Novartis investigational iptacopan Phase III study demonstrates clinically meaningful and statistically significant proteinuria reduction in patients with C3 glomerulopathy (C3G)

Ad hoc announcement pursuant to Art. 53 LR

• Phase III APPEAR-C3G study met its primary endpoint, demonstrating superiority of iptacopan vs. placebo in proteinuria reduction at six-month analysis¹; the safety profile of iptacopan was consistent with previously reported data^1-3
• C3 glomerulopathy (C3G) is an ultra-rare complement-mediated kidney disease, with approximately 50% of patients progressing to kidney failure within 10 years of diagnosis^4-7; currently no treatments address the underlying cause of C3G^7-9
• Novartis plans to review results with global health authorities to enable potential submissions in 2024; data will be submitted for presentation at an upcoming medical meeting
• Iptacopan demonstrated positive Phase III results in IgA nephropathy (IgAN) at the interim analysis and a late-stage development program is ongoing across four investigational indications^10-14

Read full press release here