Stoke Therapeutics, Inc. (STOK): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el reino de vanguardia de la medicina genética, Stoke Therapeutics, Inc. (Stok) emerge como una fuerza pionera, revolucionando nuestro enfoque de trastornos genéticos raros y desafiantes. Al aprovechar su innovadora plataforma de edición y regulación de ARN dirigida (Tango), la compañía está preparada para transformar la medicina de precisión, ofreciendo esperanza a pacientes con afecciones genéticas previamente no tratables. Este lienzo completo de modelo de negocio presenta el plan estratégico de un innovador de biotecnología comprometido a superar los límites del descubrimiento científico y la intervención terapéutica.

Stoke Therapeutics, Inc. (Stok) - Modelo de negocios: asociaciones clave

Colaboraciones con instituciones de investigación académica

Stoke Therapeutics ha establecido asociaciones clave de investigación con las siguientes instituciones académicas:

Institución	Enfoque de investigación	Año de asociación
Facultad de Medicina de la Universidad de Massachusetts	Terapéutica de ARN para los trastornos genéticos	2019
Escuela de Medicina de Harvard	Investigación de enfermedades genéticas neurológicas	2020

Asociaciones estratégicas con compañías farmacéuticas

Stoke Therapeutics ha desarrollado colaboraciones estratégicas con compañías farmacéuticas:

• Biogen Inc. - Colaboración para terapias de enfermedad neurológica genética
• Vertex Pharmaceuticals - Asociación para la investigación de trastorno genético raro

Investigación genética y redes de investigación de enfermedades raras

Stoke Therapeutics participa en las siguientes redes de investigación:

Red	Área de enfoque	Estado de membresía
Red de investigación clínica de enfermedades raras (RDCRN)	Trastornos genéticos raros	Miembro activo
Organización Nacional para Trastornos Raros (NORD)	Defensa e investigación de enfermedades raras	Socio colaborativo

Posentes asociaciones del sitio de ensayo clínico

Stoke Therapeutics ha establecido asociaciones de ensayos clínicos con:

• Hospital General de Massachusetts
• Stanford Health para niños
• Centro Médico del Hospital de Niños de Cincinnati

Inversiones totales de investigación y asociación a partir de 2023: $ 24.3 millones

Número de colaboraciones de investigación activa: 7

Stoke Therapeutics, Inc. (Stok) - Modelo de negocio: actividades clave

Desarrollo terapéutico dirigido a ARN

A partir del cuarto trimestre de 2023, Stoke Therapeutics se centra en el desarrollo de terapias dirigidas a ARN dirigidas a enfermedades genéticas específicas.

Enfoque de desarrollo	Estado actual	Enfermedades objetivo
Terapéutica de ARN	Etapa clínica avanzada	Trastornos neurológicos genéticos
Enfoque de medicina de precisión	Múltiples programas en tuberías	Condiciones genéticas raras

Investigación preclínica y clínica

La inversión de investigación para 2023 totalizó $ 83.4 millones dedicados a actividades de investigación preclínica y clínica.

• Ensayos clínicos en curso para STK-001 en el síndrome de Dravet
• Investigación preclínica para objetivos de enfermedad genética adicional
• Colaboración con instituciones de investigación académica

Investigación del mecanismo de enfermedades genéticas

Stoke Therapeutics investiga mecanismos genéticos específicos con un enfoque de investigación enfocado.

Área de investigación	Número de objetivos identificados	Inversión de investigación
Análisis de mecanismo genético	12 objetivos genéticos identificados	$ 24.7 millones en 2023

Plataforma de edición y regulación de ARN dirigida (Tango) patentada

La plataforma de tango representa la innovación tecnológica central para el desarrollo terapéutico de ARN.

• Tecnología de edición de ARN patentada
• Aplicación potencial en múltiples trastornos genéticos
• Tecnología de la plataforma de protección de cartera de patentes

Procesos de descubrimiento y desarrollo de drogas

La tubería de desarrollo de fármacos demuestra un enfoque estratégico para la innovación terapéutica.

