Apellis Provides Update on Ongoing Regulatory Review of Pegcetacoplan for GA in the European Union
- None.
- Negative trend vote from CHMP on MAA for pegcetacoplan
- Disappointment among company executives and the retina community
Conference call today at 8:30 a.m. ET
WALTHAM, Mass., Dec. 14, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today an update on the ongoing review of its marketing authorization application (MAA) for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
Following the oral explanation meeting on December 13, Apellis was informed of a negative trend vote on the MAA for pegcetacoplan. Apellis expects the CHMP to adopt a negative opinion at its next meeting, which is scheduled to be held January 22-25, 2024. If a negative opinion is adopted, Apellis plans to appeal the outcome and seek re-examination of the opinion.
“We are very disappointed in this outcome and saddened for the millions of GA patients in Europe who have no treatment for this leading cause of blindness,” said Jeffrey Eisele, Ph.D., chief development officer, Apellis. “We firmly believe in the strength of the Phase 3 results, and we will continue working closely with the CHMP, the retina community, and patient advocacy groups with the goal of bringing pegcetacoplan to GA patients in Europe.”
The MAA is based on results from the Phase 3 OAKS and DERBY studies at 24 months, which were published in The Lancet. Treatment with both every-other-month and monthly pegcetacoplan reduced GA lesion growth with increasing treatment effects over time and showed a well-demonstrated safety profile. Additionally, pegcetacoplan preserved visual function longer in multiple post hoc Phase 3 analyses that have been presented at medical congresses.
More than 2.5 million people in Europe are living with GA,1 a progressive and irreversible form of vision loss that severely impairs independence and quality of life.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss the CHMP trend vote on Thursday, December 14 at 8:30 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website. A replay of the webcast will be available for 30 days following the event.
About the Phase 3 OAKS and DERBY Studies
OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections across a broad and heterogenous 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 and a leading cause of blindness worldwide, impacting more than one million Americans and five million people worldwide.2,3 It is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. The vision loss caused by GA severely impairs independence and quality of life by making it difficult to participate in daily activities. On average, it takes only 2.5 years for GA lesions to start impacting the fovea, which is responsible for central vision.4
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. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological 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. These statements include, but are not limited to, statements regarding the appeal and re-examination of the MAA if CHMP issues a negative opinion for intravitreal pegcetacoplan and the safety profile of intravitreal pegcetacoplan. 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 benefit/risk profile of SYFOVRE following the reported events of retinal vasculitis will impact our commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all, including the impact on the likelihood and timing of such approvals; 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 Quarterly Report on Form 10-Q filed on November 1, 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
1Apellis data on file reflecting analysis of Colijin et al., 2016; Wong et al. 2014; Rudnicka et al., 2014; Korb et al., 2014; Piermarocchi et al., 2011; Fernandez-Arias et al., 2011; Augood et al., 2006.
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. Ophthalmology 2012;119:571–580.
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 Health 2014;2:e106–116.
4 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
FAQ
What is the latest update from Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) regarding its marketing authorization application (MAA) for pegcetacoplan?
What are the results of the Phase 3 OAKS and DERBY studies for pegcetacoplan?
What is the plan of Apellis Pharmaceuticals, Inc. after receiving the negative trend vote from CHMP?
