24/7 Market News- MeiraGTx Reports Positive AAV-GAD Study Data

October 15, 2024

DENVER, Colo., Oct 15, 2024 (247marketnews.com)- MeiraGTx (NASDAQ: MGTX) reported positive top-line data from its clinical bridging study of its AAV-GAD for the treatment of Parkinson’s disease, MGT-GAD-025, which is a 6-month, three-arm, randomized, double-blind, sham-controlled study.

The top-line data indicated that AAV-GAD was safe and well tolerated, with no serious adverse events related to AAV-GAD treatment, and after 26 weeks a substantial 18-point average improvement from baseline in UPDRS Part 3 “off” medication score was demonstrated in the high dose group (p=0.03), compared to no significant change in the sham or low dose groups.

The AAV-GAD study’s principal investigator and executive chair of the Rockefeller Neuroscience Institute at West Virginia University, Dr. Ali Rezai, M.D., commented, “These safety and outcome results are excellent. 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.”

Alexandria Forbes, Ph.D., MeiraGTx’s president and CEO, added, “With material made using our proprietary production process at commercial scale, we have demonstrated that AAV-GAD is safe at all doses studied, including a higher dose than previously tested. We have now treated a total of 58 patients in this development program in 3 independent multicenter clinical studies and have seen no SAEs related to AAV-GAD treatment.

“With the completion of this randomized, double-blinded bridging study, we have also demonstrated with even very small numbers of subjects that AAV-GAD treatment results in significant and clinically meaningful changes in key efficacy endpoints in Parkinson’s disease. For the UPDRS Part 3 in the “off” state, a change of 5 to 10 points is considered clinically meaningful. The 18-point change observed in the high dose arm in this study underscores the very substantial impact of AAV-GAD treatment in these Parkinson’s patients. Similarly, for the PDQ-39, where a 2 to 4-point change is considered clinically meaningful, the 8-point and 6-point changes observed in the high and low dose groups, respectively, again indicate a substantial and clinically meaningful impact of AAV-GAD treatment.

“These data demonstrate the impact of using highly targeted local delivery of gene-based therapy to correct the aberrant circuitry that results from the depletion of dopamine in the brain of idiopathic Parkinson’s patients as the disease progresses. AAV-GAD treatment is designed to normalize circuit function in all forms of Parkinson’s disease with its potential benefit not limited to any single type of Parkinson’s. The significant, substantial, and clinically meaningful changes observed in this small, sham-controlled study provide us with a clear path forward in our clinical development strategy and underpin our discussions with regulators in the US, Europe, and Japan with the goal of initiating a Phase 3 study to support approval of this disease-modifying treatment globally.”

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