Apellis Announces Five Oral Presentations in Geographic Atrophy (GA) Highlighted at the ASRS Annual Scientific Meeting
Apellis Pharmaceuticals (Nasdaq: APLS) announced significant new data from Phase 3 studies at the American Society of Retina Specialists Annual Meeting. The results support the efficacy of pegcetacoplan in slowing lesion growth in geographic atrophy (GA) associated with age-related macular degeneration (AMD). The company submitted a New Drug Application to the FDA in June 2022, with a decision on acceptance expected in August 2022. Pegcetacoplan has no current approved treatments for GA, highlighting an urgent need for effective therapies for this debilitating condition.
- Efficacy of pegcetacoplan indicated by consistent p-values below 0.05 in GA lesion growth studies.
- FDA New Drug Application submitted for pegcetacoplan, with decision expected in August 2022.
- No approved treatments for GA currently available, emphasizing the ongoing need for effective solutions.
WALTHAM, Mass., July 14, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that five oral presentations and two on-demand papers are being highlighted at the American Society of Retina Specialists Annual Scientific Meeting taking place July 13 -16, 2022 in New York City.
New data include an analysis from the Phase 3 DERBY and OAKS studies, which showed the mean rate (slope) of GA lesion growth at 18 months with both monthly and every-other-month intravitreal pegcetacoplan, an investigational, targeted C3 therapy for geographic atrophy (GA) secondary to age-related macular degeneration. The results were consistent with previously reported 18-month data, with all p-values (nominal) below 0.05.
“Our presence at ASRS reinforces our leadership in retina and the potential of pegcetacoplan to become the first treatment for GA,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “GA causes irreversible vision loss and has no approved treatments, so there is an urgent unmet need to slow the progression of this debilitating disease.”
Apellis announced the submission of a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in June 2022. The FDA decision on NDA filing acceptance is expected in August 2022. Apellis also plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.
Symposium presentation details:
Phase 3 DERBY and OAKS Studies
- Assessment of Geographic Atrophy Lesion Progression in the Phase 3 OAKS and DERBY Trials; Dry AMD 1 Symposium; July 14; 9:06 - 9:10 a.m. ET
- Safety of Intravitreal Pegcetacoplan for Geographic Atrophy (GA): Results from the OAKS and DERBY Trials; Dry AMD 1 Symposium; July 14; 9:16 - 9:20 a.m. ET
- Relationship Between Number of Intermediate-Large Drusen and Geographic Atrophy (GA) Lesion Growth Rate in the Sham Groups of the OAKS, DERBY, and FILLY Trials; Dry AMD 3 Symposium; July 16; 10:59 - 11:03 a.m. ET
GA Disease Burden
- Retrospective Cohort Analysis of Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration Followed Up for 3 Years; Dry AMD 2 Symposium; July 15; 9:30 - 9:36 a.m. ET
- Characterizing Real-World Functional Outcomes in Patients with Geographic Atrophy: An IRIS Registry Analysis; Dry AMD 2 Symposium; July 15; 9:36 - 9:42 a.m. ET
Papers on demand:
- MOSAIC: A Qualitative Study of the Clinical, Humanistic, and Financial Burden of Geographic Atrophy (GA) Among Patients and Caregivers
- Detailed Baseline Demographics and Disease Characteristics of Patients with Geographic Atrophy (GA) Enrolled in the Phase 3 OAKS and DERBY Clinical Trials
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. Secondary functional endpoints will be evaluated after all patients have received treatment for 24 months.
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 5 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. 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 complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, 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 timing of anticipated regulatory actions or submissions. 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 FILLY, 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 FILLY, 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 May 4, 2022 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.
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Investor Contact:
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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.
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.
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.
FAQ
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