The Research Institute of Hospital Universitari Vall d'Hebron, Spain, and Ocera Therapeutics have reported the preliminary data from the first part of the Phase 2 study evaluating the OCR-002 (ornithine phenylacetate) drug, used for the treatment of cirrhosis of the liver and upper gastrointestinal (GI) bleeding.
Developed by Ocera Therapeutics, OCR-002 is an injectable agent that removes toxic, circulating ammonia from the blood to treat or prevent hepatic encephalopathy (HE), a neuropsychiatric complication of acute liver failure associated with increased levels of circulating ammonia.
OCR-002 works by lowering circulating blood levels of ammonia, while enabling alternate metabolic pathways in the muscle and kidney in patients with liver failure.
The ongoing Phase 2 clinical trial will enrol 48 patients with cirrhosis of the liver and the completed open-label first phase of the study confirmed the overall effectiveness, safety and tolerability of OCR-002.
The second phase is a double-blind placebo-controlled study that will measure the primary endpoint of ammonia plasma concentration, with improvement in HE as a secondary endpoint.
The data from the study of upper GI bleed in patients with liver cirrhosis demonstrated that OCR-002 is well tolerated up to 10g in 24 hours and provides a rapid and durable ammonia reduction to normal levels after 36 hours of treatment.
Hospital Universitari Vall d'Hebron spokesperson and lead investigator of the study, Juan Cordoba, said the early data showed that OCR-002 is well tolerated, rapidly normalises hyperammonemia and has the potential to prevent the development of hepatic encephalopathy.
"Cirrhosis patients that present with acute upper gastrointestinal bleeding have a 30% chance of developing hepatic encephalopathy and require intensive care," Cordoba added.
"A medicine that can quickly remove the toxic ammonia may help prevent or reverse this life-threatening condition, minimise the need for intensive care and shorten hospitalisation."
The findings from the trial were presented at the 15th International Symposium for Hepatic Encephalopathy and Nitrogen Metabolism (ISHEN) in Grenaa, Denmark.