Dr. Falk Pharma’s PSC drug meets endpoints in Phase III trial

Dr. Falk Pharma's norucholic acid (NCA) has shown promising results in a Phase III trial for primary sclerosing cholangitis (PSC), a rare disease where the immune system attacks bile ducts in the liver, causing fibrosis. In the pivotal NUC-5 trial (NCT03872921), NCA significantly outperformed placebo in achieving combined primary endpoints: partial normalization of liver enzyme levels (alkaline phosphatase) and no disease progression on histology. NCA also showed superiority in several secondary endpoints, with a similar safety and tolerability profile to placebo.

The trial, which involved 301 PSC patients, administered either 1,500mg of NCA or a placebo over 192 weeks. There are no approved therapies for PSC, and liver transplantation remains the most effective treatment. NCA's success marks a watershed moment for PSC treatment, offering new hope for patients. Topline data from the 96-week analysis will be presented at the 2025 EASL Congress.

13-05-2025