Stoke Therapeutics, Inc. (Stok): Business Model Canvas [Jan-2025 Mis à jour]

Dans le royaume de la pointe de la médecine génétique, Stoke Therapeutics, Inc. (Stok) émerge comme une force pionnière, révolutionnant notre approche des troubles génétiques rares et difficiles. En tirant parti de leur plate-forme révolutionnaire de montage et de régulation de l'ARN ciblé (Tango), la société est prête à transformer la médecine de précision, offrant de l'espoir aux patients souffrant de conditions génétiques auparavant non traitables. Cette toile complète du modèle commercial dévoile le plan stratégique d'un innovateur biotechnologique déterminé à repousser les limites de la découverte scientifique et de l'intervention thérapeutique.

Stoke Therapeutics, Inc. (Stok) - Modèle commercial: partenariats clés

Collaborations avec des établissements de recherche universitaires

Stoke Therapeutics a établi des partenariats de recherche clés avec les établissements universitaires suivants:

Institution	Focus de recherche	Année de partenariat
École de médecine de l'Université du Massachusetts	Thérapeutique à l'ARN pour les troubles génétiques	2019
École de médecine de Harvard	Recherche de maladies génétiques neurologiques	2020

Partenariats stratégiques avec les sociétés pharmaceutiques

Stoke Therapeutics a développé des collaborations stratégiques avec les sociétés pharmaceutiques:

• Biogen Inc. - Collaboration pour les thérapies génétiques des maladies neurologiques
• Vertex Pharmaceuticals - Partenariat pour la recherche de troubles génétiques rares

Recherche génétique et réseaux de recherche de maladies rares

Stoke Therapeutics participe aux réseaux de recherche suivants:

Réseau	Domaine de mise au point	Statut d'adhésion
Réseau de recherche clinique de maladies rares (RDCRN)	Troubles génétiques rares	Membre actif
Organisation nationale pour les troubles rares (NORD)	Plaidoyer et recherche rares	Partenaire collaboratif

Partenariats potentiels de sites d'essai cliniques

Stoke Therapeutics a établi des partenariats d'essais cliniques avec:

• Hôpital général du Massachusetts
• Santé des enfants de Stanford
• Centre médical de l'hôpital pour enfants de Cincinnati

Recherche totale et investissements en partenariat à partir de 2023: 24,3 millions de dollars

Nombre de collaborations de recherche active: 7

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: Activités clés

Développement thérapeutique ciblé par l'ARN

Depuis le Q4 2023, Stoke Therapeutics se concentre sur le développement de thérapies ciblées par l'ARN ciblant des maladies génétiques spécifiques.

Focus de développement	État actuel	Target Diseases
Thérapeutique à l'ARN	Étape clinique avancée	Troubles neurologiques génétiques
Approche de la médecine de précision	Plusieurs programmes en pipeline	Conditions génétiques rares

Recherche préclinique et clinique

L'investissement en recherche pour 2023 a totalisé 83,4 millions de dollars dédiés aux activités de recherche préclinique et clinique.

• Essais cliniques en cours pour STK-001 dans le syndrome de Dravet
• Recherche préclinique pour des cibles de maladies génétiques supplémentaires
• Collaboration avec les établissements de recherche universitaires

Enquête sur le mécanisme des maladies génétiques

Stoke Therapeutics étudie les mécanismes génétiques spécifiques avec une approche de recherche ciblée.

Domaine de recherche	Nombre de cibles identifiées	Investissement en recherche
Analyse du mécanisme génétique	12 cibles génétiques identifiées	24,7 millions de dollars en 2023

Plateforme de montage et de réglementation de l'ARN ciblé propriétaire (Tango)

La plate-forme Tango représente l'innovation technologique de base pour le développement thérapeutique à l'ARN.

• Technologie de montage de l'ARN propriétaire
• Application potentielle sur plusieurs troubles génétiques
• Portefeuille de brevets Protéger la technologie de la plate-forme

Processus de découverte et de développement de médicaments

Le pipeline de développement de médicaments démontre une approche stratégique de l'innovation thérapeutique.

