MeiraGTx’s Gene Therapy Shows Significant Improvements in Motor Function and Quality of Life in Parkinson’s Disease Trial

Clinical Trial Success:

MeiraGTx's gene therapy, AAV-GAD, has shown promising results in a Phase 2 clinical trial for treating Parkinson's disease, demonstrating both safety and efficacy.

Motor Function Improvement:

The high dose group in the trial experienced a statistically significant 18-point average improvement in motor examination scores (UPDRS Part 3) at week 26, indicating substantial improvement in motor function.

Quality of Life Enhancement:

Significant improvements in quality of life were observed, with the high dose group showing an 8-point improvement in PDQ-39 scores, and the low dose group showing a 6-point improvement, compared to a 0.2-point worsening in the sham group.

Safety and Tolerability:

The therapy was found to be safe and well-tolerated, with no serious adverse events (SAEs) related to AAV-GAD treatment reported in the study.

Future Plans:

MeiraGTx plans to discuss the results with regulators in the U.S., Europe, and Japan to initiate a Phase 3 study, aiming for global approval of the disease-modifying treatment.

Mechanism of Action:

AAV-GAD works by delivering the glutamic acid decarboxylase (GAD) enzyme to the subthalamic nucleus in the brain, which catalyzes the conversion of glutamic acid into GABA, helping to alleviate motor symptoms in Parkinson's disease.

Global Impact:

Parkinson’s disease affects nearly one million people in the U.S. and over 10 million worldwide, making it the second most common neurodegenerative disease after Alzheimer’s.