Group 1 - Rocket Pharmaceuticals is positioned as a compelling gene therapy company due to its focus on therapies that provide a life-long effect, particularly in cardiovascular and hematology areas, contrasting with many gene therapies that have limited durability, especially those targeting the liver [1] - The company may initiate a beneficial long position in its stock or related derivatives within the next 72 hours, indicating potential investor interest and confidence in its future performance [1]

Core Viewpoint - Rocket Pharmaceuticals reported a narrower loss per share in Q4 2024 compared to estimates, but did not generate any revenue due to the absence of marketed products in its portfolio [1][2][7]. Financial Performance - In Q4 2024, Rocket Pharmaceuticals incurred a loss of $0.62 per share, which was better than the Zacks Consensus Estimate of a loss of $0.71 per share and a loss of $0.64 per share in the same quarter last year [1]. - For the full year 2024, the company reported a loss of $2.73 per share, an improvement from a loss of $2.92 in 2023 [7]. - The company did not record any revenues in both Q4 2024 and the full year 2024 [2][7]. Expenses - General and administrative expenses increased by 17.7% year over year to $25.3 million, attributed to higher commercial preparation expenses [3]. - Research and development expenses were $37.4 million, down 10.3% from the previous year, due to reduced manufacturing and development costs [3]. Cash Position - As of December 31, 2024, Rocket Pharmaceuticals had cash, cash equivalents, and investments totaling $372.3 million, up from $235.7 million on September 30, 2024 [4]. - Management anticipates that this cash balance will support operations and capital expenditures through the third quarter of 2026 [4]. Pipeline Developments - The company is developing Kresladi for treating severe leukocyte adhesion deficiency-I (LAD-I), but received a complete response letter from the FDA in June 2024, requesting additional information [8][9]. - Rocket Pharmaceuticals has initiated a rolling biologics license application (BLA) for RP-L102, aimed at treating Fanconi anemia, which is also under review in the EU [10]. - Dosing is ongoing in a phase II study for RP-A501, targeting Danon disease, with updates expected in the first half of 2025 [11]. - RP-A601, another AAV-based gene therapy candidate, is in phase I development for arrhythmogenic cardiomyopathy, with initial data anticipated in the first half of 2025 [12].

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].

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].

Group 1 - The CEO of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), Dr. Gaurav Shah, was interviewed a year ago, marking the last coverage of the company in two years, with the previous report being in February 2023 [1] - The Total Pharma Tracker offers tools for DIY investors, including a software that provides extensive curated research material for any ticker [1] - The company provides hands-on support for investors through in-house experts who identify the best investable stocks along with buy/sell strategies and alerts [2]

A downtrend has been apparent in Rocket Pharmaceuticals (RCKT) lately with too much selling pressure. The stock has declined 12.5% over the past four weeks. However, given the fact that it is now in oversold territory and Wall Street analysts are majorly in agreement about the company's ability to report better earnings than they predicted earlier, the stock could be due for a turnaround.Guide to Identifying Oversold StocksWe use Relative Strength Index (RSI), one of the most commonly used technical indicat ...

Key TakeawaysJefferies initiated a "buy" rating on Rocket Pharmaceuticals based on the biotech firm's experimental gene therapies.Jefferies also set a price target of $29.The analysts said they were especially optimistic about Rocket's treatment for a rare genetic heart condition, which they believe could be a $1 billion opportunity for the company. Shares of Rocket Pharmaceuticals (RCKT) jumped 5.5% intraday Wednesday as Jefferies initiated coverage of the biotech company with a "buy" rating on optimism ab ...

On Monday, Rocket Pharmaceuticals, Inc. RCKT presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.Danon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability.Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock SlidesDanon disease is caused by mutatio ...

Rocket Pharmaceuticals, Inc. (RCKT) incurred a loss of 71 cents per share in the third quarter of 2024, narrower than the Zacks Consensus Estimate of a loss of 78 cents. In the year-ago quarter, the company had reported a loss of 75 cents per share.In the absence of any marketed product in its portfolio, Rocket Pharmaceuticals did not record any revenues during the third quarter.Stay up-to-date with all quarterly releases: See Zacks Earnings Calendar.More on RCKT's Q3 ResultsIn the reported quarter, general ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3475813 (State ...