[Report Overview](index=1&type=section&id=Report%20Overview) [Strategic and Financial Highlights](index=1&type=section&id=Strategic%20and%20Financial%20Highlights) Rocket Pharmaceuticals announced a strategic pivot to prioritize its AAV cardiovascular gene therapy platform, alongside a significant organizational restructuring aimed at reducing costs. The company reported a cash position of $271.5 million, providing an operational runway into the second quarter of 2027, and appointed Chris Stevens as the new Chief Operating Officer - Strategic focus has been refined to prioritize the development of the AAV cardiovascular gene therapy platform, specifically RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM)[1](index=1&type=chunk) - An organizational restructuring is underway, expected to reduce headcount by approximately **30%** and lower the 12-month cash burn by nearly **25%**[1](index=1&type=chunk)[8](index=8&type=chunk) - The company reported cash, cash equivalents, and investments of approximately **$271.5 million**, which is expected to fund operations into the second quarter of **2027**[1](index=1&type=chunk)[9](index=9&type=chunk) - Chris Stevens has been appointed as the new Chief Operating Officer[1](index=1&type=chunk)[6](index=6&type=chunk) [CEO's Statement](index=1&type=section&id=CEO%27s%20Statement) The CEO, Gaurav Shah, M.D., highlighted the second quarter of 2025 as an inflection point where the company sharpened its focus on its high-value AAV cardiovascular platform. He noted that this strategic shift, which includes pausing investments in the Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) programs, strengthens the company's financial foundation and positions it as a leader in gene therapies for inherited cardiomyopathies - The company is fortifying its path to sustained value creation by focusing on programs with the highest value, conserving cash, and driving an efficient organization[3](index=3&type=chunk) - Rocket is pausing additional investments in its Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs and is exploring strategic alternatives to advance them externally[3](index=3&type=chunk)[8](index=8&type=chunk) - The CEO expressed confidence that the recent reorganization ensures the company is appropriately resourced to execute on near-term milestones with nearly **two years of capital**[3](index=3&type=chunk) [Recent Progress and Updates](index=2&type=section&id=Recent%20Progress%20and%20Updates) [Pipeline Updates](index=2&type=section&id=Pipeline%20Updates) The company provided key updates on its clinical pipeline. The Phase 2 study for RP-A501 (Danon disease) was placed on clinical hold by the FDA following a serious adverse event. In contrast, RP-A601 (PKP2-ACM) and RP-A701 (BAG3-DCM) received positive regulatory designations (RMAT and Fast Track, respectively), advancing them toward pivotal trials and clinical entry. The FDA review for KRESLADI™ (LAD-I) is ongoing, with a BLA submission expected by year-end 2025 [RP-A501 (Danon Disease)](index=2&type=section&id=RP-A501%20(Danon%20Disease)) The Phase 2 pivotal study for RP-A501 was placed on clinical hold by the FDA following a patient death, prompting a root cause analysis - The Phase 2 pivotal study for RP-A501 is on clinical hold by the FDA as of May 23, **2025**, following a Serious Adverse Event (SAE) that unfortunately led to a patient's death[4](index=4&type=chunk) - A root cause analysis is underway, focusing on the recent addition of a C3 inhibitor to the pre-treatment regimen[4](index=4&type=chunk) [RP-A601 (PKP2-ACM)](index=2&type=section&id=RP-A601%20(PKP2-ACM)) RP-A601 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation, with the company engaging the FDA on a potential pivotal trial design following encouraging Phase 1 data - Received FDA Regenerative Medicine Advanced Therapy (RMAT) designation in July **2025**[1](index=1&type=chunk)[4](index=4&type=chunk) - Following encouraging initial Phase 1 data presented at ASGCT, Rocket is actively engaging with the FDA on a potential pivotal trial design[1](index=1&type=chunk)[4](index=4&type=chunk) [RP-A701 (BAG3-DCM)](index=2&type=section&id=RP-A701%20(BAG3-DCM)) The IND application for RP-A701 was cleared by the FDA, and the program received Fast Track designation, with Phase 1 trial start-up activities now underway - The Investigational New Drug (IND) application was cleared by the FDA in June **2025**[4](index=4&type=chunk) - RP-A701 was granted FDA Fast Track designation, and Phase 1 trial start-up activities are now underway[1](index=1&type=chunk)[4](index=4&type=chunk) [KRESLADI™ (LAD-I)](index=2&type=section&id=KRESLADI%E2%84%A2%20(LAD-I)) The FDA is reviewing additional CMC information for KRESLADI™, with the company expecting to submit the complete BLA to resolve the Complete Response Letter by year-end 2025 - The FDA is conducting a review of limited additional Chemistry Manufacturing and Controls (CMC) information for KRESLADI™[4](index=4&type=chunk) - The company anticipates submitting the complete Biologics License Application (BLA) to resolve the Complete Response Letter before the end of **2025**[4](index=4&type=chunk) [Corporate and Operational Updates](index=3&type=section&id=Corporate%20and%20Operational%20Updates) Rocket has executed a strategic corporate restructuring, reducing its workforce by approximately 30% to align with its prioritized focus on the AAV cardiovascular platform. This move is expected to cut operating expenses by nearly 25% over the next year. As part of this realignment, the company has paused further investment in its Fanconi Anemia and Pyruvate Kinase Deficiency programs. Additionally, Chris Stevens was appointed as the new Chief Operating Officer - Implemented a workforce reduction of approximately **30%** across all functions to align with the pipeline prioritization plan[8](index=8&type=chunk) - The restructuring and other cost-saving measures are expected to reduce operating expenses by nearly **25%** over the next **12 months**[8](index=8&type=chunk) - As part of the realignment, the company is pausing additional investments in its Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) programs[8](index=8&type=chunk) - Appointed Chris Stevens, a seasoned executive with **25 years of experience** from Spark Therapeutics, as Chief Operating Officer[6](index=6&type=chunk) [Second Quarter 2025 Financial Results](index=3&type=section&id=Second%20Quarter%202025%20Financial%20Results) [Key Financial Metrics](index=3&type=section&id=Key%20Financial%20Metrics) For the second quarter of 2025, Rocket Pharmaceuticals reported a net loss of $68.9 million, or $0.62 per share, a slight improvement from the $69.6 million net loss in Q2 2024. The company reduced both R&D and G&A expenses year-over-year and ended the quarter with a solid cash position of $271.5 million Key Financial Metrics (in millions) | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Cash, Cash Equivalents & Investments | $271.5M | N/A | - | | R&D Expenses | $42.7M | $46.3M | ($3.6M) | | G&A Expenses | $25.0M | $27.4M | ($2.4M) | | Net Loss | ($68.9M) | ($69.6M) | $0.7M | | Net Loss Per Share | ($0.62) | ($0.74) | $0.12 | - The decrease in R&D expenses was primarily driven by lower costs for manufacturing, direct materials, and professional fees[9](index=9&type=chunk) - The decrease in G&A expenses was mainly due to reduced commercial preparation and compensation-related expenses[15](index=15&type=chunk) [Restructuring Expenses and Financial Guidance](index=4&type=section&id=Restructuring%20Expenses%20and%20Financial%20Guidance) The company incurred approximately $3.5 million in restructuring and related charges during the first half of 2025. Management expects its current cash, cash equivalents, and investments of $271.5 million will be sufficient to fund operations into the second quarter of 2027 - Approximately **$3.5 million** in restructuring and related charges were incurred in the first half of **2025**[10](index=10&type=chunk) - The company's cash runway is expected to extend into the second quarter of **2027**, excluding any potential proceeds from a Priority Review Voucher[9](index=9&type=chunk) [Consolidated Financial Statements](index=6&type=section&id=Consolidated%20Financial%20Statements) The consolidated statements of operations detail the company's financial performance for the three and six months ending June 30, 2025, showing a reduction in total operating expenses compared to the prior year. The balance sheet reflects total assets of $421.0 million and total stockholders' equity of $354.2 million as of June 30, 2025 Consolidated Statements of Operations (in thousands) | Statement of Operations | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Research and development | $42,658 | $46,345 | | General and administrative | $25,020 | $27,367 | | Restructuring | $3,471 | $0 | | **Total operating expenses** | **$71,149** | **$73,712** | | **Loss from operations** | **($71,149)** | **($73,712)** | | **Net loss** | **($68,919)** | **($69,646)** | | **Net loss per share** | **($0.62)** | **($0.74)** | Consolidated Balance Sheets (in thousands) | Balance Sheet | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and investments | $271,494 | $372,336 | | Total assets | $420,979 | $527,700 | | Total liabilities | $66,768 | $64,466 | | Total stockholders' equity | $354,211 | $463,234 | [Corporate Information](index=4&type=section&id=Corporate%20Information) [About Rocket Pharmaceuticals, Inc.](index=4&type=section&id=About%20Rocket%20Pharmaceuticals%2C%20Inc.) Rocket Pharmaceuticals is a late-stage biotechnology company developing a pipeline of genetic therapies for rare and complex disorders. The company utilizes a multi-platform approach, with an adeno-associated viral (AAV) vector-based portfolio for cardiovascular diseases and a lentiviral (LV) vector-based portfolio for hematology disorders - The company's AAV cardiovascular portfolio includes late-stage programs for Danon Disease and early-stage programs for PKP2-arrhythmogenic cardiomyopathy and BAG3-associated dilated cardiomyopathy[12](index=12&type=chunk) - The LV hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA), and Pyruvate Kinase Deficiency (PKD)[13](index=13&type=chunk) [Forward-Looking Statements](index=5&type=section&id=Forward-Looking%20Statements) This section serves as a cautionary notice to investors, stating that the press release contains forward-looking statements that are subject to significant risks and uncertainties. These statements, which are not guarantees of future performance, pertain to the company's plans, clinical trial timings, regulatory outcomes, and financial projections. Readers are advised not to place undue reliance on them - The press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of **1995**[16](index=16&type=chunk) - These statements involve risks and uncertainties that could cause actual results to differ materially from expectations, including the ability to realize benefits from restructuring, trial outcomes, and regulatory decisions[16](index=16&type=chunk)

