- Design
- Open-label extension of phase 3 RCTs (GALE after OAKS and DERBY)
- Population
- Eyes with geographic atrophy, early vs delayed (24 months) pegcetacoplan
- Primary outcome
- GA growth, retinal tissue preserved, progression to scotoma
- Effect
- Growth reduced up to 24% vs projected sham; non-subfoveal tissue preserved 3.16 vs 1.11 mm²
GALE was the open-label extension of the phase 3 OAKS and DERBY trials of pegcetacoplan for geographic atrophy. Eyes treated from the start had 48 months of pegcetacoplan; sham eyes crossed over at 24 months.
Over 48 months, continuous treatment reduced lesion growth by up to 24% against projected sham, equivalent to 1.88 mm² of retina. In non-subfoveal atrophy, early treatment preserved up to 3.16 mm² against 1.11 mm² for delayed treatment. The risk of progressing to absolute scotoma at the central 4 and 16 test points was reduced by 32% and 43%. Safety was consistent with the parent trials.
Pegcetacoplan slows anatomical growth; functional benefit has been harder to show. The comparison with projected sham and the delayed-start design are weaker than the original randomised comparison.
- Discuss complement inhibition early with patients whose atrophy spares the fovea
- Explain that treatment slows growth rather than restoring vision
- Monthly or every-other-month injections are required indefinitely
- Monitor for conversion to neovascular AMD, a known risk with complement inhibitors
- Its licensing and availability in India are not established
Why it matters
It suggests the window for treatment is before the fovea is involved.
Don't overread it
Open-label extension data compared with projected sham; functional benefit remains modest.
The statistics, in plain English
Comparisons against projected sham use a model of what would have happened, not a concurrent control group, which makes them less reliable than the original randomised phase.
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