Mirum's rare liver disease drug meets main goal in mid-stage study

May 4 (Reuters) - Mirum Pharmaceuticals MIRM.O said on Monday its experimental drug, volixibat, met the main goal of a mid-stage study by reducing severe itching or pruritus in patients with a type of rare liver disease.

• The study enrolled 158 patients and tested volixibat, given at 20 mg twice daily, against placebo. The main analysis included 111 patients with moderate to severe itching, while 47 with mild itching were assessed separately.

• Primary sclerosing cholangitis, or PSC, causes inflammation, scarring and narrowing of bile ducts, leading to bile buildup, liver damage, and potentially liver failure.

• In the main group, volixibat cut itch scores by 2.72 points from baseline, versus 1.08 points for placebo, giving a placebo-adjusted benefit of 1.64 points. Mirum said the result was statistically significant.

• The results support the potential for volixibat to become the first treatment for cholestatic pruritus in patients with PSC, the company said.

• "The takeaway here is very straightforward – trial is clearly positive & data is in line with expectations," said Evercore ISI analyst Gavin Clark-Gartner.

• Mirum said it has a pre-application meeting with the U.S. Food and Drug Administration scheduled for summer 2026 and plans to file for approval in the second half of 2026.