– Despite treatment with standard-of-care anti-seizure medicines, children with Dravet syndrome experienced high seizure burden and plateaued in neurodevelopment, resulting in a widening gap relative to children with typical development – – Findings underscore the urgent need for new medicines that target the underlying genetic cause of Dravet syndrome to improve long-term outcomes – CAMBRIDGE, Mass. and BEDFORD, Mass., Nov. 17, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc ...

Summary of Stoke Therapeutics FY Conference Call Company Overview - **Company**: Stoke Therapeutics (NasdaqGS:STOK) - **Industry**: Biotechnology, focusing on genetic diseases, specifically Dravet syndrome and other haploinsufficient diseases Key Points and Arguments Leadership Transition - Ian Smith transitioned from interim CEO to full-time CEO, having been associated with Stoke for nearly three years, including roles as a board member and advisor [2][3] Product and Pipeline - The primary focus is on a treatment for Dravet syndrome, a genetic disorder characterized by severe seizures due to a lack of NAV1.1 protein in the brain, caused by a mutated SCN1A gene [6][10] - The treatment aims to upregulate the NAV1.1 protein, addressing the root cause of the disease, leading to significant reductions in seizure frequency (up to 80-85% median reduction) and potential improvements in neurodevelopment [10][11] - The company is currently in Phase 3 trials, with a pipeline that includes other haploinsufficient diseases in Phase 1 or preclinical stages [4][6] Clinical Data and Efficacy - The treatment has shown sustained and durable reductions in seizures over nearly four years, which is unique compared to traditional anti-seizure medications [10][14] - Cognitive and behavioral improvements have been observed, with children showing enhanced communication and motor skills as measured by the Vineland 3 score [11][12] - The safety profile is generally well-tolerated, with 90% of patients from the Phase 1/2 study continuing into the open-label extension (OLE) study [13][14] Phase 3 Study Details - The Phase 3 study includes a six-week screening period, with a primary endpoint focused on seizure reduction at week 28 and secondary endpoints at week 52 [19][20] - The study is well-powered with a 90% confidence level for a 0.01 result, and over 25 patients have been dosed so far [20][23] - Full enrollment of 170 patients is expected by the second half of 2026, with data readout anticipated in the second half of 2027 [23][24] Regulatory and Commercial Strategy - The company received breakthrough therapy designation from the FDA for the treatment of Dravet syndrome in December 2024, acknowledging the safety and efficacy profile [25][26] - A multidisciplinary meeting with the FDA is scheduled for December to discuss the drug's mechanism of action and safety data, with the potential to expedite the approval pathway [25][27] - The commercial opportunity for Dravet syndrome is significant, with an estimated 15,000 to 20,000 diagnosed patients in the U.S. and a similar number outside the U.S. [31][32] Other Programs - The company is also developing a treatment for Autosomal Dominant Optic Atrophy (ADOA) in Phase I, targeting the OPA1 gene to improve mitochondrial function and vision [34][35] - A preclinical program for SYNGAP1, which involves seizures and neurodevelopment issues, is expected to have a development candidate by early 2026 [37] Financial Health - The company reported a strong financial position with approximately $400 million in cash, expected to fund operations through mid-2028, including the Phase 3 study and other programs [38] Additional Important Information - The treatment's administration involves lumbar puncture, which may lead to transient elevations in cerebrospinal fluid (CSF) protein, but no clinical manifestations have been observed [15][16] - The company is focused on educating the advocacy community and key opinion leaders to drive demand for the study [23][24]

Core Viewpoint - Stoke Therapeutics, Inc. (STOK) has received a Zacks Rank 2 (Buy) upgrade due to an upward trend in earnings estimates, which is a significant factor influencing stock prices [1][3]. Earnings Estimates and Stock Price Movement - The Zacks rating system emphasizes the importance of changing earnings estimates in determining near-term stock price movements, making it a valuable tool for investors [2][4]. - The correlation between earnings estimate revisions and stock price movements is strong, particularly influenced by institutional investors who adjust their valuations based on these estimates [4]. Company Performance and Outlook - The upgrade for Stoke Therapeutics indicates an improvement in the company's underlying business, suggesting that investors may respond positively by driving the stock price higher [5]. - Analysts have raised their earnings estimates for Stoke Therapeutics, with the Zacks Consensus Estimate increasing by 75.1% over the past three months [8]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with a proven track record of performance, particularly for Zacks Rank 1 stocks, which have averaged a +25% annual return since 1988 [7]. - The upgrade to Zacks Rank 2 places Stoke Therapeutics in the top 20% of Zacks-covered stocks, indicating strong potential for market-beating returns in the near term [10].

Core Insights - Stoke Therapeutics reported a quarterly loss of $0.65 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.54, marking a year-over-year increase from a loss of $0.47 per share [1] - The company achieved revenues of $10.63 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 69.03% and showing significant growth from $4.89 million in the same quarter last year [2] - Stoke Therapeutics shares have increased approximately 136.6% year-to-date, significantly outperforming the S&P 500's gain of 16.5% [3] Earnings Outlook - The future performance of Stoke Therapeutics' stock will largely depend on management's commentary during the earnings call and the company's earnings outlook [4] - The current consensus EPS estimate for the upcoming quarter is -$0.65 with projected revenues of $6.13 million, while the estimate for the current fiscal year is $0.28 on revenues of $183.79 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Stoke Therapeutics belongs, is currently ranked in the top 39% of over 250 Zacks industries, indicating a favorable outlook compared to lower-ranked industries [8]

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 ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2025 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________ to ____________ Commission File Number: 001-38938 Stoke Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) (State or other ju ...

EX 99.1 Stoke Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Updates – Global Phase 3 EMPEROR study patient recruitment ongoing in the U.S., UK, and Japan with more than 20 patients randomized to zorevunersen or sham; Study on track to complete enrollment in second half of 2026 – – As of September 30, 2025, the Company had $328.6 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to mid-2028 – – Webcast and conference call for analys ...

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]