Wave Life Sciences Ltd. (NASDAQ:WVE) saw its stock decline 17% after providing an update from its ongoing RestorAATion-2 trial of WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD).
The clinical-stage biotech company reported that its RNA editing oligonucleotide achieved sustained production of serum AAT protein at levels linked to reduced risk of AATD-related liver and lung disease after repeated 200 mg doses. Wave highlighted this as the first 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 rising to 7.2 µM, substantially higher than the 4.8 µM achieved during the single-dose phase. A single 400 mg dose produced 12.8 µM total AAT and 5.3 µM M-AAT, with the company noting that the ongoing 400 mg multidose cohort could yield further increases.
Wave indicated that data from both the 200 mg and 400 mg cohorts support monthly or less frequent subcutaneous dosing. Data from the 400 mg monthly multidose cohort are expected in Q1 2026.
WVE-006 has remained well tolerated with a favorable safety profile so far, with all adverse events mild to moderate and no serious adverse events or discontinuations reported.
GSK holds the exclusive global license for WVE-006, with development and commercialization responsibilities transferring to GSK once Wave completes the RestorAATion-2 study. Wave is eligible for up to $525 million in milestone payments, plus tiered royalties on net sales.
Wave Life Sciences stock price
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