Preliminary data from ASPIRO, the ongoing Phase 1/2 Clinical
Study of AT132, expected in fourth quarter of 2017
SAN FRANCISCO, Oct. 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 Medicines and Healthcare Products Regulatory
Agency (MHRA) has approved the Clinical Trial Authorisation (CTA)
application for AT132, the Company's gene therapy product candidate
being developed to treat X-Linked Myotubular Myopathy
(XLMTM). This is the first European CTA approval for the
AT132 program, and it allows Audentes to initiate work with
clinical study sites in the United
Kingdom to enroll patients into ASPIRO, the ongoing Phase
1/2 clinical study of AT132. Preliminary data from ASPIRO is
expected to be available in the fourth quarter of 2017.
"This CTA approval represents another important milestone for
our AT132 program," stated Matthew R.
Patterson, President and Chief Executive Officer. "We
recently announced dosing of the first patient in ASPIRO at a U.S.
clinical study site, and we are pleased to be working closely with
the European XLMTM community as we continue to execute on our
global plans to develop AT132 as a potentially transformative
product to treat this devastating rare disease."
In addition to ASPIRO, the clinical development program for
AT132 includes RECENSUS, a retrospective medical chart review, and
INCEPTUS, a prospective natural history run-in study.
Audentes has recently presented data from both the RECENSUS and
INCEPTUS studies which confirm and expand upon the understanding of
the significant disease burden of XLMTM on patients, families and
the healthcare system.
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 and a half
years to date. Audentes is developing AT132 in collaboration
with Genethon (www.genethon.fr).
About ASPIRO, the 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 estimated timing for preliminary
data from ASPIRO, the potential of AT132 to be a transformative
product for the treatment of XLMTM. 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
Director, Investor Relations
415.818.1033
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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