- First seven treated patients now ventilator
independent and able to rise to a standing position or walk
- Continued significant and durable
improvements in respiratory function and developmental motor
milestones in all treated patients
- AT132 continues to be generally
well-tolerated with a manageable safety profile across both dose
cohorts
- Enrollment of pivotal expansion cohort nearly
complete; on track to submit a BLA in the United States in mid-2020
and a MAA in Europe in the second half of 2020
- Oral presentation scheduled to begin at
1:00pm Central European Summer Time (CEST)/7:00am Eastern Time
(ET). Slides used during today’s presentation will be available on
the Investor + Media section of the Audentes website for
approximately 30 days.
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 new positive data from ASPIRO, the clinical trial
evaluating AT132 in patients with X-Linked Myotubular Myopathy
(XLMTM). The data are being presented today at the 24th
International Annual Congress of the World Muscle Society by Dr.
James J. Dowling, Hospital for Sick Children, Toronto, Canada,
during the Clinical Trial Highlights 7 session scheduled to begin
at 1:00pm Central European Summer Time (CEST)/7:00am Eastern Time
(ET).
"The new ASPIRO data shared today builds upon the encouraging
efficacy and safety profile seen to date with AT132,” stated Dr.
Dowling. “Treated patients across both dose cohorts show
significant reductions in ventilator dependence and the progressive
attainment of developmental motor milestones, suggesting that AT132
has the potential to deliver transformative benefit to patients and
families living with XLMTM.”
“We remain focused on our goal of rapidly progressing AT132
toward global regulatory approvals,” stated Natalie Holles,
President and Chief Operating Officer of Audentes. “Importantly, we
have fully enrolled 14 patients into the ASPIRO dose escalation
cohorts, and plan to complete enrollment of the ASPIRO pivotal
expansion cohort imminently. We remain on track to submit a BLA in
the United States in mid-2020 and a MAA in Europe in the second
half of 2020.”
Data Summary
The newly reported data include safety and efficacy assessments
as of the August 7, 2019 data cut-off date for 12 patients enrolled
in the ASPIRO dose escalation cohorts. The data includes 48 weeks
or more of follow-up for seven patients enrolled in Cohort 1
(1x1014 vector genomes per kilogram (vg/kg); six treated and one
untreated control) and 24-48 weeks of follow-up for five patients
in Cohort 2 (3x1014 vg/kg; four treated and one untreated control).
Key assessments include neuromuscular function as assessed by the
achievement of motor milestones and improvement in CHOP INTEND
score, and respiratory function as assessed by reduction in
ventilator dependence and improvement in maximal inspiratory
pressure (MIP). Today’s presentation does not include new muscle
biopsy data.
Efficacy
Patients receiving AT132 have achieved significant and durable
reductions in ventilator dependence, an endpoint considered to be
closely correlated with morbidity and mortality in XLMTM patients.
To date, the first seven patients treated (all six treated patients
in Cohort 1 and the first patient treated in Cohort 2) have
achieved ventilator independence. All treated patients continue to
show gains in neuromuscular function, with the first seven patients
treated achieving the ability to rise to a standing position, or
walk.
Safety
AT132 has been generally well-tolerated and has shown a
manageable safety profile across both dose groups. Since the last
data update in May 2019, there has been one new serious adverse
event (SAE) in Cohort 2, an episode of joint swelling that resolved
without treatment. Results to date indicate no clinically
meaningful differences in the safety and tolerability profile of
AT132 between the 1x1014 vg/kg and 3x1014 vg/kg dose cohorts.
Next steps in the AT132 development program include the
completion of enrollment and follow-up of patients in the ASPIRO
pivotal expansion cohort, designed to confirm the safety and
efficacy profile of AT132 at a dose of 3x1014 vg/kg, and
preparations for filing of a Biologics License Application (BLA)
for AT132 in the United States planned in mid-2020 and filing of a
Marketing Authorization Application (MAA) in Europe planned for the
second half of 2020.
About 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 X-linked Myotubular
Myopathy
Audentes is developing AT132, an AAV8 vector containing a
functional copy of the MTM1 gene, for the treatment of 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. 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 of regulatory filings for AT132 and planned
activities for the ASPIRO pivotal expansion. 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/20191005005003/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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