IPSEY News & Events

Ipsen announced that new Primary Biliary Cholangitis data with IQIRVO from the ELATIVE trial will be presented in two late-breaking sessions, at The Liver Meeting( 2025, hosted by the American Association for the Study of Liver Diseases. In interim data from the ELATIVE long-term, open label extension trial, which includes over three years of follow-up in 115 patients with PBC, observed that IQIRVO delivers sustained improvements in biomarkers of cholestasis and stabilization in markers of fibrosis, with IQIRVO treatment, suggesting potential for slowing disease progression. In addition, consistent trends in improvements were shown in fatigue and pruritus symptoms. At week 182, 72% of patients receiving IQIRVO maintained a biochemical response, with a 47% reduction in alkaline phosphatase from baseline. The proportion of patients achieving normal ALP levels remained consistent with previously presented Phase III results from the ELATIVE trial. Improvements in patients with moderate-to-severe fatigue were sustained, with similar results observed for pruritus and data also confirmed a long-term, well-characterized safety profile with no new safety signals. In a second late-breaking presentation of a further analysis of the ELATIVE trial, showing the relationship between changes in the expression of fatigue-associated proteins and reported outcomes of fatigue in patients on IQIRVO, was presented. The clinical findings from this analysis are consistent with previously published mechanistic data, suggesting that PPAR alpha/delta agonist activation may modulate key pathways involved in energy metabolism and mitochondrial function. Fatigue remains one of the most common and burdensome symptoms for patients with PBC. Currently, there are no therapies approved to address it, however, clinically meaningful improvements in fatigue have been observed with IQIRVO, which is a PPAR alpha/delta agonist, versus placebo, in the ELATIVE trial with around one in two patients reporting a significant reduction in fatigue severity4 Together, these data support further research into how IQIRVO may help address fatigue in PBC.

Ipsen announced that Japan's Ministry of Health, Labour and Welfare has granted regulatory approval for Bylvay for the treatment of pruritus associated with progressive familial intrahepatic cholestasis. PFIC is a group of rare genetic disorders in which bile acid accumulates in the liver, leading to progressive liver damage and potentially liver failure. The condition severely impacts quality of life through debilitating symptoms such as severe itching, caused by the bile accumulation in the liver and bloodstream, which can cause skin mutilation, sleep disruption, irritability, and impaired cognitive and social development. The approval from the MHLW was based on data from a Phase III, open-label study conducted in Japan, which evaluated the efficacy and safety of odevixibat in pediatric patients with PFIC types 1 and 2. The study confirmed improvements in serum bile acid levels and pruritus consistent with the global Phase III PEDFIC results. The Phase III clinical development plan for Bylvay in Japan was managed by Jadeite Medicines Inc and as part of the strategic collaboration, the new drug application for this indication was transferred to Ipsen headquartered in Tokyo, Japan. Ipsen will also be responsible for commercialization of Bylvay in Japan.