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)

    • Treatment transition strategies and dosing strategies
    • Safety and efficacy in specific subpopulations
    • Biomarkers in clinical management
    • Quality of Life (QoL) and effect on Activities of Daily Living (ADL)
    • Longer-term treatment effects
    • Biomarkers of disease severity
    • Epidemiology of PNH
    • Further characterization and measurement of PNH symptoms (e.g., brain fog)
    • Screening for PNH in risk groups

  • Nephrology
    Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G)

    • Further disease characterization in patients with either IC-MPGN or C3G
    • Biomarkers of disease severity and disease progression in patients with IC-MPGN/C3G
    • Novel clinical study endpoints of disease severity and disease progression in IC-MPGN/C3G
    • Role of innate immune system in patients with IC-MPGN/C3G

  • Neurology
    Amyotrophic lateral sclerosis (ALS)

    • Biomarkers of disease progression in patients with ALS
    • Biomarkers or prognosis in patients with ALS
    • Novel clinical study endpoints of disease severity and disease progression in patients with ALS
    • Role of innate immune system in patients with ALS
    • Role of immune system in disease progression in ALS
    • Role of innate immune system in neurological disease

  • Ophthalmology
    Geographic Atrophy (GA)

    • Long term (> 5yr) natural history data on geographic atrophy (GA) patients, including impact of progression on patients with low vision
    • Assessing structural changes in the retina during GA progression
    • Development of tests to evaluate visual function in patients with GA
    • Relationship between choroidal neovascularization (CNV) and the development of GA
    • Identification and validation of biomarkers of disease progression
    • Real world effectiveness of pegcetacoplan in patients with GA
    • Effectiveness and safety of pegcetacoplan in GA patient phenotypes not assessed in clinical trials (including but not limited to: bilateral GA patients treated in both eyes; GA patients with lesions approximately 500 microns or further from the foveal center)
    • Effectiveness and safety of pegcetacoplan in GA patients with different dosing intervals and dose escalation or de-escalation.

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