Mirum Pharmaceuticals, Inc. (MIRM) Business Model Canvas

Mirum Pharmaceuticals, Inc. (MIRM): Business Model Canvas [Dec-2025 Updated]

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You're digging into Mirum Pharmaceuticals, Inc.'s business model right now because, frankly, the story has changed: they are executing on commercialization and finally pushing toward GAAP profitability. Honestly, seeing projected 2025 net product sales landing between $500 million and $510 million, backed by $378.0 million in cash at the end of Q3, shows their rare disease focus is paying off big time. This canvas lays out the nine precise components-from their high-touch patient relationships to the pipeline fueling future growth-that explain exactly how Mirum Pharmaceuticals, Inc. is building a sustainable, revenue-generating rare disease platform.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Key Partnerships

You're looking at how Mirum Pharmaceuticals, Inc. structures its external relationships to get its rare disease therapies to market and advance its pipeline. For a company focused on orphan drugs, these alliances aren't just helpful; they're the engine for global reach and clinical development. The structure here is definitely leaning on established players for territories where Mirum is still building its footprint.

Takeda for LIVMARLI commercialization in Japan and other territories

The partnership with Takeda Pharmaceutical Company Limited for LIVMARLI (maralixibat) in Japan is a cornerstone. This exclusive licensing deal started back in September 2021 for rare cholestatic pediatric liver diseases. The real payoff came when Takeda secured approval from the Japanese Ministry of Health, Labour, and Welfare on March 27, 2025, making LIVMARLI the first and only treatment for cholestatic pruritus in Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) there.

The financial structure is key here: Mirum is eligible to receive a percentage of Takeda's annual net sales. Specifically, the commercial supply agreement, which kicked off in October 2024, dictates royalty payments ranging from high double digits declining to mid double digits over the first four years from launch, stabilizing at mid double digits thereafter. This revenue stream supports Mirum's overall guidance; they are projecting global net product sales between $420 million and $435 million for 2025. For context, net product sales for the first nine months of 2025 hit $253.6 million, a 70% year-over-year increase, largely driven by LIVMARLI.

The global reach of LIVMARLI through partners is substantial:

  • Approved in more than 40 countries for ALGS.
  • Approved in more than 30 countries for PFIC.
  • The company's Q3 2025 revenue was $133.01 million, with full year 2025 revenue guided toward the upper end of $500 million to $510 million.

Here's a quick look at the patient populations LIVMARLI addresses across its approvals:

Indication US Approval Age (Minimum) EU Approval Age (Minimum)
ALGS Cholestatic Pruritus Three months and older Two months and older
PFIC Cholestatic Pruritus 12 months and older Three months and older

Global network of clinical trial sites and investigators for pipeline studies

Advancing the pipeline requires a robust network of clinical investigators, especially for late-stage studies. Mirum Pharmaceuticals is actively running several trials that depend on these external clinical sites globally. For instance, the VANTAGE study, evaluating volixibat for Primary Biliary Cholangitis (PBC), is being conducted across the United States as well as other countries around the globe, offering an 'at-home' option monitored via telemedicine and home visits. Enrollment for the VISTAS study (volixibat for PSC) was completed in September 2025, with top-line data anticipated in the second quarter of 2026.

The pipeline diversification into neurology is also site-dependent. Mirum enrolled the first patient in the Phase 2 BLOOM study for MRM-3379 in Fragile X Syndrome (FXS) in December 2025. FXS affects an estimated 50,000 males in the U.S. and E.U.. Top-line data from this study is expected in 2027. This network must manage complex logistics, as evidenced by the fact that travel compensation may be provided if a study location isn't nearby.

Specialty pharmacies and distributors for rare disease drug delivery

Getting drugs like LIVMARLI, CHOLBAM, and CTEXLI to patients with rare diseases requires specialized distribution partners. Mirum confirms that all three approved medications have established reimbursable pathways with most payers. This is critical in a market where, as of January 2025, 382 unique specialty drugs were managed through manufacturer-defined limited or exclusive pharmacy networks.

