Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA
medicines company committed to delivering life-changing treatments
for people battling devastating diseases, today announced the
initiation of dosing in healthy volunteers in the RestorAATion
clinical trial program, which is investigating WVE-006 as a
potential treatment for alpha-1 antitrypsin deficiency (AATD).
WVE-006 is a first-in-class, GalNAc-conjugated RNA
editing oligonucleotide (AIMer). It is designed to restore
circulation of healthy, wild-type alpha-1 antitrypsin (M-AAT)
protein and reduce dysfunctional Z-AAT protein, thereby potentially
addressing AATD-related lung disease, liver disease, or both.
“Initiating dosing in RestorAATion represents an
important milestone for the alpha-1 community, where treatment
options are limited and there are no medicines that address the
underlying genetic mutation that most commonly causes AATD. In
preclinical studies, WVE-006 led to potent and durable RNA editing
and restoration of AAT protein up to 30 micromolar, underscoring
the impact of our novel chemistry. WVE-006 has the potential to
transform the treatment paradigm for this disease, and we are
well-positioned to achieve this vision as part of our collaboration
with GSK,” said Paul Bolno, MD, MBA, President and Chief Executive
Officer of Wave Life Sciences. “Moreover, with the first RNA
editing therapeutic to ever be dosed in humans, we are making a
significant contribution to the scientific field by bringing an
entirely new class of medicines into clinical development, and we
expect to continue unlocking the potential of RNA editing more
broadly.”
The RestorAATion clinical program includes healthy
volunteers (RestorAATion-1) as well as individuals with AATD who
have the homozygous PiZZ mutation (RestorAATion-2) 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 deliver
proof-of-mechanism data in individuals with AATD in 2024.
“This represents an important milestone for GSK’s
growing oligonucleotide pipeline,” said Tony Wood, Chief Scientific
Officer, GSK. “Throughout this year, we’ve demonstrated our
commitment to realizing the potential of oligonucleotide
therapeutics in areas of unmet need for patients. Beyond WVE-006,
we’re also excited to continue our collaboration with Wave Life
Sciences using their best-in-class PRISM™ platform.”
With initiation of dosing in RestorAATion, Wave has
achieved its first WVE-006 milestone in its collaboration with GSK,
resulting in a $20 million payment to Wave. For WVE-006, Wave is
eligible to receive up to $505 million in additional development,
launch, and sales-related milestone payments, as well as tiered
royalties on net sales, from GSK. Development and commercialization
responsibilities will transfer to GSK after Wave completes the
RestorAATion-2 study.
Beyond WVE-006, Wave is advancing a pipeline of
wholly owned RNA editing therapeutics designed to either correct or
upregulate mRNA across a range of high impact 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. At its R&D
Day in September 2023, Wave shared in vivo and in vitro
proof-of-concept data on several undisclosed targets, achieving at
least 2-fold mRNA upregulation in liver and kidney targets and more
than 60% mRNA correction in liver and lung targets.
About Alpha-1 Antitrypsin
DeficiencyAlpha-1 antitrypsin deficiency (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 insufficient levels of circulating M-AAT
protein, which protects the lungs from proteolytic enzymes, and it
leads to liver disease due to aggregation of misfolded Z-AAT
protein in hepatocytes. There are approximately 200,000 patients in
the United States and Europe who have Z mutations on both alleles,
known as the PiZZ genotype. Augmentation therapy via delivery of
AAT protein is currently the only treatment option for AATD lung
disease and requires weekly intravenous infusions. There are no
treatments for AATD liver disease, other than liver
transplantation.
About WVE-006 WVE-006 is a
first-in-class, GalNAc-conjugated and subcutaneously administered
RNA editing oligonucleotide designed to correct the single base
mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele,
thereby enabling restoration and circulation of functional M-AAT
protein. In preclinical studies, WVE-006 demonstrated potent and
durable editing of SERPINA1 Z transcript in mice, restoration of
AAT protein up to 30 micromolar, and improvement in several markers
of liver disease. WVE-006 is also highly specific with no evidence
of bystander editing. Together, these data demonstrate the
potential of WVE-006 to address AATD-related liver disease, lung
disease, or both.
About AIMersWave’s A-to-I RNA
editing oligonucleotides (AIMers) are designed to target single
bases on an RNA transcript and recruit proteins that exist in the
body, called ADAR (adenosine deaminases acting on RNA) enzymes,
which naturally possess the ability to change an adenine (A) to an
inosine (I), which cells read as guanine (G). This approach enables
both the correction of G-to-A point mutations, as well as the
modulation of RNA to upregulate protein expression, modify
protein-protein interactions, or alter RNA folding and processing.
AIMers enable simplified delivery and avoid the risk of permanent
changes to the genome and irreversible off-target effects with
DNA-targeting approaches. AIMers are short in length, fully
chemically modified, and use novel chemistry, including proprietary
PN backbone modifications and chiral control, which make them
distinct from other ADAR-mediated editing approaches.
About Wave Life SciencesWave
Life 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.
Forward-Looking Statements 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 future
performance and results of our clinical programs; our expectations
and anticipated timing for delivering proof-of-mechanism clinical
data in AATD patients treated with WVE-006; 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,
and RNA editing broadly, to address diseases of many different
tissues and cell types; the potential benefits of our AIMers
compared with other RNA base editing approaches; the potential
benefits that our “edit-verse” map may offer to identify new RNA
editing targets; and the potential achievement of development,
launch and sales-related milestones for WVE-006 under our GSK
collaboration 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 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 circumstance.
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
AATD Community Contact:Chelley
Casey+1 617-949-2900ccasey@wavelifesci.com
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