Core Viewpoint - Rosen Law Firm is reminding investors who purchased Rocket Pharmaceuticals, Inc. securities during the specified Class Period of the upcoming lead plaintiff deadline on August 11, 2025 [1] Group 1: Class Action Details - Investors who purchased Rocket Pharmaceuticals securities between September 17, 2024, and May 26, 2025, may be entitled to compensation without any out-of-pocket fees through a contingency fee arrangement [2] - A class action lawsuit has already been filed, and interested parties must move the Court to serve as lead plaintiff by August 11, 2025 [3] - Investors can join the class action by visiting the provided link or contacting the law firm for more information [6] Group 2: Law Firm Credentials - Rosen Law Firm has a strong track record in securities class actions, having achieved the largest securities class action settlement against a Chinese company at the time [4] - The firm was ranked No. 1 by ISS Securities Class Action Services for the number of settlements in 2017 and has consistently ranked in the top 4 since 2013, recovering hundreds of millions for investors [4] - In 2019, the firm secured over $438 million for investors, and its founding partner was recognized as a Titan of Plaintiffs' Bar by Law360 in 2020 [4] Group 3: Case Allegations - The lawsuit alleges that Rocket Pharmaceuticals made false and misleading statements regarding the effectiveness and safety of RP-A501, which was overstated [5] - It is claimed that the introduction of a novel immunomodulatory agent to increase RP-A501's effectiveness raised the risk of Serious Adverse Events (SAEs) for patients [5] - The lawsuit asserts that when the true details were revealed, investors suffered damages due to the misleading public statements made by the defendants [5]

