Wave Life Sciences Announces Approval of First Clinical Trial Application for RestorAATion-2 Trial of WVE-006 in Individuals with Alpha-1 Antitrypsin Deficiency (AATD)

Proof-of-mechanism data for WVE-006 in individuals with AATD remain on track for 2024

CAMBRIDGE, Mass., April 30, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of WVE-006, the company’s first-in-class RNA editing oligonucleotide, which is being developed for the treatment of alpha-1 antitrypsin deficiency (AATD). WVE-006 is GalNAc-conjugated and subcutaneously administered; it does not use a lipid nanoparticle (LNP) delivery system.

“The approval of our first CTA for the RestorAATion-2 clinical trial of WVE-006 marks an important milestone as we continue extending our leadership in RNA editing. It is also important for the alpha-1 community as WVE-006 has the potential to enable correction of the disease-causing RNA mutation and provide a single therapeutic option regardless of whether patients have AATD liver disease, lung disease or both,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Our rapid progress in dose escalating healthy volunteers enabled us to demonstrate the translation of safety and pharmacokinetics of WVE-006 in humans and quickly identify a starting dose level that, based on preclinical data, is expected to engage the target in patients. With proof-of-mechanism data from RestorAATion-2 expected later this year, we look forward to the opportunity to provide clinical demonstration of RNA editing and proof-of-concept for our wholly owned pipeline of RNA editing candidates.”

RestorAATion-2 is a Phase 1b/2a open label study designed to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of WVE-006 in individuals with AATD who have the homozygous Pi*ZZ mutation. The trial includes both single ascending dose (SAD) and multiple ascending dose (MAD) portions. The company remains on track to deliver proof-of-mechanism data, as measured by restoration of M-AAT protein in serum, in 2024.

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 addition to WVE-006, Wave continues to advance its wholly owned RNA editing pipeline across a range of high-impact GalNAc-hepatic and extra-hepatic targets. The company’s discovery and development efforts in RNA editing are powered by its proprietary “edit-verse,” which leverages genetic datasets and deep learning models to identify new RNA editing targets and edit sites. These targets leverage easily accessible biomarkers, offer efficient paths to proof-of-concept in humans, and represent meaningful commercial opportunities.