WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–WAVE Life Sciences Ltd. (NASDAQ:WVE), a genetic medicines company
focused on developing stereopure nucleic acid therapies for patients
impacted by rare diseases, today reaffirmed its commitment to advance
next-generation nucleic acid therapies to address the significant unmet
need of patients diagnosed with Duchenne Muscular Dystrophy (DMD).
WAVE’s DMD program is based on preclinical data demonstrating an
approximate 25-fold improvement in exon-skipping efficiency compared to
drisapersen and eteplirsen, suggesting the potential for improved
potency and an enhanced ability to restore the production of functional
dystrophin. In addition, WAVE’s proprietary muscle targeting technology
has demonstrated substantial improvement in distribution to critical
tissues in animal models, including skeletal muscle, diaphragm, and
heart.
“We recognize the acute need of the Duchenne community for therapeutic
options to address this devastating disease, and also appreciate
regulators’ requirements for strong, well-validated, scientific
evidence. Our goal is to fulfill both of these needs by bringing forward
optimally designed drugs through robust clinical trials,” said Paul
Bolno, M.D., MBA, President and CEO of WAVE Life Sciences. “Based on the
strong preclinical data we’ve seen to date, we are highly encouraged
that we are on track to develop exon-skipping medicines that maximize
potency with a favorable safety profile. In addition, we plan to conduct
rigorous, well-designed clinical trials that explore various predictive
biomarkers and evaluate comprehensive endpoints.”
WAVE’s planned clinical trials will include both ambulatory and
non-ambulatory patients in order to evaluate preservation of walking
ability, heart and lung function.
WAVE’s initial DMD candidate skips exon 51 in the dystrophin gene. The
company will initiate IND-enabling studies in 2016 and intends to
initiate its first clinical trial of the candidate in the second half of
2017. WAVE will also leverage its novel stereopure chemistry platform to
advance therapies targeting additional DMD-related exons to expand the
potential impact of its promising approach to a broader group of
patients.
WAVE will present data at the upcoming Parent Project Muscular
Dystrophy’s (PPMD) Annual Connect Conference being held June 26 – 29,
2016 in Orlando, Florida.
“We are excited whenever a new company enters the Duchenne space,” said
Pat Furlong, Founding President and CEO of PPMD. “Individuals with
Duchenne need and deserve options, and we are hoping this will become
another tool in our arsenal.”
As part of its commitment to developing better medicines for DMD, WAVE
has extended its ongoing research collaboration with the University of
Oxford to advance stereopure nucleic acid therapies for DMD across
exons. Through the collaboration, renowned researcher Matthew Wood,
M.D., Ph.D., Professor of Neuroscience, Department of Physiology,
Anatomy and Genetics, Medicine Sciences Division, University of Oxford,
and his team will continue to work with WAVE to use the company’s
proprietary platform to enhance oligonucleotide approaches, including
exon-skipping, to address the rare genetic muscle disease.
“The data we have seen to date using WAVE’s novel approach to
exon-skipping in DMD is very promising. I believe that academia and
industry, working together, may be on the verge of a veritable medical
revolution where we can potentially effectively and durably treat
genetically based diseases such as DMD,” said Professor Wood.
“Collaborations between academia and industry are critical now more than
ever in order to collectively harness the latest scientific advancements
to rapidly progress therapies for patients. We look forward to expanding
our collaboration with the WAVE team and advancing the potential and
benefits of stereopure oligonucleotide approaches for DMD.”
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic
neuromuscular disorder caused by mutations in the gene that encodes for
the protein dystrophin. Dystrophin is needed for muscle maintenance and
operation. Because of the genetic mutations, the body cannot produce
functional dystrophin. Without dystrophin, muscles cannot work properly,
and patients experience progressive loss of strength and serious medical
complications involving the heart and lungs. DMD affects approximately
one in 3,500 newborn boys around the world; approximately 13% have
mutations in Exon 51. There are currently no approved disease-modifying
drugs available to treat DMD. Exon-skipping drugs are designed to “skip
over” the mutated exon, enabling the gene to once again code for and
produce functional dystrophin.
About WAVE Life Sciences
At WAVE Life Sciences, we are driven by an unwavering passion and
commitment to deliver on our mission of confronting challenging diseases
by developing transformational therapies and empowering patients. We are
utilizing our innovative and proprietary synthetic chemistry drug
development platform to design, develop and commercialize stereopure
nucleic acid therapeutics that precisely target the underlying cause of
rare and other serious genetically defined diseases. Given the
versatility of our chemistry platform, WAVE’s deep, diverse pipeline
spans multiple modalities including antisense, exon-skipping, and
single-stranded RNAi. For more information, please visit www.wavelifesciences.com and
follow us on Twitter at @WAVELifeSci
and LinkedIn.
Forward Looking Statements
This press release contains forward-looking statements concerning our
goals, beliefs, expectations, strategies, objectives and plans, and
other statements that are not necessarily based on historical facts,
including statements regarding the following: our commencing of
IND-enabling studies and clinical trials involving our exon 51
candidate; the future performance and results of our exon 51 candidate
in clinical trials; our identification of future candidates targeting
DMD and their therapeutic potential; the ability of industry and
academia to effectively treat DMD; and the potential of our stereopure
approach and nucleic acid therapeutics. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important factors, including the following: the
ability of our IND-enabling studies to produce data sufficient to
support the filing of an IND for our exon 51 candidate and the timing
thereof; our ability to demonstrate the therapeutic benefits of our exon
51 candidate in clinical trials; the success of our platform in
identifying additional viable DMD candidates; the continued development
and acceptance of nucleic acid therapeutics as a class of drugs; and
competition from others developing therapies for similar uses, as well
as the information under the caption “Risk Factors” contained in our
Annual Report on Form 10-K filed with the Securities and Exchange
Commission (SEC) and in other filings we make with the SEC from time to
time. We undertake no obligation to update the information contained in
this press release to reflect subsequently occurring events or
circumstances.