Latest news with #GauravShah
Business Wire
07-08-2025
- Business
- Business Wire
Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Progress
CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the second quarter ending June 30, 2025. 'The second quarter of 2025 marked an important inflection point for Rocket as we refined our strategic focus around our AAV cardiovascular gene therapy platform and took multiple decisive steps to strengthen our financial foundation and thoughtfully adjust to market dynamics. We are fortifying our path to sustained value creation by leaning into programs with the highest value, conserving cash, and driving an efficient, agile organization,' said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. 'With regulatory alignment to resolve the clinical hold for RP-A501 in Danon disease in progress, momentum building for RP-A601 in PKP2-ACM as it advances toward a pivotal Phase 2 trial, and RP-A701 preparing to enter the clinic for the treatment of BAG3-DCM, Rocket is positioned as the leader in the development of gene therapies for inherited cardiomyopathies. While we are pausing additional investments in the FA and PKD programs, we are deeply grateful and committed to the patient communities and are exploring strategic alternatives to advance these programs externally. Finally, our recent reorganization ensures we are appropriately resourced to execute on our near-term milestones with nearly two years of capital.' Recent Pipeline and Operational Updates Investigation into the Serious Adverse Event (SAE) from the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing. Rocket previously disclosed an SAE, related to clinical complications from capillary leak syndrome and unfortunately leading to the patient's death. In response, Rocket paused dosing and initiated a root cause analysis, focusing on the recent addition of a C3 inhibitor to the pre-treatment regimen. On May 23, 2025, FDA placed the trial on clinical hold for further evaluation. Rocket is actively working with FDA, independent safety monitors, and clinical experts to ensure patient safety and resume the trial. While the clinical hold remains in place, the company cannot provide guidance on trial completion timing. Actively engaging with FDA on the advancement of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) in potential pivotal trial following initial positive Phase 1 data at the 28 th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). In July, Rocket received FDA Regeneration Medicine Advanced Therapy (RMAT) designation for RP-A601 for PKP2-ACM. Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy outcomes of RP-A601 for PKP2-ACM were presented at ASGCT in May 2025. Rocket is engaging with FDA on potential pivotal trial design to evaluate the efficacy and safety of RP-A601. Phase 1 trial start-up activities are underway for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). In June, an Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-DCM, received clearance from FDA. RP-A701 was recently granted FDA Fast Track designation, designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions that fill an unmet medical need. This enables increased communication with FDA, the potential for accelerated approval, and permits a rolling Biologics License Application (BLA) review. The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. BAG3-DCM is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality. Rocket estimates that the prevalence of BAG3-DCM in the U.S. is as many as 30,000 individuals. FDA review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADI TM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). Rocket previously disclosed that FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I. The Company continues to work with senior leaders and reviewers from FDA's Center for Biologics Evaluation and Research and submission of complete BLA to resolve Complete Response Letter is anticipated before the end of 2025. Ongoing strategic corporate restructuring and pipeline prioritization. As part of its broader strategic reorganization, Rocket implemented a workforce reduction of approximately 30%, across all functions, to align with a pipeline prioritization plan focused exclusively on its AAV cardiovascular gene therapy platform. This restructuring, together with other cost-saving measures, is expected to reduce operating expenses by nearly 25% over the next 12 months. The reorganization enables the company to focus on its late‑stage AAV gene therapy programs in Danon disease, PKP2‑ACM, and BAG3‑DCM, while advancing regulatory activities for KRESLADI™ in severe LAD‑I. As part of this realignment, Rocket is pausing additional investments in its Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. The company continues to evaluate options to advance the FA program with health authorities