Ipsen shares long-term elafibranor efficacy and itch-related quality of life data in primary biliary cholangitis patients

Ipsen, a global biopharmaceutical company, recently unveiled promising new data from the ELATIVE® Phase III clinical trial at the European Association for the Study of the Liver (EASL) Congress. The trial results demonstrate the sustained efficacy of elafibranor in managing primary biliary cholangitis (PBC), a progressive autoimmune liver disease, over a 78-week treatment period.

The ELATIVE® Phase III trial, which is a multi-center, randomized, double-blind, placebo-controlled study, evaluated the efficacy and safety of elafibranor for patients with PBC who exhibited inadequate response or intolerance to ursodeoxycholic acid (UDCA), the current first-line therapy. The trial enrolled 161 patients who were divided into two groups, receiving either elafibranor 80mg or a placebo, with some continuing their UDCA treatment as well.

At the 78-week mark, the trial data revealed that 70% of patients treated with elafibranor met the composite endpoint of biochemical response, compared to 0% of patients on placebo. The composite endpoint was defined by achieving certain levels of alkaline phosphatase (ALP) and total bilirubin (TB), both of which are critical predictors of PBC progression. These results underline the potential of elafibranor to slow down disease progression and improve liver function by reducing liver injury markers.

Moreover, the itch-related quality of life for patients with moderate-to-severe pruritus saw significant improvements. This was measured using the PBC-40 and 5-D Itch questionnaires. Patients treated with elafibranor reported greater reductions in overall itch severity, better sleep quality, and fewer disruptions in daily functioning compared to those on placebo. For instance, by week 52, 58% of patients on elafibranor reported reduced itching duration versus 27% on placebo. Additionally, 80% of the elafibranor group experienced no sleep disturbance or only occasional delays, in contrast to 30% in the placebo group.

Sandra Silvestri, M.D., Executive Vice President and Chief Medical Officer at Ipsen, emphasized the importance of these findings. She noted that despite existing treatments, a substantial number of PBC patients continue to suffer from worsening disease and debilitating symptoms. The long-term data from the ELATIVE study highlight elafibranor's potential to offer a new, effective treatment option, ultimately aiding in the preservation of native liver function and improving the quality of life for patients.

Dr. Christopher Bowlus, a Professor of Gastroenterology and Hepatology from the University of California Davis, also stressed the dual importance of managing disease progression and alleviating distressing symptoms for PBC patients. He highlighted that the new data provide further evidence of elafibranor's ability to improve key prognostic markers of disease progression, as well as its potential to enhance the quality of life by reducing pruritus severity.

PBC is a rare liver disease characterized by the gradual destruction of the liver's small bile ducts due to an autoimmune response. If untreated, this can lead to severe scarring and eventual liver failure. PBC symptoms, including fatigue and itching, can significantly impact a person’s quality of life, making the management of the disease crucial even for asymptomatic individuals.

In addition to the ELATIVE trial data, Ipsen also presented findings from its broader rare liver disease portfolio, including treatments for progressive familial intrahepatic cholestasis and Alagille syndrome, further establishing its commitment to addressing unmet needs in liver disease treatment.