Summary of Stoke Therapeutics Conference Call Company Overview - **Company**: Stoke Therapeutics (NasdaqGS:STOK) - **Focus**: Development of treatments for genetic diseases, specifically Dravet syndrome and Autosomal Dominant Optic Atrophy (ADOA) [2][6] Key Points on Dravet Syndrome - **Disease Understanding**: Dravet syndrome is a severe form of epilepsy affecting young children, leading to significant developmental challenges [2][3] - **Clinical Data**: Initial phase 1/2 data showed an 80% reduction in seizures on top of standard care medications, with durable effects observed over nearly four years [4][9] - **Cognitive and Behavioral Benefits**: Improvements in cognition and behavior were noted, measured by the Vineland-3 assessment, indicating potential for neurodevelopmental progress in children who typically plateau after 18 months [5][10] - **Phase 3 Program**: Enrollment for the phase 3 study is expected to complete in Q2 2026, with data anticipated in mid-2027 [6][28] Market Opportunity - **Patient Population**: Approximately 36,000-40,000 patients globally, with 16,000 in the U.S. and 6,000 currently addressable [50][51] - **Pricing Strategy**: The company aims to position its treatment as a disease-modifying therapy, potentially aligning pricing with similar therapies like Spinraza [52][54] Regulatory Pathway - **FDA Interaction**: The company is pursuing accelerated approval but faced challenges due to the FDA's request for additional data. A follow-up discussion is planned to present data against patient baseline rather than cross-study comparisons [34][35][36] - **Optimal Label**: The goal is to achieve a label that reflects both seizure reduction and cognitive improvements, which is crucial for market positioning and pricing discussions [41][42] ADOA Development - **New Indication**: The first patient for ADOA was dosed recently, targeting a genetic loss of eyesight by upregulating the OPA1 gene to improve mitochondrial function and vision [56][57] - **Market Size**: The ADOA market is estimated at around 16,000 patients in the top seven geographies [58] Financial Position - **Cash Reserves**: The company reported approximately $400 million in cash at the end of 2025, with funding from Biogen covering 30% of Dravet R&D costs, providing a runway into 2028 [60]

BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, today announced that the first patient has been dosed in the Phase 1 OSPREY study of STK-002, an investigational medicine for the treatment of Autosomal Dominant Optic Atrophy (ADOA). ADOA is a rare genetic disease that causes progressive and irreversible vision loss in both eyes starting in the first de. ...

New Headquarters Will Support Company's Work to Deliver zorevunersen, a Potential Disease-Modifying Medicine, to Patients with Dravet Syndrome and Expansion of Research EffortsLease is One of the Largest in Greater Boston Life Sciences Over the Past YearWALTHAM, Mass., Jan. 27, 2026 /PRNewswire/ -- Anchor Line Partners, LLC and Northwood Investors, LLC announced today that they have secured a significant long-term lease with biotechnology company Stoke Therapeutics (Nasdaq: STOK) at 245 Fifth Avenue in Wal ...

Core Insights - Stoke Therapeutics is a clinical-stage biotechnology company focused on developing disease-modifying therapies for Dravet syndrome and ADOA using its TANGO RNA platform [1] Company Overview - The company is currently advancing Zorevunersen, which is in Phase 3 clinical trials and has presented four-year positive Open Label Extension (OLE) data [1]

Company Overview - Stoke Therapeutics (STOK) is a clinical-stage biotechnology company focused on developing disease-modifying therapies for Dravet syndrome and ADOA using its TANGO RNA platform [1]. Product Development - The company’s lead product, Zorevunersen, is currently in Phase 3 clinical trials and has demonstrated positive four-year open-label extension (OLE) data [1]. - Another product in development is STK-002, aimed at addressing ADOA [1].

