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Over $5 5.70 + 22% MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson’s Disease
Source: GlobeNewswire Inc.
MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced top-line data from its clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease, MGT-GAD-025.
MGT-GAD-025 is a 6-month, three-arm, randomized, double-blind, sham-controlled study using AAV-GAD drug product manufactured by MeiraGTx at its wholly-owned facilities with its commercial platform process. Participants had idiopathic Parkinson's disease, a history of levodopa responsiveness for at least 12 months, and a UPDRS Part 3 score of ≥25 points in the "off" state. Fourteen subjects were randomized to one of three groups (high dose n=5, low dose n=5, and sham n=4).
Subjects received either AAV-GAD infused bilaterally into the subthalamic nucleus or a sham procedure in a blinded fashion. The total dose per treated participant was 7.0×1010 vg (low dose group) or 21×1010 vg (high dose group). The primary objective of the study was to evaluate the safety and tolerability of AAV-GAD, with exploratory efficacy endpoints including the mean change from baseline to Week 26 in MDS-UPDRS Part 3 (motor examination) scores in the “off” state and the Parkinson’s Disease Questionnaire (PDQ-39) score, a key patient-reported quality of life measure in Parkinson’s disease. 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.
Top-line data summary:
AAV-GAD was safe and well tolerated, with no serious adverse events (SAEs) related to AAV-GAD treatment.
At Week 26, a statistically significant 18-point average improvement from baseline in UPDRS Part 3 “off” medication score was demonstrated in the high dose group (p=0.03), with no significant change in the sham or low dose groups.
Significant improvements from baseline in the disease-specific, patient-reported quality of life PDQ-39 score were demonstrated in both the high and low dose groups with no significant change in the sham group at Week 26:
In the high dose AAV-GAD group, the PDQ-39 score improved by 8 points from baseline (p=0.02), the low dose group improved by 6 points from baseline (p=0.04), while the 0.2 point worsening in the sham surgery group was not statistically significant.
A dose response in PDQ-39 score was observed, with 100% of participants in the high dose group, 60% of participants in the low dose group, and 25% of participants in the sham surgery group reporting an improvement.
For the PDQ-39 score, there was a trend to significance between the high dose and sham surgery groups at 6 months (n=4 evaluable per group).
Dr. Ali Rezai, M.D., executive chair of the Rockefeller Neuroscience Institute at West Virginia University (WVU), past president of the Congress of Neurological Surgeons, and principal investigator of the AAV-GAD study, stated, “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.”
“We are excited about these impressive clinical data in Parkinson’s disease,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “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.”
Dr. Forbes continued, “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.”
About AAV-GAD
Parkinson’s disease (PD) is the second most common neurodegenerative disease after Alzheimer’s, with nearly one million people in the U.S. currently living with Parkinson’s disease and approximately 90,000 new patients diagnosed annually in the U.S. There are more than 10 million people worldwide currently living with PD. Most individuals with PD initially respond to dopamine replacement therapy, yet for a large percentage of patients, over time, this type of treatment is no longer sufficiently helpful while adverse effects of medication can also occur, leading to a considerable reduction in quality of life and the ability to function effectively. The cause of Parkinson’s disease is unknown for a majority of patients, while a much smaller percentage have a known genetic cause, but in all cases, there is dysfunction of the key circuits that control movement. AAV-GAD is an investigational gene therapy designed to reprogram these 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. AAV-GAD is delivered via a one-time infusion through a minimally invasive procedure, using a MeiraGTx proprietary device that allows infusion of the equivalent of one drop of gene therapy solution into the subthalamic nucleus, a key regulator of the circuits responsible for normal movement.
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, MeiraGTx has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness, MeiraGTx has core capabilities in viral vector design and optimization and a transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the delivery of metabolic peptides, including GLP-1, GIP, Glucagon, and PYY, using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
stockguard
5 años hace
MeiraGTx (NASDAQ:MGTX) Given Buy Rating at Piper Jaffray Companies
Piper Jaffray Companies restated their buy rating on shares of MeiraGTx (NASDAQ:MGTX) in a research note issued to investors on Monday, Stock Target Advisor reports. Piper Jaffray Companies currently has a $40.00 price target on the stock.
