Novartis' Investigational Therapy Shows Promising Action In Rare Kidney Disease

Novartis AG NVS announced topline results from the pre-specified interim analysis of the Phase 3 APPLAUSE-IgAN study at nine months of iptacopan, an investigational factor B inhibitor targeting the alternative complement pathway.

The study demonstrated superiority over placebo in reducing proteinuria (protein in urine). It provided a clinically meaningful and statistically significant proteinuria reduction on top of supportive care in patients with IgA nephropathy (IgAN), a complement-mediated disease. 

In the study, the safety profile of iptacopan (200 mg twice daily) was consistent with previously reported data.

The study continues to evaluate iptacopan’s ability to slow IgAN progression by measuring the estimated glomerular filtration rate (eGFR) slope over 24 months – the primary endpoint at the study end with topline results expected in 2025. 

It is estimated that approximately 25 people per million worldwide are newly diagnosed with IgAN each year. Up to 30% of people with IgAN with higher proteinuria (≥1 g/day) may progress to kidney failure within ten years.

Iptacopan is under review by regulators following Phase 3 results in paroxysmal nocturnal hemoglobinuria. 

Iptacopan is also being investigated in Phase 3 studies for C3 glomerulopathy, atypical hemolytic uremic syndrome, and immune complex membranoproliferative glomerulonephritis. 

With the recent acquisition of Chinook Therapeutics, the Novartis renal portfolio expands with two additional late-stage medicines developing for IgAN, complementing the existing pipeline. 

Novartis intends to submit for possible accelerated approval with the FDA in 2024. 

Price Action: NVS shares are down 0.30% at $101.55 during the premarket session on the last check Monday.