Summary of Stoke Therapeutics FY Conference Call Company Overview - **Company**: Stoke Therapeutics (NasdaqGS:STOK) - **CEO**: Ian Smith, in role since March 2025 - **Focus**: Upregulating protein expression for haplo-insufficient diseases, primarily targeting genetic conditions like Dravet syndrome and Autosomal Dominant Optic Atrophy (ADOA) [2][3] Key Developments - **Lead Medicine**: Zorevunersen, targeting Dravet syndrome by upregulating the SCN1A gene to increase NaV1.1 expression, aiming to reduce seizures and improve neurodevelopment [3][4] - **Clinical Trials**: - Currently in Phase 3 for Dravet syndrome with a target enrollment of 150 patients, approximately 60 patients have been randomized, and another 60 are in the screening phase [5][6] - Enrollment expected to complete by Q2 2026, with potential NDA submission in 2027 or early 2028 [6][20] - **Long-term Data**: Patients have been followed for up to four years, showing durability in seizure reduction (up to 85%) and improvements in neurocognition [7][10][18] Financial Position - **Funding**: Approximately $400 million available, sufficient to fund operations through 2028 [8][9] - **Collaboration**: Partnership with Biogen, which licensed commercial rights to zorevunersen and funds 30% of R&D development [21][22] Market Opportunity - **Patient Population**: Estimated 40,000 patients with Dravet syndrome across key geographies (15-20,000 in the U.S. and Europe) [20][21] - **Commercial Potential**: Anticipated robust demand for a disease-modifying treatment, with market research indicating high clinician willingness to prescribe [41][42] Regulatory Pathway - **Breakthrough Designation**: Zorevunersen has received this designation, allowing for expedited development discussions with the FDA [27][28] - **FDA Interaction**: Productive discussions with the FDA regarding the potential for filing based on Phase 1, 2, and OLE data, although the timeline for filing may not be significantly shortened [28][30] Pipeline and Future Plans - **Next Steps**: - Completion of Phase 3 enrollment and subsequent NDA filing [24][25] - Initiation of clinical trials for ADOA (STK-002) and SYNGAP1, with expectations for early efficacy results by the end of 2026 [50][51] - **Regulatory Strategy**: Focus on demonstrating improvement in vision for ADOA, which could shorten clinical study durations [52][53] Additional Insights - **Data Integrity**: Emphasis on the importance of long-term data and observed outcomes in regulatory discussions to support claims of disease modification [45][46] - **Market Positioning**: Differentiation from existing seizure medications by positioning zorevunersen as a true disease-modifying treatment [48][49] This summary encapsulates the critical points discussed during the Stoke Therapeutics FY Conference Call, highlighting the company's strategic focus, clinical advancements, financial health, and regulatory strategies.

Group 1 - The article does not provide any relevant content regarding company or industry insights [1]

Zorevunersen for Dravet Syndrome - Stoke Therapeutics is advancing zorevunersen as a potential disease-modifying therapy for Dravet Syndrome[9] - Phase 3 study (EMPEROR) enrollment is expected to complete in Q2 2026 with data readout in mid-2027 to support NDA[12, 38] - Approximately 330 patients have been identified globally for the EMPEROR study, with ~60 in screening and ~60 randomized[12, 39] - Data from ongoing OLE studies show continuing improvements in cognition and behavior over 36 months[24] - Zorevunersen demonstrated significant reduction in major motor seizure frequency at 6 months compared to natural history data, with an 8248% reduction compared to a 2043% reduction in the natural history group (P=00181)[26] - In OLE studies, 86% of patients experienced CSF protein elevation, classified as a TEAE in 45%[31] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - A Phase 1 study of STK-002 for ADOA has been initiated in the UK and Europe[11] Financial Status and Collaboration - Stoke Therapeutics had approximately $3917 million in cash, cash equivalents, and marketable securities as of December 31, 2025, with funding anticipated into 2028[12] - A strategic collaboration with Biogen exists for the development and commercialization of zorevunersen, with Biogen holding exclusive commercialization rights outside of the US, Canada, and Mexico[48] - The Biogen collaboration includes $165 million upfront, shared development costs, and potential milestone payments of $385 million[49] Market Opportunity - There are approximately 38,000 patients with Dravet syndrome across 7 major markets[40, 44]

Clinical Development and Regulatory Pathways - The Company expects to complete enrollment of 150 patients in the second quarter of 2026, with a data readout in mid-2027 anticipated to support the submission of a New Drug Application (NDA) to the FDA[11] - The Company held a multidisciplinary meeting with the FDA to discuss the ongoing clinical development of zorevunersen, exploring potential expedited regulatory pathways, with ongoing discussions for additional information[11] - The Company plans to initiate a rolling NDA submission in the first half of 2027[11] - The Company anticipates that the ongoing EMPEROR study will provide critical data for future product development and regulatory submissions[5] - The Company is focused on advancing zorevunersen to treat Dravet syndrome, with expectations for significant clinical trial results[10] Financial Position and Projections - The Company has approximately $391.7 million in cash, cash equivalents, and marketable securities as of December 31, 2025, which, combined with eligible proceeds from the Biogen collaboration, is expected to fund operations into 2028[11] - The preliminary financial information is subject to completion of financial closing procedures and may differ from actual results in the audited financial statements[6] Strategic Collaborations and Risk Management - The Company is collaborating with Acadia Pharmaceuticals and Biogen International to enhance its development activities and achieve strategic goals[10] - The Company is actively managing risks related to global business conditions, including inflation and geopolitical conflicts, which may impact operations[10] Intellectual Property Strategy - The Company is committed to protecting its intellectual property as part of its overall strategy for product commercialization[10]

Core Viewpoint - Stoke Therapeutics, Inc. is advancing its investigational medicine zorevunersen in collaboration with Biogen, targeting Dravet syndrome as a potential first-in-class disease-modifying treatment [1] Group 1: Company Overview - Stoke Therapeutics is focused on restoring protein expression through RNA medicine [1] - The company is developing zorevunersen as its lead investigational medicine [1] Group 2: Development Updates - The company announced accelerated timelines for the completion of enrollment in the clinical trial [1] - A Phase 3 data readout from the EMPEROR study is expected soon [1]