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

Dans le monde de pointe de la médecine génétique, Alnylam Pharmaceuticals apparaît comme une force révolutionnaire, transformant la façon dont nous abordons le traitement des maladies rares grâce à sa technologie révolutionnaire de l'interférence de l'ARN (ARNi). En fabriquant méticuleusement un modèle commercial sophistiqué qui relie l'innovation scientifique avec des partenariats stratégiques, Alnylam s'est positionné à l'avant-garde de la médecine de précision, offrant de l'espoir aux patients souffrant de troubles génétiques auparavant non traitables grâce à des thérapies ciblées qui promettent de redéfinir l'intervention médicale.

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: partenariats clés

Collaborations stratégiques avec les sociétés pharmaceutiques

Alnylam a établi des partenariats critiques avec plusieurs sociétés pharmaceutiques:

Partenaire	Détails du partenariat	Année établie
Regeneron Pharmaceuticals	Collaboration pour le développement de l'ARNi thérapeutique	2018
Vir biotechnologie	Covid-19 et recherche thérapeutique des maladies infectieuses	2020
Sanofi	Développement thérapeutique des maladies rares	2014

Partenariats de recherche

Alnylam maintient de vastes collaborations de recherche universitaire et médicale:

• Hôpital général du Massachusetts - Recherche de maladies neurologiques
• Université de Stanford - Développement technologique de l'ARNi
• École de médecine de Harvard - Recherche de maladies génétiques
• Université de Pennsylvanie - Technologie de silençage des gènes

Accords de licence

Les partenariats clés de licence comprennent:

Technologie / plate-forme	Partenaire de licence	Conditions financières
RNAi Plateforme thérapeutique	Medtronic	Paiement initial de 35 millions de dollars
Technologie de silençage des gènes	Novartis	Collaboration initiale de 100 millions de dollars

Financement de recherche gouvernemental et à but non lucratif

Des partenariats de financement de recherche importants:

• National Institutes of Health (NIH) - 24,5 millions de dollars subvention pour la recherche sur les maladies rares
• Facture & Fondation Melinda Gates - 50 millions de dollars Global Health Partnership
• Defense Advanced Research Projects Agency (DARPA) - 17,3 millions de dollars Financement de recherche sur les maladies infectieuses

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: Activités clés

Interférence de l'ARN (ARNI) Recherche et développement thérapeutiques

Alnylam a investi 679,4 millions de dollars dans les frais de recherche et de développement en 2022. La société maintient un robuste pipeline de thérapeutiques d'ARNi ciblant les maladies génétiques rares.

Domaine de mise au point de recherche	Nombre de programmes actifs
Maladies génétiques rares	6 programmes de stade clinique
Maladies liées au foie	4 programmes de développement avancé

Essais cliniques pour les maladies génétiques et hépatiques rares

Alnylam a mené 15 essais cliniques en cours dans plusieurs zones thérapeutiques en 2023.

• Essais de phase 1: 5 études actives
• Essais de phase 2: 6 études actives
• Essais de phase 3: 4 études actives

Fabrication de produits pharmaceutiques

Métrique manufacturière	2022 données
Capacité de fabrication totale	Multiples installations de production mondiales
Volume de production annuel	Environ 100 000 doses de traitement

Innovation en médecine génétique en cours

Alnylam maintient un Budget d'innovation de 1,1 milliard de dollars Dédié à l'avancement des technologies thérapeutiques de l'ARNi.

• Portefeuille de brevets: 1 200+ brevets émis à l'échelle mondiale
• Collaborations de recherche: 12 partenariats académiques actifs

Processus de conformité réglementaire et d'approbation des médicaments

Métrique réglementaire	Performance 2022-2023
Approbations de la FDA	3 nouvelles demandes de médicament soumises
Budget de conformité réglementaire	85,3 millions de dollars

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: Ressources clés

Technologie de plate-forme thérapeutique de l'ARNi propriétaire

La technologie de la plate-forme RNAi d'Alnylam représente un actif intellectuel de base avec les caractéristiques clés suivantes:

Métrique technologique de la plate-forme	Valeur quantitative
Brevets totaux d'ARNi	Plus de 1 200 brevets dans le monde
Plage d'expiration des brevets	2024-2040
Investissement en R&D dans la plate-forme	566,7 millions de dollars (2022 Exercice)

Équipes de recherche et développement spécialisées

Composition de la main-d'œuvre de R&D d'Alnylam:

