MeiraGTx Announces Positive Phase 2 Data for AAV-GAD Gene Therapy in Parkinson’s Disease, Paving Way for Phase 3 Trial

Positive Phase 2 Data:
MeiraGTx has announced positive data from its clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease, showing significant and clinically meaningful improvements in motor function and quality of life.

Study Design:
The randomized, double-blind, sham-controlled study involved 14 patients with idiopathic Parkinson’s disease, with participants receiving either a high dose, low dose, or sham treatment.

Efficacy:
The high-dose group showed an 18-point average improvement in the Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3, and significant improvements in the Parkinson’s Disease Questionnaire (PDQ-39) score, indicating improved quality of life.

Safety:
No serious adverse events (SAEs) related to AAV-GAD treatment were reported, demonstrating the therapy’s safety and tolerability.

Future Plans:
MeiraGTx is working towards a Phase 3 trial to further explore the therapy, with discussions ongoing with regulators in the US, Europe, and Japan.

Disease Impact:
Parkinson’s disease is a severe neurodegenerative disorder affecting over 10 million people worldwide, with current treatments often failing to manage symptoms effectively over time.

Gene Therapy Mechanism:
AAV-GAD is designed to deliver the GAD gene to brain cells, boosting GABA levels to normalize brain signaling and alleviate Parkinson’s symptoms.

Clinical Development:
MeiraGTx has treated a total of 58 patients in this development program across three independent multicenter clinical studies, with no SAEs related to AAV-GAD treatment.