Apellis to Seek FDA Approval of its Dry AMD Drug
Known as APL-2, the drug met its primary endpoint in the OAKS clinical trial but not the DERBY trial
The biopharmaceutical company Apellis announced that combined results from two Phase 3 clinical trials, DERBY and OAKS, showed that its drug APL-2 (pegcetacoplan) reduced the progression of geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD). Monthly and every-other-month treatment with APL-2 reduced GA lesion growth by 17 percent and 14 percent, respectively, in the two trials. Treatments with the drug are made by injections into the vitreous, the soft gel in the middle of the eye. A total of 1,258 people were enrolled in the studies.
The company plans to submit a New Drug Application (NDA) for APL-2 to the U.S. Food and Drug Administration (FDA) in the first half of 2022. If approved by the FDA, the drug would be the first treatment available for GA.
While the OAKS trial met its primary endpoint of GA lesion growth, the DERBY trial did not. In the OAKS trial, monthly and every-other-month treatment with APL-2 reduced GA lesion growth by 22% and 16%, respectively, compared to sham (placebo) at 12 months. In the DERBY trial, APL-2 reduced lesion growth by 12% and 11% with monthly and every-other-month treatment, respectively, compared to sham at 12 months.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 that causes central vision loss due to loss of cells. There are currently no FDA-approved treatments for GA.