Financial Performance - The company reported a net loss of $65.6 million for the nine months ended September 30, 2025, compared to a net loss of $55.6 million for the same period in 2024, indicating an increase in losses year-over-year [129]. - The company reported no revenue for the three months ended September 30, 2025, and September 30, 2024, and generated $0.9 million in revenue for the nine months ended September 30, 2024 [149]. - The net loss for the three months ended September 30, 2025, was $20.953 million, compared to a net loss of $20.217 million for the same period in 2024, indicating an increase in loss of 3.6% [148]. - The company expects to continue incurring significant operating losses and will require additional capital to fund operations and R&D activities [166]. - For the nine months ended September 30, 2025, the company used $59.7 million in cash for operating activities, reflecting a net loss of $65.6 million [174]. - Cash used in operating activities for the nine months ended September 30, 2024, was $53.1 million, with a net loss of $55.6 million [175]. Research and Development - The company is developing NGN-401 for the treatment of Rett syndrome, with a primary endpoint of a 35% responder rate in the ongoing Embolden trial, which involves 20 participants [109]. - Interim data from the Phase 1/2 trial of NGN-401 showed that all pediatric participants gained a total of 35 developmental milestones/skills across key clinical domains [111]. - The company discontinued the NGN-101 program for CLN5 Batten disease after the RMAT application was denied, indicating a strategic shift in focus [125]. - The Embolden trial is expected to enroll across 13 sites, with completion of enrollment anticipated within three to six months [110]. - The company entered into a Master Research Collaboration with the University of Edinburgh, which was extended through December 2026, to support pipeline development [132]. - The increase in R&D expenses was driven by higher costs in the Rett syndrome program, including a $4.0 million rise in clinical trial costs for NGN-401 [154]. - The company anticipates needing substantial additional capital to support ongoing operations and product development [130]. - The company expects significant increases in research and development expenses over the next several years due to expanded clinical trials and personnel costs [139]. Expenses - Research and development expenses increased to $17.241 million for the three months ended September 30, 2025, compared to $16.285 million for the same period in 2024, reflecting an increase of 5.9% [148]. - Total operating expenses for the three months ended September 30, 2025, were $23.798 million, up from $22.180 million in 2024, representing a year-over-year increase of 7.3% [148]. - General and administrative expenses for Q3 2025 were $6.6 million, a 11.2% increase from $5.9 million in Q3 2024 [157]. - For the nine months ended September 30, 2025, general and administrative expenses reached $21.4 million, up 30.2% from $16.4 million in the same period of 2024 [158]. - The company anticipates that general and administrative expenses will increase to support ongoing R&D activities [159]. Cash and Investments - As of September 30, 2025, the company had an accumulated deficit of $327.9 million and cash, cash equivalents, and short-term investments totaling $265.4 million [129]. - Interest income for the three months ended September 30, 2025, was $2.739 million, an increase from $1.842 million in the same period in 2024 [148]. - Interest income increased by $0.9 million for Q3 2025 compared to Q3 2024, primarily due to a significant increase in cash and short-term investments [160]. - Net cash used in investing activities for the nine months ended September 30, 2025, was $15.6 million, primarily due to purchases of short-term investments totaling $226.8 million [176]. - For the nine months ended September 30, 2025, net cash provided by financing activities was $9.8 million, mainly from open market sales of common stock [178]. Regulatory and Compliance - The FDA granted clearance for the Investigational New Drug (IND) application for NGN-401 in January 2023, and the program was selected for the FDA's START Pilot Program in June 2024 [108][123]. - All treatment-related adverse events in the Phase 1/2 trial of NGN-401 were Grade 1 or Grade 2 in severity, with no evidence of hemophagocytic lymphohistiocytosis (HLH) reported at the 1E15 vg dose level [121][122]. - The company has federal and state net operating loss carryforwards of $319.8 million and $39.6 million, respectively, which may offset future taxable income [146]. - The company recorded a total contingent value rights (CVR) liability of $638,000 as of September 30, 2025, compared to $1.1 million as of December 31, 2024 [188]. - The company does not have any off-balance sheet arrangements as defined by SEC rules [195]. Facilities and Lease Obligations - The company established a fully operational cGMP facility in Houston, Texas, to manufacture products for research and clinical studies, enhancing control over product quality and development timelines [126]. - The company has a lease obligation for its New York headquarters expiring in January 2028, covering approximately 6,000 square feet [181]. - The Houston manufacturing facility lease, covering 42,342 square feet, expires in August 2029, with options for two additional five-year terms [182]. - The company has entered into a License Agreement with the University of Edinburgh, which includes potential milestone payments of up to $30.3 million based on regulatory and sales achievements [191].
Aquinox(NLTX) - 2025 Q3 - Quarterly Report