Rocket Pharmaceuticals(RCKT) - 2024 Q4 - Annual Results

Financial Performance - Rocket Pharmaceuticals reported a net loss of $258.7 million or$2.73 per share for the year ended December 31, 2024, compared to a net loss of $245.6 million or$2.92 per share for 2023[11]. - Research and development expenses decreased to $171.2 million in 2024 from$186.3 million in 2023, primarily due to a reduction in manufacturing and development costs[11]. - General and administrative expenses increased to $102.0 million in 2024 from$73.3 million in 2023, driven by higher commercial preparation and legal expenses[11]. - As of December 31, 2024, Rocket had cash, cash equivalents, and investments of approximately $372.3 million, expected to fund operations into the third quarter of 2026[10]. - The company raised net proceeds of$182.5 million through a public offering of approximately 15.2 million shares at a price of $12.50 per share[8]. Clinical Development - Dosing is ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease, with a program update anticipated in the first half of 2025[4]. - Initial data from the Phase 1 study of RP-A601 for PKP2-ACM is expected in the first half of 2025 after completing enrollment in the low dose cohort[7]. - Rocket is progressing with the rolling Biologics License Application (BLA) for RP-L102 for Fanconi Anemia, with final module submission anticipated in 2025[7]. - The Phase 1 study of RP-A501 demonstrated safety and meaningful efficacy, with all evaluable patients showing a ≥10% reduction in LV mass index at 12 months[7]. Regulatory Affairs - Regulatory reviews are ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia) with the FDA requesting additional Chemistry Manufacturing and Controls (CMC) information[7].