GlobeNewswire Inc | Nov 10, 2021 07:00AM EST

November 10, 2021

-- Pegcetacoplan, an investigational, targeted C3 therapy, has the potential to become the first treatment for GA, a leading cause of blindness worldwide

WALTHAM, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that data from the Phase 3 DERBY and OAKS studies will be presented as part of three oral presentations at the American Academy of Ophthalmology (AAO) Annual Meeting to be held November 12 15, 2021 in New Orleans, Louisiana. The studies evaluated the efficacy and safety of intravitreal pegcetacoplan, an investigational, targeted C3 therapy, in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In the studies, monthly and every-other-month pegcetacoplan showed a clinically meaningful reduction of GA lesion growth with a favorable safety profile in a broad population.

There is currently no treatment for the millions of people worldwide that suffer from geographic atrophy, a progressive and irreversible disease that ultimately leads to blindness, said Rishi Singh, M.D., investigator of the DERBY study and retina specialist and staff surgeon at Cleveland Clinic Florida. The Phase 3 data to be presented at AAO continue to support pegcetacoplans safety and efficacy profile with both monthly and every-other-month dosing and reinforce that this investigational therapy is a breakthrough for people living with GA.

Both monthly and every-other-month pegcetacoplan met the primary endpoint in OAKS, significantly reducing GA lesion growth. While narrowly missing statistical significance in DERBY, pegcetacoplan reduced GA lesion growth with both monthly and every-other-month treatment. Additionally, in both studies, pegcetacoplan demonstrated an even greater reduction in GA lesion growth in patients with extrafoveal lesions, supporting treatment with pegcetacoplan earlier in the disease progression.

Apellis plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.

Presentation details:

-- Treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration with Pegcetacoplan: Updates on the Randomized Phase 3 DERBY and OAKS Trials RET10 Friday, Nov. 12 3:38-3:44 p.m. CT -- Dry AMD: Are We on the Verge of a Treatment? SYM35 Monday, Nov. 15 9:45-11:00 a.m. CT -- Efficacy and Safety of Intravitreal Pegcetacoplan in GA: Results from the Phase 3 DERBY and OAKS Trials PA064 Monday, Nov. 15 12:18-12:25 p.m. CT

About DERBY and OAKSDERBY (621 patients enrolled) and OAKS (637 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 geographic atrophy (GA) secondary to age-related macular degeneration (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 (p-value less than .05) at 12 months.Patients in DERBY and OAKS will continue to receive masked treatment for 24 months.

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 GA,1 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, more than five million people have GA globally including approximately one million people inthe UnitedStates.2,3 There are currently noapproved treatments for GA.

About Pegcetacoplan for Geographic Atrophy (GA)Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the bodys immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplanwas granted Fast Track designation by the U.S. Food and Drug Administration (FDA) forthe treatment of geographic atrophy.

About ApellisApellis 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, pleasevisit http://apellis.com.

Apellis Forward-Looking StatementStatements 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 results of the DERBY and OAKS trials are sufficient to support regulatory submissions; whether a submission for approval of intravitrealpegcetacoplanfor GA on the basis of the DERBY and OAKS trials will be accepted by the FDA or foreign regulatory agencies; whether intravitrealpegcetacoplanwill receive approval from the FDA or equivalent foreign regulatory agencies for GA when expected or at all;and other factors discussed in the Risk Factors section of Apellis Quarterly Report on Form 10-Q with the Securities and Exchange Commission onNovember 8, 2021and 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, whetheras a result ofnew information, future events or otherwise.

MediaContact:Lissa Pavlukmedia@apellis.com617.977.6764

Investor Contact:Meredith Kayameredith.kaya@apellis.com617.599.8178

1Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals.Am J Ophthalmol2019;198:223261.2Rudnicka 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.Ophthalmology2012;119:571580.3Wong 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 Health2014;2:e106116.