Late-breaking elafibranor primary sclerosing cholangitis (PSC) data demonstrates favorable safety profile and significant efficacy in second potential rare liver disease indication

• Elafibranor showed a favorable safety profile and demonstrated dose-dependent efficacy over 12 weeks for people living with PSC, a rare liver disease that currently has no approved treatment options
• Patients treated with elafibranor versus placebo showed significant improvements in liver biochemical parameters, including alkaline phosphatase (ALP)
• Stabilization of non-invasive markers of liver fibrosis were observed in patients on elafibranor versus placebo
• A significant improvement in pruritus was observed in patients on elafibranor 120 mg versus placebo

PARIS, FRANCE, 24 April 2025 Ipsen (Euronext: IPN; ADR: IPSEY) will be presenting data from the late-breaking abstract on elafibranor in the investigational Phase II ELMWOOD study at the European Association for the Study of the Liver (EASL) congress as an oral presentation, on 10 May at 11.15 CET. For the first time data highlighting the potential of elafibranor in treating people living with primary sclerosing cholangitis (PSC) will be presented. PSC is a rare liver disease that currently has no approved treatment options.

"These results are a testament to our ongoing commitment to advancing potential treatments for rare liver diseases where there is a significant unmet need and few options for patients currently exist,” said Christelle Huguet, PhD, Executive Vice President and Head of Research and Development, Ipsen. “These results are encouraging and reinforce elafibranor’s action as a PPARα/δ agonist in potentially treating multiple liver diseases, like PSC.”

The Phase II ELMWOOD trial data (LB25222/OS089) demonstrated a positive safety and tolerability profile and efficacy benefits for patients with PSC treated with elafibranor versus those treated with a placebo. In this 12-week study, 68 patients with PSC were randomized to receive either elafibranor 80 mg or 120 mg or placebo. The primary endpoint was the safety and tolerability of elafibranor. Treatment-emergent adverse events were experienced by 68.2%, 78.3% and 69.6% of patients on elafibranor 80 mg, 120 mg and placebo, respectively. Adverse events leading to treatment discontinuation occurred more commonly in patients on placebo (8.7%) than elafibranor 80 mg (4.5%) or 120 mg (4.3%). Serious adverse events occurred in 4.3% of patients on placebo and none on elafibranor.¹

Efficacy results showed that patients on elafibranor had significant dose-dependent reductions in alkaline phosphatase (ALP), with patients on elafibranor 80 mg and 120 mg having significant reductions at week 12 versus placebo (−103.2 U/L and −171.1 U/L vs +32.1 U/L; p < 0.0001), and improvements observed as early as week 4. Similar findings were seen in other biochemical liver parameters, including alanine aminotransferase (ALT) and gamma-glutamyl transferase (GGT), which are important biochemical markers of disease progression.

Patients on elafibranor also had stabilization in Enhanced Liver Fibrosis (ELF), a non-invasive marker of liver fibrosis, versus patients on placebo at week 12. Additionally, patients on elafibranor 120 mg experienced improvements in pruritus compared with patients on placebo according to the Worst Itch Numeric Rating Scale (WI NRS) score (-0.96 vs -0.28; p<0.05).¹

“These data from the ELMWOOD trial are encouraging, demonstrating positive safety and efficacy data for elafibranor as a potential treatment for PSC, where none currently exist,” said Cynthia Levy, MD, Professor of Clinical Medicine and Hepatology, University of Miami Miller School of Medicine, Miami, Florida. “PSC is a serious liver disease and currently, liver transplantation is the only treatment that can significantly improve the prognosis. These findings support further investigation in larger longer-term trials to fully evaluate the potential of elafibranor in PSC.”

PSC is a rare, chronic liver disease characterized by inflammation and scarring of the bile ducts, which can lead to liver damage and eventually liver failure. The exact cause of PSC is unknown, but it is often associated with other autoimmune conditions, such as inflammatory bowel disease. Symptoms of PSC can include itching, fatigue, jaundice, and abdominal pain. Over time, PSC can result in complications like bile duct infections, liver cirrhosis, and an increased risk of liver cancer. Currently, there are no FDA or EMA approved therapies for the treatment of PSC.

About ELMWOOD
The ELMWOOD phase II study is a randomized, double-blind, placebo-controlled study which evaluated the safety and efficacy of elafibranor in treating PSC, a rare liver disease. Conducted over 12 weeks, the trial involved 68 patients who were randomized to receive either elafibranor (80 mg or 120 mg) or a placebo. The primary endpoint was the safety and tolerability of elafibranor versus placebo. Additional endpoints included change from baseline in liver biochemistry values, non-invasive markers of fibrosis, and patient-reported outcomes, including pruritus according to the Worst Itch Numeric Rating Scale (WI NRS). The open-label 96-week extension evaluating the safety and efficacy of elafibranor 120 mg remains ongoing.

About elafibranor
Elafibranor is an oral peroxisome proliferator-activated receptor (PPAR) agonist, which exerts an effect on PPARα and PPARδ. Activation of PPARα and PPARδ decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters. Activation of PPARα and PPARδ also has anti-inflammatory effects by acting on different pathways. In 2019, elafibranor was granted Breakthrough Therapy Designation by the U.S Food and Drug Administration (FDA) in adults with Primary Biliary Cholangitis (PBC) who have an inadequate response to ursodeoxycholic acid (UDCA) the existing first-line therapy for PBC. Elafibranor under the brand name IQIRVO^® was granted U.S. FDA accelerated approval in June 2024, EU conditional approval by the European Commission (EC) in September 2024 and UK Medicines and Healthcare products Regulatory Agency (MHRA) approval in October 2024, for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The FDA, EC and MHRA approvals are contingent on the further verification of clinical benefit. Elafibranor was developed by GENFIT. Ipsen licensed the exclusive worldwide rights (except China, Hong Kong, Taiwan and Macau) to elafibranor from GENFIT in 2021.
Please see full Prescribing Informationfor IQIRVO in the U.S.
Please see full Prescribing Information for IQIRVO in the E.U.

ENDS

About Ipsen
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References

1. Levy. C. et al. Elafibranor for primary sclerosing cholangitis: The ELMWOOD phase II randomised controlled trial. European Association for the Study of the Liver (EASL) congress, 2025. Abstract LB25222 
   
   

Attachment

• Ipsen PR_EASL 2025 IQIRVO PSC_24042025