Release Details

• SYFOVRE reduced nonsubfoveal GA lesion growth by over 40% (monthly) in Year 3 compared to projected sham in the GALE extension study
  
  
• Well-demonstrated safety profile consistent with previously reported clinical data
  
  
• Approximately 92% of patients enrolled in GALE completed the first year of the study, demonstrating robust long-term compliance
  
  
• Data were presented during an oral presentation at the American Academy of Ophthalmology (AAO) Annual Meeting
  

A Media Snippet accompanying this announcement is available by clicking on this link.

WALTHAM, Mass., Nov. 04, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced data from the GALE extension study following 3 years of continuous treatment with SYFOVRE^® (pegcetacoplan injection), the first-ever FDA-approved treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The data were reported during an oral presentation at the American Academy of Ophthalmology (AAO) Annual Meeting.

“We are continuing to see increasing SYFOVRE treatment effects year-over-year and a more than 40% reduction in nonsubfoveal lesion growth in the third year,” said Caroline Baumal, M.D., chief medical officer, Apellis. “Additionally, SYFOVRE is the only treatment that offers dosing beyond 12 months, and these results demonstrate the importance of early and continuous treatment over time.”  

SYFOVRE continued to demonstrate increasing treatment effects over time. In Year 3, SYFOVRE (all p-values nominal):

• Reduced GA lesion growth with both monthly (35%; p<0.0001) and every-other-month (EOM) (24%; p<0.0001) treatment compared to the projected sham arm.
• Reduced nonsubfoveal GA lesion growth with both monthly (42%; p<0.0001) and EOM (28%; p=0.0015) treatment compared to the projected sham arm.
• Reduced GA lesion growth by 19% (p<0.0001) after one year of SYFOVRE treatment (combined monthly and EOM), compared to the sham treatment period, in patients who crossed over from the sham group.
  

“These three-year data further solidify the long-term durability of both every-other-month and monthly SYFOVRE, including the increasing treatment effects over time,” said Jeffrey S. Heier, M.D., presenting author and director, retina service and director, retinal research, Ophthalmic Consultants of Boston. “GA is a chronic disease that requires long-term management to slow its devastating progression. With the largest dataset collected in GA, I am very encouraged about the potential of SYFOVRE to make a meaningful difference for patients who for so long had no options.”

An analysis of the untreated fellow eye further validated the treatment effects observed in Year 3. In patients with bilateral GA, lesions are known to grow at similar rates in both eyes. The fellow eye analysis serves as an important additional control to assess treatment effect.

The safety profile of SYFOVRE in Year 3 remained consistent with previously reported data. During the first year of GALE, the rate of new-onset investigator-reported exudative AMD was 7.1% (monthly) and 2.3% (EOM). There was one serious adverse event of ischemic optic neuropathy (ION) in the monthly group between Months 24 to 30, which was previously reported, and one case of endophthalmitis between Months 30 to 36. The rate of intraocular inflammation (IOI) was 0.26% per injection from Months 0 to 36, which does not include the four cases linked to the 2018 impurity. Zero events of retinal vasculitis have been observed in the SYFOVRE clinical trial program, following more than 24,000 injections to date.

Approximately 92% of patients enrolled in GALE completed the first year of the study, demonstrating robust long-term treatment compliance.

About the GALE Long-Term Extension Study
GALE (n=792) is a Phase 3, multicenter, open-label, extension study to evaluate the long-term efficacy and safety of SYFOVRE^® (pegcetacoplan injection) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The objectives of the study are to evaluate the long-term incidence and severity of ocular and systemic treatment emergent adverse events as well as change in the total area of GA lesions as measured by fundus autofluorescence. More than 80-percent of participants who completed the OAKS and DERBY studies entered the GALE study. GALE also includes 10 patients who were previously enrolled in the Phase 1b study of pegcetacoplan for GA.

Patients included in the 3-year GALE lesion growth reduction analyses were in the SYFOVRE treatment arms through Month 24 in the OAKS and DERBY studies and remained on the same regimen in GALE. Sham-treated patients in the Phase 3 OAKS and DERBY studies were eligible to transition to SYFOVRE treatment in GALE after Month 24, so a projected sham arm was used to estimate the growth of GA lesions without treatment between Months 24 and 36. The projected sham arm was estimated as the average 12-month mean rate of change in the OAKS and DERBY sham arms through Month 24.

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 SYFOVRE^® (pegcetacoplan injection) 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 SYFOVRE in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.

In Phase 3 studies at 24 months, both every-other-month and monthly SYFOVRE reduced GA lesion growth with increasing effects over time and showed a well-demonstrated safety profile.

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 SYFOVRE^® (pegcetacoplan injection)
SYFOVRE^® (pegcetacoplan injection) is the first and only approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration.

Marketing applications are currently under review with five regulatory agencies worldwide. A decision in the EU is expected in early 2024, and decisions in Canada, Australia, Switzerland, and the United Kingdom are expected in the first half of 2024.

U.S. Important Safety Information for SYFOVRE^® (pegcetacoplan injection)
CONTRAINDICATIONS

• SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation
  

WARNINGS AND PRECAUTIONS

• Endophthalmitis and Retinal Detachments
  • Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately.
• Neovascular AMD
  • In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration.
• Intraocular Inflammation
  • In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE.
• Increased Intraocular Pressure
  • Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed.

ADVERSE REACTIONS

• Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage.
  

Please see accompanying full Prescribing Information for more information.

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-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 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 safety profile of SYFOVRE. 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 these reported events will impact our commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all, including the impact of the reported events of retinal vasculitis 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 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 

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.
³ Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.