Dyne Therapeutics：z-rostudirsen BLA 提交按計劃於 2026 年第二季進行

Dyne 已完成與 FDA 的正面 pre-BLA 會議，並按計劃於 2026 年第二季提交 z-rostudirsen 用於 exon 51 DMD 的美國加速核准 BLA。公司亦計劃於 2026 年第二季啟動全球確證性 Phase 3 試驗，假設獲得優先審查及核准，美國上市目標為 2027 年第一季。

關鍵事實

申報時間: 2026年5月11日上午11:35
事件: 申請已送件
方向: 正面
公司: DYNDyne Therapeutics
資產: z-rostudirsen
來源: SEC 8-K
可信度: 有出處

來源摘錄

EX-99.1 2 dyn-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Dyne Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Highlights - Positive pre-BLA meeting completed with FDA for z-rostudirsen in exon 51 DMD; on track for BLA submission in Q2 2026 and potential launch in Q1 2027 - - Positive cardiopulmonary results and long-term dystrophin data from Phase 1/2 DELIVER trial of z-rostudirsen in exon 51 DMD presented at MDA conference - - Completion of enrollment in registrational expansion cohort of Phase 1/2 ACHIEVE trial of z-basivarsen in DM1 on track for Q2 2026; global confirmatory Phase 3 HARMONIA trial initiated - - New preclinical data to be presented at ASGCT underscore differentiated capability of clinically validated FORCE TM platform to cross the blood-brain

SEC 8-K