MeiraGTx Holdings plc announced positive top-line data from its clinical bridging study, MGT-GAD-025, of AAV-GAD for the treatment of Parkinson’s disease. The study successfully met its primary objective of safety and tolerability, with no serious adverse events related to AAV-GAD treatment observed across a total of 58 patients in three independent clinical studies.
The data demonstrated significant and clinically meaningful improvements from baseline for key efficacy endpoints at 26 weeks. Specifically, the high dose group showed an 18-point average improvement in Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 'off' medication score, which was statistically significant (p=0.03). This improvement is considered substantial, as a change of 5 to 10 points is clinically meaningful.
Furthermore, significant improvements were observed in the Parkinson’s Disease Questionnaire (PDQ-39) score, a key patient-reported quality of life measure. The high dose group improved by 8 points (p=0.02) and the low dose group by 6 points (p=0.04) from baseline at 26 weeks. These changes are also considered substantial, given that a 2 to 4-point change is clinically meaningful for the PDQ-39. The company plans to engage with global regulatory agencies to initiate a Phase 3 registrational study based on these results.
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