Apellis Announces Validation of Four Marketing Applications for Pegcetacoplan for Geographic Atrophy
Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that its marketing applications for intravitreal pegcetacoplan, targeted at treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD), have been validated by regulatory bodies in Canada, Australia, the UK, and Switzerland. Decisions from these authorities are anticipated in the first half of 2024. Following its first approval in the U.S., this move marks a significant step towards worldwide access to pegcetacoplan. The applications were based on Phase 3 OAKS and DERBY study results, demonstrating meaningful reductions in GA lesion growth. Additionally, a marketing authorization application is under review by the European Medicines Agency, with an expected decision in early 2024.
- Marketing applications for pegcetacoplan validated in Canada, Australia, UK, and Switzerland.
- Anticipation of approval decisions in the first half of 2024, indicating potential for expanded market access.
- Phase 3 studies (OAKS and DERBY) showed clinically meaningful reductions in GA lesion growth with a strong safety profile.
- Dependence on upcoming approval decisions, which are not guaranteed.
- Market performance impacted by potential delays or rejections from regulatory authorities.
- Applications are under review in Canada, Australia, the United Kingdom, and Switzerland
- Approval decisions expected in the first half of 2024
WALTHAM, Mass., April 21, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that the company has received validation of its marketing applications by regulatory authorities in Canada, Australia, the United Kingdom, and Switzerland for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Decisions by local regulatory authorities in these countries are expected in the first half of 2024.
“With our first approval of pegcetacoplan in the U.S. and multiple global decisions to be expected within the next year, we are closer than ever to achieving our goal of bringing pegcetacoplan to patients worldwide,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “We look forward to working with regulators in Canada, Australia, the United Kingdom, and Switzerland to deliver the first potential treatment for GA in these countries.”
The marketing applications were validated via the Access Consortium Work-Sharing Initiative, which supports coordinated review across multiple health authorities. This initiative has the potential to offer an efficient regulatory pathway and lead to timely access to treatments for patients.
Results from the Phase 3 OAKS and DERBY studies at 24 months are the basis of the applications. In the studies, treatment with both every-other-month and monthly pegcetacoplan showed clinically meaningful reductions of GA lesion growth with effects that increased over time and a well-demonstrated safety profile.
A marketing authorization application for pegcetacoplan is under review by the European Medicines Agency with a decision expected in early 2024.
About OAKS and DERBY
OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated the efficacy of monthly and every-other-month pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.
About Geographic Atrophy (GA)
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness that impacts more than five million people worldwide, including one million people in the United States.1,2 This progressive disease can severely impair visual function, independence, and quality of life as it takes on average 2.5 years for GA lesions to encroach the fovea, which is responsible for central vision.3 GA is caused by destruction of retinal cells through irreversible lesion growth that is driven by excessive complement activation.4 C3 is the only target that can precisely control the complement cascade due to its central location.
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. SYFOVRE™ (pegcetacoplan injection) is approved in the United States for the treatment of GA secondary to age-related macular degeneration.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first and only therapy for geographic atrophy, a leading cause of blindness around the world. With nearly a dozen clinical and pre-clinical programs underway, we believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
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. 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 pegcetacoplan will be commercially available when expected; whether clinical trials of pegcetacoplan indicate an apparent positive effect that is greater than the actual positive effect, whether pegcetacoplan will receive approval from foreign regulatory agencies for GA when expected or at all; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 21, 2023 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 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 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
4 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.
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