Apellis Announces Plans to Submit 24-Month Phase 3 Data to the FDA for Pegcetacoplan NDA for Geographic Atrophy (GA)
- Inclusion of the 24-month data has the potential to strengthen the product profile at launch, with minimal impact to launch timing
- Submission will be a Major Amendment to the New Drug Application (NDA), extending the review period by three months with an expected PDUFA target action date in February 2023
WALTHAM, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals Inc (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today provided an update on its New Drug Application (NDA) for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
The company plans to submit the 24-month efficacy data from the Phase 3 DERBY and OAKS studies as part of its application. As previously announced, these data showed robust and consistent effects with monthly and every-other-month pegcetacoplan treatment in both studies. The 24-month safety data were previously submitted as part of the 120-day update. The submission is planned for November 2022 and will be a Major Amendment to the NDA, extending the review period by three months with an expected Prescription Drug User Fee Act (PDUFA) target action date in February 2023.
“We look forward to sharing with the FDA the 24-month Phase 3 data, which demonstrate robust and increasing effects over time. We were able to complete the full 24-month data package faster than expected, providing us with an opportunity to submit these data prior to the November 26 PDUFA date. These longer-term data have the potential to provide an even stronger product profile at launch,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder, Apellis. “We expect the submission will have minimal impact to the timing of the launch, which was originally planned for January. Assuming a February PDUFA date, we would be prepared to launch immediately following an approval.”
Dr. Francois continued, “We will work closely with the FDA as they complete their review of the pegcetacoplan NDA and look forward to bringing the first potential therapy to people living with GA, who currently have no approved treatment for this relentless and debilitating disease.”
Apellis remains on track to submit an EU marketing authorization application, which will also include the 24-month results, to the European Medicines Agency by the end of 2022.
About the Phase 3 OAKS and DERBY Studies
OAKS (n=621) and DERBY (n=637) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies was to evaluate 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 at 12 months. Patients continued to receive masked 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 five 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.
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 submissions and decisions. 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 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’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 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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3Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
4Seddon, 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.