MeiraGTx’s Parkinson’s Gene Therapy Candidate Shows Promise in Clinical Trial, Stock Soars
MeiraGTx Holdings (MGTX) is experiencing a surge in its stock price after its gene therapy candidate, AAV-GAD, for treating Parkinson’s disease (PD), demonstrated positive results in a clinical bridging study. The study successfully met its primary objective of safety and tolerability, while also revealing significant improvements in key efficacy endpoints. This news has sent MGTX’s stock soaring by 14.9%.
The MGT-GAD-025 study, which investigated the efficacy of AAV-GAD for PD, showcased substantial and clinically meaningful advancements from baseline in key efficacy endpoints after 26 weeks of treatment. Notably, the high-dose group experienced a statistically significant 18-point average improvement from baseline in the UPDRS Part 3 (motor examination) “off” medication score at week 26. In contrast, no significant changes were observed in the sham or low-dose groups.
Furthermore, MeiraGTx reported significant dose-dependent improvements from baseline in the disease-specific, patient-reported quality of life PDQ-39 score in both the high and low-dose groups. The sham group, however, did not exhibit any significant changes at 26 weeks.
Importantly, the investigational gene therapy was found to be generally safe and well-tolerated. No serious adverse events related to AAV-GAD treatment were observed.
Addressing a Significant Unmet Medical Need
Parkinson’s disease, the second most common neurodegenerative disorder after Alzheimer’s, affects nearly one million individuals in the United States alone, with an estimated 90,000 new cases diagnosed annually. Globally, over 10 million people are living with PD. While initial treatment with dopamine replacement therapy often proves effective, its efficacy tends to diminish over time, leading to adverse side effects that significantly impact quality of life and daily functioning. This unmet medical need underscores the importance of innovative treatments like AAV-GAD.
While the cause of PD remains unknown for most, a small percentage of cases have a genetic basis. Regardless of the underlying cause, PD disrupts movement control circuits. AAV-GAD employs a novel mechanism of action aimed at correcting these aberrant circuits, which arise from dopamine depletion in the brain of idiopathic PD patients as the disease progresses.
Using MeiraGTx’s proprietary delivery instrument, AAV-GAD is administered as a one-time infusion into the subthalamic nucleus, a key regulator of the circuits responsible for normal movement.
MeiraGTx’s Next Steps for AAV-GAD
Based on the encouraging data from the MGT-GAD-025 study, MeiraGTx is preparing to discuss these results with regulatory bodies in the United States, EU, and Japan. The goal is to potentially initiate a phase III study that would support the global approval of this disease-modifying treatment. This signifies a crucial step towards making AAV-GAD available to patients who desperately need effective options.
Beyond AAV-GAD, MeiraGTx has multiple other programs in clinical development. These include the phase III Lumeos study for X-linked retinitis pigmentosa, phase I/II programs in achromatopsia and RPE65-deficiency, a phase II study for radiation-induced xerostomia, and a separate mid-stage study for Sjogren’s syndrome. The company’s commitment to developing innovative gene therapies for a range of diseases highlights its dedication to improving patient lives.
