28 May 2025 | 9:26AM EDT Rocket Pharmaceuticals Inc. (RCKT): Clinical Hold on Ph. 2 DD Trial Puts Largest Late-Stage Oppy At Risk; Downgrade to Sell w/ $2 PT On Tuesday (5/27), RCKT announced that the FDA placed a clinical hold on its pivotal Ph. 2 Danon disease (DD) trial. The hold was issued on 5/23 after a patient treated in early May experienced capillary leak syndrome that emerged ~1 week after RP-A501 dosing. Although there were some early signs the condition was beginning to improve, the patient subs ...

LOS ANGELES, May 29, 2025 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Rocket Pharmaceuticals, Inc. (“Rocket” or “the Company”) (NASDAQ: RCKT) investors that the firm has initiated an investigation into possible securities fraud and may file a class action on behalf of investors. Rocket investors that lost money on their investment are encouraged to contact Lesley Portnoy, Esq. Investors are encouraged to contact attorney Lesley F. Portnoy, by phone 310-692-8883 or email: info@portnoylaw.com, to discuss ...

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices by Rocket Pharmaceuticals and its officers or directors following a serious adverse event in a clinical trial that led to a significant drop in the company's stock price [1][3]. Group 1: Company Developments - On May 27, 2025, Rocket Pharmaceuticals announced an unexpected serious adverse event (SAE) related to its investigational gene therapy RP-A501 for Danon disease, which involved clinical complications from capillary leak syndrome [3]. - Following the SAE, Rocket voluntarily paused further dosing in the Phase 2 pivotal trial, and the FDA placed a clinical hold on the trial for further evaluation [3]. - The patient involved in the SAE subsequently passed away due to an acute systemic infection [3]. Group 2: Market Reaction - The announcement of the SAE resulted in Rocket's stock price falling by $3.94 per share, a decrease of 62.84%, closing at $2.33 per share on May 27, 2025 [3]. Group 3: Legal Context - Pomerantz LLP, a prominent firm in corporate and securities class litigation, is representing investors in the investigation regarding Rocket Pharmaceuticals [1][4].

Shares of Rocket Pharmaceuticals (RCKT) plunged 62.8% on Tuesday after the company announced a patient death in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.The company confirmed that one of the patients in the study experienced an acute systemic infection, an unexpected serious adverse event, which resulted in his untimely death.Following the turn of events, RCKT has voluntarily paused further dosing in the study evaluatin ...

About this content About Angela Harmantas Angela Harmantas is an Editor at Proactive. She has over 15 years of experience covering the equity markets in North America, with a particular focus on junior resource stocks. Angela has reported from numerous countries around the world, including Canada, the US, Australia, Brazil, Ghana, and South Africa for leading trade publications. Previously, she worked in investor relations and led the foreign direct investment program in Canada for the Swedish government ...

Rocket Pharmaceuticals (RCKT) Clinical Update Summary Company Overview - **Company**: Rocket Pharmaceuticals (RCKT) - **Focus**: Investigational gene therapy for Dannon disease, specifically RPA-501 Key Points from the Conference Call Clinical Trial Update - A serious adverse event (SAE) occurred in a patient enrolled in the Phase 2 pivotal trial for RPA-501, related to capillary leak syndrome [4][5] - The trial has been placed on clinical hold by the FDA following the SAE, which resulted in the patient's unfortunate passing due to a systemic infection [5][7] - Rocket is conducting a comprehensive root cause analysis and is in active dialogue with the FDA regarding the situation [4][7] Protocol Changes and Safety Measures - A novel immune suppression agent, a C3 inhibitor, was introduced to the pretreatment regimen to mitigate complement activation risks [4][12] - The introduction of the C3 inhibitor was based on prior evidence of complement activation in Dannon disease [12][62] - The company is evaluating whether the new agent may have influenced immune responses unexpectedly [6][7] Patient Treatment and Outcomes - Two patients have experienced capillary leak syndrome, with one patient showing a reduced course of complications after the introduction of the C3 inhibitor [18][21] - The company had planned to treat additional patients by mid-year, but the clinical hold has paused these plans [27] Financial and Operational Outlook - Rocket expects existing resources to fund operations into 2027, excluding potential proceeds from the sale of Priority Review Vouchers (PRVs) [9] - The company is prioritizing investments in its AAV platform while conducting a strategic review of its pipeline [8] Future Steps and Communication - Rocket is committed to transparency and open communication with the Dannon community and stakeholders [8][70] - The company is working with the FDA to define next steps and is confident in finding a path forward for RPA-501 [7][70] Additional Insights - The C3 inhibitor used is a licensed product, and there was a previous instance of sepsis in a Phase 1 study, but the patient recovered fully [66] - The company has not observed capillary leak syndrome in patients who did not receive the C3 inhibitor [59] - The FDA has been responsive and collaborative in discussions regarding the trial [71] Conclusion Rocket Pharmaceuticals is facing significant challenges with its RPA-501 clinical trial due to a serious adverse event. The company is actively working with the FDA to address safety concerns and is committed to maintaining transparency with stakeholders while ensuring patient safety. The financial outlook remains stable, with resources expected to last into 2027.

