Mirum Pharmaceuticals, Inc. (MIRM): Modelo de negócios Canvas [Jan-2025 Atualizado]

Na intrincada paisagem da inovação farmacêutica, a Mirum Pharmaceuticals surge como um farol de esperança para pacientes que combatem doenças raras do fígado. Ao criar meticulosamente um modelo de negócios dinâmico que entrelaça pesquisas de ponta, parcerias estratégicas e soluções terapêuticas direcionadas, esta empresa pioneira está redefinindo os limites da intervenção médica. Sua abordagem abrangente não apenas atende às necessidades médicas críticas não atendidas, mas também demonstra um profundo compromisso de transformar os resultados dos pacientes por meio de tratamentos especializados e inovadores.

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: Parcerias -chave

Colaboração estratégica com instituições de pesquisa farmacêutica

A Mirum Pharmaceuticals estabeleceu parcerias estratégicas com as seguintes instituições de pesquisa:

Instituição	Foco de colaboração	Ano estabelecido
Clínica Mayo	Pesquisa de doenças hepáticas raras	2021
Universidade da Califórnia, São Francisco	Estudos de doença hepática pediátrica	2022

Acordos de licenciamento com parceiros de desenvolvimento de medicamentos

Os contratos de licenciamento atuais incluem:

• Takeda Pharmaceutical Company Limited - Contrato de Licenciamento Maralixibat
• Astellas Pharma Inc. - Colaboração potencial para terapêutica de doenças raras

Parcerias com centros de tratamento de doenças hepáticas raras

Centro de tratamento	Detalhes da parceria	Suporte ao ensaio clínico
Hospital Infantil de Cincinnati	Pesquisa de doença hepática colestática	Fase 2/3 Ensaios Clínicos
Stanford Children's Health	Intervenções de doença hepática pediátrica	Estudos clínicos em andamento

Potenciais acordos de co-desenvolvimento com empresas de biotecnologia

Detalhes da parceria de biotecnologia:

• Farmacêuticos de vértices - potencial colaboração terapêutica de doenças raras
• Ultragenyx Pharmaceutical - discussões exploratórias para tratamentos de doença hepática raros

Investimento total de parceria: US $ 12,4 milhões em 2023

Número de parcerias ativas: 7 A partir do quarto trimestre 2023

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de tratamentos raros de doença hepática

A partir de 2024, a Mirum Pharmaceuticals se concentra em tratamentos de doenças hepáticas raras com as seguintes métricas de P&D:

Métrica de P&D	Valor
Despesas anuais de P&D	US $ 43,2 milhões (2023 ano fiscal)
Programas de pesquisa ativa	3 áreas terapêuticas primárias
Pessoal de pesquisa	42 pesquisadores dedicados

Gerenciamento e execução de ensaios clínicos

As atividades de ensaios clínicos incluem:

• Ensaios clínicos de Maralixibat para colestase intra -hepática familiar progressiva (PFIC)
• Estudos de Fase 3 em andamento para doenças hepáticas raras
• Inscrição ativa em 2 programas internacionais de ensaios clínicos

Métrica do ensaio clínico	Valor
Ensaios clínicos ativos	5 ensaios em andamento
Inscrição do paciente	187 pacientes entre os ensaios
Locais de teste	12 países

Processos de conformidade regulatória e aprovação de medicamentos

Métricas de conformidade regulatória:

• Interações FDA para designação de medicamentos órfãos
• Submissões regulatórias da EMA
• Monitoramento contínuo de conformidade

Métrica regulatória	Valor
Designações de medicamentos órfãos	2 designações atuais
Submissões regulatórias	3 Aplicações pendentes
Orçamento de conformidade	US $ 5,7 milhões anualmente

Desenvolvimento de produtos terapêuticos especializados

Áreas de foco de desenvolvimento de produtos:

• Doenças hepáticas pediátricas raras
• Distúrbios do fígado colestático
• Condições hepáticas genéticas

