Small Cap-MeiraGTx Reveals Encouraging Data From Parkinson's Study Showing Significant, Clinically Meaningful Changes in Key Efficacy Endpoints

On Tuesday, MeiraGTx Holdings plc MGTX released topline data from its clinical bridging study of AAV-GAD for Parkinson’s disease, MGT-GAD-025.

MGT-GAD-025 is a 6-month, three-arm, randomized, double-blind, sham-controlled study using AAV-GAD drug product manufactured by MeiraGTx at its wholly-owned facilities with its commercial platform process.

Participants had idiopathic Parkinson’s disease, a history of levodopa responsiveness for at least 12 months, and a UPDRS Part 3 score of ≥25 points in the “off” state.

Fourteen subjects were randomized to one of three groups (high dose n=5, low dose n=5, and sham n=4)

The primary objective of the study was to evaluate the safety and tolerability of AAV-GAD, with exploratory efficacy endpoints including the mean change from baseline to Week 26 in MDS-UPDRS Part 3 (motor examination) scores in the “off” state and the Parkinson’s Disease Questionnaire (PDQ-39) score, a key patient-reported quality of life measure in Parkinson’s disease.

AAV-GAD was safe and well tolerated, with no serious adverse events (SAEs) related to AAV-GAD treatment.

At Week 26, the high-dose group demonstrated a statistically significant 18-point average improvement from baseline in the UPDRS Part 3 “off” medication score (p=0.03), with no significant change in the sham or low dose groups.

Significant improvements from baseline in the disease-specific, patient-reported quality of life PDQ-39 score were demonstrated in both the high and low-dose groups with no significant change in the sham group at Week 26:

• In the high-dose AAV-GAD group, the PDQ-39 score improved by 8 points from baseline (p=0.02), the low-dose group improved by 6 points from baseline (p=0.04), while the 0.2 point worsening in the sham surgery group was not statistically significant.
• A dose-response in PDQ-39 score was observed, with 100% of participants in the high-dose group, 60% in the low-dose group, and 25% in the sham surgery group reporting an improvement.
• For the PDQ-39 score, there was a trend to significance between the high dose and sham surgery groups at six months (n=4 evaluable per group).

Subjects who completed this trial may enroll in a long-term follow-up study, in which they will be monitored for five years after treatment.

Price Action: MGTX stock is up 13.2% at $5.25 at last check Tuesday.

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