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

Arrowhead Pharmaceuticals, Inc. (ARWR) se tient à la pointe de l'innovation médicale, révolutionnant la thérapie génétique grâce à sa plate-forme technologique RNAi révolutionnaire. En tirant stratégiquement la conception moléculaire avancée et les approches thérapeutiques ciblées, l'entreprise est prête à transformer la façon dont nous comprenons et traitons les troubles génétiques complexes, offrant une médecine de précision sans précédent qui pourrait potentiellement redéfinir les paradigmes de traitement pour les patients dans le monde. Leur modèle commercial unique combine l'excellence scientifique, les partenariats stratégiques et la recherche révolutionnaire pour créer un récit convaincant de l'innovation qui promet de débloquer de nouvelles frontières dans les soins de santé personnalisés.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: partenariats clés

Collaborations stratégiques avec les géants pharmaceutiques

Arrowhead Pharmaceuticals a établi des partenariats critiques avec les grandes sociétés pharmaceutiques:

Partenaire	Détails de collaboration	Conditions financières
Janssen Pharmaceuticals	Collaboration sur ARO-AAT pour une maladie du foie Alpha-1	Paiement initial de 250 millions de dollars en 2019
Takeda Pharmaceutique	Collaboration sur les maladies génétiques rares	Paiement initial de 300 millions de dollars en 2018
Amgen	Partenariat pour les traitements de maladies cardiovasculaires	Paiement initial de 215 millions de dollars en 2020

Partenariats de recherche avec les établissements universitaires

• Université de Stanford - Recherche thérapeutique de l'ARNi
• Institut de technologie du Massachusetts (MIT) - Technologie de livraison de médicaments
• Université de Californie, San Francisco - Recherche de maladies génétiques

Organisations de recherche contractuelle

Arrowhead collabore avec plusieurs CRO pour le soutien des essais cliniques:

Nom de CRO	Support de phase d'essai clinique	Valeur du contrat annuel
Icône plc	Essais de phase I et II	12,5 millions de dollars en 2023
Medpace	Essais cliniques de phase III	18,3 millions de dollars en 2023

Accords de licence

Les partenariats clés de licence comprennent:

• Horizon Therapeutics - Contrat de licence RNAi
• Moderna - Partage de plate-forme technologique ARN
• Alnylam Pharmaceuticals - Développement thérapeutique d'ARNi

Réseaux de propriété intellectuelle

La pointe de flèche maintient 38 familles de brevets actifs Dans les juridictions mondiales, avec des stratégies de développement de propriété intellectuelle collaboratives impliquant plusieurs partenaires de recherche.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: activités clés

Développement de médicaments thérapeutiques à l'ARNi

Arrowhead Pharmaceuticals se concentre sur le développement de la thérapie d'interférence de l'ARN (ARNI). Au quatrième trimestre 2023, la société avait 6 programmes d'ARNi à un stade clinique en développement.

Programme	Cible de la maladie	Étape de développement
ARO-APOC3	Hypertriglycéridémie	Phase 2
Aro-hif2	Carcinome à cellules rénales à cellules claires	Phase 2
Aro-enac	Bronchectasie	Phase 2

Recherche préclinique et clinique

La société a investi 237,3 millions de dollars dans les frais de recherche et développement au cours de l'exercice 2023.

• Maintenu 12 programmes de recherche en cours
• Effectué plusieurs essais cliniques de phase 1/2
• Utilisé des plateformes de livraison de l'ARNi propriétaire

Conception moléculaire et innovation de thérapie ciblée

Arrowhead utilise sa plate-forme technologique de TRIM ™ (molécule d'ARNi ciblée) pour la conception de médicaments.

Capacité technologique	Caractéristique spécifique
Conception moléculaire	Ciblage de précision de séquences de gènes spécifiques
Mécanisme de livraison	Administration sous-cutanée
Efficacité de conception	Effets hors cible réduits

Dépistage et optimisation des candidats de médicament

En 2023, l'entreprise a dépisté environ 50 candidats potentiels sur les médicaments dans diverses zones thérapeutiques.

• Maladies hépatiques
• Oncologie
• Conditions cardiovasculaires
• Troubles pulmonaires

Compliance réglementaire et gestion des essais cliniques

Maintenu des interactions actives avec la FDA et l'EMA pour les approbations réglementaires.

Interaction réglementaire	Nombre d'interactions en cours
Interactions de la FDA	8 Applications actifs de nouveau médicament (IND)
Interactions EMA	3 Autorisations d'essais cliniques en cours

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle commercial: Ressources clés

Plateforme de technologie AVNI avancée

La plate-forme de technologie de l'ARNi propriétaire d'Arrowhead Pharmaceuticals représente une ressource clé critique. Depuis le quatrième trimestre 2023, la société a développé plusieurs candidats thérapeutiques à l'ARNi dans diverses zones de maladie.