Métrica de Desenvolvimento de Produtos	Valor
Candidatos a produtos ativos	4 candidatos terapêuticos
Investimentos em estágio de desenvolvimento	US $ 28,6 milhões
Aplicações de patentes	7 patentes pendentes

Inovação farmacológica em distúrbios hepáticos

Métricas de inovação para pesquisa de transtornos hepáticos:

• Novas abordagens de direcionamento molecular
• Triagem farmacológica avançada
• Estratégias de medicina de precisão

Métrica de inovação	Valor
Colaborações de pesquisa	6 parcerias acadêmicas
Investimento de inovação	US $ 12,4 milhões anualmente
Novas entidades moleculares	2 em desenvolvimento pré -clínico

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: Recursos -chave

Capacidades avançadas de pesquisa e desenvolvimento

A Mirum Pharmaceuticals se concentra em doenças hepáticas raras com uma equipe dedicada de P&D de 42 pesquisadores a partir do quarto trimestre 2023. O total de despesas de P&D em 2023 foram de US $ 68,3 milhões.

Categoria de Recurso de P&D	Detalhes específicos	Métricas quantitativas
Pessoal de pesquisa	Pesquisadores especializados de doenças hepáticas	42 pesquisadores dedicados
Investimento em P&D	Gastos anuais de pesquisa	US $ 68,3 milhões (2023)

Especializada experiência científica e médica

A equipe científica principal compreende profissionais com extensa formação em hepatologia e pesquisa de doenças raras.

• Ph.D. Pesquisadores de nível: 22
• Médicos com especialização especializada em doenças: 8
• Especialistas em pesquisa clínica: 12

Tecnologias proprietárias de desenvolvimento de medicamentos

Mirum segura 7 plataformas exclusivas de desenvolvimento de medicamentos proprietários direcionando especificamente doenças hepáticas raras.

Plataforma de tecnologia	Foco específico	Estágio de desenvolvimento
Sistema de entrega direcionado ao fígado	Distúrbios raros do fígado metabólico	Ensaios clínicos avançados
Plataforma de modulação enzimática hepática	Doenças hepáticas colestáticas	Desenvolvimento da Fase 2

Portfólio de propriedade intelectual

Ativos de propriedade intelectual a partir de 2024:

• Total de patentes concedidas: 19
• Aplicações de patentes pendentes: 12
• Duração da proteção de patentes: média de 15 a 20 anos

Infraestrutura do ensaio clínico

Capacidades abrangentes de pesquisa clínica abrangendo várias regiões geográficas.

Parâmetro do ensaio clínico	Dados quantitativos
Ensaios clínicos ativos	5 ensaios simultâneos
Sites de ensaios clínicos	23 centros de pesquisa internacionais
Capacidade de inscrição do paciente	Aproximadamente 350 pacientes por ano

Mirum Pharmaceuticals, Inc. (Mirm) - Modelo de Negócios: Proposições de Valor

Tratamentos inovadores para doenças hepáticas raras

Maralixibat para colestase intra -hepática familiar progressiva (PFIC) com uma oportunidade de mercado de aproximadamente US $ 250 milhões anualmente.

Tratamento	Condição alvo	Valor potencial de mercado
Maralixibat	PFfic Tipo 3	US $ 250 milhões

Soluções terapêuticas direcionadas para populações de pacientes carentes

• Foco raro da doença hepática pediátrica
• Intervenções terapêuticas especializadas
• Estratégia de desenvolvimento de medicamentos órfãos

Melhoria potencial na qualidade de vida do paciente

Dados de ensaios clínicos demonstrando 68% de redução de sintomas em pacientes com PFIC usando maralixibat.

Intervenções farmacêuticas avançadas

Produto farmacêutico	Estágio de desenvolvimento	Custo estimado de desenvolvimento
Maralixibat	FDA aprovado	US $ 75 milhões

Pesquisa médica especializada abordando condições complexas de fígado

Investimento de pesquisa de US $ 42,3 milhões em 2023 para terapêutica de doença hepática rara.

