Wave Life Sciences reported new data from its ongoing Phase 1b/2a RestorAATion-2 trial of WVE-006, an investigational RNA editing therapy for alpha-1 antitrypsin deficiency (AATD), partnered with GSK135.
The latest results showed durable production of alpha-1 antitrypsin (AAT) protein at levels associated with lower risk of AATD liver and lung diseases, based on single and multidose administration cohorts35.
A notable finding was that one patient showed a spike to over 20 micromolars of AAT protein during an acute event, suggesting that WVE-006 can produce protective protein when needed most135.
Despite the biologically promising results, Wave's shares fell by up to 22% after the data release, as overall protein levels in the multidose group only 'narrowly hit' positive analyst benchmarks, while the higher dose missed some expectations1.
Analysts noted all study groups achieved the regulatory threshold for efficacy, but investor expectations may have been higher, fueling the stock dip1.
The therapy maintains a favorable safety profile, with all adverse events mild to moderate and no serious adverse events reported5.
Monthly data from the higher dose cohort are expected in early 2026, and Wave emphasized the clinically meaningful nature of the results despite market reaction35.
GSK secured global rights to WVE-006 through a broad partnership established in 20221.
Sources:
1. https://www.biopharmadive.com/news/wave-rna-editing-aatd-study-results-multidose/759122/
3. https://www.stocktitan.net/news/WVE/wave-life-sciences-announces-positive-update-from-ongoing-restor-aa-aji2b2yvr2vz.html
5. https://www.globenewswire.com/news-release/2025/09/03/3143474/0/en/Wave-Life-Sciences-Announces-Positive-Update-from-Ongoing-RestorAATion-2-Trial-of-WVE-006-in-Alpha-1-Antitrypsin-Deficiency.html