Here are a few analogies for Mirum Pharmaceuticals:

• Like a **Vertex Pharmaceuticals** specializing in rare liver and cholestatic diseases.
• An emerging **Alexion Pharmaceuticals**, but focused specifically on rare liver and cholestatic disorders.

• LIVMARLI: An investigational oral drug for the treatment of progressive familial intrahepatic cholestasis, Alagille syndrome, and biliary atresia disease.
• Volixibat: A drug under development for the treatment of intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis.

Mirum Pharmaceuticals (MIRM), as a biopharmaceutical company developing and commercializing prescription drugs, sells primarily to other companies rather than directly to individuals. Its major customers are typically large pharmaceutical wholesalers and distributors, who then supply the drugs to hospitals, clinics, and specialty pharmacies for dispensing to patients.

The major customers are:

• Cencora, Inc. (formerly AmerisourceBergen Corporation) - Symbol: COR
• McKesson Corporation - Symbol: MCK
• Cardinal Health, Inc. - Symbol: CAH

Chris Peetz, Chief Executive Officer

Chris Peetz is a co-founder of Mirum Pharmaceuticals. He previously served as Chief Executive Officer of Flashlight Therapeutics, Inc.. Peetz also held the role of Chief Financial Officer and Head of Corporate Development at Tobira, which was acquired by Allergan plc in 2016. Prior to Tobira, he served as Vice President, Finance and Corporate Development of Jennerex Biotherapeutics, a company that was acquired by a Korean group. He held various positions at Onyx Pharmaceuticals, Inc. (now Amgen Inc.). Peetz was an entrepreneur-in-residence at Frazier Healthcare Partners, a private equity firm, since May 2017, which ultimately led to the creation of Mirum.

Eric Bjerkholt, Chief Financial Officer

Eric Bjerkholt joined Mirum as Chief Financial Officer in September 2023. Before Mirum, he served as Chief Financial Officer at Chinook Therapeutics, Inc. and prior to that, held the CFO position at Aimmune Therapeutics, Inc.. Earlier in his career, he worked in healthcare investment banking at J.P. Morgan & Company, Inc. for nearly a decade.

Peter Radovich, President & Chief Operating Officer

Peter Radovich previously served as Executive Vice President, Operations at Global Blood Therapeutics (GBT) from 2014 to 2020. From 2006 to 2014, he worked at Onyx Pharmaceuticals, Inc. (now Amgen, Inc.) in various roles, including Vice President, Program Leadership, and commercial and corporate strategy. He also held product marketing positions at Chiron Corporation (now Novartis AG).

Joanne Quan, M.D., Chief Medical Officer

Joanne Quan served as Chief Medical Officer at Nuvig Therapeutics and, from 2018 to 2021, held the same role at Modis Therapeutics, a company that was acquired by Zogenix, Inc. (and subsequently, UCB). Her experience includes clinical development leadership positions at Eiger Biopharmaceuticals, InterMune, Arena Pharmaceuticals, Bayhill Therapeutics, ALZA Corporation (Johnson and Johnson), Genentech, and PathoGenesis.

Jean-Luc Girardet, Ph.D., Chief Technical Officer

Jean-Luc Girardet previously served as Senior Vice President of Pharmaceutical Sciences at Brii Biosciences from 2018 to 2022. From 2007 to 2018, he held executive leadership roles at Ardea Biosciences, a member of the AstraZeneca Group. Earlier in his career, he worked at Valeant Pharmaceuticals, Ribapharm, and ICN Pharmaceuticals.

Key Risks to Mirum Pharmaceuticals (MIRM)

1. Clinical Trial Outcomes and Regulatory Approvals: Mirum Pharmaceuticals' business relies significantly on the successful completion of clinical trials and subsequent regulatory approvals for its product candidates. There is no guarantee that ongoing or future clinical trials for drugs like Volixibat (for conditions such as primary sclerosing cholangitis and primary biliary cholangitis) will yield positive results, even if interim analyses have been favorable. Additionally, the company faces risks associated with regulatory challenges and the possibility that regulatory outcomes may not fully support the valuation already embedded in the stock. The company's filings explicitly mention "risks and uncertainties with the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies and the potential that estimated prevalences are materially inaccurate".
2. Competition and Market Access: Mirum operates in a highly competitive biopharmaceutical industry, particularly within the sector for cholestatic liver diseases. Direct competition comes from companies developing similar therapies, such as Ipsen with its drug Bylvay (odevixibat), which targets progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS)—indications for which Mirum's LIVMARLI is also approved. The company faces challenges in maintaining pricing power and market access in a healthcare system that is sensitive to costs. The threat of competitors obtaining patents that limit or interfere with Mirum's ability to market its products, along with the high costs associated with defending intellectual property rights, also presents a constant challenge.
3. Product Safety and Side Effects: The safety profiles and potential side effects of Mirum's approved and investigational drugs pose a risk. LIVMARLI, the company's lead product, has been associated with drug-induced liver injury (DILI), with some patients in trials experiencing hepatic decompensation events and elevations of liver tests. In severe cases, DILI attributable to LIVMARLI led to one patient requiring a liver transplant and another patient dying. Other common adverse reactions for LIVMARLI include diarrhea, abdominal pain, and fat-soluble vitamin deficiency, which can worsen during treatment. Similarly, Volixibat, another key pipeline candidate, has reported common adverse events such as mild to moderate gastrointestinal issues, primarily diarrhea.

