Stoke Therapeutics, Inc. (Stok): Modelo de negócios Canvas [Jan-2025 Atualizado]

No domínio de ponta da medicina genética, a Stoke Therapeutics, Inc. (Stok) surge como uma força pioneira, revolucionando nossa abordagem a distúrbios genéticos raros e desafiadores. Ao alavancar sua inovadora plataforma de edição e regulamentação de RNA direcionada (Tango), a empresa está pronta para transformar a medicina de precisão, oferecendo esperança a pacientes com condições genéticas anteriormente intratáveis. Essa lona abrangente do modelo de negócios revela o plano estratégico de um inovador de biotecnologia comprometido em ultrapassar os limites da descoberta científica e da intervenção terapêutica.

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: Parcerias -chave

Colaborações com instituições de pesquisa acadêmica

A Stoke Therapeutics estabeleceu as principais parcerias de pesquisa com as seguintes instituições acadêmicas:

Instituição	Foco na pesquisa	Ano de parceria
Faculdade de Medicina da Universidade de Massachusetts	RNA Therapeutics para distúrbios genéticos	2019
Escola de Medicina de Harvard	Pesquisa neurológica de doenças genéticas	2020

Parcerias estratégicas com empresas farmacêuticas

A Stoke Therapeutics desenvolveu colaborações estratégicas com empresas farmacêuticas:

• Biogen Inc. - Colaboração para terapias de doenças neurológicas genéticas
• VERTEX Pharmaceuticals - Parceria para Pesquisa de Transtorno Genético Raro

Pesquisa genética e redes de pesquisa de doenças raras

Stoke Therapeutics participa das seguintes redes de pesquisa:

Rede	Área de foco	Status de associação
Rede de Pesquisa Clínica de Doenças Raras (RDCRN)	Distúrbios genéticos raros	Membro ativo
Organização Nacional para Distúrbios Raros (Nord)	Advocacia e pesquisa de doenças raras	Parceiro colaborativo

Potenciais parcerias do local do ensaio clínico

A Stoke Therapeutics estabeleceu parcerias de ensaios clínicos com:

• Hospital Geral de Massachusetts
• Stanford Children's Health
• Centro Médico de Hospital Infantil de Cincinnati

Pesquisa total e investimentos em parceria em 2023: US $ 24,3 milhões

Número de colaborações de pesquisa ativa: 7

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: Atividades -chave

Desenvolvimento terapêutico direcionado a RNA

A partir do quarto trimestre 2023, a Stoke Therapeutics se concentra no desenvolvimento de terapias direcionadas a RNA direcionadas a doenças genéticas específicas.

Foco de desenvolvimento	Status atual	Doenças alvo
RNA Therapeutics	Estágio clínico avançado	Distúrbios neurológicos genéticos
Abordagem de medicina de precisão	Vários programas em pipeline	Condições genéticas raras

Pesquisa pré -clínica e clínica

O investimento em pesquisa para 2023 totalizou US $ 83,4 milhões dedicados a atividades de pesquisa pré -clínica e clínica.

• Ensaios clínicos em andamento para STK-001 na síndrome de Dravet
• Pesquisa pré -clínica para alvos adicionais de doenças genéticas
• Colaboração com instituições de pesquisa acadêmica

Investigação do mecanismo de doença genética

A Stoke Therapeutics investiga mecanismos genéticos específicos com uma abordagem de pesquisa focada.

Área de pesquisa	Número de metas identificadas	Investimento em pesquisa
Análise do mecanismo genético	12 alvos genéticos identificados	US $ 24,7 milhões em 2023

Plataforma de edição e regulamentação de RNA direcionadas proprietárias (Tango)

A plataforma de tango representa a inovação tecnológica central para o desenvolvimento terapêutico de RNA.

• Tecnologia de edição de RNA proprietária
• Aplicação potencial em vários distúrbios genéticos
• Portfólio de patentes Proteger a tecnologia da plataforma

Processos de descoberta e desenvolvimento de medicamentos

O pipeline de desenvolvimento de medicamentos demonstra abordagem estratégica da inovação terapêutica.

Estágio de desenvolvimento	Número de programas	Custo estimado de desenvolvimento
Estágio pré -clínico	4 programas	US $ 42,3 milhões
Estágio clínico	2 programas	US $ 61,9 milhões

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: Recursos -chave

Plataforma de tecnologia de tango proprietária

A plataforma de tecnologia Tango da Stoke Therapeutics (aumento direcionada da produção de genes nucleares) se concentra no desenvolvimento de terapêuticas antisense de ácido oligonucleico (ASO). A partir de 2024, a plataforma tem como alvo doenças genéticas com mecanismos específicos mediados por RNA.

