WVE 最新10-Q变化

相对上期新增的文字 · 来源:10-Q · 2026-07-30

• We expect to initiate combination and maintenance trials of WVE-007 in the second half of 2026.

• We have shared interim data from the 200 mg and 400 mg single and multidose cohorts, as well as data from the 600 mg single dose cohort of RestorAAtion-2. Key highlights include:

• Robust, dose-dependent reductions of circulating, mutant Z-AAT from baseline: 70.5% in the 200 mg biweekly dose cohort (seven doses) and 67.7% in the 400 mg monthly dose cohort (four doses).

• Robust, dose-dependent restoration of wild-type M-AAT protein (canonical M-AAT) as a percentage of total circulating AAT: 64.4% in the 200 mg biweekly dose cohort (seven doses) and 58.7% in the 400 mg monthly dose cohort (four doses).

• Restoration of dynamic AAT response: Three instances of dynamic and rapid production of serum AAT protein due to acute phase responses were observed across RestorAATion-2 as indicated by concurrent C-reactive protein (CRP) and AAT elevation; this included a total AAT level of 20.6 µM and M-AAT level…

相对上期删除的文字 · 来源:10-Q · 2026-04-28

• We expect to initiate the Phase 2a portion of INLIGHT in the second quarter of 2026. We expect to initiate combination and maintenance trials of WVE-007 in 2026.

• In September 2025, we announced positive data from the 200 mg single and multidose (n=8), and 400 mg single dose (n=8) cohorts of the ongoing RestorAATion-2 study. Key highlights included:

• Following a single 200 mg dose of WVE-006, a total AAT level of 20.6 µM, including a M-AAT level of 10.3 µM, was observed in one individual during an acute phase response due to a kidney stone. These data demonstrate that treatment with WVE-006 enables endogenous regulation and dynamic increased sec…

• In the 200 mg multidose cohort, we observed 11.9 µM of total AAT and M-AAT of 7.2 µM, which was significantly increased from levels achieved during the single dose portion of the cohort. M-AAT levels reached 64.4% of total AAT, and mutant Z-AAT protein declined from baseline by 60.3%.

• In the 400 mg single dose cohort, we observed total AAT of 12.8 µM and M-AAT of 5.3 µM.