RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea)

Structure/Sequence

Molecular FormulaC22H24O4S

InChIKeyAFLFKFHDSCQHOL-UHFFFAOYSA-N

CAS Registry824932-88-9

Clinical Trials associated with Elafibranor

NCT07387549

/ RecruitingPhase 3

A Phase III, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis

The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death.
In this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up.
During the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety.
Following the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.

Start Date14 May 2026

Sponsor / Collaborator

Ipsen SA

DRKS00039209

/ RecruitingNot Applicable

Safety and efficacy of elafibranor therapy in patients with primary biliary cholangitis and liver cirrhosis. - Elafibranor in patients with primary biliary cholangitis and liver cirrhosis.

Start Date16 Mar 2026

Sponsor / Collaborator-

NCT06730061

/ Active, not recruitingPhase 3

A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease.
PBC is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis).
PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. In this study all participants will receive a daily dose of elafibranor (the study intervention).
The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease.
There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored.
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to approximately 6 years

Start Date16 Jan 2025

Sponsor / Collaborator

Ipsen SA

100 Clinical Results associated with Elafibranor

100 Translational Medicine associated with Elafibranor

100 Patents (Medical) associated with Elafibranor

167

Literatures (Medical) associated with Elafibranor

01 Jul 2026·Gastroenterology & hepatology

A Review of Therapies for Primary Biliary Cholangitis.

Article

Author: Kowdley, Kris V ; Alamgir, Mariam ; Bhushan, Sheena ; Sohal, Aalam

Primary biliary cholangitis (PBC) is a rare, chronic, progressive autoimmune cholestatic liver disease that can lead to cirrhosis if left untreated. Ursodeoxycholic acid remains the standard first-line therapy; however, approximately 40% of patients exhibit an inadequate or partial biochemical response, and approximately 5% to 10% of patients are intolerant to the drug. Consequently, there remains a substantial need for effective second-line agents. Until recently, obeticholic acid (OCA) and fibrates were the only available second-line therapies. However, the therapeutic landscape for PBC has evolved significantly with the US Food and Drug Administration conditional approval of elafibranor and seladelpar as second-line therapies for patients without cirrhosis. Notably, OCA has been withdrawn as an available treatment option, further shifting the current treatment paradigm. This article aims to examine the current pharmacologic landscape of first-line and second-line PBC treatments, with an emphasis on clinical considerations and strategies for individualized treatment selection to optimize patient outcomes.

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

01 Jun 2026·TRENDS IN MOLECULAR MEDICINE

The emerging role of PPARs in primary biliary cholangitis

Review

Author: Palomer, Xavier ; Wahli, Walter ; Rodríguez-Calvo, Ricardo ; Vázquez-Carrera, Manuel ; García-Mateo, Sandra

Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease characterized by autoimmune-mediated destruction of intrahepatic bile ducts, leading to fibrosis, cirrhosis, and liver failure. Ursodeoxycholic acid remains the first-line treatment, but up to 40% of patients respond inadequately and continue to experience fatigue and pruritus. This therapeutic gap has recently been addressed by the approval of two new drugs, elafibranor and seladelpar, which activate peroxisome proliferator-activated receptors (PPARs). This review explores recently unveiled molecular mechanisms underlying the effectiveness of PPAR-targeting drugs in PBC, focusing on their effects on cellular immune regulation, bile acid production and toxicity, and hepatic fibrosis. Additionally, we examine current knowledge and ongoing challenges that will influence the roles of PPAR agonists in improving PBC treatment.

