Audentes Therapeutics presents new ‘positive’ interim data from ASPIRO

Audentes Therapeutics announced new positive interim data from ASPIRO, the Phase 1/2 clinical trial of AT132 for the treatment of X-linked Myotubular Myopathy. The data will be presented at the 23rd International Annual Congress of the World Muscle Society by Nancy Kuntz, MD, Principal Investigator at Ann & Robert H. Lurie Children’s Hospital of Chicago, Medical Director of Mazza Foundation Neuromuscular Program and Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine. The oral presentation by Dr. Kuntz will be held during a session on new therapeutic modalities scheduled to begin at 9:30 a.m. ET on October 5, 2018. The newly reported data include follow-up assessments ranging from 4 to 48 weeks for the eight patients enrolled in ASPIRO to date, including the seven patients enrolled in Cohort 1 (1×1014 vg/kg; six treated and one untreated control) and one patient enrolled to date in Cohort 2 (3×1014 vg/kg). Key assessments include neuromuscular function as measured by CHOP INTEND; respiratory function as measured by maximal inspiratory pressure and ventilator dependence; and vector transduction, transgene expression and histological improvement as assessed via muscle biopsy. All treated patients continue to show meaningful improvements in neuromuscular and respiratory function, with no new treatment-related SAEs reported since the last scientific update in May 2018.
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