Audentes Therapeutics announced continuing positive interim data from the group of patients receiving the first dose of the gene therapy clinical trial ASPIRO, including muscle biopsy results from the first three patients treated in the study and the week 24 efficacy analysis for Patient 3.

Audentes Therapeutics reported:

–          Muscle biopsy data demonstrate highly efficient tissue transduction as indicated by vector copy number, robust myotubularin protein expression as assessed by western blot, and significant improvement in histology in first three treated patients at 24-week time point

–          Efficacy analysis updated to include week 24 assessments for Patient 3, demonstrating significant improvements in neuromuscular and respiratory function and reduction in ventilator dependence

–          No serious adverse events in any patient since the last data update provided at ASGCT in May 2018

–          Proceeding with per protocol dose escalation from 1×1014 vector genomes per kilogram (vg/kg) to 3×1014 vg/kg

Read the Audentes Therapeutics 7th August Press Release here

This is very positive and significant news. Audentes Therapeutics have however reminded the patient community that:

“It is important to note:

– we cannot make any conclusions on the interim findings of the clinical trial until after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analysed
– making conclusions about interim data may not accurately predict the full risk/benefit profile of an investigational product
-after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analysed, more complete information about safety and efficacy of this investigational gene therapy product will become available to the community.”

Audentes Therapeutics letter 7th August

Audentes Therapeutics has now analysed the muscle biopsies gathered from the 3 initial Cohort 1 patients at 24 weeks following administration.  The data, along with the safety and efficacy assessments from all patients, has been reviewed with the independent Data Monitoring Committee and the decision to escalate the dose in line with the planned protocol is the next step to determine the optimal dose.  Audentes Therapeutics expects to provide an update on these plans later in 2018 at the 23rd International Congress of the World Muscle Society, October 2–6, 2018.