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Avexis Completes Phase I Trial Patient Enrolment

08 January 2016

Avexis has announced that patient enrolment is complete in a Phase I clinical trial of their gene therapy drug, scAAV9.CB.SMN.

Originally named ChariSMA, but referred to as AVXS-101 in their most recent press release, the Avexis drug makes use of harmless viruses to restore SMN protein levels, and has been shown to improve disease symptoms in SMA model mice (for further information click here).

The Phase I trial has been designed to primarily test the safety and tolerability of the drug in children diagnosed with Type I SMA before six months of age (Click here for more information). Patients were also selected based on their having just two copies of the backup Survival Motor Neuron 2 (SMN2) gene.

This early stage trial includes two treatment groups:

Group 1 has three patients aged 6-7 months (at the time of treatment) who were given a lower virus dose, while Group 2 includes 12 patients aged 1-8 months and treated with a higher virus dose.

Patients are being monitored weekly in the first month after treatment and will be assessed at monthly intervals for up to 2 years. As a secondary measure, the trial also aims to measure drug effectiveness by recording the time taken for an adverse, disease-related event to occur.

Further Information

Avexis press release

scAAV9.CB.SMN clinical trial website