in alignment with its refined strategic focus and broader corporate priorities. In July, Rocket appointed Chris Stevens as Chief Operating Officer. Chris Stevens is a highly seasoned executive with 25 years of experience across technical operations, product strategy, and general management, bringing immense commercial technical operations experience to Rocket. Stevens most recently served as the Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics (subsidiary of Roche), where he successfully led teams across manufacturing, supply chain, quality, compliance, engineering, EHS, and facilities management, playing a key role in delivering gene therapies to patients. Second Quarter 2025 Financial Results Cash position. Cash, cash equivalents and investments as of June 30, 2025, were $271.5 million. Rocket expects such resources, excluding any potential proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI TM, will be sufficient to fund its operations into the second quarter of 2027. R&D expenses. Research and development expenses were $42.7 million for the three months ended June 30, 2025, compared to $46.3 million for the three months ended June 30, 2024. The decrease of $3.7 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $2.3 million, professional fees of $2.0 million and building supplies and consumables of $0.8 million, offset by an increase in clinical trial expenses of $1.2 million. G&A expenses. General and administrative expenses were $25.0 million for the three months ended June 30, 2025, compared to $27.4 million for the three months ended June 30, 2024. The decrease in G&A expenses was primarily driven by decreases in commercial preparation related expenses of $1.4 million and compensation and benefits expense of $0.9 million. Net loss. Net loss was $68.9 million or $0.62 per share (basic and diluted) for the three months ended June 30, 2025, compared to $69.6 million or $0.74 (basic and diluted) for the three months ended June 30, 2024. Shares outstanding. 107,884,420 shares of common stock were outstanding as of June 30, 2025. Restructuring Expenses and Financial Guidance Restructuring expenses. Approximately $3.5 million in restructuring and restructuring-related charges were incurred in the first half of 2025. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's ability to realize the intended benefits of the restructuring plan and reduction in workforce, expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to fund its operations into the second quarter of 2027. There can be no assurance that the restructuring plan or the planned reduction in workforce will have the intended effect on the Company's operational results and strategic decisions, that any anticipated charges and any anticipated cost savings associated with the restructuring plan or the reduction in workforce will achieve their intended benefits. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Business Wire
24-07-2025
- Business
- Business Wire
Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs
BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced a strategic corporate reorganization and pipeline prioritization aimed at maximizing near-term value, extending operational runway into the second quarter of 2027, and positioning the company for sustained long-term growth. The restructuring and reprioritization initiative focuses Rocket's resources on the adeno-associated virus (AAV) cardiovascular platform, comprised of clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM), as well as submitting the completed responses to the CRL for KRESLADI™ (formerly RP-L201) for severe leukocyte adhesion deficiency-I (LAD-I). The transition reflects the company's focus on its late-stage AAV pipeline, a strategy previously announced in January 2025. The company will implement a reduction in workforce of approximately 30%, which, along with other planned cost-saving initiatives, is expected to reduce Rocket's 12-month operating expenses by nearly 25%. Rocket anticipates that its existing cash resources, excluding any potential proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI™, will fund operations into the second quarter of 2027. 'We are grateful for the remarkable contributions of our entire team and the significant progress we've made in advancing genetic therapies for rare diseases,' said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. 