SAN FRANCISCO, April 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 the U.S. Food and Drug Administration (FDA) has
cleared the investigational new drug (IND) application for AT132,
the Company's gene therapy product candidate to treat X-Linked
Myotubular Myopathy (XLMTM). The IND is now active and
Audentes plans to initiate ASPIRO, the multicenter, multinational,
open-label, ascending dose Phase 1/2 clinical study of
AT132. Preliminary data from ASPIRO is expected to be
available in the fourth quarter of 2017.
Matthew R. Patterson, President
and Chief Executive Officer, stated, "FDA clearance of the AT132
IND is an important milestone for Audentes that builds upon the
momentum established with the recently cleared IND for AT342 to
treat Crigler-Najjar Syndrome and the initiation of internal cGMP
manufacturing for both AT342 and AT132." Mr. Patterson
continued, "Based on the promising preclinical data set for the
program, we believe AT132 has the potential to significantly
improve the lives of patients living with this devastating
disease. We look forward to working closely with the XLMTM
medical community and patient organizations as we execute on our
plans to enter the clinic."
In addition to ASPIRO, the clinical development program for
AT132 includes RECENSUS, a retrospective medical chart review, for
which Audentes recently announced data from an initial analysis of
112 male subjects. This analysis confirmed and expanded upon
the understanding of the medical burden of XLMTM on patients,
families and the healthcare system. Audentes is also
conducting INCEPTUS, a clinical assessment and Phase 1/2 run-in
study. The primary objectives of INCEPTUS are to characterize
the disease course and natural history of children with XLMTM,
assess the burden of disease on XLMTM patients and caregivers,
identify subjects for potential enrollment in ASPIRO, and serve as
a longitudinal baseline and within-patient control for
ASPIRO. As of April 1, 2017, 15
of the up to 16 subjects have been enrolled in INCEPTUS. Audentes
plans to report preliminary data from INCEPTUS in mid-2017.
About AT132 for X-Linked Myotubular 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 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.
About ASPIRO, the Planned Phase 1/2 Clinical Study of
AT132
ASPIRO is designed as a multicenter, multinational,
open-label, ascending dose study to evaluate the safety and
preliminary efficacy of AT132 in approximately 12 XLMTM patients
less than five years of age. The study is expected to include nine
AT132 treated subjects and three delayed-treatment concurrent
control subjects. Primary endpoints include safety (adverse
events and certain laboratory measures) and efficacy (assessments
of neuromuscular and respiratory function). Secondary
endpoints include the burden of disease and health related
quality-of-life, and muscle tissue histology and biomarkers. The
primary efficacy analysis is expected to be conducted at 12 months,
with interim evaluations expected to be conducted at earlier time
points. After the primary 12-month assessment, subjects are
expected to be followed for another four years to assess long term
safety, durability of effect and developmental progression.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a biotechnology company focused
on developing and commercializing gene therapy products 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: the ability to manufacture AT132 and
AT342 in sufficient quantities to supply ASPIRO and VALENS,
respectively; the ability to initiate ASPIRO and report preliminary
data from ASPIRO by the fourth quarter of 2017; the potential of
AT132 to significantly improve the lives of patients; and the
ability to report preliminary data from INCEPTUS in
mid-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:
Jeffrey Gruis
415.818.1015
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.