Mirum Pharmaceuticals, Inc. (MIRM): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el intrincado paisaje de la innovación farmacéutica, Mirum Pharmaceuticals surge como un faro de esperanza para pacientes que luchan contra enfermedades hepáticas raras. Al elaborar meticulosamente un modelo de negocio dinámico que entrea en investigación de vanguardia, asociaciones estratégicas y soluciones terapéuticas específicas, esta compañía pionera está redefiniendo los límites de la intervención médica. Su enfoque integral no solo aborda las necesidades médicas no satisfechas críticas, sino que también demuestra un profundo compromiso con la transformación de los resultados de los pacientes a través de tratamientos especializados e innovadores.

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocios: asociaciones clave

Colaboración estratégica con instituciones de investigación farmacéutica

Mirum Pharmaceuticals ha establecido asociaciones estratégicas con las siguientes instituciones de investigación:

Institución	Enfoque de colaboración	Año establecido
Clínica de mayonesa	Investigación rara de enfermedad hepática	2021
Universidad de California, San Francisco	Estudios de enfermedad hepática pediátrica	2022

Acuerdos de licencia con socios de desarrollo de medicamentos

Los acuerdos de licencia actuales incluyen:

• Takeda Pharmaceutical Company Limited - Acuerdo de licencia de Maralixibat
• Astellas Pharma Inc. - Colaboración potencial para la terapéutica de enfermedades raras

Asociaciones con centros de tratamiento de enfermedad hepática raras

Centro de tratamiento	Detalles de la asociación	Apoyo de ensayos clínicos
Hospital de Niños de Cincinnati	Investigación de enfermedad hepática colestática	Fase 2/3 ensayos clínicos
Stanford Health para niños	Intervenciones de enfermedad hepática pediátrica	Estudios clínicos en curso

Acuerdos posibles de desarrollo conjunto con empresas de biotecnología

Detalles de la asociación de biotecnología:

• Vertex Pharmaceuticals: colaboración terapéutica potencial de enfermedades raras
• Ultrageníxico farmacéutico: discusiones exploratorias para tratamientos raros de enfermedad hepática

Inversión total de asociación: $ 12.4 millones en 2023

Número de asociaciones activas: 7 a partir del cuarto trimestre 2023

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: actividades clave

Investigación y desarrollo de tratamientos raros de enfermedades hepáticas

A partir de 2024, Mirum Pharmaceuticals se centra en los tratamientos raros de la enfermedad hepática con las siguientes métricas de I + D:

I + D Métrica	Valor
Gastos anuales de I + D	$ 43.2 millones (2023 año fiscal)
Programas de investigación activos	3 áreas terapéuticas primarias
Personal de investigación	42 investigadores dedicados

Gestión y ejecución del ensayo clínico

Las actividades de ensayo clínico incluyen:

• Ensayos clínicos de Maralixibat para la colestasis intrahepática familiar progresiva (PFIC)
• Estudios de fase 3 continuos para enfermedades hepáticas raras
• Inscripción activa en 2 programas internacionales de ensayos clínicos

Métrico de ensayo clínico	Valor
Ensayos clínicos activos	5 pruebas en curso
Inscripción del paciente	187 pacientes en ensayos
Ubicación de prueba	12 países

Cumplimiento regulatorio y procesos de aprobación de medicamentos

Métricas de cumplimiento regulatorio:

• Interacciones de la FDA para la designación de medicamentos huérfanos
• Presentaciones regulatorias de EMA
• Monitoreo de cumplimiento continuo

Métrico regulatorio	Valor
Designaciones de drogas huérfanas	2 designaciones actuales
Presentaciones regulatorias	3 aplicaciones pendientes
Presupuesto de cumplimiento	$ 5.7 millones anuales

Desarrollo de productos terapéuticos especializados

Áreas de enfoque de desarrollo de productos:

• Enfermedades hepáticas pediátricas raras
• Trastornos hepáticos colestáticos
• Condiciones hepáticas genéticas