Métrica de tecnologia	Dados específicos
Aplicações de patentes	17 patentes emitidas a partir do quarto trimestre 2023
Investimento em P&D	US $ 78,3 milhões em 2023

Experiência especializada em pesquisa genética

A empresa mantém uma equipe de pesquisa focada especializada em terapias genéticas.

• Pessoal de pesquisa total: 124 funcionários
• Pesquisadores no nível de doutorado: 63
• Especialistas genéticos: 42

Portfólio de propriedade intelectual

A Stoke Therapeutics mantém uma estratégia de propriedade intelectual robusta.

Categoria IP	Quantidade
Total de famílias de patentes	23
Aplicações de patentes pendentes	8

Instalações de pesquisa e desenvolvimento

Localizado em Bedford, Massachusetts, com aproximadamente 48.000 pés quadrados de laboratório e espaço de pesquisa.

Pessoal científico e médico qualificado

A empresa emprega profissionais especializados com formação científica avançada.

• Total de funcionários: 214 (em dezembro de 2023)
• Pessoal de pesquisa: 124
• Equipe de Desenvolvimento Clínico: 36
• Pessoal de fabricação: 22

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: proposições de valor

Abordagens inovadoras de tratamento de doenças genéticas

A Stoke Therapeutics se concentra no desenvolvimento da terapêutica direcionada a RNA, com ênfase específica em distúrbios genéticos raros. A partir do quarto trimestre 2023, o candidato a produtos principal da empresa, STK-001, tem como alvo a síndrome de Dravet com uma potencial oportunidade de mercado de aproximadamente US $ 500 milhões anualmente.

Área terapêutica	Condição alvo	Tamanho potencial de mercado
Distúrbios genéticos raros	Síndrome de Dravet	US $ 500 milhões
Doenças neurológicas	Epilepsias relacionadas à SCN1A	US $ 350 milhões

Medicina de precisão direcionada a distúrbios genéticos raros

O aumento direcionado da empresa da plataforma de produção de genes nucleares (Tango) da Companhia permite intervenções genéticas precisas.

• Desenvolveu tecnologia terapêutica direcionada a RNA proprietária
• Concentre -se em condições genéticas com opções de tratamento limitadas
• Potencial para abordar os distúrbios genéticos em nível molecular

Potencial para abordar condições genéticas anteriormente intratáveis

A Stoke Therapeutics relatou o pipeline de pesquisa direcionado a vários distúrbios genéticos raros com necessidades médicas não atendidas significativas.

Estágio de pesquisa	Número de programas	Condições direcionadas
Pré -clínico	3-4 programas	Distúrbios neurológicos raros
Desenvolvimento Clínico	1-2 programas	Síndrome de Dravet

Soluções terapêuticas direcionadas a RNA

Em 2023, a Stoke Therapeutics investiu aproximadamente US $ 75 milhões em pesquisa e desenvolvimento para o avanço das tecnologias terapêuticas de RNA.

• Tecnologia proprietária da plataforma de tango
• Abordagem de intervenção genética de precisão
• Potencial para modular a expressão gênica

Intervenções personalizadas de doenças genéticas

A abordagem da empresa permite estratégias terapêuticas direcionadas para mutações genéticas específicas.

Característica da tecnologia	Valor único
Especificidade genética	Direcionamento molecular preciso
Personalização do tratamento	Intervenções genéticas específicas do paciente

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com grupos de defesa do paciente

Em 2024, a Stoke Therapeutics mantém parcerias ativas com 7 organizações de defesa de pacientes com doenças raras focadas em distúrbios genéticos.

Tipo de grupo de defesa	Número de parcerias ativas	Áreas de foco
Grupos de transtornos genéticos raros	7	Condições neurológicas genéticas

Colaboração da comunidade científica

A Stoke Therapeutics colabora com 12 instituições de pesquisa acadêmica e 5 centros de pesquisa farmacêutica globalmente.

• 12 parcerias de pesquisa acadêmica
• 5 Colaborações do Centro de Pesquisa Farmacêutica
• A pesquisa se concentra em terapias genéticas direcionadas a RNA

Comunicação transparente sobre o progresso da pesquisa

A empresa hospeda 4 eventos de comunicação científica anualmente, incluindo conferências de investidores e simpósios de pesquisa.