The distribution landscape is concentrated, but Mirum must navigate it carefully. In 2024, specialty pharmacies collectively dispensed an estimated $265 billion in specialty pharmaceuticals. A key dynamic is exclusivity; analysis suggests 34% of specialty drugs have exclusive networks (only one pharmacy). Mirum's strategy relies on securing access through these specialty channels to ensure appropriate patients can start and stay on therapy, supported by their Patient Assistance Program.

Patient advocacy groups for disease awareness and support programs

For rare diseases, advocacy groups are essential partners, not just for awareness but for shaping clinical understanding. Mirum Pharmaceuticals demonstrated this commitment by actively engaging with clinicians, researchers, and advocates at major medical meetings like NASPGHAN 2025 and AASLD 2025. This engagement helps Mirum further understand and enhance care for these communities.

The partnership extends into trial design itself. Patients and families play a crucial role in how Mirum evaluates its investigational treatments, including the development of patient-centric tools such as surveys, pruritus severity scales, and e-diaries to better capture the burden of rare diseases. Furthermore, Mirum supports access through an expanded access program open across multiple countries for eligible ALGS and PFIC patients, working with dedicated healthcare teams to navigate insurance and financial support.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Key Activities

You're looking at the engine room of Mirum Pharmaceuticals, Inc. (MIRM) right now, which is all about driving revenue from current products while simultaneously pushing high-stakes pipeline assets through late-stage trials. The key activities are heavily weighted toward commercial execution and clinical milestones as of late 2025.

Commercializing three approved rare disease medicines (LIVMARLI, CHOLBAM, CTEXLI)

The commercial engine is clearly running hot, with the company raising its full-year 2025 revenue guidance to between $500 million and $510 million. This performance is anchored by LIVMARLI, which management projects will become a billion-dollar brand. The focus here is maximizing penetration in existing markets and leveraging new formulations.

Here's a snapshot of the commercial performance through the third quarter of 2025:

Metric Value (Q3 2025) Year-over-Year Change
Total Global Net Product Sales $133.0 million Up 47.1%
LIVMARLI Net Product Sales $92.2 million Up 56%
LIVMARLI U.S. Net Sales $64 million N/A
LIVMARLI Ex-U.S. Net Sales $28 million N/A
Bile Acid Medicines (CHOLBAM/CTEXLI) Net Product Sales $40.8 million Up 31%

For the first nine months of 2025, LIVMARLI's net product sales hit $253.6 million, which is a 70% increase year over year. The company achieved its first quarter of positive net income in Q3 2025, reporting approximately $3 million, which definitely shows the impact of this commercial success. In the Alagille syndrome market in the U.S., LIVMARLI has achieved approximately 50% market penetration.

Conducting pivotal Phase 2b/3 trials for Volixibat (PSC, PBC)

The key activity here is advancing Volixibat, an investigational oral IBAT inhibitor, through two potentially registrational studies targeting areas with no approved therapies. This is about setting up the next major revenue driver. The progress is concrete, with one trial finishing enrollment and the other on track to finish soon.

  • VISTAS (PSC): Enrollment complete as of September 2025.
  • VISTAS Topline Data Expected: Second quarter of 2026.
  • VANTAGE (PBC): Expected to complete enrollment in 2026.
  • VANTAGE Topline Data Expected: First half of 2027.

The prior interim analysis for the VANTAGE study in PBC already showed statistically significant improvement in pruritus, plus meaningful reductions in serum bile acids and improvements in fatigue. You know, fatigue and sleep are huge quality-of-life issues in these chronic diseases, so that data is important.

Expanding LIVMARLI label via the EXPAND Phase 3 study

This activity focuses on broadening the utility of the flagship product into ultra-rare settings of cholestatic pruritus beyond the current approved indications. The EXPAND study is designed to enroll up to 90 people, ages 6 months and older, across multiple global sites. The timeline for this expansion is set for 2026 and beyond, which is a defintely a longer-term catalyst compared to the Volixibat data.

Key milestones for the EXPAND study include:

  • Target Population Estimate: Approximately 500 U.S. patients with ultra-rare cholestatic pruritus.
  • Enrollment Completion Target: Expected in 2026.
  • Topline Data Expected: First half of 2027.

Executing global regulatory filings and market access strategies

This involves securing and expanding market access for the current portfolio and advancing pipeline assets through regulatory pathways. The team secured several key approvals in 2025, which immediately added to the commercial base. For instance, the FDA approved a new tablet formulation of LIVMARLI earlier this year, which is seeing high conversion rates among eligible patients, suggesting good uptake due to patient convenience.