Core Viewpoint - Rocket Pharmaceuticals, Inc. is facing a class action securities lawsuit due to alleged securities fraud related to the safety and clinical trial protocol of its RP-A501 treatment, which resulted in significant stock price decline after the FDA placed a clinical hold on the study [1][2]. Group 1: Lawsuit Details - The lawsuit seeks to recover losses for investors affected by alleged securities fraud between September 17, 2024, and May 26, 2025 [1]. - Defendants allegedly provided misleading statements about the safety of RP-A501 while concealing serious adverse events, including participant deaths during the clinical trial [2]. - Following the announcement of the FDA's clinical hold on May 27, 2025, Rocket's stock price plummeted from $6.27 to $2.33 per share, a decline of approximately 37% in one trading day [2]. Group 2: Next Steps for Investors - Investors who suffered losses during the relevant timeframe have until August 11, 2025, to request appointment as lead plaintiff, although participation does not require this role [3]. - Class members may be entitled to compensation without any out-of-pocket costs or fees [3]. Group 3: Firm Background - Levi & Korsinsky, LLP has a strong track record in securities litigation, having secured hundreds of millions for shareholders over the past 20 years [4]. - The firm has been recognized in ISS Securities Class Action Services' Top 50 Report for seven consecutive years as a leading securities litigation firm in the United States [4].

