Apellis Reports Top-Line Results from Phase III Studies Evaluating Pegcetacoplan for Geographic Atrophy

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Apellis Pharmaceuticals has announced the top-line results from two Phase III studies, evaluating intravitreal pegcetacoplan as a treatment for geographic atrophy.

Apellis Pharmaceuticals, a global biopharmaceutical company, has announced, in a Sep. 9, 2021 press release, the top-line results from two Phase III studies, DERBY and OAKS, which evaluated intravitreal pegcetacoplan as a treatment for geographic atrophy (GA).

The two studies involved a total of 1258 adults with GA secondary to age-related macular degeneration (AMD) and based on the outcomes, the company now plans to submit a new drug application (NDA) to FDA in the first half of 2022. The therapy was granted fast track designation for the treatment of GA by FDA in 2018 and has already been approved by FDA for use in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in May 2021.

Pegcetacoplan is an investigational targeted C3 therapy that has been designed to regulate excessive activation of the complement cascade, which can lead to the onset and progression of many serious diseases. In the OAKS study, the primary endpoint for both monthly and every-other-month treatment with pegcetacoplan was met, where the treatment arm demonstrated a significant reduction in GA lesion growth compared to the pooled sham group. Although the DERBY trial did not meet the primary endpoint, there was still a reduction of GA lesion growth compare with the pooled sham results. The therapy demonstrated a favorable safety profile in both studies.

“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, MD, principal investigator of the DERBY study and director, retina service and director, retinal research, Ophthalmic Consultants of Boston, in the press release. “Pegcetacoplan demonstrated a clinically meaningful slowing of disease progression with an even stronger effect in GA patients with extrafoveal lesions.”


“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 III study,” added Federico Grossi, MD, PhD, chief medical officer, Apellis, in the press release. “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,” said Cedric Francois, MD, PhD, co-founder and chief executive officer, Apellis, in the press release. “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.”

Source: Apellis