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Today's Featured Story
Mirum Pharma: A Rare Disease Growth Story to WatchWritten by Chris Markoch. Article Posted: 5/20/2026. 
Key Points
- Mirum Pharmaceuticals posted strong revenue growth as its lead drug Livmarli continues gaining momentum.
- The company’s growing pipeline and commercial infrastructure could create multiple long-term growth opportunities.
- MIRM stock pulled back after earnings due to higher expenses and a convertible note offering, but analysts still see significant upside.
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Mirum Pharmaceuticals (NASDAQ: MIRM) is a late-stage biotechnology company making meaningful progress toward its mission of treating rare diseases with no or limited treatment options. Mirum recently reported its Q1 2026 earnings, highlighted 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 accounting 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.
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The company also raised its full-year revenue guidance to a range of $660 million to $680 million. At the midpoint, that would represent a 26% YOY increase. However, since the earnings report, MIRM is down about 12% despite bullish analyst sentiment. What Makes Mirum a Compelling Speculative Play?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 strong sequential momentum. Livmarli is currently in a late-stage trial that could expand the drug’s label to include Alagille syndrome. Topline data for the Phase 3 study is scheduled for December. Cholbam and Ctexli provide a revenue floor that single-asset biotechs do not 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 commercial infrastructure advantage. Mirum has spent years building relationships with hepatologists and transplant specialists who treat rare liver disease patients. That distribution moat is expensive to replicate and gives new pipeline drugs a faster path to adoption than a typical commercial-stage launch would suggest. For investors willing to wait, that foundation is the real asset. Why Is MIRM Dropping?The simple reason for MIRM's decline is that this is still a mid-cap company that is not profitable and, although revenue is growing, is still in the early stages. Short sellers are reacting to a specific data point in the earnings report: the company’s operating expense was much higher in the quarter ($949 million) due mostly to one-time costs associated with its acquisition of Bluejay. This highlights the primary risk with Mirum and many biotech stocks. The company needs to turn positive trial data into durable, reimbursed revenue. Mirum is doing that; now it must show that the revenue can grow in a way that makes the company profitable. To that end, management said that its research and development (R&D) is funded and that it expects to be operating cash flow positive next year, with GAAP profitability by 2028. However, the more complex answer comes from the company’s proposed offering of $600 million in convertible senior notes due 2032. The announcement came with an overallotment option for an additional $90 million, offered in a private placement to qualified institutional buyers. On the surface, there’s nothing too alarming about the announcement, nor will it necessarily be dilutive. And bullish investors can argue that some of the money may go toward additional acquisitions of revenue-generating drugs. The Post-Earnings Dip Can Be a Buyable OpportunityRetail investors who bought into MIRM around the time it went public in 2019 have benefited from the company’s strong growth. In fact, over the past five years, MIRM is up more than 450%, including approximately 115% growth in the past 12 months. 
It's a reminder that investors with patience and conviction can be rewarded when these companies execute, as Mirum certainly has. That raises the question of how much upside is still out there. The Mirum analyst forecasts on MarketBeat have a consensus price target of $137.08 as of May 19. That’s more than 40% above the stock’s price as of this writing. Those ratings account for favorable topline readings for Maralizibat in late 2026. There’s also more upside in the company’s pipeline. The catch? Short interest of around 17% means that there will be some selling pressure on any rally, even if it’s not central to Mirum’s bull case. |
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