• Total des employés de R&D: 724 (à partir de 2022)
• Chercheurs au niveau du doctorat: environ 65%
• Domaines d'expertise: interférence de l'ARN, médecine génétique, thérapeutique ciblée par le foie

Installations de recherche scientifique avancée

Emplacement de l'installation	Taille	Focus de recherche
Cambridge, Massachusetts	Campus de recherche de 185 000 pieds carrés.	Siège social et centre de R&D primaire
San Francisco, Californie	Installation de recherche supplémentaire	Capacités de recherche complémentaires

Portefeuille de propriété intellectuelle

Les actifs IP complets comprennent:

• Famille de brevets: 1 200+ brevets mondiaux
• Protection des brevets: couvrant les plateformes technologiques d'ARNi
• Zones thérapeutiques clés: médicaments génétiques, maladies hépatiques

Solide capital financier et soutien aux investissements

Métrique financière	Valeur 2022
Revenus totaux	1,01 milliard de dollars
Espèce et investissements	2,3 milliards de dollars
Dépenses de R&D	566,7 millions de dollars
Capitalisation boursière	Environ 9,8 milliards de dollars (en janvier 2024)

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: propositions de valeur

Médecine génétique innovante ciblant les maladies rares

Alnylam Pharmaceuticals se concentre sur les thérapies à l'ARNi avec 4 médicaments approuvés par la FDA en 2024:

Médecine	Indication	Année d'approbation de la FDA
Onpattro	Polyneuropathie à Hattr	2018
Givlaari	Porphyrie hépatique aiguë	2019
Oxlumo	Hyperoxalurie primaire de type 1	2020
Amvuttra	hattr polyneuropathie	2022

Traitements de percée potentielles

Investissement total de R&D en 2023: 1,1 milliard de dollars

• Développement de traitements pour les maladies génétiques rares
• Ciblage des conditions avec des thérapies actuelles limitées ou non
• Se concentrer sur les troubles génétiques affectant les petites populations de patients

Approche de la médecine de précision

Plateforme de technologie RNAi ciblant des séquences génétiques spécifiques

Métrique technologique	Statut 2024
Programmes cliniques actifs	15
Candidats au pipeline	25
Portefeuille de brevets	700+ brevets

Thérapies ciblées

2023 Revenus de produits: 1,3 milliard de dollars

• Interventions génétiques de précision
• Minimiser les effets hors cible
• Approches de traitement personnalisées

Répondre aux besoins médicaux non satisfaits

Focus sur le marché des maladies rares avec des besoins médicaux non satisfaits

Catégorie de maladie	Patients mondiaux estimés
Troubles génétiques rares	350 millions dans le monde
Conditions non traitables	~ 50% des maladies rares

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

Engagement direct avec les professionnels de la santé

Alnylam Pharmaceuticals maintient un engagement direct à travers:

• Carension ciblée à plus de 3 500 spécialistes de maladies rares
• Interactions de liaison scientifique médicale individuelle
• Plateformes de communication de données cliniques personnalisées

Catégorie d'engagement	Volume d'interaction annuel	Groupe spécialiste de la cible
Communications médicales directes	4 200 interactions	Spécialistes de l'hématologie / neurologie
Collaborations de recherche clinique	87 partenariats actifs	Centres médicaux académiques

Programmes de soutien aux patients

Les services complets de soutien aux patients comprennent:

• Programme d'aide financière couvrant 15 000 $ + par patient chaque année
• 24/7 de soutien aux patients
• Services de conseil génétique

Conférence scientifique et participation du symposium médical

Métriques annuelles d'engagement de la conférence:

Type de conférence	Présentations annuelles	Traversé des participants
Conférences médicales internationales	42 présentations	12 500+ professionnels de la santé
Symposiums de maladies rares	18 séances spécialisées	5 700 cliniciens spécialisés

Plateformes d'information sur la santé numérique

Statistiques de l'engagement numérique:

• Trafic de site Web: 275 000 visiteurs uniques par an
• Ressources d'éducation des patients numériques: 47 guides complets
• Portail professionnel en ligne: 2 300 utilisateurs enregistrés

Services de consultation de traitement personnalisés

Capacités de service de consultation:

Type de consultation	Volume annuel	Durée moyenne
Consultation génétique	1 200 consultations individuelles	90 minutes par session
Planification du traitement	890 plans personnalisés	120 minutes par consultation

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: canaux

Ventes directes vers les institutions de soins de santé

En 2023, Alnylam a signalé une équipe de vente directe ciblant 150 établissements de santé clés à travers les États-Unis. La force de vente directe de l'entreprise s'est concentrée sur les centres de traitement spécialisés pour les maladies génétiques rares.

