Wave Life Sciences Ltd. announced positive data from the 200 mg single and multidose and 400 mg single dose cohorts of the ongoing Phase 1b/2a RestorAATion-2 study evaluating WVE-006 as a treatment for alpha-1antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously delivered A-to-I RNA editing oligonucleotide (AIMer) developed with Wave's best-in-class oligonucleotide chemistry platform. 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.

Furthermore, these data support monthly or less frequent dosing. Collectively, its data highlight WVE-006's potential to provide a transformative treatment option for people living with AATD. WVE-006 is aGalNAc-conjugated., subcutaneously delivered A- to-I RNA editing oligon nucleotide (AIMer).

WVE-006 continues to be well tolerated with a favorable safety profile to date. All adverse events (AEs) were mild to moderate in intensity, and there were no SAEs or discontinuations. "Wave's clinical trial provides two important findings.

First, RNA editing resulted in the endogenous production of M protein in ZZ individuals. Second, serendipitously the study observed that the stress of a kidney stone was associated with marked upregulation of M protein production. These results are extremely exciting," said Stephen Rennard, MD, Professor of Medicine in the Division of Pulmonary, Critical Care and Sleep Medicine at the University of Nebraska Medical Center.

GSK has the exclusive global license for WVE-006. Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion- 2 study. In total, Wave is eligible for up to $525 million in milestones, as well as tiered royalties on net sales, for WVE-006.

Behind WVE-006, Wave is continuing to advance a wholly owned pipeline of RNA editing candidates which utilize its proprietary chemistry in a variety of hepatic and extrahepatic tissues. Analysts planning to participate during the Q&A portion of the live call can join the conference call at the audio-conferencing link here. Once registered, participants will receive the dial-in information.

Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website.