Dr. Falk Pharma’s norucholic acid (NCA) drug has demonstrated potential in a Phase III trial for primary sclerosing cholangitis (PSC).
In the pivotal, Phase III NUC-5 trial, (NCT03872921), NCA showed statistical superiority over placebo in achieving the combined primary endpoints of partial normalisation of blood levels of a liver enzyme linked to PSC (alkaline phosphatase) and no progression of disease stage on histology.
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Significant superiority of NCA was also observed in multiple secondary endpoints and safety and tolerability was similar between the NCA and placebo groups.
Topline data from the 96-week analysis of the NUC-5 trial will be presented at 2025 EASL Congress, taking place 7-10 May in Amsterdam.
The double-blind, placebo-controlled trial enrolled 301 patients with PSC, who receive either 1,500mg NCA or placebo for a total of 192 weeks.
PSC is a rare, progressive disease in which the immune system attacks the bile ducts in the liver, which leads to fibrosis, or the formation of scar tissue. A considerable proportion of patients develop bile duct, liver, or colorectal cancer, while many others eventually progress to cirrhosis of the liver.
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By GlobalDataThere are no approved treatments available and currently, the most effective treatment option is liver transplantation. Patients can also be treated with cholestyramine help relieve itching caused by bile acids. Other treatments include antibiotics for infections and supplements for vitamin deficiencies.
In 2015, Intercept’s Ocaliva (obeticholic acid) received orphan drug designation in the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for PSC and in 2017, it showed a benefit during its Phase II trial. However, in 2020, Intercept deprioritised the drugs development in PSC due to lack of clarity on the regulatory pathway for the indication.
Head of the Division of Gastroenterology and Hepatology at the Medical University of Vienna, Austria, Prof. Michael Trauner said: “Finding a medicine that effectively treats PSC has been a challenge in the field of hepatology for decades. After so many disappointments in this space, the first positive results from a Phase III study on PSC is a watershed moment for people with PSC, their families, physicians and the entire PSC community. The results of this study will not only advance patient care but will also give researchers new insights into the disease itself.”
Across the 16 major pharmaceutical markets (16MM: Australia, Brazil, Canada, China, France, Germany, India, Italy, Japan, Mexico, Russia, South Africa, South Korea, Spain, UK, and US), GlobalData epidemiologists estimate diagnosed incident cases of PSC to reach 17,685 in 2029, with most cases due to be reported from China, a total 33.2% of the cases.
GlobalData is the parent company of Clinical Trials Arena.
