12 Best High Short Interest Stocks With Highest Upside Potential

7. Mirum Pharmaceuticals Inc. (NASDAQ:MIRM)

Average Upside Potential: 47.54%

Mirum Pharmaceuticals Inc. (NASDAQ:MIRM) is one of the best high short interest stocks with highest upside potential. On May 30, Mirum Pharmaceuticals reported positive results from its rare liver disease programs at the EASL International Liver Congress 2026. The Phase 2b VISTAS study for volixibat in primary sclerosing cholangitis/PSC demonstrated rapid, sustained, and clinically meaningful reductions in pruritus, supporting the company’s plan for an NDA submission in H2 2026.

Data from the Phase 2b AZURE-1 study of brelovitug in chronic hepatitis delta virus/HDV showed strong efficacy, with high rates of virologic response and ALT normalization at Week 24, even in patients with advanced disease. Mirum expects topline data from its Phase 3 AZURE trials later in 2026, with potential BLA submission in 2027.

Additionally, real-world evidence for the established therapy LIVMARLI (maralixibat) in progressive familial intrahepatic cholestasis/PFIC showed improved event-free survival when compared to an external control cohort. These results reinforce the long-term clinical benefits of LIVMARLI in reducing the risk of surgical biliary diversion, liver transplantation, and death in PFIC patients.

Mirum Pharmaceuticals Inc. (NASDAQ:MIRM) is a biotech company that develops and commercializes novel therapies for rare & orphan diseases.

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