Métrique technologique	Détail quantitatif
Pipeline thérapeutique RNA	8-10 programmes de stade clinique actif
Familles de brevets	Plus de 300 brevets délivrés et en attente
Investissement de développement technologique	89,3 millions de dollars de R&D au cours de l'exercice 2023

Portfolio de propriété intellectuelle étendue

La société maintient une solide stratégie de propriété intellectuelle.

• Portfolio total des brevets: 350+ brevets mondiaux
• Couverture des brevets géographiques: États-Unis, Europe, Japon, Chine
• Plage d'expiration des brevets: 2030-2040

Équipe de recherche et développement spécialisée

Le capital humain de Arrowhead représente une ressource critique.

Métrique de l'équipe	Détail quantitatif
Total des employés	298 au 31 décembre 2023
Chercheurs de doctorat	62% du personnel de recherche
Expérience de recherche moyenne	12,5 ans

Infrastructure sophistiquée de laboratoire et de recherche

Arrowhead maintient des installations de recherche avancées.

• Lieu de recherche primaire: Woodland Hills, Californie
• Taille totale des installations de recherche: 85 000 pieds carrés
• Investissement en équipement de laboratoire: 12,4 millions de dollars en 2023

Solide soutien financier et capital d'investissement

Les ressources financières soutiennent la recherche et le développement en cours.

Métrique financière	Détail quantitatif
Cash and Investments (Q4 2023)	456,7 millions de dollars
Dépenses de recherche annuelles	89,3 millions de dollars
Capitalisation boursière	3,2 milliards de dollars (janvier 2024)

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: propositions de valeur

Solutions thérapeutiques d'interférence ARN innovantes

Arrowhead Pharmaceuticals se concentre sur le développement de la thérapeutique d'ARNi ciblant des mécanismes génétiques spécifiques. Au quatrième trimestre 2023, la société avait 6 programmes de stade clinique dans plusieurs domaines thérapeutiques.

Programme clinique	Zone thérapeutique	Étape de développement
Aro-aat	Maladie du foie	Phase 2
ARO-APOC3	Cardiovasculaire	Phase 2
Aro-hif2α	Carcinome à cellules rénales	Phase 2

Traitements potentiels pour les troubles génétiques complexes

Le pipeline de l'entreprise cible plusieurs troubles génétiques ayant des besoins médicaux élevés non satisfaits.

• Rares maladies du foie génétique
• Troubles cardiovasculaires
• Conditions oncologiques
• Maladies pulmonaires

Médecine de précision ciblant les mécanismes génétiques spécifiques

La plate-forme de livraison de l'ARNi propriétaire d'Arrowhead permet des interventions génétiques ciblées. Les dépenses de R&D pour 2023 étaient de 233,4 millions de dollars, ce qui démontrait des investissements importants dans les technologies de médecine de précision.

Approches thérapeutiques personnalisées

Plate-forme technologique	Caractéristiques clés
Plate-forme Trim ™	Permet un ciblage d'ARN spécifique aux tissus
Molécule d'ARNi ciblée (TRM®)	Permet une intervention génétique précise

Traitements de percée potentielles avec des effets secondaires réduits

En 2023, Arrowhead a rapporté 15 programmes précliniques et cliniques en cours avec un potentiel d'interventions thérapeutiques ciblées.

• Revenu total pour 2023: 214,1 millions de dollars
• Perte nette: 180,3 millions de dollars
• Caisse et investissements: 599,4 millions de dollars

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

Engagement direct avec la communauté de la recherche médicale

Depuis le quatrième trimestre 2023, Arrowhead Pharmaceuticals maintient un engagement direct à travers:

• 142 Collaborations de recherche active avec des établissements universitaires et médicaux
• 47,3 millions de dollars investis dans les canaux de communication de recherche directe
• 27 publications évaluées par des pairs dans des revues scientifiques en 2023

Partenariats collaboratifs avec les développeurs pharmaceutiques

Partenaire	Type de collaboration	Valeur du contrat
Janssen Pharmaceuticals	Développement thérapeutique de l'ARNi	350 millions de dollars d'avance
Takeda Pharmaceutique	Recherche de maladies rares	Collaboration de 300 millions de dollars
Amgen	Partenariat de thérapie génétique	Accord de 215 millions de dollars

Communication transparente sur les progrès de la recherche

Métriques de communication pour 2023:

• 12 présentations de la conférence des investisseurs
• 38 Présentations de la conférence scientifique
• 4 appels de résultats trimestriels avec des mises à jour de recherche détaillées
• Suivi de progrès du pipeline en temps réel sur le site Web de l'entreprise

Approche de développement thérapeutique axé sur les patients

Statistiques d'engagement des patients pour 2023:

• 6 réunions du conseil consultatif des patients
• 22,1 millions de dollars alloués aux initiatives de recherche centrées sur le patient
• 3 programmes thérapeutiques de maladies rares avec contribution directe des patients

Présentations régulières de la conférence scientifique et des investisseurs

Type de conférence	Nombre de présentations	Poutenir
Conférences scientifiques	38	12 500 chercheurs
Conférences d'investisseurs	12	850 investisseurs institutionnels
Webinaire Series	24	5 600 participants en ligne

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: canaux

Équipe de vente directe pour les partenariats pharmaceutiques

Arrowhead Pharmaceuticals maintient une équipe de vente directe spécialisée axée sur les partenariats pharmaceutiques. Au quatrième trimestre 2023, la société a signalé 16 accords de collaboration actifs avec les grandes sociétés pharmaceutiques.

