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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