SAN FRANCISCO, April 30, 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 data related to its product
candidates AT132 for the treatment of X-linked Myotubular Myopathy
(XLMTM), AT342 for the treatment of Crigler Najjar Syndrome, and
AT982 for the treatment of Pompe disease will be presented at the
21st Annual Meeting of the American Society of Gene and Cell
Therapy (ASGCT) to be held in Chicago,
IL on May 16-19th, 2018.
Six abstracts will be presented during the meeting, highlighting
the company's advances in the research, development and manufacture
of its portfolio of innovative AAV-based gene therapy products.
![Audentes Therapeutics, Inc. Audentes Therapeutics, Inc.](https://mma.prnewswire.com/media/391266/Audentes_Therapeutics___Logo.jpg)
"ASGCT offers an exciting opportunity for Audentes to share new
clinical and preclinical data from across our portfolio of gene
therapy product candidates," stated Matthew
R. Patterson, President and Chief Executive Officer.
"In particular, we are looking forward to providing the next
interim data update from the first dose cohort of patients in
ASPIRO, the Phase 1/2 clinical study of our product candidate AT132
to treat XLMTM. These presentations demonstrate our
commitment to advancing AAV gene therapy as a therapeutic modality,
and to rapidly developing new potential medicines for patients
living with severe, life-threatening rare diseases."
Clinical presentations:
ASIPRO Phase 1/2 Gene Therapy Trial in X-Linked Myotubular
Myopathy (XLMTM): Preliminary Safety and Efficacy Findings (Kuntz,
et al.)
Abstract number: 7
Session Title: Musculoskeletal Diseases I
Oral Presentation: Wednesday May 16, 2018 10:30am - 10:45am
Room: International Ballroom South
ASPIRO is a Phase 1/2 study to evaluate safety and preliminary
efficacy of AT132 in XLMTM patients less than five years of
age. Updated interim data on the first dose cohort
(1x1014 vector genomes/kg of AT132) will be presented,
including up to 24-week data for the first four subjects
enrolled.
The ASPIRO Trial for X-Linked Myotubular Myopathy:
Carefully Taking Systemic AAV Treatments to the Next Level
(Gray)
Session Title: New Directions and Clinical Trials for
Muscle and Skeletal Disorders
Symposium Presentation: Friday May 18,
2018 9:00am – 9:30am
Room: Salon A-1/2
An update on the ASPIRO clinical trial of AT132 in XLMTM
patients will be presented and related to previous clinical results
for systemic AAV gene therapies and new developments in vector
design and manufacturing.
Gene Therapy for Crigler-Najjar Syndrome with AT342, a
Liver-Targeted AAV8-UGT1A1 Vector – Preliminary Safety and Efficacy
Results from a Phase 1/2 Study (VALENS) (Prasad, et
al.)
Abstract number: 538
Session Title: Metabolic, Storage, Endocrine, Liver and
Gastrointestinal Diseases II
Poster Presentation: Thursday May 17, 2018 5:15pm - 7:15pm
Room: Stevens Salon C, D
VALENS is a Phase 1/2 study to evaluate the safety and
preliminary efficacy of AT342 in Crigler-Najjar patients greater
than one year of age. The preliminary safety and efficacy
profile of AT342 dosed at 1.5x1012 vg/kg will be
presented.
Non-clinical presentations:
A Novel Hybrid Promoter Directing AAV-Mediated Expression of
Acid Alpha-Glucosidase to Liver, Muscle and CNS Yields Optimized
Outcomes in a Mouse Model of Pompe Disease (Heffner, et
al.)
Abstract number: 236
Session Title: Metabolic, Storage, Endocrine, Liver and
Gastrointestinal Diseases I
Poster Presentation: Wednesday May 16,
2018 5:30pm - 7:30pm
Room: Stevens Salon C, D
AT982 is an AAV8 vector which utilizes a novel hybrid promoter
designed to provide tissue-specific neuromuscular transgene
expression and enhance immune tolerance to hGAA.
Bioanalytical data from multiple tissue types support the clinical
translation of AT982 as an optimized vector for the treatment of
Pompe disease.
