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SOURCE Audentes Therapeutics, Inc.
- Announced positive interim data in the first three patients dosed in ASPIRO, the Phase 1/2 clinical study of AT132 to treat X-Linked Myotubular Myopathy (XLMTM). Plan to dose three additional patients in the first cohort of ASPIRO and to report additional interim data in the second quarter of 2018.
- Initiated dosing in VALENS, the Phase 1/2 clinical study of AT342 to treat Crigler-Najjar Syndrome. No safety signals observed to date and plan to provide preliminary results in the second quarter of 2018.
- Selected clinical development candidate to treat Pompe disease. Plan to file IND mid-2018 and initiate a Phase 1/2 clinical trial of AT982 in the fourth quarter of 2018.
- Strengthened balance sheet with a follow-on financing of approximately $217.2 million in net proceeds in January 2018.
SAN FRANCISCO, March 8, 2018 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today reported its financial results for the fourth quarter and full year ended December 31, 2017 and provided an update on the company's recent achievements and anticipated upcoming milestones.
"2017 was a transformational year marked by significant achievements across our portfolio of gene therapy product candidates," stated Matthew R. Patterson, President and Chief Executive Officer. "Among our many accomplishments, we are particularly pleased to have recently reported positive interim data from the first dose cohort of patients in ASPIRO, the Phase 1/2 clinical study of our product candidate AT132 to treat XLMTM. These promising initial data add to the growing body of evidence of the utility of AAV-based gene therapy to treat rare neuromuscular diseases, and we are optimistic that they signal a turning point in our ability to bring meaningful benefit to patients and families living with XLMTM."
Mr. Patterson continued, "As we look forward to the year ahead, we are excited by our many anticipated upcoming pipeline catalysts, including sharing additional data from our ASPIRO study in XLMTM, new data from our VALENS study in Crigler-Najjar, and advancing additional product candidates into the clinic for Pompe disease and CASQ2-CPVT. With our recently completed follow-on offering, we are well capitalized to continue our mission to develop transformative gene therapy products for patients living with serious, life-threatening rare diseases."
Recent Achievements & Upcoming Key Events
Fourth Quarter and Full Year 2017 Financial Results
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its fourth quarter 2017 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and dial the conference ID 5890848 to access the call.
A replay of the webcast will be available on the Audentes website for approximately 30 days.
About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases. We are currently conducting Phase 1/2 clinical studies of our lead product candidates AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT342 for the treatment of Crigler-Najjar Syndrome. We have two additional product candidates in development, including 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 www.audentestx.com.
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: anticipated clinical milestones, the timing and nature of clinical development activities, the timing of the release of data from ongoing clinical trials, the timing of regulatory filings, the expected benefits of the company's product candidates and the use and adequacy of cash reserves. 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 commercialize its product candidates, the timing and results of preclinical and clinical trials, fund development activities and achieve development goals, establish and scale-up manufacturing processes that comply with regulatory requirements, 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.
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