(RTTNews) - MeiraGTx Holdings plc (MGTX), a vertically integrated, clinical-stage genetic medicines company, announced Tuesday positive top-line data from MGT-GAD-025, its clinical bridging study of AAV-GAD for the treatment of Parkinson's disease.
In pre-market activity on the Nasdaq, MeiraGTx shares were gaining around 14.2 percent to trade at $5.2983
Parkinson's disease or PD is the second most common neurodegenerative disease after Alzheimer's.
AAV-GAD is an investigational gene therapy designed to reprogram dysfunctional brain circuits through the local production of GABA, a chemical neurotransmitter that can help restore more normal activity to these critical cells in any form of PD.
The company noted that the primary study objective of safety and tolerability was met in the MGT-GAD-025, a 6-month, three-arm, randomized, double-blind, sham-controlled study using AAV-GAD drug product manufactured by MeiraGTx at its facilities with its commercial platform process.
The company noted that AAV-GAD is delivered though a one-time infusion through a minimally invasive procedure, using a MeiraGTx proprietary device.
In the trial, fourteen subjects, who had idiopathic Parkinson's disease, were randomized to one of three groups, of high dose, low dose and sham, and they received either AAV-GAD infused bilaterally into the subthalamic nucleus or a sham procedure in a blinded fashion.
The primary objective of the study was to evaluate the safety and tolerability of AAV-GAD, with exploratory efficacy endpoints.
Subjects who completed this trial may enroll in a long-term follow-up study (NCT05894343), where they will be monitored for a total of five years post-treatment.
Ali Rezai, executive chair of the Rockefeller Neuroscience Institute at West Virginia University, and principal investigator of the AAV-GAD study, stated, "The extent of motor score improvements in patients who received the high dose treatment combined with significant quality of life improvement measures are very encouraging for both patients and physicians."
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