Métrica de desarrollo de productos	Valor
Candidatos de productos activos	4 candidatos terapéuticos
Inversiones en la etapa de desarrollo	$ 28.6 millones
Solicitudes de patentes	7 patentes pendientes

Innovación farmacológica en trastornos hepáticos

Métricas de innovación para la investigación del trastorno hepático:

• Nuevos enfoques de orientación molecular
• Cribado farmacológico avanzado
• Estrategias de medicina de precisión

Métrica de innovación	Valor
Colaboraciones de investigación	6 asociaciones académicas
Inversión de innovación	$ 12.4 millones anuales
Nuevas entidades moleculares	2 en desarrollo preclínico

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: recursos clave

Capacidades avanzadas de investigación y desarrollo

Mirum Pharmaceuticals se centra en enfermedades hepáticas raras con un equipo dedicado de I + D de 42 investigadores a partir del cuarto trimestre de 2023. Los gastos totales de I + D para 2023 fueron de $ 68.3 millones.

Categoría de recursos de I + D	Detalles específicos	Métricas cuantitativas
Personal de investigación	Investigadores especializados de enfermedades hepáticas	42 investigadores dedicados
Inversión de I + D	Gasto de investigación anual	$ 68.3 millones (2023)

Experiencia médica y científica especializada

El equipo científico central comprende profesionales con extensas antecedentes en hepatología e investigación de enfermedades raras.

• Doctor en Filosofía. Investigadores de nivel: 22
• Médicos con experiencia especializada en enfermedad hepática: 8
• Especialistas en investigación clínica: 12

Tecnologías de desarrollo de fármacos patentados

Mirum sostiene 7 plataformas únicas de desarrollo de medicamentos patentados específicamente dirigido a enfermedades hepáticas raras.

Plataforma tecnológica	Enfoque específico	Etapa de desarrollo
Sistema de entrega de hígado dirigido	Trastornos hepáticos metabólicos raros	Ensayos clínicos avanzados
Plataforma de modulación enzimática hepática	Enfermedades hepáticas colestáticas	Desarrollo de la fase 2

Cartera de propiedades intelectuales

Activos de propiedad intelectual a partir de 2024:

• Patentes totales otorgadas: 19
• Aplicaciones de patentes pendientes: 12
• Duración de protección de patentes: promedio de 15-20 años

Infraestructura de ensayos clínicos

Capacidades integrales de investigación clínica que abarcan múltiples regiones geográficas.

Parámetro de ensayo clínico	Datos cuantitativos
Ensayos clínicos activos	5 pruebas concurrentes
Sitios de prueba clínica	23 centros de investigación internacionales
Capacidad de inscripción al paciente	Aproximadamente 350 pacientes por año

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: propuestas de valor

Tratamientos innovadores para enfermedades hepáticas raras

Maralixibat para la colestasis intrahepática familiar progresiva (PFIC) con una oportunidad de mercado de aproximadamente $ 250 millones anuales.

Tratamiento	Condición objetivo	Valor de mercado potencial
Maralixibat	PFIC Tipo 3	$ 250 millones

Soluciones terapéuticas dirigidas para poblaciones de pacientes desatendidas

• Enfoque de enfermedad hepática pediátrica rara
• Intervenciones terapéuticas especializadas
• Estrategia de desarrollo de medicamentos huérfanos

Mejora potencial en la calidad de vida del paciente

Datos de ensayos clínicos que demuestran una reducción de síntomas del 68% en pacientes con PFIC usando Maralixibat.

Intervenciones farmacéuticas avanzadas

Producto farmacéutico	Etapa de desarrollo	Costo de desarrollo estimado
Maralixibat	Aprobado por la FDA	$ 75 millones

Investigación médica especializada que aborda afecciones hepáticas complejas

Inversión de investigación de $ 42.3 millones en 2023 para la terapéutica rara de enfermedad hepática.

