Stoke Therapeutics近日宣布，其针对Dravet综合征的潜在疾病修饰疗法zorevunersen的全球III期EMPEROR研究已完成全部162名患者的入组工作。这一里程碑的达成，标志着该药物向提交新药申请、尽快惠及患者迈出了关键一步。
此前，公司预计将在2026年第二季度完成约150名患者的入组。此次公布的162名患者入组数量超出了这一预期。入组患者来自美国、英国和日本等多个国家和地区，他们将被随机分配接受zorevunersen或安慰剂治疗。
Zorevunersen是一款首创的反义寡核苷酸药物，旨在通过增加脑细胞中非突变SCN1A基因拷贝的功能性NaV1.1蛋白表达，从根本上治疗Dravet综合征。该药物已获得美国食品药品监督管理局的突破性疗法认定和孤儿药资格。
公司的目标是在2027年中公布EMPEROR研究的顶线数据，并计划在2027年上半年启动滚动新药申请，目标是在2027年底或2028年初实现潜在上市。公司当前拥有约4.11亿美元的资金，足以支持该药物研发直至潜在的上市阶段。
责任编辑：张俊 SF065
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