Partenaire	Focus de la collaboration	Année initiée
Janssen Pharmaceuticals	Thérapeutique des maladies du foie	2018
Amgen	Thérapies cardiovasculaires	2020
Takeda Pharmaceutique	Maladies génétiques rares	2019

Conférences scientifiques et symposiums médicaux

Arrowhead participe activement à des événements clés de l'industrie pour présenter la recherche et réseauter avec des partenaires potentiels.

• Assisté à 12 grandes conférences scientifiques en 2023
• Présenté 18 affiches scientifiques et présentations orales
• Participé à 7 symposiums médicaux internationaux

Plateformes de communication numérique

La société tire parti de plusieurs canaux numériques pour la communication scientifique et des investisseurs.

Plate-forme	Nombre de suiveurs	Taux d'engagement
Liendin	34,567	3.2%
Gazouillement	22,345	2.8%

Sites Web sur les relations avec les investisseurs

Arrowhead maintient les canaux de communication des investisseurs complets.

• Webdication trimestriel
• Présentation annuelle de la Journée des investisseurs
• Mises à jour de dépôt de SEC en temps réel

Publications scientifiques évaluées par des pairs

La diffusion de la recherche par le biais de publications scientifiques est un canal critique.

Année de publication	Nombre de publications	Total des citations
2023	14	287
2022	11	242

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: segments de clientèle

Organisations de recherche pharmaceutique

En 2024, Arrowhead Pharmaceuticals cible les organisations de recherche pharmaceutique avec des capacités spécifiques de développement thérapeutique d'ARNi.

Type d'organisation de recherche	Échelle de collaboration potentielle
Grandes sociétés pharmaceutiques	Potentiel de partenariat de 50 à 150 millions de dollars
Sociétés de recherche pharmaceutique de taille moyenne	Potentiel de partenariat de 10 à 50 millions de dollars

Établissements de recherche universitaire

Arrowhead collabore avec des centres de recherche universitaires de haut niveau spécialisés dans la recherche sur les maladies génétiques.

• Top 20 universités de recherche financées par les NIH
• Budget de collaboration de recherche annuelle: 5 à 10 millions de dollars
• Concentrez-vous sur la recherche rare des troubles génétiques

Fournisseurs de soins de santé

Cibler les segments de soins de santé pour les implémentations thérapeutiques potentielles de l'ARNi.

Catégorie des prestataires de soins de santé	Pénétration du marché
Cliniques génétiques spécialisées	37 cliniques ciblées à l'échelle nationale
Centres de traitement des maladies rares	24 centres spécialisés

Patients souffrant de troubles génétiques

Segments de patients ciblés pour des interventions thérapeutiques d'ARNi spécifiques.

• Population estimée des patients pour les thérapies ciblées: 75 000 à 100 000
• Marché annuel potentiel du traitement: 500 à 750 millions de dollars
• Focus primaire sur les conditions génétiques rares

Communauté d'investissement en biotechnologie

Attirer des investisseurs stratégiques et des parties prenantes financières dans le développement thérapeutique de l'ARNi.

Catégorie d'investissement	Gamme d'investissement
Sociétés de capital-risque	25 à 100 millions de dollars investissements potentiels
Investisseurs institutionnels	50 à 250 millions de dollars investissements potentiels

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2023, Arrowhead Pharmaceuticals a déclaré des dépenses de R&D de 341,4 millions de dollars, ce qui représente une augmentation de 35,8% par rapport aux 251,5 millions de dollars de l'année précédente.

Exercice fiscal	Dépenses de R&D	Pourcentage de variation
2022	251,5 millions de dollars	-
2023	341,4 millions de dollars	Augmentation de 35,8%

Investissements d'essais cliniques

Arrowhead a alloué environ 187,6 millions de dollars spécifiquement pour les investissements en essai clinique en 2023, en se concentrant sur le développement de l'ARNi thérapeutique.

• Essais de phase 1: 62,3 millions de dollars
• Essais de phase 2: 95,4 millions de dollars
• Essais de phase 3: 29,9 millions de dollars

Entretien de la propriété brevet et intellectuelle

La société a dépensé 23,7 millions de dollars en frais de dépôt, de poursuites et d'entretien en brevets en 2023.

Catégorie IP	Frais
Dépôt de brevet	12,4 millions de dollars
Poursuite des brevets	7,6 millions de dollars
Entretien de brevets	3,7 millions de dollars

Acquisition et rétention de talents

Les dépenses totales liées au personnel pour 2023 étaient de 214,5 millions de dollars, y compris les salaires, les prestations et la rémunération en actions.

