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]

Core Viewpoint - Rocket Pharmaceuticals (RCKT) has experienced a significant downtrend, with a 12.5% decline over the past four weeks, but it is now in oversold territory, suggesting a potential turnaround due to improved earnings expectations from analysts [1]. Group 1: Stock Performance and Technical Indicators - The stock's Relative Strength Index (RSI) reading is at 26.81, indicating that the heavy selling pressure may be exhausting, which could lead to a price rebound [5]. - Stocks oscillate between overbought and oversold conditions, and the RSI helps identify potential reversal points, making it a useful tool for investors seeking entry opportunities [3]. Group 2: Earnings Estimates and Analyst Consensus - Over the last 30 days, the consensus EPS estimate for RCKT has increased by 8.5%, reflecting strong agreement among analysts regarding improved earnings for the current year [6]. - RCKT holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, indicating a strong potential for a near-term turnaround [7].

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 ...

Regulatory Approvals and Clinical Trials - The FDA accepted the BLA and granted priority review for RP-L201 for the treatment of severe LAD-I, with approval expected in 2025[97]. - Treatments in the FA Phase 2 studies were completed in 2023, and a BLA submission was initiated on September 26, 2024[97]. - The company has received FDA clearance for an IND for RP-A601 and has initiated a Phase 1 study for this program[99]. - The company is in the process of providing additional requested information to the FDA regarding RP-L201 following a CRL issued in June 2024[97]. - The Phase 1 study for RP-A601 targeting PKP2-ACM has been initiated, assessing safety and preliminary efficacy in at least six adult patients with ICDs[125]. - The global Phase 2 pivotal trial of RP-A501 for DD will evaluate efficacy and safety in 12 patients, with a primary endpoint focused on improvements in LAMP2 protein expression and reductions in left ventricular mass[119]. - The company has reached alignment with the FDA on the global Phase 2 pivotal trial, which includes a pediatric safety run-in[119]. - The ongoing Phase 2 study's primary endpoint is to achieve bone marrow restoration exceeding a 10% mitomycin-C resistance threshold[135]. Financial Performance and Expenses - R&D expenses decreased by $4.5 million to $42.3 million for the three months ended September 30, 2024, compared to $46.8 million for the same period in 2023[170]. - Total operating expenses increased by $3.995 million to $69.424 million for the three months ended September 30, 2024, compared to $65.429 million for the same period in 2023[169]. - General and administrative expenses increased by $8.5 million to $27.1 million for the three months ended September 30, 2024, compared to $18.6 million for the same period in 2023[171]. - R&D expenses decreased by $10.7 million to $133.9 million for the nine months ended September 30, 2024, compared to $144.6 million for the same period in 2023[173]. - General and administrative expenses increased by $24.8 million to $76.6 million for the nine months ended September 30, 2024, compared to $51.8 million for the same period in 2023[174]. - The company reported a net loss of $66.719 million for the three months ended September 30, 2024, compared to a net loss of $61.899 million for the same period in 2023[169]. - The company incurred net losses of $198.4 million for the nine months ended September 30, 2024, compared to $245.6 million for the year ended December 31, 2023[178]. - As of September 30, 2024, the accumulated deficit was $1.16 billion, up from $959.4 million as of December 31, 2023[178]. - Cash used in operating activities was $162.8 million for the nine months ended September 30, 2024, compared to $165.6 million for the same period in 2023[180]. - The company had $235.7 million in cash, cash equivalents, and investments as of September 30, 2024[178]. - Future viability depends on generating cash from operations or raising additional capital, which may dilute existing stockholders[179]. - The company has not generated any revenue and has incurred losses since inception, with significant R&D efforts required for drug candidates[176]. - The company experienced negative cash flows from operations each year since inception[178]. Product Development and Efficacy - RP-A501 is currently in a Phase 1 clinical trial for Danon Disease, with seven patients treated and ongoing assessments of efficacy[110]. - The Phase 1 study of RP-A501 showed positive efficacy updates, with improvements in key biomarkers and functional measures reported[114]. - As of the latest data extraction, all six patients in the Phase 1 study of RP-A501 showed signs of improvement or stabilization, with significant reductions in A hsTnl (up to 98%) and A BNP (up to 90%) in pediatric and adult patients[118]. - The company has successfully produced 2 cGMP RP-A501 batches with superior specifications compared to Phase I material, potentially optimizing the safety profile of the product[117]. - RP-L102 has treated a total of 14 patients in clinical trials, with 12 patients from the U.S. and EU completing treatment[134]. - As of April 17, 2023, RP-L102 demonstrated sustained genetic correction in 8 of 12 evaluable patients and phenotypic correction in 7 of 12 patients[136]. - RP-L201 has achieved 100% overall survival at 12 months post-infusion in a cohort of nine severe LAD-I patients[141]. - RP-L301 has shown sustained efficacy in adult patients, including hemoglobin normalization and transfusion independence[149]. - The Phase 2 pivotal trial for RP-L301 is designed to support accelerated approval with a primary endpoint of ≥1.5 point hemoglobin improvement at 12 months[152]. Corporate Strategy and Market Position - The company has global commercialization and development rights to all product candidates under royalty-bearing license agreements[100]. - The company aims to develop into a fully integrated biotechnology company targeting rare pediatric diseases with substantial unmet needs[154]. - The company completed the acquisition of Renovacor, gaining access to a gene therapy program designed to deliver a fully functional BAG3 gene[130]. - The company has raised approximately $1.0 billion from investors through equity and convertible debt financing since inception[155]. - The company has not generated any revenue from product sales and does not expect to do so in the near future[156]. - The company anticipates continued increases in general and administrative expenses due to headcount growth and operational complexity as a public company[166]. Market and Disease Insights - The prevalence of Danon Disease is estimated to be between 15,000 to 30,000 patients in the U.S. and EU[108]. - BAG3-DCM is estimated to affect up to 30,000 individuals in the U.S., with no current therapies directly targeting the underlying mechanism of the disease[128]. - The average lifespan of a Fanconi Anemia patient is estimated to be 30 to 40 years, with a prevalence of approximately 4,000 patients in the U.S. and EU[133].

Exhibit 99.1 Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Progress Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association's 2024 Late-Breaking Scientific Sessions Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025 Rolling BLA submissi ...

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...