Apellis Reiterates Plans to Submit a New Drug Application in the First Half of 2022 for Pegcetacoplan for Geographic Atrophy Following FDA Feedback
Apellis Pharmaceuticals (Nasdaq: APLS) announced it received formal FDA feedback supporting its plans to submit a New Drug Application (NDA) for intravitreal pegcetacoplan aimed at treating geographic atrophy (GA) due to age-related macular degeneration. The NDA will utilize data from the Phase 3 DERBY and OAKS studies, alongside the Phase 2 FILLY study, all deemed adequate by the FDA. Apellis aims to submit the NDA in H1 2022 without needing additional trials. Over 1,500 patients participated in these studies, highlighting the company's commitment to addressing GA, a leading cause of blindness.
- FDA feedback supports NDA submission plans for pegcetacoplan.
- Studies involve over 1,500 patients, enhancing data credibility.
- No additional studies required for NDA submission, streamlining approval process.
- None.
WALTHAM, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that it received formal, written feedback from the U.S. Food and Drug Administration (FDA) that further reinforces the company’s plans to submit a New Drug Application (NDA) for intravitreal pegcetacoplan for geographic atrophy (GA) secondary to age-related macular degeneration. The NDA will be supported by efficacy and safety data from the Phase 3 DERBY and OAKS studies and the Phase 2 FILLY study.
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 Federico Grossi, M.D., Ph.D., chief medical officer, Apellis. “We look forward to working closely with the FDA to bring the first potential treatment to people living with GA, a devastating disease that leads to blindness.”
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 U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.
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 http://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, 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 Securities and Exchange Commission filed on November 8, 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:
Lissa Pavluk
media@apellis.com
617.977.6764
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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