Alnylam Pharmaceuticals, Inc. (ALNY): Modelo de Negocio Canvas [Actualizado en Ene-2025]

En el mundo de vanguardia de la medicina genética, Alnylam Pharmaceuticals surge como una fuerza revolucionaria, transformando cómo abordamos el tratamiento de enfermedades raras a través de su innovadora tecnología de interferencia de ARN (ARNi). Al elaborar meticulosamente un modelo de negocio sofisticado que une la innovación científica con asociaciones estratégicas, Alnylam se ha posicionado a la vanguardia de la medicina de precisión, ofreciendo esperanza a pacientes con trastornos genéticos previamente no tratables a través de terapias específicas que prometen redefinir la intervención médica.

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: asociaciones clave

Colaboraciones estratégicas con compañías farmacéuticas

Alnylam ha establecido asociaciones críticas con varias compañías farmacéuticas:

Pareja	Detalles de la asociación	Año establecido
Regeneron Pharmaceuticals	Colaboración para el desarrollo terapéutico de RNAi	2018
Vir biotecnología	Covid-19 e investigación terapéutica de enfermedades infecciosas	2020
Sanofi	Desarrollo terapéutico de la enfermedad rara	2014

Asociaciones de investigación

Alnylam mantiene extensas colaboraciones de investigación académica y médica:

• Hospital General de Massachusetts - Investigación de enfermedades neurológicas
• Universidad de Stanford - Desarrollo de tecnología RNAi
• Harvard Medical School - Investigación de enfermedades genéticas
• Universidad de Pensilvania - Tecnología de silenciamiento de genes

Acuerdos de licencia

Las asociaciones clave de licencias incluyen:

Tecnología/plataforma	Socio de licencia	Términos financieros
Plataforma terapéutica RNAi	Medtrónico	$ 35 millones en el pago por adelantado
Tecnología de silenciamiento de genes	Novartis	Colaboración inicial de $ 100 millones

Financiación de investigación gubernamental y sin fines de lucro

Asociaciones de financiación de investigación significativas:

• Institutos Nacionales de Salud (NIH) - Subvención de $ 24.5 millones para la investigación de enfermedades raras
• Factura & Fundación Melinda Gates - Global Health Partnership de $ 50 millones
• Agencia de Proyectos de Investigación Avanzada de Defensa (DARPA) - Financiación de la investigación de enfermedades infecciosas de $ 17.3 millones

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: actividades clave

Investigación y desarrollo de drogas terapéuticas de interferencia de ARN (ARNi)

Alnylam invirtió $ 679.4 millones en gastos de investigación y desarrollo en 2022. La compañía mantiene una sólida cartera de terapias RNAi dirigidas a enfermedades genéticas raras.

Área de enfoque de investigación	Número de programas activos
Enfermedades genéticas raras	6 programas de etapa clínica
Enfermedades relacionadas con el hígado	4 programas de desarrollo avanzado

Ensayos clínicos para enfermedades genéticas e hepáticas raras

Alnylam realizó 15 ensayos clínicos en curso en múltiples áreas terapéuticas en 2023.

• Ensayos de fase 1: 5 estudios activos
• Ensayos de fase 2: 6 estudios activos
• Ensayos de fase 3: 4 estudios activos

Fabricación de productos farmacéuticos

Métrico de fabricación	Datos 2022
Capacidad de fabricación total	Múltiples instalaciones de producción global
Volumen de producción anual	Aproximadamente 100,000 dosis de tratamiento

Innovación continua de medicina genética

Alnylam mantiene un Presupuesto de innovación de $ 1.1 mil millones dedicado a avanzar en tecnologías terapéuticas de ARNi.

