AstraZeneca and Alexion Report Positive Phase III Results for Ultomiris in IgA Nephropathy Treatment

Photo by Anthony Devlin/Getty Images for AstraZeneca

(IN BRIEF) AstraZeneca and Alexion have reported positive interim Phase III results for Ultomiris in treating immunoglobulin A nephropathy, showing a significant reduction in proteinuria compared to placebo. The therapy demonstrated rapid effects as early as week 10 and met its primary endpoint at week 34, highlighting its potential as a disease-modifying treatment. IgAN is a progressive kidney disease that can lead to kidney failure, and current treatment options remain limited for many patients. The study’s findings support the role of complement inhibition in reducing inflammation and improving outcomes. With a consistent safety profile and ongoing evaluation of long-term kidney function, the companies plan to pursue regulatory submissions and further validate Ultomiris as a treatment option for this condition.

(PRESS RELEASE) CAMBRIDGE, 21-Apr-2026 — /EuropaWire/ — AstraZeneca and its rare disease division Alexion have reported positive interim results from the Phase III I CAN clinical trial evaluating Ultomiris (ravulizumab) in adults with immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The findings show that the therapy achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in proteinuria at week 34, measured by 24-hour urine protein creatinine ratio.

The results indicate that Ultomiris delivered a rapid effect, with reductions in proteinuria observed as early as week 10. The study continues, with the second primary endpoint—change in estimated glomerular filtration rate (eGFR), a key measure of kidney function—set to be evaluated at week 106.

IgAN is a rare and progressive kidney disease caused by abnormal immune responses, where IgA protein deposits trigger inflammation and damage in the kidneys. Over time, this can lead to chronic kidney disease and, in severe cases, end-stage kidney disease requiring dialysis or transplantation. The condition affects more than half a million people across major global markets, with a significant proportion at risk of progression.

Jonathan Barratt, a lead investigator in the trial and Professor of Renal Medicine at the University of Leicester, noted that despite advancements in care, many patients continue to experience disease progression. He highlighted that targeting the complement system—a central driver of inflammation in IgAN—may offer a new approach to reducing proteinuria and potentially altering disease outcomes.

Marc Dunoyer, Chief Executive Officer of Alexion, emphasized that the findings reinforce the potential of terminal complement inhibition as a disease-modifying strategy. He pointed to the rapid and sustained reduction in proteinuria as an encouraging sign for future treatment options and confirmed plans to submit the data to regulatory authorities in key markets.

The safety profile observed during the trial was consistent with previous experience of Ultomiris, with no new safety concerns identified. The company also intends to present the full data set at an upcoming scientific meeting.

The I CAN trial is a global, randomized, double-blind, placebo-controlled study involving approximately 510 participants across 28 countries. Patients received either Ultomiris or placebo in addition to standard therapy over a 106-week period, with dosing administered intravenously at regular intervals following an initial loading dose.

Ultomiris is a long-acting complement C5 inhibitor designed to provide sustained suppression of the complement cascade, a component of the immune system that, when overactivated, can damage healthy tissues. The therapy is already approved for several rare conditions, including paroxysmal nocturnal haemoglobinuria, atypical haemolytic uraemic syndrome, generalized myasthenia gravis, and neuromyelitis optica spectrum disorder.

The broader clinical development programme for Ultomiris continues to explore its potential across multiple immune-mediated diseases, reflecting ongoing efforts to address significant unmet medical needs in nephrology and immunology.

SOURCE: AstraZeneca