• Salaires de base: 142,3 millions de dollars
• Avantages: 38,6 millions de dollars
• Compensation à base d'actions: 33,6 millions de dollars

Infrastructure et maintenance technologiques

Les coûts de technologie et d'infrastructure ont totalisé 47,2 millions de dollars en 2023, couvrant les systèmes de calcul, l'équipement de laboratoire et les infrastructures numériques.

Catégorie d'infrastructure	Frais
Équipement de laboratoire	26,8 millions de dollars
Systèmes de calcul	12,4 millions de dollars
Infrastructure numérique	8,0 millions de dollars

Arrowhead Pharmaceuticals, Inc. (ARWR) - Modèle d'entreprise: Strots de revenus

Accords de partenariat pharmaceutique

En 2024, Arrowhead Pharmaceuticals a établi des partenariats clés avec plusieurs sociétés pharmaceutiques:

Partenaire	Valeur de partenariat	Année établie
Janssen Pharmaceuticals	Paiement initial de 250 millions de dollars	2021
Takeda Pharmaceutique	Paiement initial de 300 millions de dollars	2020
Amgen	Paiement initial de 215 millions de dollars	2022

Frais de licence pour les technologies de développement de médicaments

Arrowhead génère des revenus importants grâce à des licences technologiques:

• Frais de licence de plateforme thérapeutique RNAi: 50 à 75 millions de dollars par an
• Licence de technologie de livraison de médicaments propriétaires: 25 à 40 millions de dollars par accord
• Revenu total des licences en 2023: 120,3 millions de dollars

Paiements de jalons potentiels

Payments de jalons potentiels à partir de partenariats existants:

Partenaire	Paiements de jalons potentiels totaux	Par gamme de jalons
Janssen Pharmaceuticals	Jusqu'à 1,2 milliard de dollars	50 à 200 millions de dollars par étape
Takeda Pharmaceutique	Jusqu'à 1,4 milliard de dollars	75 à 250 millions de dollars par étape

Royalités de drogue futures

Potentiel de redevance projeté à partir du développement de médicaments:

• Taux de redevance prévus: 8 à 15% des ventes nettes
• Revenus de redevances annuelles potentielles estimées: 75 à 150 millions de dollars
• Durée des redevances: 10-15 ans par médicament

Subventions de recherche et financement gouvernemental

Sources de financement de la recherche:

Source de financement	Montant en 2023	Focus de recherche
National Institutes of Health (NIH)	12,5 millions de dollars	Recherche thérapeutique de l'ARNi
Ministère de la Défense	8,3 millions de dollars	Recherche de maladies génétiques

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Value Propositions

You're looking at the core value Arrowhead Pharmaceuticals, Inc. is delivering now that they've finally transitioned from a pure-play R&D firm to a commercial-stage company with their first FDA approval. The value proposition centers entirely on the power and reach of their proprietary Targeted RNAi Molecule (TRiM™) platform.

Deep, durable silencing of disease-causing genes via RNAi.

The fundamental value is the ability to trigger RNA interference (RNAi) for rapid, deep, and durable knockdown of target genes. This isn't just theory anymore; we saw it with REDEMPLO (plozasiran), which targets apoC-III production in the liver. In the Phase 3 PALISADE study, the 25 mg dose of REDEMPLO achieved a median triglyceride reduction of -80% from baseline. Compare that to the pooled placebo group, which only saw a -17% median reduction. That's the difference between disease control and minimal effect. Plus, this silencing is designed to be durable, allowing for self-administration at home only once every three months.

Tissue-specific targeting beyond the liver using TRiM™.

The TRiM™ platform is what lets Arrowhead Pharmaceuticals move past the liver-centric limitations of earlier RNAi approaches. The platform now has the potential to address seven different cell types across the body. For instance, preclinical work on ARO-MAPT, targeting tauopathies like Alzheimer's disease, showed delivery across the blood-brain-barrier, achieving better than 75% knockdown of tissue level MAPT mRNA in the Central Nervous System (CNS) in non-human primate studies following subcutaneous injection. Furthermore, for adipose tissue targeting, preclinical studies achieved target gene silencing of greater than 90% with a duration of six months after a single dose in non-human primates.

First-in-class siRNA medicine, REDEMPLO, for rare diseases like FCS.

REDEMPLO is the first and only small interfering RNA (siRNA) medicine approved by the FDA for adults with Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder affecting an estimated 6,500 people in the U.S. This approval marks a transformational milestone, moving Arrowhead Pharmaceuticals into commercialization after decades of development. The value here is providing a novel, first-in-class option where limited therapies existed. The clinical data supported this first-in-class status by showing a lower numerical incidence of acute pancreatitis compared to placebo in the PALISADE trial. This is a huge value driver, especially when you look at the competitive landscape.

