AMSTERDAM– NorthSea Therapeutics B.V., (‘NST’), a biotech company developing novel and innovative strategies for the treatment of NASH and other metabolic diseases, today announces it has initiated a Phase 1 study with SEFA-6179 in adult subjects, targeting the initial orphan indication of the treatment of IFALD.
IFALD is a multifactorial condition characterized by the development of hepatic inflammation, cholestasis and steatosis in patients with intestinal failure and/or short bowel syndrome. It occurs after prolonged use of total parenteral (intravenous) nutrition. The development of hepatic fibrosis, which can progress to cirrhosis and liver failure, is a major concern for patients with this condition. No approved drug therapy exists for this population.
The Phase 1 SEFA-6179 clinical trial is being conducted in the United Kingdom and is expected to enroll approximately 90 healthy participants. The study is a randomized, placebo-controlled, single and multiple ascending dose trial, evaluating the safety, tolerability and pharmacokinetics of SEFA-6179. The final results are anticipated in Q3 2022 and will support the initiation of a global Phase 2 study in patients with IFALD, scheduled to start in Q1 2023.
Professor Palle Bekker Jeppesen, Head of the Department of Intestinal Failure and Liver Diseases at Rigshospitalet in Copenhagen, stated: “SEFA-6179 has shown a broad treatment effect on key biomarkers of liver injury as well as improvements in liver histology in multiple pre-clinical models. The availability of an oral therapy could optimize the management of this liver disease with high medical need. It could deliver significant clinical benefit to patients on long-term parenteral nutrition with a high risk of IFALD.”
NST has a library of Structurally Engineered Fatty Acids (SEFAs) which are all new chemical entities with unique differentiated physicochemical properties and biological targets.
Rob de Ree, NST’s CEO, commented: “The commencement of this trial is a key milestone in NST’s journey to become a multi-asset company with a robust clinical pipeline of SEFAs. We are excited to develop our first candidate for an orphan indication, especially for one where patients currently have no therapeutic options available.”
SEFA-6179 is a novel, orally-administered, highly potent, synthetic, medium chain fatty acid analog. The molecule directly targets the liver and has demonstrated robust treatment of cholestasis, steatosis and fibrosis in multiple pre-clinical models. The unique pharmacologic properties of SEFA-6179 facilitate intestinal uptake, which is critical for ensuring optimal drug absorption in patients with intestinal failure and short bowel syndrome. Unlike natural fatty acids, NST’s SEFAs are designed to prevent rapid use as an energy source, allowing for optimal drug exposure.
