Apellis Announces Three Oral Presentations of the Phase 3 DERBY and OAKS Data in Geographic Atrophy (GA) to be Highlighted at the AAO Annual Meeting
Apellis Pharmaceuticals (APLS) announced the presentation of Phase 3 DERBY and OAKS trial data for pegcetacoplan at the AAO Annual Meeting from Nov 12-15, 2021. Pegcetacoplan, an investigational C3 therapy, demonstrated clinically meaningful reductions in geographic atrophy (GA) lesion growth in patients with age-related macular degeneration. While achieving the primary endpoint in OAKS, it narrowly missed in DERBY. The company plans to file a New Drug Application with the FDA in the first half of 2022, marking a significant step toward addressing a condition with no approved treatments worldwide.
- Phase 3 studies show pegcetacoplan significantly reduced GA lesion growth.
- Monthly and every-other-month dosing options demonstrated a favorable safety profile.
- The company plans to submit a New Drug Application (NDA) with the FDA in H1 2022.
- DERBY trial narrowly missed statistical significance in reducing GA lesion growth.
- Pegcetacoplan, an investigational, targeted C3 therapy, has the potential to become the first treatment for GA, a leading cause of blindness worldwide
WALTHAM, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that data from the Phase 3 DERBY and OAKS studies will be presented as part of three oral presentations at the American Academy of Ophthalmology (AAO) Annual Meeting to be held November 12 – 15, 2021 in New Orleans, Louisiana. The studies evaluated the efficacy and safety of intravitreal pegcetacoplan, an investigational, targeted C3 therapy, in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In the studies, monthly and every-other-month pegcetacoplan showed a clinically meaningful reduction of GA lesion growth with a favorable safety profile in a broad population.
“There is currently no treatment for the millions of people worldwide that suffer from geographic atrophy, a progressive and irreversible disease that ultimately leads to blindness,” said Rishi Singh, M.D., investigator of the DERBY study and retina specialist and staff surgeon at Cleveland Clinic Florida. “The Phase 3 data to be presented at AAO continue to support pegcetacoplan’s safety and efficacy profile with both monthly and every-other-month dosing and reinforce that this investigational therapy is a breakthrough for people living with GA.”
Both monthly and every-other-month pegcetacoplan met the primary endpoint in OAKS, significantly reducing GA lesion growth. While narrowly missing statistical significance in DERBY, pegcetacoplan reduced GA lesion growth with both monthly and every-other-month treatment. Additionally, in both studies, pegcetacoplan demonstrated an even greater reduction in GA lesion growth in patients with extrafoveal lesions, supporting treatment with pegcetacoplan earlier in the disease progression.
Apellis plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.
Presentation details:
- Treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration with Pegcetacoplan: Updates on the Randomized Phase 3 DERBY and OAKS Trials – RET10 – Friday, Nov. 12 – 3:38-3:44 p.m. CT
- Dry AMD: Are We on the Verge of a Treatment? – SYM35 – Monday, Nov. 15 – 9:45-11:00 a.m. CT
- Efficacy and Safety of Intravitreal Pegcetacoplan in GA: Results from the Phase 3 DERBY and OAKS Trials – PA064 – Monday, Nov. 15 – 12:18-12:25 p.m. CT
About DERBY and OAKS
DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections 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 .05) at 12 months. Patients in DERBY and OAKS will continue to receive masked treatment for 24 months.
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, including whether the results of the 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 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 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.
FAQ
What are the results of the Phase 3 DERBY and OAKS trials for pegcetacoplan?
What is the significance of the AAO Annual Meeting for Apellis Pharmaceuticals?
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What is geographic atrophy and why is it significant?
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