The biopharmaceutical company Apellis has treated the first patient in its Phase 3 clinical trial program for APL-2, a compound designed to slow the progression of advanced dry age-related macular degeneration (AMD) also known as geographic atrophy (GA).
The Phase 3 program consists of two clinical trials: DERBY and OAKS. Each will enroll 600 patients. Trial participants will receive intravitreal injections of APL-2, or a sham procedure (placebo), monthly or every other month. The injections are made into the vitreous, the soft gel in the middle of the eye.
In the Phase 2 clinical trial, monthly injections of APL-2 slowed the growth of GA lesions by 29 percent. GA lesions are the regions in the retina where loss of cells occurs. Cell loss correlates with vision loss.
APL-2 is designed to slow the progression of GA by inhibiting C3, a protein in the complement system. While complement is part of the human immune system that wards off harmful bacteria and viruses, it can cause damage if not controlled properly. Researchers have strong evidence that an overactive complement system is involved in the death of retinal cells in AMD.
According to Apellis, 1 million people in the United States have GA, a progressive condition which causes central vision loss. There are currently no FDA-approved treatments for GA.