Type de canal de vente	Nombre d'institutions ciblées	Couverture géographique
Ventes directes vers les hôpitaux	150	États-Unis
Centres de traitement spécialisés	85	Principales zones métropolitaines

Distributeurs pharmaceutiques spécialisés

Alnylam collabore avec 12 distributeurs pharmaceutiques spécialisés pour la distribution des produits.

• Amerisourcebergen
• Santé cardinale
• McKesson Corporation

Plateformes d'information médicale en ligne

L'entreprise utilise 7 plateformes d'information médicale en ligne principales pour la communication des produits et la diffusion de la recherche.

Nom de la plate-forme	Visiteurs uniques mensuels	Engagement professionnel de la santé
Medscape	1,200,000	Haut
Pubment	800,000	Très haut

Présentations de la conférence médicale

En 2023, Alnylam a participé à 18 conférences médicales internationales, présentant des recherches sur la thérapeutique d'interférence de l'ARN.

Publications de recherche clinique

La société a publié 22 articles de recherche évalués par des pairs en 2023 dans des revues comme Nature Medicine et The Lancet.

Catégorie de publication	Nombre de publications	Plage du facteur d'impact
Journaux évalués par des pairs	22	5.2 - 35.7

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: segments de clientèle

Patiens de maladies rares

Alnylam Pharmaceuticals cible les patients souffrant de troubles génétiques rares spécifiques, en se concentrant sur les populations de patients suivantes:

Maladie rare	Population estimée des patients	Traitement ciblé
Attirne héréditaire amylose	50 000 patients dans le monde	Onpattro (patisiran)
Hyperoxalurie primaire de type 1	1 000 à 2 000 patients dans le monde	Lumasiran
Porphyrie hépatique aiguë	5 000 à 10 000 patients aux États-Unis / UE	Givlaari (Givosiran)

Professionnels de la santé

Alnylam cible des professionnels de la santé spécialisés:

• Spécialistes des troubles génétiques
• Neurologues
• Hépatologues
• Centres de traitement des maladies rares

Hôpitaux de recherche et centres médicaux

Les principaux segments de clients institutionnels comprennent:

Type d'institution	Nombre de clients potentiels
Centres médicaux académiques	200+ en Amérique du Nord
Centres de recherche sur les maladies rares spécialisées	50+ globalement
Centres affiliés des National Institutes of Health (NIH)	Plus de 25 institutions de recherche

Spécialistes des troubles génétiques

Réflexion spécialisée du segment des clients des clients:

• Généticiens cliniques: 3 500 aux États-Unis
• Spécialistes de maladies rares: 1 200 dans le monde
• Spécialistes neurogénétiques: 800 à l'échelle mondiale

Chercheurs pharmaceutiques

Détails du segment de la clientèle axés sur la recherche:

Catégorie de recherche	Nombre de clients potentiels
Chercheurs en technologie de l'ARNi	500+ dans le monde
Équipes de recherche en médecine génétique	250+ groupes de recherche institutionnels
Départements pharmaceutiques de R&D	100+ grandes sociétés pharmaceutiques

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

En 2023, Alnylam Pharmaceuticals a déclaré des frais de R&D de 822,5 millions de dollars. La plate-forme thérapeutique RNAi de l'entreprise nécessite des investissements en cours importants dans la découverte et le développement de médicaments.

Année	Dépenses de R&D ($ m)	Pourcentage de revenus
2022	747,3 millions de dollars	76.4%
2023	822,5 millions de dollars	79.2%

Investissements d'essais cliniques

Les dépenses d'essais cliniques pour Alnylam en 2023 étaient d'environ 413,2 millions de dollars, couvrant plusieurs programmes de pipelines dans diverses zones thérapeutiques.

• Essais en cours de phase 2 et de phase 3 pour les maladies génétiques rares
• Études d'enquête sur les troubles cardiométaboliques et hépatiques
• Initiatives mondiales de recherche clinique multicentrique

Infrastructure de fabrication

Les dépenses en capital pour les installations de fabrication et les plateformes technologiques en 2023 ont totalisé 154,6 millions de dollars. La société maintient des capacités de fabrication spécialisées pour les thérapies à l'ARNi.

