Alnylam Pharmaceuticals, Inc. (Alny): Modelo de Negócios Canvas [Jan-2025 Atualizado]

No mundo de ponta da medicina genética, a Alnylam Pharmaceuticals surge como uma força revolucionária, transformando como abordamos o tratamento de doenças raras através de sua inovadora tecnologia de interferência de RNA (RNAi). Ao elaborar meticulosamente um modelo de negócios sofisticado que preenche a inovação científica com parcerias estratégicas, o Alnylam se posicionou na vanguarda da medicina de precisão, oferecendo esperança a pacientes com distúrbios genéticos anteriormente incomparáveis ​​por meio de terapias direcionadas que prometem redefinir a intervenção médica.

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negócios: Parcerias -chave

Colaborações estratégicas com empresas farmacêuticas

A Alnylam estabeleceu parcerias críticas com várias empresas farmacêuticas:

Parceiro	Detalhes da parceria	Ano estabelecido
Regeneron Pharmaceuticals	Colaboração para o desenvolvimento da RNAi Therapeutics	2018
Vir Biotecnology	CoVID-19 e pesquisa terapêutica de doenças infecciosas	2020
Sanofi	Desenvolvimento terapêutico de doenças raras	2014

Parcerias de pesquisa

Alnylam mantém extensas colaborações acadêmicas e de pesquisa médica:

• Hospital Geral de Massachusetts - Pesquisa em Doenças Neurológicas
• Universidade de Stanford - Desenvolvimento de Tecnologia RNAi
• Faculdade de Medicina de Harvard - Pesquisa de Doenças Genéticas
• Universidade da Pensilvânia - Tecnologia de silenciamento de genes

Acordos de licenciamento

As principais parcerias de licenciamento incluem:

Tecnologia/plataforma	Parceiro de licenciamento	Termos financeiros
RNAi Plataforma terapêutica	Medtronic	Pagamento antecipado de US $ 35 milhões
Tecnologia de silenciamento de genes	Novartis	Colaboração inicial de US $ 100 milhões

Financiamento de pesquisa do governo e sem fins lucrativos

Parcerias significativas de financiamento de pesquisa:

• Institutos Nacionais de Saúde (NIH) - US $ 24,5 milhões para pesquisa de doenças raras
• Conta & Melinda Gates Foundation - US $ 50 milhões Global Health Partnership
• Agência de Projetos de Pesquisa Avançada de Defesa (DARPA) - US $ 17,3 milhões de financiamento de pesquisa de doenças infecciosas

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negócios: Atividades -chave

Interferência de RNA (RNAi) Pesquisa e Desenvolvimento Terapêutico de Drogas

A Alnylam investiu US $ 679,4 milhões em despesas de pesquisa e desenvolvimento em 2022. A Companhia mantém um oleoduto robusto da RNAi Therapeutics visando doenças genéticas raras.

Área de foco de pesquisa	Número de programas ativos
Doenças genéticas raras	6 programas de estágio clínico
Doenças relacionadas ao fígado	4 programas de desenvolvimento avançado

Ensaios clínicos para doenças genéticas e hepáticas raras

A Alnylam conduziu 15 ensaios clínicos em andamento em várias áreas terapêuticas em 2023.

• Ensaios de fase 1: 5 estudos ativos
• Ensaios de fase 2: 6 estudos ativos
• Fase 3 Ensaios: 4 estudos ativos

Fabricação de produtos farmacêuticos

Métrica de fabricação	2022 dados
Capacidade total de fabricação	Várias instalações de produção global
Volume anual de produção	Aproximadamente 100.000 doses de tratamento

Inovação de medicina genética em andamento

Alnylam mantém a Orçamento de inovação de US $ 1,1 bilhão dedicado ao avanço das tecnologias terapêuticas da RNAi.

