Summary of Rocket Pharmaceuticals FY Conference Call Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Gene Therapy - **Focus**: Development of gene therapies for rare diseases, particularly in cardiomyopathy and hematology Key Points Company and Product Pipeline - Rocket Pharmaceuticals operates as a dual platform company with both in vivo AAV-based cardiac programs and ex vivo lentiviral-based hematology programs in development [2][3] - The company has six disclosed programs, including three cardiac programs targeting Danon Disease, PKP2-arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, collectively affecting at least 100,000 patients in the U.S. and Europe [2][3] - The lentiviral portfolio includes Leukocyte Adhesion Deficiency-I, Fanconi Anemia, and Pyruvate Kinase Deficiency, with a decision to pause new spending on the latter two to focus resources on AAV-based cardiac programs [3][4] Financial and Operational Updates - A workforce reduction was implemented to prioritize certain programs and extend cash runway, with cash expected to last into Q2 2027 [4] - The company aims to be first, best, and only in class for its gene therapy products, targeting diseases with increasing market sizes [3][4] Clinical Trials and Regulatory Interactions - The pivotal phase two trial for Danon Disease was put on clinical hold due to increased risk of TMA (Thrombotic Microangiopathy) but was lifted in less than three months after modifications to the treatment regimen [9][10] - The trial will start in early 2026, with a three-month troponin run-in period before patient treatment begins [10] - The pivotal trial design includes a 12-patient trial with composite endpoints of protein expression and LV mass index reductions, agreed upon with the FDA [24][30] Market Opportunity - The prevalence of Danon Disease is estimated at 15,000 to 30,000 patients in the U.S. and Europe, with ongoing efforts to identify more patients through genetic testing [34] - The company is positioned to potentially file for commercialization in the next few years, contingent on trial outcomes [35][36] Future Catalysts - Upcoming catalysts include the PDUFA date for CRISLATI, clarity on PKP2 trial design, and updates on the Danon program and BAG3 phase one start [61][62] - The company plans to proceed cautiously with its programs, focusing on one at a time to ensure successful execution [62] Additional Insights - The company has learned valuable lessons from the Danon Disease program that will inform future trials, particularly regarding safety monitoring and regulatory interactions [38][39] - The choice of vectors for gene therapy is strategic, with AAV9 used for Danon Disease and RH74 for PKP2 and BAG3, based on regulatory experience and patient community feedback [55] Conclusion - Rocket Pharmaceuticals is actively advancing its gene therapy programs with a focus on rare diseases, navigating regulatory challenges, and preparing for future commercialization opportunities while ensuring patient safety and efficacy in its clinical trials [61][62]

Summary of Rocket Pharmaceuticals (RCKT) 2025 Conference Call Company Overview - **Company**: Rocket Pharmaceuticals (RCKT) - **Focus**: Gene therapy for rare diseases, particularly pediatric conditions with high unmet needs Key Points Discussed Danan Syndrome Program - The FDA lifted the clinical hold on the Danan syndrome program in record time, under three months, indicating the program's value and the FDA's collaboration [3][4] - The company is focusing on diseases with high unmet needs, particularly rare and often fatal pediatric diseases [4] - The cardiac portfolio has been prioritized, with three key programs targeting different types of cardiomyopathy, representing over 100,000 patients in the U.S. and Europe [7] Clinical Trials and Safety Monitoring - The company has adjusted dosing protocols to mitigate risks associated with thrombotic microangiopathy (TMA) observed in previous trials [12][14] - A new dosing strategy has been established, moving from a higher dose of 6.7e13 to a recalibrated dose of around 4e13, which aligns with FDA recommendations [16][27] - The monitoring protocol for TMA has evolved, incorporating a combination of rituximab, sirolimus, and steroids, with a focus on early detection of complement activation [18][19] Patient Enrollment and Community Response - Initial enrollment faced challenges due to safety events, but subsequent patient recruitment has been rapid, with ten patients enrolled in just over two months after initial delays [35] - The patient community remains supportive despite setbacks, recognizing the fatal nature of the disease and the potential benefits of the trial [33] Future Directions and Milestones - The company anticipates treating three new patients in early 2026, following necessary regulatory approvals and monitoring protocols [40][41] - The FDA has not mandated an increase in patient enrollment beyond 12 for the pivotal trial, allowing the company to focus on achieving a positive trial outcome [43][44] - Upcoming milestones include updates on patient treatment, trial design alignment with the FDA, and epidemiological data to support patient identification [59] Lessons Learned and Application to Other Programs - Insights from the Danan program regarding TMA and dosing are being applied to the PKP2 program, with stringent patient selection criteria to avoid complications [46][48] - The company is also exploring the use of immunofluorescence for more accurate protein localization in the PKP2 program, moving away from traditional Western blot methods [49] BAG3 Program - The BAG3 program is set to begin Phase 1 trials next year, with a focus on dilated cardiomyopathy, which has a clear endpoint of ejection fraction (EF) improvement [60][61] Additional Important Information - The company is developing a fourteen-gene panel to screen for mutations that may increase the risk of complement activation, enhancing patient safety [32] - The overall sentiment from the community and investigators remains optimistic, with a strong belief in the efficacy of gene therapy for devastating rare diseases [33] This summary encapsulates the critical discussions and insights from the Rocket Pharmaceuticals conference call, highlighting the company's strategic focus, clinical advancements, and community engagement.

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].