Mirum Pharmaceuticals MIRM announced that the phase III EXPAND study, which evaluated Livmarli (maralixibat) for treating cholestatic pruritus in patients aged six months and older with ultra-rare cholestatic liver diseases, including biliary atresia, has met its primary endpoint.
What Did MIRM’s EXPAND Study Data Show?
Data from the study showed that treatment with Livmarli led to a statistically significant improvement in cholestatic pruritus compared with placebo in the primary analysis population of pediatric patients.
Treatment with Livmarli showed a mean 1.81-point improvement from baseline in the average worst-daily ItchRO(Obs) severity score during weeks 13-20, compared with a 0.83-point improvement for placebo. Treatment with Livmarli also achieved a 69% reduction in serum bile acids versus placebo.
The EXPAND study also met all five prespecified secondary endpoints, including the three endpoints evaluated in patients with biliary atresia and a native liver. In these patients, Livmarli demonstrated a statistically significant improvement in cholestatic pruritus compared with placebo.
Based on the positive data from the EXPAND study, Mirum plans to submit a supplemental new drug application (sNDA) for Livmarli to the FDA in the first half of 2027.
In March 2026, Mirum completed enrollment in the phase III EXPAND study.
Year to date, shares of Mirum have increased 9% compared with the industry’s rise of 3.7%.
Image Source: Zacks Investment Research
MIRM Thrives on Livmarli Sales Uptake
Mirum’s lead marketed drug, Livmarli, is presently approved for treating cholestatic pruritus in patients with Alagille syndrome worldwide. The drug is also approved for treating certain patients with progressive familial intrahepatic cholestasis in the United States and Europe.
The FDA has also approved a new tablet formulation of Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis. The oral tablet, which was launched in the United States in June 2025, is likely to offer convenience for older patients.
Sales of Livmarli have been rising steadily since its approval and launch worldwide. In the first half of 2026, Livmarli generated sales worth $242.5 million, indicating an increase of 50.2% on a year-over-year basis. The continued demand for Livmarli is driving the top line and the momentum is expected to continue for the rest of 2026. Reflecting the strong performance of its business so far in 2026, Mirum now expects total revenues to be in the range of $680-$700 million in 2026, compared with the previous expectation of $660-$680 million.
Potential label expansion of Livmarli is likely to drive sales further in the future quarters.
MIRM's Zacks Rank & Other Stocks to Consider
Mirum currently carries a Zacks Rank #2 (Buy).
Some other top-ranked stocks in the biotech sector are Werewolf Therapeutics HOWL, Arvinas ARVN and Eton Pharmaceuticals ETON, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Werewolf Therapeutics’ 2026 loss per share have narrowed from $1.49 to 89 cents, while loss per share estimates for 2027 have narrowed from $2.20 to 59 cents during the same time. HOWL’s shares have gained 55% year to date.
Werewolf Therapeutics’ earnings beat estimates in each of the trailing four quarters, with the average being 47.49%.
Over the past 60 days, estimates for Arvinas’ 2026 bottom line have improved from a loss of 27 cents per share to earnings of 5 cents, while loss per share estimates for 2027 have narrowed from $3.44 to $3.37 during the same time. ARVN shares have declined 37.1% year to date.
Arvinas’ earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 279.07%.
Over the past 60 days, estimates for Eton Pharmaceuticals’ 2026 earnings per share have risen from 75 cents to $1.13, while estimates for 2027 have increased from $1.91 to $1.94 during the same time. ETON shares have skyrocketed 226.8% year to date.
Eton Pharmaceuticals’ earnings beat estimates in one of the trailing four quarters, while missing the same on the remaining three occasions, with the average negative surprise being 11.00%.
7 Best Stocks for the Next 30 Days
Just released: Experts distill 7 elite stocks from the current list of 220 Zacks Rank #1 Strong Buys. They deem these tickers "Most Likely for Early Price Pops."
Since 1988, the full list has beaten the market more than 2X over with an average gain of +23.9% per year. So be sure to give these hand picked 7 your immediate attention.
See them now >>Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
Mirum Pharmaceuticals, Inc. (MIRM): Free Stock Analysis Report
Arvinas, Inc. (ARVN): Free Stock Analysis Report
Eton Pharmaceuticals, Inc. (ETON): Free Stock Analysis Report
Werewolf Therapeutics, Inc. (HOWL): Free Stock Analysis Report
This article originally published on Zacks Investment Research (zacks.com).