Today, 1st May, at a meeting of the American Society of Gene and Cell Therapy, Audentes announced new positive data from ASPIRO their Phase1/2 clinical trial for the gene therapy product AT132.

The information being presented was 48 weeks follow up for 6 treated patients (Cohort 1), and 24 weeks follow up for 3 more treated patients (Cohort 2). The patients ranged in age from 8 months to 6 years and all were enrolled in the pre-trial study INCEPTUS.

Cohort 1 received the first dose that the study was designed to try, and Cohort 2 received the second dose.

Significant and sustained improvements’ have been seen in all patients including;

– improvements in respiratory function, with reduction in ventilator usage for all patients including 4 of the patients achieving ventilator independence
– achievement of motor milestones such as head control, sitting unaided, crawling and step taking
– muscle biopsies taken at 24 and 48 weeks show marked improvements in the muscle structure, with improvements continuing to be seen from 24 to 48 weeks. The muscle biopsies of the children who received the second dose (cohort 2) showed a more rapid improvement in muscle structure.

From a safety perspective, the treatment was generally well tolerated and there seemed to be no difference in reaction between the 6 patients dosed with dose 1 and the three patients dosed with dose 2. What reactions there were (some liver reactions, nausea and mild heart inflammation) which were only experienced by some patients, have been effectively managed, and have not returned.

The next steps include using this data to identify and agree on the ideal dosage for this gene therapy, and further discussion with the regulators in Europe (EMA) and the US (FDA) about possible pathways to licensing this treatment.

For more details please read Audentes 1st May press release.

Letter from Audentes to patient community
English 01MAY2019 XLMTM Patient Letter
French 01MAY2019 XLMTM Patient Letter FR
German 01MAY2019 XLMTM Patient Letter DE
Italian 01MAY2019 XLMTM Patient Letter IT
Polish 01MAY2019 XLMTM Patient Letter PL
Spanish 01MAY2019 XLMTM Patient Letter ES

Update 14.5.19 – What is the status of enrollment for INCEPTUS and ASPIRO?

INCEPTUS is currently closed for enrollment and will likely remain closed for the time being as we continue to gather ongoing longitudinal data on the children currently in the study. There is a possibility, based on discussions with the regulators, that additional patients may be enrolled into INCEPTUS in a very limited manner. This will be discussed at a site to site level with the Principal Investigators. Regardless, if patients and families are keen to take part in the study, they should contact their local PI and ask to be placed on the waiting list that is maintained at each site.
ASPIRO is actively enrolling patients.
Each clinical trial site maintains a wait-list which they manage for patients interested in participating in the clinical trials. These sites continue to evaluate potential patients on these wait-lists based on the protocol criteria and in case there is a future need to enroll more patients into the clinical trials in the case that regulators require this. In addition to the inclusion/exclusion criteria listed on, each clinical trial site may have additional considerations, such as capacity at their site. The overall number of patients in the clinical trial is also limited.

Gene Therapy, the first treatment candidate for XLMTM, is explained here on the ASGCT website and in an educational video.

Please note this important message from Audentes:

“We would like to ask for your continued partnership in understanding the need to refrain from any discussions (including social media, media, online, telephone or in-person communications) about how children in the ASPIRO clinical trial may be doing while the trial is in progress. This includes friends, families and patient groups. Please refrain from proactively asking parents of children enrolled in the ASPIRO clinical trial for information regarding their child’s medical status. This is critical in helping to maintain the integrity of the data coming out of the trial.

Our hope is to demonstrate the safety and efficacy of the investigational gene therapy product candidate to meet the needs of the regulators, such that it will benefit as many children and families affected by XLMTM as possible, in the shortest amount of time. We do this best by running a robustly controlled and scientifically disciplined clinical trial and we need your help in making sure this occurs”.