Wave Life Sciences Unveils Breakthrough in RNA Editing and Reports First Clinical Evidence of Mechanism in Humans – WAVE Life Sciences (NASDAQ:WVE)

Wave Life Sciences Ltd. WVE The stock is trading higher on Wednesday after the company reported 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 disease can cause lung and liver damage.

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

Also Read: Wave Life Sciences Stock Rises on Promising Preliminary Data from a Mid-Term Study of a Muscle Disease Drug.

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

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

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

Furthermore, restoration of 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 on day 15, accounting for more than 60% of total AAT.

An increase from baseline in neutrophil elastase inhibition was consistent with production of functional M-AAT.

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

The RestorAATion-2 study is ongoing and Wave expects to publish multidose data in 2025.

GSK plc GSK holds the exclusive global license for WVE-006.

Development and commercialization responsibility will transfer to GSK after Wave completes the RestorAATion-2 trial.

Wave is eligible to receive up to $525 million in milestones and sales-based royalties.

Price promotion: WVE shares were up 74.4% at $14.93 at last check on Wednesday.

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Image created with artificial intelligence via Midjourney.