APLS Apellis Pharmaceuticals Inc

Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.

“We are deeply disappointed by this outcome, which leaves millions of Europeans with GA without a treatment for this irreversible form of blindness,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder, Apellis. “This CHMP opinion was made despite broad support for pegcetacoplan from the European retina community and multiple dissenting votes by CHMP members who supported a path to approval.”

“We have seen the profound impact this medicine has had for GA patients in the U.S. and remain committed to expanding access to this crucial treatment, focusing on serving unmet patient needs in the U.S. and other regions globally,” Dr. Francois continued.

“It is disappointing to know that we will not be able to provide access to this treatment to GA patients in the EU,” said Frank G. Holz, OAKS and DERBY study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany. “As an ophthalmologist and retina specialist, I have seen how patients with GA lose their ability to read, drive, and even see faces. The Phase 3 data for pegcetacoplan are clinically meaningful and showed the potential to make a difference for EU patients.”

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.1,2 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.3

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 ApellisApellis 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-ever 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 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. 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 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 27, 2024 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: Tracy Vineis media@apellis.com617.420.4839

Investor Contact: Meredith Kaya meredith.kaya@apellis.com617.599.8178 

1Rudnicka 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;119:571–580.2Wong 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:e106–116.3Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.