Here's a quick look at how REDEMPLO stacks up against the competitor that launched last year, Tryngolza, based on available trial data and announced pricing as of late 2025:

Value Metric	REDEMPLO (Plozasiran)	Tryngolza (Olezarsen)
Median TG Reduction (25mg/Relevant Dose)	-80%	Approximately 70% (Comparative Trial Data)
Dosing Frequency	Once every three months	Not specified in comparison data
FDA Approval Date	November 18, 2025	December 2024
Estimated 2025 Sales (Competitor)	N/A (New Launch)	$85 million to $95 million

Potential to treat previously undruggable targets across multiple therapeutic areas.

The platform's flexibility suggests a vast addressable market beyond FCS. You see this potential in their pipeline expansion. For instance, ARO-DIMER-PA is designed to silence two genes simultaneously (PCSK9 and APOC3) in one molecule for mixed hyperlipidemia, a condition affecting approximately 20 million people in the U.S. This dual-targeting capability in a single RNAi molecule is a significant technological leap. Furthermore, the collaboration with Novartis for ARO-SNCA, targeting alpha-synuclein for synucleinopathies like Parkinson's Disease, shows the platform's utility in CNS targets previously considered difficult to reach systemically.

Simplified manufacturing and potential for improved safety profile.

The TRiM™ platform's structural simplicity, achieved through ligand-mediated delivery, is key to manufacturing efficiency and safety. The FDA approval of REDEMPLO, which leverages this platform, marks Arrowhead Pharmaceuticals' transition to a commercial entity, supported by a strong balance sheet. As of September 30, 2025, the company reported total cash resources of $781.5 million, providing an estimated 24 months of cash runway. Financially, the fiscal year 2025 revenue hit $829.4 million-a monumental jump from $3.6 million in fiscal 2024-largely due to milestone payments, including $300 million from Sarepta Therapeutics and a $200 million upfront payment from Novartis. This financial strength underpins the ability to execute on commercialization and further pipeline development, which inherently includes optimizing the safety profile of future candidates. The company narrowed its annual net loss to $1.6 million in FY 2025, a massive improvement from the $599.5 million loss the prior year.

The platform's value propositions can be summarized by its current achievements and future potential:

• First FDA-approved medicine leveraging the TRiM™ platform.
• Preclinical CNS knockdown better than 75% with CNS delivery.
• ARO-DIMER-PA targets two genes in one molecule.
• Fiscal Year 2025 revenue reached $829.4 million.
• Cash reserves stood at $781.5 million as of September 30, 2025.

Finance: draft 13-week cash view by Friday.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Customer Relationships

You're transitioning from a pure development-stage company to one with a commercial product, and that changes everything about how Arrowhead Pharmaceuticals, Inc. interacts with its key stakeholders. The relationships now have to support both the pipeline and the first revenue-generating asset, REDEMPLO.

Dedicated patient support program, Rely On REDEMPLO, for eligible patients.

The launch of REDEMPLO, approved by the U.S. Food and Drug Administration (FDA) on November 18, 2025, was immediately followed by the rollout of the Rely On REDEMPLO patient support program. This program is designed to shepherd patients through every stage of their treatment journey with the siRNA medicine. For the estimated 6,500 people in the U.S. living with genetic or clinical Familial Chylomicronemia Syndrome (FCS), this support is critical, especially since REDEMPLO is self-administered at home via a subcutaneous injection once every three months. A key component of this relationship management is the provision of financial assistance options for eligible patients. Furthermore, Arrowhead Pharmaceuticals announced the One-REDEMPLO pricing model, establishing one consistent price across current and potential future indications, set at $60,000 per year. This unified pricing strategy is a direct attempt to simplify the payer and patient experience, moving away from variable pricing based on indication. That's a clear signal about how they view long-term patient commitment.

High-touch, direct engagement with specialist physicians (e.g., lipidologists).

Commercial success for REDEMPLO hinges on deep engagement with the specialized medical community that manages FCS. The prescriber base is tightly defined, comprising specialist physicians such as lipidologists, endocrinologists, preventive cardiologists and internal medicine physicians with a focus on lipid disorders. To ensure the drug gets into the hands of those who need it quickly after the November 2025 approval, Arrowhead Pharmaceuticals is targeting approximately 5,000 health care professionals through direct, personal engagement at launch. This high-touch approach is necessary because these specialists often operate within complex multidisciplinary teams that include gastroenterologists and specialized dietitians, all of whom need to be educated on the first and only FDA-approved siRNA medicine for FCS.

Strategic, long-term collaboration management with large pharma partners.

For a company like Arrowhead Pharmaceuticals, managing strategic, long-term collaborations is as much a customer relationship as managing patients; these partners fund significant portions of the pipeline. The fiscal year 2025 results clearly show the financial weight of these relationships, with full-year revenue hitting $829.4 million, driven entirely by these agreements. You need to track these milestones closely, as they directly impact the balance sheet, which stood at $781.5 million in total cash resources as of September 30, 2025. Here's the quick math on the two major recent deals:

Partner	Program/Asset	Upfront Payment Received	Total Potential Milestones
Sarepta Therapeutics	ARO-DM1 (for DM1)	Not specified for upfront, but earned $300 million in FY2025 milestones (including a recent $200 million payment).	Not specified in detail, but previously earned a $100 million milestone.
Novartis	ARO-SNCA (for synucleinopathies) and other targets	$200 million	Up to $2 billion, plus tiered royalties up to the low double digits.

