Wave Life Sciences Reveals RNA Editing Breakthrough, Reports First-Ever Clinical Proof-Of-Mechanism In Humans

Wave Life Sciences Ltd. WVE stock is trading higher on Wednesday after the company revealed proof-of-mechanism data from the ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 in alpha-1 antitrypsin deficiency (AATD), an inherited genetic disorder that causes low levels of a protein (AAT) that protects the lungs.

The condition can cause lung and liver damage.

WVE-006 is designed to address AATD-related lung disease, liver disease, or both.

Also Read: Wave Life Sciences Stock Surges On Promising Interim Data From Mid-Stage Study Of Muscular Disorder Drug.

The company adds that today’s proof-of-mechanism data are the first-ever clinical demonstration of RNA editing in humans.

These data are from the first single-dose cohort (200 mg) in RestorAATion-2 and include the first two patients with “ZZ” AATD (Pi*ZZ AATD) to reach day 57.

Individuals with Pi*ZZ AATD do not naturally produce wild-type alpha-1 antitrypsin (M-AAT) protein; therefore, the presence of M-AAT protein is confirmation of successful editing of mutant Z-AAT mRNA.

Additionally, restoring 50% M-AAT would be consistent with the heterozygous “MZ” genotype with low risk of AATD lung and liver disease.

Circulating wild-type M-AAT protein in plasma reached a mean of 6.9 micromolar at day 15, representing more than 60% of total AAT.

Increases in neutrophil elastase inhibition from baseline were consistent with the production of functional M-AAT.

WVE-006 has been well-tolerated and has a favorable safety profile to date. All adverse events in RestorAATion-2 and the ongoing RestorAATion-1 trial of healthy volunteers are mild to moderate, with no serious adverse events reported.

The RestorAATion-2 trial is ongoing, and Wave expects to share multidose data in 2025.

GSK plc 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 and sales-based royalties.

Price Action: WVE stock is up 74.4% at $14.93 at last check Wednesday.

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Image created using artificial intelligence via Midjourney.