Financial Data and Key Metrics Changes - The company ended Q3 2025 with $328.6 million in cash, cash equivalents, and marketable securities, and raised an additional $48.7 million post-quarter through an ATM [20][21] - Total revenue for the quarter was $10.6 million, up from $4.9 million in Q3 2024, primarily due to a $6.7 million increase from collaboration with Biogen [20][21] - Net loss for Q3 was $38.3 million, or $0.65 per share, compared to a net loss of $26.4 million, or $0.47 per share, in the prior year, driven by increased operating expenses [20][21] Business Line Data and Key Metrics Changes - The company has made significant progress in the clinical development of zorevunersen, with over 200 patients identified for the phase 3 study, an increase from 150 previously reported [5][18] - R&D expenses rose to $37.7 million from $22.2 million a year ago, reflecting spending on the phase 3 study and early-stage pipeline advancements [20][21] - SG&A expenses increased to $16 million from $12.7 million, indicating investment in commercial capabilities [20][21] Market Data and Key Metrics Changes - The company is preparing for a meeting with the FDA to discuss expedited regulatory pathways for zorevunersen, which has received breakthrough therapy designation [6][19] - The market opportunity for zorevunersen is growing due to increased awareness of Dravet syndrome and the potential disease-modifying effects of the drug [6][19] Company Strategy and Development Direction - The company aims to rapidly scale its business to reach as many patients as possible with zorevunersen, focusing on medical affairs and commercial leadership [7][8] - There is a strategic emphasis on developing additional disease-modifying medicines, particularly in haploinsufficient disease areas [8][9] - The company is also launching a disease awareness campaign to improve diagnosis rates of Dravet syndrome through genetic testing [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the company's growth trajectory and the potential impact of zorevunersen on patients with Dravet syndrome [3][22] - The company is committed to educating the medical community and increasing awareness of Dravet syndrome [22] - Management highlighted the importance of the upcoming FDA meeting and the potential for expedited pathways to bring zorevunersen to market [19][22] Other Important Information - The company is actively recruiting for the phase 3 Emperor study, with 30 sites in the U.S., U.K., and Japan [17][18] - The first patient was randomized in August, and over 20 patients have been randomized so far [18] - The company anticipates completing enrollment in the second half of 2026, with data expected in late 2027 [39] Q&A Session Summary Question: What potential scenarios might be anticipated based on the PDD meeting with the FDA? - The company is preparing to present extensive data, including four-year safety data, to the FDA, which could lead to expedited pathways or a request to continue with the phase 3 study [26][27] Question: How clinically meaningful are the cognitive and behavioral improvements on the VABS-III data? - A change of one to three points on the VABS-III scale is considered clinically meaningful, with significant improvements observed in communication skills [31][33] Question: Can the company pursue early approval based solely on seizure reduction? - Yes, the company could seek approval based on seizure reduction, but it intends to pursue a label that reflects the disease-modifying potential of zorevunersen [37] Question: What are the common reasons for screen failure in the Emperor study? - Common reasons include not meeting the minimum seizure count required for enrollment, illness during the screening period, or family commitments [78] Question: How does the company plan to handle the differences in dosing schedules between phase 1/2 and phase 3 studies? - The company acknowledges the differences but believes the data from phase 1/2 and the open-label extension supports the dosing regimen planned for phase 3 [66]

Stoke Therapeutics Third Quarter 2025 Business Update Webcast for Investors & Analysts November 4, 2025 © COPYRIGHT 2025 | STOKE THERAPEUTICS | 1 Forward-Looking Statements and Other Legal Notices This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "us") for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter(s) or Stoke or any officer, director, e ...