Managing these relationships means ensuring pipeline progress meets contractual obligations, like the recent $200 million milestone earned from Sarepta Therapeutics following a drug safety committee review for ARO-DM1.

Regulatory and medical affairs engagement with the FDA and global agencies.

The relationship with regulatory bodies is perhaps the most critical for a biotech transitioning to commercial status. The successful FDA approval of REDEMPLO on November 18, 2025, followed the FDA's acceptance of the New Drug Application (NDA) on January 17, 2025, with a PDUFA target action date of November 18, 2025. This approval was based on the Phase 3 PALISADE study, which showed a median triglyceride reduction of -80% from baseline. Beyond the initial FCS indication, Arrowhead Pharmaceuticals plans to submit approval applications for plozasiran to other regulatory authorities in 2025, and ongoing late-stage studies, like SHASTA-3 and SHASTA-4, are designed to support a supplemental NDA for broader use, with top-line data expected in the third quarter of 2026. This continuous, data-driven engagement with the FDA and global agencies is what unlocks the next phase of commercial opportunity.

Finance: review the cash runway projection into fiscal 2028 based on current burn rate and expected milestone receipts by end of Q1 2026.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Channels

You're transitioning Arrowhead Pharmaceuticals, Inc. from a development focus to a commercial entity with REDEMPLO. Here's how they are getting the product and the data to the market as of late 2025.

Direct Commercial Sales Force for the US Market Launch of REDEMPLO

Arrowhead Pharmaceuticals, Inc. transitioned to a commercial-stage company following the U.S. FDA approval of REDEMPLO (plozasiran) on November 19, 2025, for familial chylomicronemia syndrome (FCS). The company stated its commercial team was prepared to 'hit the ground running' upon approval. The launch is supported by the Rely On REDEMPLO patient support program.

The pricing strategy channels the drug through a unified One-REDEMPLO pricing model, setting a yearly Wholesale Acquisition Cost (WAC) at $60,000 per patient, regardless of future indications. This is significantly lower than the competitor Tryngolza's WAC of $595,000. The estimated US population for FCS is approximately 6,500 people.

Specialty Pharmacies and Distributors for Drug Fulfillment (Drug in Channel)

Following the FDA approval, Arrowhead Pharmaceuticals, Inc. confirmed they have drug in channel, indicating the fulfillment pathway is active. REDEMPLO is self-administered at home via a subcutaneous injection once every three months. While specific specialty pharmacy partners aren't detailed, the company relies on this network for drug fulfillment for its first commercial product.

Out-Licensing Partners (e.g., Sanofi, Amgen) for Ex-US Market Access

Business development and licensing remain critical to the Arrowhead Pharmaceuticals, Inc. model, providing significant non-product revenue inflows. The fiscal year 2025 revenue totaled $829 million, driven entirely by these agreements with Sarepta, Sanofi, and GSK.

Here are the key financial terms and partner activities as of late 2025:

Partner	Program/Scope	Upfront Payment (USD)	Total Potential Milestones (USD)	Royalty/Other Terms
Sarepta Therapeutics	CNS/Muscle targets (ARO-DM1 milestone earned)	$500 million (cash) + $325 million (equity)	Up to $10 billion total (per program terms)	Tiered royalties up to the low double digits.
Novartis	ARO-SNCA (Parkinson's) + additional targets (TRiM platform)	$200 million	Up to $2 billion	Tiered royalties up to the low double digits.
Sanofi (Visirna via)	REDEMPLO in Greater China	N/A (Visirna received $130 million upfront)	Up to $265 million (for plozasiran milestones)	N/A
Takeda/Amgen	Fazirsiran / Olpasiran	N/A (Milestones/Royalties from prior deals)	N/A	N/A

The Sarepta deal included a $325 million equity investment priced at $27.25 per share, representing a 35% premium. Arrowhead also earned a $200 million milestone from Sarepta in the period leading up to the earnings call.

Scientific Publications and Medical Conferences to Disseminate Clinical Data

Dissemination of clinical data is channeled through participation in major medical meetings. Arrowhead Pharmaceuticals, Inc. was scheduled to present data at several key events in late 2025, supporting both the approved indication and pipeline expansion efforts.

Key participation and data presentation channels include:

• American Heart Association (AHA) Scientific Sessions 2025 (November 7-10, 2025).
• American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting 2025 (November 7-11, 2025).
• Jefferies Global Healthcare Conference 2025 (November 17-20, 2025).
• TD Cowen's Virtual Treatment Advancements in Obesity & Related Disorders (November 24, 2025).

