Ipsen, Genfit Build Case for Rare Liver Disease Drug with Phase III Data
Pictured: Illustration of diseased liver/iStock, Mohammed Haneefa Nizamudeen
Ipsen and partner Genfit on Monday posted promising data from the Phase III ELATIVE trial for their investigational dual peroxisome activated receptors agonist elafibranor, further building the candidate’s case for regulatory approval in primary biliary cholangitis.
More than half of patients treated with elafibranor achieved the study’s primary endpoint of biochemical response, defined as having normal bilirubin concentrations alongside a substantial decrease in alkaline phosphatase levels, maintained below a particular threshold. Only 4% of placebo comparators met these biochemical response criteria.
Elafibranor also normalized alkaline phosphatase levels in 15% of treated patients, compared to none in the placebo group.
In terms of safety, ELATIVE found that elafibranor was well-tolerated with an adverse event profile that was similar to placebo. Common side effects included abdominal pain, diarrhea, nausea and vomiting.
In a statement, Christelle Huguet, executive vice president and head of R&D at Ipsen, said elafibranor could be a “paradigm-changing treatment” for primary biliary cholangitis (PBC) and satisfy patients’ needs for an effective second-line therapy option.
The pharma partners presented these data Monday in a late-break oral session during The Liver Meeting 2023 of the American Association for the Study of Liver Diseases, with a simultaneous publication in The New England Journal of Medicine.
Monday’s data drop follows a topline readout in June 2023, which showed that elafibranor could elicit a significantly higher rate of clinically meaningful cholestasis response than placebo as well as induce stronger pruritus improvements, as quantified by the PBC Worst Itch Numerical Rating Scale score.
Ipsen and Genfit are gearing up for a regulatory filing for elafibranor, which will include data from ELATIVE, according to the companies’ announcement on Monday.
Designed to be orally available and taken once a day, elafibranor is a small molecule drug candidate that targets and activates both the alpha and data peroxisome activated receptors (PPAR), improving lipid metabolism, as well as reducing inflammation, fibrosis and steatosis—all disease hallmarks of PBC.
Elafibranor was initially being developed by Genfit, which decided in July 2020 to discontinue the development of the candidate for non-alcoholic steatohepatitis (NASH) following disappointing Phase III data.
Ipsen picked the candidate up in December 2021 as part of a long-term strategic global partnership with Genfit. Ipsen made a $130 million upfront payment for the PPAR agonist, along with access to future clinical programs from Genfit.