Apellis C3 Therapy slows GA progression

Apellis Announces New Analysis Demonstrating Targeted C3 Therapy Pegcetacoplan Slows Progression of Early Disease in Patients with Geographic Atrophy (GA)

October 3, 2020

  • Post hoc analysis of Phase 2 FILLY study shows 39% reduction in rate of progression from nascent GA , an earlier form of disease, to GA in patients treated with pegcetacoplan monthly vs. sham.
  • First ever observation of slowed nascent GA progression in a Phase 2 study signals potential benefits of earlier intervention with pegcetacoplan in patients with GA
  • Data support hypothesis that targeting C3 with pegcetacoplan addresses an underlying cause of disease , excessive complement activation, as evidenced by slowed progression of nascent GA to GA.

WALTHAM, Mass., Oct. 03, 2020 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc.(Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the results of a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The post hoc analysis found that monthly treatment with pegcetacoplan significantly reduced the rate of progression from nascent GA to GA in areas of the retina outside of existing GA lesions. The data were presented today in a late-breaking oral session at the European Society of Retina Specialists 2020 Virtual(EURETINA).

Pegcetacoplan is the only targeted C3 therapy in Phase 3 clinical trials for GA, a complement-driven eye disease that causes blindness, affects approximately five million people globally and has no approved treatment. In the post hoc analysis, progression from nascent GA to GA was observed in 50.0% of the pegcetacoplan-treated group and 81.8% of the sham-treated group (p=0.02). The 39% reduction in progression from nascent GA to GA indicates that pegcetacoplan slows the rate of progression of AMD in areas of the retina outside of GA lesions. Progression from large drusen (deposits in the retina that are a hallmark of AMD) to nascent GA or GA at Month 12 was observed in 22.6% of patients in the pegcetacoplan-treated group and 33.3% of patients in the sham-treated group (p=0.31).

“Our findings from this post-hoc analysis demonstrate that intravitreal pegcetacoplan, a targeted C3 therapy, significantly lowers the rate of progression from nascent GA to GA inpatients when compared to sham controls,” said Srini Vas Sadda, M.D., President & Chief Scientific Officer of the Doheny Eye Institute and lead investigator. “This study provides exciting evidence to support further exploration of the potential of pegcetacoplan for earlier intervention in the course of GA.”
The post hoc analysis, a collaboration with the Doheny Image Reading Research Lab, included FILLY patients from the monthly pegcetacoplan-treated group (n=42) and sham-treated group (n=69) who completed the Month 12 study visit and who did not develop exudative or neovascular AMD. The objective of the analysis was to investigate the effects of pegcetacoplan on complement-driven progression of AMD outside of GA lesions.
“Patients with geographic atrophy experience changes in the retina that progress from the earlier stages of AMD to the beginning of atrophy and ultimately irreversible vision loss,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “This post hoc analysis of the FILLY study demonstrates that pegcetacoplan slows early disease progression and may have the potential to delay the onset of GA and vision loss in patients.”
Earlier this year, Apellis announced the completion of patient enrollment in the ongoing Phase 3 DERBY and OAKS studies investigating pegcetacoplan in patients with GA secondary to AMD. Top-line results from these pivotal trials are expected in Q3 2021.

About Pegcetacoplan (APL-2)

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate 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 paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy(GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

About Geographic Atrophy (GA)

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, and C3is the only target to precisely control complement overactivation. 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.
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.
There are currently no approved treatments for GA.


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 in pegcetacoplan-treated patients compared to sham.

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 auto-fluorescence (FAF).

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 best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. 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 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 July 30, 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
Argot Partners


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