Mirum Pharmaceuticals, a biopharmaceutical company dedicated to transforming the treatment of rare diseases, has etched its name as a formidable player in the pharmaceutical landscape. With a steadfast focus on innovative therapies and a commitment to improving the lives of patients, Mirum has navigated the complexities of the industry with remarkable agility.
Company Background
Incorporated in the State of Delaware on May 2, 2018, and headquartered in Foster City, California, Mirum Pharmaceuticals began operations in November 2018. The company's journey started with a significant milestone when it entered into an Assignment and License Agreement with Shire International GmbH (later acquired by Takeda Pharmaceutical Company Limited). This agreement granted Mirum an exclusive, royalty-bearing worldwide license to develop and commercialize its two initial product candidates, Livmarli and volixibat. As part of this agreement, Mirum was also assigned license agreements held by Shire with other third parties, laying the foundation for its future growth.
Product Development and Approvals
Mirum's first major success came with the approval of Livmarli, an orally administered, minimally-absorbed ileal bile acid transporter (IBAT) inhibitor. In 2021, Livmarli received approval for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the United States and subsequently in several other countries. The company's commitment to expanding its therapeutic reach was further demonstrated in 2023 when Livmarli gained additional approvals for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) in the U.S. and for the treatment of PFIC in the European Union.
Strategic Acquisitions
A transformative moment in Mirum's history occurred in August 2023 with the completion of the Bile Acid Portfolio Acquisition from Travere Therapeutics, Inc. This strategic move significantly expanded Mirum's commercial portfolio and bolstered its expertise in rare diseases. The acquisition brought two approved therapies under Mirum's umbrella: Chenodal, approved for the treatment of radiolucent stones in the gallbladder, and Cholbam, approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and for the adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease.
In 2023, Mirum further solidified its position in the rare disease landscape with the strategic acquisition of Travere Therapeutics' bile acid portfolio, which includes the approved medicines Chenodal and Cholbam. This transformative deal not only expanded Mirum's commercial footprint but also bolstered its expertise in rare genetic disorders, particularly in the realm of cerebrotendinous xanthomatosis (CTX) and bile acid synthesis disorders.
Financials
Mirum's financial performance has been equally impressive, with the company reporting net product sales of $90.3 million in the third quarter of 2024, a remarkable 89% increase from the same period in the previous year. This strong commercial execution has prompted Mirum to raise its full-year 2024 guidance to $330 to $335 million in net product revenue.
The company's financial trajectory has been on an upward trend. For the fiscal year 2023, Mirum reported revenue of $186.37 million, although it incurred a net loss of $163.415 million. The operating cash flow for 2023 was negative $70.944 million, with free cash flow at negative $91.053 million. However, the most recent quarter (Q3 2024) showed significant improvement, with revenue reaching $90.38 million, representing an 89% increase from Q3 2023. The net loss for Q3 2024 was reduced to $14.23 million, and the company achieved positive operating cash flow of $3.984 million and free cash flow of $13.803 million.
Geographically, Mirum's revenue is primarily derived from the United States market. In Q3 2024, 81.8% of net product sales ($73.91 million) came from the US, while the remaining 18.2% ($16.40 million) was generated from international markets.
Liquidity
The company's liquidity position remains strong, with $293.8 million in cash, cash equivalents, and investments as of September 30, 2024. This financial flexibility allows Mirum to continue investing in its pipeline, expanding its commercial operations, and pursuing strategic opportunities that align with its long-term growth objectives.
As of September 30, 2024, Mirum's debt-to-equity ratio stood at 1.369, indicating a balanced capital structure. The company's cash and cash equivalents amounted to $222.97 million, providing a solid foundation for ongoing operations and future investments. Mirum also has access to a $200 million at-the-market (ATM) equity offering program initiated in November 2023, with the full $200 million capacity still available as of September 30, 2024. The company's current ratio of 3.340 and quick ratio of 3.154 further underscore its strong short-term liquidity position.
Pipeline and Innovation
The company's pipeline continues to evolve, with the recent in-licensing of MRM-3379, a selective phosphodiesterase 4D (PDE4D) inhibitor, for the treatment of Fragile X syndrome. This addition aligns with Mirum's strategic focus on rare genetic neurological disorders, further diversifying its portfolio and showcasing the team's ability to identify and acquire promising assets.
Mirum's commitment to innovation is also evident in the progress of its clinical programs. The company's lead asset, Livmarli, is currently being evaluated in the Phase 3 EXPAND study, which aims to explore its potential in additional settings of cholestatic pruritus. Additionally, the company's IBAT inhibitor, volixibat, has recently been granted breakthrough therapy designation by the FDA for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC), underscoring its promise in addressing this unmet medical need.
Approved Medicines and Product Pipeline
Mirum's approved medicines segment includes three key products: LIVMARLI (maralixibat) oral solution, Cholbam (cholic acid) capsules, and Chenodal (chenodiol) tablets. LIVMARLI is approved for multiple indications related to cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) in various markets. Cholbam is FDA-approved for bile acid synthesis disorders and peroxisomal disorders, while Chenodal is approved for radiolucent stones in the gallbladder and has received medical necessity recognition for cerebrotendinous xanthomatosis (CTX).
The company's product pipeline includes volixibat, which is being developed for cholestatic liver diseases such as primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). Mirum is conducting Phase 2b clinical trials for volixibat in both indications. Additionally, the company is exploring expansion opportunities for Livmarli through the Phase 3 EXPAND study, set to initiate in the second half of 2024. The recently in-licensed MRM-3379 for Fragile X Syndrome is expected to enter Phase 2 clinical trials in 2025.
Challenges and Risks
However, Mirum is not without its challenges. The company operates in a highly competitive and regulated industry, and its success is contingent upon its ability to navigate the complexities of the healthcare landscape, secure favorable reimbursement policies, and maintain a robust intellectual property portfolio. Additionally, the company's reliance on third-party manufacturers and distributors introduces supply chain risks that must be carefully managed.
Management and Future Outlook
Despite these obstacles, Mirum has demonstrated its resilience and adaptability. The company's experienced management team, led by CEO Chris Peetz, has navigated the industry's challenges with exceptional foresight, positioning Mirum as a rising titan in the rare disease therapeutics arena.
As Mirum continues to execute on its strategic priorities, shareholders can expect the company to remain at the forefront of innovation, driving meaningful advancements in the treatment of rare and debilitating conditions. With a diversified product portfolio, a strong financial foundation, and a steadfast commitment to patient-centric innovation, Mirum Pharmaceuticals is poised to create lasting value for its stakeholders. The company's increased guidance for full-year 2024 net product revenue of $330 to $335 million reflects management's confidence in the continued growth trajectory and successful execution of Mirum's commercial strategy.