Financial Performance and Liquidity
Revenues for the fourth quarter of 2025 reached $30.3 million, an increase from $21.2 million in the prior year period. Full-year 2025 revenues totaled $170.4 million, up from $83.3 million in 2024, primarily driven by $72.9 million in upfront license revenue and $11.8 million in service revenue from the Nippon Shinyaku collaboration, alongside higher Zolgensma and Itvisma royalties. The company reported a fourth-quarter net loss of $67.1 million, or $1.30 per share, compared to a net loss of $51.2 million, or $1.01 per share, in the fourth quarter of 2024. The full-year net loss narrowed to $193.9 million from $227.1 million in the previous year. REGENXBIO ended 2025 with $240.9 million in cash, cash equivalents, and marketable securities, which is projected to fund operations into early 2027.
Operating Expenses
Research and development expenses for the fourth quarter rose to $59.6 million, bringing the full-year total to $228.3 million. This increase was largely attributed to personnel costs and clinical trial expenses associated with the pivotal trials for RGX-202. General and administrative expenses also increased to $22.4 million for the quarter and $82.9 million for the full year, driven by professional services and corporate advisory fees. The company’s full-year loss from operations was $161.2 million.
Pipeline and Operational Developments
The company advanced its Duchenne muscular dystrophy program, reporting that patients treated with the pivotal dose of RGX-202 demonstrated an average improvement of 7.4 points on the North Star Ambulatory Assessment compared to the expected disease trajectory at 18 months. Topline pivotal data for RGX-202 is anticipated in early Q2 2026. In retinal diseases, topline pivotal data for subretinal delivery of sura-vec is expected in Q4 2026. Concurrently, the neurodegenerative pipeline faced regulatory setbacks, with the FDA placing clinical holds on both RGX-111 and RGX-121 following a report of an intraventricular CNS tumor in an RGX-111 trial participant. Additionally, the FDA issued a complete response letter for the RGX-121 Biologics License Application in February 2026.