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 Information for IQIRVO in the U.S.
Please see full Prescribing Information for IQIRVO in the E.U.
ENDS
About Ipsen
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.
Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 80 countries.
Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.
Ipsen contacts
Investors
- Khalid DEOJEE | + 33 6 66 01 95 26 | khalid.deojee@ipsen.com
Media
- Sally Bain | +1 8573200517 | sally.bain@ipsen.com
- Anne Liontas | + 33 7 67 34 72 96 | anne.liontas.ext@ipsen.com
Disclaimers and/or Forward-Looking Statements
The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com.
References
- 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
