Early trial results testing maralixibat for hard-to-treat itching due by year’s end
Rare liver disease patients still being recruited for global EXPAND study
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A global clinical trial testing Mirum Pharmaceuticals‘ maralixibat for treatment-resistant itching, or pruritus, associated with rare cholestatic conditions is expected to complete patient enrollment — now underway at more than 30 sites in a dozen countries — in the first half of this year.
Early data are then expected in the final months of the year, according to a Mirum press release announcing financial and corporate updates for 2025 and spanning 2026.
The ongoing Phase 3 trial, called EXPAND (NCT06553768), is recruiting an estimated 90 participants at 35 locations across North and South America, Europe, and the Middle East. It’s open to individuals 6 months and older who have cholestatic liver disease with pruritus that has not responded to other therapies.
Participants in the trial are randomly assigned to receive either maralixibat or a placebo for 20 weeks, or about five months. This is followed by another 20 weeks in which all will receive the experimental medication. The main goal is to determine whether maralixibat reduces itch severity across these liver-related conditions.
Cholestasis refers to a slowed or blocked flow of bile, a digestive fluid produced by the liver that normally travels to the intestines through tubes called bile ducts. When the flow of bile is blocked, bile builds up in the liver and leaks into the bloodstream, which can set the stage for irreversible liver damage and cause symptoms such as itching.
Maralixibat already approved as Livmarli for 2 liver diseases
Maralixibat is designed to reduce bile buildup by increasing the amount of bile excreted in a person’s stool. The therapy is currently approved in the U.S., the European Union, and Japan under the name Livmarli to treat pruritus associated with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC), two rare genetic disorders that cause cholestasis.
The EXPAND trial is open to individuals with other rare cholestatic conditions who will receive an oral solution containing 300 microgram/kg maralixibat or the placebo, given once daily in the first week and then twice daily thereafter.
Livmarli was originally developed as an oral solution, but a tablet version of the therapy was cleared for use in the U.S. last year. According to Mirum, net sales of Livmarli in 2025 totaled more than $300 million.
“2025 was an excellent year for Mirum, reflecting the strength and scalability of our purpose-built rare disease operating model,” said Chris Peetz, CEO of Mirum. “We delivered strong global commercial growth, advanced multiple late-stage clinical programs and are expanding our pipeline.”
The company noted that the timing of EXPAND has been “accelerated,” with “top-line data now expected [in the fourth quarter of] 2026.”
