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MeiraGTx Holdings plc (MGTX+2.63%) has filed its Form 10-K filing for the fiscal year ended December 31, 2024.
The company is a clinical-stage genetic medicines firm with a focus on gene therapy. It has a broad pipeline of late-stage clinical programs targeting conditions such as Parkinson's disease, radiation-induced xerostomia, and inherited retinal dystrophies.
MeiraGTx operates manufacturing facilities in London, UK, and Shannon, Ireland, which are expected to supply current clinical and preclinical programs and meet potential commercial production needs.
The company is developing a gene regulation platform using synthetic riboswitch technology for precise gene expression control. This platform is being applied to biologic therapeutics, cell therapy for oncology and autoimmune diseases, and long-term intractable pain.
MeiraGTx has a strategic collaboration with Johnson & Johnson Innovative Medicine for the research, development, and commercialization of gene therapies for inherited retinal diseases. An upfront payment of $65 million was received, with potential future contingent consideration of up to $350 million.
In October 2023, MeiraGTx entered into an Investment Agreement with Sanofi Foreign Participations, issuing 4 million ordinary shares for $30 million. Sanofi has a right of first negotiation for the use of MeiraGTx's riboswitch gene regulation technology for certain targets.
The company is developing AAV-hAQP1 for the treatment of radiation-induced xerostomia (RIX) and Sjogren’s syndrome, both of which are conditions associated with impaired saliva production. The product is currently undergoing a Phase 1 dose escalation clinical trial in the United States.
MeiraGTx is also developing AAV-GAD for the treatment of Parkinson’s disease. A Phase 2 clinical trial has shown improvements in motor scores and patient-reported quality of life measures. The product was observed to be well-tolerated with no treatment-related serious adverse events.
The company is also developing AAV-RPE65 for the treatment of retinal dystrophy associated with mutations in the RPE65 gene. The FDA has granted orphan drug designation to AAV-RPE65 for the treatment of Leber congenital amaurosis caused by mutations in the RPE65 gene.
MeiraGTx has developed a potentially transformative gene regulation platform using synthetic riboswitch technology, which allows for the precise expression of any transgene under the control of oral small molecules. This technology is being applied to the delivery of metabolic peptides and cell therapy for various diseases.
The company owns and operates GMP manufacturing facilities in London, UK, and Shannon, Ireland, which are expected to supply current clinical and preclinical programs as well as third-party supply obligations through regulatory approval and potential commercial production.
MeiraGTx has received orphan drug designations from the FDA and the European Commission for several of its gene therapy product candidates. However, there is no guarantee that these designations will lead to faster development or regulatory review or approval.
The company is subject to extensive regulation by governmental authorities in the U.S., UK, EU, and other jurisdictions, which govern the research, development, testing, manufacture, labeling, packaging, promotion, storage, advertising, distribution, marketing, post-approval monitoring and reporting, and export and import of products.
MeiraGTx has incurred significant losses since its inception and anticipates continued losses for the foreseeable future. Additional capital will be required to fund operations, and there is no guarantee that such capital will be available on acceptable terms.
The regulatory landscape for gene therapy products is complex and evolving, making it difficult to predict the time and cost of obtaining regulatory approval for product candidates. Adverse developments in preclinical studies or clinical trials conducted by others in the field of gene therapy and gene regulation products may also impact the company's regulatory approval process.
The company has received several regulatory designations, including orphan drug designation from the FDA and European Commission for multiple product candidates. However, these designations do not guarantee marketing exclusivity or faster regulatory review or approval.
MeiraGTx's strategy includes completing clinical development and obtaining regulatory approval for its pipeline of gene therapy candidates, advancing its preclinical pipeline, and pursuing strategic collaborations with biotechnology and pharmaceutical companies to leverage its capabilities, manufacturing capacity, and proprietary gene regulation technology.
This content was summarized by generative artificial intelligence using public filings retrieved from SEC.gov. The original data was derived from the MeiraGTx Holdings plc annual 10-K report dated March 13, 2025. To report an error, please email earnings@qz.com.