Apellis Announces Two New Publications from the Positive Phase 2 FILLY Study Evaluating Pegcetacoplan in Geographic Atrophy

Rhea-AI Summary

Apellis Pharmaceuticals (APLS) announced the publication of post hoc analyses from the Phase 2 FILLY study, validating the efficacy of pegcetacoplan, a C3 therapy for geographic atrophy (GA) secondary to AMD. The articles, featured in Ophthalmology and the American Journal of Ophthalmology, confirm consistent results across patient subgroups, indicating a significant unmet need in GA treatment, which currently lacks approved therapies. With over 1,200 patients enrolled in ongoing Phase 3 studies, Apellis aims to provide a breakthrough solution for approximately five million GA patients globally.

Positive

• Efficacy data from Phase 2 FILLY study published in two leading journals supports pegcetacoplan's potential.
• Ongoing Phase 3 studies (DERBY and OAKS) have enrolled over 1,200 patients, demonstrating strong clinical engagement.

Negative

• None.

• Data published in Ophthalmology and the American Journal of Ophthalmology underscore the potential of pegcetacoplan, an investigational targeted C3 therapy, for geographic atrophy
  

WALTHAM, Mass., March 10, 2021 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that two leading journals, Ophthalmology and the American Journal of Ophthalmology, published post hoc analyses from the Phase 2 FILLY study of intravitreal pegcetacoplan for geographic atrophy (GA) secondary to AMD. GA is a leading cause of blindness that affects approximately five million people around the world^1,2 and has no approved treatments.

• The analysis, “Impact of Baseline Characteristics on Geographic Atrophy Progression in the FILLY Trial Evaluating the Complement C3 Inhibitor Pegcetacoplan,” was published in the American Journal of Ophthalmology, and showed that efficacy results across patient subgroups were consistent with the FILLY results.
  
  
• Published in Ophthalmology, “Characterizing New-Onset Exudation in the Randomized Phase 2 FILLY Trial of Complement Inhibitor Pegcetacoplan for Geographic Atrophy,” further clarified characteristics of investigator-determined exudations reported in the study.
  

“Geographic atrophy remains the most significant unmet need in the retina, and we are committed to advancing the first medicine for people living with this relentless disease,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “These data from two new analyses of our positive Phase 2 FILLY study reinforce the potential of pegcetacoplan in GA, and we look forward to sharing results from our Phase 3 GA studies in the third quarter of this year.”

Pegcetacoplan is currently being evaluated in two pivotal studies, DERBY and OAKS, which enrolled more than 1,200 patients with GA.

About Pegcetacoplan (APL-2)
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 FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and EMA. For additional information regarding pegcetacoplan 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. 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 GA³, 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 FILLY
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 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 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 www.apellis.com.

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 in respect of the expected closing of the exchanges. 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 conditions for the closing of the exchanges will be satisfied 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
media@apellis.com
617.997.3484

Investor Contact:
Argot Partners
apellis@argotpartners.com 
212.600.1902 

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 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. 

FAQ

What did the Phase 2 FILLY study reveal about pegcetacoplan for geographic atrophy?

The Phase 2 FILLY study revealed that pegcetacoplan showed consistent efficacy across patient subgroups, reinforcing its potential for treating geographic atrophy.

When will Apellis Pharmaceuticals share results from its Phase 3 studies for pegcetacoplan?

Apellis Pharmaceuticals plans to share results from its Phase 3 studies in the third quarter of the year.

What is geographic atrophy and why is it significant?

Geographic atrophy is a leading cause of blindness, affecting approximately five million people globally, and currently has no approved treatments.

How is pegcetacoplan administered in clinical trials?

Pegcetacoplan is administered as an intravitreal injection monthly or every other month for 12 months during clinical trials.

What are the Phase 3 studies for pegcetacoplan called?

The Phase 3 studies for pegcetacoplan are called DERBY and OAKS.