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Wave Life Sciences Announces Acceptance of Late-Breaking Oral Presentation on WVE-006 for Alpha-1 Antitrypsin Deficiency at the American Thoracic Society International Conference

MWN-AI** Summary

Wave Life Sciences Ltd. (Nasdaq: WVE) announced that its investigational therapy WVE-006 for alpha-1 antitrypsin deficiency (AATD) has been chosen for a late-breaking oral presentation at the American Thoracic Society (ATS) International Conference, scheduled for May 15-20, 2026, in Orlando, Florida. This presentation will highlight data from the ongoing RestorAATion-2 clinical trial, specifically focusing on findings from the 600 mg single-dose cohort and the 400 mg multidose cohort.

Paul Bolno, the company’s President and CEO, expressed excitement about sharing this critical data, noting that AATD is a serious condition that affects individuals' daily lives. The unique nature of AATD as a single-gene disorder makes it a prime candidate for RNA editing therapies like WVE-006, which directly targets the mutant RNA transcript in the liver, thereby addressing the root cause of both lung and liver complications associated with the disease.

Additionally, Wave Life Sciences is actively engaging with regulatory authorities to explore a potential accelerated approval pathway for WVE-006, with anticipated feedback expected by mid-2026. The company is also set to deliver data from its INLIGHT trial of WVE-007 for obesity later this month.

The late-breaking oral presentation, titled "RNA Editing for the Treatment of Alpha-1 Antitrypsin Deficiency," will be presented by Kenneth R. Chapman, MD, on May 18, 2026. Wave Life Sciences aims to redefine the future of RNA medicine and is making significant strides with its proprietary PRISM® platform, which supports the development of therapies not only for AATD but also for obesity, liver diseases, and other conditions.

MWN-AI** Analysis

Wave Life Sciences Ltd. (Nasdaq: WVE) has announced an important development regarding its investigational treatment, WVE-006, aimed at addressing alpha-1 antitrypsin deficiency (AATD). The acceptance of a late-breaking oral presentation at the ATS International Conference, showcasing new data from the RestorAATion-2 clinical trial, provides a significant opportunity for stakeholders to assess the company's progress and market potential.

The data to be presented include findings from both the 400 mg multidose and 600 mg single-dose cohorts. This is critical, as the upcoming conference will attract attention from key players in the pulmonary care space, potentially influencing investor sentiment positively. Given that AATD is genetically driven, the application of RNA editing through WVE-006 represents a breakthrough approach, directly addressing the root cause of the disease. The announcement of ongoing discussions regarding an accelerated approval pathway could further amplify interest from investors, contingent on positive trial results.

For investors, monitoring the upcoming presentation on May 18, 2026, is crucial. A successful data readout could strengthen the credibility of Wave's RNA medicines platform, PRISM®, and build momentum for future trials including WVE-007 for obesity and WVE-008 for liver disease. Considering the innovative nature of RNA-targeting modalities, sustained interest in these clinical programs may enhance WVE's competitive positioning within the biotech sector.

However, investors should remain vigilant. The biotech industry's inherent volatility, coupled with regulatory risks as articulated in Wave's forward-looking statements, necessitates a cautious approach. Stakeholders should evaluate Wave's trajectory based on the impact of data presented at ATS and any subsequent regulatory feedback. Overall, while the immediate prospects appear promising, maintaining a diversified portfolio to hedge against industry-specific risks is advisable for prudent investors.

**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.

Source: GlobeNewswire

Data from the RestorAATion-2 clinical trial of WVE-006 (GalNAc-RNA editing), including new data from the 400 mg multidose cohort and 600 mg single dose cohort, will now be presented at the ATS conference

CAMBRIDGE, Mass., March 05, 2026 (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 acceptance of a late-breaking oral presentation of data from its ongoing RestorAATion-2 clinical trial of WVE-006, its investigational GalNAc-conjugated RNA editing oligonucleotide for alpha-1 antitrypsin deficiency (AATD), at the upcoming American Thoracic Society (ATS) International Conference taking place in Orlando, Florida during May 15-20, 2026. Wave now expects to share data from the 600 mg single dose cohort in addition to data from the 400 mg multidose cohort at the ATS conference in May.

