AstraZeneca PLC's (LSE:AZN, NASDAQ:AZN) Ultomiris has met its primary endpoint in a phase III trial targeting immunoglobulin A nephropathy (IgAN), a rare inflammatory kidney disease that can progress to kidney failure.
The results showed a statistically significant reduction in proteinuria, the presence of excess protein in urine that indicates kidney damage.
The interim results from the I CAN trial showed Ultomiris, known generically as ravulizumab, reduced proteinuria based on a 24-hour urine protein creatinine ratio (UPCR) at week 34, with reductions observed as early as week 10.
IgAN affects more than 560,000 people across the US, EU and Japan and occurs when abnormal proteins trigger immune complexes that deposit in the kidneys, activating the body's complement system, a branch of the immune response, and driving inflammation that progressively damages kidney tissue.
Ultomiris works by blocking the C5 protein in the terminal complement cascade, the final stage of this immune response, preventing the body from attacking its own kidney cells.
Jonathan Barratt, professor of renal medicine at the University of Leicester and a trial investigator, said many patients with IgAN continue to progress to kidney failure despite advances in care, and described the results as promising.
Marc Dunoyer, chief executive of Alexion, AstraZeneca Rare Disease, said the company intends to file the data with regulatory authorities in key markets and will seek accelerated approval.
The safety profile was consistent with Ultomiris's established record, with no new concerns identified.
The trial's second primary endpoint, measuring the rate of kidney filtration at week 106, will be assessed at the final analysis when the full study completes.