Financial Performance - Revenue for Q3 2025 was $10,632,000, a 117% increase compared to $4,894,000 in Q3 2024[18] - Net loss for Q3 2025 was $38,347,000, compared to a net loss of $26,430,000 in Q3 2024[18] - Basic net income (loss) per share for Q3 2025 was $(0.65), compared to $(0.47) for Q3 2024[18] - The company reported comprehensive income (loss) of $(37,997,000) for Q3 2025, compared to $(26,249,000) for Q3 2024[18] - For the nine months ended September 30, 2025, the company reported a net income of $51,049, compared to a net loss of $78,499 for the same period in 2024[26] - For the three months ended September 30, 2025, the company reported a net loss of $38.3 million, compared to a net loss of $26.4 million for the same period in 2024[122] - For the nine months ended September 30, 2025, revenue was $183.0 million, a significant increase of $169.1 million compared to $13.9 million in 2024[160] Expenses - Research and development expenses for Q3 2025 were $37,696,000, up 69% from $22,205,000 in Q3 2024[18] - Total operating expenses for Q3 2025 were $53,723,000, a 54% increase from $34,897,000 in Q3 2024[18] - Research and development expenses for the three months ended September 30, 2025, totaled $37.7 million, an increase from $22.2 million in the same period in 2024[145] - Sales, general and administrative expenses for the three months ended September 30, 2025, were $16.0 million, an increase of $3.3 million from $12.7 million in 2024[157] - Research and development expenses for the nine months ended September 30, 2025, totaled $96.2 million, an increase of $30.5 million from $65.7 million in 2024[161] - Sales, general and administrative expenses for the nine months ended September 30, 2025, were $45.9 million, up $9.9 million from $36.0 million in 2024[163] Assets and Liabilities - Total assets increased to $360,264,000 as of September 30, 2025, from $271,555,000 as of December 31, 2024[15] - Total stockholders' equity rose to $308,105,000 as of September 30, 2025, compared to $229,021,000 as of December 31, 2024[15] - Cash and cash equivalents decreased to $83,394,000 as of September 30, 2025, from $127,983,000 as of December 31, 2024[15] - Total accrued and other current liabilities as of September 30, 2025, were $30.1 million, an increase from $18.6 million as of December 31, 2024[59] - The company had an accumulated deficit of $439.8 million as of September 30, 2025[169] Cash Flow - Net cash provided by operating activities was $76,039 for the nine months ended September 30, 2025, a significant improvement from a net cash used of $63,658 in 2024[26] - Cash, cash equivalents, and restricted cash at the end of the period were $84,115, down from $150,585 at the end of September 2024[42] - For the nine months ended September 30, 2025, net cash provided by operating activities was $76.0 million, attributed to a net income of $51.0 million and non-cash charges of $25.6 million[174] - During the same period, cash used in operating activities in 2024 was $63.7 million, primarily due to a net loss of $78.5 million[175] Financing Activities - The company issued approximately 7.0 million shares of common stock under a Controlled Equity Offering Sales Agreement for net proceeds of $61.0 million as of September 30, 2025[30] - The company completed an underwritten public offering in April 2024, resulting in net proceeds of approximately $119.9 million after deducting underwriting costs[31] - The company completed a public offering of 5,555,557 shares at $13.50 per share, resulting in net proceeds of approximately $119.9 million after costs[117] Collaborations and Agreements - The Company entered into a License and Collaboration Agreement with Acadia Pharmaceuticals in January 2022, receiving an upfront payment of $60.0 million and is eligible for up to $245.0 million in milestone payments for the SYNGAP1 program[72][73]. - The Company entered into a License and Collaboration Agreement with Biogen on February 14, 2025, receiving an upfront payment of $165.0 million and is eligible for up to $50.0 million in development milestone payments and $335.0 million in commercial milestone payments[81]. - The Company recognized revenue of $162.3 million related to the Biogen collaboration for the nine months ended September 30, 2025[139] Clinical Development - The company initiated a Phase 3 study, EMPEROR, for its investigational new medicine zorevunersen in May 2025, with the first patient dosed in August 2025[113] - The company plans to enroll the first patient in a Phase 1 study for STK-002, targeting autosomal dominant optic atrophy (ADOA), in the second half of 2025[115] - The company is actively recruiting patients for the global Phase 3 EMPEROR study of zorevunersen, with over 150 patients identified as potential candidates[134] - The Clinical Trial Authorization (CTA) for STK-002 was authorized by the UK Medicines and Healthcare Products Regulatory Agency in April 2023, with the Phase 1 trial announced in August 2025[204] Risks and Uncertainties - The company is subject to risks and uncertainties typical of early-stage biotechnology firms, including competition and the need for significant capital for product development[34] - The company expects to incur net losses for the foreseeable future, with increasing research and development expenses as it seeks regulatory approvals for product candidates[123] - The company has a history of operating losses and anticipates continuing to incur losses for the foreseeable future, necessitating additional funding to support research and development efforts[206] - The company faces various risks, including the potential for significant delays in product development and regulatory approval processes[202] Market and Regulatory Considerations - The company plans to discuss expedited regulatory pathways for zorevunersen with the FDA, leveraging its Breakthrough Therapy Designation[134] - The success of zorevunersen and STK-002 depends on multiple factors, including regulatory approvals, market acceptance, and reimbursement status[206] - Clinical trials for zorevunersen and STK-002 must demonstrate safety and efficacy to satisfy regulatory authorities[213] Internal Controls - The company recognizes that internal controls over financial reporting may not prevent all errors or fraud, providing only reasonable assurance of achieving control objectives[199] - There have been no changes in internal control over financial reporting that materially affect the company's internal controls during the reporting period[198]

