Wave Life Sciences Ltd. (WVE) saw its stock skyrocket on Wednesday, fueled by the company’s announcement of promising proof-of-mechanism data from its ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 for alpha-1 antitrypsin deficiency (AATD). This inherited genetic disorder causes low levels of a protein (AAT) that protects the lungs, potentially leading to lung and liver damage.
WVE-006 is designed to directly address the root cause of AATD by targeting the genetic defect responsible for the deficiency. The data released by Wave represent a landmark achievement, showcasing the first-ever clinical demonstration of RNA editing in humans. This groundbreaking technology holds the potential to revolutionize the treatment of genetic disorders.
The data comes from the first single-dose cohort (200 mg) of the RestorAATion-2 study, focusing on individuals with the most severe form of AATD, known as Pi*ZZ AATD. These individuals lack the ability to naturally produce the wild-type alpha-1 antitrypsin (M-AAT) protein. The presence of M-AAT protein in the treated patients signifies a successful editing of the mutant Z-AAT mRNA. Moreover, the restored M-AAT levels reached a mean of 6.9 micromolar at day 15, representing over 60% of total AAT. This increase in functional M-AAT is a crucial step towards mitigating the risks of lung and liver disease associated with AATD.
The safety profile of WVE-006 remains positive, with all adverse events reported in the RestorAATion-2 and RestorAATion-1 trials categorized as mild to moderate. No serious adverse events have been reported.
This positive news comes as Wave continues its RestorAATion-2 trial, with multidose data expected to be released in 2025. GSK plc (GSK) has secured an exclusive global license for WVE-006 and will assume development and commercialization responsibilities upon completion of the RestorAATion-2 study. Wave stands to benefit from up to $525 million in milestones and sales-based royalties based on the success of WVE-006.
With the strong clinical data, promising safety profile, and potential for a significant market impact, Wave Life Sciences is undoubtedly drawing attention in the pharmaceutical industry. The future of AATD treatment is looking brighter than ever, with WVE-006 poised to become a leading therapeutic option for those suffering from this debilitating genetic disorder.
