Mirum gains as Phase 3 LIVMARLI trial meets primary endpoint in liver disease

Investing.com -- Mirum Pharmaceuticals Inc (NASDAQ: MIRM) shares advanced 2.9% in premarket trading after the rare disease specialist announced positive topline results from its Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) in patients with ultra-rare cholestatic liver diseases.

The study achieved its primary endpoint by demonstrating a statistically significant reduction in cholestatic pruritus compared with placebo in pediatric patients, while also meeting all five prespecified secondary endpoints.

Patients receiving maralixibat experienced a mean 1.81-point improvement from baseline in their worst-daily itch severity score compared to a 0.83-point improvement for placebo, representing a placebo-adjusted treatment difference of 0.98 points (p=0.0004).

Furthermore, the treatment drove a 69.0% relative reduction in serum bile acids compared with placebo (p<0.0001), reinforcing the therapy’s targeted biological impact on bile acid transport.

In the subgroup of patients with biliary atresia and a native liver, maralixibat delivered a pronounced 1.15-point placebo-adjusted improvement in itch severity (p=0.0002) alongside a 73.1% reduction in serum bile acids.

The safety profile remained consistent with previous clinical experience, with no new safety signals observed during the 20-week double-blind period, though diarrhea occurred in 64.0% of treated patients compared with 11.1% in the placebo group.

On the back of these clinical findings, 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 successful expansion into additional ultra-rare cholestatic indications positions Mirum to potentially broaden LIVMARLI's addressable commercial market beyond its existing approvals in Alagille syndrome and progressive familial intrahepatic cholestasis.

Original Article

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