Financial Performance - Revenue for Q3 2025 was $10.6 million, an increase of 116.3% from $4.9 million in Q3 2024, driven by agreements with Acadia and Biogen[9] - Revenue for the nine months ending September 30, 2025, was $183.0 million, a significant increase from $13.9 million for the same period in 2024[9] - Net income for the nine months ended September 30, 2025, was $51.0 million, or $0.85 per diluted share, compared to a net loss of $78.5 million, or $1.48 per share, for the same period in 2024[9] - Revenue for Q3 2025 was $10,632,000, a 117% increase compared to $4,894,000 in Q3 2024[25] Expenses and Losses - Net loss for Q3 2025 was $38.3 million, or $0.65 per share, compared to a net loss of $26.4 million, or $0.47 per share, for the same period in 2024[9] - Total operating expenses for Q3 2025 were $53,723,000, up from $34,897,000 in Q3 2024, representing a 54% increase[25] - Research and development expenses for Q3 2025 increased to $37.7 million from $22.2 million in Q3 2024, reflecting increased activities to support zorevunersen[9] - Research and development expenses for the nine months ended September 30, 2025, were $96,227,000, an increase from $65,710,000 in the same period of 2024[25] Cash and Assets - As of September 30, 2025, the company had $328.6 million in cash, cash equivalents, and marketable securities, expected to fund operations until mid-2028[9] - Cash and cash equivalents as of September 30, 2025, were $83,394,000, down from $127,983,000 as of December 31, 2024[23] - Total assets increased to $360,264,000 as of September 30, 2025, compared to $271,555,000 as of December 31, 2024[23] - Stockholders' equity rose to $308,105,000 as of September 30, 2025, from $229,021,000 as of December 31, 2024[23] Clinical Development - The global Phase 3 EMPEROR study is actively recruiting patients, with more than 20 patients randomized and approximately 35 additional patients in the screening period[3][4] - The company plans to present new data from the zorevunersen clinical development program at the American Epilepsy Society 2025 Annual Meeting in December[6] - The company is scheduled to meet with the FDA before year-end 2025 to discuss zorevunersen's safety and efficacy data under the Breakthrough Therapy Designation[6] Collaborations - The company is collaborating with Biogen and Acadia for the development of zorevunersen and STK-002[19] - The company sold approximately 1.8 million shares of common stock since September 30, 2025, raising $48.7 million after commissions[9] Interest Income - The company reported interest income of $3,463,000 for Q3 2025, slightly down from $3,545,000 in Q3 2024[25] Future Outlook - The company anticipates development goals to be achieved by mid-2028[19]

Core Viewpoint - Stoke Therapeutics, Inc. is focused on RNA medicine to restore protein expression and is developing zorevunersen as a potential disease-modifying treatment for Dravet syndrome [1] Company Overview - Stoke Therapeutics is a biotechnology company dedicated to harnessing the body's potential through RNA medicine [1] - The company has a lead investigational medicine, zorevunersen, which is positioned as a first-in-class treatment for Dravet syndrome [1] Upcoming Events - Management will host a webcast and conference call for analysts and investors on November 4, 2025, at 4:30 p.m. E [1]

