Cambridge, Mass. — September 3, 2025 — Leads & Copy — Wave Life Sciences Ltd. (Nasdaq: WVE) has announced positive data from its Phase 1b/2a RestorAATion-2 study of WVE-006 for alpha-1 antitrypsin deficiency (AATD). The study demonstrated therapeutically restored physiological serum AAT production in a Pi*ZZ individual during a non-drug related acute phase response. Repeat 200 mg doses of WVE-006 achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases (11.9 µM total AAT, 7.2 µM M-AAT).
A single 400 mg dose achieved 12.8 µM total AAT and 5.3 µM M-AAT. Data from the 200 mg and 400 mg cohorts support monthly or less frequent subcutaneous dosing. The 400 mg monthly multidose cohort data is expected in 1Q 2026. WVE-006 continues to be well tolerated with a favorable safety profile to date.
Wave will host an investor conference call and webcast today at 8:30 a.m. ET.
Approximately 200,000 people in the US and Europe living with AATD are homozygous for the Z mutation (Pi*ZZ) in the SERPINA1 gene, which can lead to severe lung disease, liver disease, or both. WVE-006 is designed to address both lung and liver manifestations of AATD by correcting the Z mutation in SERPINA1 mRNA, thereby converting Z-AAT to wild-type M-AAT without altering the endogenous transcriptional regulation of the SERPINA1 gene.
According to Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences, the data represents a significant clinical milestone, with WVE-006 enabling the dynamic generation of wild-type M-AAT protein when needed.
According to Stephen Rennard, MD, Professor of Medicine in the Division of Pulmonary, Critical Care and Sleep Medicine at the University of Nebraska Medical Center, Wave’s clinical trial provides two important findings: RNA editing resulted in the endogenous production of M protein in ZZ individuals; the stress of a kidney stone was associated with marked upregulation of M protein production, suggesting that RNA editing has the potential to restore endogenous M protein production and its normal regulation in AATD.
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 eligible for up to $525 million in milestones, as well as tiered royalties on net sales, for WVE-006.
Kate Rausch, VP, Corporate Affairs and Investor Relations, +1 617-949-4827
Source: Wave Life Sciences
