PARIS, FRANCE, 24 JULY 2026 - Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Phase III BOLD trial evaluating Bylvay versus placebo for patients living with biliary atresia (BA) who have already undergone a Kasai hepatoportoenterostomy (HPE) did not meet the primary endpoint of improvement in native liver survival vs placebo. Current topline data is in line with the well-established safety profile of odevixibat in approved indications. Characterized as a rapidly progressive and complex condition, BA is the leading cause of paediatric liver transplant, with no approved therapeutic options beyond Kasai HPE or liver transplant surgery.
"Biliary atresia is a rare and serious liver disease that affects babies. Progressing rapidly and with no effective medical treatments, many children develop severe liver damage and biliary atresia remains the number 1 cause of liver transplantation in children, often before the age of 2. Research remains limited, leaving patients, families and clinicians with very few therapeutic options," said lead investigator Dr Saul J. Karpen MD PhD FAASLD, Pediatric Hepatologist and Chief Scientific Officer, Stravitz-Sanyal Institute for Liver Disease and Metabolic Health, Virginia Commonwealth University. "As the first global Phase III trial in biliary atresia, BOLD has generated the most comprehensive dataset ever assembled in this disease. Although the study did not meet its primary endpoint, the commitment of participating children and families has produced valuable insights that will deepen our understanding of biliary atresia and provide a crucial basis for future research and patient care. As biliary atresia is a heterogeneous disease with variable clinical presentation and progression, further analyses of this comprehensive dataset may provide important insights into disease biology and help us better understand whether outcomes differ across patient subgroups."
"This outcome is disappointing for patients living with this serious disease and their families," said Christelle Huguet, PhD, EVP Head of R&D, Ipsen. "These results reflect the significant challenge biliary atresia presents as a complex, rare paediatric cholestatic liver disease which has so far evaded all therapeutic attempts beyond surgery. We would like to sincerely thank the patients, caregivers, investigators, and the broader biliary atresia community who dedicated their time and effort to the BOLD trial. Their commitment has been instrumental in helping advance our understanding of biliary atresia and the needs of those living with this challenging disease."
About Bylvay (odevixibat)
Odevixibat is a once-daily selective and potent ileal bile acid transport (IBAT) inhibitor that reduces bile acid reabsorption in the intestine. Through IBAT inhibition, the bile acids are instead diverted and excreted with faeces. Bylvay (odevixibat) is approved in the U.S. as the first treatment for cholestatic pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC) across all types, and for pruritus in patients with Alagille Syndrome (ALGS). In the EU Bylvay is approved for the treatment of PFIC, with orphan exclusivity granted, and is additionally marketed as KAYFANDA for ALGS.
About the BOLD Phase III trial
BOLD is a Phase III randomized, double-blind, placebo-controlled, trial (NCT04336722) investigating the efficacy and safety of odevixibat compared to placebo in patients with biliary atresia who have undergone a Kasai hepatoportoenterostomy (HPE). It is the largest trial evaluating disease modification in biliary atresia. The trial enrolled 254 patients across 19 countries who had undergone Kasai HPE surgery within the first 90 days of life. Patients received oral odevixibat 120mcg/kg/day or placebo once daily for up to 104 weeks. The primary endpoint was native liver survival, defined as time from randomization to first occurrence of liver transplant or death at week 104. An open-label extension study currently ongoing (BOLD-EXT) is evaluating the longer-term safety and efficacy of odevixibat in patients who have completed the BOLD trial. A decision regarding the continuation of patients in the open-label extension will be made following a comprehensive review of the full trial data.
About biliary atresia
Biliary atresia (BA) is a rare, serious pediatric cholestatic liver disease affecting 1 in 5,000-20,000 newborns in which the bile ducts inside and/or outside the liver are blocked, absent, or scarred, preventing bile from draining from the liver into the intestine. Bile then builds up in the liver, causing progressive inflammation, fibrosis, cirrhosis and ultimately liver failure if untreated. The condition appears in newborns and young infants, typically within the first weeks to months of life, often through persistent jaundice, pale stools and dark urine. Biliary atresia is the most common cause of pediatric liver transplantation worldwide. While an early Kasai HPE surgical procedure can restore bile flow and delay disease progression, it does not always cure the condition and many patients will require a liver transplant. Currently there are no approved medical treatments options for BA.
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 66 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 0517 |
| 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.
