SAN FRANCISCO, Nov. 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 the publication of "A Multicenter, Retrospective Medical
Record Review of X-Linked Myotubular Myopathy: The RECENSUS Study,"
in Muscle & Nerve. This publication from RECENSUS,
a study sponsored by Audentes, expands upon the data first
presented at the Muscular Dystrophy Association (MDA) Scientific
Conference in March 2017, and
reinforces the high mortality and significant disease burden
experienced by children with XLMTM and their families.
The results from 112 boys enrolled in RECENSUS form one of the
largest peer-reviewed publications of data illustrating the
substantial clinical, humanistic and economic burden of XLMTM.
In this dataset, overall mortality was 44% (64% of patients
≤18 months of age; 32% of patients >18 months of age). At
birth, 95% of the boys were hypotonic and 90% required some form of
respiratory support. After the immediate neonatal period,
once patients have been stabilized, almost half (48%) required
24-hour ventilation, while the remaining boys averaged 8.5 hours
per day on a ventilator. 60% of the boys in the study had
undergone a tracheostomy. Critically, the data show that
infants with XLMTM spend, on average, 35% of their first year of
life in the hospital and undergo an average of 3.7 surgeries during
that time.
These data are consistent with previously published reports
demonstrating that XLMTM is a devastating, life-threatening
disorder that manifests early in the neonatal period and has
considerable, ongoing unmet medical need. The data also illustrate
that the time from presentation of symptoms to a confirmed
diagnosis of XLMTM is declining (from a mean of 35.1 months in the
period 1996-2000, to 4.4 months in the period 2011-2014).
This is likely due to an increasing awareness of XLMTM
coupled with improved diagnostic techniques, and may help with
appropriate intervention and management of patients early in their
lives.
"RECENSUS has established one of the largest data sets of
genetically-confirmed XLMTM in the world, and this analysis makes a
vital contribution to our understanding of this disease," stated
Dr. Alan Beggs, PhD, Director of The
Manton Center for Orphan Disease Research at Boston Children's
Hospital, Sir Edwin and Lady Manton Professor of Pediatrics at
Harvard Medical School, and Principal
Investigator of the RECENSUS study. "The RECENSUS study more
completely defines the disease burden and management of XLMTM, and
demonstrates the devastating impact that an XLMTM diagnosis has on
the lives of patients and their families."
The RECENSUS study was the first step in the clinical
development program for AT132, the Audentes product candidate in
development for the treatment of XLMTM. Following RECENSUS,
Audentes initiated INCEPTUS, a clinical assessment and Phase 1/2
run-in study, in July 2016. The
primary objectives of INCEPTUS are to prospectively characterize
the disease course and natural history of children with XLMTM and
to assess the burden of disease on XLMTM patients and caregivers.
INCEPTUS also serves to identify subjects for potential
enrollment in ASPIRO, the Phase 1/2 clinical study of AT132, as
well as act as a longitudinal baseline and within-patient control
for ASPIRO. Preliminary data reported from INCEPTUS confirm
the significant neuromuscular and respiratory deficits experienced
by XLMTM patients, and provide insight into the relevance and
sensitivity of assessments used in ASPIRO, a Phase 1/2 clinical
study designed to assess the safety and preliminary efficacy of
AT132. Audentes initiated ASPIRO in September 2017 and plans to report preliminary
clinical data from the study in early January 2018.
The publication can be accessed at the following link:
http://onlinelibrary.wiley.com/doi/10.1002/mus.26018/full.
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 approximately 5 years
to date. Audentes is developing AT132 in collaboration with
Genethon (www.genethon.fr).
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 are currently conducting a Phase 1/2 clinical study of our lead
product candidate AT132 for the treatment of X-Linked Myotubular
Myopathy (XLMTM) and have three additional product candidates in
development, including 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 timing of preliminary
data from ASPIRO and the potential of INCEPTUS to serve as a
longitudinal baseline and within patient control for ASPIRO.
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, Investor Relations
415.818.1033
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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