Core Insights - Ipsen announced new data on IQIRVO for Primary Biliary Cholangitis (PBC) from the ELATIVE trial, to be presented at The Liver Meeting 2025 [1] Group 1: Clinical Trial Results - Interim data from the ELATIVE trial showed that IQIRVO led to sustained improvements in cholestasis biomarkers and stabilization of fibrosis markers over three years in 115 patients [2] - At week 182, 72% of patients on IQIRVO maintained a biochemical response, with a 47% reduction in alkaline phosphatase (ALP) from baseline [2] - Improvements in fatigue and pruritus symptoms were consistent, with a well-characterized safety profile and no new safety signals identified [2][4] Group 2: Expert Commentary - Dr. Cynthia Levy emphasized the importance of long-term treatment data for PBC, noting that IQIRVO shows effective treatment with a reassuring safety profile [3] - Sandra Silvestri highlighted IQIRVO's potential to manage disease progression markers and improve quality of life for PBC patients [4] Group 3: Mechanistic Insights - Further analysis from the ELATIVE trial indicated a relationship between changes in fatigue-associated proteins and reported fatigue outcomes in patients treated with IQIRVO [3] - The data suggests that IQIRVO, as a PPAR α/δ agonist, may modulate pathways involved in energy metabolism and mitochondrial function, addressing fatigue in PBC patients [3][6] Group 4: Trial Design and Background - The ELATIVE trial is a Phase III, multi-center, randomized, double-blind, placebo-controlled study evaluating elafibranor 80mg once daily versus placebo for PBC patients with inadequate response to ursodeoxycholic acid (UDCA) [5] - The trial enrolled 161 patients, with ongoing open-label long-term extension [5] Group 5: Product Information - IQIRVO (elafibranor) is an oral PPAR agonist that improves cholestasis and has anti-inflammatory effects, granted Breakthrough Therapy Designation by the FDA in 2019 [7] - It received accelerated approval from the FDA in June 2024 and conditional approval from the EMA and UK MHRA in late 2024 for treating PBC [7] Group 6: Disease Context - Primary Biliary Cholangitis is a rare autoimmune liver disease affecting approximately 100,000 people in the US and 165,000 in Europe, primarily women [8]

Core Insights - Ipsen announced new data on IQIRVO (elafibranor) at the European Association for the Study of the Liver congress, highlighting its efficacy in treating primary biliary cholangitis (PBC) [1] Group 1: Clinical Data and Efficacy - Additional analyses from the ELATIVE study indicate that patients with PBC treated with IQIRVO experienced greater improvements in fatigue after 52 weeks compared to placebo, with 42.9% of IQIRVO patients showing improvement versus 31.3% in the placebo group [2] - Among patients with moderate-to-severe fatigue at baseline, 66.7% of those treated with IQIRVO achieved clinically meaningful improvements, compared to 31.3% in the placebo group [2] - The positive effect of IQIRVO on fatigue appears to occur independently of its effect on pruritus [2] Group 2: Mechanistic Insights - A comprehensive proteomic analysis revealed changes in the expression of over 20 proteins involved in inflammation, immune response, and key symptomatic domains of PBC, including fatigue, in patients treated with IQIRVO [3] - The observed effects on fatigue-associated proteomic signatures are linked to PPARα activation [3][6] Group 3: Treatment Context and Approvals - IQIRVO is an oral, once-daily PPAR agonist that improves cholestasis and has anti-inflammatory effects, and it was granted Breakthrough Therapy Designation by the FDA in 2019 [5] - The drug received accelerated approval from the FDA in June 2024, conditional approval from the European Commission in September 2024, and approval from the UK MHRA in October 2024 for treating PBC in adults with inadequate response to UDCA [5] - Ipsen licensed the exclusive worldwide rights to elafibranor from GENFIT in 2021, and IQIRVO is currently undergoing regulatory processes with other authorities [5] Group 4: Disease Context - PBC is a rare autoimmune liver disease affecting approximately 100,000 people in the US and 165,000 in Europe, predominantly women, and can lead to severe complications if untreated [4]