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Stoke Therapeutics, Inc. (STOK): Business Model Canvas [Dec-2025 Updated] |
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You're looking to map out the actual business engine of Stoke Therapeutics now that they've hit a major inflection point with the Biogen deal and the zorevunersen Phase 3 trial. Honestly, this isn't just a science story anymore; it's a commercial setup, backed by a \$328.6 million cash balance as of September 30, 2025, and fueled by that \$165 million upfront payment received in Q1 2025. We need to see how their proprietary TANGO platform translates into revenue streams and what costs they are taking on as they build out their North American sales force. Here's the quick math on their late 2025 model-dive into the nine building blocks below to see the full picture.
Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Key Partnerships
You're looking at the core alliances that fuel Stoke Therapeutics, Inc.'s engine, especially how they are funding the late-stage push for zorevunersen and pipeline expansion. These aren't just handshake deals; they come with hard numbers that define the company's current financial footing.
Biogen: Ex-North America Development and Commercialization for Zorevunersen
The agreement with Biogen, which closed in February 2025, is a massive validation and a key cash flow driver. Stoke Therapeutics received an upfront payment of $165 million upon closing the deal for ex-North America rights to zorevunersen (STK-001) for Dravet syndrome (DS). This collaboration is structured to share the burden of the global Phase 3 EMPEROR study, with Biogen covering 30% of external clinical development costs while Stoke covers the remaining 70%. Beyond the initial cash infusion, Stoke stands to earn up to $385 million in development and commercial milestone payments, plus tiered royalties starting from low double digits to high teens on net sales in Biogen's territories. For the nine months ending September 30, 2025, revenue recognized from the Biogen Agreement totaled $150.8 million. This partnership, combined with Stoke's existing position, is expected to fund operations through to mid-2028.
Acadia Pharmaceuticals: Collaboration for the SYNGAP1 Program Development
Stoke Therapeutics' alliance with Acadia Pharmaceuticals focuses on several neurodevelopmental targets, notably the SYNGAP1 program. Acadia made an upfront payment of $60 million to Stoke. For the SYNGAP1 program specifically, the firms agreed to jointly share global research, development, and commercialization responsibilities, dividing future costs and profits 50-50. Stoke is also eligible for up to $907 million in total milestone payments and royalties from this collaboration. Revenue recognized related to the Acadia Agreement for the nine months ending September 30, 2025, was $6.8 million.
Clinical Research Organizations (CROs) and Trial Execution
The execution of the global Phase 3 EMPEROR study for zorevunersen relies heavily on external partners, including CROs to manage the complex logistics. This study is designed to enroll 150 patients with DS who have a confirmed variant in the SCN1A gene. As of the third quarter of 2025, the study was actively recruiting in the U.S., UK, and Japan, with more than 20 patients already randomized to either zorevunersen or sham. Enrollment is on track to complete in the second half of 2026, with European sites expected to initiate in the first half of 2026. The primary endpoint is the reduction in major motor seizure frequency over a 52-week treatment period.
Academic Medical Centers and Patient Advocacy Groups
Key sites for the EMPEROR trial are major Academic Medical Centers across the US, UK, EU, and Japan, providing the necessary infrastructure and specialist expertise for rare disease clinical trials. Stoke Therapeutics has noted enthusiasm from clinicians regarding the trial design. Patient Advocacy Groups are essential for reaching the target population, which for Dravet syndrome is approximately 38,000 patients across major markets. These groups help with disease awareness and the recruitment of eligible participants who must be on at least one stable anti-seizure medication and have tried at least two prior therapies.
Here's a quick look at the financial contribution of these major deals as of the third quarter of 2025:
| Partnership/Metric | Financial/Statistical Number | Timeframe/Context |
|---|---|---|
| Biogen Upfront Payment | $165 million | February 2025 Closing |
| Acadia Upfront Payment | $60 million | Total Deal |
| Total Potential Milestones (Biogen) | Up to $385 million | Zorevunersen |
| Total Potential Milestones/Royalties (Acadia) | Up to $907 million | SYNGAP1 Program |
| Biogen Cost Share (External Dev.) | 30% covered by Biogen | Zorevunersen Clinical Development |
| SYNGAP1 Cost/Profit Share | 50-50 split | Global Research/Development/Commercialization |
| YTD Revenue from Partnerships (9 Months) | $183.0 million | Ending September 30, 2025 |
| Cash as of September 30, 2025 | $328.6 million | Cash, cash equivalents, and marketable securities |
| EMPEROR Study Enrollment (as of Q3 2025) | Over 20 patients randomized | Phase 3 Zorevunersen Trial |
The combined revenue from these deals helped push Stoke Therapeutics' Q3 2025 revenue to $10.6 million, beating analyst forecasts by over 70%.
Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Key Activities
You're looking at the core engine driving Stoke Therapeutics, Inc. (STOK) right now-the actual work they are doing to bring their RNA medicines to market. It's all about execution on the late-stage trial and preparing the ground for a potential launch in North America.
Research and Development (R&D): Advance the TANGO platform and pipeline expansion
Stoke Therapeutics is heavily investing in its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, which is used to develop Antisense Oligonucleotides (ASOs). This R&D focus supports both the lead candidate and pipeline growth. Research and development expenses for the three months ended September 30, 2025, were $37.7 million, up from $22.2 million for the same period in 2024. Year-to-date through September 30, 2025, R&D expenses totaled $96.2 million. Pipeline expansion includes STK-002 for Autosomal Dominant Optic Atrophy (ADOA), with its Phase 1 (OSPREY) study now underway. The pipeline also includes work on the SYNGAP1 program.