'Our prioritization reflects a commitment to the programs with the most compelling near-term opportunities for patients and to setting a foundation for Rocket's long-term growth and success. Together, our cardiovascular programs have demonstrated strong potential for impact in areas of high unmet need in substantial patient populations.' Rocket is advancing its leadership in cardiovascular gene therapy by strategically leveraging its AAV platform to address monogenic cardiomyopathies with high unmet need. With a portfolio spanning Danon disease, PKP2-ACM, and BAG3-DCM, Rocket is uniquely positioned to develop one-time, potentially curative treatments for distinct molecular subtypes of heart failure. Collectively, these programs target the major genetically defined causes of hypertrophic, arrhythmogenic, and dilated cardiomyopathies which represent a significant portion of inherited heart disease and impact approximately more than 100,000 patients in the U.S. and EU. Based on business and strategic priorities and as part of its realignment, Rocket anticipates delays associated with the Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. FDA approval of RP-L102 is no longer anticipated in 2026. These adjustments reflect a disciplined approach to capital allocation and a strategic reprioritization of assets. Additional information is expected to be provided with second quarter earnings updates as the company continues to evaluate strategic options. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's ability to realize the intended benefits of the restructuring plan and reduction in workforce, expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to fund its operations into the second quarter of 2027. There can be no assurance that the restructuring plan or the planned reduction in workforce will have the intended effect on the Company's operational results and strategic decisions, that any anticipated charges and any anticipated cost savings associated with the restructuring plan or the reduction in workforce will achieve their intended benefits. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Yahoo
27-05-2025
- Business
- Yahoo
Patient dies in trial of Rocket gene therapy for heart condition
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A patient who received an experimental gene therapy developed by Rocket Pharmaceuticals has died following treatment, the company disclosed Tuesday. According to Rocket, the patient experienced what's known as capillary leak syndrome, where plasma and proteins seep from blood vessels into surrounding tissue, and later died from an acute systemic infection. The Food and Drug Administration ordered the study's halt Friday, while Rocket works with clinical trial monitors and other experts to investigate the cause. The company said it is focusing on a 'novel immune suppression agent' it recently added to a pre-treatment preparatory regimen. Rocket had proactively modified its pre-treatment regimen in attempt to reduce the risk of an immune response known as 'complement activation,' which it previously saw evidence of in the Phase 2 trial. Two patients received the immune suppression agent, company CEO Gaurav Shah explained on a conference call Tuesday morning. Rocket was able to reduce the treatment course the second patient received after seeing early signs of capillary leak syndrome in the first. The company is now exploring whether the additional drug it used — a so-called C3 inhibitor — 'may have influenced immune responses in an unexpected or paradoxical way,' said Shah, who noted that the only difference in protocol for these two patients was the change in preparatory regimen. Shah said Rocket is confident there remains a path forward for its gene therapy, which is designed to treat a rare inherited disorder known as Danon disease. The condition results in the accumulation of sugars and certain cellular components in cardiac muscle, leading to heart failure and early death. 'This is a deeply tragic loss. We are committed to fully understanding the circumstances surrounding it objectively and neutrally,' said Shah. 'We are also immensely grateful to the family for their contribution to this important clinical research and their commitment to help advance science for the broader Danon community.' Now, however, Rocket must prove to the FDA, researchers and its investors that its treatment, dubbed RP-A501, remains safe. It's a position many other gene therapy developers have found themselves in after unexpected patient deaths in trials. So far, investors appear skeptical Rocket will have an easy answer. Shares in the company slumped by more than 60% in Tuesday morning trading, erasing hundreds of millions of dollars from the company's market capitalization. Rocket emphasized that the introduction of the new immune agent was limited only to the Danon program, and not used in the company's other pipeline projects. The company said it can't predict when it may be able to resume the Phase 2 trial, which was expected to enroll 12 patients. RP-A501 is built around a version of the gene, called LAMP2B, that's mutated in people with Danon. The replacement DNA is encased in an engineered viral shell known as an adenovirus-associated serotype 9 capsid. The therapy is infused, and is meant to be a one-time treatment.
