AstraZeneca (AstraZeneca (AZN)) just got some good news in its quest to treat a rare kidney disease. The company announced Monday that its drug Ultomiris (ravulizumab) met a prespecified interim endpoint in a Phase 3 trial for immunoglobulin A nephropathy (IgAN), a condition that can lead to kidney failure.
The study, called I CAN, showed that patients treated with Ultomiris had a 46.6% reduction in proteinuria — that's protein in the urine, a key marker of kidney damage — from baseline at week 34. The placebo group saw only a 5.6% reduction. The placebo-adjusted treatment effect was 43.4%, and the result was statistically significant (p<0.0001).
Proteinuria is a big deal in IgAN because it's a sign that the kidneys are leaking protein instead of filtering it properly. Lowering it is a major goal for treatments.
The benefit appeared early. At week 10, Ultomiris-treated patients already had a 36.7% reduction in proteinuria, compared to 8.5% for placebo. And the effect held steady through week 34. The company said the benefit was seen across different patient subgroups, including those with varying demographics, baseline clinical profiles, and disease severity.
Jonathan Barratt, a professor of renal medicine at the University of Leicester and an investigator in the trial, explained that terminal complement activation — part of the immune system — plays a big role in inflammation and kidney function decline in IgAN. The interim findings suggest that blocking that pathway with Ultomiris could be a disease-modifying approach for patients with this rare condition.
Gianluca Pirozzi, Senior Vice President and Head of Development, Regulatory and Safety at Alexion, AstraZeneca's rare disease unit, said the results showed an early and meaningful reduction in proteinuria, including in patients at higher risk of disease progression. He added that the findings support the role of terminal complement inhibition in IgAN and will help advance regulatory filings in key markets.
On the safety front, nothing new to worry about. The safety profile was consistent with what's already known about Ultomiris, with no new safety concerns identified.
As for the stock, AstraZeneca shares were down 1.51% at $183.14 at the time of publication on Monday, according to market data.
These results are a positive step for patients with IgAN, a disease that currently has limited treatment options. If Ultomiris continues to perform well, it could become an important new therapy.