Core Points - A securities class action lawsuit has been filed against Rocket Pharmaceuticals, Inc. for alleged material misstatements and omissions affecting investors [1][3] - The lawsuit pertains to the period from September 17, 2024, to May 26, 2025, during which investors may seek compensation [2] - The lawsuit claims that Rocket Pharmaceuticals failed to disclose significant safety-related updates regarding its RP-A501 clinical trial, leading to a fatal adverse event and subsequent FDA clinical hold [3] Company Information - Rocket Pharmaceuticals is headquartered in Cranbury, New Jersey, and specializes in gene therapies for rare and life-threatening diseases [2] - Following the public confirmation of the trial hold and patient death on May 28, 2025, Rocket Pharmaceuticals' stock price plummeted from $6.27 to $2.33 [3]

NEW YORK, Aug. 5, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Rocket Pharmaceuticals, Inc. ("Rocket" or the "Company") (NASDAQ: RCKT) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Rocket investors who were adversely affected by alleged securities fraud between September 17, 2024 and May 26, 2025. Follow the link below to get more information and be contacted by a member of our team: https://zlk.com/pslra-1/rocket-pharmaceuticals ...

Core Viewpoint - A class action lawsuit has been filed against Rocket Pharmaceuticals, alleging violations of the Securities Exchange Act of 1934 related to misleading information about its clinical trials and safety protocols [1][4][5]. Group 1: Lawsuit Details - The lawsuit, titled Ho v. Rocket Pharmaceuticals, Inc., is pending in the District of New Jersey and seeks to represent purchasers of Rocket Pharmaceuticals' securities [1]. - A subsequent case, Yankov v. Rocket Pharmaceuticals, Inc., has also been filed [1]. - The allegations include that Rocket Pharmaceuticals provided misleading information regarding its Phase 2 trial of RP-A501 for Danon disease while concealing serious risks, including participant deaths [4]. Group 2: Clinical Trial Issues - The lawsuit claims that Rocket Pharmaceuticals failed to disclose a critical amendment to the trial protocol that introduced a new immunomodulatory agent, which was linked to serious adverse events [4]. - On May 27, 2025, the U.S. FDA placed a clinical hold on the RP-A501 study after a patient died, which was not disclosed to investors at the time of the protocol amendment [5]. Group 3: Legal Process - Investors who suffered losses can seek to be appointed as lead plaintiff in the class action lawsuit, with motions due by August 11, 2025 [2][6]. - The lead plaintiff will represent the interests of all class members and can choose their legal representation [6]. Group 4: Firm Background - Robbins Geller Rudman & Dowd LLP is a leading law firm in securities fraud litigation, having recovered over $2.5 billion for investors in 2024 alone [7]. - The firm has been ranked 1 in securing monetary relief for investors in securities class actions for four out of the last five years [7].

Core Viewpoint - Rocket Pharmaceuticals, Inc. is facing a class action lawsuit due to allegations of providing misleading statements regarding the safety and clinical trial protocol of its RP-A501 treatment, which resulted in significant stock price decline after the FDA placed a clinical hold on the study following a Serious Adverse Event [3][4]. Group 1: Allegations and Events - The class period for the lawsuit is from September 17, 2024, to May 26, 2025 [3]. - Allegations include that Rocket Pharmaceuticals made overwhelmingly positive statements while concealing material adverse facts about RP-A501's safety, including the risk of Serious Adverse Events (SAEs) [3]. - The company amended the trial protocol to introduce a new immunomodulatory agent without informing shareholders, which led to inflated stock prices [3]. - On May 27, 2025, Rocket announced the FDA's clinical hold on the RP-A501 Phase 2 study after a patient suffered a SAE, ultimately resulting in death [3]. - Following this announcement, Rocket's stock price plummeted from $6.27 per share on May 23, 2025, to $2.33 per share on May 27, 2025, marking a decline of approximately 37% in one trading day [3]. Group 2: Next Steps for Shareholders - Shareholders are encouraged to register for the class action lawsuit by August 11, 2025, to potentially become lead plaintiffs [4]. - Registered shareholders will receive updates through a portfolio monitoring software regarding the case's status [4]. - Participation in the case incurs no cost or obligation for shareholders [4]. Group 3: Law Firm Information - The Gross Law Firm is a nationally recognized class action law firm dedicated to protecting investors' rights against deceit and fraud [5]. - The firm aims to ensure companies adhere to responsible business practices and seeks recovery for investors who suffered losses due to misleading statements [5].