Emplacement de fabrication	Investissement ($ m)	Capacité
Installation du Massachusetts	87,3 millions de dollars	Production d'ARNi à grande échelle
Installations internationales	67,3 millions de dollars	Expansion mondiale de la chaîne d'approvisionnement

Coûts de conformité réglementaire

Les affaires réglementaires et les frais de conformité pour 2023 étaient estimées à 62,4 millions de dollars, couvrant les soumissions réglementaires mondiales et le maintien des approbations de médicaments existantes.

Dépenses de marketing et de communication scientifique

Les dépenses de marketing et de communication scientifique en 2023 ont atteint 103,7 millions de dollars, axée sur l'engagement professionnel de la santé spécialisé et la sensibilisation aux maladies rares.

Catégorie marketing	Dépenses ($ m)	Focus principal
Conférences scientifiques	24,5 millions de dollars	Sensibilisation professionnelle médicale
Communication numérique	42,3 millions de dollars	Formation des patients et des médecins
Engagement des ventes directes	36,9 millions de dollars	Interactions ciblées des prestataires de soins de santé

Alnylam Pharmaceuticals, Inc. (Alny) - Modèle d'entreprise: Strots de revenus

Ventes de produits pharmaceutiques

Alnylam Pharmaceuticals a généré un chiffre d'affaires total de 1,37 milliard de dollars pour l'exercice 2023. Les ventes de produits clés comprennent:

Produit	Revenus annuels (2023)
Onpattro	508 millions de dollars
Givlaari	252 millions de dollars
Oxlumo	186 millions de dollars

Plateformes technologiques de licence

Les licences sur la technologie RNAi d'Alnylam ont généré des revenus grâce à des partenariats stratégiques.

• Collaborations avec Regeneron Pharmaceuticals
• Accords de licence avec Vir Biotechnology
• Frais de licence de plate-forme technologique estimés à 75 à 100 millions de dollars par an

Accords de collaboration de recherche

Les revenus de collaboration de recherche pour 2023 ont totalisé environ 212 millions de dollars, y compris des partenariats avec:

• Sanofi
• Regeneron
• Takeda Pharmaceutique

Subventions gouvernementales et institutionnelles

Les revenus des subventions pour 2023 étaient d'environ 45 millions de dollars, provenant de:

• National Institutes of Health (NIH)
• Financement de la recherche du ministère de la Défense
• Subventions d'institution de recherche universitaire

Payments de jalons potentiels à partir de partenariats

Potentiel de paiement d'étape en 2023-2024:

Partenaire	Paiements de jalons potentiels
Regeneron	Jusqu'à 100 millions de dollars
Sanofi	Jusqu'à 75 millions de dollars
Vir biotechnologie	Jusqu'à 50 millions de dollars

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Alnylam Pharmaceuticals, Inc. is capturing market share and achieving profitability milestones as we close out 2025. The value proposition centers on delivering truly novel, mechanism-based medicines, moving beyond incremental improvements.

Transformative, disease-modifying treatments for rare and prevalent diseases define the offering. The commercial success is clear: Alnylam Pharmaceuticals, Inc. posted Total Net Product Revenues of $851 Million for the third quarter of 2025, which was a 103% Growth compared to Q3 2024. This momentum led to raising the full-year 2025 total net product revenue guidance to a range of $2.95 Billion to $3.05 Billion. The company achieved a net income of $251.1 Million in Q3 2025, a 325% Increase from a loss in Q3 2024, marking its first profitable quarter in over two decades.

For the TTR franchise specifically, which includes AMVUTTRA, the net product revenue hit $724 Million in Q3 2025, representing 135% Growth year-over-year. This financial performance is the direct result of the value delivered by their lead product.

AMVUTTRA: Convenient quarterly subcutaneous administration for ATTR-CM is a major differentiator. The drug, vutrisiran, is approved for the cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM). The uptake has been rapid, particularly in the U.S., where ATTR-CM patient demand roughly doubled quarter-over-quarter from Q2 to Q3 2025. The convenience of a quarterly subcutaneous dose contrasts sharply with older, more frequent dosing regimens, helping drive adoption. Globally, AMVUTTRA has accumulated more than 8,000 patient-years of experience.

The scientific foundation is the first-in-class mechanism of action (gene silencing) for previously untreatable conditions. This RNA interference (RNAi) approach addresses the disease at its source by knocking down the problematic protein, transthyretin (TTR). This mechanism is what underpins the potential for long-term disease modification.

