**公司及行业概述** - **公司**：Stoke Therapeutics，专注于基因疗法，主要项目针对Dravet综合征（一种罕见儿科遗传病）和常染色体显性视神经萎缩（ADOA）[1][3][5][8] - **行业**：生物技术，聚焦罕见病治疗，涉及中枢神经系统（CNS）和眼科疾病领域[1][8] --- **核心观点与论据** **1 Dravet综合征项目进展** - **临床数据**： - 患者用药3-4年后显示癫痫发作显著减少（具体数据未量化），且效果持久[5][6] - 认知和行为改善长期持续，例如患者恢复表达/接受性沟通能力（Vineland评分提升8-9分，临床意义阈值为2-3分）[6][45][49] - 安全性：累计给药超800剂，无临床显著不良反应[18][50] - **机制**：通过上调Nav1.1蛋白（Dravet的根源缺陷）改善症状[7] - **监管进展**： - 2024年获FDA突破性疗法认定（针对综合征整体，非仅癫痫）[11] - 计划2024年下半年与FDA讨论加速审批路径（可能跳过III期）[12][17] **2 ADOA项目启动** - **机制**：通过上调OPA1基因增强线粒体功能，改善视力[8][51] - **临床前数据**：非人灵长类模型中显示视力提升（非仅稳定）[52] - **计划**：2024年下半年启动I/II期试验[8][53] **3 商业化与合作伙伴** - **Biogen合作**： - 覆盖北美以外市场（70+国家），Biogen承担30%研发成本[9][25][27] - 目标利用Biogen的全球渠道加速上市（如欧洲、日本）[30][32] - **定价策略**：北美自主定价，其他地区由Biogen主导[34] **4 财务与资本** - **现金储备**：3.55亿美元，预计资金充足至2028年（覆盖III期数据读出）[9] --- **其他重要细节** - **III期试验设计**： - 170名患者，主要终点为28周癫痫减少，次要终点52周认知改善（Vineland-3）[36][37] - 预筛选150名患者，预计2025年下半年完成入组，2027年数据读出[37][38] - **潜在加速路径**： - 可能基于长期安全性和疗效数据争取有条件批准，但无明确生物标志物（需脑活检）[19][20] - 欧洲或英国可能更灵活[32] - **风险因素**： - 脑脊液蛋白升高（与给药方式相关，无临床影响）[50] - III期试验需平衡揭盲风险与中期分析需求[40] --- **关键数据引用** - **财务**：3.55亿美元现金[9] - **临床**：800+剂量给药，3-4年安全性数据[18][50] - **疗效**：Vineland评分提升8-9分（阈值2-3分）[45][49] （注：部分数据如癫痫减少百分比未明确量化，需后续披露）

Stoke Therapeutics (STOK) Q2 2025 Earnings Call August 12, 2025 04:30 PM ET Speaker0Good afternoon and welcome to Stokes Therapeutics second quarter twenty twenty five conference call. I'm Tommy Leggett, Chief Financial Officer at Stokes. Joining me for prepared remarks are Ian Smith, our Interim Chief Executive Officer Doctor. Barry Tico, Chief Medical Officer and Doctor. Kimberly Parkerson, Head of Neurology Clinical Development.In addition, Jason Hoyt, our Chief Patient Officer will participate in Q and ...

Stoke Therapeutics Second Quarter 2025 Business Update Webcast for Investors & Analysts August 12, 2025 © COPYRIGHT 2025 | STOKE THERAPEUTICS | 1 Agenda CEO Opening Remarks Ian F. Smith, Interim Chief Executive Officer & Director Phase 3 Design and Progress Barry Ticho, M.D., Ph.D., Chief Medical Officer Long-Term 36-Month OLE Data Kimberly Parkerson, M.D., Ph.D., SVP, Head of Neurology Clinical Development Pipeline Update: ADOA Preclinical Data and Development Plan Barry Ticho, M.D., Ph.D., Chief Medical O ...

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 June 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 jurisdi ...

EX 99.1 – Webcast and conference call for analysts and investors at 4:30PM Eastern Time today – BEDFORD, Mass., August 12, 2025 – Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the second quarter end ...

