On Tuesday, MeiraGTx Holdings plc (NASDAQ: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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