Cash of $972M; Z-rostudirsen BLA in 2Q:26; ACHIEVE DM1 Data in 1Q:27

研报英文原文证据摘录

Cash of $972M; Z-rostudirsen BLA in 2Q:26; ACHIEVE DM1 Data in 1Q:27

showing that muscle-adjusted dystrophin to 18.33% at 12 months or later, indicating the Shares Out (mil) 165.3

benefit of continued treatment. Further, z-rostudirsen treated boys showed numerical Form 10Q as of May 8th

improvements in lung function (FVC) and heart function (LVEF) at 24 months, which Market Cap. (mil) US$2,910.9

contrast with the steady decline observed in natural history data. The safety profile Total Assets ($mil) 1,080

remains favorable. Dyne will file the BLA for z-rostudirsen in 2Q:26 and seek priority Avg Daily Vol (000) 2,070

Book Value/Share US$5.23 review. We anticipate accelerated approval and U.S. launch in 1Q:27. Dyne plans to

Price/Book 337%

initiate a confirmatory Phase III trial in 2Q:26. Beyond the lead z-rostudirsen (Exon Net Cash Per Share US$4.98

51) program, Dyne is also leveraging its FORCE platform to advance a pipeline of 1Q:26 cash less Hercules debt

additional exon-skipping candidates, including DYNE-253, DYNE-245, DYNE-244, and Debt to Total Capital 13.8%

DYNE-255. Div Yield 0.00%

Fiscal Year End Dec

• Z-basivarsen ACHIEVE Data in 1Q:27; Confirmatory Trial Initiated. Dyne expects

Price Performance - 1 Year

to exceed target enrollment of >60 DM1 patients in the registrational ACHIEVE cohort

of z-basivarsen with data anticipated in 1Q:27. Dyne plans to file a BLA with potential USD

accelerated approval in early 3Q:27 with potential launch in 1Q:28. Dyne has initiated the 25

confirmatory Phase III HARMONIA trial to randomize ~150 DM1 patients (1:1) to 6.8mg/ 20

kg z-basivarsen q8W or placebo for 48 weeks followed by a 24-week OLE. The primary

endpoint is change from baseline in 5-time sit to stand (5TSS), and HARMONIA is well 15