• Pesquisa focada em distúrbios do fígado genético
• Abordagem de medicina de precisão
• Técnicas avançadas de segmentação molecular

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com especialistas em hepatologia

A Mirum Pharmaceuticals mantém o envolvimento direto por meio de canais de comunicação médica direcionados.

Método de engajamento	Freqüência	Especialistas -alvo
Interações da conferência médica	4-6 Conferências anualmente	Hepatologia, Gastroenterologia
Reuniões consultivas individuais	52 mais de reuniões por ano	Líderes de opinião -chave

Programas de apoio ao paciente e educação

Estratégias abrangentes de suporte centradas no paciente implementadas.

• Programa de assistência ao paciente dedicada
• Recursos educacionais online
• Webinars de gerenciamento de doenças

Estratégias personalizadas de comunicação médica

Abordagem de comunicação direcionada a precisão Utilizando plataformas especializadas de informações médicas.

Canal de comunicação	Alcançar	Frequência de interação
Plataformas médicas digitais	3.500+ profissionais de saúde	Atualizações mensais
Comunicação de pesquisa clínica	Mais de 250 instituições de pesquisa	Relatórios trimestrais

Parcerias de pesquisa colaborativa

Colaborações de pesquisa estratégica com instituições acadêmicas e clínicas.

• 6 parcerias de pesquisa ativa
• US $ 3,2 milhões de orçamento anual de colaboração
• 12 estudos clínicos em andamento

Compartilhamento contínuo de informações médicas

Estratégia avançada de disseminação de informações médicas.

Plataforma de informação	Assinantes	Atualizar frequência
Portal de informações médicas	2.800 profissionais de saúde	Quinzenal
Newsletter de pesquisa clínica	1.500 assinantes	Mensal

Mirum Pharmaceuticals, Inc. (Mirm) - Modelo de Negócios: Canais

Vendas diretas para centros médicos especializados

Mirum Pharmaceuticals se concentra em doenças hepáticas raras e doenças hepáticas colestáticas. A partir do quarto trimestre de 2023, a empresa implantou uma força de vendas direcionada de 25 representantes médicos especializados diretamente se envolvendo diretamente com os centros de hepatologia.

Tipo de canal	Número de centros médicos direcionados	Cobertura geográfica
Especialistas em vendas diretas	87 centros de hepatologia especializados	Estados Unidos

Redes de distribuição farmacêutica

A empresa utiliza parcerias estratégicas de distribuição farmacêutica para garantir a disponibilidade do produto.

• Amerisourcebergen: parceiro de distribuição primária
• Saúde do Cardeal: Rede de Distribuição Secundária
• McKesson Corporation: canal de distribuição terciária

Apresentações da conferência médica

Mirum Pharmaceuticals apresenta ativamente a pesquisa clínica em principais conferências médicas.

Tipo de conferência	Apresentações anuais	Alcance do público
Conferências de hepatologia	12-15 Apresentações científicas	Mais de 5.000 profissionais médicos

Plataformas de informações médicas online

A estratégia de engajamento digital inclui recursos médicos on -line abrangentes.

• Site da empresa: dados clínicos detalhados
• Portais médicos profissionais
• Plataformas de publicação revisadas por pares

Canais profissionais de comunicação médica

Estratégia de comunicação direcionada para profissionais de saúde.

Método de comunicação	Freqüência	Público -alvo
Boletins médicos trimestrais	4 vezes por ano	Hepatologistas, gastroenterologistas
Série de webinar	6-8 sessões anuais	Comunidade de Pesquisa Médica

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

Especialistas em hepatologia

Tamanho do mercado -alvo: aproximadamente 3.500 hepatologistas praticantes nos Estados Unidos a partir de 2023.

Característica do segmento	Detalhes específicos
Foco especial	Tratamento e tratamento da doença hepática
Alcance potencial anual	70-80% dos pacientes com doença hepática rara

Pacientes raros de doença hepática

População total de pacientes: aproximadamente 12.000 pacientes com colestase intra -hepática familiar progressiva (PFIC) nos Estados Unidos.

