In the written feedback, the FDA stated that they do not make a distinction between Phases, provided a clinical trial is adequate and well controlled, and that all three studies appear to be adequate and well controlled. Based on this feedback, Apellis remains on track to submit an NDA in the first half of 2022 and believes that no additional studies will be needed for the NDA submission.
“We are pleased that the FDA’s feedback is consistent with our plans to submit an NDA for pegcetacoplan in GA based on data from more than 1,500 patients across three randomized, well-controlled studies,” said
About DERBY and OAKS
DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies evaluating the efficacy and safety of intravitreal pegcetacoplan 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 0.05) at 12 months. Patients in DERBY and OAKS will continue to receive masked treatment for 24 months.
About FILLY
The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled study evaluating the efficacy and safety of intravitreal pegcetacoplan in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the study was to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in square root GA lesions from baseline as measured by fundus autofluorescence (p-value less than 0.1) at 12 months followed by six months of monitoring after the end of treatment.
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 in the United States.2,3 There are currently no approved 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 body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan was granted Fast Track designation by the
About Apellis
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, whether the results of the FILLY, DERBY, and OAKS trials are sufficient to support regulatory submissions; whether a submission for approval of intravitreal pegcetacoplan for GA on the basis of the FILLY, DERBY and OAKS trials will be accepted by the FDA or foreign regulatory agencies; whether intravitreal pegcetacoplan will 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
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Investor Contact:
Meredith Kaya
meredith.kaya@apellis.com
617.599.8178
1 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.
2 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.
3 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.
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