Recent regulatory and market access achievements include:

  • FDA approval for CTEXLI tablets in Q1 2025.
  • Approval of LIVMARLI in tablet formulation in the U.S. in 2025.
  • Approval of LIVMARLI in Japan in Q1 2025, expanding global presence.
  • The PFIC launch exceeded expectations, largely due to market access strategies leveraging new patient identification via genetic testing.

Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Key Resources

You're looking at the core assets Mirum Pharmaceuticals, Inc. (MIRM) is leaning on right now, late in 2025. These aren't just line items; they are the foundation supporting their rare disease focus.

The approved rare disease portfolio is the immediate engine. Mirum Pharmaceuticals, Inc. has three key marketed products: LIVMARLI (maralixibat), CHOLBAM (cholic acid), and CTEXLI (chenodiol) tablets. The commercial performance in the third quarter of 2025 really shows how this portfolio is performing right now.

Product Group Q3 2025 Net Product Sales Growth Driver Detail
LIVMARLI $92.2 million 56% growth year-over-year
Bile Acid Medicines (CHOLBAM and CTEXLI) $40.8 million 31% growth year-over-year
Total Global Net Product Sales $133.0 million 47% growth year-over-year

This product base is supported by a strong balance sheet. As of September 30, 2025, Mirum Pharmaceuticals, Inc. held unrestricted cash, cash equivalents, and investments totaling $378.0 million. That's a solid war chest, especially since the company reported its first-ever positive net income of approximately $3 million in that same quarter.

Next up, the intellectual property protecting the IBAT inhibitor platform-which includes maralixibat (LIVMARLI) and volixibat-is a critical, long-term asset. You should know that LIVMARLI is protected by 9 US drug patents filed between 2024 and 2025, with an estimated generic launch date around Oct 05, 2043. Still, there's some near-term noise; Sandoz, Inc. filed an Abbreviated New Drug Application challenging five of these Orange Book-listed patents in November 2025. Mirum Pharmaceuticals, Inc. plans to enforce its IP vigorously, but this litigation introduces a variable to the long-term exclusivity view.

The specialized rare disease commercial and medical affairs teams are the people who make the products move. You can see the investment in these functions reflected in the Selling, General and Administrative (SG&A) expenses for the third quarter of 2025, which totaled $61.9 million. This team is focused on driving adoption for the current portfolio and supporting pipeline advancements. Here are some of the key areas they support:

  • LIVMARLI label expansion via the Phase 3 EXPAND study.
  • Commercial execution across Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) indications.
  • Support for the recently approved CTEXLI for cerebrotendinous xanthomatosis (CTX).

The company's commitment to R&D, which hit $43 million in Q3 2025, also represents a key resource in the form of scientific talent and ongoing pipeline development, including the volixibat studies for Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC). Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Value Propositions

You're looking at the core value Mirum Pharmaceuticals, Inc. (MIRM) delivers to its customers-the patients and caregivers navigating these incredibly rare conditions. It's all about providing the first, or the best, option where little else existed.

The commercial engine is clearly LIVMARLI (maralixibat), which is positioned as a potential billion-dollar brand, driving significant top-line growth. For the third quarter of 2025 alone, LIVMARLI net product sales hit $92.2 million, marking a 56% growth over the third quarter of 2024. This strong performance helped Mirum Pharmaceuticals, Inc. achieve its first quarter of positive net income, reported at approximately $3 million for Q3 2025, on total revenues of $133.0 million. The company has since raised its full-year 2025 revenue guidance to a range of $500 to $510 million.

First and only approved treatment for cholestatic pruritus in ALGS patients (LIVMARLI)

For patients with Alagille syndrome (ALGS), the value proposition centers on addressing cholestatic pruritus (itch), a symptom that affects up to 88% of this population and is considered the most burdensome.

  • LIVMARLI is approved in the U.S. for ALGS patients 3 months and older.
  • In Europe, the authorization covers ALGS patients 2 months and older.
  • The drug works by blocking bile acid reabsorption, reducing systemic bile acid levels.