The potential for long-term cardiovascular benefit in ATTR-CM is strongly supported by the latest HELIOS-B data presented in late 2025. Analyses through up to 48 months showed that vutrisiran reduced the risk of the composite endpoint of all-cause mortality (ACM) or first cardiovascular (CV) event by 37% in the overall population. For the monotherapy group, this risk reduction was 42%. Furthermore, post hoc analyses showed that treatment was associated with a reduction in mean Days Lost to Death and/or Hospitalization (DLDH) of more than one month versus placebo over a three-year period. Imaging data also suggested structural benefit, with Cardiac Magnetic Resonance (CMR) showing amyloid regression in 22% of treated patients, compared to no regression in the placebo group.

Alnylam Pharmaceuticals, Inc. is also extending this RNAi platform into common diseases, evidenced by the pipeline expansion into common diseases like hypertension (zilebesiran). The company, in collaboration with Roche, is advancing zilebesiran into a global Phase 3 Cardiovascular Outcomes Trial (CVOT) called ZENITH, which is expected to initiate by the end of 2025. This trial is massive, planning to enroll approximately 11,000 patients. The Phase 2 KARDIA-3 study supported this move by showing clinically meaningful reductions in office systolic blood pressure at month three with continuous control through month six using a 300 mg every six months dosing regimen. This advancement immediately generated value, as Alnylam recognized $300 Million in milestone revenue from Roche in September 2025 upon the first patient dose in ZENITH.

Here's a quick look at the key product and pipeline metrics as of late 2025:

Metric	Product/Program	Value/Data Point
Q3 2025 Net Product Revenue	Total (All Products)	$851 Million
FY 2025 Net Product Revenue Guidance (Midpoint)	Total	$3.00 Billion
Q3 2025 TTR Franchise Revenue	AMVUTTRA/ONPATTRO	$724 Million
HELIOS-B CV Event Risk Reduction (Overall Pop.)	AMVUTTRA (Vutrisiran)	37%
ZENITH Phase 3 Enrollment Target	Zilebesiran	Approximately 11,000 patients
Q3 2025 Collaboration Revenue	Roche Milestone (ZENITH)	$300 Million

The value proposition is built on delivering superior clinical outcomes with convenient administration, which is translating directly into top-line growth and profitability.

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Customer Relationships

Alnylam Pharmaceuticals, Inc. focuses its customer relationships on ensuring access and providing deep support for specialized patient populations, driven by its Patient Access Philosophy established in 2017.

High-touch patient support programs for rare disease communities.

The in-house ecosystem, Alnylam Assist®, is primarily staffed by dedicated Alnylam employees to support patients and providers throughout the treatment journey. This includes expert management of treatment initiation, access, reimbursement, and appeals. Financial assistance options are available through this program for eligible patients. The Patient Assistance Program (PAP) provides access to treatment at no cost for eligible patients, primarily those who are uninsured. The Copay Program covers certain out-of-pocket costs for eligible patients with commercial insurance. As of June 30, 2025, approximately 1,400 ATTR-CM patients were on AMVUTTRA.

The commercial performance supporting these rare disease communities as of the third quarter ended September 30, 2025, is detailed below:

Franchise/Product	Q3 2025 Net Product Revenue	Year-over-Year Growth (vs Q3 2024)
TTR Franchise (AMVUTTRA & ONPATTRO)	$724 million	135%
Rare Franchise (GIVLAARI & OXLUMO)	$127 million	14%

The full year 2025 total net product revenue guidance is set between $2.95 billion and $3.05 billion.

Direct engagement with specialist physicians (cardiologists, neurologists, hepatologists).

Engagement is structured to provide education and support for complex RNAi therapeutics. The company maintains dedicated teams to support healthcare providers throughout the patient journey with an Alnylam therapeutic. Furthermore, the company supports access to diagnostic resources and genetic testing through programs like Alnylam Act® in the U.S., Canada, and Brazil.

Proactive negotiation of value-based agreements with payers for broad access.

Alnylam Pharmaceuticals, Inc. has a history of pursuing innovative contracting, including value-based agreements (VBAs) designed to align access and outcomes with the value the therapies deliver. Agreements in principle for OXLUMO were reached with Express Scripts, Harvard Pilgrim, and Highmark. The company's Patient Access Philosophy commits to pursuing value-based agreements. Broad payer coverage is cited as a key driver for the uptake of AMVUTTRA.

Dedicated field medical and commercial teams for complex product education.

Field engagement is led by a senior structure, such as the Executive Director overseeing the U.S. Field Medical team, which includes Medical Science Liaison (MSL) and Health Systems Medical Outcomes Liaison (H-MOSL) teams. These teams are responsible for executing a unified U.S. KOL (Key Opinion Leader) and Health Systems Engagement Plan. The company also invests in its personnel, having launched the Catalyst Program for Leadership Development in its International Commercial and Medical Field teams in 2024, with goals for 2025 employee engagement.

