SAN FRANCISCO,
April 8, 2019 /PRNewswire/ --
Conference call and webcast Monday, April 8, 2019 at 8:30 am ET
Webcast may be
accessed via the Investor and Media page of the Audentes
website
Call may be accessed by dialing
(833) 659-8620 (U.S.) or (409) 767-9247 (international) and using
conference ID# 8879216
Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading
AAV-based genetic medicines company focused on developing and
commercializing innovative products for serious rare neuromuscular
diseases, today announced it has expanded its scientific platform
and pipeline to advance vectorized antisense treatments for the
treatment of Duchenne muscular dystrophy (DMD) and myotonic
dystrophy type 1 (DM1). This approach combines the delivery
power of AAV with the precision tools of antisense
oligonucleotides, or ASOs, to develop potential best-in-class
therapeutic candidates for these devastating neuromuscular
diseases. To accelerate these promising new programs,
Audentes has entered into a licensing agreement and will
collaborate with Nationwide Children's Hospital, utilizing the
expertise of Kevin M. Flanigan, M.D.
and Nicolas S. Wein, Ph.D., two
recognized leaders in the field of genetic medicines for
neuromuscular diseases.
"Today's announcement represents a significant step
forward in expanding our scientific platform and deepening our
pipeline of product candidates for neuromuscular diseases with high
unmet medical need," stated Matthew R.
Patterson, Chairman and Chief Executive Officer. "We
see tremendous potential in combining AAV with validated
oligonucleotide-based approaches to treat diseases that are not
amenable to traditional AAV-based gene replacement. We
believe this approach, combined with our in-house large-scale cGMP
manufacturing capability, can deliver best-in-class therapies for
the treatment of Duchenne muscular dystrophy and myotonic
dystrophy."
"We are excited to be collaborating with Audentes to
advance these novel, highly differentiated approaches for DMD and
DM1," stated Dr. Flanigan, Director of Nationwide
Children's Center for Gene Therapy.
Vectorized exon skipping uses an AAV vector to deliver an
antisense sequence designed to induce cells to skip over faulty or
misaligned sections of genetic code, leading to the expression of a
more complete, functional protein. For the treatment of DMD,
this approach has the potential to provide significant advantages
over microdystrophin gene replacement strategies that produce a
substantially truncated protein, which may limit the degree and
durability of disease correction, as well as existing ASO
therapies, whose efficacy is limited by poor biodistribution to
muscle tissue.
Audentes and Nationwide Children's are collaborating to
develop AT702, an AAV-antisense candidate designed to induce exon 2
skipping for DMD with duplications of exon 2 and mutations in exons
1-5 of the dystrophin gene. In preclinical studies of mice
with exon 2 duplications, AT702 demonstrated robust
proof-of-concept with dose-dependent increases in production of
wild type or near-wild type length dystrophin protein and
improvements in muscle function. Audentes is
currently conducting additional preclinical work and expects to
commence a Phase 1/2 study at Nationwide Children's in the fourth
quarter of 2019.
Separate from the Nationwide Children's collaboration,
Audentes is also conducting preclinical work to advance AT751 and
AT753, additional vectorized exon skipping candidates, to treat DMD
patients with genotypes amenable to exon 51 and exon 53
skipping. Both AT751 and AT753 utilize the same vector
construct backbone as AT702, enabling a potentially accelerated
path into clinical development. With these initial programs,
Audentes is targeting more than 25% of patients with DMD, and has
plans to leverage its vectorized exon skipping platform to develop
further product candidates to address up to 80% of DMD patients
over time.
In addition, Audentes and Nationwide Children's are
evaluating vectorized RNA knockdown and vectorized exon skipping
for myotonic dystrophy type 1 (DM1). Both approaches are
designed to prevent the accumulation of toxic dystrophia
myotonica-protein kinase (DMPK) RNA in affected cells, thereby
restoring normal cellular function. RNA knockdown and exon skipping
have both been clinically validated in studies with ASOs. As
with DMD, combining these approaches with AAV delivery is expected
to overcome the biodistribution limitations of ASO-based
therapies. Preclinical studies are underway, and
Audentes expects an IND for the selected product candidate, AT466,
to be filed in 2020.
Audentes plans to leverage its proprietary AAV gene
therapy technology platform, consisting of end-to-end internal
expertise from vector construct engineering to state-of the-art
large-scale cGMP manufacturing, to rapidly advance these programs
from discovery through clinical development. Internal process
and analytical development, fill-finish, and QC testing
capabilities complete the company's fully-integrated approach to
production and release of product candidates for clinical and
commercial use. The current 1,000-liter scale
manufacturing operation provides enough capacity for global
commercialization of the company's lead program AT132, as well as
continued clinical development of pipeline programs, and the
facility is designed for expansion to include an additional 8,000
liters of production capacity.