Étape de développement	Nombre de programmes	Coût de développement estimé
Étape préclinique	4 programmes	42,3 millions de dollars
Étape clinique	2 programmes	61,9 millions de dollars

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: Ressources clés

Plate-forme technologique de tango propriétaire

La plate-forme technologique du Tango (augmentation ciblée de la sortie du gène nucléaire) de Stoke Therapeutics se concentre sur le développement de la thérapie antisens de l'acide oligonucléique (ASO). En 2024, la plate-forme cible les maladies génétiques avec des mécanismes spécifiques à médiation par l'ARN.

Métrique technologique	Données spécifiques
Demandes de brevet	17 Brevets délivrés au quatrième trimestre 2023
Investissement en R&D	78,3 millions de dollars en 2023

Expertise spécialisée de la recherche génétique

La société maintient une équipe de recherche ciblée spécialisée dans les thérapies génétiques.

• Personnel de recherche total: 124 employés
• Rechercheurs au niveau du doctorat: 63
• Spécialistes génétiques: 42

Portefeuille de propriété intellectuelle

Stoke Therapeutics maintient une solide stratégie de propriété intellectuelle.

Catégorie IP	Quantité
Familles totales de brevets	23
Demandes de brevet en instance	8

Installations de recherche et de développement

Situé à Bedford, Massachusetts, avec environ 48 000 pieds carrés d'espace de laboratoire et de recherche.

Personnel scientifique et médical qualifié

L'entreprise emploie des professionnels spécialisés avec des antécédents scientifiques avancés.

• Total des employés: 214 (en décembre 2023)
• Personnel de recherche: 124
• Équipe de développement clinique: 36
• Personnel de fabrication: 22

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: propositions de valeur

Approches de traitement des maladies génétiques innovantes

Stoke Therapeutics se concentre sur le développement de la thérapie ciblée par l'ARN avec un accent spécifique sur les troubles génétiques rares. Au quatrième trimestre 2023, le candidat principal de la société, STK-001, cible le syndrome de Dravet avec une opportunité de marché potentielle d'environ 500 millions de dollars par an.

Zone thérapeutique	Condition cible	Taille du marché potentiel
Troubles génétiques rares	Syndrome de Dravet	500 millions de dollars
Maladies neurologiques	Épilepsies liées à SCN1A	350 millions de dollars

Médecine de précision ciblant les troubles génétiques rares

L'augmentation ciblée propriétaire de la société de la plate-forme de sortie de gènes nucléaires (Tango) permet des interventions génétiques précises.

• Élaboré une technologie thérapeutique ciblée par l'ARN propriétaire
• Concentrez-vous sur les conditions génétiques avec des options de traitement limitées
• Potentiel pour aborder les troubles génétiques au niveau moléculaire

Potentiel pour résoudre les conditions génétiques auparavant inextraitables

Stoke Therapeutics a rapporté des pipelines de recherche ciblant plusieurs troubles génétiques rares ayant des besoins médicaux non satisfaits importants.

Étape de recherche	Nombre de programmes	Conditions ciblées
Préclinique	3-4 programmes	Troubles neurologiques rares
Développement clinique	1-2 programmes	Syndrome de Dravet

Solutions thérapeutiques ciblées par l'ARN

En 2023, Stoke Therapeutics a investi environ 75 millions de dollars de recherche et développement pour l'avancement des technologies thérapeutiques de l'ARN.

• Technologie de plate-forme de tango propriétaire
• Approche d'intervention génétique de précision
• Potentiel pour moduler l'expression des gènes

Interventions de maladies génétiques personnalisées

L'approche de l'entreprise permet des stratégies thérapeutiques ciblées pour des mutations génétiques spécifiques.

Caractéristique technologique	Valeur unique
Spécificité génétique	Ciblage moléculaire précis
Personnalisation du traitement	Interventions génétiques spécifiques au patient

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: relations clients

Engagement direct avec les groupes de défense des patients

En 2024, Stoke Therapeutics entretient des partenariats actifs avec 7 organisations de défense des patients atteints de maladies rares axées sur les troubles génétiques.

Type de groupe de plaidoyer	Nombre de partenariats actifs	Domaines de concentration
Groupes de troubles génétiques rares	7	Conditions neurologiques génétiques

Collaboration communautaire scientifique

Stoke Therapeutics collabore avec 12 établissements de recherche universitaires et 5 centres de recherche pharmaceutique dans le monde.