Summary of Rocket Pharmaceuticals (RCKT) Investor Conference Call - May 15, 2025 Company Overview - **Company**: Rocket Pharmaceuticals (RCKT) - **Focus**: Development of gene therapies, specifically RPA-601 for PKP2 arrhythmogenic cardiomyopathy (ACM) Key Industry Insights - **Disease Context**: PKP2 ACM is an inherited heart disease affecting approximately 50,000 adults and children in the US and Europe, characterized by life-threatening arrhythmias and sudden cardiac death [9][10] - **Current Treatments**: Existing treatments include ICDs, beta blockers, antiarrhythmics, and heart transplants, which do not modify disease progression [11][12] Core Findings from the Conference Call - **Phase I Trial Results**: Preliminary data from the Phase I trial of RPA-601 indicated that the treatment was generally well tolerated, with no dose-limiting toxicities reported [15][29] - **Efficacy Indicators**: All three patients in the trial showed early signs of disease modification, with improvements in multiple biomarkers and clinical parameters [7][29] - **Dosage Decision**: The final dose for the next phase of development is set at 8E13 vector genomes per kilogram, based on safety and efficacy data from the first cohort [6][32] Safety and Efficacy Data - **Patient Cohort**: The first cohort consisted of three patients, all of whom had mild symptoms of heart failure and had been on prior treatments [14][25] - **Adverse Events**: Most adverse events were mild or moderate, with one patient experiencing serious adverse events likely related to immunosuppression rather than the gene therapy itself [15][16] - **Biomarker Improvements**: Patients showed demonstrable cardiac transduction of RPA-601, with vector copy numbers ranging from 3 to 8 per diploid nucleus [16][31] Clinical Outcomes - **Functional Improvements**: Two patients improved from NYHA class two to class one, indicating a significant reduction in heart failure symptoms [25][32] - **Quality of Life**: KCCQ scores improved significantly, with one patient showing a 34-point improvement and another a 41-point improvement [26][32] - **Arrhythmia Burden**: Preliminary indications of reduced arrhythmia burden were observed, with one patient experiencing a 63% reduction in PVCs [28][29] Future Development Plans - **Next Steps**: The company plans to design the next phase of development, including a potential pivotal trial for PKP2 ACM [32] - **Patient Population**: Future trials may include younger patients and those without ICDs, aiming to address the disease earlier [71][72] - **Market Potential**: Approximately 80% of PKP2 ACM patients have ICDs, indicating a significant addressable market for the therapy [77] Additional Considerations - **Regulatory Pathways**: The company is exploring accelerated approval pathways and is in ongoing discussions with the FDA regarding trial design and endpoints [100][116] - **Manufacturing Capabilities**: Rocket Pharmaceuticals has built out its manufacturing facility to support both the PKP2 program and other ongoing projects [88][89] Conclusion - **Overall Assessment**: RPA-601 shows promise as a transformative therapy for PKP2 ACM, with preliminary data indicating safety and efficacy. The company is positioned to advance to pivotal trials, with a focus on addressing significant unmet medical needs in this patient population [30][32]

