Wave Life Sciences Ltd. (NASDAQ:WVE) stock is trading lower on Wednesday after the company shared data from the ongoing Phase 1b/2a RestorAATion-2 Trial of WVE-006 in alpha-1 antitrypsin deficiency.
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Alpha-1 antitrypsin (AAT) deficiency is an inherited genetic disorder that reduces the production of the AAT protein, which is crucial for protecting the lungs and liver.
The data reported on Wednesday include RestorAATion-2 cohort 1 (200 mg, n=8), where participants received a single subcutaneous dose of WVE-006 and then received seven subcutaneous doses administered every other week under the multidose protocol, as well as the single dose portion of cohort 2 (400 mg, n=8).
Production of M-AAT protein indicates WVE-006 led to RNA editing in all treated participants, with durable production of serum AAT at levels associated with a lower risk of liver and lung disease following repeat 200 mg doses.
Data from both the 200 mg and 400 mg (single dose only) cohorts support a monthly or less frequent subcutaneous dosing regimen. Dosing is ongoing in the 400 mg multidose cohort with a monthly dosing regimen, for which Wave expects to deliver data in the first quarter of 2026.
WVE-006 continues to be well-tolerated with a favorable safety profile to date.
GSK Plc (NYSE:GSK) has the exclusive global license for WVE-006. Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion-2 study.
Wave is continuing to advance a wholly owned pipeline of RNA editing candidates. The company plans to share new preclinical data from these programs at Research Day in the fall 2025 and to initiate clinical development of additional RNA editing programs in 2026.
WVE Price Action: WVE stock is down 21.41% at $7.56 at publication on Wednesday.
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