REGENXBIO(RGNX) - 2024 Q4 - Annual Results

Financial Performance - Revenues for Q4 2024 were $21.2 million, a decrease of 4.5% from $22.2 million in Q4 2023; full-year revenues were $83.3 million, down 7.7% from $90.2 million in 2023[13]. - Net loss for Q4 2024 was $51.2 million, or $1.01 per share, compared to a net loss of $62.9 million, or $1.43 per share, in Q4 2023; full-year net loss was $227.1 million, or $4.59 per share, down from $263.5 million, or $6.02 per share, in 2023[16]. - Total revenues for the year ended December 31, 2024, were $83,328,000, a decrease of 7.5% compared to $90,242,000 for the year ended December 31, 2023[25]. - Net loss for the year ended December 31, 2024, was $227,102,000, compared to a net loss of $263,494,000 for the year ended December 31, 2023, representing a 13.8% improvement[25]. - The company reported a comprehensive loss of $223,414,000 for the year ended December 31, 2024, compared to a comprehensive loss of $252,522,000 for the year ended December 31, 2023, indicating a 11.5% improvement[25]. - The company reported a loss from operations of $233,346,000 for the year ended December 31, 2024, compared to a loss of $268,128,000 for the year ended December 31, 2023, reflecting a 13.0% improvement[25]. Cash and Assets - Cash, cash equivalents, and marketable securities totaled $244.9 million as of December 31, 2024, down from $314.1 million as of December 31, 2023, primarily due to cash used for operating activities[11]. - Cash and cash equivalents increased to $57,526,000 as of December 31, 2024, compared to $34,522,000 as of December 31, 2023, an increase of 66.6%[24]. - Total assets decreased to $465,989,000 as of December 31, 2024, from $573,970,000 as of December 31, 2023, a decline of 18.8%[24]. - Total liabilities decreased to $206,338,000 as of December 31, 2024, down from $262,228,000 as of December 31, 2023, a reduction of 21.4%[24]. Research and Development - Research and development expenses for Q4 2024 were $50.4 million, down from $55.7 million in Q4 2023; full-year expenses were $208.5 million, down from $232.3 million in 2023[14]. - Research and development expenses for the year ended December 31, 2024, were $208,522,000, down 10.2% from $232,266,000 in 2023[25]. - The pivotal trial for RGX-202 is nearly 50% enrolled, with completion expected in 2025 and a BLA filing anticipated in mid-2026[7]. - The pivotal data for the retinal franchise, including ABBV-RGX-314, is expected in 2026, with ongoing planning for diabetic retinopathy studies[6]. Strategic Partnerships - The strategic partnership with Nippon Shinyaku includes an upfront payment of $110 million and up to $700 million in milestone payments for RGX-121 and RGX-111[12]. - REGENXBIO submitted a BLA for clemidsogene lanparvovec (RGX-121) in March 2025, with potential FDA approval expected in the second half of 2025[6]. - REGENXBIO expects its cash runway to extend into the second half of 2026, supported by the $110 million upfront payment from Nippon Shinyaku[17]. Operating Expenses - General and administrative expenses for Q4 2024 were $20.1 million, an increase from $19.1 million in Q4 2023; full-year expenses were $76.6 million, down from $88.5 million in 2023[15]. - Operating expenses for the year ended December 31, 2024, totaled $316,674,000, a decrease of 11.6% from $358,370,000 in 2023[25]. - The weighted-average common shares outstanding increased to 49,509,000 for the year ended December 31, 2024, from 43,734,000 in 2023, an increase of 13.2%[25].