Following the release of encouraging findings from its clinical research, shares of MeiraGTx Holdings plc (NASDAQ: MGTX) have seen a significant boost, reaching $5.25, or 13.15%, in value. Top-line data from the company’s clinical bridging trial, MGT-GAD-025, which focused on AAV-GAD as a possible Parkinson’s disease therapy, was released.
Promising Safety and Efficacy Results
The safety and efficacy outcomes reported by MeiraGTx (MSTX) are particularly encouraging. Notably, motor scores and quality of life significantly improved for individuals undergoing the high-dose therapy. Both patients and medical professionals stand to benefit greatly from these advancements.
The clinical data, derived from material produced using MGTX’s proprietary commercial platform process, indicate that AAV-GAD is safe across all dosage levels studied, including higher doses not previously tested. To date, MeiraGTx has treated a total of 58 patients across three independent multicenter clinical trials, with no serious adverse events associated with AAV-GAD treatment.
Significant Clinical Impact
With the completion of this randomized, double-blind bridging study, MeiraGTx has demonstrated that AAV-GAD treatment leads to substantial and clinically meaningful changes in key efficacy endpoints for Parkinson’s disease. A change of 5 to 10 points in UPDRS Part 3 is considered clinically significant, and the observed 18-point improvement in the high-dose group underscores the treatment’s potential impact.
Similarly, for the PDQ-39 assessment, the changes of 8 points in the high-dose group and 6 points in the low-dose group further emphasize the treatment’s effectiveness.
Path Forward for Global Approval
The findings highlight the potential of targeted local delivery of gene-based therapy to address the dopamine depletion characteristic of idiopathic Parkinson’s disease. AAV-GAD treatment aims to normalize circuit function across various forms of the disease.
The significant clinical improvements observed in this study provide MeiraGTx with a clear path forward in its clinical development strategy, bolstering discussions with regulatory authorities in the U.S., Europe, and Japan to initiate a Phase 3 study aimed at global approval of this disease-modifying therapy.