Mirum Pharmaceuticals (symbol: MIRM) has the following addressable market sizes for its main products:

• LIVMARLI (maralixibat) for Alagille syndrome: The estimated patient population is between 4,000 and 5,500 in the United States and Europe combined.
• LIVMARLI (maralixibat) for Progressive Familial Intrahepatic Cholestasis (PFIC): null
• LIVMARLI (maralixibat) for Biliary Atresia: null
• Volixibat for Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC): The estimated worldwide total addressable market is more than $1 billion.
• Volixibat for Intrahepatic Cholestasis of Pregnancy (ICP): null

Mirum Pharmaceuticals (MIRM) is poised for significant revenue growth over the next two to three years, driven by the expansion of its approved therapies and the anticipated launch of new product indications. Key drivers include:

1. Continued Growth of LIVMARLI in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis (PFIC): LIVMARLI, Mirum's lead product, is expected to maintain strong growth through increased demand and expanding international reimbursement. The company reported robust growth in LIVMARLI net product sales, including an 89% increase in 2023 over 2022, and projected continued increases in 2024 and beyond. International business has expanded to 18 countries with reimbursed access to LIVMARLI, contributing to durable growth.
2. LIVMARLI Label Expansion into Additional Rare Cholestatic Pruritic Disorders (EXPAND Study): Mirum anticipates a significant revenue boost from the potential expansion of LIVMARLI's use beyond Alagille syndrome and PFIC. The Phase 3 EXPAND study, which completed enrollment, is scheduled to report data in the fourth quarter of 2026. Success in this study could lead to an additional $300 million to $400 million in annual sales for the LIVMARLI franchise by extending its reach to a broader set of rare cholestatic pruritic disorders.
3. Launch and Growth of Volixibat in Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC): The development of Volixibat for adult cholestatic conditions, specifically PSC (VISTAS study) and PBC (VANTAGE study), represents a substantial future revenue opportunity. Interim analyses for both studies were scheduled for June 2024, with topline data for the VISTAS study anticipated in 2026. There are currently no approved therapies for PSC and no approved therapies to treat cholestatic pruritus in PBC, positioning Volixibat to address significant unmet medical needs.
4. Sustained Performance of Acquired Bile Acid Medicines (CHOLBAM and CHENODAL): The integration of CHOLBAM and CHENODAL into Mirum's portfolio has strengthened its position in rare diseases. The company expects steady growth for these two medicines, contributing to overall product sales. In Q1 2024, CHOLBAM and CHENODAL net sales were $26.1 million. Additionally, CTEXLI (chenodiol), approved by the FDA for cerebrotendinous xanthomatosis (CTX), adds another growth driver.

Share Repurchases

• Mirum Pharmaceuticals reported no share repurchases as of December 31, 2025.

Share Issuance

• In late 2025, Mirum Pharmaceuticals announced private placements totaling approximately $268.5 million, including a $68.5 million placement with TCGX for 1,000,000 shares at $68.48 per share, to fund clinical development and commercial activities.
• The company's additional paid-in capital increased significantly, reaching $0.982 billion in 2025, representing a 12.84% increase from 2024.
• Additional paid-in capital also rose by 8.33% to $0.87 billion in 2024 and by 50.12% to $0.803 billion in 2023.

Inbound Investments

• Mirum Pharmaceuticals secured approximately $268.5 million through private placements with institutional investors, including TCGX, in late 2025/early 2026.
• These investments were intended to support clinical development and commercial activities, particularly following the acquisition of Bluejay Therapeutics.

Outbound Investments

• Mirum Pharmaceuticals acquired Bluejay Therapeutics in January 2026 to further develop brelovitug for chronic hepatitis D.
• The company's recent business development activities also included the acquisition of the Travere bile-acid portfolio.

Capital Expenditures

• Capital expenditures for Mirum Pharmaceuticals were approximately $0.954 million in the last 12 months leading up to early March 2026.
• Historical annual capital expenditures have been relatively low, with figures such as -$0.6 million for 2025.
• Projections indicate a significant increase in capital expenditures, with an expected $128 million for 2026.