Core Insights - Stoke Therapeutics, Inc. presented two-year data from the FALCON study, focusing on Autosomal Dominant Optic Atrophy (ADOA) [1] - The study involved 47 participants and was showcased at the 2025 American Academy of Ophthalmology Annual Meeting [1] Company Overview - Stoke Therapeutics is a biotechnology company that aims to restore protein expression through RNA medicines [1] - The company is dedicated to leveraging the body's potential in its therapeutic approaches [1] Study Details - The FALCON study is a prospective natural history study specifically targeting individuals with ADOA [1] - The results from this study provide significant insights into the condition of ADOA [1]

Core Insights - Stoke Therapeutics, Inc. (NASDAQ:STOK) is identified as a promising growth stock with a Buy rating and a price target of $35 set by H.C. Wainwright analyst Ananda Ghosh [1][2] Company Overview - Stoke Therapeutics is a biotech company focused on developing RNA-based medicines aimed at increasing protein expression to treat various diseases [3] - The company's lead drug, Zorevunersen, is specifically designed for treating Dravet syndrome [3] Drug Potential - Zorevunersen has shown promising results in a 36-month open-label extension study, indicating a long-term reduction in major motor seizure frequency and improvements in cognitive and behavioral aspects [2] - The drug is positioned to potentially become the first disease-modifying therapy for Dravet syndrome, differentiating itself from existing treatments that primarily focus on symptomatic relief [2][3]

Core Insights - Zorevunersen shows potential as a disease-modifying treatment for Dravet syndrome, with significant improvements in cognition and behavior observed over two years, contrasting with minimal changes in standard care [1][2][3] - In open-label extension studies, 95% of patients reported improvements in overall clinical status after three years of treatment with zorevunersen [1][2] Company Overview - Biogen Inc. and Stoke Therapeutics are collaborating on the development of zorevunersen, an investigational antisense oligonucleotide aimed at treating Dravet syndrome by increasing functional NaV1.1 protein production [8][9] - Zorevunersen has received orphan drug designation from the FDA and EMA, as well as Breakthrough Therapy Designation for specific mutations in the SCN1A gene [8] Clinical Data - The ongoing open-label extension studies indicate long-term benefits of zorevunersen on seizures, cognition, and behavior, supporting its potential for disease modification [3][4] - Safety data from the studies show that zorevunersen is generally well tolerated, with treatment-emergent adverse events (TEAEs) reported in 30% of patients in Phase 1/2a studies and 53% in open-label extension studies [4] Disease Context - Dravet syndrome is characterized by severe seizures and significant cognitive and behavioral impairments, with over 90% of patients experiencing seizures despite the best available treatments [7] - The estimated prevalence of Dravet syndrome in the U.S. is around 16,000 patients, with no approved disease-modifying therapies currently available [7][17]

Core Insights - Stoke Therapeutics and Biogen announced longer-term follow-up analyses from ongoing open-label extension studies of zorevunersen, indicating its potential as a disease-modifying medicine for Dravet syndrome [1] Company Overview - Stoke Therapeutics is a biotechnology company focused on restoring protein expression through RNA medicine [1] - Biogen Inc. is a biotechnology company that collaborates with Stoke Therapeutics on the zorevunersen studies [1] Product Development - The ongoing studies of zorevunersen are aimed at evaluating its effectiveness in treating Dravet syndrome, a severe form of epilepsy [1] - The new results from the studies support the potential of zorevunersen as a disease-modifying treatment [1]

Core Insights - Stoke Therapeutics, Inc. (STOK) shares increased by 11.7% to close at $30.5, with a notable trading volume compared to normal sessions, and a total gain of 23.7% over the past four weeks [1][2] Company Developments - The rise in stock price is linked to increased investor confidence in Stoke Therapeutics' lead candidate, zorevunersen, which is being developed as a first-in-class potential disease-modifying therapy for Dravet syndrome [2] - The company appointed Ian F. Smith as the new chief executive officer earlier this week [2] Financial Expectations - The company is projected to report a quarterly loss of $0.56 per share, reflecting a year-over-year decline of 19.2%, while revenues are expected to reach $5.7 million, an increase of 16.6% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Stoke Therapeutics is part of the Zacks Medical - Biomedical and Genetics industry, which includes other companies like Janux Therapeutics, Inc. (JANX), whose stock closed 6.7% higher at $24.2, but has seen a return of -4.6% over the past month [5] - Janux Therapeutics' consensus EPS estimate has changed by +0.1% over the past month to -$0.6, representing a year-over-year change of -17.7% [6]