Mirum Pharmaceuticals Meets Primary Endpoint In Phase 3 LIVMARLI Study

(RTTNews) - Mirum Pharmaceuticals (MIRM) announced positive topline results from the Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) for cholestatic pruritus in patients aged six months and older with ultra-rare cholestatic liver diseases, including biliary atresia.

The study enrolled 52 patients who received maralixibat or placebo during a 20-week double-blind period. In pediatric patients, maralixibat improved itch severity by 1.81 points compared with 0.83 points for placebo, achieving a statistically significant treatment difference of 0.98 points (p=0.0004).

Maralixibat also reduced serum bile acids by 70.1% from baseline, compared with 3.5% for placebo, representing a 69% treatment effect (p<0.0001). In patients with biliary atresia and a native liver, the treatment also significantly improved itch severity (p=0.0002).

Safety findings were consistent with previous clinical experience, with no new safety signals observed.

Mirum plans to submit a supplemental New Drug Application to the U.S. FDA in the first half of 2027 based on the EXPAND results. LIVMARLI is already approved for cholestatic pruritus associated with Alagille syndrome and progressive familial intrahepatic cholestasis.

The stock closed Friday's trading at $86.01, up 5.73%. In the premarket trading, shares are up 2.90% at $88.50.