Dyne, DMD 대상 z-rostudirsen의 Q2 2026 BLA 제출 계획

The company announced it is on track to submit a BLA for U.S. Accelerated Approval of z-rostudirsen in Q2 2026, following positive topline Phase 1/2 DELIVER trial results. A potential U.S. launch is guided for Q1 2027 assuming Priority Review and approval. The registrational expansion cohort met its primary endpoint with statistically significant dystrophin expression and showed functional improvement across all six prespecified endpoints.

핵심 사실

제출: 2026년 3월 2일 PM 12:35
이벤트: 신청 제출
방향: 긍정적
기업: DYN다인 세라퓨틱스
자산: z-rostudirsen
출처: SEC 8-K
신뢰도: 출처 확인
배수 변화: 기저 대비 7배 변화
기능적 평가변수: 6개 사전 지정 탑라인 기능적 평가변수 모두에서 위약 대비 개선
디스트로핀 발현: 6개월 시점 정상 대비 5.46% (p<0.0001)

출처 발췌

EX-99.1 2 dyn-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Dyne Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Recent Business Highlights - Planned submission for U.S. Accelerated Approval of z-rostudirsen on track for Q2 2026; potential launch in Q1 2027 - - Positive topline results reported from Phase 1/2 DELIVER trial of z-rostudirsen in exon 51 skip amenable Duchenne muscular dystrophy (DMD); additional long-term data to be presented at MDA - - Completion of enrollment in registrational expansion cohort of Phase 1/2 ACHIEVE trial of z-basivarsen in myotonic dystrophy type 1 (DM1) expected in Q2 2026; Phase 3 trial design to be presented at MDA - - Advancing four development candidates for the potential treatment of DMD amenable to skipping of exons 53, 45, 4

SEC 8-K