Full Press Release Details
Wave Life Sciences Highlights Growth Strategy for Building the Leading RNA Medicines Company in Annual
Presented preclinical proof-of-concept
data for new, wholly owned program targeting INHBE for metabolic disorders, including obesity, which uses Wave s best-in-class GalNAc-siRNA capabilities and novel
genetic insights from GSK
WVE-006 industry s first-ever RNA editing clinical
candidate on track for dosing in RestorAATion clinical trial in 4Q 2023 and first human proof-of-mechanism data in 2024
Advancing multiple, wholly owned RNA editing targets using correction and upregulation approaches; leveraging proprietary deep-learning model
to unlock additional targets across the edit-verse
Wave platform chemistry enabling extra-hepatic delivery in RNA
Wave expects to select five new clinical candidates by year-end 2025,
including INHBE in 4Q 2024
CAMBRIDGE, Mass., September 28, 2023 Wave Life Sciences Ltd. (Nasdaq: WVE), a
clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today held a virtual analyst and investor R&D Day, which highlighted the company s growth strategy for building
the leading RNA medicines company. The event also featured comments from Tony Wood, PhD, Chief Scientific Officer at GSK, and Carolyn Buser-Doepner, PhD, Vice President of the Novel Human Genetics Research Unit at GSK, regarding GSK s strategic
collaboration with Wave. An archived recording of the webcast and presentation is available here.
Today, we are witnessing a new era in
human genetics, where emerging insights on both rare and prevalent diseases are unlocking new target opportunities. Wave is uniquely positioned to capitalize on these insights with our multimodal platform and we are advancing an innovative pipeline
with potential to drive significant value for patients and families, as well as for investors, said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. We are bringing an exciting, genetics-based approach to
the metabolic disease and obesity space with our first siRNA program targeting INHBE. This program is designed to deliver healthy, sustainable weight loss to tens of millions of patients in the US and Europe alone, while also avoiding drawbacks of
current therapies. In addition to high-impact silencing targets like INHBE, we have a near-term pipeline focus on protein restoration and repair with our RNA editing and splicing capabilities. We anticipate advancing five new clinical candidates by year-end 2025, which will illustrate how Wave is reimagining what s possible in the treatment of human disease.
Following on the heels of WVE-006, the industry s first-ever RNA editing clinical candidate, we are making
great progress building a pipeline of wholly owned RNA editing programs using correction and mRNA upregulation. The targets shared today are all indicative of our ability to reach new areas of disease biology, both within the liver and beyond.
Moreover, we are leveraging our deep learning model to expand the universe of novel A-to-G targets where we can address diseases with reduced protein expression,
said Chandra Vargeese, PhD, Chief Technology Officer at Wave Life Sciences. Today we also announced significant progress in siRNA, where our data demonstrate
best-in-class silencing for both GalNAc-siRNA in liver and unconjugated siRNA in CNS. Finally, we are excited about new clinical data
suggesting WVE-N531 reached satellite cells in boys from the Part A WVE-N531 study, which underscores the power of
our novel chemistry and potential for a leading exon skipping franchise in Duchenne muscular dystrophy. Together these data indicate we are at the precipice of a transformative period for Wave.
Highlights from Wave s R&D Day:
wholly owned siRNA program driven by clinical genetics:
WVE-006 clinical program for alpha-1 antitrypsin deficiency (AATD):
Growing pipeline with high-value RNA editing targets:
WVE-N531 for Duchenne muscular dystrophy (DMD) and future
WVE-003 clinical program for Huntington s disease (HD):
About Wave Life Sciences
Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop
best-in-class medicines across multiple therapeutic modalities using PRISM, the company s proprietary discovery and drug development platform that enables the
precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.
To find out more, please visit www.wavelifesciences.com and follow Wave on X (formerly Twitter) @WaveLifeSci.
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 around advancing our pipeline of RNA editing programs and our understanding on the anticipated therapeutic benefits thereof, including the anticipated timing of delivering five new clinical
candidates; our understandings about metabolic disorders, including obesity, along with our understandings of INHBE and its correlation to a healthy metabolic profile; our expectations for our GalNAc-conjugated small interfering RNA (siRNA) program
targeting INHBE, and the anticipated therapeutic benefits thereof, including the potential to treat metabolic disorders, such as obesity, including the anticipated timing of announcing an INHBE candidate for metabolic disorders; our expectations for
our GalNAc-conjugated RNA editing oligonucleotides, and the anticipated therapeutic benefits thereof, including the potential of WVE-006 to treat AATD and the anticipated timing to deliver proof-of-mechanism data in AATD; our expectations for the Phase 2 study of WVE-N531
(FORWARD-53), including the anticipated timing of such data, and the potential multiexon strategy that may arise as a result thereof, including the potential of our DMD franchise; our expectations on timing to
deliver the multidose data from our SELECT-HD trial to enable decision-making; the future performance and results of our clinical programs; 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 our expectations regarding the continued progress of our GSK collaboration. 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 Securities and Exchange Commission (SEC), as amended, and in other
filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.