Specific data presented included a poster presentation on the Phase 3 study of Zodasiran at AHA, and multiple presentations on Fazirsiran at AASLD.

For pipeline expansion, enrollment is complete for the SHASTA-3 and SHASTA-4 Phase III trials, with top-line data expected in the third quarter of 2026, supporting a supplemental New Drug Application submission planned before the end of 2026.

The company's cash and investments stood at $919 million as of the earnings announcement date, following significant licensing inflows.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Customer Segments

You're looking at the customer base for Arrowhead Pharmaceuticals, Inc. (ARWR) as they transition from a pure R&D focus to having their first commercial product, REDEMPLO, on the market in late 2025. This segment breakdown shows who is buying their technology or who is the patient population for their approved/pipeline drugs.

The primary patient segments are defined by rare and severe lipid disorders, which are the initial focus for their FDA-approved medicine, REDEMPLO (plozasiran).

• Patients with Familial Chylomicronemia Syndrome (FCS), an estimated 6,500 people in the U.S.
• Patients with Severe Hypertriglyceridemia (SHTG) at high risk of acute pancreatitis, estimated at approximately 1 million in the U.S.
• Patients with Homozygous Familial Hypercholesterolemia (HoFH) targeted by zodasiran, with its Phase III trial enrolling about 60 subjects over age 12.

The pricing for the approved drug, REDEMPLO, is set uniformly across indications, which is a key factor for payer and physician adoption.

Customer Segment Detail	Relevant Metric	Value/Amount
FCS Patient Population (U.S. Estimate)	Genetic or Clinical FCS Patients	6,500
REDEMPLO Annual WAC	Wholesale Acquisition Cost	$60,000
High-Risk SHTG Population (U.S. Estimate)	Triglycerides $\geq$ 880 mg/dL or $>$ 500 mg/dL with prior pancreatitis	Approx. 1 million
HoFH Trial Enrollment (YOSEMITE)	Subjects Randomized	Approx. 60

Specialist physicians, such as endocrinologists and cardiologists, are the prescribers, directly interfacing with the FCS patient segment, which saw REDEMPLO achieve median triglyceride reductions around 80% from baseline in the PALISADE Phase III study.

The third major customer segment involves large pharmaceutical and biotechnology companies who license Arrowhead Pharmaceuticals, Inc. (ARWR)'s Targeted RNAi Molecule (TRiM™) platform technology. These partnerships are critical, as they drove the vast majority of the company's revenue in fiscal year 2025.

• Total Fiscal Year 2025 Revenue: $829 million (or $829.4 million).
• Revenue derived entirely from license and collaboration agreements.
• Fiscal Year 2025 Net Loss narrowed to $2 million (or loss of $0.01 per share), compared to a loss of $599 million in fiscal 2024.

Specific high-value partners define this segment, providing significant upfront payments and milestone revenue.

Partner Company	Agreement/Milestone Event (FY2025)	Financial Impact (FY2025 Revenue/Payment)
Sarepta Therapeutics	Milestone payment earned for ARO-DM1 (SRP-1003)	$300 million total milestone earned in FY2025 (including $200 million in Q4 FY2025)
Novartis	Upfront payment for ARO-SNCA collaboration	$200 million upfront payment
Sanofi	Revenue recognized from license agreement	$130 million
Novartis (Total Potential)	Future milestone payments	Up to $2 billion
Sarepta Therapeutics (Total FY2025)	Revenue recognized from arrangement	Roughly $697 million

The company's cash position also reflects the importance of these corporate customers; cash and investments totaled $919 million as of September 30, 2025.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Cost Structure

You're looking at the cost side of Arrowhead Pharmaceuticals, Inc. (ARWR) as they transition from a pure development company to one with a commercial product, REDEMPLO. The spending profile is dominated by the high burn rate required to advance their RNAi pipeline.

Dominant Research and Development (R&D) expenses represent the largest cost driver. For the fiscal year 2025 ended September 30, 2025, Research and Development expenses totaled $607.2 million (reported as $607,159 thousand). This compares to $505.9 million in the prior fiscal year.

The General and Administrative (G&A) costs are also scaling up, reflecting the commercialization build-out now that REDEMPLO is approved. For FY2025, General and Administrative expenses were $123.9 million (reported as $123,943 thousand). This is a notable increase from the G&A of $98.8 million (reported as $98,761 thousand) in FY2024. Total operating expenses for FY2025 reached $731.1 million (reported as $731,102 thousand).

The R&D spend is heavily weighted toward late-stage work. Here's a breakdown of where that investment is going:

• Costs to run clinical trials, including clinical manufacturing costs.
• Expense related to active preclinical-stage programs.
• Late-stage development of plozasiran and SHTG studies.

Specifically, nearly two-thirds (2/3) of the clinical trial spend in fiscal year 2025 was attributed to the late-stage development of plozasiran and the SHTG registrational studies. Zodasiran is also in a Phase 3 clinical trial.

