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WAVE Life Sciences 2017 Pipeline Update
Three Lead Neurology Programs to
“2017 will be an important year for WAVE as we transition our two lead
candidates in Huntington’s disease and our exon-skipping candidate in
Duchenne Muscular Dystrophy into clinical trials,” said
Updated clinical development plan for lead Huntington’s disease (HD)
programs WVE-120101 and WVE-120102
Informed by discussions with
the
As part of the company’s HD global development strategy, WAVE remains on
track to file a Clinical Trial Application (CTA) in
WVE-120101 and WVE-120102 each target a distinct patient population,
which together account for over two-thirds of the HD population. Each
therapeutic candidate is designed to selectively silence mRNA transcript
produced by the disease-causing mutant huntingtin (HTT) allele in order
to reduce the mutant HTT protein while leaving the healthy HTT allele
intact to produce normal functioning protein. If approved, WVE-120101
and WVE-120102 would be the first allele-specific therapies for
Huntington’s disease patients. Huntington’s disease is an autosomal
dominant genetic disorder, involving the HTT gene, characterized by
chorea, psychiatric illness and cognitive decline. HD is a devastating
condition that is invariably fatal affecting over 30,000 symptomatic
individuals in
Duchenne Muscular Dystrophy program entering clinic in 2H 2017
WAVE
has selected its stereopure exon-skipping candidate, WVE-210201, to
target deletions of Exon 51. Pre-clinical quantitative Western blot
studies of WAVE’s DMD Exon 51 candidate demonstrated 52% dystrophin
protein restoration as compared with normal skeletal muscle tissue
lysates, versus approximately 1% when testing other exon-skipping
therapies. WAVE is developing clinical trial protocol in collaboration
with the DMD community and intends to include both ambulatory and
non-ambulatory patients in the study as well as those previously treated
with other exon skipping therapies. GMP manufacturing is underway to
support planned clinical trials to ensure adequate supply for current
and planned studies. The company is on track to initiate a global
clinical program in the second half of 2017.
Continued progress unlocking value in adjacent therapeutic areas
WAVE
continues to make progress under its collaboration with Pfizer to
develop genetically targeted therapies for the treatment of metabolic
diseases, including NASH and NAFLD, with three programs advancing
through lead-optimization, including ApoC-III programs. The
collaboration leverages WAVE’s stereochemistry platform across antisense
and RNAi modalities and incorporates GalNAc and Pfizer’s hepatic
targeting technology. The remaining two Pfizer collaboration programs
are expected to commence by
Establishing internal GMP manufacturing capabilities
To
provide internal GMP manufacturing capabilities and increase control and
visibility of our manufacturing supply chain, WAVE recently signed a
lease for a manufacturing facility of approximately 90,000 square feet
in Lexington, MA. This new facility supplements WAVE’s existing
About
At
Forward Looking Information
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: the anticipated timing of
our IND filings and the commencement of our clinical trials; the design
and anticipated goals of our clinical trials; the future performance and
results of our programs in clinical trials; the progress and potential
benefits of our collaborations with partners; our identification of
future candidates and their therapeutic potential; the anticipated
therapeutic benefits of stereopure therapies compared to other
therapies; our advancing of therapies across multiple modalities and the
anticipated benefits of that strategy; our future growth; and the
potential of our stereopure approach and nucleic acid therapeutics
generally. 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 preclinical
programs to produce data sufficient to support the filing of INDs and
the timing thereof; our ability to continue to build and maintain the
company infrastructure and personnel needed to achieve our goals; the
clinical results of our programs, which may not support further
development of product candidates; actions of regulatory agencies, which
may affect the initiation, timing and progress of clinical studies; our
effectiveness in managing future clinical trials and regulatory
processes; the success of our platform in identifying viable candidates;
the continued development and acceptance of nucleic acid therapeutics as
a class of drugs; our ability to demonstrate the therapeutic benefits of
our stereopure candidates in clinical trials, including our ability to
develop candidates across multiple therapeutic modalities; our ability
to obtain, maintain and protect intellectual property; our ability to
enforce our patents against infringers and defend our patent portfolio
against challenges from third parties; our ability to raise additional
capital as needed; 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 (
View source version on businesswire.com: http://www.businesswire.com/news/home/20170106005730/en/
Source:
Media and Investor Contact:
WAVE Life Sciences
Jillian
Connell, 617-949-2981
Head of Investor Relations
jconnell@wavelifesci.com