Rescue of Genetic Cardiac Arrhythmias by AAV Delivery of the
Cardiac Calsequestrin Gene (Newman, et al.)
Abstract number:
147
Session Title: Cardiovascular and Pulmonary Diseases I
Poster Presentation: Wednesday May 16,
2018 5:30pm - 7:30pm
Room: Stevens Salon C, D
AT307 is an AAV8 vector which expresses a codon-optimized
version of the human CASQ2 coding sequence driven by the desmin
promoter. Data in a mouse model of CASQ2-CPVT demonstrate
robust, dose-dependent correction of this inherited cardiac
arrhythmia.
Comparison of Intracoronary versus Peripheral Vein Delivery
of AAV for Gene Therapy in a Pig Model (Lawn, et
al.)
Abstract number: 766
Session Title: Cardiovascular and Pulmonary Diseases II
Poster Presentation: Friday May 18,
2018 5:45pm - 7:45pm
Room: Stevens Salon C, D
Gene therapy is being investigated as a treatment option for
inherited cardiac mutations that lead to sudden cardiac death in
children and young adults, but requires efficient cardiac delivery
of transgenes. Data support the utility of systemic delivery
of AAV to address myocardial diseases.
Formulation Optimization for a Gene Therapy Parenteral
Product (Haque, et al.)
Abstract number: 712
Session Title: AAV Vectors III
Poster Presentation: Friday May 18,
2018 5:45pm - 7:45pm
Room: Stevens Salon C, D
Results detail experiments undertaken to optimize the
formulation for AT132 to support long-term stability.
Sponsorship:
In addition to allowing Audentes to
highlight its substantial accomplishments over the past year, ASGCT
provides an opportunity to celebrate the work of early pioneers who
have made meaningful contributions to advancing gene therapy
science. At this year's ASGCT Audentes is pleased to sponsor
the Outstanding Achievement Award Lecture, which will feature a
presentation by Jean Bennett MD,
PhD, University of Pennsylvania Scheie
Eye Institute, titled, Seeing the Light with Retinal Gene Therapy:
From Fantasy to Reality.
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 January 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 orphan drug designation in both
the United States and European
Union, and Rare Pediatric Disease and Fast Track designations by
the FDA.
About AT342 for Crigler-Najjar Syndrome
AT342 is the
Audentes product candidate being developed to treat Crigler-Najjar
Syndrome, a rare monogenic disease characterized by severely high
levels of unconjugated bilirubin that can cross the blood brain
barrier and result in irreversible neurological damage and
death. Crigler-Najjar is caused by mutations in the gene
encoding the UGT1A1 enzyme, which converts unconjugated bilirubin
to a water-soluble form that can be excreted from the body.
AT342 is comprised of an AAV8 vector containing a functional
copy of the UGT1A1 gene. 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. In February 2018, Audentes announced that it dosed
the first patient in VALENS, a multicenter, ascending dose Phase
1/2 clinical study to evaluate the safety and preliminary efficacy
of AT342 in approximately 12 Crigler-Najjar patients greater than
one year of age.
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 AT982 for Pompe disease
AT982 is the Audentes
product candidate being developed to treat Pompe disease, a
serious, progressive genetic disease characterized by severe muscle
weakness, respiratory failure leading to ventilator dependence and,
in infants, increased cardiac mass and heart failure. In untreated
infants, the disease is often fatal due to cardio-respiratory
failure within the first year of life, and in adults the disease is
progressive and life-limiting with significant ventilator and
wheelchair use. Pompe disease is caused by mutations in the gene
encoding the lysosomal enzyme alpha-glucosidase, or GAA, which
results in a deficiency of GAA protein and leads to the
accumulation of glycogen. The incidence of Pompe disease is
approximately one in 40,000 births. AT982 consists of an AAV8
vector that delivers a GAA gene expression cassette containing a
novel hybrid promoter designed to increase GAA activity in targeted
tissues, including skeletal and cardiac muscle, the nervous system
and the liver. Audentes holds exclusive global rights to both
AAV8 and AAV9 in Pompe disease from REGENXBIO.
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 timing and nature of clinical
development activities and the expected benefits 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, 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:
Paul Laland
415.519.6610
plaland@audentestx.com
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