Clinically meaningful, sustained benefit for rare cholestatic liver diseases

The sustained efficacy data from the clinical programs is a key differentiator, especially given that unmanageable itch was a common reason for liver transplant in many ALGS patients. Here's the quick math on the impact:

Clinical Endpoint/Timeframe Patient Response/Outcome Data Point
First Year of Treatment (Once-Daily) Clinically meaningful improvement in pruritus (ItchRO score of 1 or more) More than 80% of patients experienced this
Long-Term Treatment (Nearly 4 Years) Relief from cholestatic pruritus maintained Patients experienced little to no itch for the vast majority (95%) of days on treatment
6 Years Post-Start (for responders) Transplant-free survival 93% of those with more than a 1-point itch reduction were alive without a transplant

Also, an analysis of adolescents showed that for those who started LIVMARLI before age 16, the improvement in pruritus and reduction in blood bile acids persisted into late adolescence or adulthood.

Convenient single tablet formulation of LIVMARLI for older patients

Recognizing that treatment adherence can shift with age, Mirum Pharmaceuticals, Inc. delivered a formulation improvement. The FDA approved a new tablet formulation of LIVMARLI for cholestatic pruritus in ALGS and PFIC patients. This tablet is expected to improve compliance and persistence, especially among older patients.

Treatments for ultra-rare bile acid synthesis disorders and CTX

Beyond LIVMARLI, the value proposition includes established treatments for other ultra-rare genetic disorders, diversifying the company's commercial base. Sales from these Bile Acid Medicines, which include CHOLBAM and CTEXLI, were $40.8 million in Q3 2025, growing 31% year-over-year.

For you, the analyst, this means a diversified revenue stream anchored by a high-growth specialty product:

  • CHOLBAM (cholic acid): Approved for bile acid synthesis disorders due to single enzyme defects; an estimated 200 to 300 patients are current candidates for this therapy.
  • CTEXLI (chenodiol): FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.

Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Customer Relationships

You're focused on how Mirum Pharmaceuticals, Inc. builds and maintains its connection with the specialized patient and physician communities it serves. In the rare disease space, this relationship isn't just about selling a drug; it's about comprehensive support because the diseases are chronic and the patient pool is small and highly specific.

High-touch support programs for patients and caregivers in rare diseases

Mirum Pharmaceuticals, Inc. deploys the Mirum Access Plus (MAP) program to shepherd patients and their families through the treatment journey for all its medicines, including LIVMARLI, CHOLBAM, CHENODAL, and CTEXLI. This high-touch approach is critical for adherence in chronic conditions. MAP experts help by explaining the process of getting medication, working with healthcare teams to sort out insurance coverage, and identifying eligibility for financial support. For LIVMARLI, eligible patients with commercial insurance can pay as little as $\mathbf{\$10}$ out of pocket per fill. Furthermore, the Mirum Patient Assistance Program (PAP) offers free medication for up to $\mathbf{1}$ calendar year for patients without insurance coverage, with eligibility reviewed annually. This level of support directly impacts patient persistence on therapy.

The commercial success reflects this focus, with Q3 2025 global net product sales reaching $\mathbf{\$133.0}$ million, and the full-year 2025 revenue guidance updated to $\mathbf{\$500}$ to $\mathbf{\$510}$ million. This strong execution in a niche market points to effective patient access strategies.

Here's a look at the commercial performance driving these relationships as of late 2025:

Product Category Q3 2025 Net Product Sales Year-over-Year Growth (vs Q3 2024) Market Penetration/Status
LIVMARLI $\mathbf{\$92.2}$ million $\mathbf{56\%}$ Projected to be a billion-dollar brand
Bile Acid Medicines (CHOLBAM/CTEXLI) $\mathbf{\$40.8}$ million $\mathbf{31\%}$ CHOLBAM candidates estimated at $\mathbf{200}$ to $\mathbf{300}$ patients

Direct engagement with specialized hepatologists and pediatric gastroenterologists

Engagement with the core prescribing specialists is non-negotiable in ultra-rare diseases. Mirum Pharmaceuticals, Inc. demonstrates this commitment through active participation in key medical congresses. For instance, the company aligned with clinicians and researchers at major events like NASPGHAN 2025 and AASLD 2025, focusing on rare liver disease awareness. This isn't just networking; it's about fostering community ties and engaging in serious dialogues to better understand the care landscape. For a condition like Primary Sclerosing Cholangitis (PSC), where Mirum is pursuing a label expansion for Volixibat, the estimated U.S. patient population is roughly $\mathbf{30,000}$, yet market research suggests inadequate discussion of pruritus with physicians, highlighting an educational opportunity for Mirum's specialists to address.

