MeiraGTx Reports Third Quarter 2025 Financial and Operational Results

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]