Audentes Therapeutics Announces Presentation of Data at the American Society of Gene and Cell Therapy Annual Meeting

SAN FRANCISCO, May 3, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing 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, and AT342 for the treatment of Crigler Najjar Syndrome, will be presented at the 20th Annual Meeting of the American Society of Gene and Cell Therapy to be held in Washington, DC on May 10-13^th, 2017.  Four abstracts will be presented during the meeting, underscoring the company's leadership position and significant advances in the research, development and manufacture of highly innovative AAV-based gene therapy products.

John T. Gray, Ph.D, Senior Vice President, Research and Development, commented, "We are excited by the opportunity to present important data that support the advancement of our product candidates to treat XLMTM and Crigler-Najjar Syndrome.  We plan to initiate Phase 1/2 clinical studies of both in mid-2017, and to report preliminary results from each program by year-end."

Details of the presentations are provided below:

Abstract number: 216
Title: Phillips, et. al., Nonhuman Primate Safety and Potency of an AAV Vector for XLMTM Produced by Transient Transfection at 500L
Session title: Musculo-skeletal Diseases I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30 PM
Room: Exhibit Hall A & B South

Summary: Administration of AT132 produced at 500L scale demonstrated an encouraging safety profile and high potency, providing support for its advancement into clinical testing for XLMTM patients.  Specifically, an intravenous dose of 8 x 10¹⁴ vg/kg in NHPs resulted in no significant treatment related adverse events or safety findings and greater than normal levels of myotubularin protein expression in skeletal muscle.  

Abstract number: 674
Title: Greig, et. al., Evaluation of Efficacy and Safety in a Dose-Escalating Nonclinical Study of a Clinical Candidate Vector in a Mouse Model of Crigler-Najjar
Session title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases
Session Date/Time: Friday, May 12, 5:45 – 7:45 PM  
Room: Exhibit Hall A & B South

Summary: Administration of AT342 resulted in no significant treatment-related adverse events or safety findings and rapidly reduced serum bilirubin levels in the mouse model of Crigler-Najjar syndrome. 

Abstract number: 212
Title: DuPont, et. al., RNA-Seq Analysis of Canine X-Linked Myotubular Myopathy Muscles Before and After Gene Therapy
Session title: Musculo-skeletal Diseases I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30 PM 
Room: Exhibit Hall A & B South

Summary: Bioinformatic comparison of RNA-Seq data from diseased and gene therapy treated muscle reveals gene expression patterns that correlate with therapeutic correction of X-Linked Myotubular Myopathy in dogs.

Abstract number: 103
Title: Guo, et. al., RNA-Seq Tag Counting: An Approach for Quantifying the Relative Expression of Vector Derived and Endogenous Genes in rAAV-Treated Non-Clinical Models
Session title: AAV Vectors I
Session Date/Time: Wednesday, May 10, 5:30 PM – 7:30 PM
Room: Exhibit Hall A & B South

Summary: A novel bioinformatic method called 'RNA-Seq Tag Counting' processes RNA-Seq data to generate quantitative gene expression values with robust linearity and reproducibility.

About AT132 to Treat X-Linked Mytubular 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 a protein called 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.  Multiple studies in animal models of XLMTM have demonstrated that a single administration of AT132 was well tolerated and significantly improved disease symptoms and survival rates, with no significant AT132-related adverse events or safety findings.  In one study these effects have lasted more than four years to date. The U.S. Food and Drug Administration has cleared the IND for AT132, and Audentes plans to initiate ASPIRO, the Phase 1/2 study of AT132 in mid-2017.  Preliminary data from ASPIRO is expected to be available by the end of 2017.

About AT342 to Treat Crigler-Najjar Syndrome   
AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene for the treatment of Crigler-Najjar Syndrome, a rare monogenic disease characterized by severely high levels of unconjugated bilirubin in the blood and risk of irreversible neurological damage and death.  The current standard of care for Crigler-Najjar Syndrome is persistent phototherapy, usually for longer than 12 hours per day. Phototherapy wanes in effectiveness beginning around age four, and a liver transplant may be required for survival.  A single administration of AT342 has generated durable, dose-responsive and clinically-relevant decreases in total bilirubin levels in a mouse model of Crigler-Najjar, with no significant AT342-related adverse events or safety findings.  The U.S. Food and Drug Administration has cleared the IND for AT342, and Audentes plans to initiate VALENS, the Phase 1/2 study of AT342 in mid-2017.  Preliminary data from VALENS is expected to be available by the end of 2017.

About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing gene therapy product candidates for patients living with serious, life-threatening rare diseases.  We have four product candidates in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, 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: whether Audentes has or will maintain a leadership position in the research, development and manufacture of highly innovative AAV-based gene therapy products; and, whether Audentes will initiate Phase 1/2 studies in its XLMTM and Crigler-Najjar programs in mid-2017, or report preliminary data from such studies by the end of 2017.  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:
Thomas Soloway, CFO
415.818.1040
[email protected]

Media Contact:
Paul Laland
415.519.6610
[email protected]

SOURCE Audentes Therapeutics, Inc.

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