241

News (Medical) associated with Elafibranor

31 Jul 2026

·www.fiercebiotech.com

‘It’s making a difference’: Madrigal CEO talks strategy behind MASH shopping spree

Not content with navigating Rezdiffra’s trajectory to blockbuster territory, Madrigal Pharmaceuticals has spent 2026 shopping for a range of drugs that the company hopes could work alongside its metabolic dysfunction-associated steatohepatitis (MASH) trailblazer. Madrigal kicked off the year paying $50 million to Pfizer for the rights to a DGAT2i inhibitor, before spending $60 million the following month on six preclinical siRNA programs from China’s Ribo Life Science and its Swedish subsidiary Ribocure Pharmaceuticals that Madrigal wants to use to silence genes implicated in MASH. The company explained at the time that both deals were aimed at securing therapies that could be combined with Rezdiffra. The biopharma was back at the dealmaking table in May, paying $25 million upfront to Arrowhead Pharmaceuticals for a phase 1-stage RNA interference therapeutic that had previously been rejected by Johnson & Johnson. Early clinical data had suggested the asset could reduce liver fat in a mutation found in 30% of MASH patients, including high prevalence in Hispanic populations. Combined with a preclinical GLP-1 agonist licensed from China’s CSPC Pharmaceutical for $120 million upfront last year, Madrigal now has a diverse array of MASH assets coming up behind Rezdiffra. So what’s the strategy behind this rapid pipeline expansion? “The question early on was what mechanisms we wanted,” Madrigal CEO Bill Sibold told Fierce in an interview at the company’s office in Waltham, Massachusetts. “My answer was: we want all of them,” Sibold continued. “We want anything that makes sense mechanistically or has proven already to have an effect on MASH.” It’s a sign of just how much Rezdiffra has changed the game since being approved as the first-ever treatment for MASH in 2024. Before that point, Sibold said he had counted 23 drugs across the industry that had failed to treat the fatty liver disease previously known as nonalcoholic steatohepatitis (NASH). This rollcall of failure meant MASH had become “the graveyard of drug development,” lumped in with other difficult-to-treat conditions like sepsis and lupus, he remembered. The disease occurs when fat builds up in the liver, causing inflammation and eventually cell death. It is the leading cause of liver transplants for women and the second biggest cause for men in the U.S. “Industry had been really interested in this area for a long time, but nobody could crack the nut,” Sibold said. Many companies tried and failed to find a breakthrough. Gilead’s ASK1 inhibitor selonsertib and its LOXL2 enzyme-targeting simtuzumab both flopped in the clinic, as did Genfit’s dual PPAR alpha/delta agonist elafibranor and Conatus Pharmaceuticals' pan-caspase inhibitor emricasan. Rezdiffra’s origins go back to Roche. Madrigal founder Rebecca Taub, Ph.D., left the Swiss pharma’s metabolic unit to work for VIA Pharmaceuticals, which licensed the thyroid hormone receptor (THR)-β agonist. Madrigal assumed the rights to that agreement when it was founded in 2011 and continued to develop the therapy. Sibold remembers getting a call from a recruiter about the Madrigal CEO position in 2023 while he was president for North America at Sanofi, having helped launch immunology blockbuster Dupixent. “There's no way I'm going to leave this to go to that,” he remembers of his initial reaction to the offer. But as Sibold dug into the company, educating himself on the trials and the science behind its lead asset, he became convinced that this was a unique opportunity to make a special impact. The year after Sibold joined up, Madrigal had secured Rezdiffra’s historic FDA approval. As well as the company's headquarters in West Conshohocken, Pennsylvania, the company now also has an office in Massachusetts—which Fierce visited—as well as an international outpost in Switzerland. “There were so many Big Pharma companies trying to solve [MASH], and this little Pennsylvania company of very few people had the tenacity, the guts and the grit to get there,” Sibold said. Madrigal reported $958.4 million in annual net sales for the drug in 2025. By the second quarter of this year, Rezdiffra was bringing in $364.3 million, equivalent to year-over-year growth of 71%. Sibold told Fierce that it wasn’t always this way. Early on, a MASH advocate told him: “We didn't know whether to believe you at first because it seemed like such a huge hill to climb that you guys didn't even have an approved product. But you are doing exactly what you said you would do.” After establishing itself as the market leader in MASH, what’s next? Does the company stay in its lane, expand into immunology targets or look at new indications? For Sibold, the answer lies in Madrigal’s five-word purpose: leading the fight against MASH. The biopharma wants to continue working with the MASH community it has come to know so well to build out its pipeline with more effective and comprehensive treatments for different subpopulations. Rather than generate data and hope to get bought by a bigger fish, Sibold said he is planning for an independent and sustainable company 10 to 20 years down the line. His vision is for MASH treatments to grow like other specialty markets, such as rheumatoid arthritis, psoriasis or inflammatory bowel disease, with dozens of products that generate tens of billions of dollars in annual sales. “We feel like MASH will emerge like that, and as a result, we want to have the portfolio of products that can best help all or subpopulations of patients,” Sibold said. Unlike a Big Pharma, where MASH may be one of a myriad of indications being focused on, Sibold suggested Madrigal's strength comes from the fact it is committed to the disease. This approach is reflected in trial enrollment and feedback, he said. “The community values expertise, and I think we've made a conscious decision to be experts in MASH,” he said. “Through expertise comes insight into what might be the next best mechanism, or the next best mechanism to combine with your current mechanism, or the best way to design the next trial.” Because reducing liver fat is so important to preventing or treating MASH, GLP-1s are moving into the space and posing a potential threat. Last year, Novo Nordisk’s Wegovy was approved to treat MASH, and some people have warned Sibold that Madrigal could be crushed by GLP-1s in the future. Instead, the CEO insisted more activity in the space is a good thing for Madrigal. “It's great to have another company who's putting resources into diagnosis, awareness and treatment,” he said. “There are some patients that are getting Wegovy, but it is helping to grow the market. It's not to our detriment.” But other Big Pharmas are circling. Last year saw GSK acquire the FGF21 analog efimosfermin from Boston Pharmaceuticals for $1.2 billion upfront, while Roche picked up a similar drug called pegozafermin as part of its $3.5 billion buyout of 89bio . Madrigal may be looking to become the MASH version of what Vertex Pharmaceuticals has achieved in cystic fibrosis, but that doesn’t mean Sibold and his team couldn’t eventually broaden their focus. “Would we look at other diseases? Yes. We would in time, or if the biology takes us there,” he told Fierce. But for now, the company is leaning into MASH specialization, developing relationships with patient groups, clinicians and researchers while raising Rezdiffra’s profile and growing a specialized portfolio of complementary therapies. “It's been fun. It's making a difference. It's helping patients,” Sibold said. “This is as good as it gets.

