SAN FRANCISCO, Sept. 21, 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 it has commenced dosing of patients in ASPIRO, a Phase
1/2 clinical trial of AT132 for the treatment of X-Linked
Myotubular Myopathy (XLMTM). ASPIRO is a multicenter,
multinational, open-label, ascending dose study to evaluate the
safety and preliminary efficacy of AT132 in approximately twelve
XLMTM patients less than five years of age. Preliminary data
from ASPIRO is expected to be available in the fourth quarter of
2017.
"XLMTM is a severe rare disease characterized by profound muscle
weakness, respiratory failure and early death," stated Suyash Prasad, M.D., Senior Vice President and
Chief Medical Officer. "Over 50 percent of the boys affected
by XLMTM do not live to celebrate their second birthday, and no
currently approved treatment options exist for these patients,
their families and care-givers."
Dr. Prasad continued, "We are grateful to the many expert
collaborators, patient advocacy organizations and parents who have
supported Audentes and helped guide our efforts. Most of all
we are thankful to the children and families affected by XLMTM for
their participation in ASPIRO. We look forward to working together
with our partners to advance AT132 as a potentially transformative
therapy to treat this devastating disease."
In addition to ASPIRO, the clinical development program for
AT132 includes RECENSUS, a retrospective medical chart review, for
which Audentes has previously announced data from an initial
analysis of 112 male subjects. This analysis confirmed and
expanded upon the understanding of the significant disease burden
of XLMTM on patients, families and the healthcare system. Audentes
is also conducting INCEPTUS, a prospective natural history run-in
study. The primary objectives of INCEPTUS are to characterize
the clinical condition of children with XLMTM, identify subjects
for potential enrollment in ASPIRO, and serve as a longitundinal
baseline and within-patient control for ASPIRO. Audentes plans
to announce preliminary data from INCEPTUS in the third quarter of
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 and a half years
years. 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.
About Genethon
Genethon, located in Evry, France, is a non-profit R&D organization
dedicated to the development of biotherapies for orphan genetic
diseases, from research to clinical validation. Genethon is
specialized in the discovery and development of gene therapy drugs
and has multiple ongoing programs at clinical, preclinical and
research stage for neuromuscular, blood, immune system, liver and
eye diseases.
Discover Genethon's pipeline:
http://www.genethon.fr/produits/
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 preliminary data from
ASPIRO, the timing of data presentation from INCEPTUS and the
ability of INCEPTUS to serve as a longitudinal baseline and within
patient control for ASPIRO, and the potential of AT132 to be a
transformative therapy for patients living with 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:
Thomas Soloway, CFO
415.818.1040
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.