Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-006, the First-ever RNA Editing Clinical Candidate, and Plans for Upcoming Virtual “R&D Day”
WVE-006 is the first-ever RNA editing program to enter clinical development and is designed to restore production and circulation of functional, wild-type alpha-1 antitrypsin (AAT) protein and reduce levels of mutant Z-AAT protein, thereby addressing alpha-1 antitrypsin deficiency-related lung disease, liver disease or both
Wave expects to initiate dosing in the first-in-human clinical program in 4Q 2023 and deliver AAT protein restoration data from AATD patients treated with WVE-006 in 2024
Under its collaboration with GSK, Wave is eligible to receive substantial milestone payments for WVE-006 in 2023 and beyond
Wave plans to host a virtual “R&D Day” on
“With the submission of the first CTA for WVE-006, we have officially initiated clinical development of the industry’s first-ever RNA editing therapeutic candidate,” said
The current clinical development plan for WVE-006 includes healthy volunteers as well as individuals with AATD who have the homozygous PiZZ mutation, and is designed to provide an efficient path to proof-of-mechanism as measured by restoration of M-AAT protein in serum. Wave expects to initiate dosing in healthy volunteers in the fourth quarter of 2023 and deliver proof-of-mechanism data in individuals with AATD in 2024.
“WVE-006 is on a path to potentially deliver the first-ever proof-of-mechanism clinical data for an RNA editing therapeutic. Positive data would be a pivotal milestone for people living with AATD and would unlock the potential of RNA editing more broadly. Additionally, WVE-006 is a foundational component of our collaboration with GSK and carries with it meaningful milestone and royalty payments, including near-term clinical milestones,” said
Earlier this year, Wave commenced its strategic collaboration with GSK to advance transformative RNA medicines using Wave’s multimodal RNA platform, including WVE-006. Wave received
Wave expects that its cash and cash equivalents will be sufficient to fund operations into 2025. Wave does not include future milestones or other contingent payments in its cash runway.
Wave Virtual R&D Day
A link to register for the event is available here. A live webcast of the event can be accessed by visiting “Investor Events” on the investor relations section of the
About WVE-006 and AATD
WVE-006 is a clinical-stage GalNAc-conjugated investigational RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that is commonly caused by a G-to-A point mutation (“Z allele”) in the SERPINA1 gene. This mutation leads to lung disease due to a lack of wild-type alpha-1 antitrypsin (M-AAT) function in lungs, and it leads to liver disease due to aggregation of misfolded Z-AAT protein in hepatocytes. There are approximately 200,000 patients in
A-to-I(G) RNA editing oligonucleotides (“AIMers”) are designed to edit specific sites on an RNA transcript, thereby avoiding permanent changes to the genome that occur with DNA-targeting approaches. Rather than using an exogenous editing enzyme, AIMers recruit proteins that exist in the body, called ADAR enzymes, which naturally edit certain adenine (A) bases to inosine (I). Because I is read as G (guanine) by the cellular translational machinery, sequence-directed editing with ADAR has the potential to revert transcripts with single G-to-A point mutations that cause genetic diseases. This approach redirects a natural system for therapeutic purposes, enables simplified delivery without viral particles or liposomes, and avoids the risk of irreversible off-target effects of DNA-targeting approaches. AIMers are short in length, fully chemically modified, and use novel chemistry, including proprietary PN backbone modifications and chiral control, that make them distinct from other ADAR-mediated editing approaches.
About Wave Life Sciences
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our expectations for our GalNAc-conjugated RNA editing oligonucleotides (AIMers), including WVE-006, and the anticipated therapeutic benefits thereof, including the potential of WVE-006 to treat AATD; the anticipated timing to initiate dosing in the first-in-human clinical program with WVE-006, and the associated delivery of AAT protein restoration data from AATD patients treated with WVE-006; the future performance and results of our clinical programs; our expectations for delivering proof-of-mechanism clinical data for an RNA editing therapeutic; our understanding that WVE-006 is the most advanced candidate for AATD designed to restore functional wild-type AAT protein and reduce Z-AAT protein aggregation; our expectations regarding the ability of our AIMers to address diseases of many different tissues and cell types; the potential benefits of our AIMers compared with other RNA base editing approaches; and the potential achievement of milestones under our collaborations and receipt of cash payments therefor. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release and actual results may differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the
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Source: Wave Life Sciences USA, Inc.