• Portfólio de patentes: 1.200 mais de patentes emitidas globalmente
• Colaborações de pesquisa: 12 parcerias acadêmicas ativas

Processos de conformidade regulatória e aprovação de medicamentos

Métrica regulatória	2022-2023 Desempenho
Aprovações da FDA	3 novas solicitações de drogas enviadas
Orçamento de conformidade regulatória	US $ 85,3 milhões

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negócios: Recursos -chave

Tecnologia de plataforma terapêutica RNAi proprietária

A tecnologia de plataforma RNAi de Alnylam representa um Principal de ativo intelectual Com as seguintes características -chave:

Métrica de tecnologia da plataforma	Valor quantitativo
Total de patentes RNAi	Mais de 1.200 patentes em todo o mundo
Faixa de expiração da patente	2024-2040
Investimento em P&D em plataforma	US $ 566,7 milhões (2022 ano fiscal)

Equipes especializadas de pesquisa e desenvolvimento

Composição da força de trabalho de R&D de Alnylam:

• Funcionários totais de P&D: 724 (a partir de 2022)
• Pesquisadores no nível de doutorado: aproximadamente 65%
• Áreas de especialização: interferência de RNA, medicina genética, terapêutica direcionada ao fígado

Instalações de pesquisa científica avançadas

Localização da instalação	Tamanho	Foco na pesquisa
Cambridge, Massachusetts	185.000 pés quadrados campus de pesquisa	Sede e centro de P&D primário
São Francisco, Califórnia	Instalação de pesquisa adicional	Recursos de pesquisa complementares

Portfólio de propriedade intelectual

Os ativos IP abrangentes incluem:

• Família de patentes: 1.200 mais de patentes globais
• Proteção de patentes: cobrindo plataformas de tecnologia RNAi
• Áreas terapêuticas -chave: medicamentos genéticos, doenças hepáticas

Forte capital financeiro e apoio de investimentos

Métrica financeira	2022 Valor
Receita total	US $ 1,01 bilhão
Dinheiro e investimentos	US $ 2,3 bilhões
Despesas de P&D	US $ 566,7 milhões
Capitalização de mercado	Aproximadamente US $ 9,8 bilhões (em janeiro de 2024)

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de Negócios: Proposições de Valor

Medicina genética inovadora visando doenças raras

A Alnylam Pharmaceuticals se concentra na RNAi Therapeutics com 4 medicamentos aprovados pela FDA a partir de 2024:

Medicamento	Indicação	Ano de aprovação da FDA
Onpattro	Polyneuropatia em Hattr	2018
Givlaari	Porfiria hepática aguda	2019
Oxlumo	Hiperoxalúria primária tipo 1	2020
Amvuttra	Hattr Polyneuropatia	2022

Potenciais tratamentos inovadores

Investimento total de P&D em 2023: US $ 1,1 bilhão

• Desenvolvimento de tratamentos para doenças genéticas raras
• Condições de direcionamento com terapias limitadas ou não atuais
• Focando em distúrbios genéticos que afetam pequenas populações de pacientes

Abordagem de medicina de precisão

Plataforma de tecnologia RNAi direcionando sequências genéticas específicas

Métrica de tecnologia	2024 Status
Programas clínicos ativos	15
Candidatos a pipeline	25
Portfólio de patentes	Mais de 700 patentes

Terapias direcionadas

2023 Receita do produto: US $ 1,3 bilhão

• Intervenções genéticas de precisão
• Minimizar os efeitos fora do alvo
• Abordagens de tratamento personalizadas

Atendendo às necessidades médicas não atendidas

Foco no mercado de doenças raras com altas necessidades médicas não atendidas

Categoria de doença	Pacientes globais estimados
Distúrbios genéticos raros	350 milhões em todo o mundo
Condições não tratadas	~ 50% das doenças raras

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com profissionais médicos

Alnylam Pharmaceuticals mantém o envolvimento direto por meio de:

• Extensão direcionada para mais de 3.500 especialistas em doenças raras
• Interações com ligação científica médica individual
• Plataformas personalizadas de comunicação de dados clínicos

Categoria de engajamento	Volume anual de interação	Grupo Especialista Target
Comunicações médicas diretas	4.200 interações	Especialistas em Hematologia/Neurologia
Colaborações de pesquisa clínica	87 parcerias ativas	Centros Médicos Acadêmicos

Programas de apoio ao paciente

Serviços abrangentes de apoio ao paciente incluem:

