Paris, France — November 7, 2025 — Leads & Copy — Ipsen (Euronext: IPN; ADR: IPSEY) has announced new data regarding IQIRVO® (elafibranor) in patients with Primary Biliary Cholangitis (PBC). Data from the ELATIVE trial will be presented in two late-breaking sessions at The Liver Meeting® 2025, which is hosted by the American Association for the Study of Liver Diseases (AASLD).
Interim data from the ELATIVE long-term, open-label extension trial, which includes over three years of follow-up in 115 PBC patients, showed that IQIRVO provides sustained improvements in cholestasis biomarkers and stabilizes fibrosis markers. This suggests the potential for slowing disease progression with IQIRVO treatment. Fatigue and pruritus symptom improvements also showed consistent trends.
Seventy-two percent of patients receiving IQIRVO maintained a biochemical response at week 182, with a 47% reduction in alkaline phosphatase (ALP) from baseline. The percentage of patients achieving normal ALP levels remained consistent with previously presented Phase III results from the ELATIVE trial. Sustained improvements were seen in patients with moderate-to-severe fatigue, with similar results for pruritus. Data confirmed a long-term, well-characterized safety profile with no new safety signals.
Dr. Cynthia Levy, Professor of Medicine, Division of Digestive Health and Liver Diseases, University of Miami, noted that PBC does not impact all patients the same way, making long-term treatment benefit data on disease biomarkers, liver tests, and symptom impact important. Dr. Levy added that the preliminary results, indicating potential improvement in pruritus and fatigue, are very encouraging. The ELATIVE trial data confirms elafibranor is an effective long-term treatment, with a reassuring and well-characterized safety profile.
A second late-breaking presentation of a further analysis of the ELATIVE trial showed the relationship between changes in the expression of fatigue-associated proteins and reported outcomes of fatigue in patients on IQIRVO. These clinical findings are consistent with previously published mechanistic data, suggesting that PPAR α/δ agonist activation may modulate key pathways involved in energy metabolism and mitochondrial function.
Fatigue is a common and burdensome symptom for PBC patients. Clinically meaningful fatigue improvements have been observed with IQIRVO in the ELATIVE trial versus placebo, with about one in two patients reporting a significant reduction in fatigue severity. These data support further research into how IQIRVO may help address fatigue in PBC.
Sandra Silvestri, MD, PhD, Executive Vice President, Chief Medical Officer, Ipsen, said that these findings underscore IQIRVO’s potential as a long-term treatment option that manages markers of disease progression and symptoms that impact the quality of life of people living with PBC, while also helping to understand the mechanisms behind fatigue.
ELATIVE is a multi-center, randomized, double-blind, placebo-controlled Phase III clinical trial, with an open-label long-term extension (NCT04526665). It is evaluating the efficacy and safety of elafibranor 80mg once daily versus placebo for PBC patients with an inadequate response or intolerance to ursodeoxycholic acid (UDCA). The trial enrolled 161 patients who were randomized 2:1 to receive elafibranor 80mg once daily or placebo. Patients with an inadequate response to UDCA continued to receive UDCA in combination with elafibranor or placebo, while patients unable to tolerate UDCA received only elafibranor or placebo. Patients continued their assigned treatment after Week 52 until all patients had completed their treatment, or for a maximum of 104 weeks. The open-label long-term extension of ELATIVE remains ongoing.
Iqirvo (elafibranor) 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). 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.
Primary biliary cholangitis (PBC) is a rare, autoimmune liver disease where a build-up of bile and toxins and chronic inflammation cause irreversible fibrosis of the liver and destruction of the bile ducts. Impacting approximately 100,000 people in the US and 165,000 people in Europe, 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.
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Henry Wheeler, Investor, henry.wheeler@ipsen.com +33 7 64 47 11 49
Khalid Deojee, Investor, khalid.deojee@ipsen.com +33 6 66 01 95 26
Sally Bain, Media, sally.bain@ipsen.com +1 857 320 0517
Anne Liontas, Media, anne.liontas.ext@ipsen.com +33 0767347296
Source: Ipsen
