SAN FRANCISCO, June 5, 2018 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing innovative gene therapy
products for patients living with serious, life-threatening rare
diseases, today announced that the European Medicines Agency (EMA)
has granted Priority Medicines (PRIME) designation to AT132 for the
treatment of X-Linked Myotubular Myopathy (XLMTM). The PRIME
designation is intended to enhance interaction and early dialogue
with developers of promising therapies to optimize development
plans and speed evaluation so that these medicines may reach
patients as early as possible.
"We are pleased that AT132 has been accepted into the PRIME
program, which is analogous to the Breakthrough Therapy and
Regenerative Medicine and Advanced Therapy designations from the
U.S. Food and Drug Administration," stated Mary S. Newman, Senior Vice President,
Regulatory Affairs. "This PRIME designation has been enabled
by the positive interim data reported from the first dose cohort of
ASPIRO, the Phase 1/2 clinical trial of AT132, and we look forward
to collaborating with the EMA to accelerate the clinical
development of this potentially transformative therapy for XLMTM
patients."
About PRIME Designation
The PRIME program was launched
by the EMA in March 2016, and the
designation is designed to aid and expedite the regulatory process
for investigational medicines that may offer a major therapeutic
advantage over existing treatments, or benefit patients without
treatment options. The PRIME designation provides appointment
of a rapporteur, early dialogue and scientific advice at key
development milestones, and the potential to qualify products for
accelerated review earlier in the application process.
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 the protein
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. In May 2018, Audentes
reported positive interim data from the first dose cohort of
ASPIRO, a multicenter, ascending dose Phase 1/2 clinical
study to evaluate the safety and preliminary efficacy of AT132 in
approximately 12 XLMTM patients less than five years of age. The
preclinical development of AT132 was conducted in collaboration
with Genethon (www.genethon.fr).
AT132 has been granted PRIME and Orphan Drug designations by the
EMA, and Rare Pediatric Disease, Fast Track and Orphan Drug
designations by the FDA.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a biotechnology company focused
on developing and commercializing innovative gene therapy products
for patients living with serious, life-threatening rare
diseases. We are currently conducting
Phase 1/2 clinical studies of our lead product candidates
AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and
AT342 for the treatment of Crigler-Najjar Syndrome. We have
two additional product candidates in development, including 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 of the company to work
with the EMA to accelerate the clinical development and potential
approval 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:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contact:
Paul Laland
415.519.6610
plaland@audentestx.com
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SOURCE Audentes Therapeutics, Inc.