• Portafolio de patentes: más de 1,200 patentes emitidas a nivel mundial
• Colaboraciones de investigación: 12 asociaciones académicas activas

Cumplimiento regulatorio y procesos de aprobación de medicamentos

Métrico regulatorio	Rendimiento 2022-2023
Aprobaciones de la FDA	3 nuevas solicitudes de drogas presentadas
Presupuesto de cumplimiento regulatorio	$ 85.3 millones

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: recursos clave

Tecnología de plataforma terapéutica RNAi patentada

La tecnología de plataforma RNAi de Alnylam representa un activo intelectual central Con las siguientes características clave:

Métrica de tecnología de plataforma	Valor cuantitativo
Patentes totales de RNAi	Más de 1.200 patentes en todo el mundo
Rango de vencimiento de patentes	2024-2040
I + D Inversión en plataforma	$ 566.7 millones (2022 año fiscal)

Equipos especializados de investigación y desarrollo

Composición de la fuerza laboral de I + D de Alnylam:

• Empleados totales de I + D: 724 (a partir de 2022)
• Investigadores a nivel de doctorado: aproximadamente el 65%
• Áreas de especialización: interferencia de ARN, medicina genética, terapéutica dirigida al hígado

Instalaciones avanzadas de investigación científica

Ubicación de la instalación	Tamaño	Enfoque de investigación
Cambridge, Massachusetts	185,000 sq. Ft. Campus de investigación	Sede y centro de I + D primario
San Francisco, California	Investigación adicional	Capacidades de investigación complementarias

Cartera de propiedades intelectuales

Los activos IP integrales incluyen:

• Familia de patentes: más de 1,200 patentes globales
• Protección de patentes: cubierta de plataformas de tecnología RNAi
• Áreas terapéuticas clave: medicamentos genéticos, enfermedades hepáticas

Fuerte capital financiero y respaldo de inversión

Métrica financiera	Valor 2022
Ingresos totales	$ 1.01 mil millones
Efectivo e inversiones	$ 2.3 mil millones
Gasto de I + D	$ 566.7 millones
Capitalización de mercado	Aproximadamente $ 9.8 mil millones (a partir de enero de 2024)

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocio: propuestas de valor

Medicina genética innovadora dirigida a enfermedades raras

Alnylam Pharmaceuticals se centra en la terapéutica de ARNi con 4 medicamentos aprobados por la FDA a partir de 2024:

Medicamento	Indicación	Año de aprobación de la FDA
Oncattro	Polineuropatía en Hattr	2018
Givlaari	Porfiria hepática aguda	2019
Oxlumo	Hiperoxaluria primaria tipo 1	2020
Amvuttra	Polineuropatía Hattr	2022

Posibles tratamientos innovadores

Inversión total de I + D en 2023: $ 1.1 mil millones

• Desarrollo de tratamientos para enfermedades genéticas raras
• Condiciones de orientación con terapias limitadas o no actuales
• Centrarse en los trastornos genéticos que afectan a las pequeñas poblaciones de pacientes

Enfoque de medicina de precisión

Plataforma de tecnología RNAi dirigida a secuencias genéticas específicas

Métrica de tecnología	Estado 2024
Programas clínicos activos	15
Candidatos de tuberías	25
Cartera de patentes	Más de 700 patentes

Terapias dirigidas

2023 Ingresos del producto: $ 1.3 mil millones

• Intervenciones genéticas de precisión
• Minimizar los efectos fuera del objetivo
• Enfoques de tratamiento personalizados

Abordar las necesidades médicas no satisfechas

Enfoque del mercado de enfermedades raras con altas necesidades médicas no satisfechas

Categoría de enfermedades	Pacientes globales estimados
Trastornos genéticos raros	350 millones en todo el mundo
Condiciones no tratables	~ 50% de enfermedades raras

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: relaciones con los clientes

Compromiso directo con profesionales médicos

Alnylam Pharmaceuticals mantiene la participación directa a través de:

• Alcance dirigido a más de más de 3,500 especialistas en enfermedades raras
• Interacciones de enlace científico médico uno a uno
• Plataformas de comunicación de datos clínicos personalizados

Categoría de compromiso	Volumen de interacción anual	Grupo especializado en Target
Comunicaciones médicas directas	4.200 interacciones	Especialistas en hematología/neurología
Colaboraciones de investigación clínica	87 asociaciones activas	Centros médicos académicos

Programas de apoyo al paciente

Los servicios integrales de apoyo al paciente incluyen:

