MeiraGTx(MGTX) - 2024 Q4 - Annual Results

Collaboration and Partnerships - MeiraGTx reported a strategic collaboration with Hologen AI, including a $200 million upfront payment and a joint venture with an additional $230 million committed for the development of AAV-GAD for Parkinson's disease[3]. - The company is eligible to receive up to $285 million upon the first commercial sales of botaretigene sparoparvovec (bota-vec) in the U.S. and EU, along with a commercial supply agreement with Johnson & Johnson[17]. - Sanofi purchased $30 million of ordinary shares in MeiraGTx and holds a right of first negotiation for the use of its riboswitch gene regulation technology[22]. Clinical Developments - Positive data from a clinical study of AAV-GAD showed a significant improvement of 18 points in the Unified Parkinson's Disease Rating Scale (UPDRS) Part 3 in the high dose group at 26 weeks (p=0.03) and improvements in the Parkinson's Disease Questionnaire (PDQ-39) score[8]. - AAV2-hAQP1 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Grade 2/3 radiation-induced xerostomia (RIX), with a potential BLA filing anticipated in 2026[4]. - The efficacy of rAAV8.hRKp.AIPL1 for LCA4 was published in The Lancet, showing meaningful responses in 100% of treated children, with plans to submit a Marketing Authorization Application (MAA) under exceptional circumstances this year[12]. - The company received FDA Rare Pediatric Disease Designations for four therapies targeting rare inherited retinal diseases, including rAAV8.hRKp.AIPL1 for LCA4[4]. Financial Performance - Cash, cash equivalents, and restricted cash decreased to $105.7 million as of December 31, 2024, from $130.6 million as of December 31, 2023, representing a decline of approximately 19%[23]. - Service revenue for the year ended December 31, 2024, was $33.3 million, compared to no service revenue in 2023[23]. - License revenue dropped to $0 for the year ended December 31, 2024, from $14.0 million in 2023, due to the termination of a collaboration agreement[24]. - General and administrative expenses increased to $54.2 million in 2024, up from $47.3 million in 2023, marking an increase of approximately 15%[26]. - Research and development expenses rose to $119.5 million in 2024, compared to $103.8 million in 2023, an increase of about 15%[27]. - Net loss attributable to ordinary shareholders for the year ended December 31, 2024, was $147.8 million, or $2.12 per share, compared to a net loss of $84.0 million, or $1.49 per share in 2023[31]. - Total operating expenses for 2024 were $197.5 million, compared to $151.1 million in 2023, reflecting an increase of approximately 31%[38]. - Gain on sale of nonfinancial assets decreased to $28.4 million in 2024 from $54.2 million in 2023, a decline of about 48%[30]. - Total assets decreased to $269.8 million as of December 31, 2024, from $326.7 million as of December 31, 2023, a reduction of approximately 17%[40]. - Total liabilities increased to $201.9 million as of December 31, 2024, compared to $188.6 million as of December 31, 2023, an increase of about 7%[40]. Manufacturing and Compliance - MeiraGTx plans to submit a commercial MIA license application for its UK manufacturing facility in Q2 2025, following successful inspections and compliance with GMP requirements[16]. - MeiraGTx's Shannon facility in Ireland received a first-of-its-kind license for gene therapy manufacturing, allowing it to produce material for clinical trials[19].