AstraZeneca Plc’s drug Ultomiris met a primary endpoint in a late-stage trial for the rare kidney disease IgA nephropathy, showing a statistically significant reduction in urine protein at 34 weeks.
"The interim I CAN results demonstrate that blocking terminal complement activation... may play a promising role in reducing proteinuria," Jonathan Barratt, a professor at the University of Leicester and an investigator on the I CAN trial, said.
In the prespecified interim analysis of the Phase 3 trial, the reduction in proteinuria was observed as early as week 10. The trial will continue toward its other primary endpoint, which is the change in kidney function as measured by eGFR at week 106. The safety profile was consistent with previous studies, with no new concerns identified.
The positive data moves AstraZeneca a step closer to competing with Travere Therapeutics Inc.’s Filspari, which gained US approval for IgA nephropathy in February 2023. With more than 560,000 people diagnosed with IgAN in the US, Europe and Japan, the market is a significant opportunity for drugmakers focused on rare diseases.
Go Deeper IgA nephropathy, or IgAN, is a progressive inflammatory kidney disease caused by the buildup of abnormal IgA proteins. This deposition activates the body’s complement system, leading to inflammation that damages the kidneys' ability to filter blood and can ultimately lead to end-stage kidney disease requiring dialysis or transplant.
Ultomiris, known chemically as ravulizumab, is a C5 complement inhibitor. It works by blocking the terminal complement cascade, a part of the immune system that, when overactivated, can cause the body to attack its own cells. The drug is already approved to treat several other rare blood and neurological disorders.
What's Next AstraZeneca said it plans to file the interim data with regulatory authorities to seek accelerated approval in key markets.
The success is a key development for AstraZeneca’s rare disease unit, Alexion, as it seeks to expand the applications for Ultomiris. The full data from the I CAN trial, expected at week 106, will be crucial for securing full approval and for competing on long-term kidney function preservation. Investors will be watching for regulatory filings and the potential market entry of a new major competitor in the IgAN space.
This article is for informational purposes only and does not constitute investment advice.
