MeiraGTx Reports $147.8 Million Net Loss for 2024, Highlights Clinical Advancements and Strategic Collaborations

MeiraGTx Holdings plc, a clinical-stage genetic medicines company, reported net losses of $147.8 million for the year ended December 31, 2024, compared to $84.0 million in 2023. Total operating expenses increased to $197.5 million in 2024 from $151.1 million in 2023, primarily due to increased research and development costs and expenses associated with being a public company. The company attributed the increased expenses to advancements in clinical trials for several product candidates, including AAV-hAQP1 for xerostomia and AAV-GAD for Parkinson's disease, as well as expansion of research activities and internal manufacturing capabilities. The company had an accumulated deficit of approximately $702.0 million as of December 31, 2024.

Significant developments during 2024 included the completion of several Phase 1/2 clinical trials for inherited retinal diseases (IRDs), including AAV-CNGB3 and AAV-CNGA3 for achromatopsia and AAV-RPE65 for RPE65-associated retinal dystrophy. The company also completed enrollment and dosing in a Phase 1 bridging study for AAV-GAD in Parkinson's disease, reporting positive preliminary safety and efficacy data. In the area of xerostomia, MeiraGTx initiated a Phase 2 clinical trial (AQUAx2) for AAV-hAQP1, and received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Furthermore, the company entered into a strategic collaboration with Sanofi, receiving $30 million in funding and granting Sanofi a right of first negotiation for its riboswitch gene regulation technology. A significant transaction with Johnson & Johnson Innovative Medicine involved the sale of assets related to the RPGR product for an upfront payment of $65 million and potential future milestone payments totaling $350 million.

Operational highlights included the continued operation of GMP manufacturing facilities in London and Shannon, which produced GMP clinical trial material for eight different indications. The company also reported receiving various regulatory authorizations for its manufacturing facilities, including a Manufacturer’s/Importer’s Authorization (MIA) for QC testing in Shannon. As of December 31, 2024, MeiraGTx employed 381 people, 375 of whom were full-time employees. The company's pipeline includes clinical and preclinical programs targeting various diseases, including additional IRDs, neurodegenerative diseases, and salivary gland disorders, leveraging its proprietary riboswitch technology platform.

In a separate major transaction completed in early 2025, MeiraGTx entered into a strategic collaboration with Hologen AI, receiving $200 million in upfront cash consideration and forming a joint venture to further develop its AAV-GAD program for Parkinson's disease. This joint venture, Hologen Neuro AI Ltd., will receive up to an additional $230 million in funding from Hologen. MeiraGTx will retain a 30% ownership stake in the joint venture and lead clinical development and manufacturing.

Looking ahead, MeiraGTx anticipates continued losses for the foreseeable future and will require additional capital to fund operations. The company expects its cash resources to be sufficient to fund operations into 2027, but this is contingent upon several factors, including the receipt of milestone payments from Johnson & Johnson Innovative Medicine and the successful completion of its clinical development programs. The company plans to submit a Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 in the UK and is engaging with the FDA to discuss a path forward for regulatory approval in the U.S. The company also plans to submit an application for a commercial MIA license for its UK manufacturing facility in the second quarter of 2025.