On announcing 3rd quarter financial results for Audentes Therapeutics today, on 6th November, Matthew R. Patterson, Chairman and Chief Executive Officer stated “In the third quarter, we continued to see exciting progress in the clinical development of AT132”  and the press release also provided an update on the progress seen with the dosed patient cohorts in the AT132 x-linked myotubular myopathy AAV gene therapy programme.

To date, three patients in Cohort 1 (at the lowest dose) have achieved ventilator independence following a dose of AT132, and all other patients showed significant reductions in ventilator dependence which is ‘an unprecedented result for children with congenital myopathies that have been ventilated since birth’.  They also confirm that they have completed enrollment of Cohort 2 of ASPIRO, including three patients treated at a dose of 3×1014 vg/kg and one enrolled as the Cohort 2 untreated control.  Details about their other programmes and manufacturing capability for the AAV viral vector were also shared, stating that their ‘current facility, scale and process yield provide sufficient capacity for the expected global commercial needs of XLMTM and Crigler-Najjar markets, and ongoing clinical supply for current and planned pipeline programs’. You can read the full press release here.

A new XLMTM Patient Letter from Audentes Therapeutics provides more detailed information about the AT132 AAV gene therapy programme for x-linked myotubular myopathy, including a statement about patient inclusion in INCEPTUS and ASPIRO.

In Q3 2018 Audentes Therapeutics plans to engage with FDA and EMA to discuss development plans and the potential registration pathway for AT132 AAV gene therapy for x-linked myotubular myopathy within USA and Europe.

The next update will be provided by Audentes Therapeutics in Q1 2019.

Patient letter in other languages

XLMTM Patient Letter 06NOV2018 Polish

XLMTM Patient Letter 06NOV2018 Italian

XLMTM Patient Letter 06NOV2018 French

XLMTM Patient Letter 06NOV2018 Spanish

XLMTM Patient Letter 06NOV2018 German