Topline results from DERBY, OAKS may pave way to first treatment for geographic atrophy
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Topline results from the phase 3 DERBY and OAKS trials, released by Apellis Pharmaceuticals earlier this month, showed potential for pegcetacoplan to significantly reduce lesion growth in geographic atrophy.
“It is an extremely positive step forward. Pegcetacoplan may have the potential to become the first treatment available for this condition,” Frank G. Holz, MD, PhD, Euretina president, said at the virtual Euretina congress.
Pegcetacoplan is a C3 inhibitor designed to regulate overactivation of the complement cascade. The DERBY and OAKS trials investigated the safety and efficacy of this new compound in 1,258 patients with geographic atrophy (GA) secondary to AMD.
“OAKS was the first trial to meet its primary endpoint in a GA trial,” Holz said. Significant reduction in GA was achieved with both monthly and every other month dosing. Lesion growth was decreased by 22% and 16%, respectively, as compared with sham. DERBY narrowly missed its primary endpoint but showed a clear trend, consistent with OAKS and phase 2/3 studies.
“In a prespecified combined analysis of the data from the two trials, we see that both monthly and [every other month] dosing reduced lesion growth by 17% and 14%, respectively,” Holz said.
Subgroup analysis showed that patients who had the highest benefits from the treatment were those with extrafoveal lesions and foveal sparing at baseline. These patients exhibited consistent reduction in GA lesion growth, by 26% with monthly dosing and 23% with every other month administration.
“Taken together, these studies and the phase 2 FILLY study all demonstrated the impact of C3 inhibition with pegcetacoplan in the progression of GA,” Holz said.
The safety profile was favorable in both trials, he said. Exudative phenomena were observed in 6% and 4% of the monthly and every other month arms as compared with 2.4% in the sham group. Endophthalmitis rate was low, 0.047% in a total of 6,331 injections. There were 13 cases of intraocular inflammation but no events of retinal vasculitis or vascular occlusion.
“These data are highly encouraging. They are the first positive phase 3 data that show efficacy and safety to treat GA, a major unmet need in ophthalmology. This will have an enormous impact on affected patients,” Holz said.
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Holz FG. Extension to the presentation: Emerging clinical data in GA. Presented at: Euretina congress; Sept. 9-12, 2021 (virtual meeting). Satellite symposium sponsored by Apellis.
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