FORCE Data @ASGCT Shows Impressive CNS Delivery; Z-ros BLA in 2Q:26

研报英文原文证据摘录

FORCE Data @ASGCT Shows Impressive CNS Delivery; Z-ros BLA in 2Q:26

q2W doses of 3mg/kg siRNA with a CNS- Shares Out (mil) 165.3

optimized TfR1 Fab decreased MAPT RNA by ~75% consistently across deep and Form 10Q as of May 8th

cortical brain regions. KD was stable for at least 4 weeks. MAPT KD translated into Market Cap. (mil) US$3,048.1

a >50% reduction in tau protein in both the entorhinal cortex and hippocampus, key Total Assets ($mil) 1,080

regions of tau accumulation in Alzheimer's disease. Dyne also showed that in humanized Avg Daily Vol (000) 2,053

Book Value/Share US$5.23 mice, subcutaneous siRNA could KD MAPT to a similar level as IV administration. We

Price/Book 353%

are impressed by the depth of MAPT KD with systemic delivery, especially in hard-to- Net Cash Per Share US$4.98

reach deep brain regions. We see blockbuster potential for tau-lowering therapies for 1Q:26 cash less Hercules debt

the treatment of tauopathies like Alzheimer's Disease, progressive supranuclear palsy, Debt to Total Capital 13.8%

and many cases of Parkinson's Disease. Div Yield 0.00%

Fiscal Year End Dec

• Z-rostudirsen BLA in 2Q:26; Potential Approval by 1Q:27. Dyne previously reported

Price Performance - 1 Year

that 20mg/kg q4W Z-rostudirsen (n=21) increased muscle-content adjusted dystrophin

by 4.63% to 5.46% vs. a 0.03% increase with placebo (n=7) to 0.95% at 6 months. USD

(p<0.0001) The safety profile remains favorable. Dyne will file the BLA for z-rostudirsen 25

in 2Q:26 and seek priority review. We anticipate accelerated approval and U.S. launch 20

in 1Q:27. Dyne plans to initiate a confirmatory Phase III trial in 2Q:26. Beyond the lead z-

rostudirsen (Exon 51) program, Dyne is also leveraging its FORCE platform to advance 15