In May Audentes released new data at the American Society for Gene and Cell Therapy (ASGCT) to include more positive feedback about their AT132 gene therapy trial programme (ASPIRO), which is aimed at patients with x-linked myotubular myopathy (MTM1) aged up to five years.
Outcomes from nine patients in total were shared at the meeting. Chairman and Chief Executive Officer of Audentes, Matthew R Patterson said “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. We look forward to important next steps for the program, including selection of the optimal dose and further discussions with regulators in the U.S. and Europe regarding possible pathways to license applications.”
- Audentes Therapeutics presents new positive data from ASPIRO, the phase 1/2 clinical trial of AT132 for X-linked myotubular myopathy, at 22nd annual meeting of the American Society of Gene and Cell Therapy
In October Audentes presented new positive data from ASPIRO, the clinical trial evaluating AT132 in patients with X-Linked myotubular myopathy, at the 24th International Annual Congress of the World Muscle Society. The data was presented by Dr James J Dowling from the Toronto Hospital for Sick Children. Dr Dowling said “The new ASPIRO data shared today builds upon the encouraging efficacy and safety profile seen to date with AT132. Treated patients across both dose cohorts show significant reductions in ventilator dependence and the progressive attainment of developmental motor milestones, suggesting that AT132 has the potential to deliver transformative benefit to patients and families living with XLMTM.”
Natalie Holles, President and Chief Operating Officer of Audentes said “We remain focused on our goal of rapidly progressing AT132 toward global regulatory approvals. Importantly, we have fully enrolled 14 patients into the ASPIRO dose escalation cohorts, and plan to complete enrolment of the ASPIRO pivotal expansion cohort imminently. ”
- Audentes Therapeutics presents new positive data from ASPIRO, the clinical trial evaluating AT132 in patients with X-Linked myotubular myopathy (XLMTM), at the 24th International Annual Congress of the World Muscle Society
In November the organisation reported its third quarter 2019 financial results and provided a corporate update. Matthew R Patterson, Chairman and Chief Executive Officer stated “AT132 continues to show a promising safety and efficacy profile in patients with XLMTM, with the first seven treated patients now ventilator independent. Beyond AT132, we are excited about the significant momentum building across our entire pipeline of development candidates.”
Share your experience with x linked myotubular myopathy (xlmtm): a survey for the US X linked myotubular myopathy community
Also in November, Audentes Therapeutics launched the xlmtm parent and caregiver community survey in the US. Initially the survey is live for US based families, with UK and German versions coming in early 2020.
The survey aims to gather important data related to the quality of life and economic impacts of xlmtm and to create a more thorough understanding of the quality of life and economic impact of xlmtm on families and caregivers. The results of this survey will be published and shared with the xlmtm community and may be shared with health policy makers and insurance companies to support patient access to future treatments.
More detailed information about the study is available below.