RP-A501

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 quarter ending June 30, 2024. “ Over the quarter, Rocket has been advancing its clinical pipeline as we progressed our RP-A501 and RP-A601 cardiac programs, targeting Danon disease and PKP2-ACM, and continued to actively enroll patients,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “ At ASGCT, we shared follow-up data from across our hematology portfolio including 4-year follow-up data for KRESLADI to treat patients with severe LAD-I, demonstrating a 100% survival rate. In parallel, we have been preparing for the anticipated FDA approval of KRESLADI.” Recent Pipeline and Operational Updates Continued advancement of Phase 2 pivotal study of RP-A501 for Danon Disease . Enrollment in the Phase 2 pivotal study of RP-A501 to treat Danon Disease is actively progressing. Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Granted orphan medicinal product designation from the European Commission (EC) for RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM). In May, Rocket announced orphan medicinal product designation from the EC for RP-A601 for the treatment of PKP2-ACM. Enrollment in the Phase 1 study is ongoing. Details of the study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412. Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the European Union (EU). The designation qualifies for financial and regulatory benefits including protocol assistance from the European Medicines Agency during clinical development, access to centralized marketing authorization, and a 10-year period of marketing exclusivity after product approval. U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information underway for KRESLADI ™ for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). In June, Rocket announced that the FDA requested limited additional CMC information to complete its review of KRESLADI ™ (marnetegragene autotemcel; marne-cel) to treat severe LAD-I. The Company is working with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research to support the approval of KRESLADI ™ . Long-term KRESLADI ™ follow-up data from the global Phase 1/2 study were presented at the American Society of Gene and Cell Therapy (ASGCT) 27 th Annual Meeting. Data demonstrated survival of 100% in the absence of allogeneic hematopoietic stem cell transplantation from 18 to 45 months with a well-tolerated safety profile in all nine patients with severe LAD-I. Progressed Fanconi Anemia (FA) program through regulatory and clinical milestones. Regulatory filings and review for RP-L102 for the treatment of FA are on track with health authorities in the U.S. and Europe. Results from the global Phase 1/2 study of RP-L102 were presented at the ASGCT 27 th Annual Meeting. Previously disclosed data demonstrated genetic and phenotypic correction combined with hematologic stabilization extending to 42 months with polyclonal integration patterns. Furthered Pyruvate Kinase Deficiency (PKD) program through clinical milestones. Updated data from the Phase 1 study of RP-L301 for PKD were presented at the ASGCT 27 th Annual Meeting. Sustained and clinically meaningful hemoglobin improvement and well-tolerated safety profile were observed in PKD patients up to 36 months after RP-L301 treatment. The global Phase 2 pivotal study of RP-L301 for PKD has been initiated. Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06422351. Progressing BAG3-associated dilated cardiomyopathy preclinical program. Nonclinical, IND-enabling studies are ongoing. Second Quarter Financial Results Cash position. Cash, cash equivalents and investments as of June 30, 2024, were $278.8 million. R&D expenses. Research and development expenses were $91.6 million for the six months ended June 30, 2024, compared to $97.8 million for the six months ended June 30, 2023. The decrease of $6.2 million in R&D expenses was driven by decreases in manufacturing and development and direct costs of $14.9 million. The decreases were partially offset by increases in the costs for compensation and benefits of $1.2 million due to increased R&D headcount, professional fees of $3.4 million, laboratory supplies of $0.6 million, non-cash stock compensation expense of $1.1 million, and clinical trial costs of $1.4 million. G&A expenses. General and administrative expenses were $49.5 million for the six months ended June 30, 2024, compared to $33.2 million for the six months ended June 30, 2023. The increase in G&A expenses was primarily driven by increased commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $9.5 million, legal expenses of $3.3 million, and non-cash stock compensation expense of $1.4 million. Net loss. Net loss was $131.7 million or $1.40 per share (basic and diluted) for the six months ended June 30, 2024, compared to $124.0 million or $1.55 (basic and diluted) for the six months ended June 30, 2023. Shares outstanding. 90,956,613 shares of common stock were outstanding as of June 30, 2024. Financial Guidance Cash position. As of June 30, 2024, Rocket had cash, cash equivalents and investments of $278.8 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs. 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 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. 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. For more information about Rocket, please visit www.rocketpharma.com 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 2026. 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, 2023, filed February 27, 2024 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. Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Operating expenses: Research and development $ 46,345 $ 51,383 $ 91,572 $ 97,754 General and administrative 27,367 17,374 49,515 33,197 Total operating expenses 73,712 68,757 141,087 130,951 Loss from operations (73,712 ) (68,757 ) (141,087 ) (130,951 ) Interest expense (471 ) (468 ) (942 ) (936 ) Interest and other income, net 2,294 846 5,323 2,754 Accretion of discount on investments, net 2,243 2,678 5,006 5,097 Net loss $ (69,646 ) $ (65,701 ) $ (131,700 ) $ (124,036 ) Net loss per share - basic and diluted $ (0.74 ) $ (0.82 ) $ (1.40 ) $ (1.55 ) Weighted-average common shares outstanding - basic and diluted 93,746,243 80,472,362 93,759,894 79,965,755 June 30, 2024 December 31, 2023 Cash, cash equivalents, and investments $ 278,825 $ 407,495 Total assets 446,411 566,341 Total liabilities 61,776 73,767 Total stockholders' equity 384,635 492,574

2024年6月28日，Rocket Pharmaceuticals宣布美国食品和药物管理局（FDA）针对其公司在研基因疗法KRESLADI™ （marnegragene autotemcel; marne-cel）的生物制剂许可申请发出了一份完整答复函 (CRL)，其中FDA要求提供有限的额外化学制造和控制 (CMC) 信息以完成审查。 此前，该机构以需要更多时间审查生产过程为由，将其决定推迟了三个月。 Kresladi（曾用名RP-L201）是一款用以治疗严重白细胞粘附缺陷-I（LAD-I）的在研基因疗法，该疗法通过慢病毒对患者的自体造血干细胞进行基因工程化改造，以递送ITGB2基因的功能性拷贝。该基因编码β-2整合素成分CD18，这是一种促进白细胞粘附并使其从血管中渗出以抵抗感染的关键蛋白质。 该疗法获美国FDA授予再生医学先进疗法（RMAT）认定、罕见儿科疾病认定和快速通道资格，在欧盟拥有PRIME和先进治疗药物（ATMP）资格，并且在两个地区拥有孤儿药资格。 KRESLADI™全球1/2期研究的积极一线数据显示，在18至42个月的随访期间，所有9名LAD-I患者在输液后12个月（以及整个随访期间）的总存活率均为100%。数据还显示，与治疗前相比，重大感染的发生率大幅下降，同时有证据表明，LAD-I相关皮肤病变得到了缓解，伤口修复能力得到了恢复。所有主要和次要终点均已达到，所有患者对 KRESLADI™ 的耐受性良好，未发生与治疗相关的严重不良事件。 Rocket与FDA生物制剂评估与研究中心 (CBER) 的高层领导会面，就所需的有限范围的额外CMC信息达成一致，以支持KRESLADI™尽快获得批准。Rocket Pharma 首席执行官、医学博士Gaurav Shah说："FDA 是一个密切的合作者，它了解大量未满足的医疗需求、明确的临床益处以及患者及时就医的重要性，这一点令人欣慰。CBER 领导层的直接参与和为患者迅速提供这种疗法的承诺让我们对原发性免疫缺陷社区充满了希望。" 关于 Rocket Pharmaceuticals Rocket Pharmaceuticals是一家全面整合的后期生物技术公司，正在推进一条可持续的基因疗法研究管线，旨在纠正复杂罕见疾病的根源。Rocket的创新性多平台方法使我们能够为每种适应症设计最佳基因疗法，创造出具有潜在变革性的选择，使患有毁灭性罕见疾病的患者能够获得长久而充实的生活。 Rocket公司基于慢病毒（LV）载体的血液学产品组合包括针对范可尼贫血症（FA）的RP-L102、白细胞粘附缺陷 I 型（LAD-I）的Kresladi、 以及治疗丙酮酸激酶缺乏症（PKD）的RP-L301。 Rocket 公司基于腺相关病毒（AAV）载体的心血管产品组合包括针对达农病的在研基因疗法RP-A501，目前该疗法正在进行关键2期试验；针对PKP2-致心律失常性心肌病的基因疗法RP-A601，以及针对BAG3相关性扩张型心肌病的临床前项目。 参考资料：公司公告