Financial Overview and Outlook for Mirum Pharmaceuticals

Mirum Pharmaceuticals is a biopharmaceutical company dedicated to transforming the treatment of rare diseases. The company has three approved medicines - LIVMARLI, CHOLBAM, and CHENODAL/CTEXLI - and is developing additional product candidates for rare cholestatic conditions.

In 2024, Mirum reported net losses of $87.9 million, an improvement from $163.4 million in 2023. The company’s accumulated deficit stood at $644.2 million as of the end of 2024, compared to $556.2 million a year earlier. Mirum ended 2024 with $293.3 million in cash, cash equivalents, restricted cash and investments, up from $286.3 million at the end of 2023.

Revenue Trends

Mirum’s total revenue grew significantly in 2024, reaching $336.9 million compared to $186.4 million in 2023. This was driven by strong growth in product sales, which increased from $178.9 million in 2023 to $336.4 million in 2024.

The increase in product sales was due to several factors:

• Sales of Mirum’s Bile Acid Medicines (Cholbam and Chenodal/Ctexli), which the company acquired in August 2023 through the Bile Acid Portfolio Acquisition.
• Continued commercialization of Livmarli in the U.S. for the treatment of Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), as well as in certain international markets.

Mirum’s license and other revenue declined from $7.5 million in 2023 to $0.5 million in 2024, as the company did not achieve any significant milestones from its licensing agreements in the more recent year.

Looking ahead, Mirum expects total product sales to continue increasing on an annual basis as it grows sales of its approved medicines. However, the company’s license revenue remains uncertain and dependent on its partners achieving future milestones.

Expenses and Profitability

Mirum’s total operating expenses increased from $295.5 million in 2023 to $424.5 million in 2024, driven by the following:

Cost of Sales:

• Increased from $47.0 million in 2023 to $81.6 million in 2024, primarily due to higher royalty expenses, amortization of acquired intangibles, and product costs associated with the Bile Acid Medicines.

Research and Development (R&D) Expenses:

• Grew from $102.6 million in 2023 to $140.6 million in 2024, reflecting increased spending on the development of Livmarli, volixibat, and the newly in-licensed MRM-3379 program.
• Key R&D investments included the Livmarli EXPAND label expansion study, the volixibat VISTAS and VANTAGE Phase 2b trials in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), and the upfront payment for the MRM-3379 in-licensing deal.

Selling, General & Administrative (SG&A) Expenses:

• Increased from $145.9 million in 2023 to $202.2 million in 2024, driven by higher personnel costs, marketing/promotional expenses, and other administrative costs to support the company’s commercial activities and global expansion.

The significant increase in operating expenses, combined with the loss of $49.1 million related to the termination of the revenue interest purchase agreement (RIPA) in 2023, resulted in Mirum reporting a net loss of $87.9 million in 2024, an improvement from the $163.4 million net loss in the prior year.

Going forward, Mirum expects its cost of sales to increase in line with higher product sales, but remain relatively stable as a percentage of revenue. R&D expenses are also likely to rise as the company advances its pipeline, while SG&A costs are expected to grow to support the commercialization of its approved medicines globally.

Financial Position and Liquidity

Mirum ended 2024 with a strong cash position, holding $293.3 million in cash, cash equivalents, restricted cash and investments. This represented an increase from the $286.3 million in cash and cash equivalents at the end of 2023.

The company’s cash balance was bolstered by several key financing activities in 2023:

• $202.2 million in net proceeds from a private placement of 8 million shares of common stock, used to finance the upfront payment for the Bile Acid Portfolio Acquisition.
• $305.3 million in net proceeds from the issuance of $316.3 million in convertible notes, a portion of which was used to repurchase and terminate the RIPA.

Mirum believes its current cash resources, combined with expected revenue growth from product sales, will be sufficient to fund its operations and development activities for the foreseeable future. However, the company may need to raise additional capital through equity offerings, debt financing, or other means to support its long-term growth plans and pipeline advancement.

Key Strengths and Risks

Mirum’s key strengths include:

1. Diversified rare disease portfolio: The company has three approved medicines - Livmarli, Cholbam, and Chenodal/Ctexli - addressing various cholestatic and bile acid-related disorders. This provides revenue diversification.

2. Robust pipeline: Mirum is advancing several promising product candidates, including volixibat for cholestatic liver diseases and the newly in-licensed MRM-3379 for Fragile-X Syndrome, which offer potential long-term growth.

3. Experienced management team: The company’s leadership has deep expertise in rare disease drug development and commercialization.

4. Strong financial position: Mirum ended 2024 with a healthy cash balance to fund its operations and pipeline development.

However, Mirum also faces several risks:

1. Reliance on approved products: The company’s near-term success is heavily dependent on the continued growth of its approved medicines, particularly Livmarli. Any setbacks in these products could significantly impact Mirum’s financial performance.

2. Pipeline development risks: There is no guarantee that Mirum’s product candidates in development will successfully navigate the clinical trial process and obtain regulatory approvals.

3. Competition and pricing pressure: Mirum may face competition from other therapies, which could impact the commercial potential of its products. Pricing and reimbursement challenges are also a concern.

4. Potential for additional capital needs: While Mirum’s current cash position is strong, the company may need to raise additional funds in the future to support its long-term growth plans and pipeline advancement.

Conclusion

Mirum Pharmaceuticals has made significant progress in 2024, with strong revenue growth driven by its approved medicines and the addition of the Bile Acid Medicines portfolio. The company’s diversified rare disease focus, robust pipeline, and experienced management team position it well for continued success.

However, Mirum must navigate the risks inherent in the biopharmaceutical industry, including the need to successfully develop its product candidates, maintain the commercial momentum of its approved products, and secure adequate funding to support its long-term growth. Investors will be closely watching Mirum’s ability to execute on its strategic priorities and deliver value for shareholders in the years ahead.