Rocket Pharmaceuticals (RCKT) 2025 Conference September 03, 2025 03:20 PM ET Speaker0All right. I think we're ready to get started. Welcome, everyone. I'm Josh Schimmer from the Cantor Biotech Equity Research Team and very pleased to introduce from Rocket Pharmaceuticals, Gaurav Shah, Chief Executive Officer. Gaurav, great to see you again, as always, and a very recent, very positive update on the Danan syndrome program.So, us a quick snapshot of where ROCCAT is in advancing your gene therapy portfolio and ...

Core Viewpoint - Rocket Pharmaceuticals' shares increased by 28.9% after the FDA lifted the clinical hold on its pivotal phase II study for RP-A501, a gene therapy for Danon disease [1][7]. Group 1: Clinical Study Updates - The clinical hold was imposed in May due to a serious adverse event resulting in a patient's death, prompting the company to pause dosing [2]. - The FDA has now allowed the study to resume with a recalibrated lower dose of RP-A501 (3.8 x 10¹³ GC/kg), administered to three patients sequentially with a minimum four-week interval [3]. - Six patients have been treated in the pivotal phase II study, with further updates expected after reviewing data from the next three patients [4]. Group 2: Company Focus and Pipeline - Rocket Pharmaceuticals is prioritizing the development of adeno-associated virus (AAV) gene therapies for rare cardiovascular conditions, with RP-A501 being the most advanced candidate [8]. - The company is also developing another AAV-based candidate, RP-A601, for arrhythmogenic cardiomyopathy, and plans to initiate clinical studies for RP-A701 targeting dilated cardiomyopathy [10]. Group 3: Market Performance - Rocket Pharmaceuticals' shares have declined by 70.2% year-to-date, contrasting with a 3% rise in the industry [5].

Core Insights - Rocket Pharmaceuticals Inc. experienced a significant increase in stock price following the FDA's decision to lift the clinical hold on its Phase 2 trial for RP-A501 targeting Danon disease [1][5] - The clinical hold was resolved in under three months, allowing the trial to resume with a recalibrated dosing strategy [1][3] Company Developments - The FDA's hold was initially placed due to a patient death linked to an acute systemic infection during the study [2] - Rocket Pharmaceuticals has addressed the FDA's concerns, leading to the resumption of the pivotal study with a new dose of 3.8 x 10¹³ GC/kg of RP-A501 [3][4] - The adjusted dosing aligns with the efficacy observed in the Phase 1 study and aims to ensure safety and efficacy in the ongoing trial [4] Treatment Protocol Adjustments - The revised treatment regimen will discontinue the prophylactic use of a C3 complement inhibitor while retaining sirolimus, rituximab, and steroids [5] - A lower threshold for administering a C5 inhibitor (eculizumab) will be specified to manage complement activation [5] - To date, six patients with Danon disease have been treated in the Phase 2 study with RP-A501 [5]

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] - The Rosen Law Firm emphasizes the importance of selecting qualified legal counsel with a proven track record in securities class actions [4] Group 2: Case Allegations - The lawsuit alleges that Rocket Pharmaceuticals made false and misleading statements regarding the effectiveness and safety of its product RP-A501, which was overstated [5] - Specific claims include that RP-A501 was less effective than represented, and changes to its clinical trial protocol increased the risk of Serious Adverse Events [5]

Core Viewpoint - Faruqi & Faruqi, LLP is investigating potential claims against Rocket Pharmaceuticals due to misleading statements regarding the safety of its clinical trial RP-A501, which led to significant investor losses [2][4][5]. Group 1: Legal Investigation and Class Action - Faruqi & Faruqi, LLP is encouraging investors who suffered losses in Rocket Pharmaceuticals to contact them for legal options [1]. - A federal securities class action has been filed against Rocket Pharmaceuticals, with a deadline of August 11, 2025, for investors to seek the role of lead plaintiff [2]. - The firm has a history of recovering hundreds of millions of dollars for investors since its establishment in 1995 [3]. Group 2: Misleading Statements and Clinical Trial Issues - The complaint alleges that Rocket Pharmaceuticals provided overly positive statements while concealing material adverse facts about the safety of RP-A501 and the clinical trial protocol [4]. - Rocket amended the trial protocol to include a new immunomodulatory agent without informing shareholders, which contributed to artificially inflated stock prices [4]. - On May 27, 2025, the FDA placed a clinical hold on the RP-A501 Phase 2 study after a patient suffered a Serious Adverse Event, including death, which was not disclosed to investors prior to the incident [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 one trading day [6].

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 aims to recover losses for investors affected by alleged securities fraud between September 17, 2024, and May 26, 2025 [1]. - Defendants allegedly made positive statements while concealing material adverse facts about RP-A501's safety, including the risk of Serious Adverse Events (SAEs) [2]. - Following the announcement of a clinical hold by the FDA due to a SAE, Rocket's stock price plummeted from $6.27 to $2.33, 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 to be appointed as lead plaintiff [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 one of the leading securities litigation firms in the U.S. [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 Overview - 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]