AcquisitionPhase 1Drug Approval

30 Jul 2026

·www.biospace.com

Ipsen delivers excellent H1 2026 results and upgrades its full-year guidance

H1 2026 results with sales growth of 23.5% at CER 1 , or 20.4% as reported, with the portfolio beyond Somatuline ® achieving sales growth of 24.8% 1 and core operating income of €845m, growing by 28.8% as reported, with a core operating margin of 38.6% of total sales, increasing by 2.5 points Upgraded FY 2026 financial guidance 2 : total sales growth greater than 20.0% at CER (prior guidance: greater than 13.0% at CER); core operating margin greater than 37.0% of total sales (prior guidance: greater than 35.0%) Delivering on strategy to accelerate the growth of key medicines and expand the pipeline, adding multiple anticipated near-term launches of assets with blockbuster potential across the three therapeutic areas. Driven by several positive Phase III readouts, for Dysport ® in episodic and chronic migraine, Iqirvo ® in primary biliary cholangitis and the acquisitions of Kartos Therapeutics 3 and Memo Therapeutics AG PARIS, FRANCE, 30 July 2026 - Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the first half of 2026. Extract of consolidated results H1 2026 H1 2025 % change €m €m Actual TCC Total Sales 2 190,2 1 819,8 20,4 % 23,5 % Core Operating Income 844,9 655,8 28,8 % Core operating margin 38,6 % 36,0 % +2,5 pts Core Consolidated Net Profit 607,7 508,3 19,6 % Core earnings per share (fully diluted) 7,33 € 6,07 € 20,7 % IFRS Operating Income 564,8 451,6 25,1 % IFRS operating margin 25,8 % 24,8 % +1,0 pts IFRS Consolidated Net Profit 402,6 335,5 20,0 % IFRS earnings per share (fully diluted) 4,86 € 4,00 € 21,4 % Free Cash Flow 651,7 483,2 34,9 % Closing net cash/(debt) 1 004,9 487,6 n/a “Our excellent first-half performance demonstrates the strength of Ipsen’s strategy, with all three therapeutic areas contributing to growth and the portfolio beyond Somatuline continuing to accelerate,” said David Loew, Chief Executive Officer, Ipsen. “This momentum enables us to upgrade our 2026 guidance while continuing to invest in innovation and future growth. Importantly, we have significantly strengthened our late-stage pipeline in the first half, with positive Phase III lifecycle management readouts for Dysport in episodic and chronic migraine and Iqirvo in primary biliary cholangitis, alongside the completed acquisition of Memo Therapeutics AG and the proposed acquisition of Kartos Therapeutics. These transactions add two innovative clinical assets—navtemadlin and potravitug—with the potential to address significant unmet needs and create long-term value. Our expanding pipeline and focused business development strategy further contribute to Ipsen’s sustainable growth outlook and our ability to bring meaningful innovation to patients worldwide.” Full-year 2026 guidance Based on the strong performance in the first half, Ipsen upgrades its financial guidance for 2026: ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Total sales growth greater than 20.0%, at constant currency. Based on the average level of exchange rates in June 2026, an adverse effect on total sales of around 1% of currencies is expected Core operating margin greater than 37.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external innovation opportunities and assumes the dilutive impact of the acquisitions of Memo Therapeutics AG and Kartos Therapeutics Guidance 4 on total sales and core operating margin in 2026 is assuming accelerated sales growth of the portfolio excluding Somatuline and the continued growth of Somatuline sales despite potential entry of generic lanreotide. Pipeline progress since Q1 2026 Ipsen presented in May late-breaking Phase II data in Glabellar Lines for corabotase, its first-in-class recombinant neuroinhibitor (RNI™), demonstrating a rapid onset of action, a statistically superior peak effect versus placebo at Week 4 and a clinically significant sustained duration of effect, outperforming both placebo and Dysport at Week 24. Corabotase has been recognized by the WHO (World Health Organization) and USAN (United States Adopted Name) as a novel botase molecule. Ipsen announced positive topline Phase III results from the BEOND migraine program in July, with Dysport meeting the primary endpoint in both the episodic, E-BEOND and chronic, C-BEOND trials by demonstrating statistically significant reductions in monthly migraine days versus placebo. These results make Dysport the first botulinum toxin