REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights

Core Insights - REGENXBIO Inc. reported significant progress in its late-stage gene therapy programs, highlighting advancements in treatments for Duchenne muscular dystrophy, Hunter syndrome, wet AMD, and diabetic retinopathy [2][3]. Financial Performance - Cash, cash equivalents, and marketable securities totaled $302.0 million as of September 30, 2025, an increase from $244.9 million at the end of 2024, primarily due to a $110.0 million upfront payment from Nippon Shinyaku and $144.5 million from royalty monetization [7]. - Revenues for Q3 2025 were $29.7 million, up from $24.2 million in Q3 2024, driven by $5.9 million in development service revenue from the Nippon Shinyaku partnership [8]. - Research and development expenses rose to $56.1 million in Q3 2025 from $54.4 million in Q3 2024, mainly due to increased personnel and manufacturing costs [9]. - General and administrative expenses increased to $20.3 million in Q3 2025 from $19.4 million in Q3 2024, attributed to professional services and consulting [10]. - The net loss for Q3 2025 was $61.9 million, compared to a net loss of $59.6 million in Q3 2024, resulting in a basic and diluted net loss per share of $1.20 [11]. Program Highlights - RGX-202, a gene therapy for Duchenne muscular dystrophy, is advancing rapidly with topline results expected in early Q2 2026 and a Biologics License Application (BLA) submission planned for mid-2026 [5][6]. - Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy for Hunter syndrome, with a PDUFA date set for February 8, 2026 [4][12]. - Surabgene lomparvovec (sura-vec) is positioned to be the first gene therapy for chronic retinal diseases, with pivotal trial enrollment completed and topline data expected in Q4 2026 [5][7]. Operational Developments - Enrollment in the pivotal trial for RGX-202 was completed in October 2025, with ongoing confirmatory studies for ambulatory patients [6]. - REGENXBIO has begun manufacturing RGX-202 for commercial supply, anticipating a launch in 2027 [6]. - The company presented positive 12-month data for RGX-121, showing significant biomarker reductions and correlations with neurodevelopmental outcomes [12]. Future Outlook - REGENXBIO expects its cash position to fund operations into early 2027, excluding potential milestone payments from partners [13]. - The company is preparing for a conference call to discuss these results and operational highlights [14].