Rocket Pharmaceuticals(US:RCKT)2025-08-07 20:06PART I - FINANCIAL INFORMATION Financial Statements Presents the unaudited consolidated financial statements for Q2 and H1 2025, reporting a net loss with no revenue generated Consolidated Balance Sheet Highlights | Financial Metric | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and investments | $271.5 million | $372.3 million | | Total Assets | $421.0 million | $527.7 million | | Total Liabilities | $66.8 million | $64.5 million | | Total Stockholders' Equity | $354.2 million | $463.2 million | Consolidated Statements of Operations Highlights | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Revenue | $0 | $0 | | Research and development | $42.7 million | $46.3 million | | General and administrative | $25.0 million | $27.4 million | | Restructuring | $3.5 million | $0 | | Net Loss | ($68.9 million) | ($69.6 million) | | Net Loss Per Share | ($0.62) | ($0.74) | Consolidated Cash Flow Highlights | Cash Flow Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($104.8 million) | ($110.7 million) | | Net cash (used in) provided by investing activities | ($26.3 million) | $90.7 million | | Net cash provided by financing activities | $0.2 million | $2.7 million | Notes to Consolidated Financial Statements Provides details on accounting policies, liquidity risks, a corporate reorganization, and a clinical hold on the RP-A501 trial - Rocket is a late-stage biotechnology company developing gene therapies for rare cardiovascular and hematology disorders323334 - As of June 30, 2025, the company had an accumulated deficit of $1.35 billion, with cash and investments of $271.5 million expected to fund operations into Q2 202737 - In July 2025, the company initiated a strategic reorganization involving a 30% workforce reduction and a restructuring charge of approximately $3.5 million40110111 - In May 2025, the FDA placed a clinical hold on the Phase 2 trial for RP-A501 for Danon disease following a Serious Adverse Event (SAE)3954 Management's Discussion and Analysis of Financial Condition and Results of Operations Details management's perspective on financial condition, operational results, a strategic reorganization, and key clinical updates Overview and Strategy Outlines the strategic shift to prioritize the AAV cardiovascular platform, reduce operating expenses, and seek external partnerships - In July 2025, the company announced a strategic corporate reorganization to focus resources on its AAV cardiovascular platform and respond to the CRL for KRESLADI™119 - The restructuring included a 30% workforce reduction and is expected to reduce 12-month operating expenses by nearly 25%119 - The company is actively exploring external partnership options for its Fanconi Anemia (RP-L102) and Pyruvate Kinase Deficiency (RP-L301) programs173179 Pipeline Overview Reviews the company's pipeline, highlighting the clinical hold on RP-A501 and the Complete Response Letter for KRESLADI™ - The FDA placed the Phase 2 pivotal trial for RP-A501 (Danon disease) on clinical hold on May 23, 2025, after a patient experienced a Serious Adverse Event (SAE)151152 - In June 2024, the FDA issued a Complete Response Letter (CRL) for the KRESLADI™ (RP-L201) BLA for LAD-I, requesting additional CMC information166 - For the Fanconi Anemia program (RP-L102), an MAA was accepted by the EMA, but the company is now seeking external partners for regulatory filings173 Results of Operations Compares financial results for the six months ended June 30, 2025 and 2024, noting changes in R&D and G&A expenses Comparison of Operations | Metric | Six Months Ended June 30, 2025 ($M) | Six Months Ended June 30, 2024 ($M) | Change ($M) | | :--- | :--- | :--- | :--- | | R&D Expenses | 78.6 | 91.6 | (13.0) | | G&A Expenses | 53.5 | 49.5 | 4.0 | | Restructuring | 3.5 | 0.0 | 3.5 | | Net Loss | (130.3) | (131.7) | 1.4 | - The decrease in R&D expenses for the six-month period was primarily driven by lower manufacturing costs ($4.8M), professional fees ($3.6M), and a $2.7M CIRM grant207 - The increase in G&A expenses for the six-month period was primarily driven by a $4.3 million increase in legal expenses208 Liquidity and Capital Resources Assesses the company's financial position, including its cash balance, accumulated deficit, and projected cash runway into Q2 2027 - As of June 30, 2025, the company had $271.5 million in cash, cash equivalents, and investments and an accumulated deficit of $1.35 billion213 - The company believes its current financial resources are sufficient to fund operating expenses and capital expenditure requirements into the second quarter of 2027213 - Net cash used in operating activities was $104.8 million for the six months ended June 30, 2025, compared to $110.7 million for the same period in 2024215216217 Quantitative and Qualitative Disclosures About Market Risk Discusses the company's exposure to interest rate risk on its investment portfolio and the potential impact of rate changes - The company's primary market risk is interest rate sensitivity on its $271.5 million portfolio of cash, cash equivalents, and investments as of June 30, 2025223 - A hypothetical uniform 100-basis-point increase in interest rates would result in a decline of $0.7 million in the net fair value of the company's investments224 Controls and Procedures Confirms the effectiveness of the company's disclosure controls and procedures as of the end of the reporting period - Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2025226 - There were no material changes to the company's internal control over financial reporting during the quarter229 PART II - OTHER INFORMATION Legal Proceedings Details the settlement of litigation with Lexeo Therapeutics and the filing of two new securities class action lawsuits - In June 2025, the company settled litigation with Lexeo Therapeutics, Inc, with the financial impact being not material230231 - Two securities class action lawsuits were filed against the company in June and July 2025 related to public statements about the Phase 2 clinical trial for RP-A501232 Risk Factors Highlights new risks related to securities litigation costs and the potential challenges of the recent strategic restructuring - A new risk factor is the potential for substantial costs and diversion of management resources due to recently filed securities class action lawsuits235236 - The company's strategic restructuring may not achieve anticipated savings and could lead to unforeseen difficulties, unexpected costs, or attrition238239 Other Information (Items 2-6) Covers standard disclosures including unregistered equity sales, senior securities defaults, and Rule 10b5-1 trading plans - There were no unregistered sales of equity securities or defaults upon senior securities during the period240241 - No directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during the three months ended June 30, 2025243