SAN FRANCISCO, April 15, 2019 /PRNewswire/ -- 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 multiple planned presentations at the 22nd Annual
Meeting of the American Society of Gene and Cell Therapy (ASGCT) in
Washington, D.C., including new
data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in patients
with X-linked Myotubular Myopathy (XLMTM). The oral
presentation will be given by Perry B.
Shieh, M.D., Ph.D., Associate Professor of Neurology at the
Department of Neurology at UCLA Medical Center and will be held
during the Plenary Session "Presidential Symposium &
Presentation of the Top Abstracts," scheduled to begin at
2:15 pm ET.
The presentation by Dr. Shieh will include new data from ASPIRO,
with up to 48-weeks of follow-up in all patients enrolled in dose
Cohort 1 (1x1014 vg/kg), and up to 24-weeks in the
earliest patients enrolled in dose Cohort 2 (3x1014
vg/kg).
"We remain highly encouraged by the emerging profile of AT132
and are looking forward to sharing new results from ASPIRO," stated
Matthew R. Patterson, Chairman and
Chief Executive Officer. "2019 is a critical year for the
development of AT132, with important next steps that include
optimal dose selection and final agreement on regulatory submission
pathways in the US and Europe,
which we expect to achieve in the third quarter."
Audentes has a range of additional presentations planned during
the conference, including a sponsored symposium that will provide
an in-depth review of the new data from ASPIRO. Following are
details for each presentation:
Oral Presentations:
ASPIRO Phase 1/2 Gene Therapy Trial In X-Linked Myotubular
Myopathy (XLMTM): Update on Preliminary Safety And Efficacy
Findings
Abstract number: 652
Session Title: Presidential Symposium & Presentation of the Top
Abstracts
Date: Wednesday, May 1, 2019,
2:15 pm - 2:30 pm ET
Combination Therapy Reduces Existing Anti-AAV Antibody by
Logs and Allows for Safe and Efficacious Redosing
Abstract
number: 147
Session Title: Use of Immune Modulation in Gene Therapy
Date: Monday, Apr 29, 2019,
4:00 pm - 4:15 pm ET
Development of an NGS-Based Assay and Bioinformatics Workflow
for CMC Characterization of Contaminating DNA in AAV
Products
Abstract number: 700
Session Title: Advances in Cell Product and Gene Vector
Manufacturing
Date: Wednesday, May 1, 2019,
4:15 pm - 4:30 pm ET
Poster Presentation:
AAV9-U7snRNA Mediated Skipping of DMD Exon 2: Absence of
Off-target Splicing Effects as Demonstrated by
RNA-Seq
Abstract number: 814
Session Title: Musculo-skeletal Diseases
Date: Wednesday, May 1, 2019,
5:00 pm - 6:00 pm ET
Audentes-Sponsored Symposium:
Achieving New
Milestones in Neuromuscular Disease: AAV Gene Therapy for X-linked
Myotubular Myopathy
Wednesday, May 1,
2019 6:00 pm - 7:30 pm ET
Jefferson Room, Washington
Hilton
The Audentes-sponsored symposium will provide an in-depth review
of the latest developments in ASPIRO, the Phase 1/2 study of AT132
in patients with XLMTM. Speakers will offer a range of
clinical perspectives on the latest data and will include leaders
from the fields of pediatric pulmonology, neurology, and muscle
pathology.
Speakers:
Alan Boyd, MB, ChB, FRSB, FRCP, FFPM
Chief Executive
Officer, Boyd Consultants, Crewe,
UK
Fellow and Immediate Past President of the Faculty of
Pharmaceutical Medicine at the Royal College of Physicians
Suyash Prasad,
M.D.
Senior Vice President, Chief Medical Officer, Audentes
Therapeutics
Mike Lawlor, M.D.,
Ph.D.
Associate Professor, Pediatric Pathology Clinical
Neuromuscular Laboratory and Congenital Muscle Disease Tissue
Repository, Medical College of
Wisconsin
Geovanny Perez, M.D.,
MSc
Assistant Professor of Pediatrics & Pulmonary
Medicine, George Washington University School
of Medicine and Health Sciences
Perry B. Shieh, M.D.,
Ph.D.
Associate Professor of Neurology, University of California Los Angeles
About X-linked Myotubular Myopathy
X-linked
Myotubular Myopathy (XLMTM) is a serious, life-threatening, rare
neuromuscular disease that is characterized by extreme muscle
weakness, respiratory failure, and early death. Mortality rates are
estimated to be 50 percent in the first 18 months of life, and for
those patients who survive past infancy, there is an estimated 25%
mortality by the age of 10. XLMTM is caused by mutations in the
MTM1 gene that lead to a lack or dysfunction of myotubularin, a
protein that is needed for normal development, maturation, and
function of skeletal muscle cells. The disease affects
approximately 1 in 40,000 to 50,0000 newborn males.
XLMTM places a substantial burden of care on patients, families
and the healthcare system, including high rates of healthcare
utilization, hospitalization and surgical intervention. More than
80 percent of XLMTM patients require ventilator support, and the
majority of patients require a gastrostomy tube for nutritional
support. In most patients, normal developmental motor milestones
are delayed or never achieved. Currently, only supportive treatment
options, such as ventilator use or a feeding tube, are
available.
About AT132 for the treatment of XLMTM
Audentes is
developing AT132, an AAV8 vector containing a functional copy of
the MTM1 gene, for the treatment of X-linked Myotubular Myopathy
(XLMTM). AT132 may provide patients with significantly improved
outcomes based on the ability of AAV8 to target skeletal muscle and
increase myotubularin expression in targeted tissues following a
single intravenous administration.
Audentes has reported promising safety, efficacy, and muscle
biopsy data from ASPIRO, an ongoing, multicenter, ascending dose
Phase 1/2 clinical study designed to evaluate the safety and
efficacy of AT132. The preclinical development of AT132 was
conducted in collaboration with Genethon (www.genethon.fr).
AT132 has been granted Regenerative Medicine and Advanced
Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug
designations by the U.S. Food and Drug Administration (FDA), and
Priority Medicines (PRIME) and Orphan Drug designations by the
European Medicines Agency (EMA).
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.
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 potential benefits of AT132 and
the expected timing for optimal dose selection and final agreement
on regulatory submission pathways for AT132. 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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