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2024-05-10 10:50 Apellis Announces Top - Line Results from Phase 3 DERBY and OAKS Studies in Geographic Atrophy ( GA ) and Plans to Submit NDA to ... Release Details Apellis Announces Top - Line Results from Phase 3 DERBY and OAKS Studies in Geographic Atrophy ( GA ) and Plans to Submit NDA to FDA in the First Half of 2022 September 9 , 2021 OAKS met the primary endpoint for both monthly and every - other - month treatment with pegcetacoplan , demonstrating a significant reduction in GA lesion growth of 22 % ( p = 0.0003 ) and 16 % ( p = 0.0052 ) , respectively , compared to pooled sham at 12 months DERBY did not meet the primary endpoint of GA lesion growth , showing a reduction of 12 % ( p = 0.0528 ) and 11 % ( p = 0.0750 ) with monthly and every - other - month treatment , respectively , compared to pooled sham at 12 months In a prespecified analysis of the combined studies , pegcetacoplan decreased GA lesion growth in patients with extrafoveal lesions at baseline by 26 % ( p < 0.0001 ) and 23 % ( p = 0.0002 ) with monthly and every - other - month treatment , respectively Favorable safety profile in both studies ; new - onset exudations occurred in 6.0 % , 4.1 % , and 2.4 % of patients in the combined pegcetacoplan monthly , every - other - month , and sham groups , respectively Pegcetacoplan has the potential to become the first treatment for patients with GA Conference call scheduled today at 4:30 p.m. ET WALTHAM , Mass . , Sept. 09 , 2021 ( GLOBE NEWSWIRE ) -- Apellis Pharmaceuticals , Inc. ( Nasdaq : APLS ) , a global biopharmaceutical company and leader in complement , today reported top - line results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan , an investigational targeted C3 therapy , in 1,258 adults with geographic atrophy ( GA ) secondary to age - related macular degeneration ( AMD ) . GA is a leading cause of blindness that impacts more than five million people globally including one million people in the United States . 1.2 Based on results from the studies , the company 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 . " These results underscore the potential for pegcetacoplan to become the first treatment for geographic atrophy , a progressive and irreversible disease that robs patients of their vision and for which no treatment exists , " said Jeffrey S. Heier , M.D. , principal investigator of the DERBY study and director , retina service and director , retinal research , Ophthalmic Consultants of Boston . " Pegcetacoplan demonstrated a clinically meaningful slowing of disease progression with an even stronger effect in GA patients with extrafoveal lesions . " Monthly and every - other - month treatment with pegcetacoplan met the primary endpoint in OAKS , significantly reducing GA lesion growth by 22 % ( p = 0.0003 ) and 16 % ( p = 0.0052 ) , respectively , compared to pooled sham at 12 months . DERBY did not meet the primary endpoint , showing a reduction in GA lesion growth of 12 % ( p = 0.0528 ) and 11 % ( p = 0.0750 ) with monthly and every - other - month treatment , respectively , compared to pooled sham at 12 months . In a prespecified analysis of the combined DERBY and OAKS studies , monthly and every - other - month treatment with pegcetacoplan reduced GA lesion growth by 17 % ( p < 0.0001 ) and 14 % ( p = 0.0012 ) , respectively , compared to pooled sham at 12 months . In a prespecified analysis of the primary endpoint , pegcetacoplan demonstrated a greater effect in patients with extrafoveal lesions at baseline . Patients with GA typically present first with extrafoveal lesions , which then progress toward the fovea where central vision is impacted . In the combined studies , monthly and every - other - month treatment with pegcetacoplan decreased GA lesion growth by 26 % ( p < 0.0001 ) and 23 % ( p = 0.0002 ) , respectively , in patients with extrafoveal lesions compared to pooled sham at 12 months . https://investors.apellis.com/news-releases/news-release-details/apellis-announces-top-line-results-phase-3-derby-and-oaks 13 1/3