Mirum Pharmaceuticals, Inc. (MIRM) Q2 2025 Earnings Call Transcript

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

Overview

The earnings call reveals strong financial performance with a 61% revenue growth and increased cash flow. Regulatory approvals and positive pipeline progress further enhance outlook. The Q&A section highlights confidence in ongoing trials and market potential, despite management's vague revenue distribution response. The updated guidance and cash flow positivity suggest a favorable short-term stock reaction, especially for a small-cap company.

Key Financial Performance

Total Revenue $128 million, a 64% growth over the second quarter last year. The growth was driven by increased demand for Livmarli in both the U.S. and international markets, as well as solid performance from the bile acid portfolio.

Livmarli U.S. Net Product Sales $57 million. Growth attributed to increased disease awareness, broader use of genetic testing leading to more PFIC diagnoses, and synergy between Alagille syndrome and PFIC indications.

Livmarli International Net Product Sales $31 million. Growth driven by expanding reimbursement, growing demand, and strong performance in partner markets, including a successful launch in Japan by Takeda.

Bile Acid Portfolio Revenue $40 million. Growth attributed to steady demand and increased engagement following the CTEXLI approval earlier this year.

Total Operating Expense $133 million, which includes R&D expense of $46 million, SG&A expense of $63 million, and cost of sales of $23 million. Noncash stock-based compensation expense was $18 million, and intangible amortization and other noncash items were $6 million.

Cash, Cash Equivalents, and Investments $322 million as of June 30, a $29 million increase from the end of last year. The company was operating cash flow positive for the quarter.

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Operating Highlights

Livmarli: Key driver of revenue growth, with $128 million in total revenues for Q2 2025, representing 64% growth year-over-year. U.S. sales reached $57 million, driven by increased awareness and genetic testing for PFIC. International sales reached $31 million, supported by reimbursement expansion and Takeda's launch in Japan.

Single tablet formulation of Livmarli: Launched in June 2025 in the U.S., adding convenience for patients.

MRM-3379: Phase II study for Fragile X syndrome to begin by end of 2025, targeting males aged 16-45 with severe cognitive needs.

Geographic expansion: Livmarli launched in Japan by partner Takeda in June 2025, with promising initial demand.

Reimbursement expansion: International growth supported by expanding reimbursement in direct and partner markets.

Revenue growth: Achieved $128 million in Q2 2025 revenues, a 64% increase year-over-year. Full-year guidance raised to $490-$510 million.

Cash flow: Operating cash flow positive for Q2 2025, with cash and investments increasing to $322 million.

Pipeline development: Progress in VISTAS Phase IIb study for PSC, VANTAGE study for PBC, and EXPAND study for cholestatic pruritus. Phase II study for MRM-3379 in Fragile X syndrome to start by end of 2025.

Long-term vision for Livmarli: Aiming to make Livmarli a $1 billion-plus revenue brand through label expansion and continued growth in Alagille syndrome and PFIC.

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Risk or Challenges

Regulatory Risks: The company is advancing multiple clinical trials, including the VISTAS Phase IIb study and the Phase II study of MRM-3379. These trials are subject to regulatory approvals and potential delays, which could impact timelines and strategic objectives.

Market Competition: Livmarli faces competition in the rare disease market, and its ability to maintain a differentiated position with physicians and patients is critical for sustained growth.

Supply Chain Variability: International product sales, particularly through partners like Takeda, are subject to quarter-to-quarter variations due to large periodic orders, which could lead to revenue unpredictability.

Economic Uncertainties: The company’s financial performance is tied to reimbursement approvals and market demand, which could be influenced by broader economic conditions.

Strategic Execution Risks: The company’s ambitious pipeline and growth targets require effective execution across multiple fronts, including clinical trial enrollments, product launches, and market expansions.

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Guidance & Outlook

Revenue Guidance for 2025: The company has raised its full-year revenue guidance for 2025 to $490 million to $510 million, reflecting close to 50% top-line growth.

