Audentes Therapeutics announced continuing positive interim data from the group of patients receiving the first dose of the gene therapy clinical trial ASPIRO. Three more patients have been added to this first dose cohort and Audentes Therapeutics reported:
– significant improvements in neuromuscular function at the 24 week time point
– significant improvements in respiratory function at the 24 week time point
– encouraging initial efficacy at the 4 week time point in the 3 further patients
– patient 1 (from the first cohort) has achieved ventilator independence and oral feeding

Read the Audentes Therapeutics 16th May Press Release here, with further detail on efficacy data for each patient in Cohort 1

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 analyzed, more complete information about safety and efficacy of this investigational gene therapy product will become available to the community.”

Read Audentes Therapeutics letter 16th May 2018 to the patient community here

Audentes Therapeutics is currently completing analysis of the muscle biopsies gathered from initial Cohort 1 patients.  These data, along with the safety and efficacy assessments from all patients, will be reviewed with the independent Data Monitoring Committee to inform decisions regarding dose escalation.  Audentes Therapeutics expects to provide an update on these plans in the third quarter of 2018.

A very important question that emerges from the patient community at this phase in a research study is the biotechnology company’s approach to early access to the potential drug. Audentes have updated their website on their current position on Expanded Access. Read here

Patient community letter in other translations:

German – 16 May 2018 XLMTM Patient Letter_DE

Spanish – 16 May 2018 XLMTM Patient Letter_ES

French –  16 May 2018 XLMTM Patient Letter_FR

Italian – 16 May 2018 XLMTM Patient Letter_IT

Polish – 16 May 2018 XLMTM Patient Letter_PL