MeiraGTx Holdings’ 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 90,000 new cases diagnosed annually. Globally, more than 10 million people are living with PD. Initially, most patients respond well to dopamine replacement therapy, but over time, its effectiveness often diminishes and adverse side effects may arise, significantly impacting quality of life and daily functioning. This represents a high unmet medical need.

While the cause of PD remains unknown for most, a small percentage have a genetic basis. In all cases, PD involves disruptions in movement control circuits.

AAV-GAD has a novel mechanism of action that aims to correct the aberrant circuitry that results from the depletion of dopamine in the brain of idiopathic PD patients as the disease progresses. Using the company's proprietary delivery instrument, AAV-GAD is administered as a one-time infusion into the subthalamic nucleus, a key regulator of the circuits responsible for normal movement.

MGTX’s Path Forward for AAV-GAD Following Upbeat Results

Based on the encouraging data readout from the MGT-GAD-025 study, MeiraGTx is currently gearing up to discuss these results with regulatory bodies in the United States, EU and Japan to potentially initiate a phase III study that will support the approval of this disease-modifying treatment globally.

Apart from AAV-GAD, the company has multiple other programs in clinical development, including the phase III Lumeos study for X-linked retinitis pigmentosa, phase I/II programs in achromatopsia and RPE65-deficiency, phase II study for radiation-induced xerostomia and a different mid-stage study for Sjogren’s syndrome.

MeiraGTx Holdings PLC Price and Consensus

MeiraGTx Holdings PLC price-consensus-chart | MeiraGTx Holdings PLC Quote

MGTX’s Zacks Rank & Stocks to Consider

MeiraGTx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals ANIP, Alnylam Pharmaceuticals ALNY and Amicus Therapeutics FOLD, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share (EPS) have moved up from $4.69 to $4.81. EPS estimates for 2025 have improved from $5.37 to $5.86, during the same period. Year to date, shares of ANIP have gained 6.3%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.

In the past 60 days, estimates for Alnylam’s 2024 loss per share have narrowed from $1.20 to 63 cents. Loss per shares estimates for 2025 have narrowed from 34 cents to 26 cents, during the same period. Year to date, shares of ALNY have risen 50.1%.

Alnylam’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 108.53%.

In the past 60 days, estimates for Amicus’ 2024 EPS have remained constant at 20 cents. EPS estimates for 2025 have increased from 48 cents to 50 cents, during the same period. Year to date, shares of FOLD have lost 27.3%.

Amicus’ earnings beat estimates in three of the four trailing four quarters, missing the mark once, with the average surprise being 23.96%.

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