Financial Performance - Rocket Pharmaceuticals incurred a loss of 56 cents per share in Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 59 cents and an improvement from a loss of 66 cents per share in the same quarter last year [1] - The company did not record any revenues in the reported quarter, missing the Zacks Consensus Estimate for total revenues of $8 million [2] - General and administrative expenses rose 28% year over year to $28.4 million, attributed to increased commercial preparation and legal expenses [2] - Research and development expenses were $35.9 million, down 21% from the previous year due to reduced manufacturing and development costs [3] - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024, with expectations to fund operations into Q4 2026 [3] - Year to date, RCKT shares have declined 44%, compared to an 8% decline in the industry [4] Pipeline Developments - Kresladi, developed for treating severe leukocyte adhesion deficiency-I (LAD-I), received a complete response letter (CRL) from the FDA in June 2024, requesting limited additional information on the Chemistry Manufacturing and Controls (CMC) [7][8] - The company plans to file a complete BLA to resolve the CRL later in 2025 [8] - Rocket Pharmaceuticals is developing RP-L102 for treating Fanconi anemia (FA) and has initiated a rolling BLA, expecting to complete the submission in late 2025 or early 2026 [9] - Dosing is currently underway in a phase II pivotal study for RP-A501, targeting male patients with Danon disease, with clinical data readout expected in mid-2026 [10]

Financial Performance - As of March 31, 2025, Rocket Pharmaceuticals had cash, cash equivalents, and investments totaling approximately $318.2 million, expected to fund operations into the fourth quarter of 2026[8]. - Research and development expenses for Q1 2025 were $35.9 million, a decrease of $9.3 million from $45.2 million in Q1 2024, primarily due to reduced manufacturing and development costs[11]. - General and administrative expenses increased to $28.4 million in Q1 2025 from $22.1 million in Q1 2024, driven by higher legal expenses and commercial preparation costs[11]. - The net loss for Q1 2025 was $61.3 million, or $0.56 per share, compared to a net loss of $62.1 million, or $0.66 per share, in Q1 2024[11]. - The total operating expenses for Q1 2025 were $64.4 million, a decrease from $67.4 million in Q1 2024[15]. Clinical Development - The ongoing Phase 2 pivotal study of RP-A501 for Danon disease is expected to provide a program update in mid-2025, with clinical data readout anticipated in mid-2026[4]. - Initial data from the Phase 1 study of RP-A601 for PKP2-ACM is expected in May 2025, with an estimated 50,000 people affected in the U.S. and Europe[6]. - The company is on track to submit an IND for the BAG3-DCM program in mid-2025[3]. - Rocket Pharmaceuticals is progressing with the rolling Biologics License Application (BLA) for RP-L102 for Fanconi Anemia, with final module submission anticipated in late 2025 or early 2026[6]. Leadership Changes - The company appointed Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer in April 2025[5].

