Positive News from EASL Congress on Two Potential Treatments for PSC
- PSC Partners Seeking a Cure
- May 7
- 3 min read
Updated: 6 days ago
In May 2025, the European Association for Study of the Liver (EASL) hosted its annual congress in Amsterdam, Netherlands. Late-breaking oral presentations on potential treatments for PSC featured results from two clinical trials:
Dr. Falk Pharma: Norucholic acid for the treatment of primary sclerosing cholangitis: 96-week analysis of a pivotal phase 3 trial, presented by Michael Trauner
Ipsen Pharmaceuticals: Elafibranor for primary sclerosing cholangitis: the ELMWOOD phase II randomised controlled trial, presented by Cynthia Levy
PSC Partners is encouraged by the positive results from both studies, which offer hope to the community. No treatments have yet been approved for PSC, and the success of these two studies offers potential pathways to a treatment.
Norucholic Acid (NCA): Results from European Phase 3 Study in PSC
On May 7, 2025, Dr. Falk Pharma announced results from their phase 3 study of norucholic acid (also known as norursodeoxycholic acid, or norUDCA). The NCA Phase III trial was a 96 week study of 301 PSC patients, 205 patients receiving 1500 mg daily of NCA and 96 receiving a placebo. 78% of patients were concurrently taking UDCA (Ursodeoxycholic acid, or Urso/Ursodiol).
The combined primary endpoint* was reduction of alkaline phosphatase (ALP) levels** to less than 1.5x the upper limit of normal (ULN), along with no worsening of fibrosis stage. All patients in the study began with ALP levels greater than this threshold.
The following key results were achieved in the study at week 96:
A higher rate of patients taking NCA achieved the primary endpoint (15.1%) versus placebo (4.1%). Among those not concurrently taking UDCA, a larger difference was seen: 20.3% on NCA and UDCA vs 0% on placebo and UDCA.
A higher rate of patients receiving NCA had improvement in fibrosis versus placebo, and a lower rate had worsening of fibrosis versus placebo.
Other liver enzyme markers had sustained improvement for patients on NCA but not on placebo.
NCA demonstrated a favorable safety and tolerability profile, with similar rates of adverse events versus placebo.
As this study was conducted solely in European countries, approval as a treatment for PSC would only be considered in Europe based on this study alone. A separate path would have to be pursued for approval in the United States by the FDA.
Learn more:
About NCA: NCA is a derivative of UDCA, an approved treatment for PBC that is sometimes prescribed for off-label use in treatment of PSC.
Press release from Dr. Falk Pharma
*Primary endpoint: The most important outcome(s) that researchers measure to determine whether a new treatment is effective. In the case of a combined primary endpoint, both measures need to be achieved.
**ALP is a biomarker collected through blood samples for measuring and predicting disease progression in PSC.
Elafibranor: Results from International Phase 2 Study in PSC
On May 10, 2025, Ipsen Pharmaceuticals will present results from their Phase II ELMWOOD study. The ELMWOOD study was a 12 week study of 68 patients who were randomly assigned to receive 80 mg or 120 mg of elafibranor or a placebo. The primary endpoint of this study was safety and tolerability of elafibranor.
The following key results were achieved in the study:
Elafibranor showed “significant dose-dependent reductions in alkaline phosphatase (ALP)” versus placebo after 12 weeks, with greater reduction in the 120 mg treatment group: -171.1 U/L in 120 mg, -103.2 U/L in 80 mg, and +32 U/L in placebo.
Enhanced Liver Fibrosis (ELF) scores, another marker for disease progression, also stabilized in the elafibranor group compared with placebo.
Patients in the 120 mg elafibranor treatment group also experienced improvements in pruritus (PSC-related itch) versus placebo.
Elafibranor demonstrated a favorable safety and tolerability profile, with comparable rates of adverse events to placebo.
Learn more:
About Elafibranor: Elafibranor was approved by the FDA in 2019 as a Breakthrough Therapy Designation as a second-line treatment for primary biliary cholangitis (PBC), when UDCA did not succeed as a first-line treatment.
PSC Partners will continue to update the community as more information becomes available regarding this study. Please check back for updates, including on the Registry’s Ongoing Clinical Trials page.