Mirum Pharmaceuticals, Inc. (MIRM): Business Model Canvas [Jan-2025 Mis à jour]

Dans le paysage complexe de l'innovation pharmaceutique, Mirum Pharmaceuticals apparaît comme un phare d'espoir pour les patients luttant contre les maladies hépatiques rares. En fabriquant méticuleusement un modèle commercial dynamique qui mêle la recherche de pointe, les partenariats stratégiques et les solutions thérapeutiques ciblées, cette entreprise pionnière redéfinit les limites de l'intervention médicale. Leur approche complète répond non seulement aux besoins médicaux non satisfaits critiques, mais démontre également un engagement profond à transformer les résultats des patients grâce à des traitements spécialisés et innovants.

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: partenariats clés

Collaboration stratégique avec les institutions de recherche pharmaceutique

Mirum Pharmaceuticals a établi des partenariats stratégiques avec les institutions de recherche suivantes:

Institution	Focus de la collaboration	Année établie
Clinique de mayo	Recherche de maladies hépatiques rares	2021
Université de Californie, San Francisco	Études pédiatriques des maladies du foie	2022

Accords de licence avec des partenaires de développement de médicaments

Les accords de licence actuels comprennent:

• Takeda Pharmaceutical Company Limited - Contrat de licence Maralixibat
• Astellas Pharma Inc. - Collaboration potentielle pour les thérapies de maladies rares

Partenariats avec des centres de traitement des maladies hépatiques rares

Centre de traitement	Détails du partenariat	Soutien en essai clinique
Hôpital pour enfants de Cincinnati	Recherche cholestatique des maladies du foie	Phase 2/3 essais cliniques
Santé des enfants de Stanford	Interventions de maladie du foie pédiatrique	Études cliniques en cours

Accords de co-développement potentiels avec des entreprises de biotechnologie

Détails du partenariat en biotechnologie:

• Vertex Pharmaceuticals - Collaboration thérapeutique potentielle des maladies rares
• Ultragenyx Pharmaceutical - Discussions exploratoires pour les traitements de maladie du foie rares

Investissement total de partenariat: 12,4 millions de dollars en 2023

Nombre de partenariats actifs: 7 au Q4 2023

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: Activités clés

Recherche et développement de traitements de maladies hépatiques rares

En 2024, Mirum Pharmaceuticals se concentre sur les traitements de maladie du foie rares avec les mesures de R&D suivantes:

Métrique de R&D	Valeur
Dépenses annuelles de R&D	43,2 millions de dollars (2023 Exercice)
Programmes de recherche actifs	3 zones thérapeutiques primaires
Personnel de recherche	42 chercheurs dédiés

Gestion et exécution des essais cliniques

Les activités des essais cliniques comprennent:

• Essais cliniques Maralixibat pour la cholestase intrahépatique familiale progressive (PFIC)
• Études en cours de phase 3 pour les maladies hépatiques rares
• Inscription active dans 2 programmes d'essais cliniques internationaux

Métrique d'essai clinique	Valeur
Essais cliniques actifs	5 essais en cours
Inscription des patients	187 patients à travers les essais
Lieux d'essai	12 pays

Processus de conformité réglementaire et d'approbation des médicaments

Métriques de la conformité réglementaire:

• Interactions de la FDA pour la désignation de médicaments orphelins
• Soumissions réglementaires EMA
• Surveillance continue de la conformité

Métrique réglementaire	Valeur
Désignations de médicaments orphelins	2 désignations actuelles
Soumissions réglementaires	3 applications en attente
Budget de conformité	5,7 millions de dollars par an

Développement de produits thérapeutiques spécialisés

Domaines d'intervention du développement des produits:

• Maladies hépatiques pédiatriques rares
• Troubles du foie cholestatiques
• Conditions hépatiques génétiques

Métrique de développement de produits	Valeur
Produits actifs candidats	4 candidats thérapeutiques
Investissements en phase de développement	28,6 millions de dollars
Demandes de brevet	7 brevets en instance

