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]

Core Insights - Stoke Therapeutics, a biotech company focused on rare genetic diseases, experienced a notable insider sale by Chief Medical Officer Barry Ticho, who sold 5,358 shares for approximately $167,115 following significant share price gains in 2025 [1][8]. Company Overview - Stoke Therapeutics specializes in developing antisense oligonucleotide (ASO) medicines targeting severe genetic diseases, with lead candidates STK-001 for Dravet syndrome (currently in Phase I/IIa trials) and STK-002 for autosomal dominant optic atrophy (in preclinical stage) [5][7]. - As of December 5, 2025, the company's stock price was $31.19, with a market capitalization of $1.74 billion and a revenue of $205.63 million for the trailing twelve months (TTM) [4]. Financial Performance - The company reported $328.6 million in cash, cash equivalents, and marketable securities, alongside $183 million in year-to-date revenue, indicating a strong financial position expected to fund operations until 2028 [9]. Insider Activity - Barry Ticho's recent sale of shares, while smaller than his historical median sale, represented a larger proportion of his remaining holdings (21.30% compared to a long-term median of 6.84%), reflecting a decline in his available share base [6]. - The shares were sold at a weighted average price of approximately $31.19, closely aligning with recent trading levels, indicating minimal price slippage [6]. Market Context - Stoke Therapeutics' stock price has appreciated significantly, reaching a 52-week high of $38.69 on October 16, 2025, and remaining above $30 per share in December, driven by favorable clinical trial data and strategic collaborations, particularly with Biogen [8][9].

Group 1: Market Movements - Biotech stocks experienced significant fluctuations in after-hours trading, with several companies rebounding after substantial losses during regular sessions, driven by fresh clinical data, FDA designations, and financing announcements [1] - Stoke Therapeutics, Inc. (STOK) saw its stock rise to $33.95 (+11.42%) in post-market hours after closing at $30.47 (-7.67%), following positive data presentations with Biogen regarding zorevunersen for Dravet syndrome [2] - Alpha Tau Medical Ltd. (DRTS) experienced a notable increase in share price, climbing to $5.30 (+25.26%) after closing at $4.23 (+8.74%), following the treatment of the first patient in its pilot study for recurrent glioblastoma multiforme [3] Group 2: Company Announcements - Absci Corp. (ABSI) rose to $3.65 (+3.69%) after hours, building on a close of $3.52 (+2.03%), with no new announcements but previously reported first volunteer dosing in its Phase 1/2a HEADLINE study of ABS-201 [4] - Senti Biosciences, Inc. (SNTI) surged to $1.60 (+3.92%) after hours, despite a sharp decline to $1.54 (-35.56%) during regular trading, driven by the FDA granting RMAT designation to its investigational CAR-NK cell therapy for AML and other hematologic malignancies [5] - TuHURA Biosciences, Inc. (HURA) edged up to $1.18 (+0.85%) after hours, following a significant decline to $1.17 (-40.91%) at the close, announcing a registered direct offering of 9.4 million shares priced at $1.65 per share [6]