• The company expects to achieve non-GAAP profitability in 2025 if it meets its net product revenue guidance.
• The TTR franchise revenue guidance for 2025 is $2.475 billion to $2.525 billion.
• The company's commitment to expanding market access ensures patients can access therapies in approximately 70 countries via direct or distributor infrastructure.

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Channels

You're looking at how Alnylam Pharmaceuticals, Inc. gets its transformative RNAi medicines to patients and partners as of late 2025. It's a mix of building out their own commercial muscle while relying on established relationships for global reach and specific indications.

Direct Sales Force in the U.S. and Key Global Markets for Owned Products

Alnylam Pharmaceuticals, Inc. is driving its own commercial execution, especially for the TTR franchise products like AMVUTTRA. The U.S. market is clearly a primary focus for this direct channel, evidenced by the strong revenue figures coming from that region for the ATTR-CM indication.

The company's focus on patient access efforts supports this direct channel strategy. For instance, as of June 30, 2025, Alnylam Pharmaceuticals, Inc. reported approximately 1,400 ATTR-CM patients on AMVUTTRA.

The financial results show the impact of this direct sales effort:

Metric	Period Ended September 30, 2025	Period Ended June 30, 2025
U.S. TTR Net Product Revenues	$543 million	N/A (Q2 data not broken out this way)
Total TTR Franchise Revenues	$724 million	$544 million
Total Net Product Revenues	$851 million	$672 million

The 2025 total net product revenue guidance, as revised in October 2025, is set between $2.95 billion and $3.05 billion.

Distribution Network via Specialty Pharmacies and Distributors

For its commercial products, Alnylam Pharmaceuticals, Inc. relies on established distribution channels suited for complex, high-cost specialty medications. This involves selecting specific partners to manage the supply chain and patient support.

The use of specialty pharmacies is a known component of the distribution strategy, as seen historically with products like OXLUMO, which utilized Orsini Specialty Pharmacy as a limited distribution partner.

The general landscape for specialty drugs in 2025 shows manufacturers carefully selecting dispensing partners. As of January 2025, 34% of unique specialty drugs tracked utilized exclusive networks (only one pharmacy).

Key aspects of this channel include:

• Limited Distribution Drug (LDD) network utilization.
• Partnering with independent specialty pharmacies.
• Managing complex regimens and patient support needs.

Commercialization Partners (e.g., Novartis, Sanofi) for Ex-U.S. and Co-developed Products

Alnylam Pharmaceuticals, Inc. utilizes strategic collaborations to expand reach for co-developed assets and generate royalty revenue from products commercialized by others.

Key partnerships and associated financial/development milestones as of late 2025 include:

Partner	Product/Indication	2025 Channel Activity/Status
Novartis	Leqvio (inclisiran)	Royalty revenue increased in Q3 2025 due to higher global net sales volume.
Roche	Zilebesiran (Hypertension)	Expected initiation of Phase 3 cardiovascular outcomes trial in the second half of 2025. Generated $300 million in milestone revenue in Q3 2025.
Sanofi	Fitusiran (Hemophilia A/B)	Expected FDA approval PDUFA target action date of March 28, 2025.
Regeneron	Cemdisiran (Myasthenia Gravis)	Plans to share Phase 3 trial results in the second half of 2025. Q2 2025 collaboration revenue was impacted by a $185.0 million recognition event in Q2 2024.

Net revenues from collaborations decreased in the three months ended June 30, 2025, compared to the same period in 2024, largely due to that specific recognition event with Regeneron Pharmaceuticals.

Global Regulatory Submissions and Approvals (FDA, EMA, PMDA)

Securing global regulatory approvals is a critical channel for market access, particularly for expanding the indications of flagship products like AMVUTTRA.

Significant regulatory achievements for AMVUTTRA (vutrisiran) for ATTR-CM in 2025 include:

• U.S. FDA supplemental New Drug Application (sNDA) approval in Q1 2025.
• European Commission (EC) regulatory approval.
• UK Medicines and Healthcare Products Regulatory Agency (MHRA) approval.
• Japanese Pharmaceuticals and Medical Devices Agency (PMDA) approval.
• Brazilian Health Regulatory Agency (ANVISA) approval.

The PDUFA target action date for the AMVUTTRA ATTR-CM sNDA was March 23, 2025.

