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  • Science
    • PNH
    • Geographic Atrophy
    • C3G and Primary IC-MPGN
    • HSCT-TMA
    • Pipeline
  • Medical Research
    • Scientific Focus
    • Investigator Initiated Trials
    • Collaborative Research
    • Medical Writing Grants
    • Retina Fellow Research Grant
    • Exploratory Data Analysis
  • Congress & Manuscripts
    • Congress Presentations
    • Manuscripts
  • Resources
    • Medical Information
    • Early Access & Compassionate Use Program
    • Prescribing Information
    • Community Letters
  • Search
  • This website is intended only for use by U.S. Healthcare Professionals.

Our Scientific Focus

Apellis is dedicated to improving the lives of individuals living with serious disease driven by complement and supports medical and scientific research that demonstrates the highest level of scientific integrity, inclusion, and curiosity.

Hematology
Paroxysmal Nocturnal Hemoglobinuria (PNH)

  • Quality of Life (QoL) and effect on Activities of Daily Living (ADL)
  • Further characterization and measurement of PNH symptoms (e.g., brain fog)
  • Long-term effects of better treatment

Nephrology
Complement-mediated Kidney Disease

  • Characterization of burden of disease, unmet need, novel patient reported outcomes scales and meaningful clinical outcomes in patients with complement-mediated kidney disease
  • Biomarkers of disease severity and disease progression in patients with C3G, primary IC-MPGN or other complement mediated kidney disease
  • Novel clinical and/or imaging study endpoints of disease severity and disease progression in complement mediated kidney disease
  • Role of innate immune system in patients with complement mediated kidney disease glomerular diseases
  • Epidemiology or natural history of disease that support better understanding of the trajectory of biomarkers/histological/clinical parameters over the course of disease in patients with complement-mediated kidney disease
  • Understand potential benefits of C3 inhibition in novel patient populations with complement-mediated glomerular disease
  • Understand real-world use of C3 inhibition and long-term outcomes in patients with C3G or primary IC-MPGN

Ophthalmology
Geographic Atrophy (GA) 

  • Development of studies and tests to evaluate visual function in patients with GA due to AMD
  • Further understanding of the relationship between GA and neovascularization in AMD
  • Real world effectiveness of pegcetacoplan in patients with GA due to AMD
  • Effectiveness and safety of pegcetacoplan in patients with GA due to AMD with clinical features (phenotypes) not assessed in clinical trials (including but not limited to bilateral GA patients treated in both eyes; GA patients with lesions approximately 200 microns or further from the foveal center)
  • Effectiveness and safety of pegcetacoplan in GA due to AMD with different dosing intervals and dose escalation or de-escalation
  • Further understanding of biomarkers in GA to assess disease progression and/or treatment response

Other Complement-Related Diseases

  • Understanding the role of complement in other disease areas
  • Exploration of other therapeutic areas with significant unmet needs where complement inhibition may play a role
  • Explore new complement biology that could open the door to breakthrough therapies

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Privacy PolicyTerms and Conditions

©2025 Apellis Pharmaceuticals, Inc. All rights reserved. 08/25 MED-US-CORP-22-00001 v14.0
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Geographic Atrophy Risk Calculator Important Usage Information

Intended Use

•  This Geographic Atrophy (GA) Risk Calculator is only intended for disease state awareness and for use by an eye doctor to understand the factors for disease progression. This is not a diagnostic tool or a tool to assist in treatment selection.

•  All decisions about patient care and diagnosis shall be made by a patient’s physician using his or her independent clinical judgment.

•  The GA Risk Calculator predicts a potential 5-year risk of outcomes for a GA patient (time to severe visual impairment (Snellen 20/200) and time to subfoveal involvement). This tool was developed utilizing risk equations using retrospective electronic health records of GA data from Cleveland Clinic patients (n=410). Statistical tests identified the most prominent predictors for time to severe visual impairment prediction as baseline visual acuity, subfoveal lesion, age, sex, smoking, fellow eye GA and select comorbidities. Validation was conducted using the Sham arms of Oaks (ClinicalTrials.gov, NCT03525613) and Derby (NCT03525600) trials. There is no treatment-related data incorporated in the tool nor is this tool intended to make any treatment assessments or recommendations.

•  The risk calculator being presented is for illustrative purposes only. Patient disease experience and/or progression may vary and depends on many individual factors including, but not limited to, the patient’s clinical history.

Limitations

•  Patients enrolled in clinical trials are expected to differ from those seen in routine clinical practice on important risk factors, such as time from GA onset and other severity measures.

•  More frequent data collection during clinical trials (monthly) than routine practice (~quarterly) could impact estimates of time to event outcomes.

•  The risk calculator predicts 5-year outcomes. However, the rate of progression on these outcomes can change with time within those 5-years.

•  These calculated predictions do not guarantee onset of identified outcomes.

Proceed to GA Risk Calculator

Developed by Apellis Pharmaceuticals, Inc. in collaboration with The Cleveland Clinic Foundation

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