Published in Pipeline

The Lancet publishes 24-month results form phase

  • SYFOVRE demonstrated clinically meaningful reductions in GA lesion growth in as few as six doses per year

  • Only treatment approved for use beyond 12 months for GA, a chronic disease and leading cause of vision loss

Apellis Pharmaceuticals, Inc. announced that The Lancet published the positive 24-month results from the phase 3 OAKS and DERBY studies evaluating SYFOVRE (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

SYFOVRE is the only treatment approved for use beyond 12 months in GA, a chronic disease and leading cause of vision loss.

In the publication, both every-other-month and monthly SYFOVRE showed a clinically meaningful reduction of GA lesion growth with increasing effects over time and a well-demonstrated safety profile in a broad population of more than 1,200 patients.

“The increasing treatment effects of SYFOVRE over time with both monthly and every-other-month dosing are exciting to see and meaningful for the field,” said Charles Wykoff, MD, PhD, senior author and director of research, Retina Consultants of Texas. “The vision loss caused by GA takes a tremendous toll on patients and their families, impacting independence and well-being. SYFOVRE is a major advance for patients living with this progressive and relentless disease.”

“These 24-month data demonstrate that SYFOVRE is a clinically meaningful treatment for GA, and we are proud these results were published in one of the world's most prestigious peer-reviewed journals,” said Caroline Baumal, MD, chief medical officer, Apellis. “With SYFOVRE’s approval in the United States and five additional applications under review globally, we are working urgently to bring this important treatment to patients in need worldwide.”

Publication co-first authors are Jeffrey Heier, M.D. and Eleonora Lad, M.D., Ph.D.

About the phase 3 OAKS and DERBY studies

OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of SYFOVRE (pegcetacoplan injection) with sham injections across a broad and heterogenous population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated the efficacy of monthly and every-other-month SYFOVRE in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.