SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, today announced its planned presentations at the 24th International Annual Congress of the World Muscle Society in Copenhagen, Denmark, including new data from ASPIRO, the clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy. The oral presentation will be given by Dr. James J. Dowling, Hospital for Sick Children, Toronto, Canada, and will be held during the Clinical Trial Highlights 7 session of the conference on Saturday, October 5 beginning at 1:00pm Central European Summer Time (CEST)/7:00am Eastern Time (ET).
Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls). Efficacy analyses will include assessments of ventilator dependence and achievement of developmental motor milestones.
“We are excited to share new efficacy and safety data from our ASPIRO study,” stated Natalie Holles, President and Chief Operating Officer. “The data from these dose escalation cohorts, along with results from our ongoing pivotal expansion cohort, will form the basis of the AT132 BLA submission planned in mid-2020, and MAA submission planned for the second half of 2020.”
Audentes is planning several additional presentations during the conference, including a company-sponsored symposium that will provide an in-depth review of XLMTM and the ASPIRO study results. Following are details for each presentation:
ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings
Session: Clinical Trial Highlights 7
Date and time: Saturday, October 5, 1:00-2:00pm Central European Summer Time (CEST)/7:00am-8:00am Eastern Time (ET)
Abstract number: O.39
INCEPTUS Pre-Phase 1, Prospective, Non-Interventional, Natural History Run-in Study to Evaluate Subjects Aged 4 Years and Younger with X-Linked Myotubular Myopathy (XLMTM)
Session: Congenital myopathies: centronuclear and others
Date and time: October 2, 2019, 4:45pm - 6:45pm Central European Summer Time (CEST)/10:45am – 12:45pm Eastern Time (ET)
Abstract number: P.105
Altering the Treatment Paradigm: Gene Therapy for Neuromuscular Disorders
Date and time: Friday, October 4, 1:15pm–2:45pm Central European Summer Time (CEST)/7:15am – 8:45am (ET)
Location: Axelborg Hall
Agenda and Speakers:
AAV Gene Therapy for Neuromuscular Disorders
Benedikt Schoser, MD (Chair)
Friedrich-Baur Institute, Ludwig-Maximilians University, Munich, Germany
Gene Therapy for XLMTM: The ASPIRO Study
The Potential of AAV for Neuromuscular Disorders
Edward Conner, MD
Senior Vice President and Chief Medical Officer, Audentes Therapeutics, San Francisco, CA, USA
Improvements in XLMTM Muscle Pathology and Biomarkers
Michael W Lawlor, MD, PhD
Department of Pathology and Laboratory Medicine and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA
Achieving Ventilator Independence with AT132 in XLMTM
Robert Graham, MD
Division of Critical Care Medicine, Boston Children’s Hospital, Boston, MA, USA
Attaining Motor Developmental Milestones in Children with XLMTM
Laurent Servais, MD, PhD
Muscular Dystrophy UK Oxford Neuromuscular Centre, Oxford, UK and Liège University, Liège, Belgium
About X-linked Myotubular Myopathy
X-linked Myotubular Myopathy (XLMTM) is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure, and early death. Mortality rates are estimated to be 50 percent in the first 18 months of life, and for those patients who survive past infancy, there is an estimated additional 25% mortality by the age of 10. XLMTM is caused by mutations in the MTM1 gene that lead to a lack or dysfunction of myotubularin, a protein that is needed for normal development, maturation, and function of skeletal muscle cells. The disease affects approximately 1 in 40,000 to 50,0000 newborn males.
XLMTM places a substantial burden of care on patients, families and the healthcare system, including high rates of healthcare utilization, hospitalization and surgical intervention. More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support. In most patients, normal developmental motor milestones are delayed or never achieved. Currently, only supportive treatment options, such as ventilator use or a feeding tube, are available.
About AT132 for the treatment of XLMTM
Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM). AT132 may provide patients with significantly improved outcomes based on the ability of AAV8 to target skeletal muscle and increase myotubularin expression in targeted tissues following a single intravenous administration.
Audentes has reported promising safety, efficacy, and muscle biopsy data from ASPIRO, an ongoing, multicenter, ascending dose clinical study designed to evaluate the safety and efficacy of AT132. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).
AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).
About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases. We are leveraging our AAV gene therapy technology platform and proprietary manufacturing expertise to develop programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown. Our product candidates are showing promising therapeutic profiles in clinical and preclinical studies across a range of neuromuscular diseases. Audentes is a focused, experienced and passionate team driven by the goal of improving the lives of patients.
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 the ASPIRO pivotal expansion and the ASPIRO clinical data results and the timing and nature of regulatory filings for 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 and 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, 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.