to show efficacy in Phase III trials across both episodic and chronic migraine prevention. Dysport was well tolerated, with a safety pro with its established use and no new safety signals identified. Ipsen intends to submit to regulatory authorities and detailed results will be presented at a future scientific congress. Ipsen announced positive topline results from the Phase IIIb ELSPIRE study of Iqirvo in primary biliary cholangitis (PBC) in July. The study met its primary endpoint, with 85% of patients receiving Iqirvo achieving alkaline phosphatase (ALP) normalization at Week 52 versus 23% on placebo (p=<0.0001), while maintaining a safety pro with previous studies and identifying no new safety signals. ELSPIRE evaluated patients with ALP levels of 1-1.67x ULN, and supports the potential to significantly expand the addressable population for Iqirvo. Ipsen plans to submit the data to regulatory authorities and present these data at an upcoming scientific congress. Ipsen announced in July that the Phase III BOLD trial evaluating Bylvay ® (odevixibat) versus placebo in patients with biliary atresia who had already undergone a Kasai hepatoportoenterostomy did not meet its primary endpoint of improved native liver survival. Topline safety data remained consistent with odevixibat's well-established pro its approved indications, with no new signals identified. External innovation Ipsen recently announced its agreement to acquire Kartos Therapeutics, a clinical-stage biopharmaceutical company, centered on navtemadlin, a Phase III oral MDM2 inhibitor for myelofibrosis designed to restore p53 tumor-suppressor activity in patients with a suboptimal response to ruxolitinib. Top-line data from the registrational POIESIS trial are expected in 2027. Under the terms of the agreement, Kartos shareholders will receive $450m upfront and may be eligible for up to $1.3bn in milestone payments, with closing anticipated by the end of Q3 2026, subject to customary conditions. Ipsen acquired Memo Therapeutics AG in July, a clinical-stage biotechnology company focused on potravitug, a Phase II antibody targeting BK polyomavirus (BKPyV), a frequent cause of nephropathy, graft loss and transplant failure in kidney transplant recipients. Potravitug received FDA Fast Track designation in 2023 and EU Orphan Drug designation in 2025. Under the terms of the agreement, Memo Therapeutics AG shareholders received €200m upfront, with the potential for total consideration exceeding €700m through development, regulatory and sales milestones. Consolidated financial statements The Board of Directors approved the condensed consolidated financial statements on 29 July 2026. The Company’s auditors performed a limited review of the H1 2026 condensed consolidated financial statements. The interim financial report, with regards to the regulated information, will be available on ipsen.com in due course, under the Reports and Accounts tab in the Investor Relations section. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CEST. Participants can access the webcast here . Analysts can join the call and ask questions by registering here . Calendar Ipsen intends to publish its year-to-date and third-quarter sales update on 22 October 2026. Notes All financial figures are in € millions (€m). The performance shown in this announcement covers the six-month period to 30 June 2026 (H1 2026) and the three-month period to 30 June 2026 (Q2 2026), compared to the six-month period to 30 June 2025 (H1 2025) and the three-month period to 30 June 2025 (Q2 2025), respectively, unless stated otherwise. Commentary is based on the performance in H1 2026, unless stated otherwise. 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 7 64 47 11 49 Khalid Deojee khalid.deojee@ipsen.com +33 6 66 01 95 26 Media Sally Bain sally.bain@ipsen.com +1 857 320 05 17 Anne Liontas anne.liontas.ext@ipsen.com +33 7 67 34 72 96 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 and risks arising from unexpected regulatory or political changes such as changes in tax regulation and regulations on trade and tariffs, such as protectionist measures, especially in the United States; 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 . 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period. 2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions. 3 Subject to satisfaction of customary deal closing conditions 4 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions. Attachment Ipsen PR_HY 2026_30072026