Dedicated commercial team for market education and access assistance

The commercial team's primary role is education and access, which is evident in the performance of LIVMARLI. The drug is approved for cholestatic pruritus in Alagille syndrome (ALGS), and Mirum has achieved approximately $\mathbf{50\%}$ market penetration in the U.S. for this indication. This suggests the commercial team is effectively reaching the right prescribers and educating them on the product's value proposition. The introduction of the LIVMARLI tablet formulation earlier this year is also a key commercial touchpoint, expected to improve compliance and persistence among eligible patients who convert from the oral solution.

The commercial team's success is measured by the growth in their core products:

  • LIVMARLI net sales grew $\mathbf{87\%}$ year-over-year in Q2 2025.
  • The company achieved its first quarter of positive net income in Q3 2025, signaling commercial maturity.
  • The team supports access for all approved medicines: LIVMARLI, CHOLBAM, CHENODAL, and CTEXLI.

Long-term patient retention due to chronic nature of rare liver diseases

The chronic nature of diseases like ALGS and PFIC inherently supports long-term patient retention, provided the therapy is effective and well-tolerated. LIVMARLI, being an ongoing treatment for cholestatic pruritus, benefits from this dynamic. The company's focus is on ensuring patients stay on therapy, which is where the MAP program's ongoing education and support become crucial over years, not just months. The high capture rate of newly diagnosed ALGS patients further solidifies this long-term relationship foundation. It's a defintely sticky patient base.

The company's leadership, including CEO Chris Peetz who has been in the role since March 2019, emphasizes this commitment to the rare disease community, which underpins the long-term relationship strategy. Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Channels

You're looking at how Mirum Pharmaceuticals, Inc. gets its specialized, rare-disease medicines to the patients who need them, which is a critical part of their business model, especially given their focus on orphan drugs. This involves a mix of specialized logistics and direct engagement with the medical community.

Specialty pharmacy networks for drug distribution and fulfillment.

The distribution relies heavily on established specialty pharmacy networks to handle the fulfillment of their commercial products, like LIVMARLI and the Bile Acid Medicines. The success of these channels is directly reflected in the reported sales figures for the third quarter of 2025, which hit $133.0 million in global net product sales. LIVMARLI, their flagship, drove the majority of this, with net sales reaching $92.2 million in Q3 2025, representing 56% year-over-year growth. Bile Acid Medicines contributed $40.8 million in net product sales for the same period, showing 31% growth YoY. This indicates that the fulfillment and reimbursement pathways for these drugs are functioning effectively to reach patients across their approved indications. The company has an expanded access program running across multiple countries for eligible patients with ALGS and PFIC, which also utilizes specific distribution pathways.

Direct sales force targeting specialized treatment centers in the U.S.

Mirum Pharmaceuticals, Inc. deploys a focused commercial team to target specialized treatment centers in the U.S., where patients with rare liver diseases are concentrated. While the exact size of the direct sales force isn't public, the commercial focus is clear from the product performance. LIVMARLI sales in the U.S. are a key driver, accounting for approximately 69% of the total global net product sales in the third quarter of 2025. The company is executing with financial discipline to support this commercial footprint while advancing its pipeline.

International distributors and partners for ex-U.S. market access.

For markets outside the U.S., Mirum Pharmaceuticals, Inc. leans on international distributors and partners to manage commercialization and ensure patient access. The company actively manages these relationships, specifically mentioning roles focused on driving commercialization strategies in regions like Latin America and the Middle East. LIVMARLI has approvals in Europe, with specific patient age indications varying by country, such as approval for patients three months and older in Europe for ALGS. The company maintains dedicated contact points for various European regions to facilitate access and support.

Digital and in-person medical education for healthcare professionals.