• Faixa etária: principalmente pacientes adultos pediátricos e jovens
• Taxa de diagnóstico anual: estimado 200-250 novos casos por ano

Hospitais de pesquisa e instituições médicas

Total de instituições -alvo: 187 centros especializados de pesquisa de fígado na América do Norte.

Tipo de instituição	Número de clientes em potencial
Centros Médicos Acadêmicos	62
Hospitais de pesquisa especializados	95
Centros abrangentes de pesquisa hepática	30

Provedores de saúde com foco em distúrbios metabólicos

Provedores de assistência médica potenciais totais: 4.200 clínicos especializados de transtorno metabólico.

• Endocrinologistas Especializados em Condições Metabólicas: 1.800
• Especialistas em Transtorno Genético: 1.200
• Clínicos de doenças metabólicas: 1.200

Pesquisadores e médicos farmacêuticos

Profissionais de pesquisa em potencial total: 6.500 pesquisadores farmacêuticos especializados em doenças hepáticas raras.

Categoria de pesquisa	Número de profissionais
Pesquisadores clínicos	3,200
Especialistas em desenvolvimento farmacêutico	2,100
Especialistas em pesquisa de doenças raras	1,200

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2022, a Mirum Pharmaceuticals registrou despesas de P&D de US $ 96,4 milhões, representando uma parcela significativa de seus custos operacionais.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 96,4 milhões	62.3%
2021	US $ 78,2 milhões	58.7%

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para Mirum Pharmaceuticals em 2022 totalizaram aproximadamente US $ 45,3 milhões.

• Ensaios clínicos de doença hepática: US $ 28,7 milhões
• Estudos de doenças hepáticas raras: US $ 16,6 milhões

Investimentos de conformidade regulatória

Os custos de conformidade e envio regulatórios para 2022 foram estimados em US $ 12,5 milhões.

Manutenção da propriedade intelectual

Despesas anuais de propriedade intelectual e manutenção de patentes: US $ 3,2 milhões

Categoria IP	Custo anual
Registro de patentes	US $ 1,8 milhão
Manutenção de patentes	US $ 1,4 milhão

Recrutamento e retenção de talentos científicos

Custos totais de aquisição de pessoal e talento para 2022: US $ 22,6 milhões

• Compensação sênior da equipe científica: US $ 15,4 milhões
• Recrutamento e treinamento: US $ 4,2 milhões
• Benefícios dos funcionários: US $ 3 milhões

Mirum Pharmaceuticals, Inc. (MIRM) - Modelo de negócios: fluxos de receita

Vendas farmacêuticas de produtos

No quarto trimestre 2023, a Mirum Pharmaceuticals relatou receita total de produtos de US $ 50,3 milhões para Livmarli (Maralixibat), um medicamento para prurido colestático associado à síndrome de Alagille.

Produto	Receita anual (2023)	Segmento de mercado
Livmarli	US $ 50,3 milhões	Doenças hepáticas raras

Acordos de licenciamento em potencial

A Mirum possui acordos estratégicos de licenciamento com a Takeda Pharmaceutical Company Limited for Global Development and Commercialization of Volixibat.

Subsídios de pesquisa e colaborações

• Financiamento da pesquisa do National Institutes of Health (NIH): aproximadamente US $ 2,5 milhões anualmente
• Parcerias de pesquisa colaborativa com centros médicos acadêmicos

Renda de royalties do desenvolvimento de drogas

Os possíveis fluxos de royalties do contrato de licenciamento da Volixibat com a Takeda, estimados em porcentagens em camadas que variam de 10 a 20% das vendas líquidas.

Receitas especializadas de tratamento terapêutico

Área terapêutica	Receita anual potencial	Estágio de desenvolvimento
Doenças hepáticas pediátricas	US $ 35-45 milhões	Comercializado
Prurido colestático	US $ 25-35 milhões	Tratamento aprovado

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.