Mirum Pharmaceuticals(NASDAQ) MIRM

Mirum helps children and adults with rare liver diseases by providing specialized medicines. These treatments target specific conditions where bile builds up and causes severe itching or organ damage. The company works to improve life for patients who have few other medical options.

The Medicine Cabinet

• Livmarli: Oral medication used to treat severe itching in patients with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC); works by blocking the absorption of bile acids in the gut.
• Ctexli: Standard of care treatment for adults with cerebrotendinous xanthomatosis (CTX), a rare genetic metabolic disorder; received FDA approval in February 2025.
• Cholbam: Capsule used for pediatric and adult patients with specific bile acid synthesis and peroxisomal disorders; approved by the FDA in 2015.
• Volixibat: Investigational drug being tested for adult liver conditions including primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC).

Growth and Acquisitions

• Acquired the bile acid portfolio from Travere Therapeutics in August 2023, adding Cholbam and Ctexli to its commercial products.
• Completed the acquisition of Bluejay Therapeutics in January 2026, gaining the hepatitis D candidate brelovitug.
• Entered into a license agreement with Enthorin Therapeutics in October 2024 to develop a new treatment for fragile X syndrome.
• Expanded the use of Livmarli into new international markets through distribution and licensing agreements with various rare disease companies.

What Makes Mirum Different

Mirum focuses on orphan diseases, which are rare conditions affecting small numbers of people that often lack dedicated therapies. The company utilizes a specialized commercial team to reach a small, highly targeted group of medical experts and specialty pharmacies.

Key Financial Data

• Revenue reached $148.9 million in Q4 2025, up 49.8% from Q4 2024.
• Gross profit grew to $120.6 million in Q4 2025, up 57.4% year-over-year.
• Operating cash flow turned positive at $6.1 million in Q4 2025, up 219.7% from a loss in Q4 2024.
• Market capitalization increased to $4 billion by Q4 2025, up 98.4% from the previous year.
• Net loss narrowed to $5.7 million in Q4 2025, a 75.9% improvement from the $23.8 million loss in Q4 2024.

Bottom Line

Mirum has transformed from a clinical-stage developer into a commercial-stage company with multiple approved products for rare diseases. Its strategy combines internal development with strategic acquisitions to build a specialized portfolio in the orphan drug sector.