Orziloben granted Orphan Drug Designation in the U.S. and European Union

Company expects to report topline results from Phase 2 study in Q3 2026

NorthSea Therapeutics B.V. (“NorthSea” or the “Company”), a private, late-stage clinical biotechnology company leveraging its proprietary structurally engineered fatty acids (SEFAs) platform to develop novel drugs to treat rare liver diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) have granted Orphan Drug Designations (ODD) to orziloben (NST-6179) for the treatment of intestinal failure associated liver disease (IFALD). Orziloben was also previously granted rare pediatric disease designation by the FDA.

“We are pleased that orziloben has received ODD from both agencies. This recognition continues to reinforce the widespread need to treat rare liver diseases and the potential of orziloben. Orziloben is in a Phase 2 study for the treatment of IFALD, a rare, chronic life-threatening condition with no approved pharmacologic treatment options,” said Sophie Jeannin, Chief Medical Officer of NorthSea Therapeutics.

In a Phase 1 study evaluating the safety, tolerability, and pharmacokinetics (PK) of orziloben in healthy people, the drug demonstrated a favorable tolerability profile, with most treatment-emergent adverse effects (TEAEs) being mild. Orziloben is currently being evaluated in a randomized, double-blind, Phase 2, placebo-controlled study in adult patients with IFALD. Topline results are expected in the third quarter of 2026.

“IFALD is a devastating liver disease for patients with chronic intestinal failure and can progress to end stage liver disease. If approved, orziloben has the potential to be the standard of care in the U.S. for the treatment of IFALD. We are deeply committed to advancing this important medicine that has the potential to be transformative for people and families living with IFALD,” said Rob de Ree, Chief Executive Officer of NorthSea Therapeutics.

About Orziloben
Orziloben, previously known as NST-6179, is a highly bioavailable, potent, oral agent that is administered once-daily for the treatment of intestinal failure associated liver disease (IFALD). It is an investigational, fully synthetic medium chain fatty acid (MCFA) analog that, unlike long chain fatty acids, is passively absorbed from the gut and directly targets the liver via the portal vein. It is also designed to resist rapid metabolism in the liver, overcoming a primary limitation of unmodified MCFAs as drugs. Of relevance to IFALD, orziloben targets multiple fatty acid sensitive receptors that selectively target dysregulated bile acid metabolism (PPAR-α), liver inflammation (GPR84), and fibrosis (PPAR-γ).

About Intestinal Failure Associated Liver Disease (IFALD)
Intestinal Failure-Associated Liver Disease (IFALD) represents one of the most serious and life-threatening conditions in patients with intestinal failure (IF) receiving parenteral nutrition (PN). IFALD is characterized by progressive hepatic dysfunction, manifesting as persistent cholestasis, hepatic inflammation, fibrosis, steatosis, and ultimately biliary cirrhosis.¹ There are an estimated 40,000 adult patients and 3,600 pediatric patients in the United States with chronic intestinal failure. Of these, approximately 15-40% of adults and approximately 40-60% of children develop IFALD.²

About our Phase 2
Orziloben is currently in a Phase 2 trial evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) in adult patients with IFALD. This Phase 2 trial is a randomized, double-blind, placebo-controlled study, evaluating safety, tolerability, PK, and pharmacodynamic effect of orziloben 800mg once daily versus placebo for 4 weeks of treatment, and orziloben 1200mg once daily for 12 weeks in adult patients with IFALD. The trial will evaluate reductions from baseline in alkaline phosphatase, gamma-glutamyl transferase (GGT), aspartate transaminase (AST), alanine transaminase (ALT), and bilirubin. Topline data are expected in Q3 2026.

About NorthSea Therapeutics
NorthSea Therapeutics B.V. is a private, late-stage clinical biotechnology company that leverages our structurally engineered fatty acids (SEFAs) platform to develop novel drugs that target cholestatic, inflammatory, and fibrotic pathways to treat heterogeneous rare liver diseases that require a multi-modal mechanism of action (MOA). Its proprietary structurally engineered fatty acids (SEFAs) are designed to target key disease pathways, offering potential first-in-class treatments. NorthSea’s lead program is orziloben, a Phase 2 program for the treatment of intestinal failure associated liver disease (IFALD). NorthSea's investors include Forbion, Ysios Capital, venBio, Novo Seeds, Sofinnova, BGV, and New Science Ventures. Headquartered in the Netherlands, NorthSea also has a presence in the U.S. and Norway. To learn more, visit https://northseatherapeutics.com/en/.

¹Pironi et al. 2023; Cuerda et al. 2021; Di Dato et al. 2022
²Zafirovska, M.; Zafirovski, A.; Rotovnik Kozjek, N. Current Insights Regarding Intestinal Failure-Associated Liver Disease (IFALD): A Narrative Review. Nutrients 2023, 15, 3169. https://doi.org/10.3390/nu15143169

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