Yahoo
27-05-2025
- Business
- Yahoo
Rocket Pharmaceuticals Provides Update on Phase 2 Clinical Trial of RP-A501 for Danon Disease
Conference call to be held later today at 8:30 a.m. ET CRANBURY, N.J., May 27, 2025--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing an integrated pipeline of genetic therapies for rare diseases, today announced an update related to RP-A501, its investigational gene therapy for Danon disease. A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE). The SAE involved clinical complications related to a capillary leak syndrome. Rocket is conducting a comprehensive root cause analysis and remains in active dialogue with the U.S. Food and Drug Administration (FDA) and other key stakeholders, with the current focus being on the recent introduction of a novel immune suppression agent to the pre-treatment regimen that had been implemented to mitigate complement activation observed in some patients. This novel agent was specific to the AAV9-Danon program. Upon learning of the initial event, Rocket voluntarily paused further dosing in the study. On May 23, 2025, the FDA placed a clinical hold on the trial to allow for further evaluation. Rocket is deeply saddened to report that this patient has since passed away after an acute systemic infection. Rocket is working with the FDA, the Independent Data Safety Monitoring Committee, clinical investigators, and scientific experts, and is committed to ensuring the safety of all study patients while resuming the trial as expeditiously as possible. While the clinical hold remains in place, the company is unable to provide guidance on the anticipated timing for completion of the Phase 2 trial. "We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon. We are immensely grateful for the patients and families who participate in this important research," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. Rocket extends our deepest sympathies to the patient's family, caregivers, and the treating clinical team. Rocket also continues its longstanding collaboration with the Danon Foundation, whose commitment to research, advocacy, and patient support remains integral to advancing the field and improving outcomes for individuals affected by Danon disease. As of March 31, 2025, Rocket had cash, cash equivalents, and investments of $318.2 million. The company is prioritizing investment in its AAV platform while assessing ways to optimize value for the rest of the pipeline. This will translate into a reduction in overall cash spend. Rocket now expects its existing resources will be sufficient to fund operations into 2027, excluding any potential proceeds from the sale of Priority Review Vouchers that could follow the approval of therapies from the hematology portfolio. Conference Call Information Rocket will host a conference call today, May 27, 2025, at 8:30 a.m. ET. To join the call, please register at Webinar details will be posted on the Events and Presentations page of the Rocket website at RP-A501 Phase 2 Pivotal Trial Overview The global, single-arm, multi-center Phase 2 pivotal trial evaluates the efficacy and safety of RP-A501 in 12 patients with Danon disease, including a pediatric safety run-in (n=2), and a dose level of 6.7 x 1013 GC/kg. To support accelerated approval, the study assesses the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression, and reductions in left ventricular mass. Key secondary endpoint is change in troponin. Additional secondary endpoints include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire, New York Heart Association class, event free survival to 24 months and treatment emergent safety events. These endpoints could support full approval with longer-term follow-up. A global natural history study is running concurrently with the Phase 2 pivotal trial. The pediatric run-in enrolled two patients in a sequential manner with a minimum three-month follow-up prior to subsequent enrollment. In addition, all patients enrolled in the trial are required to have a three-months observational pre-treatment run-in to enable an assessment of troponin (and other biomarker) trajectories to optimally assess this key secondary endpoint. Details about the Phase 2 study can be found at under NCT identifier NCT06092034. About RP-A501 RP-A501 is Rocket's investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene. RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene ( which, when inserted into heart cells harboring mutations in the endogenous LAMP2 gene, has the potential to fully restore cardiac function at its root. RP-A501 represents a single dose treatment and is administered as an intravenous infusion. In preclinical and clinical studies, has been shown to target cardiac cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improved cardiac structure and function in patients. In 2023, the U.S. Food and Drug Administration granted regenerative medicine advanced therapy designation, and the European Medical Agency granted PRIority MEdicines (PRIME) eligibility to RP-A501. About Danon Disease Danon disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon disease is cardiac transplantation, which is associated with substantial complications and is not considered curative, representing the high unmet medical need for patients with Danon disease. In 2023, Rocket secured an ICD-10 code from the Centers for Medicare and Medicaid Services (CMS) to document patients with LAMP2 deficiency in Danon disease. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as "could," "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into 2027. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. View source version on Contacts Investors Meg Dodgemdodge@ Media Kevin Giordanomedia@