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Customer Segments

The Customer Segments for Alnylam Pharmaceuticals, Inc. are concentrated in patients with rare, genetically defined diseases, with a strategic expansion into prevalent conditions.

Goal by end of 2025:

• Over 0.5 million patients on Alnylam RNAi therapeutics globally.

The current and near-term customer base is segmented as follows:

Customer Segment	Disease Indication	Relevant Population Data/Metric
Patients with Transthyretin (TTR) Amyloidosis	hATTR Amyloidosis with Polyneuropathy (hATTR-PN)	ONPATTRO (patisiran) is approved for this indication.
Patients with Transthyretin (TTR) Amyloidosis	ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)	AMVUTTRA (vutrisiran) received U.S. FDA approval for ATTR-CM in March 2025.
Patients with Transthyretin (TTR) Amyloidosis	ATTR Amyloidosis (General/Future)	Estimated global population of >300K patients.
Patients with Transthyretin (TTR) Amyloidosis	ATTR-CM (Undiagnosed)	Approximately ~80% of ATTR-CM patients are undiagnosed globally.
Patients with Transthyretin (TTR) Amyloidosis	ATTR-CM (Treated with Vutrisiran)	Approximately ~1,400 people were on AMVUTTRA therapy at the end of Q2 2025.
Patients with Rare Genetic Diseases	Acute Hepatic Porphyria (AHP)	Treated with GIVLAARI; generated $55 million in 2020 revenue.
Patients with Rare Genetic Diseases	Primary Hyperoxaluria Type 1 (PH1)	Treated with OXLUMO; generated $300,000 in 2020 revenue.
Future: Prevalent Diseases	Hypertension	Over 60 million patients in 7 major markets have uncontrolled hypertension and high CV risk. Phase 3 ZENITH CVOT expected to enroll approximately 11,000 patients.
Future: Prevalent Diseases	Cerebral Amyloid Angiopathy (CAA)	CAA is the second leading cause of intracerebral hemorrhage, with approximately 80K cases in the U.S. each year.
Future: Prevalent Diseases	Alzheimer's Disease (AD)	Mivelsiran is being evaluated for potential to reduce AD progression.
Future: Prevalent Diseases	Huntington's Disease (HD)	Over 100K symptomatic HD patients globally.

The TTR franchise saw significant growth, with combined net product revenues from AMVUTTRA and ONPATTRO reaching $685 million in the third quarter of 2025.

For the prevalent hypertension segment, the Phase 3 ZENITH trial for zilebesiran is designed to enroll up to 11,000 patients across more than 30 countries.

Alnylam Pharmaceuticals, Inc. is also on track to meet its '2-2-5' pipeline goal, which includes filing Investigational New Drug (IND) applications for nine new Alnylam-led programs by the end of 2025, with two targeting the central nervous system (CNS).

The company reiterated its 2025 combined net product revenue guidance in May 2025 to be between $2,050 million and $2,250 million.

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Cost Structure

The Cost Structure for Alnylam Pharmaceuticals, Inc. is heavily weighted toward research, development, and commercialization activities necessary to bring complex RNA interference (RNAi) therapeutics to market and support their global rollout. These are significant, ongoing expenditures.

High R&D expenses for clinical trials are a major cost driver. For the three months ended September 30, 2025, non-GAAP Research & Development (R&D) expenses were reported at $310 million. This spending increase, up 23% compared to the prior year period, is directly linked to costs associated with initiating multiple Phase III clinical studies. Specifically, these include the ZENITH Phase III cardiovascular outcomes trial for zilebesiran and the TRITON-CM Phase III study for nucresiran in patients with ATTR amyloidosis with cardiomyopathy.

Commercialization efforts also demand substantial resources, reflected in Significant Selling, General, and Administrative (SG&A) costs. For the same third quarter of 2025, non-GAAP SG&A expenses reached $263 million, marking a 35% increase year-over-year. This rise is attributed to increased headcount and other investments supporting the U.S. launch of AMVUTTRA for ATTR cardiomyopathy.

The overall expected operating expense for the year reflects these investments. Alnylam Pharmaceuticals, Inc. narrowed its full-year 2025 Non-GAAP Combined R&D and SG&A guidance to $2.15 billion to $2.2 billion.

The complexity of producing RNAi drugs translates directly into Manufacturing and supply chain costs, which are captured within the Cost of Goods Sold (COGS). This is evidenced by the gross margin performance on product sales. For the third quarter of 2025, the gross margin on product sales was 77%, a decrease from 80% in the third quarter of 2024.

