Wave Life Sciences Announces Initiation of Dosing in Phase 2 FORWARD-53 Trial of WVE-N531 in Duchenne Muscular Dystrophy
15 Dezembro 2023 - 10:30AM
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 the Phase 2 FORWARD-53 clinical trial,
which is evaluating WVE-N531 as a treatment for boys with Duchenne
muscular dystrophy (DMD) who are amenable to exon 53 skipping.
FORWARD-53 is designed to assess functional dystrophin protein at
24 and 48 weeks with every other week dosing of WVE-N531.
“Following encouraging data from the WVE-N531 proof-of-concept
trial, we believe we are on the right path toward addressing a
significant unmet need in DMD – the generation of endogenous
dystrophin protein to levels that meaningfully impact the
trajectory of the disease,” said Anne-Marie Li-Kwai-Cheung, MChem,
MTOPRA, RAPS, Chief Development Officer at Wave Life Sciences. “Our
clinical data in boys with DMD amenable to exon 53 skipping
demonstrated the highest level of exon skipping ever observed in
the clinic, and high muscle concentrations of WVE-N531 in skeletal
muscle of 6.1 micromolar. Importantly, in our non-human primate
studies, muscle concentrations were higher in the diaphragm and
heart than in skeletal muscle. Additionally, while FORWARD-53 is
dosing every other week, we have the potential for monthly dosing
in the future. For these reasons, we are excited about the
potential of WVE-N531. We are grateful to the DMD community for
their continued support and look forward to announcing dystrophin
expression data in 2024, as well as advancing exon skipping
candidates for other mutations if FORWARD-53 is successful.”
FORWARD-53 is a potentially registrational, open-label, Phase 2
clinical trial that has enrolled 10 boys with DMD who are amenable
to exon 53 skipping. The trial is powered to evaluate functional,
endogenous dystrophin expression following 24 and 48 weeks of every
other week, intravenous dosing at 10 mg/kg. The primary endpoint is
dystrophin protein levels, and the trial is also evaluating
pharmacokinetics, digital and functional endpoints, and safety and
tolerability.
FORWARD-53 is fully enrolled, and Wave expects to deliver data,
including dystrophin expression from muscle biopsies, in 2024.
Wave’s Phase 1b/2a Part A proof-of-concept trial in boys with
DMD amenable to exon 53 skipping demonstrated high muscle
concentrations of WVE-N531 (mean of 6.1 micromolar or 42
micrograms/gram) and mean exon skipping of 53% (range: 48-62%) at
six weeks, after boys received three doses of 10 mg/kg every other
week. WVE-N531 appeared safe and well-tolerated, with all
treatment-related adverse events being mild.
Additionally, at Wave’s R&D Day in September 2023, the
company shared an analysis of muscle biopsy data from the Part A
proof-of-concept trial indicating that WVE-N531 was present in
myogenic stem cells, which are important for potential muscle
regeneration. These are the first clinical data in DMD to
demonstrate uptake in myogenic stem cells at the early, six-week
timepoint and further support the potential differentiation of
WVE-N531 from other therapeutics, including gene therapies.
About Duchenne Muscular DystrophyDuchenne
muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular
disorder caused predominantly by out-of-frame deletions in the
dystrophin gene, resulting in absent or defective dystrophin
protein. Dystrophin protein is needed for normal muscle maintenance
and operation. Because of the genetic mutations in DMD, the body
cannot produce functional dystrophin, which results in progressive
and irreversible loss of muscle function, including the heart and
lungs. Worldwide, DMD affects approximately one in 5,000 newborn
boys. Approximately 8%-10% of DMD patients have mutations amenable
to treatment with an exon 53 skipping therapy. Exon skipping aims
to address the underlying cause of DMD by promoting the production
of dystrophin protein to stabilize or slow disease progression.
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 understanding of the anticipated
therapeutic benefit of WVE-N531 as a therapy for DMD; our
expectations for our FORWARD-53 clinical trial and potential
registration; our understanding of the cause of DMD and the
potential addressable patients amenable to treatment with an exon
53 skipping therapy; our expectation that the generation of
endogenous dystrophin protein will have a positive impact on
patients with DMD; our expectations and anticipated timing for
delivering dystrophin data, including muscle biopsies, in DMD
patients treated with WVE-N531; our expectations for advancing exon
skipping candidates for other mutations in DMD; and the potential
benefits of PRISM, including our novel PN backbone chemistry
modifications, and our stereopure oligonucleotides compared with
stereorandom oligonucleotides. 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
DMD Community Contact:Chelley Casey+1
617-949-2900ccasey@wavelifesci.com
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