Core Insights - Umecrine Cognition, a portfolio company of Karolinska Development, is presenting new clinical scale data for Primary Biliary Cholangitis (PBC) at the EASL Congress 2025 in Amsterdam [1][2]. Company Overview - Karolinska Development AB holds a 73% ownership stake in Umecrine Cognition [5]. - The company focuses on identifying and investing in breakthrough medical innovations in the Nordic region, aiming to develop these into commercial products [6][8]. Drug Development - Umecrine Cognition is developing golexanolone, a drug candidate currently in a Phase 2a clinical study for PBC [2]. - The newly developed CGI-S-PBC™ scale is designed to evaluate symptom severity in PBC patients, which cannot be measured by conventional laboratory tests [3]. Clinical Research - The CGI-S-PBC™ scale expands on the PBC-40 patient-reported outcome measure and has been validated in two separate studies [3]. - Abstracts summarizing the validation and implementation of CGI-S-PBC™ will be presented at the EASL Congress, focusing on its role as a clinical trials outcome tool [4].

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]