CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease.

CRANBURY，N.J.-（商业电线）-火箭制药公司（纳斯达克股票代码：RCKT）是一家领先的晚期生物技术公司，致力于推进针对需求未得到满足的罕见疾病的综合可持续基因治疗管道，今天宣布与美国食品和药物管理局（FDA）就RP-A501治疗Danon病的全球2期关键试验达成一致。

Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male patients at age ~20 and females at age ~40, and for which there are no approved curative or disease-modifying therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe..

达农病是一种致命的遗传性心肌病，可导致20岁左右的大多数男性患者和40岁左右的女性死亡，并且尚无批准的治疗方法或可改善疾病的疗法。在美国和欧洲，该病估计影响15000至30000名患者。。

“I am very excited to announce our alignment with the FDA on our pivotal study design for RP-A501 for Danon Disease, which reflects the highly collaborative discussions with the review team and senior management at FDA’s Center for Biologics Evaluation and Research and marks the first-ever regulatory pathway to approval for a genetic treatment for heart disease.

“我非常高兴地宣布我们与FDA就达农病RP-A501的关键研究设计达成一致，这反映了与FDA生物制品评估和研究中心审查小组和高级管理层的高度合作讨论，并标志着第一个有史以来批准心脏病基因治疗的监管途径。

We believe this milestone sets us on the most efficient and rapid path to delivering this potentially transformative therapy to Danon Disease patients who would otherwise progress to heart transplantation or death,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “I would also like to highlight the work conducted by our CMC team over the past several years to establish our in-house cGMP manufacturing capabilities, which has already provided us with sufficient material for the pivotal study and should support our eventual commercialization efforts.”.

我们相信，这一里程碑为我们提供了最有效，最快速的途径，向Danon病患者提供这种潜在的变革性治疗，否则这些患者将进展为心脏移植或死亡，“火箭制药公司首席执行官Gaurav Shah博士说。“我还要强调我们的CMC团队在过去几年中开展的工作，以建立我们内部的cGMP制造能力，这已经为我们的关键研究提供了足够的材料，并应该支持我们最终的商业化努力。”。

Dr. Shah continued “As a one-time potentially curative infusion, RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options. With today’s progress in our Danon Disease program, we believe we are forging a path to bring curative gene therapies to patients affected by devastating cardiovascular diseases and broadening the possibilities for addressing the large array of inherited heart diseases through the promise of cardiac gene therapy.”.

博士。沙阿继续“作为一次性潜在治愈性输注，RP-A501有可能恢复正常的心脏功能，并为没有其他可行治疗选择的Danon病患者提供终身益处。随着今天Danon疾病计划的进展，我们相信我们正在开辟一条道路，为受破坏性心血管疾病影响的患者提供治疗性基因治疗nd通过心脏基因治疗的希望扩大了解决大量遗传性心脏病的可能性。”。

Phase 2 Pivotal Trial of RP-A501 for Danon Disease

RP-A501治疗Danon病的2期关键试验

The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.

全球单臂多中心2期关键试验将评估RP-A501在12例Danon病患者中的疗效和安全性，包括儿科安全磨合（n=2），具有自然史比较和剂量水平为6.7 x 1013 GC/kg。

To support accelerated approval, the study will assess the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression (≥ Grade 1, as measured by immunohistochemistry), and reductions in left ventricular (LV) mass.

为了支持加速批准，该研究将评估RP-A501的功效，通过基于生物标志物的共同主要终点测量，包括LAMP2蛋白表达的改善（≥级1，通过免疫组织化学测量）和左心室的减少（LV）质量。

Key secondary endpoint is change in troponin. Additional secondary endpoints will include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire (KCCQ), New York Heart Association (NYHA) class, event free survival to 24 months and treatment emergent safety events. These endpoints could support full approval with longer-term follow-up..

关键的次要终点是肌钙蛋白的变化。其他次要终点将包括利钠肽，堪萨斯城心肌病问卷（KCCQ），纽约心脏协会（NYHA）课程，无事件生存至24个月和治疗紧急安全事件。这些终点可以支持长期随访的全面批准。。

A global natural history study will serve as an external comparator and run concurrently to the Phase 2 pivotal trial.

全球自然史研究将作为外部比较，并与第二阶段关键性试验同时进行。

In-house manufacturing has been completed with sufficient high-quality drug product produced to fully supply the Phase 2 pivotal study. Potency assays have been developed and qualified in accordance with FDA guidance.

