LONDON and NEW YORK - MeiraGTx Holdings plc (NASDAQ:MGTX), a clinical-stage genetic medicines company, announced today that its gene therapy product AAV-GAD has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of Parkinson’s disease.
The RMAT designation, which expedites the development and review of promising therapies for serious conditions, was based on positive outcomes from three clinical studies. These studies showed AAV-GAD’s potential benefits when administered as a one-time treatment for Parkinson’s disease patients whose symptoms are not adequately controlled with current medications.
Dr. Alexandria Forbes, President and CEO of MeiraGTx, expressed excitement about the designation, emphasizing the clinically meaningful benefits observed in motor function and other Parkinson’s symptoms. Dr. Forbes also highlighted the significant findings from their partnership with Hologen, which applied AI technology to the Phase 2 study data, revealing potential disease-modifying changes in patients’ brain circuitry.
AAV-GAD is designed to target dysfunctional brain circuits by locally producing GABA, a neurotransmitter, through a minimally invasive procedure. The therapy aims to restore normal activity in the subthalamic nucleus, a region implicated in controlling movement.
The RMAT designation is a testament to the therapy’s potential in addressing the unmet needs of Parkinson’s disease, a serious neurodegenerative disorder affecting nearly one million people in the U.S. alone. It includes benefits similar to Fast Track and Breakthrough Therapy designations, such as increased FDA interaction and potential for accelerated approval and Priority Review.
MeiraGTx’s commitment to advancing genetic medicine is reflected in its broad pipeline and in-house manufacturing capabilities, which span across several late-stage clinical programs targeting inherited and common diseases.
This news is based on a press release statement and is intended to provide information on MeiraGTx’s recent regulatory milestone. It does not constitute an endorsement of the company or its products.
In other recent news, MeiraGTx Holdings has announced a strategic collaboration with Hologen Limited, resulting in a $200 million upfront payment and the formation of a joint venture, Hologen Neuro AI Ltd. This partnership, which aims to advance the development of treatments for Parkinson’s disease and other central nervous system disorders, includes an additional $230 million from Hologen to support the AAV-GAD program through commercialization. Concurrently, MeiraGTx has secured a new lease for expanded laboratory and office space in New York City, while terminating an existing sublease. On the financial front, RBC Capital Markets raised its price target for MeiraGTx to $13.00, maintaining an Outperform rating, reflecting confidence in the company’s strategic advancements and partnerships. Piper Sandler also reiterated an Overweight rating, with a $26.00 price target, citing upcoming catalysts like pivotal data for xerostomia treatment and the potential commercialization of botavec by Johnson & Johnson™. The company’s collaboration with Hologen AI is expected to enhance manufacturing processes and clinical development, with MeiraGTx retaining a 30% stake in the joint venture. These developments mark significant steps in MeiraGTx’s growth, showcasing its robust pipeline and strategic initiatives.
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