Ipsen to Present Elafibranor Phase II Data at EASL Congress 2025

Ipsen, 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 Congress (EASL Congress 2025) as an oral presentation, on 10th May 2025.

For the first time data highlighting the potential of elafibranor in treating people living with primary sclerosing cholangitis will be presented. Primary sclerosing cholangitis is a rare liver disease that currently has no approved treatment options.

Elafibranor showed a favorable safety profile and demonstrated dose-dependent efficacy over 12 weeks for people living with primary sclerosing cholangitis, 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. 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.

""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, executive VP and head of R&D, Ipsen. “These results are encouraging and reinforce elafibranor’s action as a PPARa/d agonist in potentially treating multiple liver diseases, like primary sclerosing cholangitis.”

The phase II ELMWOOD trial data demonstrated a positive safety and tolerability profile and efficacy benefits for patients with primary sclerosing cholangitis treated with elafibranor versus those treated with a placebo. In this 12-week study, 68 patients with primary sclerosing cholangitis 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 per cent, 78.3 per cent and 69.6 per cent 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 per cent) than elafibranor 80 mg (4.5 per cent) or 120 mg (4.3 per cent). Serious adverse events occurred in 4.3 per cent of patients on placebo and none on elafibranor.

Efficacy results showed that patients on elafibranor had significant dose-dependent reductions in alkaline phosphatase, with patients on elafibranor 80 mg and 120 mg having significant reductions at week 12 versus placebo, and improvements observed as early as week 4. Similar findings were seen in other biochemical liver parameters, including alanine aminotransferase and gamma-glutamyl transferase, which are important biochemical markers of disease progression.

Patients on elafibranor also had stabilization in enhanced liver fibrosis, 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 score.

“These data from the ELMWOOD trial are encouraging, demonstrating positive safety and efficacy data for elafibranor as a potential treatment for primary sclerosing cholangitis, where none currently exist,” said Cynthia Levy, MD, Professor of Clinical Medicine and Hepatology, University of Miami Miller School of Medicine, Miami, Florida. “Primary sclerosing cholangitis 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 primary sclerosing cholangitis.”

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

The ELMWOOD phase II study is a randomized, double-blind, placebo-controlled study which evaluated the safety and efficacy of elafibranor in treating primary sclerosing cholangitis, 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. The open-label 96-week extension evaluating the safety and efficacy of elafibranor 120 mg remains ongoing.

Elafibranor is an oral peroxisome proliferator-activated receptor agonist, which exerts an effect on PPARa and PPARd. Activation of PPARa and PPARd decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters. Activation of PPARa and PPARd also has anti-inflammatory effects by acting on different pathways. In 2019, elafibranor was granted Breakthrough Therapy Designation by the US FDA in adults with Primary Biliary Cholangitis (PBC) who have an inadequate response to ursodeoxycholic acid the existing first-line therapy for PBC. Elafibranor under the brand name IQIRVO was granted US FDA accelerated approval in June 2024, EU conditional approval by the European Commission in September 2024 and UK 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 US 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.

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