Clinical Programs and Trials - The company has two clinical-stage and one pre-clinical stage in vivo adeno-associated viral (AAV) programs, with the RP-A501 pivotal study for Danon disease (DD) completing enrollment in September 2024[105]. - The FDA accepted the BLA for RP-L201 for severe Leukocyte Adhesion Deficiency-I (LAD-I) and granted priority review, with a complete BLA submission anticipated in 2025[106]. - The RP-A501 Phase 1 clinical trial treated seven patients, with a low-dose cohort showing sustained LAMP2 protein expression and a reduction of left ventricular mass index by ≥10% at 12 months[120][123]. - The company aims to submit BLAs for its clinical programs and expand its gene therapy pipeline to target additional indications in the medium to long term[114]. - The company has received FDA clearance for an IND application for RP-A601 and initiated a Phase 1 study for this program[105]. - The RP-L102 program completed Phase 2 studies in 2023, with a rolling BLA submission initiated in September 2024 and EMA acceptance of the MAA in April 2024[106]. - The company is focusing resources on other programs and has not yet initiated enrollment in the Phase 2 RP-L301 study, despite having reached agreement with the FDA on the study design[106]. - All evaluable patients in the Phase 1 trial demonstrated improvements across tissue, laboratory, and imaging-based biomarkers, with a median improvement of 27 points in Kansas City Cardiomyopathy Questionnaire scores[130]. - The ongoing pivotal Phase 2 trial aims for a median reduction of 10% in left ventricular mass index and positive protein expression of Grade 1 or more as co-primary endpoints[133]. - RP-A501 received RMAT designation from the FDA in February 2023 and PRIME designation from the EMA in May 2023, with enrollment of 12 patients in the Phase 2 study completed in September 2024[137]. - The global Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with dilated cardiomyopathy, including a pediatric safety run-in of 2 patients[131]. - The ongoing Phase 2 study for RP-L102 has shown sustained genetic correction in 8 of 12 evaluable patients, with a favorable safety profile[154]. - The Phase 1/2 trial for RP-L201 has treated nine patients, with 100% survival without the need for allogeneic transplant[160]. - The company is conducting a global Phase 1 study for RP-L301, with positive updates showing sustained efficacy and no serious adverse events reported[166]. Market and Financial Overview - The prevalence of Danon disease is estimated to be between 15,000 to 30,000 patients in the U.S. and EU, with no specific therapies currently available[118]. - BAG3-associated dilated cardiomyopathy (BAG3-DCM) has a prevalence of approximately 30,000 individuals in the U.S., with a high unmet medical need for effective therapies[145]. - The addressable annual market opportunity for RP-L102 in the U.S. and EU is estimated to be 400 to 500 patients[151]. - The company has raised approximately $1.2 billion from investors through equity and convertible debt financing since inception, funding its operating activities[172]. - The company has not generated any revenue from product sales to date and does not expect to do so in the near future[173]. - The company had an accumulated deficit of $1.28 billion as of March 31, 2025, compared to $1.22 billion as of December 31, 2024[194]. - Cash used in operating activities was $55.8 million for the three months ended March 31, 2025, compared to $56.9 million for the same period in 2024[196]. - Net cash used by investing activities was $58.0 million for the three months ended March 31, 2025, compared to a net cash provided of $35.0 million in the same period of 2024[199][200]. - The company expects R&D expenses to increase for the foreseeable future as it continues to invest in product candidates and clinical trials[180]. - The company has $318.2 million in cash, cash equivalents, and investments as of March 31, 2025, which is expected to fund operations into the fourth quarter of 2026[194]. Research and Development Expenses - The company's direct R&D expenses are primarily external costs associated with preclinical research, process development, and clinical development activities[175]. - R&D expenses decreased by $9.3 million to $35.9 million for the three months ended March 31, 2025, compared to $45.2 million in the same period of 2024[189]. - General and administrative expenses increased by $6.3 million to $28.4 million for the three months ended March 31, 2025, compared to $22.1 million in the same period of 2024[190]. - Total operating expenses decreased by $2.99 million to $64.4 million for the three months ended March 31, 2025, compared to $67.4 million in the same period of 2024[188]. - The company believes its cash, cash equivalents, and marketable securities do not contain excessive risk, but acknowledges potential future adverse changes in market value[205]. Manufacturing and Production - The company has successfully produced multiple commercial-grade Danon AAV cGMP batches since 2022, which are expected to optimize the safety profile of RP-A501[136]. - The company has a 103,720 square foot cGMP manufacturing facility in Cranbury, New Jersey, supporting its gene therapy pipeline[170]. - The company is exploring preclinical research to expand applications of its proprietary LV and AAV program platforms for additional rare genetic diseases[171]. Investment and Financial Strategy - The company's investments are primarily in U.S. Treasury Securities, indicating a conservative investment strategy[204]. - Interest and other income decreased by $1.7 million to $1.3 million for the three months ended March 31, 2025, compared to $3.0 million in the same period of 2024[191]. - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024[204]. - A hypothetical increase in market interest rates by 100 basis points would result in a decline of $1.0 million in the net fair value of the company's investments[205]. - The company does not utilize interest rate hedging agreements or other interest rate derivative instruments, exposing it to interest rate sensitivity risk[204].