Orziloben Phase 2A Trial for IFALD Begins

NorthSea Therapeutics, a biotech firm specializing in innovative treatments for non-alcoholic steatohepatitis (NASH) and other metabolic liver diseases, has initiated a Phase 2a clinical trial for Orziloben (NST-6179) to treat intestinal failure-associated liver disease (IFALD), a condition affecting those on long-term intravenous nutrition. The study, involving up to 36 adults across North America, aims to assess Orziloben's safety, tolerability, pharmacokinetics, and pharmacodynamics, with results expected in the second half of 2025. This trial marks a significant step for the company, reinforcing its status as a multi-asset, clinical-stage entity.
IFALD is a complex, orphan liver disease characterized by hepatic inflammation, cholestasis, and steatosis, often linked to extended parenteral nutrition use. The condition can lead to hepatic fibrosis, potentially progressing to cirrhosis and liver failure. Preclinical studies have shown Orziloben's efficacy in preventing severe cholestasis and fibrosis in models of PN-induced liver injury. The drug's ability to target multiple pathogenic components of IFALD and its oral dosing potential make it a promising candidate for treating PN-induced liver disease and possibly other liver conditions.
Professor Palle Bekker Jeppesen, an expert in intestinal failure and liver diseases, has noted Orziloben's robust efficacy in pre-clinical models and its potential to become a therapeutic option for patients with limited current treatments. Orziloben, a novel, potent, synthetic medium-chain fatty acid analogue, is designed for enhanced pharmacological effects and ease of transport to the liver and cells, avoiding rapid metabolism. The drug's complete prevention of severe cholestasis and fibrosis in a pre-clinical model of PN-induced liver injury, along with its reduction of myofibroblasts and hepatic inflammation, underscores its potential as an efficacious treatment for liver diseases.
NorthSea Therapeutics, headquartered in the Netherlands, is advancing structurally engineered fatty acids for metabolic disorders, with icosabutate in clinical development for NASH and two additional compounds in various stages of clinical development. The company is supported by a consortium of investors and maintains a presence in Norway and the US.