Tipo de evento de comunicação	Frequência anual	Média do participante
Conferências de investidores	2	150-200 participantes
Simpósios de pesquisa	2	100-150 participantes

Programas de apoio ao paciente e educação

A Stoke Therapeutics fornece programas de suporte direcionados para pacientes inscritos em ensaios clínicos.

• 3 programas de apoio a pacientes dedicados
• Serviços de aconselhamento genético
• Recursos educacionais online

Interações de participantes do ensaio clínico em andamento

A partir de 2024, a Stoke Therapeutics gerencia 3 ensaios clínicos ativos com aproximadamente 85 participantes totais.

Fase de ensaios clínicos	Número de ensaios	Total de participantes
Ensaios clínicos ativos	3	85

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: canais

Comunicação direta de pesquisa médica

A Stoke Therapeutics utiliza canais de comunicação direcionados com líderes e pesquisadores de opinião -chave na medicina genética. Em 2023, a empresa apresentou 18 resumos científicos em principais conferências médicas.

Canal de comunicação	Número de interações
Apresentações diretas de pesquisa	38 apresentações científicas
Engajamento ponto a ponto	52 interações de pesquisa direcionadas

Conferências e apresentações científicas

A empresa participa ativamente de conferências especializadas em medicina genética.

• Sociedade Americana de Gene & Conferência de terapia celular: 12 apresentações
• Doença rara e Simpósio de Medicina Genética: 8 apresentações de palestras
• Conferência Internacional de Distúrbios Neurológicos: 6 Discussões de Pesquisa

Networking da indústria farmacêutica

A Stoke Therapeutics mantém parcerias estratégicas com redes de pesquisa farmacêutica.

Plataforma de rede	Parcerias ativas
Redes de pesquisa farmacêutica	7 colaborações ativas
Consórcio de Inovação da Biotecnologia	4 parcerias estratégicas

Plataformas de relações com investidores

A empresa usa vários canais para comunicação para investidores.

• Chamadas trimestrais de ganhos: 4 por ano
• Conferências de investidores: 6 apresentações anuais
• Plataformas de investidores digitais: atualizações financeiras em tempo real

Publicações científicas digitais

A Stoke Therapeutics publica pesquisas em revistas científicas digitais e impressas.

Tipo de publicação	Número de publicações
Revistas revisadas por pares	15 publicações em 2023
Plataformas de pesquisa on -line	22 submissões de pesquisa digital

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: segmentos de clientes

Pacientes de doenças genéticas raras

População alvo de pacientes: aproximadamente 25 a 30 milhões de indivíduos nos Estados Unidos com doenças genéticas raras.

Categoria de doença	População estimada de pacientes	Valor potencial de mercado
Síndrome de Dravet	1 em 15.700 indivíduos	US $ 250-350 milhões em potencial mercado
Outros distúrbios genéticos raros	5.000 a 10.000 grupos de pacientes únicos	US $ 500 milhões em potencial mercado anual

Populações de pacientes com transtorno neurológico

Concentre -se em condições neurológicas específicas com origens genéticas.

• População de pacientes com epilepsia: 3,4 milhões de pacientes nos Estados Unidos
• Pacientes de epilepsia genética: estimado 30-40% do total de casos de epilepsia
• Mercado de tratamento potencial: US $ 1,2 bilhão até 2025

Comunidades de doenças genéticas pediátricas

Demográfico alvo primário para terapias genéticas de precisão.

Faixa etária	Prevalência de doenças genéticas	Potencial de tratamento
0-18 anos	1 em cada 33 crianças afetadas	Mercado potencial de US $ 750 milhões

Instituições de Pesquisa Médica

Parcerias de pesquisa colaborativa com as principais instituições.

• Principais colaboradores de pesquisa: Harvard Medical School, MIT, Universidade de Stanford
• Financiamento anual de pesquisa: US $ 15-20 milhões em subsídios colaborativos
• Parcerias de pesquisa ativa: 7-9 colaborações institucionais

Parceiros de pesquisa farmacêutica

Colaborações da indústria farmacêutica estratégica.

Tipo de parceiro	Número de parcerias	Valor potencial de colaboração
Grandes empresas farmacêuticas	3-4 parcerias ativas	US $ 50-100 milhões em potencial valor de colaboração
Empresas de pesquisa de biotecnologia	5-6 colaborações em andamento	US $ 25-75 milhões em potencial financiamento de pesquisa

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Stoke Therapeutics registrou despesas de P&D de US $ 127,9 milhões. O foco da pesquisa da empresa se concentra principalmente em doenças genéticas raras, especificamente distúrbios neurológicos.

