Audentes’ Next Steps After Collaborative Initial Meeting with FDA

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 provided an update following the receipt of minutes from its Regenerative Medicine and Advanced Therapy (RMAT)/Type B meeting with the U.S. Food and Drug Administration (FDA) held in December 2018, regarding the company’s lead gene therapy candidate, AT132, for the treatment of X-linked Myotubular Myopathy (XLMTM).

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 FDA on program next-steps to enable submission of a Biologics License Application (BLA) for AT132.

“We’re pleased that our first discussion with the FDA under RMAT was both collaborative and productive, and provided a potential path toward BLA submission for AT132,” stated Matthew R. Patterson, Chairman and Chief Executive Officer.  “We appreciate the constructive feedback provided by the Agency and look forward to working with the FDA to advance AT132 to registration as quickly as possible.”

Following the meeting, Audentes is proceeding with its previously announced plan to enroll an additional 3-5 patients in Cohort 2 (3×10¹⁴ 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 FDA to facilitate final agreement on the path to BLA submission.  Audentes also received preliminary feedback on its CMC characterization and validation plans and will submit the requested information to the Investigational New Drug (IND) application on a continuing basis.

In pursuit of global regulatory approvals for AT132, Audentes also initiated discussions with the European Medicines Agency (EMA) in the fourth quarter of 2018 under the recently granted Priority Medicines (PRIME) designation.  In the first quarter of 2019, Audentes anticipates receiving scientific advice from the EMA that will begin to define the path toward submission of a European Marketing Authorization Application.

https://www.biospace.com/article/releases/audentes-therapeutics-announces-next-steps-in-the-development-of-at132-to-treat-xlmtm-following-collaborative-initial-meeting-with-fda-under-rmat-designation/