Ipsen Reports Positive Late-Stage Results for PBC Therapy

Rare Daily Staff

Ipsen reported that its liver disease drug Iqirvo significantly improved a key marker of disease control in people with primary biliary cholangitis, according to results from a late-stage clinical trial.

In the phase 3b ELSPIRE study, 85 percent of patients treated with Iqirvo achieved normalization of alkaline phosphatase (ALP), compared with 23 percent of those receiving placebo after one year. ALP is an enzyme associated with liver damage, and normalizing its levels is increasingly viewed as a critical goal in managing PBC.

PBC is a rare autoimmune disease in which bile accumulates in the liver, leading to inflammation and progressive damage that can ultimately result in liver failure. Although existing treatments such as ursodeoxycholic acid (UDCA) are effective for many patients, a substantial proportion do not respond adequately.

Iqirvo works by activating the nuclear receptors PPAR-alpha and PPAR-delta, which regulate bile acid metabolism and reduce liver inflammation. The drug received accelerated approval in the United States in 2024 for patients with an inadequate response to standard therapy, with regulators requiring additional data to confirm long-term clinical benefit.

The ELSPIRE trial evaluated patients with moderately elevated ALP levels who had not responded sufficiently to standard treatment. The study enrolled 92 patients across 10 countries and compared once-daily Iqirvo with placebo over 52 weeks. The drug’s safety profile was consistent with previous studies, with no new safety signals identified.

Ipsen said it plans to present full results at an upcoming medical meeting and submit the data to regulatory authorities, potentially supporting broader use of the therapy.

The findings may also expand the eligible patient population for Iqirvo. By targeting patients with less severe but still abnormal ALP levels, the drug could be used earlier in the disease course, potentially slowing progression before significant liver damage occurs.

“Normalizing ALP is not just a biochemical outcome, but a tangible objective to delay the need for a liver transplant and improve prognosis for PBC patients,” said Christelle Huguet, Head of R&D at Ipsen. “These results contribute to the growing body of evidence supporting the potential of Iqirvo to help patients achieve ALP normalization.”