Mirum Pharmaceuticals, Inc. has announced promising results from its Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) for treating cholestatic pruritus in patients with ultra-rare cholestatic liver diseases. The study met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints, including in patients with biliary atresia.
This development is significant for Mirum, as it reinforces the efficacy of LIVMARLI and could lead to expanded treatment options for patients suffering from these conditions. The pharmaceutical industry, particularly in the area of pharmaceutical preparations, is likely to see heightened competition as companies strive to innovate and improve their offerings in response to these results.
Background
Mirum Pharmaceuticals, Inc. is a Delaware-based company focused on developing treatments for rare and ultra-rare liver diseases. The company operates under the Standard Industrial Classification (SIC) code 2834, which pertains to pharmaceutical preparations. Mirum's common stock trades on the Nasdaq under the ticker symbol MIRM. Recent filings include an 8-K on October 13, 2026, detailing the results from the Phase 3 study.
What the Filing Says
According to the filing dated October 13, 2026, Mirum reported that the Phase 3 EXPAND study enrolled 52 patients, including 46 pediatric and six adult patients. The study demonstrated a statistically significant improvement in pruritus compared to placebo, with a mean 1.81-point improvement in average worst-daily ItchRO(Obs) severity score during Weeks 13-20.
Additionally, treatment with maralixibat resulted in a 70.1% reduction in serum bile acids compared to a 3.5% reduction for placebo, indicating a substantial therapeutic effect. The safety profile of maralixibat was consistent with prior clinical experiences, with no new safety signals observed.
Impact
The results from the Phase 3 EXPAND study may significantly impact patients suffering from cholestatic liver diseases, particularly biliary atresia. If approved, LIVMARLI could provide a new treatment option for these patients, enhancing their quality of life. Additionally, the pharmaceutical industry may feel pressure to innovate as competitors respond to Mirum's positive findings.
What's Next
Mirum Pharmaceuticals plans to submit a supplemental New Drug Application in the United States in the first half of 2027 based on the Phase 3 EXPAND study results. This filing will be crucial for the potential commercialization of LIVMARLI as a treatment for cholestatic liver diseases.