SAN FRANCISCO, Nov. 13, 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 Medicines and Healthcare Products Regulatory
Agency (MHRA) has approved the Clinical Trial Authorisation (CTA)
application for AT342, the Company's gene therapy product candidate
being developed to treat Crigler-Najjar Syndrome. This is the
first European CTA approval for the AT342 program, and it allows
Audentes to initiate the enrollment process at clinical sites in
the UK for VALENS, the Phase 1/2 clinical study of
AT342.
"This CTA approval represents an important milestone for our
AT342 program," stated Matthew R.
Patterson, President and Chief Executive Officer. "We
are pleased to be working closely with the European Crigler-Najjar
Syndrome community as we continue to execute on our global plans to
develop AT342 as a potentially transformative product to treat this
devastating rare disease."
In addition to VALENS, the clinical development program for
AT342 includes LUSTRO, a prospective natural history run-in study.
The primary objectives of LUSTRO are to characterize the
clinical condition of patients with Crigler-Najjar Syndrome,
identify subjects for potential enrollment in VALENS, and serve as
a longitudinal baseline and within-patient control for VALENS.
About VALENS, the Phase 1/2 Clinical Study of
AT342
VALENS is a multicenter, multinational, open-label,
ascending dose study to evaluate the safety and preliminary
efficacy of AT342 in approximately 12 Crigler-Najjar patients one
year of age and older. The study is expected to include nine AT342
treated patients and three delayed-treatment concurrent control
patients. Primary endpoints include safety (adverse events and
certain laboratory measures, including immunological parameters)
and efficacy (changes in serum bilirubin and number of hours on
phototherapy within a 24-hour period). Patients are expected
to remain on prescribed phototherapy for 12 weeks following
administration of AT342, and those with a meaningful decrease in
bilirubin at week 12 will be weaned off phototherapy over the
subsequent five-week period. Patients are expected to be
followed for a minimum of five years to assess long term safety and
durability of effect.
About AT342 for Crigler-Najjar Syndrome
AT342 is an
AAV8 vector containing a functional copy of the UGT1A1 gene for the
treatment of Crigler-Najjar Syndrome, a rare monogenic disease
characterized by severely high levels of unconjugated bilirubin in
the blood which present a significant risk of irreversible
neurological damage and death. The current standard of care
for Crigler-Najjar Syndrome is daily, persistent phototherapy,
usually for longer than 12 hours per day. Phototherapy wanes in
effectiveness as children age, and a liver transplant may be
required for survival. A single administration of AT342 has
generated durable, dose-responsive and clinically-relevant
decreases in total bilirubin levels in a mouse model of
Crigler-Najjar, with no significant AT342-related adverse events or
safety findings.
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 ability of LUSTRO to be used as
a longitudinal baseline and within patient control for VALENS and
the potential of AT342 to be a transformative therapy for
Crigler-Najjar patients. 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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