- Phase 2a study evaluating the safety, tolerability, PK, and PD of Orziloben in IFALD
- The study will enrol up to 36 adult subjects at multiple sites in North America
- Study strengthens NST’s position as a multi-asset, clinical stage company
Amsterdam, The Netherlands, 21 February 2024 – NorthSea Therapeutics B.V. (‘NST’, or the ‘Company’), a biotech company developing novel and innovative strategies for the treatment of non-alcoholic steatohepatitis (NASH) and other liver-associated metabolic diseases, today announced the dosing of the first patient in its Phase 2a clinical trial of Orziloben (NST-6179) in intestinal failure-associated liver disease (IFALD), an orphan liver disease affecting individuals on prolonged intravenous (parenteral) nutrition (PN).
The trial is a randomized, double-blind, Phase 2a, placebo-controlled study, which will be conducted at multiple sites across North America. It is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Orziloben in adult subjects with IFALD. The readout of the trial is anticipated in H2 2025.
For more information on the study, see ClinicalTrials.gov (NCT05919680).
Dosing the first patient in our Phase 2a trial for Orziloben in IFALD is a significant achievement for NorthSea Therapeutics, and is a testament to our commitment to advance innovative treatments for liver diseases. There is a critical need for effective therapies in IFALD as, to date, there are no drug therapies approved to treat this orphan indication. We believe Orziloben has the potential to make a substantial impact in addressing this unmet medical need.
Rob de Ree
NST’s CEO
IFALD is a complex condition, characterized by the development of hepatic inflammation, cholestasis and steatosis, in patients with intestinal failure and/or short bowel syndrome. It is known to be associated with prolonged administration of PN. One major concern for patients with the condition is the development of hepatic fibrosis, which can progress to cirrhosis and liver failure.
Preclinical studies have demonstrated the efficacy of Orziloben in preventing severe cholestasis, fibrosis, and other key markers of liver damage in models where PN was administered. In one pre-clinical model of PN-induced liver injury, Orziloben treatment completely prevented severe cholestasis and the development of fibrosis. Orziloben was also shown to prevent the pronounced increase in markers of liver damage in another in vivo model of PN in combination with an inflammatory stimulus (endotoxin). Furthermore, Orziloben significantly reduced the number of myofibroblasts, the main collagen producing cell in the liver, in addition to hepatic inflammation and steatosis in a model of established fibrosis. The unique ability of Orziloben to target multiple pathogenic components of IFALD, and its potential for oral dosing, make it a promising candidate for the treatment of PN-induced liver disease, as well as other liver disease indications.
Orziloben has shown consistently robust efficacy in several pre-clinical models, and has demonstrated a strong preventative effect on important disease pathophysiology components, such as cholestasis and fibrosis. If these impressive pre-clinical effects translate to clinical effects, Orziloben has the potential to become an effective therapeutic for intestinal failure patients who currently have limited treatment options.
Professor Palle Bekker Jeppesen
Head of the Department of Intestinal Failure and Liver Diseases at Rigshospitalet in Copenhagen,
If you are interested in learning more about Orziloben, the NorthSea Therapeutics team will be attending the ASPEN 2024 Nutrition Science & Practice Conference (2-5 March, Tampa, Florida). Please contact: carine.beysen@northseatherapeutics.com
For further information:
NorthSea Therapeutics B.V.
Rob de Ree (CEO)
E-mail: info@northseatherapeutics.com
Tel: +31 356993000
Instinctif Partners (Media)
Melanie Toyne-Sewell / Katie Duffell
E-mail: NorthSea@instinctif.com
Tel: +44 20 7457 2020