Engaging healthcare professionals (HCPs) through medical education and scientific exchange is a core channel for driving awareness and appropriate use of their specialized therapies. Mirum Pharmaceuticals, Inc.'s Medical Affairs team supports this by maintaining relationships with the scientific community. They provide specific contact points for Medical Information requests across key markets, which is a direct channel for HCP education and support. Here are some of the dedicated contact numbers available for Medical Information as of late 2025:

  • U.S. Medical Information: 1 855 676 4968.
  • Canada Medical Information: 1 833 548 6754.
  • Germany Medical Information: +49 89 20194073.
  • France Medical Information: 0 805 98 78 78 or +33 1 85 14 93 17.
  • United Kingdom Medical Information: 2038853479.

The company also supports investigator-initiated original research to enhance the understanding of rare liver diseases.

The overall commercial performance, which is the ultimate measure of channel effectiveness, is projected to result in full-year 2025 revenue guidance between $500 million and $510 million.

Metric Product/Segment Q3 2025 Value Year-over-Year Growth (Q3 2025 vs Q3 2024)
Global Net Product Sales Total $133.0 million Not specified directly, but total revenue grew from $90.4 million in Q3 2024.
Net Product Sales LIVMARLI $92.2 million 56%
Net Product Sales Bile Acid Medicines $40.8 million 31%
Revenue Guidance Full Year 2025 $500 to $510 million N/A

Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Customer Segments

You're looking at the core patient populations Mirum Pharmaceuticals, Inc. (MIRM) targets with its approved therapies and late-stage pipeline assets as of late 2025. Honestly, their strategy is laser-focused on ultra-rare, high-need cholestatic diseases, with a strategic expansion into other rare conditions.

The current commercial focus centers on patients with conditions where LIVMARLI (maralixibat) is approved, alongside the patient base for their Bile Acid Medicines portfolio, which includes the recently approved therapy for CTX.

Indication Patient Population Detail Approved Product/Status Prevalence/Guidance Data (US/EU)
Alagille Syndrome (ALGS) Pediatric and adult patients with cholestatic pruritus. LIVMARLI (Approved in US 3+ months, Europe 2+ months) Only between 4,000 and 5,500 patients in the U.S. and the European Union have ALGS.
Progressive Familial Intrahepatic Cholestasis (PFIC) Pediatric and adult patients with cholestatic pruritus. LIVMARLI (Approved in US 12+ months, Europe 3+ months) 1,000+ US/EU prevalence.
Cerebrotendinous Xanthomatosis (CTX) Adult patients with the rare, progressive disorder of cholesterol metabolism. CHENODAL (FDA Approved) Bile Acid Medicines net product sales were $40.8 million in the third quarter of 2025.

Mirum Pharmaceuticals, Inc. is also actively pursuing indications that represent significant future customer segments, primarily through the development of volixibat. These are not yet commercialized for these specific uses, but they represent major growth vectors.

Here's the quick math on the pipeline-driven segments you asked about:

  • Patients with Primary Sclerosing Cholangitis (PSC): Volixibat VISTAS Phase 2b study enrollment is complete; topline data expected in the second quarter of 2026.
  • Patients with Primary Biliary Cholangitis (PBC): Volixibat VANTAGE Phase 2b study enrollment is expected to complete in 2026.

To give you a sense of the current scale driving this, Mirum Pharmaceuticals, Inc. reported third quarter 2025 global net product sales of $133.0 million, and the full year 2025 revenue guidance is set at the upper end of $500 million to $510 million.

Also, keep in mind the company is building out its franchise, as evidenced by the fact that they have 3 APPROVED RARE DISEASE PRODUCTS and 4 ADDITIONAL INDICATIONS IN DEVELOPMENT in high-need orphan indications as of early 2025.

Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Cost Structure

You're looking at the core spending that fuels Mirum Pharmaceuticals, Inc.'s growth engine right now, especially as they scale their commercial footprint. The cost structure is heavily weighted toward supporting their growing product portfolio and advancing the pipeline.

High R&D expenses, totaling $43 million in Q3 2025, are necessary to fund the pipeline, which includes key studies like the VISTAS study for volixibat in primary sclerosing cholangitis (PSC) and the BLOOM Phase 2 study in Fragile X Syndrome. This R&D spend reflects the commitment to developing new therapies beyond their current liver-focused indications.

