ELATIVE, a randomised PBC trial involving 161 patients, has successfully met its primary endpoint.
Ipsen and Genfit have announced positive data from the pivotal ELATIVE phase 3 study.
During the clinical trial, the safety and efficacy of elafibranor – an investigational dual agonist – is being assessed as a treatment among patients with the rare cholestatic liver disease, primary biliary cholangitis (PBC).
The individuals in question would already have had an inadequate response or intolerance to the current standard of care therapy – ursodeoxycholic acid (UDCA).
ELATIVE, which involves 161 patients, is a randomised, multi-location, placebo-controlled phase 3 study, with an open-label long-term extension and is evaluating elafibranor at a daily 80mg dose level.
The research has duly met its primary endpoint, with 51% of participating patients on elafibranor 80mg achieving a cholestasis response, compared with 4% on placebo. Meanwhile, elafibranor was well tolerated with a safety profile consistent with that witnessed across previously reported studies.
Howard Mayer, executive vice president and head of research and development at Ipsen, reflected: “These are encouraging results that suggest elafibranor could be an effective treatment to prevent progression of PBC in patients who have received UDCA. It has a good safety profile and was well-tolerated, and could provide an important new therapeutic option for long-term treatment of patients with this debilitating condition.”
He added: “PBC is a serious condition which, if not treated properly, can lead to progression of liver disease and ultimately liver failure.”
Pascal Prigent, chief executive officer at GENFIT, concluded: “We are pleased by these results because PBC remains a disease where significant unmet medical needs exist. This long-awaited trial outcome is therefore good news for patients and for healthcare professionals who need more options to improve the clinical management of patients with PBC.
“It is also a gratifying recognition of the quality of our team’s work and of GENFIT’s ability to innovate and deliver tangible results.”
PBC is a rare autoimmune cholestatic liver disease in which bile ducts are gradually destroyed.