Core Viewpoint - The Rocket Pharmaceuticals class action lawsuit alleges violations of the Securities Exchange Act of 1934, focusing on misleading information regarding the safety and clinical trial protocol of its gene therapy product RP-A501 for Danon disease [1][4]. Group 1: Lawsuit Details - The lawsuit is titled Ho v. Rocket Pharmaceuticals, Inc., No. 25-cv-10049, and is pending in the District of New Jersey, representing purchasers of Rocket Pharmaceuticals securities [1]. - A subsequent case, Yankov v. Rocket Pharmaceuticals, Inc., No. 25-cv-13532, has also been filed [1]. - The lawsuit claims that Rocket Pharmaceuticals provided material information about its Phase 2 trial while concealing adverse facts about the safety of RP-A501, including the risk of Serious Adverse Events [4]. Group 2: Clinical Trial Issues - On May 27, 2025, Rocket Pharmaceuticals announced that the FDA placed a clinical hold on the RP-A501 Phase 2 study after a patient suffered a Serious Adverse Event, leading to death [5]. - The complaint states that the company failed to disclose a substantive amendment to the trial's protocol that introduced a new immunomodulatory agent, which was critical information for investors [4][5]. Group 3: Lead Plaintiff Process - The Private Securities Litigation Reform Act of 1995 allows any investor who purchased Rocket Pharmaceuticals securities during the Class Period to seek appointment as lead plaintiff [6]. - The lead plaintiff is typically the investor with the greatest financial interest and acts on behalf of all class members [6]. Group 4: About Robbins Geller - Robbins Geller Rudman & Dowd LLP is a leading law firm specializing in securities fraud and shareholder litigation, having recovered over $2.5 billion for investors in 2024 alone [7]. - The firm has been ranked 1 in securing monetary relief for investors in securities class action cases for four out of the last five years [7].

Core Viewpoint - Faruqi & Faruqi, LLP is investigating potential claims against Rocket Pharmaceuticals due to misleading statements regarding the safety of its clinical trial for RP-A501, which led to significant investor losses [2][4][5]. Group 1: Legal Investigation and Claims - Faruqi & Faruqi, LLP is encouraging investors who suffered losses exceeding $50,000 in Rocket Pharmaceuticals between February 27, 2025, and May 26, 2025, to discuss their legal options [1]. - The firm has set an August 11, 2025, deadline for investors to seek the role of lead plaintiff in a federal securities class action against Rocket Pharmaceuticals [2]. - The complaint alleges that Rocket Pharmaceuticals provided positive statements while concealing material adverse facts about the safety of RP-A501 and the clinical trial protocol [4]. Group 2: Clinical Trial Issues - Rocket Pharmaceuticals faced a clinical hold from the FDA on the RP-A501 Phase 2 pivotal study after a patient suffered a Serious Adverse Event (SAE), which resulted in death [5]. - The company amended the trial protocol to introduce a new immunomodulatory agent without disclosing this critical update to investors, which is a key point of contention in the lawsuit [4][5]. Group 3: Stock Price Impact - Following the announcement of the clinical hold, Rocket's stock price plummeted from $6.27 per share on May 23, 2025, to $2.33 per share on May 27, 2025, marking a decline of approximately 37% in just one trading day [6].

Core Viewpoint - Rosen Law Firm is reminding investors who purchased Rocket Pharmaceuticals securities during the specified Class Period of the upcoming lead plaintiff deadline on August 11, 2025 [1]. Group 1: Class Action Details - Investors who purchased Rocket Pharmaceuticals securities between September 17, 2024, and May 26, 2025, may be entitled to compensation without any out-of-pocket fees through a contingency fee arrangement [2]. - A class action lawsuit has already been filed, and interested parties can join by contacting the law firm [3][6]. - The lawsuit claims that Rocket Pharmaceuticals made false and misleading statements regarding the effectiveness and safety of its product RP-A501, which led to investor damages when the truth was revealed [5]. Group 2: Law Firm Credentials - Rosen Law Firm has a strong track record in securities class actions, having achieved significant settlements, including the largest securities class action settlement against a Chinese company at the time [4]. - The firm has been consistently ranked among the top firms for securities class action settlements and has recovered hundreds of millions of dollars for investors [4].