Innovation pharmacologique dans les troubles hépatiques

Métriques d'innovation pour la recherche sur les troubles hépatiques:

• Nouvelles approches de ciblage moléculaire
• Dépistage pharmacologique avancé
• Stratégies de médecine de précision

Métrique d'innovation	Valeur
Collaborations de recherche	6 partenariats académiques
Investissement en innovation	12,4 millions de dollars par an
Nouvelles entités moléculaires	2 en développement préclinique

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: Ressources clés

Capacités avancées de recherche et de développement

Mirum Pharmaceuticals se concentre sur les maladies hépatiques rares avec une équipe de R&D dédiée de 42 chercheurs au T2 2023. Les dépenses totales de R&D pour 2023 étaient de 68,3 millions de dollars.

Catégorie de ressources R&D	Détails spécifiques	Métriques quantitatives
Personnel de recherche	Chercheurs spécialisés sur les maladies du foie	42 chercheurs dédiés
Investissement en R&D	Dépenses de recherche annuelles	68,3 millions de dollars (2023)

Expertise scientifique et médicale spécialisée

L'équipe scientifique de base comprend des professionnels ayant une formation approfondie en hépatologie et en recherche de maladies rares.

• doctorat chercheurs de niveau: 22
• Médecins ayant une expertise spécialisée sur les maladies du foie: 8
• Spécialistes de la recherche clinique: 12

Technologies de développement de médicaments propriétaires

Mirum tient 7 plateformes de développement de médicaments propriétaires uniques ciblant spécifiquement les maladies hépatiques rares.

Plate-forme technologique	Focus spécifique	Étape de développement
Système de livraison ciblé par le foie	Troubles hépatiques métaboliques rares	Essais cliniques avancés
Plate-forme de modulation enzymatique hépatique	Maladies hépatiques cholestatiques	Développement de phase 2

Portefeuille de propriété intellectuelle

Actifs de la propriété intellectuelle à partir de 2024:

• Total des brevets accordés: 19
• Demandes de brevet en instance: 12
• Protection des brevets Durée: moyenne de 15 à 20 ans

Infrastructure d'essais cliniques

Capacités de recherche clinique complètes couvrant plusieurs régions géographiques.

Paramètre d'essai clinique	Données quantitatives
Essais cliniques actifs	5 essais simultanés
Sites d'essais cliniques	23 centres de recherche internationaux
Capacité d'inscription des patients	Environ 350 patients par an

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: propositions de valeur

Traitements innovants pour les maladies hépatiques rares

Maralixibat pour la cholestase intrahépatique familiale progressive (PFIC) avec une opportunité de marché d'environ 250 millions de dollars par an.

Traitement	Condition cible	Valeur marchande potentielle
Maralixibat	PFIC TYPE 3	250 millions de dollars

Solutions thérapeutiques ciblées pour les populations de patients mal desservis

• Rare Pédiatrique Fixation du foie Focus
• Interventions thérapeutiques spécialisées
• Stratégie de développement de médicaments orphelins

Amélioration potentielle de la qualité de vie des patients

Données d'essai cliniques démontrant une réduction des symptômes de 68% chez les patients PFIC à l'aide de maralixibat.

Interventions pharmaceutiques avancées

Produit pharmaceutique	Étape de développement	Coût de développement estimé
Maralixibat	Approuvé par la FDA	75 millions de dollars

Recherche médicale spécialisée concernant les conditions hépatiques complexes

Investissement en recherche de 42,3 millions de dollars en 2023 pour les thérapies rares des maladies hépatiques.

• Recherche ciblée sur les troubles du foie génétiques
• Approche de la médecine de précision
• Techniques de ciblage moléculaire avancées

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: relations avec les clients

Engagement direct avec des spécialistes de l'hépatologie

Mirum Pharmaceuticals maintient un engagement direct grâce à des canaux de communication médicale ciblés.