• Programa de Assistência Financeira, cobrindo US $ 15.000+ por paciente anualmente
• Helpline de apoio ao paciente 24 horas por dia, 7 dias por semana
• Serviços de aconselhamento genético

Conferência Científica e Participação do Simpósio Médico

Métricas anuais de engajamento da conferência:

Tipo de conferência	Apresentações anuais	Retenção dos participantes
Conferências médicas internacionais	42 apresentações	12.500 mais de profissionais de saúde
Simpósios de doenças raras	18 sessões especializadas	5.700 médicos especializados

Plataformas de informações de saúde digital

Estatísticas de engajamento digital:

• Tráfego do site: 275.000 visitantes únicos anualmente
• Recursos de educação digital do paciente: 47 guias abrangentes
• Portal de Profissional Médico Online: 2.300 usuários registrados

Serviços de consulta de tratamento personalizado

Recursos de serviço de consulta:

Tipo de consulta	Volume anual	Duração média
Consulta genética	1.200 consultas individuais	90 minutos por sessão
Planejamento de tratamento	890 planos personalizados	120 minutos por consulta

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de Negócios: Canais

Vendas diretas para instituições de saúde

Em 2023, Alnylam relatou a equipe de vendas direta direcionada a 150 instituições de saúde importantes nos Estados Unidos. A força de vendas direta da empresa se concentrou em centros de tratamento especializados para doenças genéticas raras.

Tipo de canal de vendas	Número de instituições direcionadas	Cobertura geográfica
Vendas diretas para hospitais	150	Estados Unidos
Centros de tratamento especializados	85	Principais áreas metropolitanas

Distribuidores farmacêuticos especializados

Alnylam colabora com 12 distribuidores farmacêuticos especializados para distribuição de produtos.

• Amerisourcebergen
• Cardinal Health
• McKesson Corporation

Plataformas de informações médicas online

A empresa utiliza 7 plataformas principais de informações médicas on -line para comunicação de produtos e disseminação de pesquisas.

Nome da plataforma	Visitantes únicos mensais	Engajamento profissional de saúde
Medscape	1,200,000	Alto
PubMed	800,000	Muito alto

Apresentações da conferência médica

Em 2023, Alnylam participou de 18 conferências médicas internacionais, apresentando pesquisas sobre terapêutica de interferência de RNA.

Publicações de pesquisa clínica

A empresa publicou 22 artigos de pesquisa revisados ​​por pares em 2023 em periódicos como Nature Medicine e The Lancet.

Categoria de publicação	Número de publicações	Faixa de fatores de impacto
Revistas revisadas por pares	22	5.2 - 35.7

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

Pacientes com doenças raras

Alnylam Pharmaceuticals tem como alvo pacientes com distúrbios genéticos raros específicos, concentrando -se nas seguintes populações de pacientes:

Doença rara	População estimada de pacientes	Tratamento direcionado
Amiloidose do ator hereditário	50.000 pacientes globalmente	Onpattro (Patisiran)
Hiperoxalúria primária tipo 1	1.000-2.000 pacientes em todo o mundo	Lumasiran
Porfiria hepática aguda	5.000 a 10.000 pacientes nos EUA/UE	Givlaari (Givosiran)

Profissionais de saúde

Alnylam tem como alvo profissionais médicos especializados:

• Especialistas em Transtorno Genético
• Neurologistas
• Hepatologistas
• Centros de tratamento de doenças raras

Hospitais de pesquisa e centros médicos

Os principais segmentos de clientes institucionais incluem:

Tipo de instituição	Número de clientes em potencial
Centros Médicos Acadêmicos	Mais de 200 na América do Norte
Centros de pesquisa de doenças raras especializadas	Mais de 50 globalmente
Institutos Nacionais de Saúde (NIH) Centros Afiliados	Mais de 25 instituições de pesquisa

Especialistas em Transtorno Genético

Remutação especializada do segmento de clientes médicos:

• Geneticistas clínicos: 3.500 nos Estados Unidos
• Especialistas em doenças raras: 1.200 em todo o mundo
• Especialistas neurogenéticos: 800 globalmente

Pesquisadores farmacêuticos

Detalhes do segmento de clientes focados na pesquisa:

Categoria de pesquisa	Número de clientes em potencial
Pesquisadores de tecnologia da RNAi	500+ em todo o mundo
Equipes de pesquisa de medicina genética	Mais de 250 grupos de pesquisa institucional
Departamentos de P&D farmacêuticos	Mais de 100 grandes empresas farmacêuticas

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Em 2023, a Alnylam Pharmaceuticals registrou despesas de P&D de US $ 822,5 milhões. A plataforma terapêutica RNAi da empresa requer investimento contínuo significativo em uma nova descoberta e desenvolvimento de medicamentos.

