Industry News
Breakthrough Treatment for Age-Related Vision Loss Shows Promise in New Lancet Publication
Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a pioneering pharmaceutical company, has revealed groundbreaking results in the fight against age-related macular degeneration (AMD) through its innovative treatment, SYFOVRE® (pegcetacoplan injection). The promising findings were published in a prestigious peer-reviewed journal, The Lancet, offering hope to individuals suffering from geographic atrophy (GA), a chronic disease that is the leading cause of vision loss.
The newly published research presents positive 24-month results from two crucial Phase 3 clinical trials, known as OAKS and DERBY. These trials have focused on evaluating the efficacy and safety of SYFOVRE® as a treatment for GA secondary to AMD, a debilitating condition affecting millions worldwide.
The publication reports that both monthly and every-other-month SYFOVRE® treatments have demonstrated significant reductions in GA lesion growth over time, with a well-documented safety profile across a diverse group of over 1,200 patients. These results are of particular significance, as SYFOVRE® is the only treatment to gain approval for use beyond 12 months in the management of GA.
Charles Wykoff, M.D., Ph.D., senior author and director of research at Retina Consultants of Texas, expressed his enthusiasm for the findings, stating, "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. 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."
Caroline Baumal, M.D., chief medical officer at Apellis, hailed the publication of these 24-month results as a significant milestone. She remarked, "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. 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."
The OAKS and DERBY studies are pivotal Phase 3 clinical trials involving a combined total of 1,258 participants. These studies utilized a randomized, double-masked, sham-controlled design to compare the effectiveness and safety of SYFOVRE® with sham injections in a diverse population of patients with GA secondary to AMD. The studies evaluated the efficacy of both monthly and every-other-month SYFOVRE® treatments by measuring the change in the total area of GA lesions from baseline using fundus autofluorescence.
GA is a progressive form of AMD, affecting over 5 million people worldwide, with one million in the United States alone. It is characterized by the destruction of retinal cells, leading to irreversible lesion growth, primarily driven by excessive complement activation. SYFOVRE® is designed to provide comprehensive control of the complement cascade by targeting C3, a pivotal factor in the complement system. The treatment has been approved in the United States for the management of GA secondary to AMD and is currently under review by regulatory agencies in the EU, Canada, Australia, Switzerland, and the United Kingdom, with decisions expected in early 2024.
The promising results of the SYFOVRE® treatment in the OAKS and DERBY studies offer new hope to individuals grappling with the devastating consequences of GA, bringing us one step closer to improved vision care on a global scale.