Ano	Despesas de P&D	Porcentagem do total de despesas operacionais
2022	US $ 106,3 milhões	78.5%
2023	US $ 127,9 milhões	82.1%

Investimentos de ensaios clínicos

Os gastos com ensaios clínicos para a Stoke Therapeutics em 2023 foram de aproximadamente US $ 85,4 milhões, concentrando-se no avanço do seu programa principal Stok-001 para tratamentos genéticos de epilepsia.

• Fase 1/2 ensaios clínicos para distúrbios genéticos raros
• Desenvolvimento clínico em andamento para vários candidatos terapêuticos
• Custo estimado por julgamento, variando entre US $ 10 a 15 milhões

Manutenção da plataforma de tecnologia

Os custos de manutenção da plataforma de tecnologia para 2023 foram estimados em US $ 22,6 milhões, cobrindo a biologia computacional, a infraestrutura de pesquisa de terapia genética e as tecnologias de direcionamento de RNA proprietárias.

Proteção à propriedade intelectual

As despesas de proteção da propriedade intelectual em 2023 totalizaram US $ 5,2 milhões, cobrindo o arquivamento, manutenção e proteção legal de patentes para suas tecnologias de medicina genética.

Categoria IP	Número de patentes	Custo de proteção estimado
Patentes de tecnologia central	28	US $ 3,1 milhões
Patentes de método terapêutico	15	US $ 2,1 milhões

Aquisição de talentos e custos de retenção

As despesas totais de pessoal para 2023 foram de US $ 92,3 milhões, incluindo salários, remuneração baseada em ações e custos de recrutamento.

• Compensação média anual de funcionários: US $ 215.000
• Compensação baseada em ações: US $ 37,6 milhões
• Despesas de recrutamento e treinamento: US $ 8,7 milhões

Stoke Therapeutics, Inc. (Stok) - Modelo de negócios: fluxos de receita

Vendas potenciais de produtos terapêuticos

A partir do quarto trimestre 2023, a Stoke Therapeutics não possui produtos aprovados comercialmente gerando receita direta. A empresa está focada no desenvolvimento de terapêuticas direcionadas a RNA, com o candidato principal STK-001 em ensaios clínicos para a síndrome de Dravet.

Acordos de colaboração de pesquisa

Parceiro	Detalhes do acordo	Valor potencial
Moderna	Colaboração para RNA Therapeutics	Pagamento inicial de US $ 50 milhões

Propriedade intelectual de licenciamento

A Stoke Therapeutics se mantém 14 patentes emitidas e vários pedidos de patentes relacionados à sua plataforma de RNA antisense direcionada (TARA) direcionada.

Concessão de financiamento

• Institutos Nacionais de Saúde (NIH) Subsídios de pesquisa: US $ 2,3 milhões em 2022
• Subsídios de pesquisa de doenças raras: aproximadamente US $ 1,5 milhão anualmente

Potenciais pagamentos marcantes

Parceria	Potenciais pagamentos marcantes	Estágio de desenvolvimento
Colaboração moderna	Até US $ 400 milhões em possíveis pagamentos marcantes	Desenvolvimento pré -clínico e clínico

Receita total para o ano fiscal de 2022: US $ 64,7 milhões, principalmente de acordos de colaboração e subsídios de pesquisa.

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Value Propositions

You're looking at the core promises Stoke Therapeutics, Inc. (STOK) is making to patients and the market with its pipeline, centered on zorevunersen for Dravet syndrome.

Disease-Modifying Therapy: Potential to treat the underlying genetic cause of Dravet syndrome

The value here is moving past symptom management to address the root issue. Dravet syndrome is estimated to affect up to 38,000 people across the U.S., UK, EU-4, and Japan, with about 85% presenting with an SCN1A gene mutation. Zorevunersen is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of the gene.

The clinical evidence supporting this potential disease modification comes from long-term data:

• Substantial and durable reductions in convulsive seizure frequency observed through three years of zorevunersen treatment in OLE studies.
• The Phase 3 EMPEROR study, initiated in mid-2025, is designed to evaluate this primary endpoint: percent change from baseline in major motor seizure frequency.

Controllable Dosing: ASO chronic annuity model offers dose adjustability and reversibility

While specific annuity model financial terms aren't public, the clinical dosing structure in the Phase 3 EMPEROR study gives you a concrete idea of the treatment plan. This is an intrathecal administration regimen, which is a key delivery method detail for this ASO therapy.

The proposed Phase 3 dosing regimen is:

• Two loading doses of 70mg.
• Followed by two maintenance doses of 45mg over a 52-week treatment period.

