Clinical TrialMay 18, 2026, 04:32 PM
Wave Life Sciences WVE-006 Trial Shows 71% Z-AAT Reduction, 64% M-AAT
AI Summary
Wave Life Sciences announced updated positive data from its RestorAATion-2 trial of WVE-006 for alpha-1 antitrypsin deficiency (AATD). The data showed WVE-006's potential to address both lung and liver manifestations, with significant reductions in harmful Z-AAT and restoration of healthy M-AAT, consistent with monthly dosing. The therapy also demonstrated dynamic AAT production during acute phase responses and a favorable safety profile. The company expects FDA feedback on a potential accelerated approval pathway by mid-2026.
Key Highlights
- WVE-006 reduced harmful Z-AAT by 71% in the 200 mg biweekly cohort.
- Wild-type M-AAT comprised 64% of total AAT in the 200 mg biweekly cohort.
- Total AAT reached 11.9 µM (200 mg biweekly) and 13.6 µM (400 mg monthly).
- Z-AAT reduced by 67.7% and M-AAT reached 58.7% of total AAT with 400 mg monthly dosing.
- Dynamic AAT production restored, with increases of 57.8% and 59.8% during acute phase responses.
- RNA editing effects sustained at least three months following the last dose.
- WVE-006 was generally safe and well tolerated with no liver toxicities.
- FDA feedback on a potential accelerated approval pathway expected mid-2026.
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