Manufacturing and supply chain costs are now becoming more tangible with the launch of REDEMPLO. The company established a single pricing structure for REDEMPLO, setting the annual wholesale acquisition cost (WACC) at $60,000. Clinical manufacturing costs were noted as a key driver within the overall R&D spend.

Revenue-generating activities, primarily from partnerships, help offset these costs, but the upfront and milestone payments are distinct from the core operating expenses. Consider these key non-operating/partnership-related cash inflows that impact the overall financial structure:

Revenue/Payment Type	Amount (USD)	Source/Context
Novartis Upfront Payment (ARO-SNCA)	$200 million	Global licensing and collaboration agreement
Sarepta Milestone Payments Earned (Total)	$300 million	Milestones for ARO-DM1 Phase 1/2 study
Sanofi Upfront Payment (Visirna/Greater China)	$130 million	Asset purchase agreement for four cardiometabolic candidates

Intellectual property maintenance and licensing fees are an ongoing, though less detailed, component of the cost structure, as the company continues to protect and expand its proprietary Targeted RNAi Molecule (TRiM™) platform.

Finance: draft 13-week cash view by Friday.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Canvas Business Model: Revenue Streams

You're looking at the core engine that powers Arrowhead Pharmaceuticals, Inc. (ARWR) right now: how the money actually comes in. For a company transitioning from pure R&D to commercial stage, the revenue mix tells the whole story. As of late 2025, it's overwhelmingly about partnerships.

Collaboration and License Revenue Dominance

The financial results for fiscal year 2025 show that licensing deals are the primary revenue driver. Collaboration and license revenue totaled $829.4 million for FY2025. This massive figure represents the company's success in monetizing its proprietary Targeted RNAi Molecule (TRiM™) platform through external partners. This revenue stream is critical for funding the internal pipeline development, especially as the company manages its first commercial launch.

The total FY2025 revenue of $829 million was driven entirely by these license and collaboration agreements with partners like Sarepta Therapeutics, Sanofi, and GlaxoSmithKline (GSK). This is a stark contrast to the $3.55 million reported in fiscal 2024.

Upfront and Milestone Payments from Partners

A significant portion of the 2025 revenue came from hitting specific targets within those agreements. You can see the major components contributing to that top line:

• The agreement with Novartis, announced in September 2025 for ARO-SNCA, included an upfront payment of $200 million.
• Arrowhead Pharmaceuticals, Inc. earned a $200 million milestone payment from Sarepta Therapeutics following a Drug Safety Committee review and subsequent authorization to dose escalate for ARO-DM1.
• The company also earned a $100 million milestone from Sarepta related to the first of two pre-specified enrollment targets for ARO-DM1, which was satisfied by a combination of $50 million in cash and $50 million worth of Arrowhead common stock bought back from Sarepta.

Here's a quick look at the major recognized collaboration revenue components for FY2025:

Partner	FY2025 Revenue Component	Amount (Millions USD)
Sarepta Therapeutics	Ongoing Recognition of Initial Consideration	$587
Sarepta Therapeutics	Milestone Payment (ARO-DM1)	$94
Sanofi	Upfront/Consideration Recognition	$130
GSK	Milestone Payment	$2.6

The total recognized revenue from Sarepta Therapeutics for FY2025 was approximately $697 million. That's a lot of non-dilutive cash flow.

Royalty Payments on Out-licensed Products

While specific royalty dollar amounts for FY2025 aren't broken out separately from the milestone/recognition figures, the structure is in place for future passive income. For instance, the Novartis deal makes Arrowhead Pharmaceuticals, Inc. eligible to receive tiered royalties on commercial sales up to the low double digits. Similarly, the Sarepta agreement includes potential royalties on commercial sales up to the low double digits. These payments will become a more visible stream once out-licensed products reach the market.

Initial Product Sales of REDEMPLO

Arrowhead Pharmaceuticals, Inc. transitioned into a commercial-stage company with the FDA approval of REDEMPLO (plozasiran) in November 2025 for familial chylomicronemia syndrome (FCS). FCS affects an estimated 6,500 people in the U.S. The company launched the product with a unified pricing model of $60,000 per year for current and future indications. Analyst estimates suggest initial product sales for FCS in 2025 were around $0.5 million. Honestly, the expectation is that commercial sales will have no substantial impact on 2026 financials, with growth expected post-2026 as they pursue label expansion into severe hypertriglyceridemia (sHTG).

Ongoing Revenue Recognition from Existing Deals

The revenue recognition schedule from the massive February 2025 Sarepta deal provides a steady baseline. As noted, $587 million relates to the ongoing recognition of initial Sarepta consideration in FY2025. Furthermore, the Sarepta deal includes a structure for future payments:

• $250 million to be paid in annual installments of $50 million over 5 years, with the first payment due in February 2026.
• Potential milestone payments totaling approximately $10 billion are also possible from the Sarepta agreement.

Finance: draft 13-week cash view by Friday.