Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates

Core Insights - Neurogene reported positive interim data from the NGN-401 Phase 1/2 gene therapy trial for Rett syndrome, showing multidomain, durable gains with 35 developmental milestones acquired across eight participants [1][5] - The first participant has been dosed in the Embolden registrational trial of NGN-401, with complete enrollment expected in three to six months [1][2] - The company maintains a strong cash position, with $265.4 million in cash and equivalents, providing a runway through the first quarter of 2028 [11] Clinical Development Highlights - The NGN-401 gene therapy program for Rett syndrome has advanced significantly, with 12 of 13 trial sites initiated [2][5] - Interim clinical data from the pediatric cohort (ages 4-10) demonstrated functional improvements across all participants, with gains in hand function, language, and ambulation [5] - The therapy has been well-tolerated in both pediatric and adolescent/adult cohorts, with no significant adverse effects reported [5] Financial Results - For the third quarter of 2025, R&D expenses were $17.2 million, up from $16.3 million in the same period in 2024, primarily due to increased clinical trial costs [11] - General and administrative expenses rose to $6.6 million from $5.9 million year-over-year, driven by higher employee-related costs [11] - The net loss for the quarter was $21.0 million, compared to $20.2 million in the prior year [11] Regulatory Designations - NGN-401 has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designation, indicating its potential as a significant treatment for Rett syndrome [8]