• 12 partenariats de recherche académique
• 5 collaborations du centre de recherche pharmaceutique
• Recherche sur la recherche sur les thérapies génétiques ciblées par l'ARN

Communication transparente sur les progrès de la recherche

La société organise 4 événements de communication scientifique chaque année, y compris des conférences d'investisseurs et des symposiums de recherche.

Type d'événement de communication	Fréquence annuelle	Moyenne des participants
Conférences d'investisseurs	2	150-200 participants
Symposiums de recherche	2	100-150 participants

Programmes de soutien aux patients et d'éducation

Stoke Therapeutics propose des programmes de soutien ciblés aux patients inscrits à des essais cliniques.

• 3 programmes de soutien aux patients dédiés
• Services de conseil génétique
• Ressources éducatives en ligne

Interactions des participants en cours d'essai clinique

En 2024, Stoke Therapeutics gère 3 essais cliniques actifs avec environ 85 participants au total.

Phase d'essai clinique	Nombre de procès	Total des participants
Essais cliniques actifs	3	85

Stoke Therapeutics, Inc. (Stok) - Modèle commercial: canaux

Communication de recherche médicale directe

Stoke Therapeutics utilise des canaux de communication ciblés avec les principaux leaders d'opinion et les chercheurs en médecine génétique. En 2023, la société a présenté 18 résumés scientifiques lors de grandes conférences médicales.

Canal de communication	Nombre d'interactions
Présentations de recherche directes	38 présentations scientifiques
Engagement entre pairs	52 Interactions de recherche ciblées

Conférences et présentations scientifiques

L'entreprise participe activement à des conférences spécialisées en médecine génétique.

• Société américaine de gène & Conférence de thérapie cellulaire: 12 présentations
• Symposium de maladie rare et de médecine génétique: 8 présentations d'ouverture
• Conférence internationale sur les troubles neurologiques: 6 discussions de recherche

Réseautage de l'industrie pharmaceutique

Stoke Therapeutics maintient des partenariats stratégiques avec les réseaux de recherche pharmaceutique.

Plate-forme de réseautage	Partenariats actifs
Réseaux de recherche pharmaceutique	7 collaborations actives
Consortiums d'innovation biotechnologique	4 partenariats stratégiques

Plateformes de relations avec les investisseurs

L'entreprise utilise plusieurs canaux pour la communication des investisseurs.

• Répédances trimestrielles: 4 par an
• Conférences des investisseurs: 6 présentations annuelles
• Plateformes d'investisseurs numériques: mises à jour financières en temps réel

Publications scientifiques numériques

Stoke Therapeutics publie des recherches dans des revues scientifiques numériques et imprimées.

Type de publication	Nombre de publications
Journaux évalués par des pairs	15 publications en 2023
Plateformes de recherche en ligne	22 soumissions de recherche numérique

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: segments de clientèle

Patiens de maladies génétiques rares

Population de patients cibles: environ 25 à 30 millions d'individus aux États-Unis atteints de maladies génétiques rares.

Catégorie de maladie	Population estimée des patients	Valeur marchande potentielle
Syndrome de Dravet	1 individus sur 15 700	Marché potentiel de 250 à 350 millions de dollars
Autres troubles génétiques rares	5 000 à 10 000 groupes de patients uniques	Marché annuel potentiel de 500 millions de dollars

Populations de patients du trouble neurologique

Concentrez-vous sur des conditions neurologiques spécifiques avec des origines génétiques.

• Population de patients en épilepsie: 3,4 millions de patients aux États-Unis
• Patients d'épilepsie génétiques: estimé 30 à 40% du total des cas d'épilepsie
• Marché du traitement potentiel: 1,2 milliard de dollars d'ici 2025

Communautés de maladies génétiques pédiatriques

Cible primaire démographique pour les thérapies génétiques de précision.

Groupe d'âge	Prévalence des maladies génétiques	Potentiel de traitement
0-18 ans	1 enfants sur 33 touchés	Marché potentiel de 750 millions de dollars

Institutions de recherche médicale

Partenariats de recherche collaborative avec les principaux institutions.