Cognitive/Behavioral Improvement: Data suggests benefits beyond just seizure reduction

The value proposition extends to the non-seizure comorbidities that severely impact quality of life. The EMPEROR study includes key secondary endpoints focusing on these areas, measured primarily by the Vineland-3 assessment tool.

Data from the open-label extension studies support this secondary benefit:

The OLE data demonstrated continued improvements in cognition and behavior during the 3-year OLE period, extending beyond the initial 9 months of treatment in the Phase 1/2 studies.

Addressing Haploinsufficiency: A platform approach for a large class of genetic diseases

Stoke Therapeutics, Inc. (STOK) uses its proprietary TANGO platform to target diseases caused by autosomal dominant haploinsufficiencies, where a loss of approximately 50% of normal protein expression causes the condition. This platform aims to increase protein production from the remaining healthy gene copy to restore near-normal levels.

The financial backing for pursuing this broad platform potential is substantial as of late 2025. Here's a quick look at the financial health supporting this pipeline development:

Metric	Value (as of 9/30/2025)	Period/Context
Cash, Cash Equivalents, and Marketable Securities	$328.6 million	Anticipated to fund operations to mid-2028
Total Revenue	$10.6 million	Third Quarter 2025
Net Income (Loss)	$51.0 million (Income)	Nine months ended September 30, 2025
R&D Expenses	$37.7 million	Three months ended September 30, 2025
Phase 3 Study Enrollment	More than 20 patients randomized	As of November 4, 2025

The company also has a current ratio of 6.53, showing excellent short-term financial health. Also, the stock delivered a return of 141.7% over the past year as of early December 2025.

Finance: draft Q4 2025 cash burn projection by next Tuesday.

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Customer Relationships

You're building relationships in a space where every patient interaction is critical, especially when dealing with severe genetic diseases like Dravet syndrome. Stoke Therapeutics, Inc. is currently deep in clinical engagement, which forms the backbone of its early customer relationship strategy before a commercial launch.

High-Touch Patient Support: Programs for rare disease patients and caregivers

The relationship focus is intensely high-touch, centered around the ongoing clinical evaluation of zorevunersen. For Dravet syndrome, a rare genetic epilepsy affecting approximately 38,000 patients across major markets, the unmet need is significant, with up to 57% of patients not achieving adequate seizure reduction on current medications. The most intensive relationship data comes from the Open-Label Extension (OLE) studies, which represent long-term patient commitment and support.

The OLE patient engagement numbers as of the May 30, 2025 data cut show this intensive relationship:

Time Point	Patient Count (n=)
OLE Baseline	74
Month 12	66
Month 24	44
Month 36	19

Safety evaluations across all studies involved 81 patients receiving at least one dose of zorevunersen in Phase 1/2a studies and 75 patients in the OLE studies. This level of longitudinal data collection underscores a deep, sustained relationship with participating patients and their caregivers.

Direct Sales Force (Future): Build relationships with US/Canada/Mexico prescribers

Stoke Therapeutics, Inc. is currently focused on clinical development, but the groundwork for future commercial relationships is implied by the need to prepare for market entry. The company is advancing toward a potential regulatory decision by meeting with the FDA by year-end 2025. While specific numbers for a future US/Canada/Mexico direct sales force are not yet public, the strategy centers on building relationships with prescribers once a first-in-class therapy is approved. Honestly, you'd expect the build-out to scale significantly following any positive regulatory feedback.

Key Account Management: Maintain strategic alliances with Biogen and Acadia

For Stoke Therapeutics, Inc., strategic alliances function as critical key accounts, driving both development and revenue recognition. The collaboration with Biogen for zorevunersen rights outside North America and with Acadia for the SYNGAP1 program are central to the business structure. The financial contribution from these relationships was evident in the Q3 2025 results.

Here's the quick math on revenue recognized from these key partners for the three months ended June 30, 2025:

• Revenue from the License and Collaboration Agreement with Acadia: $10.6 million.
• Revenue from the License and Collaboration Agreement with Biogen: $3.2 million.

These partnerships contributed to the year-to-date revenue reaching $183 million as of Q3 2025. Maintaining these relationships involves joint scientific engagement, such as the planned data presentations by Stoke Therapeutics and Biogen at the 2025 American Epilepsy Society (AES) Annual Meeting, taking place December 5-9.

