原始来源记录
决策级顶线结果
Dyne Therapeutics：DELIVER 试验 REC 达到主要终点，肌营养不良蛋白水平为 5.46%
注册扩展队列达到了主要终点，肌营养不良蛋白水平统计学显著增加至正常的 5.46%。在六个月时，所有六项预设临床终点均观察到功能改善，肺功能得以维持。公司仍计划于 2026 年第二季度提交美国加速批准的 BLA。
关键事实
- 申报时间
- 2025年12月8日 11:36
- 事件
- 顶线结果
- 方向
- 积极
- 来源
- SEC 8-K
- 可信度
- 有来源
- 日历
- FDA 审批决定 (PDUFA) · 2027年1月21日 · 积极
来源摘录
EX-99.1 2 d16518dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD) - Registrational Expansion Cohort (REC) met primary endpoint, demonstrating statistically significant increase in dystrophin to 5.46% at 6 months (muscle content-adjusted; p<0.0001), replicating the same 7-fold change from baseline previously observed at the registrational dose - - Functional improvement was observed across multiple clinical endpoints at 6 months in REC; lung function was preserved at 6 months - - New positive long-term results from DELIVER trial showed sustained functional improvement across all assessed endpoints through 24 months - - Continued favorable safety and tolerability