MCW research team contributes to positive clinical trial results for myotubular myopathy gene therapy

Audentes Therapeutics, Inc., recently presented new positive data on ASPIRO, a Phase 1/2 clinical trial of AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM) at the 2019 American Society of Gene and Cell Therapy (ASGCT) Conference in Washington DC. XLMTM is a severe, inherited myopathy that causes severe skeletal muscle weakness, respiratory insufficiency and death in children. Early nonclinical translational studies and the pathological evaluation of muscle biopsies from ASPIRO have been performed in the laboratory of Michael W. Lawlor, MD, PhD, associate professor of pathology and associate director of the Medical College of Wisconsin Neuroscience Research Center.

Dr. Lawlor provided an in-depth presentation of the muscle biopsy data in a talk entitled “The ASPIRO Study: Improvements in XLMTM Muscle Biology Pathology and Biomarkers” at the Audentes ASGCT 2019 Satellite Symposium on May 1.

The newly reported data include safety and efficacy assessments for 11 patients enrolled in ASPIRO as of the April 8, 2019 data cut-off date. The majority of patients have demonstrated progressive acquisition of motor developmental milestones including head control, sitting unassisted, crawling, standing with support and initiating stepping movements.

Patients receiving AT132 have achieved reductions in ventilator dependence not previously observed in chronically ventilated patients with neuromuscular disorders. Reduction of ventilator dependence is an endpoint considered to be correlated with survival. Four patients were successfully weaned off of ventilation by the 48-week timepoint, with all other treated patients demonstrating sustained and clinically meaningful reductions in ventilator use.

“We are excited to share today’s results and are working hard toward our goal of making AT132 available to patients living with XLMTM globally as rapidly as possible,” stated Matthew R. Patterson, chairman and Chief Executive Officer of Audentes.

The next steps in the AT132 development program include selection of the optimal dose in the ASPIRO study, planned to occur in the second quarter of 2019, and further interactions with the FDA and EMA, planned to occur in the third quarter of 2019, to present this most recent data update and gain further alignment on the license application submission pathways for AT132 in the United States and Europe.