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Audentes Therapeutics Announces FDA Clearance of Investigational New Drug Application for AT132 to Treat X-Linked Myotubular Myopathy
- Preliminary data from ASPIRO, the planned Phase 1 / 2 Clinical Study of AT132, expected in fourth quarter of 2017

SAN FRANCISCO, April 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 the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for AT132, the Company's gene therapy product candidate to treat X-Linked Myotubular Myopathy (XLMTM). The IND is now active and Audentes plans to initiate ASPIRO, the multicenter, multinational, open-label, ascending dose Phase 1 / 2 clinical study of AT132. Preliminary data from ASPIRO is expected to be available in the fourth quarter of 2017.

Matthew R. Patterson, President and Chief Executive Officer, stated, "FDA clearance of the AT132 IND is an important milestone for Audentes that builds upon the momentum established with the recently cleared IND for AT342 to treat Crigler-Najjar Syndrome and the initiation of internal cGMP manufacturing for both AT342 and AT132." Mr. Patterson continued, "Based on the promising preclinical data set for the program, we believe AT132 has the potential to significantly improve the lives of patients living with this devastating disease. We look forward to working closely with the XLMTM medical community and patient organizations as we execute on our plans to enter the clinic."

In addition to ASPIRO, the clinical development program for AT132 includes RECENSUS, a retrospective medical chart review, for which Audentes recently announced data from an initial analysis of 112 male subjects. This analysis confirmed and expanded upon the understanding of the medical burden of XLMTM on patients, families and the healthcare system. Audentes is also conducting INCEPTUS, a clinical assessment and Phase 1 / 2 run-in study. The primary objectives of INCEPTUS are to characterize the disease course and natural history of children with XLMTM, assess the burden of disease on XLMTM patients and caregivers, identify subjects for potential enrollment in ASPIRO, and serve as a longitudinal baseline and within-patient control for ASPIRO.  As of April 1, 2017, 15 of the up to 16 subjects have been enrolled in INCEPTUS. Audentes plans to report preliminary data from INCEPTUS in mid-2017.

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% 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 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. Audentes is developing AT132 in collaboration with Genethon (

About ASPIRO, the Planned Phase 1 / 2 Clinical Study of AT132
ASPIRO is designed as a multicenter, multinational, open-label, ascending dose study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age. The study is expected to include nine AT132 treated subjects and three delayed-treatment concurrent control subjects. Primary endpoints include safety (adverse events and certain laboratory measures) and efficacy (assessments of neuromuscular and respiratory function). Secondary endpoints include the burden of disease and health related quality-of-life, and muscle tissue histology and biomarkers. The primary efficacy analysis is expected to be conducted at 12 months, with interim evaluations expected to be conducted at earlier time points. After the primary 12-month assessment, subjects are expected to be followed for another four years to assess long term safety, durability of effect and developmental progression.

About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing gene therapy products 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

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 ability to manufacture AT132 and AT342 in sufficient quantities to supply ASPIRO and VALENS, respectively; the ability to initiate ASPIRO and report preliminary data from ASPIRO by the fourth quarter of 2017; the potential of AT132 to significantly improve the lives of patients; and the ability to report preliminary data from INCEPTUS in mid-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

Media Contact:
Jeffrey Gruis


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SOURCE Audentes Therapeutics, Inc.