SAN FRANCISCO, Aug. 21, 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 U.S. Food and Drug
Administration (FDA) granted Regenerative Medicine Advanced Therapy
(RMAT) designation to AT132 for the treatment of X-linked
Myotubular Myopathy (XLMTM).
"We are pleased that the FDA has granted RMAT designation to
AT132, an important regulatory milestone that highlights the
transformative potential of AT132 as a therapy to treat XLMTM, a
rare, congenital disease characterized by extreme muscle weakness,
respiratory failure and early death," stated Mary S. Newman, Senior Vice President,
Regulatory Affairs at Audentes. "This news follows our recent
announcement of the Priority Medicines, or PRIME, designation
received from the European Medicines Agency, and we look forward to
collaborating closely with the FDA, EMA, our XLMTM clinical experts
and the patient community as we seek to rapidly advance the AT132
development program toward global regulatory approvals."
Established under the 21st Century Cures Act, RMAT designation
is a program designed to expedite the development and approval of
regenerative medicine products, including gene therapy products.
An investigational therapy is eligible for the RMAT
designation if it is intended to treat, modify, reverse or cure a
serious or life-threatening disease or condition, and preliminary
clinical evidence indicates a potential to address unmet medical
needs for that disease or condition. The designation includes
all the benefits of the FDA's Fast Track and Breakthrough Therapy
designations, and enables the ability to work more closely and
frequently with the FDA to discuss surrogate or intermediate
endpoints to support the potential acceleration of approval and
satisfy post-approval requirements.
RMAT designation for AT132 was granted based on positive interim
clinical data from ASPIRO, the ongoing Phase 1/2 clinical study of
AT132 for XLMTM. These data show encouraging initial evidence
of safety and efficacy in the first dose cohort, including
significant improvements in neuromuscular function as assessed by
the CHOP-INTEND scale, increased respiratory function as
demonstrated by reductions in ventilator dependence and gains in
maximal inspiratory pressure (MIP), a measure of respiratory muscle
strength. In addition to these functional outcome measures,
muscle biopsy results from the first three patients treated in the
study at the 24-week timepoint demonstrate highly efficient tissue
transduction as indicated by vector copy number, robust
myotubularin protein expression as assessed by western blot, and
significant improvement in histology.
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 percent
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 August 2018, Audentes reported promising safety,
efficacy and muscle biopsy data out to the 24-week timepoint 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 Regenerative Medicine Advanced Therapy
(RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug
designations by the FDA, and Priority Medicines (PRIME) and
Orphan Drug designations by the European Medicines Agency
(EMA).
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 transformative potential of
AT132 to treat XLMTM, and the ability of the company to work with
regulatory agencies to rapidly advance the 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:
Katie Hogan
415.951.3398
khogan@audentestx.com
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SOURCE Audentes Therapeutics, Inc.