Ipsen to present new data across four rare liver diseases at EASL, including late-breaking data in PBC and PSC

• Seven abstracts, including three late-breaking presentations, will be shared on new IQIRVO^® (elafibranor) and Bylvay^®/Kayfanda^® (odevixibat) data, reinforcing the strong clinical value across Ipsen’s rare liver disease portfolio where unmet need is high and treatments are few
• In two late-breaking presentations, IQIRVO’s effect on fatigue and its relationship with pruritus in patients with primary biliary cholangitis (PBC) is shared, alongside proteomic results demonstrating IQIRVO impacts on inflammation, biochemistry, fibrosis and symptom-associated markers
• IQIRVO’s efficacy benefits are supported by long-term stabilization of non-invasive markers of liver fibrosis data and a new bone health analysis reinforces its safety profile in PBC
• Simultaneous publication in Journal of Hepatology and oral late-breaking presentation of Phase II ELMWOOD results evaluates safety and efficacy of elafibranor in primary sclerosing cholangitis
• Longer-term Bylvay/Kayfanda efficacy and safety data presented for progressive familial intrahepatic cholangitis and Alagille syndrome, two rare liver diseases

PARIS, FRANCE, 6 May 2025 – Today, Ipsen (Euronext: IPN; ADR: IPSEY) announced that seven abstracts with new data from its rare liver disease portfolio will be presented at the European Association for the Study of the Liver (EASL) congress, 7 – 10 May, in Amsterdam, Netherlands. These include two late-breaking abstracts selected for poster presentation from Ipsen’s IQIRVO^® (elafibranor) in primary biliary cholangitis (PBC) and one late-breaking abstract selected for oral presentation and simultaneous publication in the Journal of Hepatology: Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized controlled trial, on elafibranor in primary sclerosing cholangitis (PSC). Two other abstracts describing the effect of IQIRVO non-invasive markers of fibrosis and bone health in PBC were also selected for poster presentation.

In addition, two abstracts with data on odevixibat known in the EU as Bylvay^® for PFIC and Kayfanda^® for ALGS, will be shared as poster presentations. These data are from the open label extension studies in progressive familial intrahepatic cholangitis (PFIC) and Alagille syndrome (ALGS) respectively.

Upcoming presentations at EASL will reinforce the long-term efficacy benefits and safety profile of IQIRVO with data from the Phase III ELATIVE study and the ongoing open-label extension in patients with PBC:
  

• Data from the ongoing ELATIVE open-label extension demonstrate stabilization of non-invasive markers of fibrosis after two-years of treatment, suggesting IQIRVO may slow worsening of fibrosis in patients with PBC.
• An analysis of the effect of IQIRVO on bone mineral density and biomarkers of bone turnover after 52 weeks in the ELATIVE study suggests there is no negative impact on bone health in patients with PBC taking IQIRVO.

New long-term data from the open-label extension studies with Bylvay in PFIC and Kayfanda in ALGS provide a growing body of evidence for these rare liver diseases:

• Data from PEDFIC-2 on patients 18 years and older with PFIC treated with Bylvay demonstrated clinically meaningful improvements in pruritus and reductions in serum bile acid. This further characterizes the clinical benefits of long-term Bylvay therapy for a disease associated with paediatrics and commonly misdiagnosed in adults.
• For patients with ALGS, efficacy and safety outcomes were studied for patients aged 10 years and older from the ASSERT-EXT long-term extension study. Bylvay, known as Kayfanda in EU for ALGS, was found to be well-tolerated, with patients achieving clinically meaningful improvements in pruritus, reductions in serum bile acid and improvements in sleep outcomes out to 72 weeks.

With two molecules approved for three different rare cholestatic liver diseases and a pipeline with two additional late-stage indications, Ipsen is leading research and development for treatments where unmet need is high and treatments are often few or do not exist. By focusing on conditions like PSC, PBC, PFIC and ALGS, Ipsen aims to improve patient outcomes and quality of life. The company’s commitment is reflected in its rigorous clinical trials and ongoing efforts to develop effective treatments. This demonstrate Ipsen’s unwavering support for patients affected by these challenging and often debilitating diseases.

Data presentations at EASL

ENDS

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^® /Kayfanda^® (odevixibat)
Bylvay (known as Kayfanda^® in E.U. in ALGS) (odevixibat) is a once-daily non-systemic ileal bile acid transport (IBAT) inhibitor. Odevixibat was approved in July 2021 under exceptional circumstances in the E.U. under the brand name Bylvay^®, as the first drug treatment option for all types of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older, and in June 2021 in the U.S. under the brand name Bylvay^®, as the first drug treatment option for patients 3 months of age and older living with cholestatic pruritus due to PFIC. Bylvay has received orphan exclusivity for the treatment of PFIC in the E.U. and in the U.S.

Odevixibat was approved in September 2024 under exceptional circumstances in the E.U. under the brand name Kayfanda^® for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. 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.

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.

Attachment

• Ipsen PR_EASL Media Update_06052025