PARIS, France – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Phase IIIb ELSPIRE trial evaluating IQIRVO® (elafibranor) in patients with PBC with an Alkaline Phosphatase (ALP) 1-1.67 times the upper limit of normal (x ULN) met the primary endpoint with statistical significance, showing an ALP normalization rate of 85% with IQIRVO vs 23% for placebo (p=<0.0001) at Week 52. The safety profile of IQIRVO was consistent with the known profile and no new safety signals were identified. Ipsen plans to present these data at an upcoming medical meeting and submit to regulatory authorities.
“We now recognize that patients who maintain normal serum alkaline phosphatase (ALP) have the best outcomes in PBC; therefore, our treatment goals are evolving to aim for ALP normalization as the optimal treatment target for this progressive and chronic liver disease,” said Kris Kowdley, MD, Director, Liver Institute Northwest, Medical Director and Senior Scientific Advisor, Velocity Clinical Research, and Professor, Elson S. Floyd College of Medicine, Washington State University. “These data from the ELSPIRE trial show that ALP normalization is an achievable goal for patients with PBC and will hopefully improve the prognosis for people living with PBC.”
“Normalizing 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, PhD, EVP, Head of R&D, Ipsen. “These results contribute to the growing body of evidence on the potential of IQIRVO to help people achieve ALP normalisation.”
About IQIRVO® (elafibranor)
IQIRVO is an oral, once-daily, peroxisome proliferator-activated receptor (PPAR) agonist. Activation of PPARα and PPARδ decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters. IQIRVO is the first approved PPAR agonist with dual α and δ activation, shown to have complementary anti-inflammatory, anti-cholestatic, anti-fibrotic and metabolic effects. 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 in combination with ursodeoxycholic acid in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The FDA and EMA approvals are contingent on the further verification of clinical benefit. In addition to the U.S., E.U. and UK, IQIRVO is approved in Canada, Australia, Brazil and 13 other countries and is in regulatory processes with other authorities. IQIRVO was developed by GENFIT. Ipsen licensed the exclusive worldwide rights (except China, Hong Kong, Taiwan and Macau) to elafibranor from GENFIT in 2021 and expanded the geographical scope to China, Hong Kong, Taiwan and Macau in March 2026.
About the ELSPIRE Phase IIIb trial
ELSPIRE is a Phase IIIb randomized, double-blind, placebo-controlled study (NCT06383403) evaluating the efficacy and safety of elafibranor in adults with PBC who have had an inadequate response or intolerance to ursodeoxycholic acid, the existing first-line therapy for PBC. The trial enrolled 92 patients across 10 countries who had an ALP of 1-1.67 x ULN and were randomized 2:1 to receive IQIRVO 80 mg once daily or placebo. The primary endpoint of the trial was the proportion of patients with normalization of ALP at week 52. Data are based on patients in the IQIRVO treatment arm (n=62) compared to patients in the placebo treatment arm (n=30) within the treatment period, who achieved ALP normalization.
About PBC
PBC is a rare, autoimmune liver disease where a build-up of bile and toxins and chronic inflammation cause fibrosis of the liver and destruction of the bile ducts.4 With approximately 1.8 people per 100,000 diagnosed worldwide each year, 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. 5,6
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
+33 7 66 47 11 49
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Khalid Deojee
+33 6 66 01 95 26
Media
Sally Bain
+1 857 320 0517
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Anne Liontas
+33 7 67 34 72 96
References
1 Murillo Perez CF, et al. Am J Gastroenterol. 2020;115(7):1066–1074
2 European Association for the Study of the Liver Clinical Practice Guidelines: Primary Biliary Cholangitis. Available: https://easl.eu/publication/the-diagnosis-and-management-of-patients-with-primary-biliary-cholangitis/
3 American Association for the Study of Liver Diseases Practice Guidelines: Primary Biliary Cholangitis. Available: https://www.aasld.org/practice-guidelines/primary-biliary-cholangitis
4 Lleo et al. Primary biliary cholangitis. The Lancet. 2020;396:1915–1926.
5 Tan et al. Global Epidemiology of Primary Biliary Cholangitis: An Updated Systematic Review and Meta-Analysis. Clin Gastroenterol Hepatol. 2026 Mar;24(3):621-632. doi: 10.1016/j.cgh.2025.03.025.
6 Trivella et al. Primary biliary cholangitis: Epidemiology, prognosis, and treatment. Hepatol Commun. 2023 Jun 2;7(6):e0179. doi: 10.1097/HC9.0000000000000179