Apellis Pharmaceuticals, Inc. has announced longer-term data from the Phase 3 DERBY and OAKS studies, which showed that intravitreal pegcetacoplan, an investigational, targeted C3 therapy, continued to reduce geographic atrophy (GA) lesion growth and demonstrate a favorable safety profile at month 18 for the treatment of GA secondary to age-related macular degeneration (AMD). These data will be included in the New Drug Application (NDA) that the company plans to submit to the U.S. Food and Drug Administration (FDA) in the second quarter of 2022.
“It is exciting to see these positive data with pegcetacoplan, which showed continuous and potentially improving effects over time. These 18-month results provide further evidence that pegcetacoplan meaningfully slows disease progression and has the potential to preserve vision longer,” said Jeffrey S. Heier, M.D., principal investigator of the DERBY study and director, retina service and director, retinal research, Ophthalmic Consultants of Boston. “In my practice, I have seen how devastating it can be for people living with GA to lose more of their vision year after year. There is an urgent unmet need in GA, and these results reinforce the potential of pegcetacoplan to become the first-ever treatment for patients with this debilitating disease.”
In a longer-term analysis of the primary endpoint, pegcetacoplan continued to reduce GA lesion growth compared to pooled sham at month 18 (all p-values are nominal):
- In OAKS, pegcetacoplan reduced GA lesion growth with both monthly (22%; p<0.0001) and every-other-month treatment (16%; p=0.0018).
- In DERBY, pegcetacoplan reduced GA lesion growth with both monthly (13%; p=0.0254) and every-other-month treatment (12%; p=0.0332).
- Pegcetacoplan demonstrated marked improvements in DERBY during months 6-12 with reductions of 17% with monthly and 16% with every-other-month treatment compared to months 0-6, and the treatment effects were sustained through month 18. The treatment effects observed in DERBY were comparable with OAKS during months 6-18.
- Data at 18 months from the combined studies show the potential for improving treatment effects with pegcetacoplan over time. The reduction in GA lesion growth improved with monthly pegcetacoplan treatment from 13% to 21% from months 0-6 to months 12-18. The reduction in GA lesion growth improved with every-other-month pegcetacoplan treatment from 12% to 17% from months 0-6 to months 12-18.
“Pegcetacoplan is the only therapy to continuously reduce GA lesion growth across a large and broad patient population in Phase 3 studies. Building on our 12-month results, both monthly and every-other-month pegcetacoplan demonstrated clinically meaningful effects and a favorable safety profile with longer-term use,” said Federico Grossi, M.D., Ph.D., chief medical officer at Apellis. “We look forward to submitting these data to the FDA and working with regulatory agencies to bring pegcetacoplan to people living with GA around the world as quickly as possible.”
At month 18, pegcetacoplan continued to demonstrate a favorable safety profile, consistent with safety at 12 months and longer-term exposure to intravitreal injections. The rate of infectious endophthalmitis was 0.044% per injection, and the rate of intraocular inflammation was 0.23% per injection. Rates of endophthalmitis and intraocular inflammation continue to be generally in line with those reported in studies of other intravitreal therapies. No events of retinal vasculitis or retinal vein occlusion were observed. The combined rate of new-onset exudations at month 18 was 9.3%, 6.2%, and 2.9% in the pegcetacoplan monthly, every-other-month, and sham groups, respectively. Detailed data will be presented at upcoming scientific meetings.
Read the full press release here: investors.apellis.com/news-releases/news-release-details/apellis-announces-pegcetacoplan-showed-continuous-and-clinically