Wave Life Sciences (WVE) said Wednesday that its experimental RNA-editing therapy WVE-006 achieved "durable" production of serum AAT protein at levels linked to a lower risk of alpha-1 antitrypsin deficiency liver and lung diseases after repeat 200 mg doses in an ongoing phase 1b/2a trial.
The company said data from the 200 mg and 400 mg cohorts support monthly or less frequent dosing, with the 400 mg multidose cohort showing potential to achieve higher serum AAT levels. Data from the 400 mg multidose group are expected in Q1 2026.
Wave Life cited one outlier case showing "marked upregulation" of M-AAT protein, which is protective of lung function, production in a patient experiencing a kidney stone stress response.
The company also said WVE-006 remained well-tolerated with no serious adverse events or discontinuations.
Development rights for WVE-006 will transfer to GSK (GSK) after the current study is completed, Wave Life said.
Shares of the company were down more than 22% in early trading Wednesday.