Méthode d'engagement	Fréquence	Spécialistes de la cible
Interactions de la conférence médicale	4-6 conférences par an	Hépatologie, gastro-entérologie
Réunions consultatives individuelles	Plus de 52 réunions par an	Leaders d'opinion clés

Programmes de soutien aux patients et d'éducation

Des stratégies de soutien complets centrées sur le patient mis en œuvre.

• Programme d'aide aux patients dévoués
• Ressources éducatives en ligne
• Webinaires de gestion des maladies

Stratégies de communication médicale personnalisées

Approche de communication ciblée avec précision en utilisant des plateformes d'information médicale spécialisées.

Canal de communication	Atteindre	Fréquence d'interaction
Plates-formes médicales numériques	3 500+ professionnels de la santé	Mises à jour mensuelles
Communication de recherche clinique	Plus de 250 institutions de recherche	Rapports trimestriels

Partenariats de recherche collaborative

Collaborations de recherche stratégique avec des établissements universitaires et cliniques.

• 6 partenariats de recherche actifs
• Budget de collaboration de recherche annuelle de 3,2 millions de dollars
• 12 études cliniques en cours

Partage d'informations médicales continues

Stratégie avancée de diffusion d'informations médicales.

Plate-forme d'information	Abonnés	Mettre à jour la fréquence
Portail d'informations médicales	2 800 professionnels de la santé	Bihebdomadaire
Newsletter de recherche clinique	1 500 abonnés	Mensuel

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: canaux

Ventes directes vers des centres médicaux spécialisés

Mirum Pharmaceuticals se concentre sur les maladies hépatiques rares et les maladies hépatiques cholestatiques. Au quatrième trimestre 2023, la société a déployé une force de vente ciblée de 25 représentants médicaux spécialisés se livrant directement à des centres d'hépatologie.

Type de canal	Nombre de centres médicaux ciblés	Couverture géographique
Spécialistes des ventes directes	87 centres d'hépatologie spécialisés	États-Unis

Réseaux de distribution pharmaceutique

L'entreprise utilise des partenariats stratégiques de distribution pharmaceutique pour garantir la disponibilité des produits.

• Amerisourcebergen: partenaire de distribution primaire
• Cardinal Health: Réseau de distribution secondaire
• McKesson Corporation: canal de distribution tertiaire

Présentations de la conférence médicale

Mirum Pharmaceuticals présente activement la recherche clinique lors de conférences médicales clés.

Type de conférence	Présentations annuelles	Poutenir
Conférences d'hépatologie	12-15 présentations scientifiques	Plus de 5 000 professionnels de la santé

Plateformes d'information médicale en ligne

La stratégie d'engagement numérique comprend des ressources médicales en ligne complètes.

• Site Web de l'entreprise: données cliniques détaillées
• Portails médicaux professionnels
• Plateformes de publication évaluées par des pairs

Canaux de communication médicale professionnelle

Stratégie de communication ciblée pour les professionnels de la santé.

Méthode de communication	Fréquence	Public cible
Newsletters médicaux trimestriels	4 fois par an	Hépatologues, gastro-entérologues
Webinaire Series	6-8 sessions annuelles	Communauté de recherche médicale

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: segments de clientèle

Spécialistes de l'hépatologie

Taille du marché cible: environ 3 500 hépatologues pratiquants aux États-Unis à partir de 2023.

Caractéristique du segment	Détails spécifiques
Focus spécialisée	Traitement et gestion des maladies du foie
Portée potentielle annuelle	70 à 80% des patients atteints d'une maladie du foie rares

Patients atteints d'une maladie du foie

Population totale de patients: environ 12 000 patients atteints de cholestase intrahépatique familiale progressive (PFIC) aux États-Unis.

• Tranche d'âge: principalement des patients pédiatriques et jeunes adultes
• Taux de diagnostic annuel: 200-250 nouveaux cas par an

Hôpitaux de recherche et institutions médicales

Institutions cibles totales: 187 centres de recherche hépatique spécialisés en Amérique du Nord.

