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Audentes Therapeutics Provides Full Year 2017 Strategic Outlook and Financial Guidance
- Filed IND with the FDA for AT342 in Crigler-Najjar Syndrome
- Large Scale Internal cGMP Manufacturing Capability Expected to be Established for Entire Pipeline of Four Programs
- Preliminary Clinical Data from Lead Programs Expected by Year-End

SAN FRANCISCO, Jan. 9, 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 provided its full-year 2017 strategic outlook and financial guidance.

"2016 was a year marked by the achievement of significant milestones for Audentes," stated Matthew R. Patterson, President and Chief Executive Officer.  "We completed preclinical development for three programs, filed an IND for our Crigler-Najjar program, commenced key clinical activities for our XLMTM program, established large scale cGMP manufacturing at our internal manufacturing facility, and completed a successful IPO. We believe these accomplishments provide a strong foundation for a promising 2017."

Mr. Patterson continued, "As we look forward to the year ahead, we are excited by the prospect of initiating clinical trials in multiple programs.  We expect to obtain preliminary data from our lead programs by year end, highlighted by the ASPIRO phase 1 / 2 study for the treatment of XLMTM and the VALENS phase 1 / 2 study for Crigler-Najjar.  Additionally, we plan to establish internal cGMP manufacturing for our Pompe disease and CPVT programs.  By hitting these milestones, we will make meaningful progress toward achieving our goal of providing transformative gene therapy products for the patients we serve."

Mr. Patterson will provide a corporate update and 2017 outlook in a presentation at the 35th Annual JP Morgan Healthcare Conference on January 10, 2017 at 5:00 pm PST.  To access the live webcast of the presentation, please visit the Events & Presentations page within the Investors & News section of the Audentes website. Replays of the live webcast will be available on the Audentes website for approximately 30 days following the conference.

Key 2016 Accomplishments

  • Filed IND with FDA for AT342 in Crigler-Najjar Syndrome:  Represents the first IND filed by Audentes for an AAV gene therapy product.
  • Initiated large-scale cGMP production at state-of-the-art internal gene therapy manufacturing facility: The company intends to initiate clinical trials for two of its products, AT132 for X-Linked Myotubular Myopathy (XLMTM) and AT342 for Crigler-Najjar Syndrome, in 2017 with material manufactured in this facility.  The plant has been designed and commissioned as a multi-product facility intended to support commercial licensure by both the U.S. Food and Drug Administration and European Medicines Agency.
  • Commenced enrollment in INCEPTUS: INCEPTUS is a prospective clinical assessment and phase 1 / 2 run-in study designed to characterize disease presentation in boys aged three years or younger living with XLMTM. INCEPTUS is designed to serve as a longitudinal baseline and within-patient control for subjects who enroll in the ASPIRO phase 1 / 2 study.  As of January 1, 2017, 12 of the expected 12 to 15 patients have been enrolled.
  • Completed initial public offering: Strengthened the balance sheet with the completion of an initial public offering in July 2016 resulting in aggregate cash proceeds to the company of $75.2 million, net of underwriting discounts, commissions and offering costs.

2017 Outlook for Development Pipeline

AT132 for X-Linked Mytubular Myopothy
AT132 is an AAV8 vector containing a functional copy of the MTM1 gene for the treatment of 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. Multiple studies in animal models of XLMTM have demonstrated that a single administration of AT132 significantly improves disease symptoms and survival rates.  In one study, these effects have lasted approximately four years to date.

Planned 2017 Milestones

  • File IND in the first quarter of 2017
  • Complete enrollment and continue data collection in INCEPTUS, a clinical assessment and phase 1 / 2 run-in study
  • Commence enrollment in ASPIRO, a phase 1 / 2 multicenter, multinational, open-label, ascending dose, delayed-treatment concurrent control clinical study in 12 patients
  • Continue internal cGMP manufacturing to supply ASPIRO
  • Report preliminary clinical data by the end of 2017


AT342 for Crigler-Najjar Syndrome  
AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene for the treatment of Crigler-Najjar, 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 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.

Planned 2017 Milestones

  • Commence enrollment in LUSTRO, a clinical assessment and phase 1 / 2 run-in study intended to characterize the disease course, natural history, bilirubin variability and phototherapy usage in subjects with Crigler-Najjar
  • Commence enrollment in VALENS, a phase 1 / 2 multicenter, multinational, open-label ascending dose, delayed-treatment concurrent control clinical study in 12 patients
  • Conduct internal cGMP manufacturing to supply VALENS
  • Report preliminary clinical data by the end of 2017


AT982 for Pompe Disease
AT982 is an AAV9 vector containing a functional copy of the GAA gene designed to treat Pompe disease.  Pompe disease is a rare monogenic disease characterized by severe muscle weakness, respiratory failure leading to ventilator dependence and, in infants, increased cardiac mass and heart failure. Treatment with AT982 in a mouse model of Pompe disease resulted in significant improvements in weight gain, ventilation parameters, glycogen deposition and cardiac left ventricle mass. The only approved treatment for Pompe disease is enzyme replacement therapy (ERT). Although ERT is the current standard of care for the disease, it has a number of recognized limitations.

Planned 2017 Milestones

  • Establish internal cGMP manufacturing
  • Conduct additional preclinical studies in support of an IND for a phase 1 / 2 clinical study evaluating systemic administration of AT982 in patients with Pompe disease
  • Conduct preclinical proof-of-concept studies evaluating intrathecal administration of AT982
  • Report preliminary data from an investigator-sponsored study evaluating intramuscular injection of AT982 in the tibialis anterior muscle of adults with Pompe disease by the end of 2017


AT307 for CASQ2-Catecholaminergic Polymorphic Ventricular Tachycardia ( CASQ2-CPVT)
AT307 is an AAV9 vector containing a functional copy of the CASQ2 gene for the treatment of CASQ2-CPVT, a rare monogenic disease that is characterized by life-threatening arrhythmias that may lead to sudden cardiac death. Treatment with AT307 in mouse models of CASQ2-CPVT has generated highly significant reductions in ventricular arrhythmic events following a single administration.

Planned 2017 Milestones

  • Complete IND enabling preclinical studies
  • Establish internal cGMP manufacturing 
  • File IND by the end of 2017


2017 Financial Guidance
As of September 30, 2016, Audentes had cash, cash equivalents and short-term investments of $119.2 million, which includes net proceeds of $75.2 million from its initial public offering that closed in July 2016.  The current cash position is projected to fund operations into late 2018.

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: our ability to conduct IND enabling preclinical studies of AT982 and AT307; our ability to file INDs for AT132 and AT307; our ability to initiate a clinical assessment and run-in study of AT342, initiate phase 1 / 2 clinical trials of AT132 and AT342, and report preliminary data from AT132, AT342 and AT982; our ability to successfully manufacture clinical trial material for use in phase 1 / 2 studies for AT132 and AT342, and to establish internal manufacturing for AT982 and AT307; and the expectation that our current cash balance will fund operations into late 2018.  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.