Phase 3 Trial of HUS Drug Begins in Europe

An international Phase 3 study of Inmunova’s INM004 is underway across Europe and Argentina to test whether the investigational therapy can safely treat hospitalized children with haemolytic uraemic syndrome. The trial matters because HUS can cause kidney failure, long-term complications and death, and no approved specific treatment exists worldwide.

Why it matters: - Haemolytic uraemic syndrome, or HUS, can trigger acute kidney injury in children and lead to chronic kidney problems, hypertension, neurological damage and death. - A successful treatment could move care beyond supportive measures and give doctors a targeted therapy for a disease with no approved specific drug worldwide. - The trial focuses on hospitalized paediatric patients, the group most vulnerable to severe outcomes from STEC-HUS.

What happened: - A Phase 3 clinical study of INM004 is underway in Belgium, France, Germany, Italy, Romania and the United Kingdom. - The international multicentre trial is also running at 20 centres in Argentina and 23 healthcare centres in Europe. - INM004 is an investigational drug developed by Inmunova, an Argentine biotechnology company and member of Insud Pharma. - The study is testing the efficacy and safety of INM004 in hospitalized paediatric patients with a clinical diagnosis of HUS.

The details: - HUS is primarily caused by eating food or drinking liquids contaminated with Shiga toxin-producing Escherichia coli, or STEC. - The therapy contains specific polyclonal antibodies against Shiga toxin. - Those antibodies are designed to recognise and neutralise different toxin variants. - HUS is one of the leading causes of acute kidney injury in the paediatric population. - The disease can be fatal in about 3% of cases. - Symptoms that require urgent medical attention include bloody or non-bloody diarrhoea, severe abdominal pain, paleness, fatigue and reduced urination. - The study was authorized by Argentina’s ANMAT, the European Medicines Agency and the UK’s Medicines and Healthcare products Regulatory Agency. - INM004’s clinical development program has also been endorsed by the FDA and the EMA. - The FDA and the EMA granted INM004 Orphan Drug designation. - The FDA also granted INM004 Rare Pediatric Disease status. - Before Phase 3, INM004 completed Phase 1 in healthy adult volunteers and Phase 2 in paediatric HUS patients. - Phase 1 showed an adequate safety profile for its pharmacological class. - Phase 2 showed an adequate safety profile and signs of potential usefulness against the disease. - The Phase 1 findings were published in the British Journal of Clinical Pharmacology. - The Phase 2 findings were published in Pediatric Nephrology.

Between the lines: - Doctors involved in the program frame HUS as a time-sensitive disease in which early detection can change outcomes. - Dr. Gianluigi Ardissino said HUS is the most important cause of acute renal failure in previously healthy children and that current management is supportive care only. - Dr. Ramona Stroescu said STEC-HUS can quickly become systemic, with severe cases involving neurological, cardiac or pancreatic complications. - Dr. Franz Schaefer said children often reach tertiary centres with severe disease and that dialysis is the only acute option today. - Those comments underscore the gap between current care and the kind of targeted therapy INM004 is trying to test.

What's next: - The Phase 3 trial will continue collecting efficacy and safety data from hospitalised paediatric patients. - Results will determine whether INM004 can progress closer to becoming a specific treatment for HUS. - If the data are positive, the program could support a shift from supportive care toward evidence-based targeted treatment.

The bottom line: - INM004 is now in its final clinical testing stage, and the study could help define the first targeted therapy for a serious paediatric disease with no approved specific treatment.