Nov 2025 Research Roundup

30/11/2025

Research Roundup

November 2025

November has been a month of significant news across drug development, diagnostic research, and policy reform for people living with PSC and their families.

We have seen promising long-term results from advanced clinical trials, the launch of new studies targeting the gut, and major announcements regarding how the UK regulator plans to speed up access to rare disease treatments.

It is always important to remember that research takes time. Not every result will change your care straight away, but every step adds to our overall knowledge and moves us closer to approved treatments.

Ask the Expert!

If you're interested in PSC research, join me for a live online webinar with Dr Deepak Joshi at 12pm on Saturday 13 December. He'll be talking to us about PSC research announced at AASLD, the international liver conference that took place earlier this month.

Hopeful News from Drugs in Advanced Testing

Two major new drugs, currently in mid-stage (Phase II) clinical trials, released encouraging long-term data this month. This stage tests whether a drug is safe and effective in a smaller group of people. This news provides renewed hope for future treatments, as PSC currently has no proven treatment in any country.

Nebokitug (SPRING Trial) Moves Closer to Phase III

New 48-week extension data from the SPRING Phase II trial for the drug nebokitug was announced.

What it does: Nebokitug is a targeted medicine that works by changing the disease process in the liver, helping to control inflammation and scarring.
Key Results: It was safe and well tolerated for the full 48 weeks. Importantly, participants showed stable or continued improvements in markers of inflammation and scarring (fibrosis).
Why it matters: These long-term positive results provide strong support for advancing nebokitug into a larger Phase III clinical trial (the final, large study needed for approval).

Elafibranor (ELMWOOD Trial) Maintains Stable Results

Positive results were reported from the extension of the Phase II ELMWOOD trial for the drug elafibranor. This drug aims to help with inflammation, scarring, and problems with bile flow.

Key Results: Improvements in key markers of liver health were maintained over 28 weeks, and the drug remained safe. Non-invasive tests (like a Fibroscan, which measures liver stiffness) showed that scarring markers either stayed the same or improved.
Why it matters: This suggests the drug has the potential to not only stabilise liver damage but possibly help reverse some existing damage, which is highly encouraging.

New Drugs Looking at the Link Between the Gut and Liver

Two new clinical trials are exploring treatments that focus on the connection between the gut and the liver. Targeting this approach makes sense because PSC and inflammatory bowel disease (IBD) are closely related; in fact, 70–80% of people with PSC also have IBD.

LB-P8: New Trial Targets the Gut Connection

What happened: A global Phase II clinical trial for the new medicine, LB-P8, has started giving the drug to patients in the US.
Why it matters: LB-P8 is the first medicine of its kind (first in class) to be tested for PSC. It aims to adjust the gut–liver connection to slow down inflammation and scarring, offering a completely new way to tackle PSC.

CARBALIVE (CATCh Trial) Cleans Up Toxins

What happened: The CATCh trial is actively recruiting people with PSC who also have IBD to test a new drug called CARBALIVE.
Why it matters: CARBALIVE is a new treatment that you swallow. It uses tiny carbon beads that stay in your gut and act like a sponge, soaking up bad toxins. This prevents the toxins from reaching the liver, helping to keep it protected and working better.

Your Wellbeing

New research helps link diagnostic tests to quality of life, while our patient survey highlighted areas where communication could be improved.

Non-invasive Tests Help Doctors Monitor Your Wellbeing?

A new study from the University of Oxford investigated the connection between measurable signs of PSC severity and a patient's reported quality of life.

Key Finding: The study found that some non-invasive tests, such as FibroScan liver stiffness (LS), could be linked to quality of life.
Why it matters: This study shows that objective test results have value beyond tracking disease progression; they could also better monitor the impact of PSC on your physical and mental wellbeing. This is important because it addresses the historical frustration when patients feel unwell despite good test results. Although more long-term research is needed, it is a positive step to see research connecting quality of life with measurable, non-invasive markers.

Talking About Bile Duct Cancer (CCA)

Our multinational patient survey has highlighted gaps in how medical teams communicate the risk and monitoring for bile duct cancer (cholangiocarcinoma or CCA).

Key Finding: Only half of respondents reported having a planned discussion with their doctor about CCA risks. Even fewer discussed the results of their monitoring scans, like the MRCP.
Why it matters: Patients consistently reported high levels of fear and anxiety regarding CCA risk. The results show a clear need for the PSC community to work together to standardise communication around this sensitive topic and reinforce that patients should feel empowered to ask for clear explanations of their test results.

UK Policy Overhaul to Speed Up Rare Disease Treatments

This month, the Medicines and Healthcare products Regulatory Agency (MHRA), the UK's regulator, announced plans to significantly update the rules for developing rare disease therapies, including those for PSC. PSC Support has been involved in developing these plans.

What happened: The reforms are designed to tackle the specific challenges of rare disease trial design, such as small patient numbers, which make large traditional clinical trials difficult.
Why it matters: The MHRA is considering flexible new ideas to speed up access to new medicines. This pragmatic approach signals a strong commitment from the UK regulator to ensuring life-changing treatments reach people with high unmet medical needs like PSC sooner.

Final Thoughts

My main takeaway this month is one of real progress and optimism.

he clinical trial updates for elafibranor and nebokitug are encouraging, as they suggest potential treatments are safe long-term and are moving closer to final testing. This is a massive step forward.

The research showing that non-invasive tests may reflect quality of life is a positive development, helping doctors connect your test results to how you actually feel, rather than just tracking physical disease severity.

Our survey results confirm there is significant and widely-reported anxiety and fear surrounding CCA. The PSC medical community, including patient organisations, recognise the urgent need for a reliable, non-invasive screening test and we are working together to make that happen.

Finally, the UK policy changes show that the medicine regulator is committed to creating faster, more flexible pathways to get new PSC treatments to patients as soon as possible.

Don't forget to register for Dr Joshi's Ask the Expert webinar. I hope to see you there!