SAN FRANCISCO, Jan. 31, 2019 /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 provided an update following the receipt of minutes
from its Regenerative Medicine and Advanced Therapy (RMAT)/Type B
meeting with the U.S. Food and Drug Administration (FDA) held in
December 2018, regarding the
company's lead gene therapy candidate, AT132, for the treatment of
X-linked Myotubular Myopathy (XLMTM).
The goal of the meeting was to review nonclinical, clinical, and
chemistry, manufacturing, and controls (CMC) data generated to date
in the AT132 program, and to align with the FDA on program
next-steps to enable submission of a Biologics License Application
(BLA) for AT132.
"We're pleased that our first discussion with the FDA under RMAT
was both collaborative and productive, and provided a potential
path toward BLA submission for AT132," stated Matthew R. Patterson, Chairman and Chief
Executive Officer. "We appreciate the constructive feedback
provided by the Agency and look forward to working with the FDA to
advance AT132 to registration as quickly as possible."
Following the meeting, Audentes is proceeding with its
previously announced plan to enroll an additional 3-5 patients in
Cohort 2 (3x1014 vector genomes per kilogram dose level)
of ASPIRO, the Phase 1/2 study of AT132. Optimal dose
selection is expected to occur in the second quarter of 2019, after
the evaluation of the six-month biopsy results from the first three
patients dosed in Cohort 2. Subsequent to the determination
of the optimal dose, Audentes plans to provide an updated data
package to FDA to facilitate final agreement on the path to BLA
submission. Audentes also received preliminary feedback on
its CMC characterization and validation plans and will submit the
requested information to the Investigational New Drug (IND)
application on a continuing basis.
In pursuit of global regulatory approvals for AT132, Audentes
also initiated discussions with the European Medicines Agency (EMA)
in the fourth quarter of 2018 under the recently granted Priority
Medicines (PRIME) designation. In the first quarter of 2019,
Audentes anticipates receiving scientific advice from the EMA that
will begin to define the path toward submission of a European
Marketing Authorization Application.
About AT132 for X-linked Myotubular Myopathy
AT132 is
the Audentes product candidate being developed to treat XLMTM, a
disease 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. Over the course of 2018,
Audentes reported promising safety, efficacy and muscle biopsy data
from ASPIRO, a multicenter, ascending dose
Phase 1/2 clinical study designed to evaluate the safety
and 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 RMAT, Rare Pediatric Disease, Fast Track
and Orphan Drug designations by the FDA, and PRIME and Orphan
Drug designations by the 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 XLMTM, and AT342 for
the treatment of Crigler-Najjar syndrome. We have two
additional product candidates in development, AT845 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 and nature of clinical
development activities; the timing of regulatory interactions,
submissions or approvals; and, the expected safety and efficacy
profile of the company's product candidates. 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 commercialize its
product candidates, the timing and results of preclinical and
clinical trials, the company's ability to fund development
activities and achieve development goals, establish and scale-up
manufacturing processes that comply with regulatory requirements,
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.