Core Insights - MeiraGTx Holdings plc announced its financial and operational results for Q3 2025, highlighting significant advancements in its gene therapy programs and strategic collaborations, particularly with Eli Lilly in ophthalmology [1][2][3] Financial Performance - As of September 30, 2025, the company reported cash and cash equivalents of approximately $14.8 million, a decrease from $105.7 million as of December 31, 2024 [13] - Service revenue for Q3 2025 was $0.4 million, down from $10.9 million in Q3 2024, primarily due to decreased activity related to the asset purchase agreement with Johnson & Johnson [14] - General and administrative expenses increased to $13.6 million in Q3 2025 from $12.7 million in Q3 2024, attributed to higher rent, consulting fees, and share-based compensation [16] - Research and development expenses rose to $32.5 million in Q3 2025 from $26.2 million in Q3 2024, driven by increased manufacturing costs and clinical program activities [17] - The net loss attributable to ordinary shareholders for Q3 2025 was $50.5 million, or $0.62 per share, compared to a net loss of $39.3 million, or $0.55 per share, in Q3 2024 [21] Strategic Collaborations - The company entered a strategic collaboration with Eli Lilly, granting exclusive rights to its AAV-AIPL1 program for treating Leber congenital amaurosis 4 (LCA4), along with access to other gene therapy technologies [2][3][8] - Under the collaboration, MeiraGTx will receive an upfront payment of $75 million and is eligible for over $400 million in milestone payments, along with tiered royalties on licensed products [8] Clinical Development - The pivotal Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia is on track for target enrollment by the end of 2025, with potential BLA filing expected in early 2027 [2][8] - A Phase 3 study for AAV-GAD in Parkinson's disease is anticipated to start soon, following positive data from previous studies [2][8] - The company has optimized its riboswitch program for delivering native human leptin, addressing a significant unmet need in leptin deficiency treatment [2][8] Recent Developments - MeiraGTx has developed a second Specials program in the UK for treating BBS10-associated retinal dystrophy, which has received Rare Pediatric Disease Designation from the FDA [2][3] - The company is forming a joint venture with Hologen AI, which includes a $200 million upfront payment and additional funding for developing the AAV-GAD program [11][12]

Core Insights - MeiraGTx Holdings plc has made significant progress in its clinical programs, including alignment with the FDA on the Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia, which is expected to support a Biologics License Application (BLA) filing by late 2026 [1][2] - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD, a gene therapy for Parkinson's disease, indicating its potential to address serious conditions [1][5] - The company is preparing to file for Marketing Authorization Approval (MAA) in the UK and BLA in the US for AAV-AIPL1, targeting LCA4, with submissions expected in Q4 2025 [1][2] Regulatory Developments - The company has had productive interactions with the FDA and MHRA regarding multiple late-stage clinical programs and manufacturing processes [2] - AAV-GAD has shown significant disease-modifying effects in clinical trials, with plans to initiate a Phase 3 study later this year [1][6] - The FDA has completed a Good Clinical Practice inspection of the AAV-GAD bridging study, resulting in a clean inspection with no observations [6][8] Financial Performance - As of June 30, 2025, the company reported cash and cash equivalents of approximately $32.2 million, a decrease from $101.0 million a year earlier [18][19] - Service revenue increased to $3.7 million for Q2 2025, up from $0.3 million in Q2 2024, attributed to progress in process performance qualification services [19][22] - The net loss attributable to ordinary shareholders for Q2 2025 was $38.8 million, compared to a net loss of $48.6 million in Q2 2024, indicating an improvement in financial performance [28][32] Clinical Programs and Collaborations - The company is advancing its late-stage clinical programs, including AAV-hAQP1 for xerostomia and AAV-GAD for Parkinson's disease, with pivotal data readouts expected in late 2026 [1][2] - MeiraGTx is collaborating with Hologen to develop AAV-GAD, with a joint venture established to finance the program and utilize AI for data analysis [6][8] - The company is also preparing to initiate first-in-human studies using its riboswitch platform by the end of 2025 [2][20] Manufacturing and Technology - MeiraGTx has developed comprehensive in-house manufacturing capabilities, with facilities in the UK and Ireland holding necessary regulatory authorizations [15][17] - The company is focusing on optimizing its riboswitch gene regulation technology for various therapeutic applications, including metabolic diseases and chronic pain [29][30] - The manufacturing process for AAV2-hAQP1 will be in-house, ensuring consistency across different indications [5][8]