• Programa de asistencia financiera que cubre $ 15,000+ por paciente anualmente
• Línea de ayuda de apoyo al paciente 24/7
• Servicios de asesoramiento genético

Conferencia científica y participación del simposio médico

Métricas de compromiso de la conferencia anual:

Tipo de conferencia	Presentaciones anuales	Alcance de los asistentes
Conferencias médicas internacionales	42 presentaciones	Más de 12,500 profesionales de la salud
Simposios de enfermedades raras	18 sesiones especializadas	5.700 médicos especializados

Plataformas de información de salud digital

Estadísticas de compromiso digital:

• Tráfico del sitio web: 275,000 visitantes únicos anualmente
• Recursos de educación de pacientes digitales: 47 guías integrales
• Portal profesional médico en línea: 2.300 usuarios registrados

Servicios de consulta de tratamiento personalizado

Capacidades de servicio de consulta:

Tipo de consulta	Volumen anual	Duración promedio
Consulta genética	1.200 consultas individuales	90 minutos por sesión
Planificación del tratamiento	890 planes personalizados	120 minutos por consulta

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: canales

Ventas directas a instituciones de atención médica

En 2023, Alnylam informó que el equipo de ventas directas dirigida a 150 instituciones de salud clave en los Estados Unidos. La fuerza de ventas directas de la compañía se centró en centros de tratamiento especializados para enfermedades genéticas raras.

Tipo de canal de ventas	Número de instituciones específicas	Cobertura geográfica
Ventas directas a hospitales	150	Estados Unidos
Centros de tratamiento especializados	85	Principales áreas metropolitanas

Distribuidores farmacéuticos especializados

Alnylam colabora con 12 distribuidores farmacéuticos especializados para la distribución de productos.

• AmerisourceBergen
• Salud cardinal
• McKesson Corporation

Plataformas de información médica en línea

La compañía utiliza 7 plataformas principales de información médica en línea para la comunicación de productos y la difusión de investigación.

Nombre de la plataforma	Visitantes únicos mensuales	Compromiso profesional de la salud
Medidor	1,200,000	Alto
Pubmed	800,000	Muy alto

Presentaciones de conferencia médica

En 2023, Alnylam participó en 18 Conferencias médicas internacionales, Presentación de la investigación sobre la terapéutica de interferencia de ARN.

Publicaciones de investigación clínica

La compañía publicó 22 artículos de investigación revisados ​​por pares en 2023 en revistas como Nature Medicine y Lancet.

Categoría de publicación	Número de publicaciones	Rango de factores de impacto
Revistas revisadas por pares	22	5.2 - 35.7

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocios: segmentos de clientes

Pacientes con enfermedades raras

Alnylam Pharmaceuticals se dirige a pacientes con trastornos genéticos raros específicos, centrándose en las siguientes poblaciones de pacientes:

Enfermedad rara	Población de pacientes estimada	Tratamiento dirigido
Attr amiloidosis hereditaria	50,000 pacientes a nivel mundial	Onpattro (patisiran)
Hiperoxaluria primaria tipo 1	1,000-2,000 pacientes en todo el mundo	Lumasirán
Porfiria hepática aguda	5,000-10,000 pacientes en EE. UU./UE	Givlaari (Givosiran)

Profesionales de la salud

Alnylam se dirige a profesionales médicos especializados:

• Especialistas en desorden genético
• Neurólogos
• Hepatólogos
• Centros de tratamiento de enfermedades raras

Investigar hospitales y centros médicos

Los segmentos de clientes institucionales clave incluyen:

Tipo de institución	Número de clientes potenciales
Centros médicos académicos	200+ en América del Norte
Centros especializados de investigación de enfermedades raras	50+ a nivel mundial
Centros afiliados a los Institutos Nacionales de Salud (NIH)	Más de 25 instituciones de investigación

Especialistas en desorden genético

Desglose de segmento de clientes de médico especializado:

• Genetistas clínicos: 3.500 en Estados Unidos
• Especialistas en enfermedades raras: 1.200 en todo el mundo
• Especialistas neurogenéticos: 800 a nivel mundial

Investigadores farmacéuticos

Detalles del segmento de clientes centrado en la investigación:

Categoría de investigación	Número de clientes potenciales
Investigadores de tecnología RNAi	500+ en todo el mundo
Equipos de investigación de medicina genética	Más de 250 grupos de investigación institucional
Departamentos de I + D	Más de 100 compañías farmacéuticas importantes

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

En 2023, Alnylam Pharmaceuticals reportó gastos de I + D de $ 822.5 millones. La plataforma terapéutica RNAi de la compañía requiere una inversión continua significativa en el descubrimiento y el desarrollo de medicamentos novedosos.