A number of other equities analysts also recently commented on the stock. Zacks Investment Research raised shares of MeiraGTx from a hold rating to a buy rating and set a $17.00 price objective for the company in a report on Wednesday, November 13th. Chardan Capital reissued a buy rating and set a $45.00 target price on shares of MeiraGTx in a report on Monday. Finally, ValuEngine downgraded shares of MeiraGTx from a hold rating to a sell rating in a research report on Tuesday, December 3rd. One investment analyst has rated the stock with a sell rating and four have assigned a buy rating to the company’s stock. MeiraGTx has a consensus rating of Buy and a consensus target price of $33.00.
Shares of MGTX opened at $21.43 on Monday. The firm has a market cap of $791.78 million, a price-to-earnings ratio of -4.88 and a beta of 2.22. The stock has a 50 day moving average of $17.85 and a 200-day moving average of $20.48. The company has a current ratio of 6.90, a quick ratio of 6.90 and a debt-to-equity ratio of 0.11. MeiraGTx has a 52 week low of $8.53 and a 52 week high of $30.23.
MeiraGTx (NASDAQ:MGTX) last issued its quarterly earnings results on Thursday, November 7th. The company reported ($0.30) earnings per share for the quarter, beating the Zacks’ consensus estimate of ($0.52) by $0.22. The firm had revenue of $3.58 million during the quarter. On average, sell-side analysts predict that MeiraGTx will post -2.22 earnings per share for the current fiscal year.
In related news, Director Nicole Seligman bought 5,000 shares of the firm’s stock in a transaction on Tuesday, November 19th. The stock was bought at an average cost of $16.31 per share, for a total transaction of $81,550.00. Following the completion of the purchase, the director now directly owns 5,000 shares in the company, valued at approximately $81,550. The purchase was disclosed in a filing with the Securities & Exchange Commission, which is available through this hyperlink. Company insiders own 17.40% of the company’s stock.
Several institutional investors have recently made changes to their positions in the company. Orbimed Advisors LLC raised its position in shares of MeiraGTx by 29.5% during the 3rd quarter. Orbimed Advisors LLC now owns 3,349,060 shares of the company’s stock worth $53,418,000 after purchasing an additional 762,109 shares during the period. BlackRock Inc. grew its stake in MeiraGTx by 39.6% during the 2nd quarter. BlackRock Inc. now owns 789,792 shares of the company’s stock worth $21,230,000 after buying an additional 224,052 shares during the last quarter. State Street Corp increased its position in MeiraGTx by 19.3% in the third quarter. State Street Corp now owns 318,242 shares of the company’s stock worth $5,076,000 after buying an additional 51,522 shares during the period. Millennium Management LLC increased its position in MeiraGTx by 519.2% in the third quarter. Millennium Management LLC now owns 204,966 shares of the company’s stock worth $3,269,000 after buying an additional 171,866 shares during the period. Finally, JPMorgan Chase & Co. lifted its stake in MeiraGTx by 2,008.5% in the second quarter. JPMorgan Chase & Co. now owns 171,168 shares of the company’s stock valued at $4,338,000 after buying an additional 163,050 shares during the last quarter. Institutional investors and hedge funds own 42.18% of the company’s stock.
About MeiraGTx
MeiraGTx Holdings plc, a clinical-stage gene therapy company, focusing on developing treatments for patients living with serious diseases. The company develops various therapies for ocular diseases, including rare inherited blindness, as well as Xerostomia following radiation treatment for head and neck cancers; neurodegenerative diseases, such as amyothrophic lateral sclerosis; and Parkinson's diseases.
https://newsfilter.io/articles/meiragtx-nasdaqmgtx-given-buy-rating-at-piper-jaffray-companies-73eb5a44157d4419e1c8d7380a1f3fc9
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