Mirum Pharma: A Rare Disease Growth Story to Watch

Summary

Mirum Pharmaceuticals NASDAQ: MIRM is a late-stage biotechnology company that is making significant progress toward its mission to combat rare diseases with no or limited treatment options. Mirum recently reported its Q1 2026 earnings, headlined by 43% year-over-year (YOY) revenue growth. In 2025, the company reported revenue of $521.3 million, up 54%, with its lead drug Livmarli responsible for $360 million, a 69% YOY increase. Mirum has two other FDA-approved therapies—Cholbam for bile-acid synthesis disorders, and Ctexli for cerebrotendinous xanthomatosis, a rare genetic bile acid metabolism disorder.Get Mirum Pharmaceuticals alerts:Sign Up The company also raised its full-year revenue guidance to a range of $660 million to $680 million. At the midpoint, that would be a YOY increase of 26%. However, since the earnings report, MIRM is down about 12% despite bullish analyst sentiment. What Makes Mirum a Compelling Speculative Play?Mirum Pharmaceuticals TodayMIRMMirum Pharmaceuticals$96.65 +1.00 (+1.05%) As of 12:41 PM Eastern This is a fair market value price provided by Massive. Learn more.52-Week Range$42.89▼$114.99Price Target$137.08 Mirum's commercial story is unusual for a company that is not profitable: it already has three FDA-approved drugs generating real, growing revenue. As noted above, Livmarli remains the primary growth engine, with Q1 2026 net product sales of $159.9 million representing robust sequential momentum. Livmarli is currently in a late-stage trial that could expand the drug’s label to include Alagille Syndrome. Topline data for Phase 3 is scheduled for December. Cholbam and Ctexli provide a revenue floor that single-asset biotechs don't have. The pipeline adds additional future revenue opportunities: Any one of these approvals could meaningfully expand the company's addressable market. Right now, Volixibat is the furthest along. On May 4, 2026, Mirum reported that the primary endpoint was met in the VISTAS Phase 2b study. Then there's the c...