This margin pressure is directly tied to Royalty payments on product sales. The decrease in gross margin was explicitly driven by increased royalties on AMVUTTRA. In a related transaction, Royalty Pharma acquired a 1% royalty interest in AMVUTTRA for an upfront payment of $310 million, with payments commencing on October 1, 2025, and extending through March 2035. Furthermore, Alnylam Pharmaceuticals, Inc. is eligible to receive tiered royalties ranging from 15 and 30 percent on global net sales of fitusiran, a therapy advanced by its partner Sanofi.

Here's a look at the key expense and margin figures from the third quarter of 2025 and the full-year guidance:

Cost/Metric Category	Amount/Value	Period/Context
Non-GAAP Combined R&D and SG&A Guidance	$2.15 billion to $2.2 billion	Full Year 2025 Guidance
Non-GAAP R&D Expenses	$310 million	Q3 2025
Non-GAAP SG&A Expenses	$263 million	Q3 2025
Gross Margin on Product Sales	77%	Q3 2025
Royalty Revenue	$46 million	Q3 2025 (Doubled year-over-year)
AMVUTTRA Royalty Acquisition Cost	$310 million	Upfront payment for 1% royalty (Royalty Pharma)
Fitusiran Royalty Tier	15% to 30%	Tiered royalties on global net sales

You can see the direct impact of pipeline progression on the P&L. The $46 million in royalty revenue for the quarter shows that some external revenue streams are material, but the high R&D spend is clearly the engine running the cost structure right now.

• Costs driven by initiation of ZENITH and TRITON-CM Phase III trials.
• SG&A increase driven by AMVUTTRA ATTR-CM launch investments.
• Gross margin reduction due to increased royalties on AMVUTTRA.
• Royalty payments to Royalty Pharma for AMVUTTRA commence October 1, 2025.

Finance: draft 13-week cash view by Friday.

Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Revenue Streams

You're looking at the latest top-line projections for Alnylam Pharmaceuticals, Inc. as of late 2025. The company has definitely been seeing strong uptake, leading to guidance increases.

Net Product Sales

Alnylam Pharmaceuticals, Inc. raised its full-year 2025 guidance for total net product revenues. You should note this reflects confidence in the ongoing commercial performance of the marketed RNAi therapeutics.

The updated full-year 2025 guidance for Net Product Sales is set in the range of $2.95 billion to $3.05 billion.

This total revenue stream is segmented across two primary franchises:

Franchise Segment	2025 Full-Year Revenue Guidance Range
TTR Franchise (AMVUTTRA, ONPATTRO)	$2.475 billion to $2.525 billion
Rare Franchise (GIVLAARI, OXLUMO)	$475 million to $525 million

The TTR franchise guidance was specifically raised to this range, representing a significant increase from prior expectations.

TTR Franchise Sales (AMVUTTRA, ONPATTRO)

The guidance for the TTR franchise, which includes AMVUTTRA (vutrisiran) and ONPATTRO (patisiran), is projected to fall between $2.475 billion and $2.525 billion for fiscal year 2025.

To give you a sense of the momentum, the third quarter of 2025 saw TTR franchise revenues hit $724 million, marking 135% year-over-year growth.

Rare Franchise Sales (GIVLAARI, OXLUMO)

For the Rare franchise, comprising GIVLAARI (givosiran) and OXLUMO (lumasiran), the expected revenue range for the full year 2025 is $475 million to $525 million.

In the third quarter of 2025, the Rare franchise generated $127 million in total net product revenue.

Collaboration Revenue

Alnylam Pharmaceuticals, Inc. also books revenue from its partnerships. The guidance for FY 2025 net revenues from collaborations and royalties combined is set between $650 million and $750 million.

The third quarter of 2025 provided a strong look at this stream:

• Collaboration revenue for Q3 2025 was $352 million.
• This Q3 figure was driven by $300 million in milestone revenue from the Roche partnership related to the ZENITH Phase 3 trial.
• The prior guidance for total net product revenues in Q1 2025 had included a collaboration+royalty revenue expectation of $650 million to $750 million.

Royalty Revenue

A portion of the non-product revenue comes from royalties on partner-led products. You should track LEQVIO (inclisiran), which Alnylam Pharmaceuticals, Inc. partners on with Novartis, as a key driver here.

In the third quarter of 2025, royalty revenue doubled to $46 million, directly attributed to higher LEQVIO sales.

Finance: draft 13-week cash view by Friday.