Novartis Reports Positive Interim Results from Phase III APPLAUSE-IgAN Trial of First-in-Class Complement Therapy Iptacopan in IgA Nephropathy, Plans to Seek Accelerated Approval

On October 2, Novartis announced positive key results from the pre-specified interim analysis of the Phase III APPLAUSE-IgAN study at the 9th month. The specific complement B factor inhibitor Iptacopan showed superiority over placebo in reducing proteinuria (protein in urine) and provided clinically meaningful and highly statistically significant reduction in proteinuria for patients with IgA nephropathy (a complement-mediated disease) on top of supportive care. In this study, the safety profile of iptacopan (200mg, twice daily) was consistent with previously reported data. Novartis plans to seek accelerated approval from the FDA based on the interim data in 2024.

The study will continue in a double-blind manner, assessing iptacopan's ability to slow IgAN progression by measuring the slope of estimated glomerular filtration rate (eGFR) over 24 months (the primary endpoint at the end of the study), with key results expected in 2025.

It is estimated that approximately 25 people per million are newly diagnosed with IgAN worldwide each year. Up to 30% of IgAN patients with persistent high levels of proteinuria (≥1g/day) may progress to kidney failure within 10 years. Clinically, there is a need for effective targeted therapies for IgAN to slow or prevent the progression to kidney failure. Although current supportive treatments are helpful, none address the key pathogenic step in IgAN progression — the activation of the complement system.

Iptacopan was discovered and developed by Novartis to treat IgAN and other complement-mediated diseases by inhibiting Factor B, a protease essential for the alternative complement pathway.

The marketing application for iptacopan in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) is currently under regulatory review. In addition, iptacopan is presently being evaluated in pivotal studies for a range of complement-mediated diseases (CMDs), including renal diseases such as C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), membranous nephropathy (MN), lupus nephritis (LN), as well as immune thrombocytopenic purpura (ITP) and cold agglutinin disease (CAD).

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