Crestwood, Kentucky - Apellis Pharmaceuticals, Inc. announced that it has begun the Phase I clinical trial of its drug compound APL-2 in patients suffering from age-related macular degeneration (AMD).
The trial, labeled ASAP II, will focus on establishing safety of intravitreal injections of APL-2 and will be conducted in patients afflicted with wet AMD at multiple sites in the United States and Australia. Apellis plans to follow ASAP II with a larger Phase II trial in patients with geographic atrophy (the advanced stage of dry AMD) in the late spring of 2015.
Dr. Cedric Francois, the Chief Executive Officer of Apellis, commented, "We look forward to continuing the exploration of complement immunotherapy in macular degeneration alongside our other disease indications. By acting centrally in the complement cascade we believe that APL-2 provides a unique opportunity to better understand the mechanisms that might modify this difficult disease."
Apellis is a clinical stage immunotherapy company and targets the complement pathways to correct auto-immune conditions. Apellis will seek to further explore the interface between complement and adaptive immunity in a range of indications, including paroxysmal nocturnal hemoglobinuria (PNH), chronic obstructive pulmonary disease (COPD) and age-related macular degeneration (AMD).
APL-2 is a next-generation inhibitor of the class of compstatin derivatives with improved physicochemical properties. APL-2 is currently being tested in a number of Phase I clinical trials and has received Orphan Drug Designation from the FDA to treat PNH. APL-2 inhibits complement at the levels of complement factor C3, thus blocking all downstream effector pathways of the complement cascade.
About Age-related Macular Degeneration
Age-related macular degeneration (AMD) is the leading cause of severe vision loss in people over the age of 65 in the United States and other western countries. The advanced forms of AMD are classified into either choroidal neovascularization (neovascular or exudative AMD, called "wet" AMD) or dry AMD. Dry AMD is responsible for approximately 20% of all legal cases of blindness in North America. While there is treatment for wet AMD with anti-VEGF therapies such as ranibizumab (Lucentis®) and aflibercept (Eylea®), no therapy exists for dry AMD. Complement inhibition is the only mechanism known to slow the rate of progression of dry AMD, as shown by Genentech / Roche with lampalizumab in a Phase II clinical trial (MAHALO) published in 2013.