Phase 3Clinical ResultPhase 2Financial StatementOrphan Drug

14 Jul 2026

·firstwordpharma.com

Biopharma bites: Ipsen's Iqirvo notches PBC win, plus $55M for Drug Farm

Ipsen's Iqirvo helps patients with PBC and moderately elevated ALP levelsDrug Farm raises $55M to advance two lead assetsIpsen's Iqirvo helps patients with PBC and moderately elevated ALP levelsIpsen reported that its PPAR agonist Iqirvo (elafibranor) met the primary endpoint of the Phase IIIb ELSPIRE trial involving patients with primary biliary cholangitis (PBC) and alkaline phosphatase (ALP) levels 1 to 1.67 times the upper limit of normal who had an inadequate response or intolerance to first-line ursodeoxycholic acid.The study randomised 92 PBC patients to receive either 80-mg once-daily Iqirvo or placebo. At week 52, 85% of patients taking Ipsen's drug achieved ALP normalisation versus 23% for placebo (p=<0.0001). The company will present the results at an upcoming medical meeting and submit the data to regulators.Iqirvo is already cleared to treat patients with PBC, thanks to an accelerated FDA approval in 2024. However, the ELSPIRE data could double the patient population addressable by the PPAR agonist, Ipsen said. "Normalising ALP is not just a biochemical outcome, but a tangible objective to delay the need for a liver transplant and improve prognosis for PBC patients," said Christelle Huguet, EVP and head of R&D at Ipsen. "These results contribute to the growing body of evidence on the potential of Iqirvo to help people achieve ALP normalisation."Drug Farm raises $55M to advance two lead assetsDrug Farm, a biotech with offices in Shanghai and New York, closed a $55-million series D on Monday to advance its lead asset DF-003, as well as several preclinical and early-stage programmes. The round was co-led by Shanghai Pudong Leading Area Investment Center and Shanghai Puxing Collaborative Private Equity Fund Partnership Enterprise, with participation from new investors Tukar Capital, Fuzhou Xinhe Fund, Shenzhen Luohu Donghai Chempartner Investment Fund and Keyuan Pharma, as well as existing backers BioVeda China Fund, YD Capital, Jiashan State VC, Detong Capital, Wedo Capital and Biometas.Developed by Drug Farm's AI platform MedChem5, DF-003 — an ALPK1 inhibitor — is in a Phase Ib study for ROSAH syndrome. The funding will help the asset complete pivotal testing in the disease setting and generate proof-of-concept data in atherosclerotic cardiovascular disease.The fresh capital will also be used to continue clinical development of the company's next-most advanced asset, DF-006. The oral ALPK1 agonist immunomodulator has completed multiple ascending dose testing in patients with virologically suppressed, HBeAg negative, chronic hepatitis B.