Medical Science Liaisons (MSLs): Educate specialists on the TANGO mechanism

The MSL function is vital for educating specialists on the proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, which uses antisense oligonucleotides (ASOs) to selectively restore protein levels. This educational relationship-building is crucial for establishing the scientific credibility of a potential disease-modifying therapy. The company's focus is on demonstrating the potential for zorevunersen to reduce seizures and improve neurodevelopment, cognition, and behavior beyond what is achieved with standard anti-seizure medicines. While specific MSL deployment numbers aren't available, the scientific communication strategy involves presenting data at major congresses, like the 2025 AES Annual Meeting, to drive specialist understanding of the mechanism. What this estimate hides is the actual size of the MSL team required to cover the target specialist base for Dravet syndrome and the emerging ADOA program.

Finance: draft 13-week cash view by Friday.

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Channels

You're looking at how Stoke Therapeutics, Inc. plans to get zorevunersen, if approved, into the hands of patients, which is a critical part of the whole plan, especially with the Biogen deal in place.

Biogen's Global Network: Commercialization outside of North America

The channel for ex-North America sales is entirely managed by Biogen, leveraging their established global infrastructure for rare disease medicines.

Biogen receives exclusive commercialization rights for zorevunersen in all territories outside the United States, Canada, and Mexico. This arrangement is part of the February 2025 License and Collaboration Agreement. Stoke is eligible for tiered royalties ranging from low double digits to high teens on potential net sales in the Biogen territory.

This split defines the primary commercial channels:

Territory	Commercialization Lead	Financial Consideration for Stoke
United States, Canada, Mexico	Stoke Therapeutics, Inc.	Direct Sales/Distribution
Rest of World	Biogen Inc.	Tiered Royalties (low double digits to high teens)

Stoke's Direct Commercial Team: Sales and distribution in the US, Canada, and Mexico

Stoke Therapeutics retains the exclusive rights to commercialize zorevunersen within the US, Canada, and Mexico. This means building out a direct sales and distribution infrastructure for these key markets.

The company is already preparing for this commercial launch, as evidenced by financial reporting. General and administrative expenses for the three months ended March 31, 2025, were $14.7 million, compared to $10.2 million for the same period in 2024, due in part to an increase in personnel and launch readiness expense.

The target patient population for the Phase 3 EMPEROR study, which informs launch planning, includes children ages 2 to <18 with Dravet syndrome.

Specialty Pharmacies: Distribution of the final ASO product upon approval

For a complex, potentially first-in-class antisense oligonucleotide (ASO) like zorevunersen, distribution will almost certainly flow through specialty pharmacies, which manage high-touch patient support.

The specialty pharmacy landscape in 2025 shows significant concentration and complexity for manufacturers:

• Specialty pharmaceuticals account for more than half of total drug spending in the U.S.
• As of January 2025, 382 unique specialty drugs utilized a manufacturer-defined limited or exclusive network.
• 34% of these drugs had exclusive networks, meaning only one pharmacy dispensed the product.
• Health systems are projected to control 25% of the specialty pharmacy market by 2028.

Stoke Therapeutics will need to select a distribution model-open, limited, or exclusive-to ensure appropriate patient access and adherence support for Dravet syndrome patients.

Key Opinion Leaders (KOLs): Presenting data at medical congresses like AES 2025

Engaging KOLs through data presentation at major medical meetings is a crucial channel for building clinical credibility and driving adoption upon potential approval.

Stoke Therapeutics and Biogen actively presented data to the epilepsy community in late 2025:

• Data presentations for zorevunersen occurred at the 2025 American Epilepsy Society (AES) Annual Meeting.
• The meeting took place December 5-9 in Atlanta, Georgia.
• Data presented included new propensity weighted analyses derived from four years of clinical data from the Phase 1/2a and ongoing Open-Label Extension (OLE) studies.
• Key figures involved in presenting and discussing the findings included Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics, and Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen.

The pivotal Phase 3 EMPEROR study, which is expected to support global regulatory filings, began enrollment in the second quarter of 2025.

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Customer Segments

You're looking at the core patient populations Stoke Therapeutics, Inc. (STOK) is targeting with its RNA-based medicines. For a company at this stage, understanding the size and characteristics of these segments is defintely key to valuing the opportunity.

Dravet Syndrome Patients: Children and adolescents with SCN1A gene mutations

This is the primary focus, where zorevunersen (STK-001) is in a pivotal Phase 3 EMPEROR study. The market size is defined by the estimated number of people living with Dravet syndrome in key geographies. We're talking about up to 38,000 people across the U.S., UK, EU-4, and Japan. The incidence rate suggests one out of every 16,000 babies is born with the condition. A critical factor for this segment is the high level of unmet need; more than 90 percent of these patients still experience seizures even when on the best available anti-seizure medicines. The Phase 3 trial has already randomized more than 20 patients into dosing as of the third quarter of 2025, with enrollment completion anticipated in the second half of 2026. Clinical experience shows some patients have been on treatment for more than three years, with over 600 doses of zorevunersen administered to date.

