A potential new treatment for geographic atrophy (GA) moved closer to regulatory approval with the announcement of positive top-line data from the phase 3 DERBY and OAKS trials of the C3 inhibitor pegcetacoplan (Apellis Pharmaceuticals Inc.) during the EURETINA 2021 Virtual Congress.
OAKS met the primary endpoint for both monthly and every-other-month treatment with pegcetacoplan, demonstrating a significant reduction in GA lesion growth of 22% and 16%, respectively, compared to pooled sham at 12 months.
DERBY did not meet the primary endpoint of GA lesion growth, showing a reduction of 12% and 11% with monthly and every-other-month treatment, respectively, compared to pooled sham at 12 months.
Both studies are evaluating intravitreal pegcetacoplan in 1,258 adults with geographic atrophy secondary to age-related macular degeneration (AMD).
In a prespecified analysis of the combined studies, pegcetacoplan decreased GA lesion growth in patients with extrafoveal lesions at baseline by 26% and 23% with monthly and every-other-month treatment, respectively.
Pegcetacoplan was well tolerated in both studies and no events of retinal vasculitis or retinal vein occlusion were observed. Two cases of confirmed infectious endophthalmitis and one case of suspected infectious endophthalmitis were observed in the study eye out of a total of 6,331 injections (0.047%)
Based on these results, Apellis plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.
“Positive news for patients” – Frank Holz
Commenting on the phase III results of pegcetacoplan, Frank Holz, President of EURETINA, said it was very positive news for patients and clinicians.
“Geographic atrophy is a leading cause of blindness which leads to irreversible vision loss and a decline in quality of life. I think it is extremely encouraging that this is the first positive phase 3 data that show efficacy and safety to treat patients with GA. We all know the enormous impact of GA on affected patients, so I think these results represent a really positive step forward,” he said.
Jordi Monés MD, PhD, Director of the Institut de la Màcula in Barcelona, agreed that the news was very encouraging.
“It is very difficult to obtain positive results in trials involving degenerative conditions like GA. If we can slow down the rate of progression of the disease and buy some valuable time for many of our elderly patients it will make a tremendous difference to their quality of life,” he said.
Ramin Tadayoni, EURETINA Board Member, said that a new treatment for GA would herald a shift in the way clinicians need to speak to their patients about their prognosis.
“It is really good that we will be able to change the way that we talk to our patients. Before, having to tell patients that we have no treatment is really a desperate scenario for them and for us. But once we have one approved treatment, everyone is much more optimistic that this disease will one day come under control. It is a very important step forward for our patients,” he said.