Efficacy of Continuous Pegcetacoplan Treatment for Subfoveal Geographic Atrophy in Age-Related Macular Degeneration: 36-Month Results from OAKS, DERBY, and GALE Open-Label Extension.

PURPOSE: To report efficacy of intravitreal pegcetacoplan treatment over 36 months in eyes with subfoveal geographic atrophy (GA).

PATIENTS AND

METHODS

The GALE (NCT04770545) open-label extension trial adds 12 months of results to the 24-month Phase 3 OAKS (NCT03525613) and DERBY (NCT03525600) trials, representing up to 36 months of continuous pegcetacoplan treatment. They included a heterogeneous population of eyes with subfoveal GA (63%). Pegcetacoplan-treated eyes enrolling in GALE continued at the same interval of pegcetacoplan monthly (PM) or every other month (PEOM). Patients' eyes in sham monthly or every-other-month arms crossed over to receive pegcetacoplan in GALE at the same interval (sham crossover). Consequently, projected sham, calculated from prior 24-month GA growth rate of sham-observed eyes in OAKS and DERBY averaged across four 6-month segments, was the comparator for the first 12 months of GALE (months 24-36). This analysis reports results of eyes with subfoveal GA at baseline.

RESULTS

In eyes with subfoveal GA, 84% had best corrected visual acuity (BCVA) ≥20/200 and 38% had BCVA ≥20/63 at OAKS and DERBY baseline. Pegcetacoplan reduced subfoveal GA growth rate by 21% (p<0.0001) with PM and 19% (p=0.0001) with PEOM over 36 months. Increasing efficacy over time was noted between months 24 and 36; 31% reduction in subfoveal GA growth rate with PM and 25% reduction with PEOM (both p<0.0001) compared with projected sham. Microperimetry demonstrated significant reduction in formation of absolute scotomas with PM at 24 months (-2.5 number of scotomas formed; 95% confidence interval [CI]: -4.5, -0.4; p=0.0205) and 36 months (-4.0 number of scotomas formed; 95% CI: -6.8, -1.2; p=0.0050), compared to sham crossover in subfoveal GA. Safety profile in GALE was consistent with OAKS and DERBY.

CONCLUSION

Long-term efficacy of pegcetacoplan in slowing GA progression was demonstrated over 36 months in eyes with subfoveal GA.