Mirum Pharmaceuticals, Inc. (MIRM) Q3 2025 Earnings Call Transcript

Access earnings results, analyst expectations, report, slides, earnings call, and transcript.

Overview

The earnings call reflects a positive outlook with the company's first-ever positive net income, increased cash reserves, and raised revenue guidance for 2025. The Q&A section reveals confidence in product development and competitive positioning, despite some uncertainties in pricing strategies and guidance. The market cap suggests a moderate reaction, leading to an expected stock price increase of 2% to 8%.

Key Financial Performance

Revenue Third quarter revenue of $133 million, representing a nearly 50% year-over-year increase. This growth was driven by the strength and breadth of the commercial portfolio, including continued momentum from the U.S. PFIC launch and expanding demand from international markets.

LIVMARLI Net Product Sales Net product sales totaled $92 million for the quarter, with $64 million in the U.S. and $28 million internationally. Growth in the U.S. was driven by demand in Alagille syndrome and PFIC, while international growth was supported by expanding reimbursement and new launches, including Takeda's commercialization in Japan.

Bile Acid Medicines (CHOLBAM and CTEXLI) Net Product Sales Generated $41 million in net product sales this quarter, supported by increased CTX patient finding following CTEXLI's FDA approval earlier this year.

Operating Expense Total operating expense for the quarter ended September 30 was $130 million, which includes R&D expense of $43 million, SG&A expense of $62 million, and cost of sales of $26 million. Noncash stock-based compensation expense was $18 million, and intangible amortization and other noncash items were $6 million.

Net Income Generated approximately $3 million in net income, marking the first time the company achieved positive net income. This reflects the strength and scalability of the business model.

Cash, Cash Equivalents, and Investments Cash, cash equivalents, and investments were $378 million as of September 30, an $85 million increase from the beginning of the year. This growth highlights the company's financial independence and ability to fund expansion.

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Operating Highlights

LIVMARLI: Net product sales totaled $92 million for the quarter, with $64 million in the U.S. and $28 million internationally. Growth driven by Alagille syndrome and PFIC, supported by expanded diagnosis and genetic screening.

Bile Acid Medicines (CHOLBAM and CTEXLI): Generated $41 million in net product sales, with increased CTX patient finding following CTEXLI's FDA approval earlier this year.

MRM-3379: Initiated Phase II study for Fragile X syndrome, supported by preclinical data showing reversal of disease phenotype in a mouse model.

International Expansion: LIVMARLI demand grew internationally with $28 million in net product sales, supported by expanding reimbursement and launches in new geographies. First full quarter of commercialization in Japan through partner Takeda.

Revenue Growth: Third quarter revenue of $133 million, a 47% year-over-year increase, driven by strong commercial portfolio performance.

Financial Milestone: Achieved positive net income of $3 million for the first time, highlighting operating leverage in the commercial model.

Pipeline Development: Progressed clinical pipeline with three pivotal readouts expected in the next 18 months, including studies for volixibat in PSC and PBC, and LIVMARLI in ultra-rare cholestatic conditions.

Focus on Rare Diseases: Continued focus on developing treatments for rare diseases, with a broad pipeline and global footprint.

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Risk or Challenges

Regulatory and Market Risks: The company acknowledges risks and uncertainties inherent in forward-looking statements, which may cause actual results to differ materially. This includes potential regulatory hurdles and market dynamics that could impact the approval and adoption of their therapies.

Pipeline Development Risks: The success of ongoing clinical trials, such as the VISTAS Phase IIb study in PSC and the VANTAGE study in PBC, is uncertain. Delays or negative outcomes in these trials could hinder the company's ability to expand its product portfolio.

Commercial Execution Challenges: While the company has seen strong commercial performance, challenges remain in expanding genetic testing and education among adult-focused providers, which could limit the growth of LIVMARLI in broader medical segments.

International Market Variability: The company notes variability in international partner and distributor ordering patterns, which could lead to fluctuations in revenue from international markets.

Financial Sustainability Risks: Although the company achieved GAAP profitability this quarter, it does not expect consistent profitability in the near term due to ongoing investments in growth. This could pose financial sustainability challenges if revenue growth slows.

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Guidance & Outlook

Revenue Guidance: The company expects to land in the upper end of its prior full-year 2025 guidance range with $500 million to $510 million in revenues.