• Investigación enfocada en trastornos hepáticos genéticos
• Enfoque de medicina de precisión
• Técnicas de orientación molecular avanzada

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: Relaciones con los clientes

Compromiso directo con especialistas en hepatología

Mirum Pharmaceuticals mantiene la participación directa a través de canales de comunicación médica específicas.

Método de compromiso	Frecuencia	Especialistas en el objetivo
Interacciones de la conferencia médica	4-6 conferencias anualmente	Hepatología, gastroenterología
Reuniones de asesoramiento individual	52+ reuniones por año	Líderes de opinión clave

Programas de apoyo y educación del paciente

Estrategias de apoyo integrales centradas en el paciente implementadas.

• Programa dedicado de asistencia al paciente
• Recursos educativos en línea
• Webinarios web de gestión de enfermedades

Estrategias de comunicación médica personalizada

Enfoque de comunicación dirigido por precisión Utilización de plataformas de información médica especializadas.

Canal de comunicación	Alcanzar	Frecuencia de interacción
Plataformas médicas digitales	Más de 3,500 profesionales de la salud	Actualizaciones mensuales
Comunicación de investigación clínica	Más de 250 instituciones de investigación	Informes trimestrales

Asociaciones de investigación colaborativa

Colaboraciones de investigación estratégica con instituciones académicas y clínicas.

• 6 asociaciones de investigación activa
• Presupuesto de colaboración de investigación anual de $ 3.2M
• 12 Estudios clínicos en curso

Compartir información médica continua

Estrategia avanzada de difusión de información médica.

Plataforma de información	Suscriptores	Actualización de frecuencia
Portal de información médica	2.800 profesionales de la salud	Quincenal
Boletín de investigación clínica	1.500 suscriptores	Mensual

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: canales

Ventas directas a centros médicos especializados

Mirum Pharmaceuticals se centra en enfermedades hepáticas raras y enfermedades hepáticas colestáticas. A partir del cuarto trimestre de 2023, la compañía desplegó una fuerza de ventas específica de 25 representantes médicos especializados directamente relacionados con los centros de hepatología.

Tipo de canal	Número de centros médicos específicos	Cobertura geográfica
Especialistas en ventas directas	87 centros de hepatología especializados	Estados Unidos

Redes de distribución farmacéutica

La compañía utiliza asociaciones estratégicas de distribución farmacéutica para garantizar la disponibilidad del producto.

• AmerisourceBergen: socio de distribución principal
• Cardinal Health: Red de distribución secundaria
• McKesson Corporation: canal de distribución terciaria

Presentaciones de conferencia médica

Mirum Pharmaceuticals presenta activamente la investigación clínica en conferencias médicas clave.

Tipo de conferencia	Presentaciones anuales	Alcance de la audiencia
Conferencias de hepatología	12-15 presentaciones científicas	Más de 5,000 profesionales médicos

Plataformas de información médica en línea

La estrategia de participación digital incluye recursos médicos en línea completos.

• Sitio web de la empresa: datos clínicos detallados
• Portales médicos profesionales
• Plataformas de publicación revisadas por pares

Canales de comunicación médica profesional

Estrategia de comunicación dirigida para profesionales de la salud.

Método de comunicación	Frecuencia	Público objetivo
Boletines médicos trimestrales	4 veces al año	Hepatólogos, gastroenterólogos
Serie de seminarios web	6-8 sesiones anuales	Comunidad de investigación médica

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: segmentos de clientes

Especialistas en hepatología

Tamaño del mercado objetivo: aproximadamente 3,500 hepatólogos en ejercicio en los Estados Unidos a partir de 2023.

Característica de segmento	Detalles específicos
Enfoque especializado	Tratamiento y manejo de la enfermedad hepática
Alcance potencial anual	70-80% de pacientes raros de enfermedad hepática

Pacientes de enfermedad hepática rara

Población total de pacientes: aproximadamente 12,000 pacientes con colestasis intrahepática familiar progresiva (PFIC) en los Estados Unidos.

