Already active across healthcare centres in Argentina, this pivotal study will assess the efficacy and safety of what could become the first specific medication to treat this disease.

Buenos Aires, 14 August 2026.- A Phase 3 clinical study of an investigational drug for haemolytic uraemic syndrome (HUS) is currently underway in Europe, including Belgium, France, Germany, Italy, Romania and the United Kingdom. HUS is a severe disease primarily caused by consuming food or liquids contaminated with Shiga toxin-producing Escherichia coli (STEC) bacteria. This condition can affect children of any age, particularly those under five, although it can also affect adults.

The investigational drug named INM004 was developed by Inmunova, an Argentine biotechnology company and member of the global firm Insud Pharma. It is a potential treatment designed to halt the progression of HUS and prevent its severe forms. This biologic drug contains specific polyclonal antibodies against the Shiga toxin, which causes HUS. These antibodies have the advantage of being broad-spectrum: they recognise and neutralise different variants of the toxin.

This international multicentre study is being conducted across 23 healthcare centres in Europe and 20 centres in Argentina. It will assess the efficacy and safety of the therapy in hospitalised paediatric patients with a clinical diagnosis of HUS. Currently, there is no approved vaccine or specific drug available worldwide to treat the condition.

HUS is one of the leading causes of acute kidney injury in the paediatric population. It can result in lifelong complications such as chronic renal problems, hypertension, and neurological impairments, and can be fatal in approximately 3% of cases. Given its severity, acting fast upon symptoms –including bloody or non-bloody diarrhoea accompanied by severe abdominal pain, paleness, fatigue, and reduced urination– makes an immediate visit to the nearest health centre crucial for early detection.

“Encouraging outcomes from Phase 1 and 2 clinical research have cleared the path to Phase 3 to evaluate the medication’s efficacy in treating HUS”, stated Dr. Ian Roubicek, the study’s medical coordinator. “As HUS lacks specific therapeutic options, we have received strong support from both the medical community and regulatory agencies throughout the process”.

The study was authorized by Argentina’s National Administration of Drugs, Foods and Medical Devices (ANMAT), the European Medicines Agency (EMA), and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). Additionally, the clinical development program of INM004 was endorsed by the U.S. Food and Drug Administration (FDA) and the EMA, with both regulatory bodies granting Orphan Drug designation to this investigational drug. Furthermore, INM004 received Rare Pediatric Disease status from the FDA.

«HUS is a life-threatening disease and the most important cause of acute renal failure in previously healthy children. Between 40% and 50% of patients require dialysis and long-term renal sequelae are common. Current management consists solely of supportive care. The treatment under evaluation represents a specific therapy that could modify the course of HUS and improve its prognosis”, said Dr. Gianluigi Ardissino, paediatric nephrologist and referent of the Center for the Treatment and Study of Hemolytic Uremic Syndrome at the Policlinico di Milano (Italy).

Dr. Ramona Stroescu, paediatric nephrology specialist at Spitalul Clinic de Urgență pentru Copii «Louis Țurcanu» (Timisoara, Romania) noted that “STEC-HUS primarily drives acute kidney injury, but it is a systemic disease; in severe cases, we see extrarenal involvement such as neurological, cardiac, or pancreatic complications. These patients can become critically ill very quickly, meaning early detection and prompt medical attention are paramount”. She added, “This clinical trial could help shift STEC-HUS management from supportive care to a targeted, evidence-based approach, potentially reducing both acute severity and long-term consequences”.

For his part Dr. Franz Schaefer, professor of Paediatrics and head of the Paediatric Nephrology Division at Universitätsklinikum Heidelberg AöR (Germany), remarked: «STEC-HUS is rare, but still the most common cause of acute kidney failure in children beyond the neonatal age. In our tertiary care centre we see a lot of children suffering from this disease; many have a severe course and the long-term outcomes are not always good. In the acute phase we cannot offer any treatment other than dialysis. This clinical trial evaluates an innovative therapeutic concept that may help mitigating the course of HUS in children”.

Before entering the final stage of clinical research, INM004 successfully completed the first two phases of clinical studies. In Phase 1, its safety was assessed in healthy adult volunteers and showed an adequate safety profile for its pharmacological class. In Phase 2, which included paediatric patients diagnosed with HUS, the treatment showed an adequate safety profile and provided signs of being potentially useful in treating the disease. The findings of both studies were published in British Journal of Clinical Pharmacology and Pediatric Nephrology, respectively.

About HUS

Haemolytic uraemic syndrome (HUS) is a serious disease, mainly caused by consuming food or liquids (such as meat, fruits, vegetables, dairy, or water) contaminated with Shiga toxin-producing Escherichia coli (STEC) bacteria, or through direct contact with contaminated faecal matter.

This toxin, which triggers HUS, causes damage to the kidneys and, in some cases, the brain and heart. It alters blood components, resulting in a reduction of red blood cells and platelets. The condition can lead to lifelong complications for approximately half of those affected, including hypertension, neurological impairments, and chronic renal problems, and can prove fatal in about 3% of cases.

HUS can affect children of any age, primarily those under five years of age, although adults can also be affected. In fact, children and the elderly are at a higher risk of developing severe forms of the disease.

In Europe, epidemiological data from the latest report by the European Food Safety Authority (EFSA) and the European Centre for Disease Prevention and Control (ECDC) indicate that in 2024 there were 11,738 confirmed cases of Shiga toxin-producing E. coli infection (a European Union notification rate of 3.5 cases per 100,000 population), resulting in 1,411 hospitalisations. There were 417 cases of HUS reported by 23 countries, resulting in 12 fatalities. ¹

Currently, there is no vaccine or specific drug approved worldwide for the treatment of STEC-HUS. It is treated with supportive care in a hospital setting.

Symptoms and prevention

HUS typically begins with bloody or non-bloody diarrhoea, accompanied by other symptoms such as severe abdominal pain, paleness, fatigue, and reduced urine output. When these symptoms occur, immediate medical attention at the nearest healthcare facility is essential.

As preventive measures, it is necessary to maintain strict hygiene and safe food-handling practices.

• Wash hands with soap and water: Do this before and after using the toilet, changing nappies, returning from outdoors, and handling raw food.
• Ensure meat is cooked completely, particularly minced meat and derived products
• Avoid minced meat for children under 5 years old.
• Use potable water: Ensure water for drinking and washing fruits or vegetables is safe. If safe water is unavailable, boil it or add two drops of unscented, food-grade bleach per litre and let it sit for 30 minutes.
• Consume pasteurised dairy: Avoid raw or unpasteurised milk and cheese products.
• Prevent cross-contamination: Keep raw meat strictly separate from all ready-to-eat foods.
• Sanitise kitchen surfaces: Wash utensils and worktops thoroughly after any contact with raw food.
• Swim in designated areas: Avoid bathing in potentially contaminated or unmonitored waters.

About Inmunova

Inmunova is an Argentine biotechnology company dedicated to the research and development of innovative drugs for rare infectious diseases with no treatment option. Founded by scientists with recognized expertise in biotechnology, it specializes in the design of vaccines, therapeutic antibodies and immunogens, covering the entire development process: from the original idea, proofs of concept, preclinical and clinical phases, to the registration procedure and pharmacovigilance. inmunova.com/en

Reference:
1. European Food Safety Authority, European Centre for Disease Prevention and Control. The European Union One Health 2024 Zoonoses Report. EFSA J. 2025 Dec 9;23(12):e9759. Doi: 10.2903/j.efsa.2025.9759 https://www.ecdc.europa.eu/sites/default/files/documents/EUOH-%202024-Zoonoses-report.PDF