SAN FRANCISCO, Feb. 12, 2018 /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 it has commenced dosing of patients in VALENS, a Phase
1/2 clinical trial of its product candidate AT342 for the treatment
of Crigler-Najjar Syndrome. VALENS is a multicenter,
multinational, open-label, randomized, ascending dose study
designed to evaluate the safety and efficacy of AT342 in
approximately 12 Crigler-Najjar patients one year of age and
older. Audentes expects preliminary data from VALENS to be
available in the second quarter of 2018. In addition, FDA has
granted Rare Pediatric Disease and Fast Track designations for
AT342.
"Crigler-Najjar is a devastating rare disease characterized by
severe jaundice, extremely high levels of unconjugated bilirubin in
the blood, and the consequent risk of irreversible neurological
damage and death," stated Suyash
Prasad, M.D., Senior Vice President and Chief Medical
Officer. "There are no approved products to treat
Crigler-Najjar. The current standard of care requires most
patients to spend more than 10 to 12 hours per day under
phototherapy lights, and natural history data from our LUSTRO
run-in study demonstrate that even with this burdensome treatment,
bilirubin may only be reduced to levels just below those considered
to be neurotoxic."
Dr. Prasad continued, "We greatly appreciate the opportunity to
work with the Crigler-Najjar community and view the initiation of
VALENS as an important milestone in our development of AT342 for
this severe disease."
In addition to VALENS, the clinical development program for
AT342 includes LUSTRO, a prospective natural history run-in study
in Crigler-Najjar patients. The primary objectives of LUSTRO
are to characterize the disease course and natural history of
Crigler-Najjar, assess the burden of disease on patients and
caregivers, identify subjects for potential enrollment in VALENS,
and serve as a longitudinal baseline and within-patient control for
VALENS.
"We are also pleased to announce today that the FDA has granted
Rare Pediatric Disease and Fast Track designations for AT342,"
stated Matthew R. Patterson,
President and Chief Executive Officer. "These designations
provide significant benefits, including opportunities to work with
the FDA to expedite the development of AT342, 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 Crigler-Najjar patient community as
we advance the development of AT342."
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 and risk of irreversible neurological damage
and death. The current standard of care for Crigler-Najjar
Syndrome is daily, persistent phototherapy, usually for longer than
10 to 12 hours per day. Phototherapy wanes in effectiveness as
children age, and a liver transplant may be required for survival.
Data from LUSTRO, a prospective natural-history run-in study
in Crigler-Najjar patients, demonstrate that even with strict
adherence to a persistent daily phototherapy regimen, bilirubin may
only be reduced to levels just below those considered to be
neurotoxic. A single administration of AT342 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.
AT342 has been granted orphan drug designation in both
the United States and European
Union, and Rare Pediatric Disease and Fast Track designations by
the FDA.
About VALENS, the Phase 1/2 Clinical Study of AT342
VALENS is a multicenter, multinational, open-label, randomized,
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 subjects and three delayed-treatment concurrent control
subjects. 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). Subjects are expected
to remain on prescribed phototherapy for 12 weeks following
administration of AT342, and those with a prespecified decrease in
bilirubin at week 12 will be weaned off phototherapy over the
subsequent five-week period. Subjects are expected to be
followed for a minimum of five years to assess long term safety and
durability of effect.
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 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 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 timing of preliminary
data availability from VALENS, and the ability of LUSTRO to be used
as a longitudinal baseline and within patient control for
VALENS. 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
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.