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MGTX Stock Up on Upbeat Efficacy Data From Parkinson’s Disease Study

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MeiraGTx Holdings‘ (NASDAQ: MGTX) clinical bridging study of its gene therapy candidate, AAV-GAD, for treating Parkinson’s disease (PD) met its primary study objective of safety and tolerability. The stock soared 14.9% in response to the news.

MGTX’s PD Gene Therapy Candidate Betters Sham Treatment

The MGT-GAD-025 study of AAV-GAD for PD demonstrated significant and clinically meaningful improvements from baseline for key efficacy endpoints after 26 weeks of treatment.

Per the data readout, treatment with the candidate resulted in a statistically significant 18-point average improvement from baseline in the UPDRS Part 3 (motor examination) “off” medication score in the high-dose group at week 26. However, no significant change in the sham or low-dose groups was observed.

Additionally, MeiraGTx reported significant dose-dependent improvements from baseline in the disease-specific, patient-reported quality of life PDQ-39 score in both the high and low-dose groups with no significant change in the sham group at 26 weeks.

Year to date, shares of MeiraGTx have plunged 24.1% compared with the industry’s 1.8% decline.

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The investigational gene therapy was also found to be generally safe and overall well-tolerated. No serious, AAV-GAD treatment-related adverse events were observed.

Per MGTX, PD is the second most common neurodegenerative disorder after Alzheimer’s, affecting nearly one million people in the United States, with around …

Full story available on Benzinga.com

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