Ipsen to present data across five rare liver diseases at AASLD 2025

• Latest research reinforces Ipsen’s commitment to improving outcomes for patients living with rare liver diseases
• 13 abstracts, including two late-breaking abstracts from the ELATIVE study, three orals and two posters of distinction to be presented at AASLD
• Data supports the potential of IQIRVO® (elafibranor) and Bylvay® (odevixibat) to address both disease progression and symptom burden across multiple rare cholestatic liver diseases

Paris, France – October 8, 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced the presentation of 13 scientific abstracts at The Liver Meeting® 2025, hosted by the American Association for the Study of Liver Diseases (AASLD). The abstracts span five rare liver diseases: primary biliary cholangitis (PBC), primary sclerosing cholangitis (PSC), Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA), reinforcing Ipsen’s commitment to advancing research and improving outcomes for patients living with these complex and underserved rare liver diseases.

Data highlights from the IQIRVO® (elafibranor) ELATIVE Study in PBC
For PBC, Ipsen will present six abstracts from the ongoing ELATIVE study, including two late-breaking presentations (oral#5016 and poster#5030), to be released at a later date. Together these data continue to support IQIRVO’s efficacy and safety profile, towards modifying disease progression while improving multiple key symptoms including fatigue and pruritus (itch), in patients living with PBC.

• Oral #0120. Distinct protein signatures associated with pruritus, modulated by IQIRVO treatment are explored, providing new mechanistic insights into IQIRVO’s anti-pruritic effect.
• Poster #4461. Data suggest that improvements in fatigue and pruritus occur independently of biochemical cholestasis response in patients treated with IQIRVO.
• Poster #4397. ELATIVE open-label extension data provide further insights into the sustained long-term benefits of IQIRVO on stabilizing PRO-C3, a non-invasive marker of liver fibrosis.
• Poster #4522. Shows that the efficacy of IQIRVO is not impacted by the body mass index of patients living with PBC.

Data highlights from the elafibranor ELMWOOD Phase II study in PSC

• Oral #0005. In a further oral presentation, results from the 28-week interim analysis of the ongoing open-label extension of ELMWOOD will provide insights into the sustained efficacy and tolerability profile of elafibranor 120 mg in patients with PSC.

Bylvay® data highlights from across ALGS, PFIC and BA

• Poster #4406. A poster of distinction from the ASSERT and ASSERT-EXT studies discusses how irrespective of serum bile acid (sBA) response status, treatment with Bylvay led to pruritus control in patients with ALGS.
• Poster #4526. Data from the Phase III PEDFIC 2 open-label extension demonstrate that Bylvay treatment leads to rapidly reduced sBA levels and improved pruritus in most patients with MDR3 deficiency, also known as PFIC3.
• Poster #4504. In BA, Ipsen will present the BOLD and BOLD-EXT study designs, the first global Phase III program in BA. The studies aim to evaluate the effect of odevixibat on transplant-free survival in infants with BA following a Kasai procedure, addressing a critical unmet need.

Ipsen Data presentations at AASLD

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 Bylvay® (odevixibat)
Bylvay is a potent, once-daily ileal bile acid transporter inhibitor (IBATi) that acts locally in the small intestine with minimal absorption into the rest of the body (minimal systemic exposure). Bylvay has received orphan exclusivity for the treatment of PFIC in the EU and in the US. Bylvay was approved in June 2021 in the EU as the first drug to treat all types of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older, and in the US as the first treatment option for patients 3 months of age and older living with cholestatic pruritus due to PFIC. In June 2023 Bylvay was approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with ALGS and received orphan exclusivity for ALGS.  In 2024 under the brand name KAYFANDA® (odevixibat), an approval under exceptional circumstances was granted in the EU for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. Odevixibat is in late-stage development in the only Phase III clinical trial (BOLD) for the treatment of biliary atresia.

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

Investors
Henry Wheeler          henry.wheeler@ipsen.com +33 7766471149 
Khalid Deojee           khalid.deojee@ipsen.com +33 666019526

Media
Sally Bain                sally.bain@ipsen.com +1 8573200517
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

1. Summar,y, Ministry of Health Labour and Wellbeing, Japan: 001175135.pdf Last accessed: September, 2025
2. Baker A, et al. Systematic review of progressive familial intrahepatic cholestasis. Clin Res Hepatol Gastroenterol. 2019. 43(1):20–36.
3. Nayagam JS, et al. Clinical phenotype of adult‐onset liver disease in patients with variants in ABCB4, ABCB11, and ATP8B1. Hepatol Commun. 2022. 6(10):2654-2664.
4. Agarwal S, et al. Progressive Familial Intrahepatic Cholestasis (PFIC) in Indian Children: Clinical Spectrum and Outcome. J Clin Exp Hepatol. 2016. v.6(3).
5. Davit-Spraul A, et al. Progressive familial intrahepatic cholestasis. Orphanet J Rare Dis. 2009. 4(1):1-12.
6. Thompson RJ, et al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2022. 7:830–842.
7. Thompson RJ, et al. Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis 2023. JHEP Rep. 5(8):100782.