Ano	Despesas de P&D ($ M)	Porcentagem de receita
2022	US $ 747,3 milhões	76.4%
2023	US $ 822,5 milhões	79.2%

Investimentos de ensaios clínicos

Os gastos com ensaios clínicos para Alnylam em 2023 foram de aproximadamente US $ 413,2 milhões, cobrindo vários programas de pipeline em várias áreas terapêuticas.

• Ensaios de Fase 2 e 3 em andamento para doenças genéticas raras
• Estudos de investigação em distúrbios cardiometabólicos e hepáticos
• Iniciativas globais de pesquisa clínica multicêntrica

Infraestrutura de fabricação

As despesas de capital para instalações de fabricação e plataformas de tecnologia em 2023 totalizaram US $ 154,6 milhões. A empresa mantém recursos especializados de fabricação para a RNAi Therapeutics.

Local de fabricação	Investimento ($ m)	Capacidade
Instalação de Massachusetts	US $ 87,3 milhões	Produção de RNAi em larga escala
Instalações internacionais	US $ 67,3 milhões	Expansão global da cadeia de suprimentos

Custos de conformidade regulatória

Os assuntos regulatórios e as despesas de conformidade para 2023 foram estimados em US $ 62,4 milhões, cobrindo envios regulatórios globais e manutenção das aprovações existentes de medicamentos.

Despesas de marketing e comunicação científica

Os gastos com marketing e comunicação científica em 2023 atingiram US $ 103,7 milhões, focados em engajamento profissional especializado em saúde e conscientização sobre doenças raras.

Categoria de marketing	Despesa ($ m)	Foco primário
Conferências científicas	US $ 24,5 milhões	Extenção profissional médica
Comunicação digital	US $ 42,3 milhões	Educação para pacientes e médicos
Engajamento direto de vendas	US $ 36,9 milhões	Interações direcionadas para provedores de saúde

Alnylam Pharmaceuticals, Inc. (Alny) - Modelo de negócios: fluxos de receita

Vendas farmacêuticas de produtos

A Alnylam Pharmaceuticals gerou receita total de US $ 1,37 bilhão para o ano fiscal de 2023. As principais vendas de produtos incluem:

Produto	Receita anual (2023)
Onpattro	US $ 508 milhões
Givlaari	US $ 252 milhões
Oxlumo	US $ 186 milhões

Plataformas de tecnologia de licenciamento

O licenciamento de tecnologia RNAi da Alnylam gerou receita por meio de parcerias estratégicas.

• Colaborações com Regeneron Pharmaceuticals
• Acordos de licenciamento com Vir Biotechnology
• Taxas de licenciamento da plataforma de tecnologia estimadas em US $ 75-100 milhões anualmente

Acordos de colaboração de pesquisa

As receitas de colaboração de pesquisa para 2023 totalizaram aproximadamente US $ 212 milhões, incluindo parcerias com:

• Sanofi
• Regeneron
• Takeda Pharmaceutical

Subsídios governamentais e institucionais

As receitas de concessão de 2023 foram de aproximadamente US $ 45 milhões, provenientes de:

• Institutos Nacionais de Saúde (NIH)
• Financiamento do Departamento de Pesquisa de Defesa
• Graças de instituição de pesquisa acadêmica

Potenciais pagamentos marcantes de parcerias

Milestone Payment Potendent em 2023-2024:

Parceiro	Potenciais pagamentos marcantes
Regeneron	Até US $ 100 milhões
Sanofi	Até US $ 75 milhões
Vir Biotecnology	Até US $ 50 milhões

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.