Apellis Pharmaceuticals (APLS) Study Result summary

Study design and patient population

• Phase 3 VALIANT study enrolled 124 patients aged 12+ with C3G or primary IC-MPGN, rare kidney diseases affecting about 5,000 people in the US and 8,000 in Europe.

• Randomized, double-blind, placebo-controlled, multi-center trial included both adolescents and adults, with native and post-transplant kidney disease.

• 124 participants were randomized to receive pegcetacoplan or placebo plus standard of care for 26 weeks, followed by an open-label period.

• Patient demographics were similar between treatment arms; average age was 26.

• Patients on pegcetacoplan had slightly worse disease at baseline.

Efficacy results

• Pegcetacoplan achieved a 68% reduction in proteinuria at week 26 versus placebo (p<0.0001), meeting the primary endpoint.

• Consistent efficacy across all subgroups: C3G, IC-MPGN, adolescents, adults, native kidney, and transplant patients.

• Statistically significant improvements on key secondary endpoints, including composite renal endpoint and ≥50% proteinuria reduction.

• Nominally significant improvements in C3c staining and eGFR stabilization.

• Pegcetacoplan showed rapid and sustained effects, with improvements seen within four weeks.

Safety and tolerability

• Pegcetacoplan demonstrated a favorable safety and tolerability profile, consistent with previous data.

• Rates of adverse events, serious adverse events, and discontinuations were similar between treatment and placebo groups.

• No cases of meningitis or serious infections attributed to encapsulated bacteria.

• All patients completing the study enrolled in the VALE long-term extension study.