Core Viewpoint - The Rocket Pharmaceuticals class action lawsuit alleges violations of the Securities Exchange Act of 1934, focusing on misleading statements regarding the safety and clinical trial protocol of its gene therapy product RP-A501 [1][4]. Group 1: Lawsuit Details - The lawsuit is titled Ho v. Rocket Pharmaceuticals, Inc. and seeks to represent purchasers of Rocket Pharmaceuticals securities [1]. - A subsequent case, Yankov v. Rocket Pharmaceuticals, Inc., has also been filed [1]. - The lawsuit claims that Rocket Pharmaceuticals failed to disclose serious adverse events, including patient deaths, related to its clinical trials [4][5]. Group 2: Clinical Trial Allegations - Rocket Pharmaceuticals is accused of providing misleading information about its Phase 2 pivotal trial for RP-A501, particularly regarding the safety of the treatment and the introduction of a new immunomodulatory agent without proper disclosure [4]. - On May 27, 2025, the U.S. FDA placed a clinical hold on the RP-A501 study after a patient suffered a serious adverse event leading to death, which was not disclosed to investors at the time of the protocol amendment [5]. Group 3: Legal Process - Investors who purchased Rocket Pharmaceuticals securities during the class period can seek to be appointed as lead plaintiff in the lawsuit [6]. - The lead plaintiff will represent the interests of all class members and can choose a law firm to litigate the case [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 - 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 a Serious Adverse Event was reported [3][4]. Summary by Sections Allegations - The complaint alleges that Rocket Pharmaceuticals made overwhelmingly positive statements while concealing material adverse facts about RP-A501's safety and clinical trial protocol, including the risk of Serious Adverse Events (SAEs) and participant deaths [3]. - The company amended the trial protocol to include a new immunomodulatory agent without informing shareholders, leading to artificially inflated stock prices [3]. - Following the announcement of a clinical hold by the FDA due to a SAE, Rocket's stock price plummeted from $6.27 on May 23, 2025, to $2.33 on May 27, 2025, marking a decline of approximately 37% in one trading day [3]. Class Action Details - The class period for the lawsuit is defined as September 17, 2024, to May 26, 2025, and shareholders are encouraged to register for participation [3][4]. - The deadline for shareholders to seek lead plaintiff status is August 11, 2025, and there is no cost to participate in the case [4]. Firm Background - The Gross Law Firm is a nationally recognized class action law firm dedicated to protecting investors' rights against deceit and fraud, aiming to ensure responsible business practices [5].

[PART I - FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) [Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) 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](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) 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 disorders[32](index=32&type=chunk)[33](index=33&type=chunk)[34](index=34&type=chunk) - 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 2027[37](index=37&type=chunk) - In July 2025, the company initiated a strategic reorganization involving a **30% workforce reduction** and a restructuring charge of approximately **$3.5 million**[40](index=40&type=chunk)[110](index=110&type=chunk)[111](index=111&type=chunk) - 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)[39](index=39&type=chunk)[54](index=54&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Details management's perspective on financial condition, operational results, a strategic reorganization, and key clinical updates [Overview and Strategy](index=25&type=section&id=Overview%20and%20Strategy) 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](index=119&type=chunk) - The restructuring included a **30% workforce reduction** and is expected to reduce 12-month operating expenses by nearly **25%**[119](index=119&type=chunk) - The company is actively exploring external partnership options for its Fanconi Anemia (RP-L102) and Pyruvate Kinase Deficiency (RP-L301) programs[173](index=173&type=chunk)[179](index=179&type=chunk) [Pipeline Overview](index=27&type=section&id=Pipeline%20Overview) 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)[151](index=151&type=chunk)[152](index=152&type=chunk) - In June 2024, the FDA issued a Complete Response Letter (CRL) for the KRESLADI™ (RP-L201) BLA for LAD-I, requesting additional CMC information[166](index=166&type=chunk) - For the Fanconi Anemia program (RP-L102), an MAA was accepted by the EMA, but the company is now seeking external partners for regulatory filings[173](index=173&type=chunk) [Results of Operations](index=38&type=section&id=Results%20of%20Operations) 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 grant[207](index=207&type=chunk) - The increase in G&A expenses for the six-month period was primarily driven by a **$4.3 million increase** in legal expenses[208](index=208&type=chunk) [Liquidity and Capital Resources](index=39&type=section&id=Liquidity%20and%20Capital%20Resources) 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 billion**[213](index=213&type=chunk) - The company believes its current financial resources are sufficient to fund operating expenses and capital expenditure requirements into the **second quarter of 2027**[213](index=213&type=chunk) - 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 2024[215](index=215&type=chunk)[216](index=216&type=chunk)[217](index=217&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=41&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) 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, 2025[223](index=223&type=chunk) - 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 investments[224](index=224&type=chunk) [Controls and Procedures](index=42&type=section&id=Item%204.%20Controls%20and%20Procedures) 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, 2025[226](index=226&type=chunk) - There were **no material changes** to the company's internal control over financial reporting during the quarter[229](index=229&type=chunk) [PART II - OTHER INFORMATION](index=43&type=section&id=PART%20II%20-%20OTHER%20INFORMATION) [Legal Proceedings](index=43&type=section&id=Item%201.%20Legal%20Proceedings) 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 material**[230](index=230&type=chunk)[231](index=231&type=chunk) - 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-A501[232](index=232&type=chunk) [Risk Factors](index=43&type=section&id=Item%201A.%20Risk%20Factors) 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 lawsuits[235](index=235&type=chunk)[236](index=236&type=chunk) - The company's strategic restructuring may not achieve anticipated savings and could lead to unforeseen difficulties, unexpected costs, or attrition[238](index=238&type=chunk)[239](index=239&type=chunk) [Other Information (Items 2-6)](index=44&type=section&id=Items%202-6.%20Other%20Information) 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 period[240](index=240&type=chunk)[241](index=241&type=chunk) - No directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during the three months ended June 30, 2025[243](index=243&type=chunk)