Apellis Pharmaceuticals Inc. announced the release of phase 3 post-hoc analyses from the phase 3 OAKS and DERBY studies assessing Syfovre (pegcetacoplan injection) for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
Give me a refresh on Syfovre.
Syfovre contains pegcetacoplan, which has been investigated as a targeted C3 inhibition therapy.
It works by regulating excessive activation of the complement cascade, which could lead to the onset and progression of diseases.
The formulation is designed as a 15 mg/0.1 ml dose to be administered by intravitreal injection once every 25 to 60 days into each affected eye to reduce the progression of GA.
See here for a visual.
Of note, the FDA approved Syfovre for commercial distribution in February 2023 as the first and only approved therapy for GA.
What did the trials show?
Investigators in the DERBY and OAKS studies compared Syfovre with the use of sham injections by tracking the efficacy of monthly and bimonthly Syfovre in patients with GA assessed by change in the total area of GA lesions from baseline measured by fundus autofluorescence at 12 months. Patients continued to receive masked treatment for 24 months
Both monthly and bimonthly injections showed a clinically meaningful reduction—36% in DERBY and 29% in OAKS—in GA lesion growth when compared to the sham injections from months 18 to 24.
How about these analyses?
Using Spectralis optical coherence tomography (OCT), Syfovre was found to provide visual function (5.6-letter preservation) and quality-of-life benefits (4.1 points, considered clinically meaningful) in patients with extrafoveal lesions (≥0.25 mm from the foveal center).
A meaningful reduction in both photoreceptor and retinal pigmented epithelial (RPE) cell loss was also observed.
Give me some more numbers.
For every-other-month injections of Syfovre, 46% of patients in both the DERBY and OAKS trials experienced photoreceptor cell loss; 20% (OAKS) and 21% (DERBY) experienced a clinically meaningful reduction in RPE cells.
For monthly injections, 53% (OAKS) and 47% (DERBY) experienced a clinically meaningful reduction in photoreceptor cells; and 22% (OAKS) and 27% (DERBY) experienced a loss of RPE cells.
Any limitations to note?
According to the company, due to the sample size, every-other-month and monthly data of both trials were combined for both the Syfovre and sham groups.
Significance of this data?
According to Allen Chiang, MD, study author, the post-hoc analysis data supports earlier treatment with Syfovre.
“Syfovre is a game changer for GA as the first and only treatment for this relentless disease,” stated Caroline Baumal, MD, Apellis CMO,” and we look forward to exploring its potential to treat other complement-driven retinal diseases with significant unmet needs.”