Wave Life Sciences Highlights Growth Strategy for Building the Leading RNA Medicines Company in Annual R&D Day
September 28 2023 - 4:15PM
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:
First wholly owned siRNA program driven by clinical
genetics:
- Today, Wave announced its first GalNAc-conjugated small
interfering RNA (siRNA) program targeting INHBE to treat metabolic
disorders, including obesity, which impacts an estimated 47 million
individuals in the US and Europe.
- INHBE leverages novel genetic insights from GSK as part of the
Wave/GSK collaboration.
- INHBE loss-of-function heterozygous carriers exhibit a healthy
metabolic profile, including reduced waist-to-hip circumference and
reduced odds ratio of Type 2 Diabetes.
- Reduction in INHBE of 50% or greater is expected to restore a
healthy metabolic profile.
- In a preclinical study, Wave demonstrated that 62% INHBE
knockdown in diet-induced obese (DIO) mice led to 16% lower body
weight as compared to control at five weeks. In a subsequent
eight-week study, Wave demonstrated further reduction of visceral
fat resulting from INHBE knockdown, which recapitulated phenotypes
of heterozygous loss-of-function carriers with healthy metabolic
profiles.
- Wave expects to select an INHBE clinical candidate in the
fourth quarter of 2024.
- Wave also shared data demonstrating best-in-class potential of
its next-generation siRNA constructs, driven by Wave’s proprietary
chemistry, including tunable PN variants that enable delivery to a
variety of extra-hepatic tissues.
WVE-006 clinical program for alpha-1 antitrypsin
deficiency (AATD):
- Wave has initiated clinical development of WVE-006 for AATD and
recently announced submission of its first clinical trial
application (CTA).
- The current clinical development plan for WVE-006, called
RestorAATion, 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 initiate dosing with WVE-006 in healthy
volunteers in the fourth quarter of 2023 and deliver
proof-of-mechanism data in AATD in 2024.
Growing pipeline with high-value RNA editing
targets:
- Beyond WVE-006, Wave highlighted several undisclosed RNA
editing targets which span prevalent and rare liver, kidney and
lung diseases. As Wave advances its wholly owned pipeline of RNA
editing programs, it is leveraging a proprietary map of the
“edit-verse” to gain novel insights into the editable gene-disease
network and a proprietary deep learning model to identify new
targets and novel edit sites.
- Wave has the potential to advance any combination of these
targets into preclinical development to support its goal of
delivering five new clinical candidates by year-end 2025. All
targets leverage easily accessible biomarkers, offer efficient
paths to proof-of-concept in humans, and represent meaningful
commercial opportunities.
- The targets represent opportunities to correct endogenous
proteins, similarly to WVE-006 in AATD, or to upregulate mRNA to
increase endogenous protein levels. mRNA upregulation is an
application of RNA editing being pioneered at Wave, and one of its
advantages is the potential to address a range of common
diseases.
- Wave demonstrated in vivo or in vitro proof-of-concept with
several of these new targets, achieving at least 2-fold
upregulation in liver and kidney targets and more than 60%
correction in liver and lung targets.
WVE-N531 for Duchenne muscular dystrophy (DMD) and
future pipeline updates:
- Today Wave shared a new analysis of muscle biopsy data from
Part A of the Phase 1b/2a study of WVE-N531. The data indicates
that WVE-N531 was present in myogenic satellite cells, which is
important for potential muscle regeneration. To Wave’s knowledge,
these are the first clinical data in DMD to demonstrate uptake in
satellite cells at this early time point (after three biweekly
doses). In general, data for approved and investigational DMD
therapeutics that demonstrate satellite cell uptake in humans is
extremely limited.
- WVE-N531 will be investigated in a potentially registrational
Phase 2 trial called FORWARD-53. Success in this trial would unlock
a multiexon strategy where Wave can potentially address up to 40%
of the DMD population with its current DMD pipeline, which includes
discovery programs for skipping exons 51, 52, 44 and 45 in addition
to WVE-N531.
- Data from FORWARD-53 are expected in 2024.
WVE-003 clinical program for Huntington’s disease
(HD):
- WVE-003 is currently being investigated in the Phase 1b/2a
SELECT-HD clinical trial in individuals with HD, and is the most
advanced investigational HD therapeutic designed to reduce mutant
huntingtin (mHTT) protein while sparing healthy, wild-type
huntingtin (wtHTT) protein.
- The multidose portion of the SELECT-HD clinical trial is
ongoing and has been enrolling with high demand.
- Wave expects to deliver complete data from the first multidose
cohort with extended follow-up in the second quarter of 2024 to
enable decision-making, in addition to the update on single dose
and available multidose data in the second half of this year.
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 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.
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
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