Año	Gastos de I + D ($ M)	Porcentaje de ingresos
2022	$ 747.3 millones	76.4%
2023	$ 822.5 millones	79.2%

Inversiones de ensayos clínicos

Los gastos de ensayos clínicos para Alnylam en 2023 fueron de aproximadamente $ 413.2 millones, que cubren múltiples programas de tuberías en varias áreas terapéuticas.

• Ensayos continuos de fase 2 y fase 3 para enfermedades genéticas raras
• Estudios de investigación en trastornos cardiometabólicos y hepáticos
• Iniciativas globales de investigación clínica multi-centro

Infraestructura de fabricación

Los gastos de capital para instalaciones de fabricación y plataformas de tecnología en 2023 totalizaron $ 154.6 millones. La compañía mantiene capacidades de fabricación especializadas para la terapéutica RNAi.

Ubicación de fabricación	Inversión ($ m)	Capacidad
Instalación de Massachusetts	$ 87.3 millones	Producción de ARNi a gran escala
Instalaciones internacionales	$ 67.3 millones	Expansión global de la cadena de suministro

Costos de cumplimiento regulatorio

Los asuntos regulatorios y los gastos de cumplimiento para 2023 se estimaron en $ 62.4 millones, que cubren las presentaciones regulatorias globales y el mantenimiento de las aprobaciones de medicamentos existentes.

Gastos de marketing y comunicación científica

Los gastos de marketing y comunicación científica en 2023 alcanzaron $ 103.7 millones, centrados en la participación profesional especializada en la salud y la conciencia de enfermedades raras.

Categoría de marketing	Gasto ($ M)	Enfoque principal
Conferencias científicas	$ 24.5 millones	Alcance profesional médico
Comunicación digital	$ 42.3 millones	Educación para pacientes y médicos
Compromiso de ventas directas	$ 36.9 millones	Interacciones de proveedor de atención médica dirigida

Alnylam Pharmaceuticals, Inc. (ALNY) - Modelo de negocios: flujos de ingresos

Venta de productos farmacéuticos

Alnylam Pharmaceuticals generó ingresos totales de $ 1.37 mil millones para el año fiscal 2023. Las ventas clave de productos incluyen:

Producto	Ingresos anuales (2023)
Oncattro	$ 508 millones
Givlaari	$ 252 millones
Oxlumo	$ 186 millones

Plataformas de tecnología de licencia

La licencia de tecnología RNAi de Alnylam generó ingresos a través de asociaciones estratégicas.

• Colaboraciones con Regeneron Pharmaceuticals
• Acuerdos de licencia con Biotecnología VIR
• Tarifas de licencia de plataforma de tecnología estimadas en $ 75-100 millones anuales

Acuerdos de colaboración de investigación

Los ingresos por colaboración de investigación para 2023 totalizaron aproximadamente $ 212 millones, incluidas las asociaciones con:

• Sanofi
• Regenerón
• Takeda Pharmaceutical

Subvenciones gubernamentales e institucionales

Los ingresos por subvenciones para 2023 fueron de aproximadamente $ 45 millones, obtenidos de:

• Institutos Nacionales de Salud (NIH)
• Financiación del Departamento de Investigación del Departamento de Defensa
• Institución de investigación académica Subvenciones

Pagos potenciales de hitos de las asociaciones

Potencial de pago de hitos en 2023-2024:

Pareja	Pagos potenciales de hitos
Regenerón	Hasta $ 100 millones
Sanofi	Hasta $ 75 millones
Vir biotecnología	Hasta $ 50 millones

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.