Posted by Michael Wonder on 28 Sep 2017
Audentes Therapeutics announces rare paediatric disease and fast track designations for AT132 for the treatment of X-linked myotubular myopathy
27 September 2017 - Preliminary data from ASPIRO, the Phase 1 / 2 study of AT132, expected to be available in the fourth quarter of 2017.
Audentes Therapeutics today announced that the U.S. FDA has granted rare paediatric disease and fast rrack designations for AT132, the company's gene therapy product candidate being developed to treat X-Linked Myotubular Myopathy (XLMTM). In addition to these two new designations, AT132 has also received orphan drug designation from both the FDA and EMA.
Audentes recently announced the enrollment of the first patient in ASPIRO, the Phase 1 / 2 clinical trial of AT132 in patients with XLMTM. 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 has previously announced data from an initial analysis of 112 male subjects. This analysis confirmed and expanded upon the understanding of the significant disease burden of XLMTM on patients, families and the healthcare system. Audentes is also conducting INCEPTUS, a prospective natural history and run-in study. The primary objectives of INCEPTUS are to characterize the clinical condition of children with XLMTM, identify subjects for potential enrollment in ASPIRO, and serve as a longitudinal baseline and within-patient control for ASPIRO.