Here's a quick look at the clinical engagement:

• Phase 3 EMPEROR study enrollment target completion: Second half of 2026.
• Dosing regimen includes two 70mg loading doses followed by two 45mg maintenance doses.
• Safety evaluation has included 81 patients who received at least one dose.

Healthcare Payers/Systems: Insurance companies and government health programs

These entities are the ultimate revenue source, and their willingness to cover a therapy depends heavily on the clinical and financial profile. Stoke Therapeutics, Inc. is currently pre-revenue from product sales, as evidenced by their Q3 2025 revenue of $10.6 million, which was driven by collaboration funding from partners like Biogen. Year-to-date revenue through September 30, 2025, reached $183.0 million. The company posted a net loss of $38.3 million for Q3 2025. However, the balance sheet provides significant runway; as of September 30, 2025, the cash position stood at $328.6 million, expected to fund operations to mid-2028. For rare disease products like those Stoke is developing, the reimbursement strategy must account for the small market size, meaning the relative price and reimbursement level must be higher to be commercially viable.

Rare Disease Specialists: Neurologists and epileptologists treating severe genetic epilepsies

These specialists are the gatekeepers who interpret the clinical data and ultimately prescribe the medicine. The customer segment is defined by their engagement with the data supporting zorevunersen. For instance, data was presented at the 2025 American Epilepsy Society (AES) Annual Meeting. The clinical program is also engaging with the FDA under Breakthrough Therapy Designation before year-end 2025. For the ADOA pipeline, STK-002 data was presented at the 2025 American Academy of Ophthalmology (AAO) Annual Meeting.

Future Pipeline Patients: Individuals with ADOA (STK-002) and SYNGAP1 syndrome

Stoke Therapeutics, Inc. is expanding its focus to other genetic haploinsufficiency diseases. For Autosomal Dominant Optic Atrophy (ADOA), the target population is estimated at approximately 13,000 people across the U.S., UK, EU-4, and Denmark. The Phase 1 OSPREY study for STK-002 is investigating intravitreal injection doses of 0.1, 0.3, 0.5, and 0.7 mg/eye. The natural history study, FALCON, enrolled 47 patients. This ADOA population faces significant vision loss, with up to 46% registered as legally blind.

For SYNGAP1 syndrome, the patient census known to advocacy groups stood at 1,675 patients worldwide as of Q3 2025. This program is earlier, still in preclinical research, with lead optimization to identify a clinical candidate planned for 2026.

The patient numbers for the pipeline look like this:

Indication	Estimated Population (Target Markets)	Program Status (Late 2025)
Dravet Syndrome (Zorevunersen)	Up to 38,000	Phase 3 EMPEROR underway
ADOA (STK-002)	Approximately 13,000	Phase 1 OSPREY underway
SYNGAP1 Syndrome	1,675 known patients globally	Preclinical research; clinical candidate targeted for 2026

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Cost Structure

You're looking at where Stoke Therapeutics, Inc. is spending its cash to push zorevunersen through late-stage development and prepare for potential commercialization. For a clinical-stage biotech, the cost structure is dominated by science and trials, so let's break down the big buckets based on the nine months ended September 30, 2025, data.

Research and Development (R&D)

Research and Development is the engine room, and it's expensive. For the nine months ended September 30, 2025, Stoke Therapeutics reported R&D expenses of $96.2 million. This is a clear step up from the $65.7 million spent in the same period in 2024. That increase of $30.5 million is directly tied to advancing zorevunersen and growing the team to support that advancement. Honestly, this is the cost of chasing a first-in-class therapy.

The major drivers behind this R&D spend include:

• Advancement of zorevunersen activities and personnel.
• Costs associated with the global Phase 3 EMPEROR study initiation.
• Activities supporting the STK-002 program for Autosomal Dominant Optic Atrophy (ADOA).
• Lead optimization work for the SYNGAP1 program candidate identification planned for 2026.

Clinical Trial Expenses

The most significant near-term R&D outlay is the global Phase 3 EMPEROR study for zorevunersen in Dravet syndrome. Stoke Therapeutics and Biogen are heavily invested here. The study was on track to initiate in the second quarter of 2025. This is a pivotal, global, 52-week, randomized, sham-controlled trial. The cost of running a global trial of this scale, especially one involving sham control, is substantial, and it's a major component of the R&D spend.

