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Shares of Mirum Pharmaceuticals, Inc. (MIRM) closed 10% higher on Monday, marking its best day since mid-December, after the company said that its investigational drug volixibat met the primary endpoint in a mid-stage study in patients with a rare liver disease.
Primary Sclerosing Cholangitis (PSC) is a chronic, progressive, and rare liver disease characterized by inflammation, scarring, and narrowing of the bile ducts. In Mirum’s trial, volixibat delivered statistically significant and clinically meaningful reductions in cholestatic pruritus or an intense, often debilitating itching caused by liver diseases.
In the primary analysis cohort of 111 patients with moderate-to-severe itch at baseline, volixibat 20 mg twice daily produced a 2.72-point improvement from baseline on a standardized scale, compared with a 1.08-point improvement for placebo, the company said. Statistically significant itch relief was observed as early as two weeks after starting therapy and was also seen in the secondary cohort of patients with mild itch, it added.
Severe adverse events associated with the drug in the trial consisted mainly of gastrointestinal side effects and elevations in liver laboratory values such as ALT and bilirubin, the company said.
The results position volixibat as a potential approved therapy for the severe itching that is one of the most burdensome symptoms of PSC, a rare and progressive liver disease with no approved treatments, according to Mirum’s chief medical officer Joanne Quan.
Mirum said it has scheduled a pre-New Drug Application meeting with the U.S. Food and Drug Administration for this summer and plans to submit an NDA in the second half of 2026. The company added that it now expects topline data from a separate study of volixibat in primary biliary cholangitis in the first quarter of 2027.
On Stocktwits, retail sentiment around MIRM jumped from ‘bearish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘normal’ to ‘extremely high’ levels.
A Stocktwits user voiced optimism for a buyout at up $160/share price.
MIRM stock has gained about 140% over the past 12 months.
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