The trial demonstrated safety and a statistically significant improvement in both motor function and brain metabolism at one year. It also confirmed the safety and tolerability of the therapy in all twelve patients enrolled in the trial. There were no adverse events reported relating to the treatment.
Nine of the 12 patients showed an average improvement of 37%, and 5 of these patients had substantial improvement of between 40% and 65%.
“This gene therapy trial is particularly unique and the clinical data unusually promising because the treatment was confined to just one side of the brain,” stated Dr Matthew During who presented the data.
In its next trial, the company plans to infuse its treatment into both sides of the brain.
The gene transfer procedure utilized the AAV (adeno-associated virus) vector, a virus that has been used safely in a variety of clinical gene therapy trials, and the vehicle that will be used in all of the company’s first generation products, including epilepsy and Huntington’s disease.
Neurologix’s initial development efforts are focused on its core gene therapy technology, NLX, for treating Parkinson’s disease, epilepsy and Huntington’s disease.