Dyne Therapeutics：DELIVER 試驗 REC 達到主要終點，肌營養不良蛋白達 5.46%

註冊擴展隊列達到主要終點，肌營養不良蛋白顯著增加至正常的 5.46%。六個月時，所有六項預先指定的臨床終點均觀察到功能改善，肺功能得以維持。公司仍按計劃於 2026 年第二季提交美國加速批准的 BLA。

關鍵事實

申報時間: 2025年12月8日上午11:36
事件: 主要結果 (Topline)
方向: 正面
公司: DYNDyne Therapeutics
資產: z-rostudirsen
來源: SEC 8-K
可信度: 有出處
行事曆: FDA 審查決定 (PDUFA) · 2027年1月21日 · 正面

來源摘錄

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

SEC 8-K