Wave Life Sciences Announces Approval of First Clinical Trial Application for RestorAATion-2 Trial of WVE-006 in Individuals with Alpha-1 Antitrypsin Deficiency (AATD)
30 Abril 2024 - 9:30AM
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 the
approval of its first clinical trial application (CTA) for its
RestorAATion-2 clinical trial of WVE-006, the company’s
first-in-class RNA editing oligonucleotide, which is being
developed for the treatment of alpha-1 antitrypsin deficiency
(AATD). WVE-006 is GalNAc-conjugated and subcutaneously
administered; it does not use a lipid nanoparticle (LNP) delivery
system.
“The approval of our first CTA for the RestorAATion-2 clinical
trial of WVE-006 marks an important milestone as we continue
extending our leadership in RNA editing. It is also important for
the alpha-1 community as WVE-006 has the potential to enable
correction of the disease-causing RNA mutation and provide a single
therapeutic option regardless of whether patients have AATD liver
disease, lung disease or both,” said Paul Bolno, MD, MBA, President
and Chief Executive Officer of Wave Life Sciences. “Our rapid
progress in dose escalating healthy volunteers enabled us to
demonstrate the translation of safety and pharmacokinetics of
WVE-006 in humans and quickly identify a starting dose level that,
based on preclinical data, is expected to engage the target in
patients. With proof-of-mechanism data from RestorAATion-2 expected
later this year, we look forward to the opportunity to provide
clinical demonstration of RNA editing and proof-of-concept for our
wholly owned pipeline of RNA editing candidates.”
RestorAATion-2 is a Phase 1b/2a open label study designed to
evaluate the safety, tolerability, pharmacodynamics (PD) and
pharmacokinetics (PK) of WVE-006 in individuals with AATD who have
the homozygous Pi*ZZ mutation. The trial includes both single
ascending dose (SAD) and multiple ascending dose (MAD) portions.
The company remains on track to deliver proof-of-mechanism data, as
measured by restoration of M-AAT protein in serum, in 2024.
GSK has the exclusive global license for WVE-006. Development
and commercialization responsibilities will transfer to GSK after
Wave completes the RestorAATion-2 study.
In addition to WVE-006, Wave continues to advance its wholly
owned RNA editing pipeline across a range of high-impact
GalNAc-hepatic and extra-hepatic 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.
About WVE-006WVE-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 Wave Life SciencesWave 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, PRISMTM, combines multiple modalities,
chemistry innovation and deep insights in human genetics to deliver
scientific breakthroughs that treat both rare and prevalent
disorders. Its toolkit of RNA-targeting modalities includes
editing, splicing, RNA interference and antisense silencing,
providing Wave with unmatched capabilities for designing and
sustainably delivering candidates that optimally address disease
biology. Wave’s diversified pipeline includes clinical programs in
Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and
Huntington’s disease, as well as a preclinical program in obesity.
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
(formerly Twitter) 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,
including, without limitation, the potential of WVE-006 to treat
AATD; our expectations and anticipated timing for delivering
proof-of-mechanism clinical data in AATD patients treated with
WVE-006; and 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. 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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