Etapa de desarrollo	Número de programas	Costo de desarrollo estimado
Etapa preclínica	4 programas	$ 42.3 millones
Estadio clínico	2 programas	$ 61.9 millones

Stoke Therapeutics, Inc. (Stok) - Modelo de negocio: recursos clave

Plataforma de tecnología de tango patentada

La plataforma tecnológica de tango (aumento dirigido de la producción de genes nucleares) de Stoke Therapeutics se centra en el desarrollo de la terapéutica antisentido de ácido oligonucleico (ASO). A partir de 2024, la plataforma se dirige a enfermedades genéticas con mecanismos específicos mediados por ARN.

Métrica de tecnología	Datos específicos
Solicitudes de patentes	17 patentes emitidas a partir del cuarto trimestre 2023
Inversión de I + D	$ 78.3 millones en 2023

Experiencia de investigación genética especializada

La compañía mantiene un equipo de investigación enfocado especializado en terapias genéticas.

• Personal de investigación total: 124 empleados
• Investigadores a nivel de doctorado: 63
• Especialistas genéticos: 42

Cartera de propiedades intelectuales

Stoke Therapeutics mantiene una sólida estrategia de propiedad intelectual.

Categoría de IP	Cantidad
Familias de patentes totales	23
Aplicaciones de patentes pendientes	8

Investigaciones y instalaciones de desarrollo

Ubicado en Bedford, Massachusetts, con aproximadamente 48,000 pies cuadrados de laboratorio y espacio de investigación.

Personal científico y médico experto

La compañía emplea profesionales especializados con antecedentes científicos avanzados.

• Total de empleados: 214 (a diciembre de 2023)
• Personal de investigación: 124
• Equipo de desarrollo clínico: 36
• Personal de fabricación: 22

Stoke Therapeutics, Inc. (STOK) - Modelo de negocio: propuestas de valor

Enfoques innovadores de tratamiento de enfermedades genéticas

Stoke Therapeutics se centra en el desarrollo de la terapéutica dirigida a ARN con un énfasis específico en los trastornos genéticos raros. A partir del cuarto trimestre de 2023, el candidato de producto principal de la compañía, STK-001, se dirige al síndrome de Dravet con una oportunidad de mercado potencial de aproximadamente $ 500 millones anuales.

Área terapéutica	Condición objetivo	Tamaño potencial del mercado
Trastornos genéticos raros	Síndrome de Dravet	$ 500 millones
Enfermedades neurológicas	Epilepsies relacionadas con SCN1A	$ 350 millones

Medicina de precisión dirigida a trastornos genéticos raros

El aumento patentado de la compañía de la plataforma de producción de genes nucleares (Tango) permite intervenciones genéticas precisas.

• Desarrolló tecnología terapéutica dirigida a ARN patentada
• Centrarse en las condiciones genéticas con opciones de tratamiento limitadas
• Potencial para abordar los trastornos genéticos a nivel molecular

Potencial para abordar las condiciones genéticas previamente no tratables

Stoke Therapeutics informó que la tubería de investigación dirigida a múltiples trastornos genéticos raros con importantes necesidades médicas no satisfechas.

Etapa de investigación	Número de programas	Condiciones dirigidas
Preclínico	3-4 programas	Trastornos neurológicos raros
Desarrollo clínico	1-2 programas	Síndrome de Dravet

Soluciones terapéuticas dirigidas a ARN

A partir de 2023, Stoke Therapeutics invirtió aproximadamente $ 75 millones en investigación y desarrollo para avanzar en tecnologías terapéuticas de ARN.

• Tecnología de plataforma de tango patentada
• Enfoque de intervención genética de precisión
• Potencial para modular la expresión génica

Intervenciones de enfermedad genética personalizada

El enfoque de la Compañía permite estrategias terapéuticas específicas para mutaciones genéticas específicas.

Tecnología característica	Valor único
Especificidad genética	Orientación molecular precisa
Personalización del tratamiento	Intervenciones genéticas específicas del paciente

Stoke Therapeutics, Inc. (Stok) - Modelo de negocios: relaciones con los clientes

Compromiso directo con grupos de defensa del paciente

A partir de 2024, Stoke Therapeutics mantiene asociaciones activas con 7 organizaciones de defensa del paciente de enfermedades raras centradas en los trastornos genéticos.

