REGENXBIO Reports Second Quarter 2025 Financial Results and Operational Highlights

Core Insights - REGENXBIO Inc. reported strong operational momentum in advancing its gene therapy programs, particularly with RGX-121 and RGX-202, aimed at treating serious diseases such as Duchenne muscular dystrophy and Hunter syndrome [2][4][5] Financial Results - Cash, cash equivalents, and marketable securities increased to $363.6 million as of June 30, 2025, from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from Nippon Shinyaku and $144.5 million from royalty monetization [16][21] - Revenues for the second quarter of 2025 were $21.4 million, a slight decrease from $22.3 million in the same period of 2024, mainly due to a drop in Zolgensma royalties [17] - Research and development expenses rose to $59.5 million in Q2 2025 from $48.9 million in Q2 2024, driven by manufacturing and clinical trial costs [18] - General and administrative expenses increased to $19.9 million in Q2 2025 from $18.9 million in Q2 2024, attributed to personnel and consulting costs [19] - The net loss for Q2 2025 was $70.9 million, compared to a net loss of $53.0 million in Q2 2024, reflecting increased operational costs [20] Program Highlights - RGX-202 is positioned as a potential best-in-class gene therapy for Duchenne muscular dystrophy, with pivotal trial enrollment expected to complete by October 2025 [3][5] - Clemidsogene lanparvovec (RGX-121) is on track for potential FDA approval in November 2025, with successful completion of FDA inspections [4][13] - Surabgene lomparvovec (sura-vec) is advancing towards pivotal trials for diabetic retinopathy and wet AMD, with topline results expected in 2026 [7][9] Corporate Updates - In May 2025, REGENXBIO closed a $250 million royalty bond agreement, receiving $150 million upfront [11] - An amendment to the collaboration agreement with AbbVie was executed in August 2025, which includes milestone payments for the diabetic retinopathy program [14] Financial Guidance - The company expects its cash position to fund operations into early 2027, excluding potential payments from partners or monetization of a Priority Review Voucher [21]