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Audentes Therapeutics Announces Next Steps in the Development of AT132 to Treat XLMTM Following Collaborative Initial Meeting with FDA Under RMAT Designation
The goal of the meeting was to review nonclinical, clinical, and chemistry, manufacturing, and controls (CMC) data generated to date in the AT132 program, and to align with the
"We're pleased that our first discussion with the
Following the meeting, Audentes is proceeding with its previously announced plan to enroll an additional 3-5 patients in Cohort 2 (3x1014 vector genomes per kilogram dose level) of ASPIRO, the Phase 1/2 study of AT132. Optimal dose selection is expected to occur in the second quarter of 2019, after the evaluation of the six-month biopsy results from the first three patients dosed in Cohort 2. Subsequent to the determination of the optimal dose, Audentes plans to provide an updated data package to
In pursuit of global regulatory approvals for AT132, Audentes also initiated discussions with the
About AT132 for X-linked Myotubular Myopathy
AT132 is the Audentes product candidate being developed to treat XLMTM, a disease caused by mutations in the MTM1 gene, which encodes the protein 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. Over the course of 2018, Audentes reported promising safety, efficacy and muscle biopsy data from ASPIRO, a multicenter, ascending dose Phase 1/2 clinical study designed to evaluate the safety and efficacy of AT132 in approximately 12 XLMTM patients less than five years of age. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).
AT132 has been granted RMAT, Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA, and PRIME and Orphan Drug designations by the EMA.
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: the timing and nature of clinical development activities; the timing of regulatory interactions, submissions or approvals; and, the expected safety and efficacy profile of the company's product candidates. 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, the company's ability to 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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