Pipeline Milestones: Three potentially pivotal readouts are expected over the next 18 months. The VISTAS Phase IIb study in PSC is expected to announce top-line data in Q2 2026. The VANTAGE study in PBC is progressing well, with enrollment completion expected next year. The EXPAND study for LIVMARLI in additional cholestatic pruritus settings is targeting enrollment completion in 2026. A Phase II study of MRM-3379 for Fragile X syndrome has been initiated.

Market Potential: Peak revenue potential for LIVMARLI, volixibat, and MRM-3379 is expected to exceed $1 billion each.

International Expansion: LIVMARLI demand continues to grow internationally, supported by expanding reimbursement and launches in new geographies. The first full quarter of commercialization in Japan by partner Takeda showed adoption dynamics consistent with the U.S. launch.

Financial Position: The company achieved GAAP profitability in Q3 2025, generating $3 million in net income. However, consistent quarterly GAAP profitability is not yet expected as investments in growth continue. Cash, cash equivalents, and investments totaled $378 million as of September 30, 2025.

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Shareholder Return Plan

The selected topic was not discussed during the call.

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Key Q&A

Q:What are the key drivers of LIVMARLI's performance as we look ahead to 2026?

A:The key drivers include the continuation of the PFIC launch in the U.S. and internationally, with growth expected to build over time. Guidance for 2026 will likely be provided early next year.

Q:Why does the midpoint of the new guidance range imply flat sequential Q4 revenue from Q3?

A:The flat sequential revenue is due to variability in the LIVMARLI international line, which includes periodic large orders from distributors and the absence of Takeda revenue in Q4 that was present in Q3.

Q:What trends are being seen in the adoption of the solid tablet formulation of LIVMARLI?

A:The solid tablet formulation, launched in mid-June, has seen encouraging uptake with substantial switches from the liquid formulation among eligible patients (those weighing at least 25 kilos).

Q:What is the pricing strategy for volixibat?

A:The pricing strategy for volixibat is still under analysis. The company is monitoring market dynamics and considering analogs like PPARs and other products approved in PBC, which are priced at $130,000 to $150,000.

Q:What is the expectation for Paragraph IV filers and the confidence in the IP portfolio?

A:The company is confident in its IP portfolio, particularly the method patents specific to dosing of LIVMARLI. They are prepared to defend their IP if Paragraph IV filers emerge, though none have been seen to date.

Q:What are the perspectives on the best endpoint for Fragile X studies and implications for the company's program?

A:The company feels confident in its endpoint strategy, supported by strong preclinical data and engagement with the community and FDA. They are focused on validated outcomes and are optimistic about their study.

Q:What are the safety risks of IBAT inhibitors in PSC patients with inflammatory disease comorbidities?

A:No significant safety issues have been raised in the PSC study. A data monitoring committee has not suggested any protocol modifications, and the safety profile is consistent with known IBAT characteristics.

Q:What are the baseline criteria for patients in the PSC study and the importance of secondary endpoints?

A:Baseline criteria include elevated pruritus scores, representative of the PSC population. The primary regulatory focus is on pruritus, while secondary endpoints like fatigue and bile acids are important but not the main focus.

Q:What went into the decision to offer BID dosing for the EXPAND study?

A:The decision was based on empirical observations from compassionate use settings, showing great response stories at elevated dose levels.

Q:What has been the impact of the government shutdown on genetic screening programs?

A:There has been no impact observed on genetic screening programs or other related activities.

Q:What is the pace of new PFIC patient additions and the company's BD strategy?

A:The pace of new PFIC patient additions remains healthy, with efforts to educate providers about genetic cholestasis. The company continues to seek underappreciated programs for business development but has no urgency to add new products.

Q:How does the company view the competitive dynamics in PBC with the recent linerixibat PDUFA announcement?

A:The company is confident in volixibat's competitive profile, particularly its striking placebo-adjusted difference in pruritus reduction, which led to breakthrough designation. Volixibat targets both first and second-line PBC settings.

Q:Are disease-modifying effects expected with volixibat in PSC and PBC?

A:The focus is on pruritus as the primary endpoint for regulatory approval. Disease-modifying effects may take longer to observe and will be monitored over time.

Q:Review of Unclear Management Responses

A:Management avoided providing a direct answer on the pricing strategy for volixibat, stating it is still under analysis and too early to determine. Additionally, they did not provide specific baseline criteria for PSC study patients or detailed insights into secondary endpoints beyond stating their importance.

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Earnings Word Cloud

The most frequently occurring keywords in this quarter's earning call

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