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Abstract: TH-PO0483

Efficacy and Safety of Iptacopan in East Asian Patients with IgAN: Final 24-Month Results from the Phase 3 APPLAUSE-IgAN Trial

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

  • Zhang, Hong, Peking University First Hospital, Beijing, China
  • Barratt, Jonathan, The Mayer IgA Nephropathy Laboratories, University of Leicester, Leicester, United Kingdom
  • Perkovic, Vlado, University of New South Wales, Sydney, New South Wales, Australia
  • 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, Buenos Aires, Argentina
  • Maes, Bart D., AZ Delta, Roeselare, Belgium
  • Desai, Manasi Mital, Novartis Pharmaceuticals UK Ltd, London, England, United Kingdom
  • Attari, Zenab, Novartis Pharmaceuticals Limited, Hyderabad, Telangana, India
  • Jacinto-Sanders, Severina, Novartis Pharma AG, Basel, Switzerland
  • Magirr, Annabel Rose, Novartis Pharma AG, Basel, Switzerland
  • Govan, Lindsay, Novartis Pharma AG, Basel, Switzerland
  • Renfurm, Ronny, Novartis Pharma AG, Basel, Switzerland
  • Hach, Thomas, Novartis Pharma AG, Basel, Switzerland
  • Kashihara, Naoki, Kawasaki Medical School, Okayama, Japan
Background

IgAN, the most prevalent form of primary glomerulonephritis, carries a high risk of kidney failure in East Asian patients (pts). In the Ph3 APPLAUSE–IgAN trial, the complement factor B inhibitor iptacopan significantly improved eGFR slope vs placebo (PBO), reducing the rate of eGFR decline by ≈50% over 2yrs with a favorable safety profile. Here we report final data in East Asian pts.

Methods

APPLAUSE–IgAN (NCT04578834) was a randomized, double–blind, PBO–controlled, multicenter trial in adults with biopsy–confirmed IgAN and proteinuria ≥1 g/g despite optimized supportive care. This exploratory subgroup analysis included 182 East Asian pts (iptacopan 200 mg bid n=90; PBO n=92). Over 24 months, endpoints included annualized eGFR slope (primary endpoint); change in eGFR; 24–hr UPCR < 1g/g and first–morning void (FMV) UPCR; and adverse events (AEs).

Results

Baseline characteristics were balanced between treatment arms. Over 24 months, iptacopan-treated pts had a statistically significant slower eGFR (mL/min/1.73 m2/yr) decline vs PBO; eGFR slope was −2.94 vs −5.82 (difference: 2.88 [95% CI: 1.30, 4.46]; P=0.0002; 1A) and mean change in eGFR from baseline to Month 24 (M24) was −4.50 vs −9.91 (difference: 5.41 [2.12, 8.71]; P=0.0006). At M24, proportion of pts achieving 24–hr UPCR < 1g/g was higher with iptacopan (53.8%) vs PBO (22.0%); OR 5.56 (95% CI: 2.60, 11.86). Iptacopan reduced FMV UPCR by 48.9% vs PBO (95% CI: 33.5, 60.7; 1B). AE rates were similar (iptacopan: 92.2% vs PBO: 89.1%); most were mild to moderate.

Conclusion

In East Asian pts with IgAN, iptacopan improved kidney function, as shown by statistically significant and clinically meaningful improvement in total eGFR slope vs PBO and proteinuria reductions over 24 months, with a favorable safety profile. These findings were consistent with the overall APPLAUSE–IgAN population and support the clinical utility of iptacopan in pts from East Asia.

Acknowledgment

Professional medical writing assistance was provided by Nupur Chaubey (Novartis India) and Susan Simpson (Novartis Ireland) and funded by Novartis Pharma AG.

Funding

  • Commercial Support – Novartis Pharma AG