Rocket Pharmaceuticals(RCKT) - 2024 Q4 - Annual Report

Financial Performance - The net loss for the year ended December 31, 2024, was $258.7 million, compared to a net loss of $245.6 million in 2023, reflecting an increase of $13.1 million [426]. - Net cash used in operating activities was $209.7 million for the year ended December 31, 2024, compared to $194.9 million in 2023 [432]. - The company reported cash, cash equivalents, and investments of $372.3 million as of December 31, 2024, down from $407.5 million in 2023 [455]. - The company has an accumulated deficit of $1.22 billion and cash, cash equivalents, and investments totaling $372.3 million as of December 31, 2024 [426]. Research and Development - Total research and development expenses for the year ended December 31, 2024, were $171.2 million, a decrease from $186.3 million in 2023 [407]. - Direct R&D expenses for Danon disease (RP-A501) were $23.7 million in 2024, down from $29.0 million in 2023 [407]. - Research and development (R&D) expenses decreased by $15.1 million to $171.2 million for the year ended December 31, 2024, compared to $186.3 million in 2023, primarily due to decreases in manufacturing development and direct material costs [417]. - The company has two clinical-stage and one pre-clinical stage in vivo adeno-associated viral (AAV) programs, including ongoing trials for Danon disease and Plakophilin-2 Arrhythmogenic Cardiomyopathy [393]. - The company received $2.3 million in grants from CIRM for the clinical development of its AAV-based gene therapy, RP-A501, which were recorded as a reduction of R&D expenses [427]. - The FDA accepted the Biologics License Application (BLA) for RP-L201 and granted priority review, with a complete response letter anticipated in 2025 [395]. - The company has received FDA clearance for an investigational new drug (IND) application for RP-A601 and has initiated a Phase 1 study for this program [394]. Financing Activities - The company has raised net cash proceeds of approximately $1.2 billion from investors through equity and convertible debt financing since inception [400]. - The company sold a total of 4.2 million shares under the at-the-market offering program for gross proceeds of $65.8 million, with net proceeds of $63.8 million [398]. - The company completed a public offering of approximately 15.2 million shares at a price of $12.50 per share, generating net proceeds of $182.5 million [399]. - Net cash provided by financing activities was $185.7 million for the year ended December 31, 2024, primarily from a public offering that generated $182.5 million [439]. General and Administrative Expenses - The company anticipates an increase in general and administrative expenses due to expected headcount growth and increased operational complexity [413]. - General and administrative (G&A) expenses increased by $28.6 million to $102.0 million for the year ended December 31, 2024, driven by increases in commercial preparation related expenses of $17.6 million and legal expenses of $4.8 million [418]. Other Financial Information - Total other income increased by $0.4 million to $14.5 million for the year ended December 31, 2024, primarily due to an increase in interest and other income, net, of $3.0 million [419]. - Net cash provided by investing activities was $131.7 million for the year ended December 31, 2024, primarily from proceeds of $383.5 million from the maturities of investments [436]. - The company expects its current resources to be sufficient to fund operating expenses and capital expenditure requirements into the third quarter of 2026 [426]. Impairment and Assets - The company has determined there was no goodwill impairment for the years ended December 31, 2024, 2023, and 2022 [445]. - The company performed an annual impairment assessment for IPR&D assets as of December 1, with no impairment recorded for the years ended December 31, 2024 and 2023 [446]. - The company’s intangible assets include IPR&D assets, which are considered indefinite-lived until regulatory approval is obtained [446]. - The company’s accrued R&D expenses are estimated based on open contracts and purchase orders, with adjustments made as actual costs are incurred [447]. Market Sensitivity - If market interest rates were to increase by 100 basis points, the net fair value of the company's interest-sensitive marketable securities would hypothetically decline by $0.8 million [455]. - The company’s investments are primarily in U.S. Treasury Securities and Corporate Bonds, exposing it to interest rate sensitivity [455]. Stock-Based Compensation - The company’s stock-based compensation expense is recognized over the requisite service period, with performance-based PSUs recognized when performance goals are likely to be achieved [449]. Segment Reporting - The company adopted ASU 2023-07, which requires expanded disclosures for significant segment expenses effective for fiscal years beginning after December 15, 2023 [454].