Release Details

Apellis Provides 24-Month Update from Phase 1b Study of Pegcetacoplan in Patients with Geographic Atrophy

April 13, 2021
  • Post hoc analysis showed a 46% decrease in mean lesion growth in eight patients with bilateral GA comparing treated eye vs. untreated fellow eye at 24 months (p=0.007)
  • Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021

WALTHAM, Mass., April 13, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 24-month data from the Phase 1b APL2-103 study of pegcetacoplan, an investigational targeted C3 therapy, in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a leading cause of blindness that affects approximately five million people worldwide1,2 and has no treatment.

The Phase 1b study, designed to enroll approximately 12 patients with bilateral GA (disease in both eyes), was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL). Patients were dosed monthly with pegcetacoplan in one eye using the fellow eye as an untreated control.

The current post hoc analysis includes eight patients for whom data were available for at least 24 months. In this population, the growth rate of GA lesions in the treated eye was 46% (mean square root) slower than the untreated fellow eye (p=0.007). It has been shown that lesions in both eyes tend to grow at the same rate in patients with bilateral GA.3 Of the 13 enrolled patients, there were no reported cases of inflammation and two patients (16%) developed new-onset exudation during the 24-month study duration.

“I am encouraged to see the continued effect of intravitreal pegcetacoplan in patients with GA.” stated Eleonora Lad, M.D., Ph.D., Associate Professor of Ophthalmology at the Duke University Medical Center. “I believe that these findings, which demonstrate a 46% reduction in GA lesion growth at 24 months, are clinically meaningful. I am excited to learn the Phase 3 DERBY and OAKS results later this year.”

Post hoc analysis of Study APL2-103 at 24 months

A Media Snippet accompanying this announcement is available by clicking on the image or link below:

The patient population enrolled in the Phase 1b study is similar to patients enrolled in the Phase 3 DERBY and OAKS studies but allowed for more advanced disease with a wider range of baseline lesion size and lower baseline visual acuity. DERBY and OAKS use the same pegcetacoplan formulation tested in this study and top-line data are expected in the third quarter of 2021.

Patients who participated in the APL2-103 Phase 1b study will be invited to enroll in the APL2-GA-305 GALE trial. The GALE trial is an open-label extension study designed to evaluate the long-term safety and efficacy of pegcetacoplan in patients with GA who participated in the Phase 1b study or who complete DERBY and OAKS.

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

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 GA4, 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.1,2 There are currently no approved treatments for GA.

About APL2-103
The APL2-103 study is a Phase 1b, multicenter, open label, single arm, 24-month clinical trial to assess the safety of monthly intravitreal (IVT) injections of pegcetacoplan in patients diagnosed with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary outcome measures include incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs).

About DERBY and OAKS
DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.

About GALE
GALE is a Phase 3, multicenter, open label, extension study to evaluate the long-term safety and efficacy of intravitreal pegcetacoplan in patients with GA secondary to AMD. The objectives of the study are to evaluate the long-term incidence and severity of ocular and systemic treatment emergent adverse events as well as change in the total area of GA lesions as measured by fundus autofluorescence.

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

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.

Media Contact:
Mark Dole 

Investor Contact:
Argot Partners 

1 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.

2 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.

3 Sunness JS, et al. The long-term natural history of geographic atrophy from age-related macular degeneration: enlargement of atrophy and implications for interventional clinical trials. Ophthalmology. 2007 Feb; 114(2):271-7.

4 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.