Dr Falk Pharma: Dr Falk Pharma, a research-based pharmaceutical company specialising in digestive and metabolic medicine, has announced positive topline results from its pivotal phase 3 trial (NUC-5) on norucholic acid (NCA) for the treatment of primary sclerosing cholangitis (PSC), a rare and progressive liver disease with no approved therapies.
According to BERNAMA News Agency, findings from the 96-week interim analysis of the NUC-5 trial will be presented at the 2025 EASL Congress in Amsterdam during the Late Breaker session on May 10. Dr Falk Pharma Managing Director Science and Innovation, Dr Kai Pinkernell, stated that the NUC-5 is the largest PSC trial to date that included biopsy comparisons pre- and post-treatment.
Dr Pinkernell expressed gratitude towards the patients, investigators, and trial staff for their dedication and contribution, noting that a trial of this scale and duration is a major undertaking. Meanwhile, the Medical University of Vienna’s Head of the Division of Gastroenterology and Hepatology and principal investigator of the trial, Prof Michael Trauner, stated that these findings will not only support better care but also enhance the understanding of the disease.
The double-blind, placebo-controlled NUC-5 trial enrolled 301 patients with PSC, who were randomly assigned to receive either 1,500 milligrammes (mg) of NCA or a placebo over a treatment period of 192 weeks. The primary analysis conducted after 96 weeks of treatment showed that NCA led to a statistically significant achievement of the trial’s primary endpoint. This was defined as partial normalisation of alkaline phosphatase-a liver enzyme associated with PSC-combined with no progression in disease stage as assessed by liver histology.
NCA also demonstrated superiority across multiple secondary endpoints. Safety data revealed comparable rates of adverse and serious adverse events between the NCA and placebo groups. PSC is a chronic autoimmune condition that damages bile ducts in the liver, often progressing to cirrhosis or cancer. With no approved pharmaceutical treatments to date, liver transplantation remains the primary intervention for many patients.