Abstract: FR-PO0811
Efficacy and Safety of Iptacopan in Patients (Pts) from East Asia with IgAN: Interim Results from the Phase 3 APPLAUSE-IgAN Trial
Session Information
- Glomerular Clinical Trials: From Data to Impact
November 07, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Zhang, Hong, Peking University First Hospital, Beijing, China
- Perkovic, Vlado, University of New South Wales, Sydney, New South Wales, Australia
- Barratt, Jonathan, The Mayer IgA Nephropathy Laboratories, University of Leicester, Leicester, United Kingdom
- Rizk, Dana V., University of Alabama at Birmingham, Birmingham, Alabama, United States
- Rovin, Brad, The Ohio State University Wexner Medical Center, Columbus, Ohio, United States
- Trimarchi, Hernan, Hospital Britanico de Buenos Aires, Buenos Aires, Argentina
- Maes, Bart D., AZ Delta, Roeselare, Belgium
- Merkel, Tobias, Novartis Pharma AG, Basel, Switzerland
- Desai, Manasi Mital, Novartis Pharmaceuticals Limited, London, United Kingdom
- Ansari, Soudeh, Novartis Pharmaceuticals Corporation, Cambridge, Massachusetts, United States
- Hach, Thomas, Novartis Pharma AG, Basel, Switzerland
- Renfurm, Ronny, Novartis Pharma AG, Basel, Switzerland
- Kashihara, Naoki, Kawasaki Medical School, Okayama, Japan
Background
Alternative complement pathway activation is a key aspect of IgAN pathogenesis. Iptacopan, a complement factor B inhibitor, received accelerated approval by the US FDA for primary IgAN based on a 24-hour urine protein–creatinine ratio (24h-UPCR) reduction of 38.3% vs placebo (pbo) at Month (M) 9 and a favorable safety profile in the APPLAUSE-IgAN interim analysis (IA). IgAN is the most prevalent glomerulonephritis and has a high risk of progression to kidney failure in pts from East Asia. We report IA results for APPLAUSE-IgAN pts enrolled from East Asia.
Methods
APPLAUSE-IgAN is a Phase III trial in pts with IgAN and 24h-UPCR ≥1 g/g despite optimized supportive care (NCT04578834). Pts were randomized to iptacopan 200 mg bid or pbo. An IA was conducted when 250 pts in the main study population remained in or discontinued the trial by the M9 visit (interim analysis full analysis set [IA-FAS]). The safety set (SAF) comprised pts who received ≥1 dose of study treatment. These analyses were predefined and exploratory.
Results
Efficacy was analyzed in 102 pts (IA-FAS; Chinese 40.2%; Korean 28.4%; Japanese 20.6%; Other 10.8%; 51 per arm) and safety in 177 pts (SAF; iptacopan: n=88; pbo: n=89) from East Asia. Iptacopan led to adjusted geometric mean reductions from baseline of 38.0% in 24h-UPCR (95% CI 18.9, 52.6; nominal P=0.0002) and 36.5% in UPCR from first morning void (UPCR-FMV; 95% CI 17.2, 51.3; nominal P=0.0004) vs pbo at M9. A reduction in UPCR-FMV was seen as early as Week 2 in the iptacopan arm (Figure). Treatment-emergent adverse events (TEAEs) occurred in 70.5% of pts on iptacopan and 71.9% on pbo. TEAEs led to treatment discontinuation in 1.1% of pts on iptacopan and 2.2% on pbo.
Conclusion
The efficacy and safety of iptacopan was consistent between pts from East Asia and the overall main study population in the APPLAUSE-IgAN IA.
Funding
- Commercial Support – Novartis