Significant Selling, General, and Administrative (SG&A) costs were $62 million in Q3 2025. This level of spending is directly tied to the commercial expansion of Livmarli and the integration and market support for the recently acquired bile acid medicines, Cholbam and Ctexli. Honestly, scaling a commercial team and driving adoption for rare disease drugs requires substantial upfront investment in sales infrastructure and marketing efforts.

Manufacturing and Cost of Goods Sold (COGS) for the three commercial products-Livmarli, Cholbam, and Ctexli-resulted in a Cost of Sales of $26 million for the third quarter of 2025. This figure is a direct cost associated with generating the $133 million in total net product revenue reported for the same period. If onboarding takes 14+ days, churn risk rises, and similarly, any manufacturing hiccup here directly impacts gross margin.

Costs associated with global regulatory compliance and market access are embedded within the R&D and SG&A lines, as Mirum Pharmaceuticals, Inc. manages approvals and reimbursement across multiple geographies for Livmarli. While there isn't a single, isolated line item for this in the top-level reporting, the $43 million R&D and $62 million SG&A figures certainly cover the significant effort required to maintain compliance and secure payer access globally.

Here's the quick math on the operating expenses for the quarter ended September 30, 2025:

Expense Category Q3 2025 Amount (in millions USD)
Research & Development (R&D) $43 million
Selling, General, and Administrative (SG&A) $62 million
Cost of Sales (COGS) $26 million
Total Operating Expense $130.4 million

The cost structure is evolving as the company moves toward sustained profitability. You can see the shift in focus:

  • Focus on commercial execution driving SG&A.
  • Continued pipeline investment in R&D.
  • Non-cash expenses included: $18 million in stock-based compensation.
  • Non-cash expenses included: $6 million in intangible amortization.
  • The result of this spending: first-time GAAP profitability at approximately $3 million net income.

What this estimate hides is the variable nature of international ordering patterns, which can affect the timing of revenue recognition versus the fixed nature of the operating expenses you see above. Finance: draft 13-week cash view by Friday.

Mirum Pharmaceuticals, Inc. (MIRM) - Canvas Business Model: Revenue Streams

You're looking at the core ways Mirum Pharmaceuticals, Inc. brings in cash right now, which is really all about their commercial products. The company has definitely seen strong momentum from its approved therapies, leading to an updated outlook for the year.

Here's a quick look at the key revenue figures we have for the end of 2025, based on the latest reports:

Revenue Component Period / Projection Amount
Global Net Product Sales Projection Full-Year 2025 $500 million to $510 million
Total Global Net Product Sales Q3 2025 $133.0 million
LIVMARLI Net Product Sales Q3 2025 $92.2 million
Bile Acid Medicines Net Product Sales Q3 2025 $40.8 million

LIVMARLI (maralixibat) is clearly the engine driving a large part of the top line. For the third quarter of 2025, net product sales for LIVMARLI hit $92.2 million. That represents a 56% growth compared to the third quarter of 2024, showing strong adoption across its approved indications, including Alagille syndrome and Progressive Familial Intrahepatic Cholestasis (PFIC). To be fair, this single product accounted for roughly 69% of the total net product sales in that quarter.

Next up, you have the revenue from the Bile Acid Medicines portfolio, which includes CHOLBAM (cholic acid) capsules and CTEXLI (chenodiol) tablets. These products generated $40.8 million in net product sales for the third quarter of 2025. That's a 31% year-over-year growth for that segment, which is solid performance for these established therapies.

So, the revenue streams are primarily product sales, but there are other potential inflows to keep an eye on:

  • Net product sales from LIVMARLI.
  • Net product sales from CHOLBAM and CTEXLI.
  • Potential future milestone payments from international partners.

Regarding those potential future payments, Mirum Pharmaceuticals has an exclusive licensing agreement with Takeda Pharmaceutical Company Limited for the development and commercialization of LIVMARLI in Japan for indications like Alagille syndrome and PFIC. While the search results confirm this partnership and a commercial supply agreement entered into in October 2024, they don't specify any concrete, upcoming milestone payment amounts due from Takeda as of late 2025, though such structures typically include them. Finance: draft 13-week cash view by Friday.


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