Iptacopan in Patients With IgA Nephropathy From East Asia.

Introduction: IgA nephropathy (IgAN) has a high risk of progression to kidney failure in East Asia. At the month 9 interim analysis (IA) of the APPLAUSE-IgAN trial, iptacopan, a complement factor B inhibitor, demonstrated a -38.3% change from baseline in 24-hour urine protein-to-creatinine ratio (UPCR) versus placebo and had a favorable safety profile. Considering the IgAN clinical burden in East Asia, we report APPLAUSE-IgAN IA findings from East Asian patients. Methods: APPLAUSE-IgAN was a phase 3 trial (NCT04578834) in adults with biopsy-confirmed IgAN and 24-hour UPCR ≥ 1 g/g despite optimized supportive care. Patients were randomized to iptacopan 200 mg or placebo twice daily. Interim efficacy was assessed in the first 250 patients who remained in or discontinued the trial by month 9; safety was assessed in 443 patients. In these exploratory analyses, efficacy was analyzed in 102 patients (51 per group) and safety in 177 patients (iptacopan: Results: In patients from East Asia, iptacopan led to adjusted geometric mean changes from baseline of -38.0% in 24-hour UPCR (95% confidence interval [CI]: -18.9% to -52.6%) and -36.4% in UPCR from first morning void (FMV) (95% CI: -17.1% to -51.3%) versus placebo at month 9. Iptacopan led to a greater improvement in hematuria than placebo. Treatment-emergent adverse events (TEAEs) occurred in a similar proportion of patients in both groups. Conclusions: In patients from East Asia in the APPLAUSE-IgAN main study population, iptacopan led to clinically meaningful proteinuria reductions and had a favorable safety profile, consistent with results from the global main study population.