Pipeline Milestones for 2026: Three late-stage milestones are expected in 2026, including the VISTAS Phase IIb study in primary sclerosing cholangitis (PSC), which is on track to complete enrollment this quarter with top-line data expected in Q2 2026.

Livmarli Revenue Potential: Livmarli is projected to become a $1 billion-plus revenue brand in the long term, driven by growth in Alagille syndrome, PFIC, and label expansion opportunities in ultra-rare cholestasis.

MRM-3379 Phase II Study: The Phase II study for MRM-3379 in Fragile X syndrome is set to begin by the end of 2025, targeting approximately 52 male participants aged 16 to 45.

EXPAND Study for Livmarli: The EXPAND study evaluating Livmarli in additional settings of cholestatic pruritus is expected to complete enrollment in 2026.

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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 overall therapy persistence rates for Livmarli, and have they changed over the years?

A:Persistence rates for Livmarli in the Alagille indication show that 70%-75% of patients remain on therapy after one year, with much lower attrition in subsequent years. For PFIC, it is too early to comment on persistence rates.

Q:What insights can you provide about the ongoing VISTAS PSC trial?

A:The VISTAS PSC trial is progressing well. The study was conservatively powered with a standard deviation of 3, but it is expected to come closer to 2, which adds confidence. Baseline characteristics reflect the PSC population, further supporting confidence in the trial.

Q:Can you provide details on the expected revenue distribution between Livmarli and the Bile Acid business for the remainder of the year?

A:Management is not breaking down guidance by specific products but expects trends seen year-to-date to continue within the $490 million to $510 million range.

Q:What are the main drivers of growth in Livmarli sales, and how has the tablet format impacted script volumes?

A:Growth drivers include increased awareness of genetic testing and later-onset PFIC diagnoses, growth in Alagille syndrome, and strong international performance. The tablet format, introduced in June, has received positive feedback but did not impact the quarter's results.

Q:Were there any inventory impacts in the second quarter, and what is the expected Takeda order cadence?

A:Inventory impacts are only relevant to Japan and Takeda, with large periodic orders expected. There was an $11 million order in Q2, and another order is expected this year, though timing is uncertain.

Q:What progress has been made on the Fragile X trial design, and are there any next steps before starting the study?

A:The Fragile X trial design is finalized, with clearance from the IND and a Study May Proceed letter. The study is on track to enroll the first patient by the end of the year.

Q:How well penetrated is Livmarli in the Alagille and PFIC markets, and what is the potential for Livmarli as a $1 billion product?

A:Livmarli has about 50% penetration in the U.S. Alagille market, with continued growth potential. PFIC is larger than initially estimated, and the EXPAND study population is at least the size of PFIC. These factors contribute to the $1 billion potential.

Q:Are any patients in the EXPAND study already on Livmarli through compassionate use or other exceptions?

A:The EXPAND study includes patients who have not been previously treated with Livmarli to assess treatment response. The study was initiated due to high demand for compassionate use in this population.

Q:What accounts for the shift in Livmarli's market potential from $500 million to over $1 billion?

A:The $500 million figure was specific to the U.S. Alagille market, while the $1 billion potential includes Alagille, PFIC, and EXPAND indications, as well as strong international performance.

Q:What evidence supports bile acids as a driver of pruritus in PSC, and how does this differ from other diseases?

A:Bile acids play a role in PSC pruritus, but the pathophysiology differs from Alagille and PFIC. In PSC, cholestasis is the central feature, and serum bile acid levels are less critical than in other diseases.

Q:What efforts are being made to increase Livmarli penetration in Europe, and what is the potential patient population from the EXPAND study?

A:Efforts in Europe focus on adding the PFIC indication to Alagille syndrome. The EXPAND study targets various ultra-rare cholestatic pruritus populations, including biliary atresia, with significant potential to grow the patient base.

Q:Review of Unclear Management Responses

A:Management avoided providing a direct answer to the question about the expected revenue distribution between Livmarli and the Bile Acid business, stating only that trends seen year-to-date are expected to continue without breaking down guidance by specific products.

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