MeiraGTx unveiled results from a small dataset for its Parkinson’s disease gene therapy on Tuesday morning, causing its shares $MGTX to jump by as much as 24%.
In what the company called a “bridging study” featuring 14 patients, the high dose of MeiraGTx’s AAV-GAD showed a statistically significant improvement on a Parkinson’s disease measurement score known as MDS-UPDRS Part 3 after 26 weeks. Five patients in the high-dose group improved their “off” state scores by an average of 18 points from baseline, good for a p-value of 0.03.
MeiraGTx CEO Alexandria Forbes said in a statement that the company expected a five- to 10-point improvement to represent a clinically meaningful change. The sham group (four patients) and low-dose group (five patients) saw no significant changes compared to baseline. The primary endpoint was safety and tolerability, and was met.
In another exploratory endpoint, MeiraGTx said a quality-of-life measure called the PDQ-39, which uses a patient questionnaire, achieved statistical significance in both dosing groups when compared to patients’ baseline scores. There was only a “trend to significance” between the high-dose group and the sham group, MeiraGTx said.
The company is still discussing its Phase 3 plans with regulators, but expects to launch a pivotal trial in the first half of 2025, according to a corporate slide deck updated Tuesday morning.
Gene therapies for Parkinson’s disease have been attempted before, though none have been FDA-approved. MeiraGTx’s program is designed to reset the abnormal brain activity associated with the disease by targeting production of a brain chemical called gamma-aminobutyric acid, or GABA. AAV-GAD attempts to deliver a functional copy of a gene called GAD into the brain that would increase GABA production and alleviate motor symptoms.