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Apellis Completes Enrollment in Two Phase 3 Studies of the Targeted C3 Therapy, Pegcetacoplan, in Patients with Geographic Atrophy (GA)

July 7, 2020
  • Pivotal Phase 3 DERBY and OAKS studies enrolled a total of 1,259 patients
  • Top-line results expected in Q3 2021
  • Pegcetacoplan targets C3 to control the irreversible lesion growth in GA, a leading cause of blindness

WALTHAM Mass., July 07, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals Inc., (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that it completed enrollment in the Phase 3 DERBY and OAKS studies investigating intravitreal pegcetacoplan (APL-2), a targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA causes blindness and is a progressive complement-driven eye disease1,2 that affects approximately five million people globally.3,4 There are no approved therapies for GA.

“Completing enrollment in the Phase 3 DERBY and OAKS studies is a critical milestone that moves us one step closer to bringing the first treatment for geographic atrophy to patients. We are extremely grateful to all patients participating in these groundbreaking studies,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “People living with GA need a treatment and we believe targeting the complement system at C3 with pegcetacoplan has the potential to control the excessive complement activation that drives the irreversible growth of GA lesions that cause blindness. We look forward to seeing top-line results in the third quarter of 2021.”

A total of 1,259 patients are enrolled in DERBY and OAKS, which are pivotal randomized Phase 3 studies designed to compare the efficacy and safety of intravitreal pegcetacoplan with sham treatment in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the reduction in growth of GA lesion size, measured by fundus autofluorescence (FAF) at month 12 compared to baseline.

“Geographic atrophy leads to irreversible vision loss, a decline in the quality of life, and remains a major unmet need for our patients. Achieving this critical milestone reflects the importance of bringing this targeted C3 therapy to our GA patients. I want to thank my co-investigators for completing the enrollment of DERBY and OAKS, especially during the ongoing pandemic. I look forward to seeing the results next year with the hope that pegcetacoplan will enable clinicians to treat this debilitating disease,” said Jeffrey S. Heier, MD, Principal Investigator of the DERBY study and Co-President & Medical Director, Director of Retinal Research, Ophthalmic Consultants of Boston.

In July 2018, the U.S. Food and Drug Administration (FDA) granted pegcetacoplan Fast Track Designation for the treatment of GA. The Phase 3 DERBY and OAKS studies were initiated based on positive results from the Phase 2 FILLY study, which showed that monthly pegcetacoplan treatment resulted in a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham injections at 12 months.

For more information about the DERBY and OAKS studies, visit (NCT03525600) and (NCT03525613).

About Pegcetacoplan (APL-2)
Pegcetacoplan is the only investigational targeted C3 therapy in late-stage clinical trials. It is designed to control excessive complement activation, 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 including geographic atrophy (GA), paroxysmal nocturnal hemoglobinuria (PNH), cold agglutinin disease, and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of GA and the treatment of PNH. For additional information regarding our clinical trials, visit

About Geographic Atrophy (GA)
GA is an advanced form of age-related macular degeneration (AMD), the leading cause of blindness. Excessive complement activation drives the irreversible lesion growth in GA5, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1500 patients, is the only targeted C3 inhibitor being evaluated in patients 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 5 million people have GA globally.1,2  There are currently no approved treatments for GA.

About DERBY and OAKS
DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies to compare 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 compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (FAF).

The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to month 12 compared to sham.

In this study, pegcetacoplan met its primary endpoint of reducing the growth rate of the GA lesion (measured as square root transformation of GA lesion area) compared to sham after 12 months of treatment.  Pegcetacoplan administered monthly via intravitreal injection showed a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham after 12 months of treatment. With every other month administration of pegcetacoplan, a 20% (p=0.067) reduction was observed compared to sham. Statistical significance was defined as p<0.1 for this study.

Pegcetacoplan was generally well-tolerated in the Phase 2 FILLY trial. Over the full 18-month study period, a total of 26 cases of exudative AMD were reported by the investigators. These were seen more frequently in the pegcetacoplan-treated patients (18 in the monthly treatment group, 7 in the every other month treatment group and 1 in the sham control group). No negative impact on visual acuity was observed. The results of the FILLY study were published in Ophthalmology, the journal of the American Academy of Ophthalmology.

About Apellis
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and pegcetacoplan, 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 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 April 29, 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 

Investor Contact:
Sam Martin / Maghan Meyers
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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.
6Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007;357.