内部制造已经完成，生产了足够的高质量药品，以充分提供第二阶段的关键性研究。根据FDA的指导，已经开发并合格了效力测定。

Filing of the Clinical Trial Application (CTA)/Investigational Medicinal Product Dossier (IMPD) for RP-A501 to enable initiation of EU study activities is anticipated in the third quarter of this year. Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease

预计今年第三季度将提交RP-A501的临床试验申请（CTA）/研究药品档案（IMPD），以便启动欧盟研究活动。此外，火箭已经获得了一个ICD-10代码从CMS的LAMP2缺陷在达农病

About RP-A501

关于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 (AAV9.LAMP2B) 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是火箭公司用于治疗Danon病的研究性基因治疗产品，也是第一个在临床研究中证明安全性和有效性的心血管疾病基因治疗产品。达农病是由LAMP2基因突变引起的。RP-A501由含有全长野生型人LAMP2B转基因（AAV9.LAMP2B）的重组腺相关血清型9（AAV9）衣壳组成，当其插入携带内源性LAMP2突变的心脏细胞中时基因，有可能完全恢复其根部的心脏功能。

RP-A501 represents a single dose treatment and is administered as an intravenous (IV) infusion. In preclinical and clinical studies, AAV9.LAMP2B 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..

RP-A501代表单剂量治疗并且作为静脉内（IV）输注施用。在临床前和临床研究中，AAV9.LAMP2B已显示靶向心脏细胞（心肌细胞）并将功能性LAMP2B基因递送至心脏组织，最终导致患者心脏结构和功能的改善。。

About Danon Disease

关于达农病

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.

Danon病是一种罕见的X连锁遗传性疾病，由编码溶酶体相关膜蛋白2（LAMP-2）（一种重要的自噬介质）的基因突变引起。这导致自噬体和糖原的积累，特别是在心肌和其他组织中的积累，最终导致心力衰竭，对于男性患者，在青春期或成年早期经常死亡。

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. There is a high unmet medical need for patients with Danon Disease..

据估计，在美国和欧洲，该病的患病率为15000至30000。达农病唯一可用的治疗选择是心脏移植，这与严重的并发症有关，并且不被认为是治愈性的。Danon病患者的医疗需求很高。。

About Rocket Pharmaceuticals, Inc.

关于火箭制药公司。

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases.

Rocket Pharmaceuticals，Inc.（纳斯达克股票代码：RCKT）正在推进旨在纠正复杂和罕见儿童疾病根本原因的综合且可持续的研究性遗传疗法管道。该公司与平台无关的方法使其能够为每种适应症设计最佳治疗方案，为患有罕见遗传疾病的患者创造潜在的变革性选择。

Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure 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 rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

火箭公司使用基于慢病毒载体（LVV）的基因治疗的临床项目用于治疗范可尼贫血（FA），这是一种难以治疗的遗传性疾病，可导致骨髓衰竭和潜在的癌症，白细胞粘附缺乏症-I（LAD-I），一种严重的儿科遗传性疾病，可导致经常致命的复发性和威胁生命的感染，和丙酮酸激酶缺乏症（PKD），一种罕见的单基因红细胞疾病，导致红细胞破坏增加和轻度至危及生命的贫血。

Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has received IND clearance for the AAV-based gene therapy program for PKP2-arrhythmogenic cardiomyopathy (ACM) and is advancing a preclinical program for BAG3-associated dilated cardiomyopathy (DCM).

火箭使用基于腺相关病毒（AAV）的基因治疗的第一个临床计划是针对Danon病，这是一种破坏性的儿科心力衰竭病症。Rocket还获得了针对PKP2心律失常性心肌病（ACM）的基于AAV的基因治疗计划的IND许可，并且正在推进BAG3相关性扩张型心肌病（DCM）的临床前计划。

For more information about Rocket, please visit www.rocketpharma.com..

有关火箭的更多信息，请访问www.rocketpharma.com。。

Rocket Cautionary Statement Regarding Forward-Looking Statements

火箭关于前瞻性陈述的警示声明

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Danon Disease (DD), 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, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions.

本版本中有关Rocket未来预期，计划和前景的各种陈述，包括但不限于Rocket对Rocket正在开发用于治疗Danon病（DD）的候选产品的安全性和有效性的期望，Rocket正在进行的预期时间和数据读数和计划的临床试验，Rocket监管互动和计划提交的预期时间和结果，Rocket推进其Danon疾病计划的计划，包括其计划的关键试验，以及相关临床前研究和临床试验的安全性，有效性和时间安排，对于1995年“私人证券诉讼改革法案”和其他联邦证券法律规定的安全港条款而言，可能构成前瞻性声明，并存在重大风险，不确定性和假设。

You should not place reliance on these forward-looking statements, which often include words such as '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. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

您不应该依赖这些前瞻性陈述，这些陈述通常包括诸如“相信”，“期望”，“预期”，“打算”，“计划”，“将会给出”，“估计”，“寻求，“将会”，“可能”，“建议”或类似的术语，这些术语的变化或这些术语的否定。尽管Rocket认为前瞻性声明中反映的预期是合理的，但Rocket无法保证这种结果。

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 ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expe.

由于各种重要因素，实际结果可能与这些前瞻性声明所表明的结果有很大不同，包括但不限于Rocket监测COVID-19对其业务运营的影响并采取措施确保患者安全的能力，家庭和员工，患者和家属对参与Rocket正在进行的每项试验，患者登记，试验时间表和数据读数的兴趣，我们的经验。