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Audentes Therapeutics Announces First Patient Enrolled in the INCEPTUS Clinical Assessment Study of XLMTM

Study intended to serve as longitudinal baseline and within-patient control for ASPIRO, the planned Phase 1 / 2 study of AT132 for the treatment of XLMTM

SAN FRANCISCO, Aug. 17, 2016 /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 reported that the first patient has been enrolled in INCEPTUS, a prospective study designed to characterize the disease presentation in children living with X-Linked Myotubular Myopathy (XLMTM). The study is evaluating subjects prior to potential enrollment in ASPIRO, the planned Phase 1 / 2 clinical study intended to evaluate the safety and preliminary efficacy of AT132, the Audentes product candidate for treatment of XLMTM.

"The initiation of enrollment for INCEPTUS is an important first step in the clinical development of AT132 for the treatment of XLMTM and a notable milestone for Audentes," stated Dr. Suyash Prasad, Audentes Senior Vice President and Chief Medical Officer. "The burden of the disease of XLMTM to children and families is considerable, and we are grateful to those who have agreed to participate in the study," concluded Dr. Prasad.

INCEPTUS is an international study of boys with XLMTM, ages three years or younger, designed to characterize their individual disease presentation, with a specific focus on respiratory measurements and assessment of muscle strength and function. Patients enrolled in INCEPTUS will be evaluated over a three to twelve-month period prior to their potential participation in ASPIRO. INCEPTUS is designed to serve as a longitudinal baseline and within-patient control for subjects who enroll in the ASPIRO study, while also facilitating the operational aspects of ASPIRO once initiated.  

Audentes plans to file investigational new drug applications for AT132 with North American and European regulatory authorities in the first quarter of 2017. The company expects preliminary data from the ASPIRO study in the fourth quarter of 2017.

For additional information on INCEPTUS, please visit

About X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness, respiratory impairment and early mortality. There is no approved treatment for the condition. It 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. XLMTM affects approximately 1 in 50,000 newborn males worldwide. Audentes is developing AT132 for the treatment of XLMTM in collaboration with Genethon ( AT132 is a novel product based on AAV gene therapy technology.

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 products 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, statements regarding the company's anticipated clinical development and regulatory activities and the timing of clinical results. 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

Audentes Therapeutics, Inc.

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SOURCE Audentes Therapeutics, Inc.