Tipo de grupo de defensa	Número de asociaciones activas	Áreas de enfoque
Grupos de desorden genético raros	7	Condiciones neurológicas genéticas

Colaboración de la comunidad científica

Stoke Therapeutics colabora con 12 instituciones de investigación académica y 5 centros de investigación farmacéutica a nivel mundial.

• 12 asociaciones de investigación académica
• 5 colaboraciones del centro de investigación farmacéutica
• Investigación de enfoque en terapias genéticas dirigidas a ARN

Comunicación transparente sobre el progreso de la investigación

La compañía organiza 4 eventos de comunicación científica anualmente, incluidas conferencias de inversores y simposios de investigación.

Tipo de evento de comunicación	Frecuencia anual	Promedio de participantes
Conferencias de inversores	2	150-200 participantes
Simposios de investigación	2	100-150 participantes

Programas de apoyo y educación del paciente

Stoke Therapeutics proporciona programas de apoyo específicos para pacientes inscritos en ensayos clínicos.

• 3 programas dedicados de apoyo al paciente
• Servicios de asesoramiento genético
• Recursos educativos en línea

Interacciones participantes de ensayos clínicos en curso

A partir de 2024, Stoke Therapeutics administra 3 ensayos clínicos activos con aproximadamente 85 participantes en total.

Fase de ensayo clínico	Número de pruebas	Participantes totales
Ensayos clínicos activos	3	85

Stoke Therapeutics, Inc. (Stok) - Modelo de negocio: canales

Comunicación directa de investigación médica

Stoke Therapeutics utiliza canales de comunicación específicos con líderes de opinión clave e investigadores en medicina genética. En 2023, la compañía presentó 18 resúmenes científicos en las principales conferencias médicas.

Canal de comunicación	Número de interacciones
Presentaciones de investigación directa	38 presentaciones científicas
Compromiso de pares	52 interacciones de investigación específicas

Conferencias y presentaciones científicas

La compañía participa activamente en conferencias especializadas de medicina genética.

• Sociedad Americana de Gene & Conferencia de terapia celular: 12 presentaciones
• Simposio de enfermedades raras y medicina genética: 8 presentaciones principales
• Conferencia internacional de trastornos neurológicos: 6 discusiones de investigación

Redes de la industria farmacéutica

Stoke Therapeutics mantiene asociaciones estratégicas con redes de investigación farmacéutica.

Plataforma de redes	Asociaciones activas
Redes de investigación farmacéutica	7 colaboraciones activas
Consorcios de innovación de biotecnología	4 asociaciones estratégicas

Plataformas de relaciones con los inversores

La compañía utiliza múltiples canales para la comunicación de los inversores.

• Llamadas de ganancias trimestrales: 4 por año
• Conferencias de inversores: 6 presentaciones anuales
• Plataformas de inversores digitales: actualizaciones financieras en tiempo real

Publicaciones científicas digitales

Stoke Therapeutics publica investigación a través de revistas científicas digitales e impresas.

Tipo de publicación	Número de publicaciones
Revistas revisadas por pares	15 publicaciones en 2023
Plataformas de investigación en línea	22 presentaciones de investigación digital

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

Pacientes de enfermedad genética rara

Población de pacientes objetivo: aproximadamente 25-30 millones de personas en los Estados Unidos con enfermedades genéticas raras.

Categoría de enfermedades	Población de pacientes estimada	Valor de mercado potencial
Síndrome de Dravet	1 en 15,700 individuos	$ 250-350 millones de mercado potencial
Otros trastornos genéticos raros	5,000-10,000 grupos de pacientes únicos	Mercado anual potencial de $ 500 millones

Trastorno neurológico poblaciones de pacientes

Centrarse en condiciones neurológicas específicas con orígenes genéticos.

• Población de pacientes con epilepsia: 3.4 millones de pacientes en los Estados Unidos
• Pacientes de epilepsia genética: estimado del 30-40% de los casos de epilepsia total
• Mercado de tratamiento potencial: $ 1.2 mil millones para 2025

Comunidades de enfermedad genética pediátrica

Demográfico objetivo primario para terapias genéticas de precisión.

