2-year results: Demonstrated efficacy in both monthly and EOM dosing1,2
- Quick links:
- OAKS Efficacy
- DERBY Efficacy
- Subpopulation Data
- Time to Event
REDUCTION IN LESION GROWTH RATE WITH SYFOVRE VS SHAM POOLED1,2*
*Slope for baseline to Month 24 is an average of slope of baseline to Month 6, Month 6 to Month 12, Month 12 to Month 18, and Month 18 to Month 24.1
Based on a mixed effects model for repeated measures assuming a piecewise linear trend in time with knots at Month 6, Month 12, and Month 18.1
REDUCTION IN LESION GROWTH RATE WITH SYFOVRE VS SHAM POOLED1,2†
†Slope for baseline to Month 24 is an average of slope of baseline to Month 6, Month 6 to Month 12, Month 12 to Month 18, and Month 18 to Month 24.1
Based on a mixed effects model for repeated measures assuming a piecewise linear trend in time with knots at Month 6, Month 12, and Month 18.1
Demonstrated results in both monthly and EOM dosing1
Combined piecewise linear analysis did not have a prespecified statistical procedure controlling for type 1 error
See full OAKS and DERBY trial design
Contextual Information
- The prespecified key secondary endpoints on functional measures analyzed in OAKS and DERBY were BCVA reading speed, functional reading independence index, and microperimetry mean sensitivity*
- There was no statistically significant difference between the SYFOVRE arms vs sham pooled in the functional outcomes measured at 2 years
- Functional outcomes declined similarly in all 3 treatment groups over 2 years, consistent with the natural history of the disease
- The time to severe visual impairment analysis is a post hoc subgroup analysis that is not prespecified with no statistical testing hierarchy. No conclusions can be drawn from this analysis
Post Hoc Analysis Objective
- Assess the impact of SYFOVRE treatment vs sham on severe visual impairment, defined as NL-BCVA of less than 35 ETDRS letters in a post hoc analysis
Methodology
- Subgroup analysis of pooled OAKS and DERBY patients without severe visual impairment at baseline
- An event of severe visual impairment was defined as the first time a subject experienced a sustained reduction below 35 ETDRS letters for at least 4 months postbaseline without later recovery to above ≥40 letters at a subsequent assessment
- Subject without an event are censored at their last postbaseline BCVA assessment
TIME TO SEVERE VISUAL IMPAIRMENT AS BCVA <35 ETDRS LETTERS (20/200 SNELLEN EQUIVALENT) IN PATIENTS WITHOUT SEVERE VISUAL IMPAIRMENT AT BASELINE2,4
The time to severe visual impairment analysis is a post hoc subgroup analysis that is not prespecified with no statistical testing hierarchy. No conclusions can be drawn from this analysis.
Hazard ratio estimated from Cox Proportional Hazards model including patients in the modified intent-to-treat population at-risk for the event (BCVA ≥35 letters at baseline) with at least 1 postbaseline assessment. Model includes study (APL2-303 or APL2-304) + baseline GA lesion area (<7.5 mm2 or ≥7.5 mm2) + baseline presence of choroidal neovascularization in the fellow eye (Yes or No) + baseline BCVA.
*Microperimetry mean sensitivity studied in OAKS only.2
AMD=age-related macular degeneration; BCVA=best-corrected visual acuity; EOM=every other month; ETDRS=Early Treatment Diabetic Retinopathy Study; FAF=fundus autofluorescence; GA=geographic atrophy; NL-BCVA=normal luminance best-corrected visual acuity.
References: 1. SYFOVRE (pegcetacoplan injection) [package insert]. Waltham, MA: Apellis Pharmaceuticals, Inc.; 2024. 2. Data on file. Apellis Pharmaceuticals, Inc. 3. Fernández EJ, Villa-Carpes JA, Martínez-Ojeda RM, Ávila FJ, Bueno JM. Retinal and choroidal thickness in myopic young adults. Photonics. 2022;9(5):328. 4. Patel PJ, Chen FK, Rubin GS, Tufail A. Intersession repeatability of contrast sensitivity scores in age-related macular degeneration. Invest Ophthalmol Vis Sci. 2009;50(6):2621-2625. doi: 10.1167/iovs.08-2407.
