SAN FRANCISCO, Sept. 24, 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 new interim data from ASPIRO, the
Phase 1/2 clinical trial of AT132 in patients with X-linked
Myotubular Myopathy (XLMTM), will be presented at the 23rd
International Congress of the World Muscle Society (WMS) in
Mendoza, Argentina. The oral presentation will be given by
Nancy Kuntz, MD, Principal
Investigator at Lurie Children's Hospital of Chicago, Medical Director of Mazza Foundation
Neuromuscular Program and Professor of Pediatrics and Neurology at
Northwestern University Feinberg School
of Medicine, and will be held during a session on new therapeutic
modalities scheduled to begin at 9:30 a.m.
ET on October 5, 2018.
In addition to the oral presentation, Audentes plans to share five
poster presentations covering its XLMTM and Pompe disease
programs.
The presentation by Dr. Kuntz will include new interim data from
ASPIRO out to 48-weeks of follow-up in the earliest treated
patients in dose Cohort 1 (1x1014 vg/kg), and
preliminary safety and efficacy data for the sentinel patient
treated in dose Cohort 2 (3x1014 vg/kg).
"XLMTM is a severe, deadly disease with no approved treatment
options," said Matthew R. Patterson,
Chief Executive Officer. "We remain highly encouraged by the
emerging clinical profile of AT132 and are excited to share our
progress in ASPIRO at this important medical meeting."
Complete results from these studies will be announced during WMS
Congress in accordance with the meeting embargo policy.
Following are details for each presentation (all times are in
Argentina Time (ART)):
Oral presentation:
ASPIRO Phase 1/2 Gene Therapy Trial In X-Linked Myotubular
Myopathy (XLMTM): Preliminary Safety and Efficacy
Findings
Abstract number: O.17
Session Title: Selected Oral Presentations III – New
Therapeutic Approaches and Their Readout
Date: Friday, October 5, 2018, 10:30
am – 12:30 pm ART
Poster presentations:
INCEPTUS Pre-Phase 1, Prospective, Non-Interventional,
Natural History Run-in Study to Evaluate Subjects Aged 3 Years and
Younger with X-Linked Myotubular Myopathy (XLMTM): Preliminary
Findings
Abstract Number: P.136
Session Title: Congenital Myopathies (CNM)
Date: Wednesday, October 3, 2018,
4:00 pm – 5:30
pm ART
The CHOP-INTEND scale (Children's Hospital of Philadelphia
Infant Test of Neuromuscular Disorders) in X-linked Myotubular
Myopathy (XLMTM): Content Validity and Psychometric
Performance
Abstract Number: P.138
Session Title: Congenital Myopathies (CNM)
Date: Wednesday, October 3, 2018,
4:00 pm – 5:30
pm ART
Mortality and Respiratory Support in X-Linked Myotubular
Myopathy (XLMTM): The RECENSUS Study, an International,
Multicenter, Retrospective Medical Record Review of XLMTM
Abstract Number: P.139
Session Title: Congenital Myopathies (CNM)
Date: Wednesday, October 3, 2018,
4:00 pm – 5:30
pm ART
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
Abstract
Number: P.350
Session Title: Metabolic Myopathies II
Date: Friday, October 5, 2018,
5:00 pm – 6:30
pm ART
Exploring Study Design and Endpoint Selection to Evaluate
Safety, Preliminary Efficacy, and Dose Selection of AAV8 Gene
Therapy in Patients with Infantile and Late Onset Pompe Disease
(IOPD and LOPD)
Abstract Number: P.351
Session Title: Metabolic Myopathies II
Date: Friday, October 5, 2018,
5:00 pm – 6:30
pm ART
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 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 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 potential benefits 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.