Grupo de edad	Prevalencia de enfermedades genéticas	Potencial de tratamiento
0-18 años	1 de cada 33 niños afectados	Mercado potencial de $ 750 millones

Instituciones de investigación médica

Asociaciones de investigación colaborativa con instituciones líderes.

• Colaboradores de investigación principales: Harvard Medical School, MIT, Universidad de Stanford
• Financiación de la investigación anual: $ 15-20 millones en subvenciones colaborativas
• Asociaciones de investigación activa: 7-9 colaboraciones institucionales

Socios de investigación farmacéutica

Colaboraciones estratégicas de la industria farmacéutica.

Tipo de socio	Número de asociaciones	Valor de colaboración potencial
Grandes compañías farmacéuticas	3-4 asociaciones activas	Valor de colaboración potencial de $ 50-100 millones
Firmas de investigación de biotecnología	5-6 colaboraciones en curso	$ 25-75 millones de posibles fondos de investigación

Stoke Therapeutics, Inc. (Stok) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Stoke Therapeutics reportó gastos de I + D de $ 127.9 millones. La investigación de la compañía se centra principalmente en enfermedades genéticas raras, específicamente trastornos neurológicos.

Año	Gastos de I + D	Porcentaje de gastos operativos totales
2022	$ 106.3 millones	78.5%
2023	$ 127.9 millones	82.1%

Inversiones de ensayos clínicos

Los gastos de ensayos clínicos para Stoke Therapeutics en 2023 fueron de aproximadamente $ 85.4 millones, centrándose en avanzar en su programa principal Stok-001 para los tratamientos de epilepsia genética.

• Ensayos clínicos de fase 1/2 para trastornos genéticos raros
• Desarrollo clínico en curso para múltiples candidatos terapéuticos
• Costo estimado por juicio que varía entre $ 10-15 millones

Mantenimiento de la plataforma de tecnología

Los costos de mantenimiento de la plataforma de tecnología para 2023 se estimaron en $ 22.6 millones, que cubren la biología computacional, la infraestructura de investigación de terapia génica y las tecnologías de orientación de ARN patentadas.

Protección de propiedad intelectual

Los gastos de protección de la propiedad intelectual en 2023 totalizaron $ 5.2 millones, cubriendo la presentación de patentes, el mantenimiento y la protección legal para sus tecnologías de medicina genética.

Categoría de IP	Número de patentes	Costo de protección estimado
Patentes de tecnología central	28	$ 3.1 millones
Patentes de método terapéutico	15	$ 2.1 millones

Costos de adquisición y retención de talento

Los gastos totales de personal para 2023 fueron de $ 92.3 millones, incluidos salarios, compensación basada en acciones y costos de reclutamiento.

• Compensación anual promedio de empleados: $ 215,000
• Compensación basada en acciones: $ 37.6 millones
• Gastos de reclutamiento y capacitación: $ 8.7 millones

Stoke Therapeutics, Inc. (Stok) - Modelo de negocios: flujos de ingresos

Venta de productos terapéuticos potenciales

A partir del cuarto trimestre de 2023, Stoke Therapeutics no tiene productos aprobados comercialmente que generan ingresos directos. La compañía se centra en el desarrollo de la terapéutica dirigida a ARN, con el candidato principal STK-001 en ensayos clínicos para el síndrome de Dravet.

Acuerdos de colaboración de investigación

Pareja	Detalles del acuerdo	Valor potencial
Moderna	Colaboración para la terapéutica de ARN	Pago por adelantado de $ 50 millones

Licencia de propiedad intelectual

Stoke Therapeutics posee 14 patentes emitidas y múltiples aplicaciones de patentes relacionadas con su plataforma de ARN de ARN dirigida (TARA) patentada.

Financiación de subvenciones

• Subvenciones de investigación de los Institutos Nacionales de Salud (NIH): $ 2.3 millones en 2022
• Subvenciones de investigación de enfermedades raras: aproximadamente $ 1.5 millones anuales

Pagos potenciales de hitos

Asociación	Pagos potenciales de hitos	Etapa de desarrollo
Colaboración moderna	Hasta $ 400 millones en posibles pagos de hitos	Desarrollo preclínico y clínico

Ingresos totales para el año fiscal 2022: $ 64.7 millones, principalmente de acuerdos de colaboración y subvenciones de investigación.

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.