• Top Collaborateurs de la recherche: Harvard Medical School, MIT, Stanford University
• Financement de la recherche annuelle: 15-20 millions de dollars en subventions collaboratives
• Partenariats de recherche actifs: 7-9 collaborations institutionnelles

Partenaires de recherche pharmaceutique

Collaborations stratégiques de l'industrie pharmaceutique.

Type de partenaire	Nombre de partenariats	Valeur de collaboration potentielle
Grandes sociétés pharmaceutiques	3-4 partenariats actifs	Valeur de collaboration potentielle de 50 à 100 millions de dollars
Cabinets de recherche en biotechnologie	5-6 collaborations en cours	25 à 75 millions de dollars de financement de recherche potentiel

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2023, Stoke Therapeutics a déclaré des dépenses de R&D de 127,9 millions de dollars. La recherche de l'entreprise se concentre principalement sur des maladies génétiques rares, en particulier les troubles neurologiques.

Année	Dépenses de R&D	Pourcentage des dépenses d'exploitation totales
2022	106,3 millions de dollars	78.5%
2023	127,9 millions de dollars	82.1%

Investissements d'essais cliniques

Les dépenses des essais cliniques pour Stoke Therapeutics en 2023 étaient d'environ 85,4 millions de dollars, axées sur la progression de leur programme principal Stok-001 pour les traitements génétiques de l'épilepsie.

• Phase 1/2 essais cliniques pour les troubles génétiques rares
• Développement clinique en cours pour plusieurs candidats thérapeutiques
• Des coûts estimés par essai variant entre 10 et 15 millions de dollars

Maintenance de la plate-forme technologique

Les coûts de maintenance des plateformes technologiques pour 2023 ont été estimés à 22,6 millions de dollars, couvrant la biologie informatique, l'infrastructure de recherche sur la thérapie génique et les technologies de ciblage d'ARN propriétaires.

Protection de la propriété intellectuelle

Les dépenses de protection de la propriété intellectuelle en 2023 ont totalisé 5,2 millions de dollars, couvrant le dépôt, l'entretien et la protection juridique des brevets pour leurs technologies de médecine génétique.

Catégorie IP	Nombre de brevets	Coût de protection estimé
Brevets technologiques de base	28	3,1 millions de dollars
Brevets de méthode thérapeutique	15	2,1 millions de dollars

Frais d'acquisition et de rétention de talents

Les dépenses totales du personnel pour 2023 étaient de 92,3 millions de dollars, notamment les salaires, la rémunération en actions et les frais de recrutement.

• Compensation annuelle moyenne des employés: 215 000 $
• Compensation à base d'actions: 37,6 millions de dollars
• Frais de recrutement et de formation: 8,7 millions de dollars

Stoke Therapeutics, Inc. (Stok) - Modèle d'entreprise: Strots de revenus

Ventes de produits thérapeutiques potentiels

Depuis le quatrième trimestre 2023, Stoke Therapeutics n'a pas de produits approuvés commercialement générant des revenus directs. L'entreprise se concentre sur le développement de la thérapie ciblée par l'ARN, avec le candidat principal STK-001 dans les essais cliniques pour le syndrome de Dravet.

Accords de collaboration de recherche

Partenaire	Détails de l'accord	Valeur potentielle
Moderne	Collaboration pour l'ARN Therapeutics	Paiement initial de 50 millions de dollars

Licence de propriété intellectuelle

Stoke Therapeutics tient 14 brevets délivrés et plusieurs demandes de brevet liées à sa plate-forme antisens d'ARN ciblée (TARA) propriétaire.

Subventions

• Concessions de recherche des National Institutes of Health (NIH): 2,3 millions de dollars en 2022
• Subventions de recherche sur les maladies rares: environ 1,5 million de dollars par an

Paiements de jalons potentiels

Partenariat	Paiements de jalons potentiels	Étape de développement
Collaboration moderne	Jusqu'à 400 millions de dollars en paiements de jalons potentiels	Développement préclinique et clinique

Revenu total pour l'exercice 2022: 64,7 millions de dollars, principalement des accords de collaboration et des subventions de recherche.

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.