Swedish Orphan Biovitrum (SOBI) Study Result summary

Study design and patient population

• Phase III VALIANT trial enrolled 124 patients with C3G or IC-MPGN, including adolescents and adults, both with native and transplanted kidneys.

• Randomized, double-blind design comparing pegcetacoplan to placebo over 26 weeks.

• VALIANT is the largest trial in C3G and IC-MPGN, enrolling patients aged 12+.

Efficacy results

• Pegcetacoplan achieved a statistically significant 68.3% reduction in proteinuria at week 26 versus placebo (p<0.0001).

• Results were consistent across disease subtypes, age groups, and both native and post-transplant kidney disease.

• Key secondary endpoints met, including significant improvements in composite renal outcomes and ≥50% proteinuria reduction.

• Pegcetacoplan led to greater reduction in C3c kidney staining and stabilized eGFR compared to placebo.

Safety and tolerability

• Pegcetacoplan demonstrated a favorable safety profile, with adverse event rates similar to placebo and no serious infections.

• No cases of meningitis or serious infections due to encapsulated bacteria were observed.