Mirum Pharmaceuticals Highlights Encouraging Data For Rare Liver Disease Therapy From Two Mid-Stage Studies

Monday, Mirum Pharmaceuticals Inc. MIRM announced interim results from two Phase 2b studies evaluating volixibat for primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).

Interim results from the VANTAGE study evaluating volixibat in patients with PBC demonstrated a statistically significant (-3.82, p<0.0001) improvement in pruritus (itch) for volixibat and a placebo-adjusted difference of -2.32 points in the primary endpoint, p=0.0026, as measured by the Adult ItchRO scale.

Related: FDA Approves Mirum’s Livmarli For Liver Disease In Kids As Young As 5 Years: Analyst Bullish On Label Expansion Strategy.

75% of patients on volixibat achieved a greater than 50% reduction in serum bile acids.

In addition, there was a significant improvement in fatigue at week 16 with volixibat compared to the placebo.

No new safety signals were observed, and adverse events were similar between the 20 and 80 mg treatment groups.

The most common adverse event was diarrhea (77%), with all cases mild to moderate and mostly transient.

There were no clinically meaningful changes in liver biomarkers.

Based on these results, the VANTAGE PBC trial will continue with a volixibat dose of 20 mg twice daily.

Concurrently, the interim analysis for the VISTAS PSC study was conducted, and the independent data review committee recommended that the study continue with the selected volixibat dose of 20 mg twice daily, with no changes to the study.

The criteria for continuation included safety and a predefined threshold for efficacy.

Price Action: MIRM shares are up 14.6% at $29.71 at last check Monday.

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