Ipsen’s elafibranor shows promise in rare liver disease primary sclerosing cholangitis
Ipsen’s elafibranor (sold under the brand name Iqirvo) has shown promise as a treatment for primary sclerosing cholangitis (PSC), a rare liver disease that currently has no approved treatment options.Results from the phase 2 ELMWOOD study, which has been comparing two doses of the drug against placebo in 68 PSC patients, will be presented at this year’s European Association for the Study of the Liver congress.
PSC is a chronic disease characterised by inflammation and scarring of the bile ducts, which can results in liver damage and eventually liver failure. Symptoms vary, but can include itching, fatigue, abdominal pain and jaundice, with patients also experiencing serious complications such as liver cirrhosis and an increased risk of liver cancer.
Liver transplantation is currently the only treatment that can significantly improve the prognosis of PSC, underscoring the need for new approaches.
Data from ELMWOOD demonstrated a positive safety and tolerability profile, the trial’s primary endpoint, for elafibranor-treated patients versus those receiving placebo.
Efficacy benefits were also observed in the elafibranor groups. Patients receiving elafibranor experienced significant dose-dependent reductions in alkaline phosphatase at week 12 compared to placebo, with improvements seen as early as week four.
Similar results were demonstrated in other biochemical liver parameters, Ipsen said, including alanine aminotransferase and gamma-glutamyl transferase, two key biochemical markers of disease progression.
Additionally, elafibranor was associated with stabilisation in enhanced liver fibrosis compared to placebo at week 12, and patients receiving a 120mg dose of Ipsen’s drug experienced improvements in itch compared to those on placebo.
Taken orally once daily, Iqirvo is a peroxisome proliferator-activated receptor agonist that decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters.
The drug recently gained approval in the US, EU and UK under the brand name Iqirvo to treat primary biliary cholangitis, another rare liver disease.
Christelle Huguet, executive vice president and head of research and development at Ipsen, said the results from ELMWOOD “reinforce elafibranor’s action as a PPARα/δ agonist in potentially treating multiple liver diseases, like PSC”.
An open-label 96-week extension study evaluating the safety and efficacy of elafibranor 120mg in PSC is ongoing.
