On 24 December 2020, Audentes Therapeutics, an Astellas genetic medicines company, announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold for the ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy (XLMTM).
“We are grateful for the efforts of our team and investigators who have worked tirelessly to answer the FDA’s questions and we now look forward to resuming this study,” said Natalie Holles, President and Chief Executive Officer of Audentes. “We want to again extend our deepest sympathies to patients’ families impacted by the events earlier this year. We are deeply committed to the continued safe development of AT132 for the families and patients living with XLMTM, a disease with no existing treatments.”
The company is now working to complete all clinical and regulatory activities necessary to resume dosing and plans to have discussions at a future date with the regulators on the path forward toward global registration filings for AT132.
About the ASPIRO Study
ASPIRO is a two-part, multinational, randomized, open-label ascending dose trial to evaluate the safety and preliminary efficacy of AT132 in XLMTM patients less than five years of age. Primary endpoints include safety (adverse events and certain laboratory measures) and efficacy (assessments of neuromuscular and respiratory function). Secondary endpoints include the burden of disease and health-related quality-of-life, and muscle tissue histology and biomarkers.
Read more about AT132 gene therapy, and other clinical trials for MTM/CNM.
You can read and download the full press release here (PDF): Audentes Press Release 24DEC2020
