Press Release

Audentes Therapeutics to Present New Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy, at the 23rd International Congress of the World Muscle Society
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
Complete results from these studies will be announced during
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,
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:
The CHOP-INTEND scale (
Abstract Number: P.138
Session Title: Congenital Myopathies (CNM)
Date:
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:
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:
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:
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
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
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.
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
Audentes Contacts:
Investor Contact:
415.818.1033
achang@audentestx.com
Media Contact:
415.951.3398
khogan@audentestx.com
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