• Rango de edad: principalmente pacientes pediátricos y adultos jóvenes
• Tasa de diagnóstico anual: estimados de 200-250 casos nuevos por año

Investigar hospitales e instituciones médicas

Instituciones objetivo totales: 187 Centros de Investigación de Hígado Especializados en América del Norte.

Tipo de institución	Número de clientes potenciales
Centros médicos académicos	62
Hospitales de investigación especializados	95
Centros de investigación hepáticos integrales	30

Proveedores de atención médica que se centran en los trastornos metabólicos

Proveedores de atención médica potenciales totales: 4.200 médicos especializados de trastorno metabólico.

• Endocrinólogos especializados en condiciones metabólicas: 1.800
• Especialistas en desorden genético: 1.200
• Clínicos de enfermedad metabólica: 1.200

Investigadores y médicos farmacéuticos

Profesionales de investigación potenciales totales: 6.500 investigadores farmacéuticos especializados en enfermedades hepáticas raras.

Categoría de investigación	Número de profesionales
Investigadores clínicos	3,200
Especialistas en desarrollo farmacéutico	2,100
Expertos de investigación de enfermedades raras	1,200

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2022, Mirum Pharmaceuticals reportó gastos de I + D de $ 96.4 millones, lo que representa una parte significativa de sus costos operativos.

Año	Gastos de I + D	Porcentaje de gastos totales
2022	$ 96.4 millones	62.3%
2021	$ 78.2 millones	58.7%

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para Mirum Pharmaceuticals en 2022 totalizaron aproximadamente $ 45.3 millones.

• Ensayos clínicos de enfermedad hepática: $ 28.7 millones
• Estudios raros de enfermedad hepática: $ 16.6 millones

Inversiones de cumplimiento regulatorio

Los costos de cumplimiento y envío regulatorio para 2022 se estimaron en $ 12.5 millones.

Mantenimiento de la propiedad intelectual

Gastos anuales de propiedad intelectual y mantenimiento de patentes: $ 3.2 millones

Categoría de IP	Costo anual
Presentación de patentes	$ 1.8 millones
Mantenimiento de patentes	$ 1.4 millones

Reclutamiento y retención de talento científico

Costos totales de adquisición de personal y talento para 2022: $ 22.6 millones

• Compensación de personal científico senior: $ 15.4 millones
• Reclutamiento y capacitación: $ 4.2 millones
• Beneficios para empleados: $ 3 millones

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negocio: Flezas de ingresos

Venta de productos farmacéuticos

A partir del cuarto trimestre de 2023, Mirum Pharmaceuticals reportó ingresos totales del producto de $ 50.3 millones para Livmarli (Maralixibat), un medicamento para el prurito colestático asociado con el síndrome de Alagille.

Producto	Ingresos anuales (2023)	Segmento de mercado
Livmarli	$ 50.3 millones	Enfermedades hepáticas raras

Posibles acuerdos de licencia

Mirum tiene acuerdos de licencia estratégica con Takeda Pharmaceutical Company Limited para el desarrollo global y la comercialización de Volixibat.

Subvenciones y colaboraciones de investigación

• Financiación de la investigación de los Institutos Nacionales de Salud (NIH): aproximadamente $ 2.5 millones anuales
• Asociaciones de investigación colaborativa con centros médicos académicos

Ingresos de regalías del desarrollo de medicamentos

Posibles corrientes de regalías del acuerdo de licencia de Volixibat con Takeda, estimado en porcentajes escalonados que van del 10-20% de las ventas netas.

Ingresos de tratamiento terapéutico especializados

Área terapéutica	Ingresos anuales potenciales	Etapa de desarrollo
Enfermedades hepáticas pediátricas	$ 35-45 millones	Comercializado
Prurito colestático	$ 25-35 millones	Tratamiento aprobado

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.