Z-rostudirsen BLA Submitted, U.S. Launch Anticipated by 1Q:27

Research evidence excerpt

Z-rostudirsen BLA Submitted, U.S. Launch Anticipated by 1Q:27

s (n=21) achieved a statistically Form 10Q as of May 8th

significant improvement in rise from floor (RFF) velocity of 0.04 rise/second vs. pooled Market Cap. (mil) US$2,871.3

placebo (n=18, p<0.05), exceeding the MCID. Z-ros increased muscle-content adjusted Total Assets ($mil) 1,080

dystrophin by 4.63% to 5.46% vs. a 0.03% increase with placebo (n=7) to 0.95% at Avg Daily Vol (000) 2,010

Book Value/Share US$5.23 6 months. (p<0.0001). Dyne initiated the confirmatory Phase III FORZETTO trial to

Price/Book 332%

randomize 90 DMD-51 boys aged 4-18 years (1:1) to 20mg/kg z-ros or placebo in with Net Cash Per Share US$4.98

RFF at 73 weeks as the primary endpoint. Dyne is also developing additional FORCE 1Q:26 cash less Hercules debt

exon-skipping candidates including DYNE-253, DYNE-245, DYNE-244 and DYNE-255. Debt to Total Capital 13.8%

Div Yield 0.00%

• Z-basivarsen ACHIEVE Data in 1Q:27; Confirmatory Trial Enrolling. Dyne expects Fiscal Year End Dec

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

Price Performance - 1 Year

of z-basivarsen in 2Q:26 with data anticipated by 1Q:27. Dyne plans to file a BLA with

potential accelerated approval by early 3Q:27 and launch in 1Q:28. Dyne has initiated USD

the confirmatory Phase III HARMONIA trial to randomize ~150 DM1 patients (1:1) to 25

6.8mg/kg z-basivarsen q8W or placebo for 48 weeks followed by a 24-week OLE. The 20

primary endpoint of HARMONIA is change from baseline in 5-time sit to stand (5TSS),

and the trial is well-powered to detect a change of 1.5-2.0 seconds. HARMONIA is 15

also powered to detect effects on multiple secondary endpoints that include function,

strength, and CNS-related symptoms. 10