61(3):18.Ā Ā
3Ā 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. OphthalmologyĀ 2012;119:571ā580.Ā
4Ā Wong WL,Ā SuĀ 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:e106ā116.Ā
5Ā Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am JĀ OphthalmolĀ 2019;198:223ā261.āÆĀ
6Ā Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007; 357.Ā
7Ā Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration.āÆDtschĀ ArzteblĀ Int 2014; 111(8): 133ā8.āÆ