Sales, General, and Administrative (SG&A)

SG&A costs reflect the overhead needed to run the company and prepare for a potential future launch, even while still clinical-stage. For the nine months ended September 30, 2025, SG&A expenses were $45.9 million. This compares to $36.0 million in the same nine-month period of 2024. The increase of $9.9 million is attributed to personnel growth and, importantly, launch readiness expenses. That's the cost of getting the infrastructure in place before you even know if you have a product to sell.

Here's a quick look at the key operating expense data for the nine months ended September 30, 2025:

Cost Category	Amount (Nine Months Ended Sept 30, 2025)	Comparison to Prior Year (Nine Months)
Research and Development (R&D) Expenses	$96.2 million	Increased from $65.7 million in 2024
Sales, General and Administrative (SG&A) Expenses	$45.9 million	Increased from $36.0 million in 2024
Net Loss	$78.5 million	Improved from a net loss of $78.5 million in 2024 (Note: Source shows net income of $51.0M for 9M 2025 vs net loss of $78.5M for 9M 2024)

Partnership Cost-Sharing

The collaboration with Biogen for zorevunersen outside the US, Canada, and Mexico significantly impacts the cash burn rate. Under the agreement, Stoke Therapeutics pays 70 percent of the external clinical development costs for zorevunersen, with Biogen covering the remaining 30 percent. This means that while Stoke is leading the global development, a significant portion of the EMPEROR trial expense is borne by their partner, which helps manage Stoke's immediate cash outflow.

Manufacturing Costs

A core operational cost involves the manufacturing of the antisense oligonucleotide drug substance for zorevunersen and other pipeline candidates. While specific manufacturing costs aren't itemized separately from R&D in the high-level summaries, this process requires specialized chemical synthesis and quality control to produce the clinical-grade material needed for ongoing trials like EMPEROR. The costs scale with the amount of drug substance required for the Phase 3 study and subsequent regulatory stockpiling.

Finance: draft 13-week cash view by Friday.

Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Revenue Streams

You're looking at the financial scaffolding that supports Stoke Therapeutics, Inc. right now, late in 2025. The revenue streams are heavily weighted toward partnership economics, which is typical when a company is advancing a lead asset into late-stage trials.

The core of the current financial inflow comes from the collaboration with Biogen for zorevunersen, which grants Biogen exclusive rights outside the United States, Canada, and Mexico. Stoke Therapeutics retains exclusive development and commercialization rights for zorevunersen in the United States, Canada, and Mexico, setting up a potential future direct sales revenue stream in North America post-approval.

Collaboration Revenue is the immediate focus, encompassing upfront payments, milestone achievements, and cost-sharing arrangements with partners like Biogen and Acadia Pharmaceuticals. Here's a quick look at the key terms of the Biogen zorevunersen agreement:

Revenue Component	Stoke Therapeutics (STOK) Financial Detail
Biogen Upfront Payment	$165 million received in February 2025.
Potential Milestones (Biogen)	Up to $385 million in development and commercial milestone payments.
Future Royalties (Biogen Territory)	Tiered royalties ranging from low double digits to high teens on net sales.
Development Cost Sharing (Zorevunersen)	Biogen covers 30 percent; Stoke covers 70 percent of external clinical development costs.

The upfront payment from Biogen has been recognized over time as revenue. For the first quarter of 2025, revenue recognized from the License and Collaboration Agreement with Biogen was $152.4 million for the three months ended March 31, 2025. By the end of the second quarter, the cumulative revenue recognized from the Biogen Agreement for the six months ended June 30, 2025, reached $155.6 million.

Looking at the broader picture, the Total YTD 2025 Revenue, as reported for the nine months ending September 30, 2025, hit $183.0 million. This is a massive jump from $13.9 million for the same period in 2024. Honestly, it's the upfront payment amortization driving this.

The components making up that $183.0 million YTD revenue as of September 30, 2025, break down like this:

• IP license performance obligation (related to Biogen deal): $150.8 million.
• Global development activities from the Biogen Agreement: $11.5 million.
• Revenue related to the Acadia Agreement: $6.8 million.

Future Product Sales revenue for Stoke Therapeutics is tied to their retained exclusive rights for zorevunersen in the United States, Canada, and Mexico. This means direct commercial revenue generation, should the drug gain approval in those territories, is a distinct, separate stream from the Biogen ex-US/Canada/Mexico royalties. Also, remember the option Biogen has for follow-on SCN1A-targeting ASO products outside the US, Canada, and Mexico, which carries separate milestone, cost sharing, and royalty considerations.

Finance: draft 13-week cash view by Friday.