Mirum 的 LIVMARLI 三期 EXPAND 达主要终点，拟于 2027 上半年提交 sNDA 扩展胆道闭锁等超罕见胆汁淤积性肝病

Mirum Pharmaceuticals, Inc. (MIRM), a rare disease-focused biopharmaceutical company, announced positive topline results from the Phase 3 EXPAND study of LIVMARLI (maralixibat) in patients with ultra-rare cholestatic liver diseases. The study met its primary endpoint, and shares were trading higher following the announcement.
Cholestatic pruritus is persistent itching associated with impaired bile flow and the accumulation of bile acids. It can significantly affect quality of life in patients with rare liver diseases, including biliary atresia. Treatment options remain limited for several of these conditions.
LIVMARLI (maralixibat) is an orally administered ileal bile acid transporter (IBAT) inhibitor that reduces the reabsorption of bile acids in the intestine. The drug is designed to improve cholestatic pruritus and reduce serum bile acid levels. It is already approved in the U.S. for cholestatic pruritus associated with Alagille syndrome and progressive familial intrahepatic cholestasis.
Phase 3 EXPAND Trial Details
The Phase 3 EXPAND study enrolled 52 patients, including 46 pediatric and six adult participants, with ultra-rare cholestatic liver diseases. Participants were randomized to receive maralixibat at 285 micrograms per kilogram twice daily or placebo during a 20-week double-blind treatment period.
In the pediatric population, maralixibat produced a mean 1.81-point improvement in the ItchRO(Obs) severity score during Weeks 13-20, compared with a 0.83-point improvement for placebo. The placebo-adjusted treatment difference was 0.98 points (p=0.0004).
The treatment also reduced serum bile acids by 70.1% from baseline, compared with 3.5% for placebo, representing a 69% treatment effect versus placebo (p<0.0001). In patients with biliary atresia and a native liver, maralixibat significantly improved itch severity compared with placebo (p=0.0002).
Safety findings were consistent with previous clinical experience, and no new safety signals were observed. Diarrhea was the most common treatment-emergent adverse event in the maralixibat group, reported in 64% of patients compared with 11.1% in the placebo group.
Mirum's Regulatory Plans
Based on the positive EXPAND results, Mirum plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration in the first half of 2027. The filing is intended to support the potential use of LIVMARLI in additional ultra-rare cholestatic liver diseases, including biliary atresia.
Mirum's Pipeline Focus
Mirum's commercial portfolio includes LIVMARLI for Alagille syndrome and progressive familial intrahepatic cholestasis, along with CHOLBAM (cholic acid) for bile-acid synthesis disorders and CTEXLI (chenodiol) for cerebrotendinous xanthomatosis.
The company's investigational pipeline also includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis and primary biliary cholangitis; brelovitug, a monoclonal antibody in late-stage development for hepatitis delta virus; and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome.
MIRM has traded in a 52-week range of $63.23 to $130.00.
The stock closed Friday's trading at $86.01, up 5.73%.
In the premarket trading, shares are up 2.31% at $88.00.
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