Ipsen to present two late-breaking sessions at AASLD on new PBC data supporting IQIRVO®’s long-term efficacy, safety and mechanistic insights in fatigue
Summary by AI BETAClose X
- Interim data from the IQIRVO® (elafibranor) ELATIVE long-term open label extension trial demonstrate sustained biochemical response, stabilization of fibrosis markers and consistent trends in improvement of fatigue and pruritus symptoms, with a well-characterized safety profile over more than three years of treatment
- Additional new data from the ELATIVE trial highlights the potential for IQIRVO, a PPAR α/δ agonist, to beneficially impact fatigue-associated pathways linked to mitochondrialfunction
Paris, France – 7 November, 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that new Primary Biliary Cholangitis (PBC) data with IQIRVO® from the ELATIVE trial1, 2 will be presented in two late-breaking sessions, at The Liver Meeting® 2025, hosted by the American Association for the Study of Liver Diseases (AASLD).
In interim data from the ELATIVE long-term, open label extension trial, which includes over three years of follow-up in 115 patients with PBC, observed that IQIRVO delivers sustained improvements in biomarkers of cholestasis and stabilization in markers of fibrosis, with IQIRVO treatment, suggesting potential for slowing disease progression. In addition, consistent trends in improvements were shown in fatigue and pruritus symptoms. At week 182, 72% of patients receiving IQIRVO maintained a biochemical response, with a 47% reduction in alkaline phosphatase (ALP) from baseline. The proportion of patients achieving normal ALP levels remained consistent with previously presented Phase III results from the ELATIVE trial. Improvements in patients with moderate-to-severe fatigue were sustained, with similar results observed for pruritus and data also confirmed a long-term, well-characterized safety profile with no new safety signals.1
“PBC does not impact all patients in the same way. Therefore, it is important for us to have access to data associated with long-term treatment benefit on the disease biomarkers and liver tests, as well as a positive impact on symptoms. These preliminary results, which indicate a potential improvement not only in pruritus, but also in fatigue, are very encouraging,” said Dr. Cynthia Levy, Professor of Medicine, Division of Digestive Health and Liver Diseases, University of Miami. “We need to regularly monitor our patients with PBC over their lifetime and this data from the ELATIVE trial confirms that elafibranor is an effective treatment, with a reassuring, well-characterized safety profile, over the long-term.”
In a second late-breaking presentation of a further analysis of the ELATIVE trial, showing the relationship between changes in the expression of fatigue-associated proteins and reported outcomes of fatigue in patients on IQIRVO, was presented. 2 The clinical findings from this analysis are consistent with previously published mechanistic data3, suggesting that PPAR α/δ agonist activation may modulate key pathways involved in energy metabolism and mitochondrial function. Fatigue remains one of the most common and burdensome symptoms for patients with PBC. Currently, there are no therapies approved to address it, however, clinically meaningful improvements in fatigue have been observed with IQIRVO, which is a PPAR α/δ agonist, versus placebo, in the ELATIVE trial with around one in two patients reporting a significant reduction in fatigue severity.4 Together, these data support further research into how IQIRVO may help address fatigue in PBC.
“These findings underscore IQIRVO’s potential as a long-term treatment option that not only manages the markers of disease progression and symptoms that impact the quality of life of people living with PBC, but also helps us better understand the mechanisms behind fatigue,” said Sandra Silvestri, MD, PhD, Executive Vice President, Chief Medical Officer, Ipsen. “With a consistent safety profile over three years and these emerging mechanistic insights, IQIRVO is positioned to play a transformative role in the management of PBC.”
About the ELATIVE Trial
ELATIVE is a multi-center, randomized, double-blind, placebo-controlled Phase III clinical trial, with an open-label long-term extension (NCT04526665). ELATIVE is evaluating the efficacy and safety of elafibranor 80mg once daily versus placebo for the treatment of patients with PBC with an inadequate response or intolerance to ursodeoxycholic acid (UDCA), the existing first-line therapy for PBC. The trial enrolled 161 patients who were randomized 2:1 to receive elafibranor 80mg once daily or placebo. Patients with an inadequate response to UDCA would continue to receive UDCA in combination with elafibranor or placebo, while patients unable to tolerate UDCA would receive only elafibranor or placebo. Patients continued their assigned treatment after Week 52 until all patients had completed their treatment, or for a maximum of 104 weeks. The open-label long-term extension of ELATIVE remains ongoing.
About IQIRVO® (elafibranor)
Iqirvo (pronounced EYE-KER-VO) is an oral, once-daily, 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, Iqirvo was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) in adults with PBC who have an inadequate response to ursodeoxycholic acid (UDCA), the existing first-line therapy for PBC. Iqirvo was granted U.S. FDA accelerated approval in June 2024, conditional approval by the EMA in September 2024 and UK Medicines and Healthcare products Regulatory Agency (MHRA) in October 2024, for the treatment of primary biliary cholangitis (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, EMA and MHRA approvals are contingent on the further verification of clinical benefit. Iqirvo is currently in regulatory processes with other authorities. Iqirvo (elafibranor) was developed by GENFIT. Ipsen licensed the exclusive worldwide rights (except China, Hong Kong, Taiwan and Macau) to elafibranor from GENFIT in 2021.
About Primary Biliary Cholangitis
PBC is a rare, autoimmune liver disease where a build-up of bile and toxins and chronic inflammation cause irreversible fibrosis of the liver and destruction of the bile ducts. Impacting approximately 100,000 people in the US and 165,000 people in Europe, the majority being women, PBC is a lifelong condition that can worsen over time if not effectively treated and may lead to liver transplant and in some cases, premature death.
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 internal and 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 100 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
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Sally Bain sally.bain@ipsen.com +1 857 320 0517
Anne Liontas anne.liontas.ext@ipsen.com +33 0767347296
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: Long-term treatment with elafibranor leads to biochemical and symptomatic improvements for at least 3 years in patients with primary biliary cholangitis. Late-Breaking Oral Presentation # 5015. AASLD 2025, Washington D.C.
- Swain. M. et al: Elafibranor-associated changes in proteins linked to mitochondrial function correlate with fatigue improvement: Proteomic results from the ELATIVE® trial. Late-Breaking Poster Presentation # 5030. AASLD 2025, Washington D.C.
- Swain. M. et al. Elafibranor impacts inflammatory, fibrotic and symptom-associated markers in patients with primary biliary cholangitis: Proteomic results from the ELATIVE® trial. European Association for the Study of the Liver (EASL) congress, 2025. Abstract LB25202
- Jones. D. et al. Clinically significant improvements in fatigue with elafibranor in patients with primary biliary cholangitis and limited association with pruritus: Analyses from the phase III ELATIVE.® European Association for the Study of the Liver (EASL) congress, 2025. Abstract LB25220
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