Conference Call
At 8:30 a.m. Eastern Time today, April 8, 2019, Audentes management will host a
conference call and simultaneous webcast to discuss the expansion
of its AAV technology platform and new development programs for DMD
and DM1. To access a live webcast of the conference call and
the slides used during today's presentation, please visit the
Investor and Media page of the Audentes website at
www.audentestx.com. Alternatively, please call (833) 659-8620
(U.S.) or (409) 767-9247 (international) and dial the conference
ID# 8879216 to access the call. A replay of the webcast and
slides will be available on the Audentes website for approximately
30 days.
About Duchenne Muscular Dystrophy
Duchenne muscular
dystrophy (DMD) is the most common type of muscular dystrophy in
children, affecting approximately 1 in 3,500 to 5,000 male births,
with more than 300,000 patients living with the disease
worldwide. DMD is caused by mutations in the dystrophin gene,
which encodes the protein dystrophin, a structural protein involved
in maintaining muscle cell integrity. Patients with DMD
typically develop muscle weakness in the early years of life and
become wheelchair-bound in their early teens. As the disease
progresses, patients typically develop respiratory, orthopedic, and
cardiac complications. Cardiomyopathy and breathing
difficulties usually begin by the age of 20, and few individuals
with DMD live beyond their thirties. There is no cure for
DMD, and for most patients, there are no satisfactory symptomatic
or disease-modifying treatments.
About Myotonic Dystrophy Type 1
Myotonic dystrophy
type 1 (DM1), is a rare, neuromuscular disease that affects
multiple organ systems, and is characterized primarily by myotonia
and progressive muscle wasting and weakness. DM1 has several
forms which range in age of presentation and severity, including
congenital, infantile, juvenile, and adult (classic). There are
more than 100,000 patients living with DM1 across the United States, Europe, and Japan. The disease is inherited in an
autosomal dominant pattern and is caused by a mutation called a CTG
trinucleotide repeat in the dystrophia myotonica-protein kinase
(DMPK) gene. Patients with DM1 experience reduced quality of
life and shortened life expectancy. There are no disease
modifying therapies approved for DM1.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic
medicines company focused on developing and commercializing
innovative products for serious rare neuromuscular diseases.
We are leveraging our AAV gene therapy technology platform and
proprietary manufacturing expertise to develop programs across
three modalities: gene replacement, vectorized exon skipping, and
vectorized RNA knockdown. Our product candidates are showing
promising therapeutic profiles in clinical and preclinical studies
across a range of neuromuscular diseases. Audentes is a
focused, experienced and passionate team driven by the goal of
improving the lives of patients.
For more information regarding Audentes, please visit
www.audentestx.com.
About Nationwide Children's Hospital
Named to the Top
10 Honor Roll on U.S. News & World Report's 2018-19 list
of "Best Children's Hospitals," Nationwide Children's Hospital is
one of America's largest not-for-profit freestanding pediatric
health care systems providing wellness, preventive, diagnostic,
treatment and rehabilitative care for infants, children and
adolescents, as well as adult patients with congenital disease.
Nationwide Children's has a staff of more than 13,000 providing
state-of-the-art pediatric care during more than 1.4 million
patient visits annually. As home to the Department of Pediatrics of
The Ohio State University College of
Medicine, Nationwide Children's physicians train the next
generation of pediatricians and pediatric specialists. The Research
Institute at Nationwide Children's Hospital is one of the top 10
National Institutes of Health-funded freestanding pediatric
research facilities. More information is available at
NationwideChildrens.org.
Forward Looking Statements
This
press release contains forward-looking statements within the
meaning of the "safe harbor" provisions of the Private Securities
Litigation Reform Act of 1995, including, but not limited to, the
timing and nature of research and development activities, the
nature of the results of data, the timing of regulatory filings,
the expected market size of DMD and DM1 and potential penetration
into those markets, anticipated manufacturing activities and the
expected benefits of the company's product candidates and
technology platform. All statements other than statements of
historical fact are statements that could be deemed forward-looking
statements. Although the company believes that the
expectations reflected in such forward-looking statements are
reasonable, the company cannot guarantee future events, results,
actions, levels of activity, performance or achievements, and the
timing and results of biotechnology development and potential
regulatory approval is inherently uncertain. Forward-looking
statements are subject to risks and uncertainties that may cause
the company's actual activities or results to differ significantly
from those expressed in any forward-looking statement, including
risks and uncertainties related to the company's ability to advance
its product candidates, obtain regulatory approval of and
ultimately commercial its product candidates, the timing and
results of preclinical and clinical trials, the company's ability
to fund development activities and achieve development goals, the
company's ability to protect intellectual property and other risks
and uncertainties described under the heading "Risk Factors" in
documents the company files from time to time with the Securities
and Exchange Commission. These forward-looking statements
speak only as of the date of this press release, and the company
undertakes no obligation to revise or update any forward-looking
statements to reflect events or circumstances after the date
hereof.
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contacts:
Sarah
Spencer
415.957.2020
sspencer@audentestx.com
Katie Hogan
415.951.3398
khogan@audentestx.com
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SOURCE Audentes Therapeutics, Inc.