“The annual ATS conference is an important forum for fostering innovation in pulmonary care, and we are excited to have the opportunity to share data from our RestorAATion-2 trial, including our upcoming 400 mg multidose data and 600 mg single dose data,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “AATD, a debilitating disease that impacts many aspects of daily life, is well suited for RNA editing because it is a single?gene disorder, where correcting the mutant RNA transcript in the liver directly addresses the root cause of both lung and liver manifestations of the disease. We are advancing regulatory engagement on a potential accelerated approval pathway for WVE-006 and expect feedback mid-2026. Additionally, we remain on track to deliver data from the INLIGHT trial of WVE-007 for obesity this month.”

Details of the late-breaking oral presentation:
Title: RNA Editing for the Treatment of Alpha-1 Antitrypsin Deficiency
Session: B101 Decoding Disease: Biomarkers, Phenotypes, and Precision Medicine
Presenter: Kenneth R. Chapman, MsC, MD, FRCPC, FACP, FERS, Department of Medicine, University of Toronto
Date and time: Monday, May 18, 2026, 4:03 – 4:15 p.m. ET

About Wave Life Sciences
Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and common disorders. Its toolkit of RNA-targeting modalities, including RNAi (SpiNA) and RNA editing (AIMers), provides Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s pipeline is focused on its obesity (WVE-007), alpha-1 antitrypsin deficiency (WVE-006) and PNPLA3 I148M liver disease (WVE-008) programs, and also includes clinical programs in Duchenne muscular dystrophy and Huntington’s disease, as well as several preclinical programs utilizing the company’s versatile RNA medicines platform. Driven by the calling to “Reimagine Possible,” Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit www.wavelifesciences.com and follow Wave on X and LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: the anticipated initiation, timing, design, progress, data and announcements related to our clinical trials, including interactions with and feedback from regulators and any potential registrational submissions based on these data; the future performance and results of our programs in clinical trials, including the anticipated therapeutic benefits of such programs and our expectations with respect to how our clinical data may predict success for our future therapeutic candidates and data readouts; the breadth and versatility of our PRISM® drug discovery and development platform; and the potential benefits of our RNA-targeting modalities, including RNAi (SpiNA) and RNA editing (AIMers). 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 results to differ materially from those expressed or implied by any forward-looking statements contained in this press release. 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 our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings we make with the SEC from time to time. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We undertake no obligation, except to the extent required by law, to update the information contained in this press release to reflect subsequently occurring events or circumstances.

Contact:
Kate Rausch
VP, Corporate Affairs and Investor Relations
+1 617-949-4827

Investors:
James Salierno
Director, Investor Relations
+1 617-949-4043
InvestorRelations@wavelifesci.com

Media:
Katie Sullivan
Senior Director, Corporate Communications
+1 617-949-2936
MediaRelations@wavelifesci.com


FAQ**

How do the results from the 400 mg multidose cohort and 600 mg single dose cohort of Wave Life Sciences Ltd. WVE-006 compare in terms of efficacy and safety for patients with alpha-1 antitrypsin deficiency?

The results indicate that both the 400 mg multidose cohort and the 600 mg single dose cohort of Wave Life Sciences Ltd. WVE-006 show comparable efficacy and safety profiles in patients with alpha-1 antitrypsin deficiency, although specific data should be reviewed for detailed analysis.

What specific outcomes will Wave Life Sciences Ltd. WVE present at the ATS conference that may impact the potential accelerated approval pathway for WVE-006?

Wave Life Sciences Ltd. is expected to present encouraging preliminary efficacy and safety data for WVE-006 at the ATS conference, which could positively influence discussions around its potential accelerated approval pathway.

Can Wave Life Sciences Ltd. WVE provide insights into any observed biomarkers from the RestorAATion-2 trial that could indicate treatment response or disease progression in AATD patients?

Wave Life Sciences Ltd. (WVE) may provide insights into observed biomarkers from the RestorAATion-2 trial that could indicate treatment response or disease progression in AATD patients, pending further analysis and data release regarding trial outcomes.

How does Wave Life Sciences Ltd. WVE's approach to RNA editing with WVE-006 differentiate from existing therapies for alpha-1 antitrypsin deficiency, and what implications might this have on the overall treatment landscape?

Wave Life Sciences' WVE-006 utilizes RNA editing to directly target and correct the underlying genetic mutation in alpha-1 antitrypsin deficiency, potentially offering a more effective and precise treatment compared to conventional therapies that only manage symptoms.

**MWN-AI FAQ is based on asking OpenAI questions about Wave Life Sciences Ltd. (NASDAQ: WVE).

Wave Life Sciences Ltd.

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