April 29, 2017 10:38 PM ET

Biotechnology

Company Overview of Audentes Therapeutics, Inc.

Company Overview

Audentes Therapeutics, Inc., a biotechnology company, focuses on developing and commercializing gene therapy products for patients suffering from diseases caused by single gene defects in the United States. The company is developing AT132 for the treatment of X-linked myotubular myopathy; AT342 for the treatment of crigler-najjar syndrome type 1; AT982 for the treatment of pompe disease; and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. Audentes Therapeutics, Inc. has a collaboration with the University of Pennsylvania for the treatment of Crigler-Najjar. The company was founded in 2012 and is headquartered in San Francisco, California...

600 California Street

17th Floor

San Francisco, CA 94104

United States

Founded in 2012

97 Employees

Phone:

415-818-1001

Key Executives for Audentes Therapeutics, Inc.

Chief Executive Officer, President and Director
Age: 44
Total Annual Compensation: $441.8K
Chief Financial Officer and Senior Vice President
Age: 49
Total Annual Compensation: $360.5K
Chief Operating Officer and Senior Vice President
Age: 43
Total Annual Compensation: $360.5K
Chief Medical Officer, Senior Vice President and Member of Scientific & Clinical Advisory Board
Age: 46
Total Annual Compensation: $342.5K
Senior Vice President of Research & Development
Age: 53
Total Annual Compensation: $286.5K
Compensation as of Fiscal Year 2016.

Audentes Therapeutics, Inc. Key Developments

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

Audentes Therapeutics, Inc. 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. 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. 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 Therapeutics, Inc. Reports Unaudited Earnings Results for the Fourth Quarter and Full Year Ended December 31, 2016

Audentes Therapeutics, Inc. reported unaudited earnings results for the fourth quarter and full year ended December 31, 2016. For the quarter, the company reported loss from operations was $19,832,000 compared to $10,032,000 a year ago. Net loss was $19,676,000 or $0.91 per basic and diluted share compared to $9,959,000 or $4.97 per basic and diluted share a year ago. For the year, the company reported loss from operations was $60,046,000 compared to $26,726,000 a year ago. Net loss was $59,668,000 or $5.59 per basic and diluted share compared to $26,458,000 or $23.03 per basic and diluted share a year ago.

Audentes Therapeutics Announces Commencement of LUSTRO

Audentes Therapeutics, Inc. announced the commencement of LUSTRO, a prospective study designed to characterize the disease course in patients living with Crigler-Najjar Syndrome. LUSTRO will evaluate subjects prior to potential enrollment in VALENS, the planned Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT342, the Audentes product candidate being developed to treat Crigler-Najjar Syndrome. LUSTRO is an international study of approximately 16 to 18 Crigler-Najjar patients one year of age or older. The primary objectives of LUSTRO are to characterize the disease course, natural history, serum bilirubin variability and phototherapy usage of Crigler-Najjar patients. LUSTRO will also assess the burden of disease on Crigler-Najjar patients and caregivers. In addition, LUSTRO has been designed as a run-in study to identify patients for potential enrollment in VALENS, the planned Phase 1/2 study of AT342, and is intended to serve as a longitudinal baseline and within-patient control for subjects who enroll in VALENS. As LUSTRO will be conducted at the same sites and using the same assessment tools, LUSTRO will also facilitate certain operational aspects of VALENS.

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