What is Going on with APLS?
Apellis Pharmaceuticals (APLS) has reported its results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan. Intravitreal pegcetacoplan is a potential therapy that could help treat adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), a leading cause of blindness globally. The primary endpoint for the OAKS study was realized, with a reduction in GA lesion growth of about 22% over 12 months. The primary endpoint for the DERBY study was not met, with a reduction in GA lesion growth of 12%. Apellis plans on submitting a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.
What This Means for APLS?
Speaking on the importance of these results, Jeffrey S. Heier, M.D., principal investigator of the DERBY study and director, states, “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. Pegcetacoplan demonstrated a clinically meaningful slowing of disease progression with an even stronger effect in GA patients with extrafoveal lesions.”
Federico Grossi, M.D., Ph.D., chief medical officer, Apellis, states, “Our mission is to develop transformative therapies for people with complement-driven diseases and now, after decades of challenges in this complex disease, pegcetacoplan is the first investigational therapy to significantly slow the progression of GA in a large Phase 3 study. Across our ophthalmology development program, pegcetacoplan has demonstrated an efficacy and safety profile with both monthly and every-other-month dosing that we believe supports treatment for GA patients. We look forward to working with regulatory authorities to bring this medicine to patients in need as quickly as possible.”
“On the heels of our recent FDA approval in PNH, these pivotal results further reinforce the platform potential of targeting C3 across multiple diseases with few or no treatments. Apellis is singularly positioned to make a meaningful difference for patients living with a broad range of retinal, rare, and neurological diseases by targeting C3 to comprehensively control complement,” says, Cedric Francois, M.D., Ph.D., co-founder and chief executive officer at Apellis.
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Apellis Pharmaceuticals Inc is a clinical-stage biopharmaceutical company. It focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, which is an integral component of the immune system, at the level of C3 the central protein in the complement cascade.