PSC Treatment Breakthroughs 2025
PSC Research Roundup 2025:
A Year of Transformation
Research in 2025 was characterised by significant progress in the search for treatments for primary sclerosing cholangitis (PSC). We saw a shift from early-stage discovery into pivotal clinical trials and the implementation of essential new services in the UK.
Top PSC research findings in 2025
We saw progress towards treatments designed to address both the underlying causes of PSC and the symptoms that affect daily life.
- Norucholic acid (NCA, the NUC 5 trial): Early results from a pivotal Phase 3 study showed NCA was significantly superior to placebo (a dummy drug) in improving liver blood tests, with nearly a quarter of participants seeing a reversal of liver scarring. Read more.
- Nebokitug (SPRING Trial): Published in the American Journal of Gastroenterology in December 2025, 48-week data confirmed nebokitug is safe and well-tolerated. Notably, participants with moderate-to-advanced fibrosis (scarring) showed a significant reduction in liver stiffness. This success supports moving to a pivotal Phase 3 trial.
- Elafibranor (ELMWOOD Trial): Data showed reduced markers of bile flow problems and a significant reduction in the severity of itching and fatigue with elafibranor. Further data from the Open Label Extension showed lasting improvements, and the 12-week data was published in the Journal of Hepatology in January 2026. View Publication.
- Volixibat (VISTAS Trial): This trial, addressing itch in PSC with volixibat, finished recruitment in 2025. While clinical results take time to emerge, we are hopeful for early data by summer 2026.
How is PSC diagnosis and monitoring improving?
A major priority for PSC Support is establishing non-invasive tests to monitor disease and detect cancers early, and treat them.
- 3-in-1 Blood Test: Researchers in Spain developed a blood test capable of diagnosing PSC, detecting early-stage bile duct cancer (cholangiocarcinoma), and predicting future risk. Your donations have been funding this work since 2019, and we are now working to move this into the final phase of testing.
- UK Transplant Service: Launched in 2025, a specialised service now provides liver transplants for some carefully selected patients diagnosed with bile duct cancer and PSC. Watch out for our upcoming webinar with AMMF to learn more.
Looking Forward: Making Every Opportunity Count
To every person who has participated in a trial, provided a sample, or joined our Patient Panel - thank you. Your contribution is the reason we have a story to tell this year.
I often think back to my first PSC Support meeting in Oxford. For those who were there, you’ll remember the floods that year; for me, it was the start of a journey where the landscape looked very different. In 2007, there were no clinical trials. Norucholic acid (NCA) was still only being studied in mice, and the national biobank we now know as UK-PSC was only just beginning. We gave our blood samples then with a simple hope: that the genetics of PSC would be uncovered.
We have since learned that PSC is far more complex than we imagined, but we have come so far. Thanks to your voices in PSC Support surveys, we have proven that there is more to this disease than blood results. Its impact on our energy, mental health, and wellbeing is finally being taken seriously. Today, trials are tackling debilitating symptoms, measuring quality of life, and working to prevent complications before they start.
Despite these promising horizons, we cannot take progress for granted. PSC remains a devastating condition, and we still face many unknowns. We need treatments that help 100% of our community, and we need them urgently.
This means we must continue to make the most of every research opportunity. Whether it is seeking out a clinical trial, providing a sample to a research nurse, or sharing your views in a survey - it all counts.
We enter 2026 with more momentum than ever. Together, let’s keep moving towards a world without PSC.
Do drop me an email if you have any questions:
Martine Walmsley
PSC Support Head of Research Strategy
ERN RARE-LIVER Management Board and PSC Working Group Co-Lead