Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy
February 27 2024 - 8:30AM
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 its
upcoming presentations at the 2024 Muscular Dystrophy Association
(MDA) Clinical & Scientific Conference, taking place March 3-6
in Orlando, FL.
Wave’s poster presentations will highlight the best-in-class
potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which
is currently being evaluated in the Phase 2, potentially
registrational FORWARD-53 study. The presentations will also
illustrate the impact of Wave’s novel PN chemistry on pharmacology
of its exon skipping oligonucleotides. Highlights from the
presentations include:
- Data from the Phase 1b/2 proof-of-concept (Part A) study of
WVE-N531 in boys with DMD amenable to exon 53 skipping, which
demonstrate uptake of WVE-N531 in satellite cells of all
participants in the study. Satellite cells, or muscle stem cells,
are important for muscle regeneration, and this is the first
clinical evidence of satellite cell uptake for any investigational
or approved DMD therapeutic.
- Preclinical data for WVE-N531 in non-human primates, which
demonstrate that Wave’s PN chemistry significantly enhanced drug
concentrations in skeletal muscle, with even higher exposure in the
heart and diaphragm. These data suggest that WVE-N531 muscle
concentrations in the clinic may be higher in heart and diaphragm
than in skeletal muscle. In the previous Phase 1b/2 Part A study,
WVE-N531 demonstrated high skeletal muscle concentrations of
42 μg/g (42,000 ng/g) after three every-other-week doses, which
translated to best-in-class exon skipping (mean of 53%).
- Preclinical data for Wave’s exon skipping programs beyond exon
53, which reinforce the impact of PN chemistry for enabling high
tissue concentrations, exon skipping and dystrophin restoration in
preclinical models. Success with WVE-N531 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 exon
53 with WVE-N531.
“At Wave, we increasingly continue to regard exon skipping as
the preferred mechanism for altering DMD disease progression in
those amenable to this approach. Dystrophin is one of the largest
proteins in the body, and the goal of exon skipping is to enable
the body to restore its own, near full-length protein that retains
integral elements of healthy dystrophin. However, the DMD field’s
ability to realize the potential of exon skipping therapeutics and
clinically meaningful dystrophin levels has been limited by
sub-optimal potency, distribution, and durability of the existing
exon skippers,” said Anne-Marie Li-Kwai Cheung, MChem, MTOPRA,
RAPS, Chief Development Officer at Wave Life Sciences. “With our
novel chemistry, we have markedly improved on the pharmacology of
exon skipping oligonucleotides and have already demonstrated
best-in-class muscle concentrations and exon skipping, and a 25-day
half-life, in the clinic. Our optimism for WVE-N531 is further
bolstered by our satellite cell data, which indicate a potential
for WVE-N531 to repair damaged myofibers and generate new
myofibers. These data distinguish WVE-N531 from all other DMD
therapeutic approaches. We now are evaluating the ability of
WVE-N531 to restore dystrophin in the ongoing Phase 2 FORWARD-53
study and look forward to sharing 24-week data in the third quarter
of 2024.”
Details on Wave’s Presentations
Sunday, March 3, 2024
- WVE-N531 with PN Backbone Modification Significantly
Enhances Drug Concentrations in Heart, Diaphragm, and Skeletal
Muscles in Non-human Primates (Andrew Hart, Scientist II,
Wave Life Sciences)Pre-Clinical Research Poster #S146:00 PM – 8:00
PM ET
- PN-containing Oligonucleotides Yield High Levels of
Exon Skipping and Dystrophin Protein Restoration in Preclinical
Models for DMD (Abbie Maguire, Senior Scientist II, Wave
Life Sciences)Pre-Clinical Research Poster #S106:00 PM – 8:00 PM
ET
Monday, March 4, 2024
- First Clinical Evidence for Satellite Cell Targeting in
DMD: Results from Part A of a Phase 1b/2 Study of WVE-N531
(Kuldeep Singh, Senior Director and Head of Pathology, Wave Life
Sciences)Clinical Trials Poster #M1686:00 PM – 8:00 PM ET
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 best-in-class potential of
WVE-N531 in DMD; the potentially registrational nature of our Phase
2 FORWARD-53 study; the impact of our novel PN chemistry on the
pharmacology of our exon skipping oligonucleotides; our
expectations that high tissue concentrations and high exon skipping
may result in high dystrophin restoration following a sufficient
follow up period; our understanding of the anticipated therapeutic
benefit of WVE-N531 for DMD over existing therapies; our
understanding of the importance of satellite cells for muscle
regeneration; and our expectation that WVE-N531 muscle
concentrations in the clinic may be higher in heart and diaphragm
than in skeletal muscle. 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
Community Contact:Chelley Casey+1
617-949-2900ccasey@wavelifesci.com
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