Business Wire
27-05-2025
- Business
- Business Wire
Rocket Pharmaceuticals Provides Update on Phase 2 Clinical Trial of RP-A501 for Danon Disease
BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing an integrated pipeline of genetic therapies for rare diseases, today announced an update related to RP-A501, its investigational gene therapy for Danon disease. A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE). The SAE involved clinical complications related to a capillary leak syndrome. Rocket is conducting a comprehensive root cause analysis and remains in active dialogue with the U.S. Food and Drug Administration (FDA) and other key stakeholders, with the current focus being on the recent introduction of a novel immune suppression agent to the pre-treatment regimen that had been implemented to mitigate complement activation observed in some patients. This novel agent was specific to the AAV9-Danon program. Upon learning of the initial event, Rocket voluntarily paused further dosing in the study. On May 23, 2025, the FDA placed a clinical hold on the trial to allow for further evaluation. Rocket is deeply saddened to report that this patient has since passed away after an acute systemic infection. Rocket is working with the FDA, the Independent Data Safety Monitoring Committee, clinical investigators, and scientific experts, and is committed to ensuring the safety of all study patients while resuming the trial as expeditiously as possible. While the clinical hold remains in place, the company is unable to provide guidance on the anticipated timing for completion of the Phase 2 trial. 'We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon. We are immensely grateful for the patients and families who participate in this important research,' said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. Rocket extends our deepest sympathies to the patient's family, caregivers, and the treating clinical team. Rocket also continues its longstanding collaboration with the Danon Foundation, whose commitment to research, advocacy, and patient support remains integral to advancing the field and improving outcomes for individuals affected by Danon disease. As of March 31, 2025, Rocket had cash, cash equivalents, and investments of $318.2 million. The company is prioritizing investment in its AAV platform while assessing ways to optimize value for the rest of the pipeline. This will translate into a reduction in overall cash spend. Rocket now expects its existing resources will be sufficient to fund operations into 2027, excluding any potential proceeds from the sale of Priority Review Vouchers that could follow the approval of therapies from the hematology portfolio. Conference Call Information Rocket will host a conference call today, May 27, 2025, at 8:30 a.m. ET. To join the call, please register at Webinar details will be posted on the Events and Presentations page of the Rocket website at RP-A501 Phase 2 Pivotal Trial Overview The global, single-arm, multi-center Phase 2 pivotal trial evaluates the efficacy and safety of RP-A501 in 12 patients with Danon disease, including a pediatric safety run-in (n=2), and a dose level of 6.7 x 10 13 GC/kg. To support accelerated approval, the study assesses the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression, and reductions in left ventricular mass. Key secondary endpoint is change in troponin. Additional secondary endpoints include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire, New York Heart Association class, event free survival to 24 months and treatment emergent safety events. These endpoints could support full approval with longer-term follow-up. A global natural history study is running concurrently with the Phase 2 pivotal trial. The pediatric run-in enrolled two patients in a sequential manner with a minimum three-month follow-up prior to subsequent enrollment. In addition, all patients enrolled in the trial are required to have a three-months observational pre-treatment run-in to enable an assessment of troponin (and other biomarker) trajectories to optimally assess this key secondary endpoint. Details about the Phase 2 study can be found at under NCT identifier NCT06092034. About RP-A501 RP-A501 is Rocket's investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene. RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene ( which, when inserted into heart cells harboring mutations in the endogenous LAMP2 gene, has the potential to fully restore cardiac function at its root. RP-A501 represents a single dose treatment and is administered as an intravenous infusion. In preclinical and clinical studies, has been shown to target cardiac cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improved cardiac structure and function in patients. In 2023, the U.S. Food and Drug Administration granted regenerative medicine advanced therapy designation, and the European Medical Agency granted PRIority MEdicines (PRIME) eligibility to RP-A501. About Danon Disease Danon disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon disease is cardiac transplantation, which is associated with substantial complications and is not considered curative, representing the high unmet medical need for patients with Danon disease. In 2023, Rocket secured an ICD-10 code from the Centers for Medicare and Medicaid Services (CMS) to document patients with LAMP2 deficiency in Danon disease. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into 2027. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
