MeiraGTx(US:MGTX)2024-11-13 13:08Regulatory Designations and Applications - MeiraGTx received three Rare Pediatric Disease Designations (RPDD) from the FDA for its AAV8-RK-AIPL1, AAV8-RK-BBS10, and AAV5-RDH12 programs, each targeting inherited retinal diseases[1]. - The company plans to submit a Marketing Authorization Application (MAA) for AAV-AIPL1 in the UK under exceptional circumstances, with discussions ongoing with the FDA for a similar pathway in the U.S.[1][6]. Clinical Study Results - In a randomized, sham-controlled clinical study of AAV-GAD for Parkinson's disease, a statistically significant 18-point average improvement in UPDRS Part 3 "off" score was observed in the high dose group at Week 26 (p=0.03)[4]. - The PDQ-39 quality of life score improved by 8 points in the high dose AAV-GAD group (p=0.02) and by 6 points in the low dose group (p=0.04), with no significant change in the sham group[5]. Financial Performance - As of September 30, 2024, MeiraGTx had cash and cash equivalents of approximately $122.9 million, down from $130.6 million as of December 31, 2023[11][12]. - Service revenue for the three months ended September 30, 2024, was $10.9 million, attributed to process performance qualification services under the asset purchase agreement with Johnson & Johnson[12]. - Research and development expenses for the quarter were $26.2 million, a decrease from $27.9 million in the same period last year, primarily due to reduced manufacturing costs[16]. - The net loss attributable to ordinary shareholders for the quarter was $39.3 million, or $0.55 per share, an improvement from a net loss of $44.3 million, or $0.74 per share, in the prior year[20]. - Foreign currency gain was $3.5 million for the quarter, a significant improvement compared to a loss of $8.7 million in the same period last year, reflecting a change of $12.1 million[17]. - Total revenue for the three-month period ended September 30, 2024, was $10.91 million, compared to $5.10 million for the same period in 2023, representing a 114% increase[27]. - Research and development expenses for the three-month period ended September 30, 2024, were $26.24 million, slightly down from $27.86 million in the same period of 2023, a decrease of 5.8%[27]. - The net loss for the three-month period ended September 30, 2024, was $39.33 million, an improvement from a net loss of $44.30 million in the same period of 2023, reflecting an 11% reduction in losses[27]. - Total current assets decreased to $139.05 million as of September 30, 2024, down from $159.62 million at the end of December 31, 2023, a decline of 12.9%[28]. - Total liabilities increased to $203.75 million as of September 30, 2024, compared to $188.57 million at the end of December 31, 2023, an increase of 8%[28]. - The company reported cash and cash equivalents of $122.87 million as of September 30, 2024, down from $129.57 million at the end of December 31, 2023, a decrease of 5.2%[28]. - The weighted-average number of ordinary shares outstanding increased to 71,633,150 for the three-month period ended September 30, 2024, compared to 59,526,642 for the same period in 2023, an increase of 20.3%[27]. Research and Development Focus - The company has developed a proprietary manufacturing platform aimed at increasing yield and quality, with a focus on gene regulation technologies[24]. - MeiraGTx is focusing on expanding its riboswitch platform for metabolic peptides and cell therapy applications in oncology and autoimmune diseases[24]. - The company anticipates significant growth opportunities in addressing unmet needs in common diseases through its genetic medicine technologies[24].