Audentes Therapeutics Announces FDA Clearance of Investigational New Drug Application for AT132 to Treat X-Linked Myotubular Myopathy

SAN FRANCISCO, CA, USA I April 3, 2017 I Audentes Therapeutics, Inc. (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. 

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 (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.

SOURCE: Audentes Therapeutics

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