- Oral presentation of ASPIRO results to
include additional efficacy and safety data on all patients in dose
Cohort 1 and the first treated patients in dose Cohort 2
- Additional presentations include data from
INCEPTUS, a prospective natural history run-in study to
characterize the clinical condition of patients with XLMTM, and an
Audentes-sponsored Symposium, “Altering the Treatment Paradigm:
Gene Therapy for Neuromuscular Disorders”
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 its planned presentations at the 24th International
Annual Congress of the World Muscle Society in Copenhagen, Denmark,
including new data from ASPIRO, the clinical trial evaluating AT132
in patients with X-linked Myotubular Myopathy. The oral
presentation will be given by Dr. James J. Dowling, Hospital for
Sick Children, Toronto, Canada, and will be held during the
Clinical Trial Highlights 7 session of the conference on Saturday,
October 5 beginning at 1:00pm Central European Summer Time
(CEST)/7:00am Eastern Time (ET).
Dr. Dowling will present new efficacy and safety data from the
ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2
controls). Efficacy analyses will include assessments of ventilator
dependence and achievement of developmental motor milestones.
“We are excited to share new efficacy and safety data from our
ASPIRO study,” stated Natalie Holles, President and Chief Operating
Officer. “The data from these dose escalation cohorts, along with
results from our ongoing pivotal expansion cohort, will form the
basis of the AT132 BLA submission planned in mid-2020, and MAA
submission planned for the second half of 2020.”
Audentes is planning several additional presentations during the
conference, including a company-sponsored symposium that will
provide an in-depth review of XLMTM and the ASPIRO study results.
Following are details for each presentation:
Oral Presentation:
ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy
(XLMTM): Update on Preliminary Safety And Efficacy Findings
Session: Clinical Trial Highlights 7 Date and time: Saturday,
October 5, 1:00-2:00pm Central European Summer Time
(CEST)/7:00am-8:00am Eastern Time (ET) Abstract number: O.39
Poster Presentation:
INCEPTUS Pre-Phase 1, Prospective, Non-Interventional,
Natural History Run-in Study to Evaluate Subjects Aged 4 Years and
Younger with X-Linked Myotubular Myopathy (XLMTM) Session:
Congenital myopathies: centronuclear and others Date and time:
October 2, 2019, 4:45pm - 6:45pm Central European Summer Time
(CEST)/10:45am – 12:45pm Eastern Time (ET) Abstract number:
P.105
Audentes-Sponsored
Symposium:
Altering the Treatment Paradigm: Gene Therapy for
Neuromuscular Disorders Date and time: Friday, October 4,
1:15pm–2:45pm Central European Summer Time (CEST)/7:15am – 8:45am
(ET) Location: Axelborg Hall
Agenda and Speakers: AAV
Gene Therapy for Neuromuscular Disorders Benedikt Schoser, MD
(Chair) Friedrich-Baur Institute, Ludwig-Maximilians
University, Munich, Germany
Gene Therapy for XLMTM: The ASPIRO Study
The Potential of AAV for Neuromuscular
Disorders Edward Conner, MD Senior Vice President and Chief
Medical Officer, Audentes Therapeutics, San Francisco, CA, USA
Improvements in XLMTM Muscle Pathology and
Biomarkers Michael W Lawlor, MD, PhD Department of Pathology
and Laboratory Medicine and Neuroscience Research Center, Medical
College of Wisconsin, Milwaukee, WI, USA
Achieving Ventilator Independence with AT132
in XLMTM Robert Graham, MD Division of Critical Care
Medicine, Boston Children’s Hospital, Boston, MA, USA
Attaining Motor Developmental Milestones in
Children with XLMTM Laurent Servais, MD, PhD Muscular
Dystrophy UK Oxford Neuromuscular Centre, Oxford, UK and Liège
University, Liège, Belgium
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 additional 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
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 timing and nature of the ASPIRO pivotal expansion
and the ASPIRO clinical data results and the timing and nature of
regulatory filings 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 and obtain regulatory approval of and
ultimately commercialize 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190930005158/en/
Audentes Contacts:
Investor Contact: Andrew Chang
415.818.1033 achang@audentestx.com
Media Contact: Sarah Spencer
415.957.2020 sspencer@audentestx.com
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