AstraZeneca says its drug Ultomiris has delivered a statistically significant reduction in proteinuria in adults with immunoglobulin A nephropathy (IgAN), according to an interim analysis of its Phase III I CAN trial.

 

At 34 weeks, Ultomiris achieved a 43.4% placebo-adjusted reduction in 24-hour urine protein creatinine ratio (UPCR) versus placebo, meeting the study’s key interim endpoint.

 

The company said: “Ultomiris demonstrated a 46.6% reduction in 24-hour UPCR from baseline at week 34, compared to 5.6% in patients receiving placebo, resulting in a placebo-adjusted treatment effect of 43.4%.”

 

A rapid response was also observed early in treatment, with reductions seen by week 10 and sustained through week 34. Researchers reported consistency across patient subgroups, including those at higher risk of disease progression.

 

Jonathan Barratt, an investigator on the trial, said:.“For patients with IgAN, terminal complement activation is a key driver of inflammation and progressive loss of kidney function, which can frequently result in end-stage kidney disease. 

 

"These interim results show that by targeting the terminal complement pathway, Ultomiris delivered a rapid and significant reduction in proteinuria supporting its potential as a disease-modifying treatment for people living with this devastating rare disease.”

 

Safety findings were in line with the known profile of Ultomiris, with no new safety signals reported. The most common adverse events included upper respiratory tract infection, nasopharyngitis, and infusion-related reactions.

 

Gianluca Pirozzi, of Alexion, AstraZeneca Rare Disease, said: “These interim data demonstrate an early and significant reduction in proteinuria with benefit seen across subgroups, including patients who are at higher risk of disease progression. 

 

"Results underscore the important role of terminal complement inhibition in treating IgAN and highlight the potential of Ultomiris as a meaningful new treatment to address the unmet needs of this patient population. We look forward to advancing regulatory filings for approval in key markets, based on these interim data.”