Gilead Sciences’ primary biliary cholangitis (PBC) therapy, Livdelzi (seladelpar), has been shown to diminish a key marker of disease activity in a traditionally underserved portion of patients – potentially teeing the drug up to expand into a wider patient population.
The Phase III IDEAL study (NCT06060665), the data of which is due to be presented at the ongoing Liver Meeting in Colorado, enrolled adults living with PBC who have alkaline phosphatase (ALP) levels above normal yet below the 1.67× upper limit of normal (ULN) threshold.
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In the study, 75.4% of patients receiving Livdelzi achieved normalisation of ALP levels versus 16.1% in the placebo arm at week 52. In this trial, Gilead deemed patients as responders if they achieved a 15% or more decrease in ALP levels from baseline, and if blood levels of the enzyme were ≤ 1.0× the ULN.
Researchers observed this effect as early as one month into treatment, with 52.3% of patients surpassing the ALP normalisation milestone at this time point versus 6.5% in the placebo group.
The IDEAL outcomes are significant, the company says, as ALP levels in the blood represent a key marker of both disease activity and long-term outcomes in patients with PBC.
According to Cynthia Levy of the University of Miami Miller School of Medicine, IDEAL also provides evidence that Livdelzi can improve biochemical responses in a group of patients that have traditionally been left out of pivotal trials evaluating second-line therapies. “People with PBC whose alkaline phosphatase remains elevated but below 1.67 times the upper limit of normal have been largely excluded from previous pivotal trials of second-line therapies,” she commented.
“These findings can inform individualised treatment discussions, taking into account each individual’s risk of disease progression, symptoms and potential benefits and risks of additional therapy,” Levy added.
Alongside its promising efficacy, researchers found Livdelzi to be safe and tolerable in this study, as there were no new safety concerns. There were also no treatment-linked serious adverse events (SAEs) in this trial.
With these positive results in the bag, Gilead is planning to engage with global regulators to discuss these results – which could fortify the company’s efforts to secure full approval for Livdelzi. In 2024, the US Food and Drug Administration (FDA) granted the drug accelerated approval for use in combination with ursodeoxycholic acid (UDCA) in adults with PBC who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
If the drug were to secure a label expansion, it could reach a larger population of PBC patients. Analysts at GlobalData, the parent company of Clinical Trials Arena, forecast a blockbuster future for Livdelzi, as they expect the drug’s sales to peak at over $1.7bn in 2030.