Clinical Trial Execution: Manage the global Phase 3 EMPEROR study for zorevunersen
The management of the global Phase 3 EMPEROR study for zorevunersen is the most immediate, critical activity. This trial is actively recruiting patients in the U.S., UK, and Japan, with European sites expected to follow.
Here are the specifics on the EMPEROR study:
- Enrollment targets patients aged 2 to <18 with confirmed SCN1A gene variants.
- The study is designed to include 150 patients.
- The treatment period is a 52-week randomized, sham-controlled evaluation following an 8-week baseline period, totaling 60 weeks.
- Dosing involves 2 loading doses of 70 mg followed by 2 maintenance doses of 45 mg.
- The primary endpoint focuses on reductions in major motor seizure frequency.
- Data readout is expected by the end of 2027.
Open-label extension data has shown durable reductions in major motor seizures and continuing improvements in cognition and behavior.
Regulatory Strategy: Pursue expedited pathways like FDA Breakthrough Therapy Designation
Stoke Therapeutics is actively engaging with regulatory bodies to expedite zorevunersen's path to patients. Zorevunersen has been granted FDA Breakthrough Therapy Designation. The company is scheduled to meet with the FDA before year-end 2025 to discuss how to proceed under this designation. Furthermore, zorevunersen holds Orphan Drug Designation from both the FDA and EMA, alongside Rare Pediatric Disease Designation from the FDA. For STK-002, the FDA has granted Orphan Drug Designation.
Antisense Oligonucleotide (ASO) Manufacturing: Secure and scale up drug substance production
While specific drug substance production capacity numbers aren't public, the company is focused on internal capability enhancement to support future needs. The Q3 2025 report noted an increase in SG&A expenses driven by personnel and launch readiness expenses. The overall financial position is structured to support this scaling effort.
| Financial Metric | Q3 2025 Amount | Q3 2024 Amount |
| SG&A Expenses (Quarterly) | $16.0 million | $12.7 million |
| Cash, Cash Equivalents, and Marketable Securities (as of 9/30/2025) | $328.6 million | N/A |
Commercial Launch Readiness: Build out the North American sales and marketing infrastructure
Investment in commercial readiness is reflected in the operating expenses, showing a clear focus on building infrastructure for a potential launch. The collaboration with Biogen covers commercialization outside of North America, while Stoke Therapeutics retains exclusive development and commercialization rights in the United States, Canada, and Mexico. The company's current cash runway is expected to fund operations through mid-2028 and into launch readiness.
Key financial indicators supporting this readiness include:
- Q3 2025 SG&A increase of $3.3 million driven by personnel and launch readiness expenses.
- Cash runway extends to mid-2028.
- Upfront payment from Biogen collaboration was $165 million.
Finance: draft 13-week cash view by Friday.
Stoke Therapeutics, Inc. (STOK) - Canvas Business Model: Key Resources
You're looking at the core assets Stoke Therapeutics, Inc. (STOK) relies on to execute its business plan as of late 2025. These aren't just line items; they are the tangible and intangible foundations of their value proposition.
TANGO Platform: Proprietary RNA medicine technology for protein upregulation
The TANGO (Targeted Augmentation of Nuclear Gene Output) platform is the engine here. It's designed to treat diseases caused by haploinsufficiencies-situations where having only one functional copy of a gene means the body is missing about 50% of the necessary protein. Stoke uses this platform to design antisense oligonucleotides (ASOs) that tell the healthy gene copy to produce more protein, aiming to restore levels to near normal.
The platform's potential reach is quite broad, based on their bioinformatics work:
- Identified approximately 1,200 monogenic diseases amenable to TANGO.
- Identified approximately 6,500 additional genes with RNA target signatures that could be addressed.
It's a precision tool, not a permanent DNA alteration. That's a key differentiator.
Intellectual Property
The value of the TANGO platform is locked down by its intellectual property. This IP covers the ASO design and the mechanism of action itself. We see this value reflected in their recent financial activity; for the nine months ending September 30, 2025, revenue recognized related to the IP license performance obligation under the Biogen Agreement was $150.8 million.
Here's a quick snapshot of the financial context supporting ongoing R&D and IP defense:
| Financial Metric | Value as of September 30, 2025 |
| Cash, Cash Equivalents, and Marketable Securities | $328.6 million |
| Projected Cash Runway | To mid-2028 |
| Revenue from IP/License Obligations (9 months ended 9/30/2025) | $183.0 million (Total) |
Cash Reserves
You want to know if the lights stay on, right? As of September 30, 2025, Stoke Therapeutics, Inc. held $328.6 million in cash, cash equivalents, and marketable securities. Honestly, that figure is projected to fund operations out to mid-2028. That runway gives them significant breathing room to advance their pipeline without immediate capital concerns.
Clinical Data: Long-term safety and efficacy data for zorevunersen in Dravet syndrome
The lead asset, zorevunersen (STK-001), is being tested in the global Phase 3 EMPEROR study. As of September 30, 2025, more than 20 patients were randomized in that study, and they expect enrollment to wrap up in the second half of 2026. The FDA has given this candidate a Breakthrough Therapy Designation, which signals serious potential.
The long-term data from earlier studies is what really matters for conviction:
- 94% (75/80) of eligible patients from earlier phases continued into the Open-Label Extension (OLE) studies.
- Patients on the highest initial dose regimen (two or three doses of 70 mg) showed a median seizure reduction of 84.8% at 3 months post-last dose.
- Those same patients saw a median increase of eight seizure-free days per 28 days at 3 months post-last dose.
They are scheduled to review four years of safety and efficacy data with the FDA before the end of 2025. That meeting is definitely a key near-term milestone.
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.
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