文章核心观点 Biogen和Stoke Therapeutics公布评估zorevunersen 3期给药方案潜在影响的分析数据 该数据显示第68周时患者认知和行为有改善 支持将评估认知和行为的关键次要终点纳入3期EMPEROR研究 zorevunersen有望成为治疗Dravet综合征的首创疾病修饰疗法 [1] 研究结果 - 分析数据显示zorevunersen 3期给药方案使患者第68周时认知和行为有改善 与自然病史数据结果形成对比 [1] - 此前1/2a期和开放标签扩展（OLE）研究数据显示 zorevunersen在标准抗癫痫药物基础上大幅且持久降低主要运动性癫痫发作频率 并改善认知和行为 70mg负荷剂量后45mg维持剂量的患者反应可能更好 且总体耐受性良好 [2] Dravet综合征情况 - Dravet综合征是严重的发育性癫痫性脑病 以严重复发性癫痫发作以及显著的认知和行为障碍为特征 多数由SCN1A基因突变导致 超90%患者经现有最佳抗癫痫药物治疗后仍会发作 还会引发诸多并发症和较高的癫痫猝死风险 全球均有发生 美国、英国、欧盟4国和日本约有3.8万人患病 [7] zorevunersen相关信息 - zorevunersen是一种研究性反义寡核苷酸 旨在通过增加SCN1A基因非突变拷贝的NaV1.1蛋白产生来治疗Dravet综合征的根本原因 具有疾病修饰潜力 已获FDA和EMA孤儿药指定、FDA罕见儿科疾病指定和突破性疗法认定 [8] - Stoke与Biogen合作开发和商业化zorevunersen治疗Dravet综合征 Stoke保留美国、加拿大和墨西哥独家权利 Biogen获得其他地区独家商业化权利 [8] 公司情况 - Stoke Therapeutics是一家致力于利用RNA药物恢复蛋白质表达的生物技术公司 采用专有TANGO方法开发反义寡核苷酸 首个在研药物zorevunersen在Dravet综合征患者中显示出疾病修饰潜力 正进行3期研究 [9] - Biogen成立于1978年 是一家领先的生物技术公司 致力于开创创新科学 为患者提供新药 为股东和社区创造价值 [10] 研究分析方法 - 采用重复测量混合效应模型（MMRM）分析评估zorevunersen 3期给药方案对患者第68周时认知和行为的潜在影响 该模型使用1/2a期ADMIRAL研究和LONGWING OLE研究的临床数据开发 [4] - 将BUTTERFLY自然病史研究中患者的基线协变量与选定的ADMIRAL患者群体进行匹配 zorevunersen治疗患者的改善情况与BUTTERFLY研究结果形成对比 [5] - 这些数据支持选择Vineland - 3适应行为量表的五个子领域作为3期EMPEROR研究的关键次要终点 [5] 研究展示信息 - 研究结果在第16届欧洲儿科神经病学学会（EPNS）大会的癫痫II会议上展示 标题为“Zorevunersen demonstrates potential as a disease modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open - label extension studies” 演讲者为Andreas Brunklaus [6] - 演讲资料可在Stoke Therapeutics网站的投资者与新闻板块下载 [6]

Stoke Therapeutics (STOK) 2025 Conference June 04, 2025 01:25 PM ET Speaker0 Yeah. I mean, it truly does differentiate your medicine. Speaker1 Right. Are you ready? Yep. Okay. We're gonna get started with our next session. I'm Andrew Tsai, senior biotech analyst at Jefferies. Thanks for joining me today. It's my pleasure to have Stokes Interim CEO, Ian Smith, by my side today. Welcome, Ian. Speaker0 Thank you, Andrew. Speaker1 Thank you. Yep. And so maybe spend the next couple minutes discussing Stokes, wha ...

Stoke Therapeutics, Inc. (STOK) closed the last trading session at $9.63, gaining 25.2% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $22.38 indicates a 132.4% upside potential.The average comprises eight short-term price targets ranging from a low of $15 to a high of $35, with a standard deviation of $6.82. While the lowest estimate indicates an increase of 55.8% from the curren ...

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 March 31, 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 jurisd ...

Stoke Therapeutics, Inc. (STOK) came out with quarterly earnings of $1.90 per share, beating the Zacks Consensus Estimate of a loss of $0.37 per share. This compares to loss of $0.57 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 613.51%. A quarter ago, it was expected that this company would post a loss of $0.51 per share when it actually produced a loss of $0.18, delivering a surprise of 64.71%.Over the last four quarters, ...