SAN FRANCISCO, May 3, 2017 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing gene therapy products for
patients living with serious, life-threatening rare diseases, today
announced that data related to its product candidates AT132 for the
treatment of X-linked Myotubular Myopathy, and AT342 for the
treatment of Crigler Najjar Syndrome, will be presented at the 20th
Annual Meeting of the American Society of Gene and Cell Therapy to
be held in Washington, DC on
May 10-13th, 2017.
Four abstracts will be presented during the meeting, underscoring
the company's leadership position and significant advances in the
research, development and manufacture of highly innovative
AAV-based gene therapy products.
John T. Gray, Ph.D, Senior Vice
President, Research and Development, commented, "We are excited by
the opportunity to present important data that support the
advancement of our product candidates to treat XLMTM and
Crigler-Najjar Syndrome. We plan to initiate Phase 1/2
clinical studies of both in mid-2017, and to report preliminary
results from each program by year-end."
Details of the presentations are provided below:
Abstract number: 216
Title: Phillips, et. al., Nonhuman Primate Safety and Potency of an
AAV Vector for XLMTM Produced by Transient Transfection at 500L
Session title: Musculo-skeletal Diseases I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30
PM
Room: Exhibit Hall A & B South
Summary: Administration of AT132 produced at 500L scale
demonstrated an encouraging safety profile and high potency,
providing support for its advancement into clinical testing for
XLMTM patients. Specifically, an intravenous dose of 8 x
1014 vg/kg in NHPs resulted in no significant treatment
related adverse events or safety findings and greater than normal
levels of myotubularin protein expression in skeletal muscle.
Abstract number: 674
Title: Greig, et. al., Evaluation of Efficacy and Safety in a
Dose-Escalating Nonclinical Study of a Clinical Candidate Vector in
a Mouse Model of Crigler-Najjar
Session title: Metabolic, Storage, Endocrine, Liver and
Gastrointestinal Diseases
Session Date/Time: Friday, May 12, 5:45 – 7:45
PM
Room: Exhibit Hall A & B South
Summary: Administration of AT342 resulted in no significant
treatment-related adverse events or safety findings and rapidly
reduced serum bilirubin levels in the mouse model of Crigler-Najjar
syndrome.
Abstract number: 212
Title: DuPont, et. al., RNA-Seq Analysis of Canine X-Linked
Myotubular Myopathy Muscles Before and After Gene Therapy
Session title: Musculo-skeletal Diseases I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30 PM
Room: Exhibit Hall A & B South
Summary: Bioinformatic comparison of RNA-Seq data from diseased
and gene therapy treated muscle reveals gene expression patterns
that correlate with therapeutic correction of X-Linked Myotubular
Myopathy in dogs.
Abstract number: 103
Title: Guo, et. al., RNA-Seq Tag Counting: An Approach for
Quantifying the Relative Expression of Vector Derived and
Endogenous Genes in rAAV-Treated Non-Clinical Models
Session title: AAV Vectors I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30
PM
Room: Exhibit Hall A & B South
Summary: A novel bioinformatic method called 'RNA-Seq Tag
Counting' processes RNA-Seq data to generate quantitative gene
expression values with robust linearity and reproducibility.
About AT132 to Treat X-Linked Mytubular Myopathy
AT132 is the Audentes product candidate being developed to treat
XLMTM, a rare monogenic disease characterized by extreme muscle
weakness, respiratory failure and early death, with an
estimated 50% mortality rate by 18 months of age. XLMTM is
caused by mutations in the MTM1 gene, which encodes a protein
called myotubularin. Myotubularin plays an important role in
the development, maintenance and function of skeletal muscle
cells. AT132 is comprised of an AAV8 vector containing a
functional copy of the MTM1 gene. Multiple studies in animal
models of XLMTM have demonstrated that a single administration of
AT132 was well tolerated and significantly improved disease
symptoms and survival rates, with no significant AT132-related
adverse events or safety findings. In one study these effects
have lasted more than four years to date. The U.S. Food and Drug
Administration has cleared the IND for AT132, and Audentes plans to
initiate ASPIRO, the Phase 1/2 study of AT132 in mid-2017.
Preliminary data from ASPIRO is expected to be available by the end
of 2017.
About AT342 to Treat Crigler-Najjar
Syndrome
AT342 is an AAV8 vector
containing a functional copy of the UGT1A1 gene for the treatment
of Crigler-Najjar Syndrome, a rare monogenic disease characterized
by severely high levels of unconjugated bilirubin in the blood and
risk of irreversible neurological damage and death. The
current standard of care for Crigler-Najjar Syndrome is persistent
phototherapy, usually for longer than 12 hours per day.
Phototherapy wanes in effectiveness beginning around age four, and
a liver transplant may be required for survival. A single
administration of AT342 has generated durable, dose-responsive and
clinically-relevant decreases in total bilirubin levels in a mouse
model of Crigler-Najjar, with no significant AT342-related adverse
events or safety findings. The U.S. Food and Drug
Administration has cleared the IND for AT342, and Audentes plans to
initiate VALENS, the Phase 1/2 study of AT342 in mid-2017.
Preliminary data from VALENS is expected to be available by the end
of 2017.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a biotechnology company focused
on developing and commercializing gene therapy product candidates
for patients living with serious, life-threatening rare
diseases. We have four product candidates in development,
AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM),
AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the
treatment of Pompe disease, and AT307 for the treatment of the
CASQ2 subtype of Catecholaminergic Polymorphic Ventricular
Tachycardia (CASQ2-CPVT). We are a focused, experienced and
passionate team committed to forging strong, global relationships
with the patient, research and medical communities.
For more information regarding Audentes, please
visit www.audentestx.com.
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: whether Audentes has or will
maintain a leadership position in the research, development and
manufacture of highly innovative AAV-based gene therapy products;
and, whether Audentes will initiate Phase 1/2 studies in its XLMTM
and Crigler-Najjar programs in mid-2017, or report preliminary data
from such studies by the end of 2017. 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:
Thomas Soloway, CFO
415.818.1040
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.