SAN FRANCISCO, Sept. 27, 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
granted Rare Pediatric Disease and Fast Track designations for
AT132, the company's gene therapy product candidate being developed
to treat X-Linked Myotubular Myopathy (XLMTM). In addition to
these two new designations, AT132 has also received Orphan Drug
designation from both the FDA and European Medicines Agency.
Matthew R. Patterson, President
and Chief Executive Officer of Audentes, stated, "The incentives
provided by the Rare Pediatric Disease and Fast Track designations
are significant and include opportunities to work with FDA to
expedite the development of AT132, and the potential to obtain a
valuable Rare Pediatric Disease Priority Review Voucher upon
approval. We are eager to leverage these benefits and to work
closely with the FDA and XLMTM patient community as we pursue our
goal of developing AT132 to treat this devastating rare
disease."
Audentes recently announced the enrollment of the first patient
in ASPIRO, the Phase 1/2 clinical trial of AT132 in patients with
XLMTM. Preliminary data from ASPIRO is expected to be
available in the fourth quarter of 2017. 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 and 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 longitudinal
baseline and within-patient control for ASPIRO.
About the Rare Pediatric Disease Priority Review Voucher and
Fast Track Programs
A Rare Pediatric Disease designation
may be granted by the FDA to drugs and biologics intended to treat
orphan diseases affecting fewer than 200,000 patients in
the United States, primarily age
18 years or younger. The designation provides incentives to advance
the development of rare disease drugs, including access to the
FDA's expedited review and approval programs. In addition, under
the FDA's Rare Pediatric Disease Priority Review Voucher program, a
sponsor that receives approval for a biologics license application
for a rare pediatric disease may be eligible to receive a
voucher for a priority review of a subsequent marketing
application for a different product. The priority review voucher
may be used by the sponsor, sold or transferred.
The Fast Track program was created by the FDA to facilitate the
development and expedite the review of new drugs which show promise
in treating a serious or life-threatening disease and address an
unmet medical need. Drugs that receive this designation benefit
from more frequent communications and meetings with FDA to review
the drug's development plan including the design of the proposed
clinical trials, use of biomarkers and the extent of data needed
for approval. Drugs with Fast Track Designation may qualify for
priority review to expedite the FDA review process, if relevant
criteria are met.
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 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: whether Audentes will eventually
receive approval of AT132, whether it will receive a Rare Pediatric
Disease Voucher, whether such voucher will have value in the form
of expediting the review of other Audentes products or have value
to other companies seeking such a voucher, and whether benefits
afforded by the FDA's Fast Track designation will help facilitate
the development and expedited review of AT132. 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.