An Argentine biotech company, Inmunova, is running a Phase 3 trial of an experimental antibody drug that could become the first specific treatment for hemolytic uremic syndrome (HUS), a childhood kidney disease.
Argentina has the world's highest incidence of HUS, with roughly 300 to 350 children developing it each year, according to Inmunova. It usually follows infection with Shiga toxin-producing E. coli bacteria, which live in cattle and reach people through undercooked ground beef or contaminated food and water. Children under five are the most vulnerable, and avoiding undercooked ground beef remains the main precaution.
Doctors today can only treat symptoms: fluids, transfusions, blood-pressure control and dialysis, which 40% to 50% of patients need during the acute phase. Inmunova estimates the disease kills about 3% of affected children, and its technical director, Vanesa Zylberman, says HUS is the leading cause of kidney transplants in adolescence. Antibiotics are avoided because they can stress the bacteria into releasing more toxin, she says.
Inmunova's drug, INM004, takes a different route. It is made of antibodies raised in horses and designed to bind the Shiga toxin before it reaches kidney cells, and is given intravenously once HUS is diagnosed. The Phase 3 trial, registered on ClinicalTrials.gov, is randomized and placebo-controlled: all of the planned 220 children, aged nine months to 17, receive standard care, and about half also receive INM004. It began in Argentina in October 2024 and has since added hospitals in eight European countries, including Spain, Germany, France and Britain.
Earlier phases tested safety in healthy adults and then in 57 children with HUS, with results published in the British Journal of Clinical Pharmacology and Pediatric Nephrology. Inmunova describes the signals as encouraging but says efficacy has not been proven. It says an independent review around the trial's midpoint found the study should continue, and that it expects Phase 3 to run about another year.
The EMA and the FDA have granted INM004 orphan-drug status, a designation for treatments of rare diseases. Regulators will weigh the evidence on safety and efficacy only after the trial ends, so no date for commercial availability exists. If approved, Zylberman says, it would be sold to hospitals rather than to patients.