Type d'institution	Nombre de clients potentiels
Centres médicaux académiques	62
Hôpitaux de recherche spécialisés	95
Centres de recherche hépatique complets	30

Les prestataires de soins de santé se concentrent sur les troubles métaboliques

Total des prestataires de soins de santé potentiels: 4 200 cliniciens spécialisés des troubles métaboliques.

• Endocrinologues spécialisés dans les conditions métaboliques: 1 800
• Spécialistes des troubles génétiques: 1 200
• Cliniciens de la maladie métabolique: 1 200

Chercheurs pharmaceutiques et cliniciens

Professionnels de recherche potentiels totaux: 6 500 chercheurs pharmaceutiques spécialisés dans les maladies hépatiques rares.

Catégorie de recherche	Nombre de professionnels
Chercheurs en clinique	3,200
Spécialistes du développement pharmaceutique	2,100
Experts en recherche de maladies rares	1,200

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2022, Mirum Pharmaceuticals a déclaré des dépenses de R&D de 96,4 millions de dollars, ce qui représente une partie importante de leurs coûts opérationnels.

Année	Dépenses de R&D	Pourcentage des dépenses totales
2022	96,4 millions de dollars	62.3%
2021	78,2 millions de dollars	58.7%

Coûts de gestion des essais cliniques

Les dépenses d'essais cliniques pour Mirum Pharmaceuticals en 2022 ont totalisé environ 45,3 millions de dollars.

• Essais cliniques de la maladie hépatique: 28,7 millions de dollars
• Études rares des maladies du foie: 16,6 millions de dollars

Investissements de conformité réglementaire

Les frais de conformité et de soumission réglementaires pour 2022 ont été estimés à 12,5 millions de dollars.

Maintenance de la propriété intellectuelle

Dépenses annuelles sur la propriété intellectuelle et l'entretien des brevets: 3,2 millions de dollars

Catégorie IP	Coût annuel
Dépôt de brevet	1,8 million de dollars
Entretien de brevets	1,4 million de dollars

Recrutement et rétention des talents scientifiques

Total des frais d'acquisition du personnel et des talents pour 2022: 22,6 millions de dollars

• Rémunération du personnel scientifique senior: 15,4 millions de dollars
• Recrutement et formation: 4,2 millions de dollars
• Avantages sociaux: 3 millions de dollars

Mirum Pharmaceuticals, Inc. (MIRM) - Modèle d'entreprise: Strots de revenus

Ventes de produits pharmaceutiques

Depuis le quatrième trimestre 2023, Mirum Pharmaceuticals a déclaré un chiffre d'affaires total des produits de 50,3 millions de dollars pour Livmarli (Maralixibat), un médicament pour le prurit cholestatique associé au syndrome d'Alagille.

Produit	Revenus annuels (2023)	Segment de marché
Livmarli	50,3 millions de dollars	Maladies hépatiques rares

Accords de licence potentiels

Mirum a des accords de licence stratégique avec Takeda Pharmaceutical Company Limited pour le développement mondial et la commercialisation de Volixibat.

Grants de recherches et collaborations

• Financement de la recherche des National Institutes of Health (NIH): environ 2,5 millions de dollars par an
• Partenariats de recherche collaborative avec des centres médicaux universitaires

Revenus redevances du développement de la drogue

Les flux de redevances potentiels de la convention de licence Volixibat avec Takeda, estimée à des pourcentages à plusieurs niveaux allant de 10 à 20% des ventes nettes.

Revenus de traitement thérapeutique spécialisés

Zone thérapeutique	Revenus annuels potentiels	Étape de développement
Maladies hépatiques pédiatriques	35 à 45 millions de dollars	Commercialisé
Prurit cholestatique	25 à 35 millions de dollars	Traitement approuvé

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.