MeiraGTx’s AAV Gene Therapy Shows Promising Results for Parkinson’s

Significant Improvements in Motor Function and Quality of Life

The trial, named MGT-GAD-025, evaluated 14 patients with idiopathic Parkinson’s disease, divided into three groups: five patients receiving a high dose of AAV-GAD, five receiving a low dose, and four undergoing a sham procedure. The AAV-GAD therapy involves a one-time infusion of the drug into the **subthalamic nucleus**, a brain region critical to motor control. This therapy delivers a gene that promotes the production of GABA, a neurotransmitter associated with improved motor function.

At 26 weeks, the trial’s data revealed:

– High-dose group: 18-point improvement from baseline on the motor function section of the Unified Parkinson’s Disease Rating Scale (UPDRS) (p=0.03).
– Quality of life improvement: 8-point improvement in the Parkinson’s Disease Questionnaire (PDQ-39) score (p=0.02) in the high-dose group, compared to a 6-point improvement in the low-dose group (p=0.04) and no significant change in the sham group.

The trial also confirmed that AAV-GAD therapy was safe and well-tolerated across all dosage groups, with no serious adverse events (SAEs) reported.

Dr. Ali Rezai, executive chair of the Rockefeller Neuroscience Institute at West Virginia University and the study’s principal investigator, praised the results, saying, “The extent of motor score improvements and the significant quality of life benefits are very encouraging for both patients and physicians.”

A Step Closer to Phase III

MeiraGTx’s proprietary manufacturing process produced the therapy at commercial scale, demonstrating that the therapy is safe even at higher doses than previously tested. Across three independent studies, **58 patients** have now been treated with AAV-GAD, and no serious treatment-related safety concerns have emerged.

According to Dr. Alexandria Forbes, President and CEO of MeiraGTx, “Even with small patient numbers, AAV-GAD treatment results in significant and clinically meaningful changes in key efficacy endpoints in Parkinson’s disease.”

Addressing Unmet Needs in Parkinson’s Disease

Parkinson’s disease affects nearly one million people in the U.S. and more than 10 million people worldwide, with around 90,000 new U.S. cases diagnosed each year. While levodopa is the standard treatment to alleviate symptoms, its efficacy declines over time, leaving patients with limited options.

By targeting the dysfunctional brain circuits involved in movement control, AAV-GAD offers a novel therapeutic approach. The therapy’s mechanism focuses on restoring more normal activity in key cells through the production of GABA, which could offer new hope for patients with this degenerative condition.

MeiraGTx aims to continue its momentum with AAV-GAD by preparing for the next phase of clinical development. The company hopes to confirm the midphase trial’s results in a larger Phase III trial and bring the therapy closer to regulatory approval.

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