Clinical ResultPhase 3Phase 1Drug ApprovalAccelerated Approval

100 Deals associated with Elafibranor

External Link

KEGG	Wiki	ATC	Drug Bank
D11208	Elafibranor	-	DB05187

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Primary Biliary Cholangitis	United States	Ipsen Biopharmaceuticals, Inc.	10 Jun 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cholangitis, Sclerosing	Phase 3	United States	Ipsen SA	14 May 2026
Fibrosis	Phase 3	United States	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Argentina	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Australia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Belgium	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Canada	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Chile	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Colombia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Czechia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Denmark	Genfit SA	01 Mar 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EASL2026	Not Applicable	Primary Biliary Cholangitis	296	Second-line therapy	ianbaohwcx(rhciealcze) = srfhuwxvkc fujnrnxyao (jhqnmtxgic ) View more	Negative	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis	296	UDCA monotherapy (No treatment escalation)	zogxlidaxe(pzftmbhdgi) = hawisqbjxu mtuveygrmt (ktokyorigg )	Negative	27 May 2026
ELEVATE-PBC (EASL2026)	Not Applicable	Primary Biliary Cholangitis	281	Elafibranor	vfvcvpxaux(bdvnwoclku) = fprxfzamqi jjuodhrcou (shogdcycdn ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis	76	Elafibranor	sijtjdpnyu(zushevvqzc) = tbaefwqnzj eccdhlfmgc (gpvobbnqmo ) View more	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	93	Elafibranor 80 mg	rypbbuhjgn(glykaghcke) = udkeybjrsz zekmpsjewd (qbnrqnqnnc, 0.15) View more	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	159	Elafibranor 80 mg	vtutzbedkc(uualqgbzyz) = kpzjzdrbck ndpswwfilx (cbtoiikaiz ) View more	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	159	Placebo	vtutzbedkc(uualqgbzyz) = vbykgnixel ndpswwfilx (cbtoiikaiz ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	31	Elafibranor	fjwkbwrufm(hzfavwssib) = Three patients discontinued ELA due to mild intolerance; no serious adverse events occurred. chdxwzrigw (jpwzkowffo ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	426	Seladelpar	vxbwlrioce(ucjitfxnhy) = ubdgyuobbk ruekglpnld (rcaxyzngck ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	426	Elafibranor	vxbwlrioce(ucjitfxnhy) = xnnjddsaau ruekglpnld (rcaxyzngck ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis	80	Elafibranor 80 mg/day (Observation)	zaijdaupfa(frfnbibqex) = nnitonnvru dyjpariois (azlxasjddh ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	321	Elafibranor (Baseline ALP >1 to <1.67× ULN)	hbnjunivkk(ekmhcqxrxf) = odxjxunwab rglbbtzouz (emnlvjwxqw )	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	321	Elafibranor (prior 2L Tx)	hbnjunivkk(ekmhcqxrxf) = pyqiwlfwxj rglbbtzouz (emnlvjwxqw )	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	138	Elafibranor 80 mg (continuous pts)	cftmxcwrxs(kopnedrbvh) = 4 muscle-related AEs occurred in the OLE (2 continuous ELA; 2 crossover ELA), none treatment-related. In pts receiving statins (continuous ELA: 19 [20.4%]; crossover ELA: 10 [22.2%]), no new muscle-related safety signals were detected. djccpekfee (bewcaoodmv )	Positive	27 May 2026

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