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Investing.com -- Wave Life Sciences Ltd. (NASDAQ:WVE) stock fell 17% after the company released an update from its ongoing RestorAATion-2 trial of WVE-006 in Alpha-1 Antitrypsin Deficiency (AATD).
The clinical-stage biotechnology company reported that its RNA editing oligonucleotide achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses. The company highlighted this as the first-ever demonstration of therapeutically restored physiological serum AAT production in a PiZZ individual during a non-drug related acute phase response.
In the 200 mg multidose cohort, total AAT reached 11.9 micromolar (µM) with wild-type M-AAT increasing to 7.2 µM, significantly higher than levels achieved during the single dose portion (4.8 µM). A single 400 mg dose achieved 12.8 µM total AAT and 5.3 µM M-AAT, with the company noting that an ongoing 400 mg multidose cohort has potential to deliver further increases.
Wave Life Sciences indicated that data from both the 200 mg and 400 mg cohorts support monthly or less frequent subcutaneous dosing. The company expects to report data from the 400 mg monthly multidose cohort in the first quarter of 2026.
WVE-006 continues to be well tolerated with a favorable safety profile to date, with all adverse events reported as mild to moderate in intensity and no serious adverse events or discontinuations.
GSK holds the exclusive global license for WVE-006, with development and commercialization responsibilities transferring to GSK after Wave completes the RestorAATion-2 study. Wave is eligible for up to $525 million in milestones, plus tiered royalties on net sales.
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