SAN DIEGO--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today announced that the European Commission (EC), based on a positive opinion issued by the European Medicines Agency (EMA), has granted orphan medicinal product designation for the Company’s product candidate ARCT-032 to treat Cystic Fibrosis (CF).

圣地亚哥--（商业新闻短讯）--Arcturus Therapeutics Holdings Inc.（以下简称“公司”，“Arcturus”，纳斯达克：ARCT），一家全球晚期临床信使RNA药物公司，专注于开发传染病疫苗和药物，以治疗肝脏和呼吸道罕见疾病中未满足的医疗需求，今天宣布欧盟委员会（EC），根据欧洲药品管理局（EMA）发布的积极意见，已授予该公司候选产品ARCT-032治疗囊性纤维化（CF）的孤儿药品名称。

In November 2023, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARCT-032 for CF..

2023年11月，美国食品和药物管理局（FDA）为CF授予ARCT-032孤儿药名称。。

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 EU. The designation provides significant incentives to promote the development of the drug including protocol assistance, access to the centralized authorization procedure, fee reductions, and research grants, as well as 10 years of market exclusivity..

欧盟委员会指定的孤儿药品可用于预防或治疗危及生命或长期衰弱的疾病的新型疗法，这些疾病在欧盟影响不到10000人中的5人。该名称为促进该药物的开发提供了重要的激励措施，包括协议援助，获得集中授权程序，减少费用和研究资助，以及10年的市场排他性。。

“We are pleased to receive orphan medicinal product designation from the EC for ARCT-032, as it represents a significant milestone for our CF program,” said Joseph Payne, President & CEO of Arcturus Therapeutics. “We will continue to advance ARCT-032 as a potential new treatment option for people with CF regardless of their genetic mutations.”.

Arcturus Therapeutics总裁兼首席执行官约瑟夫·佩恩（JosephPayne）表示：“我们很高兴从EC获得ARCT-032孤儿药产品的称号，因为它代表了我们CF计划的一个重要里程碑。”。“我们将继续推进ARCT-032作为CF患者的潜在新治疗选择，无论其基因突变如何。”。

After successful completion of the single-ascending-dose portion of the study in healthy adults, the first participant with CF in the Phase 1b study part enrolled and completed two administrations of ARCT-032. Arcturus remains on track to share interim Phase 1b data in H1 2024.

在健康成年人成功完成单次递增剂量部分的研究后，1b期研究部分的第一名CF参与者登记并完成了两次ARCT-032给药。大角星仍有望在2024年上半年共享1b期中期数据。

About Cystic Fibrosis

关于囊性纤维化

Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis.

囊性纤维化是一种寿命缩短的疾病，在世界范围内分布。囊性纤维化跨膜电导调节剂（CFTR）基因的突变导致气道中CFTR蛋白和/或功能的减少或缺失，导致氯化物转运不足以维持气道表面稳态。

CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in some people with CF because of their underlying mutations..

CF粘液更难清除，从而阻塞气道并导致感染，炎症，呼吸衰竭或其他危及生命的并发症。目前批准的CFTR调节剂疗法旨在增加CFTR通道的功能，以帮助减轻症状，但由于其潜在的突变，对某些CF患者无效。。

About ARCT-032

关于ARCT-032

ARCT-032 utilizes Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease.

ARCT-032利用大角星的LUNAR®脂质介导的雾化平台将CFTR信使RNA递送至肺部。CF患者肺部CFTR mRNA功能拷贝的表达有可能恢复CFTR活性并减轻导致进行性肺部疾病的下游影响。

The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of CFTR expression and function in human bronchial epithelial cells..

ARCT-032计划得到了啮齿动物，雪貂和灵长类动物的临床前数据的支持，并证明了CFTR在人支气管上皮细胞中表达和功能的恢复。。

About Arcturus Therapeutics

关于大角星治疗学

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise.

Arcturus Therapeutics Holdings Inc.（纳斯达克股票代码：ARCT）成立于2013年，总部位于加利福尼亚州圣地亚哥，是一家全球晚期临床mRNA药物和疫苗公司，拥有以下技术：（i）LUNAR®脂质介导的递送，（ii）STARR®mRNA技术（sa mRNA）和（iii）mRNA药物以及药品制造专业知识。

Arcturus developed the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics.

大角星开发了世界上第一种自我扩增信使RNA（sa mRNA）新型冠状病毒疫苗，并获得批准。Arcturus正在与CSL-Seqirus进行创新mRNA疫苗的全球合作，并在日本成立了一家合资企业ARCALIS，专注于mRNA疫苗和治疗剂的生产。

Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase deficiency and cystic fibrosis, along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics.

Arcturus的管道包括可能治疗鸟氨酸转氨甲酰酶缺乏症和囊性纤维化的RNA治疗候选物，以及针对SARS-CoV-2（COVID-19）和流感的合作mRNA疫苗计划。大角星的多功能RNA治疗平台可应用于多种类型的核酸药物，包括信使RNA，小干扰RNA，环状RNA，反义RNA，自扩增RNA，DNA和基因编辑疗法。

Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications issued in the U.S., Europe, Japan, China, and other countries). For more information, visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn..

大角星的技术涵盖了其广泛的专利组合（在美国、欧洲、日本、中国和其他国家发布了400多项专利和专利申请）。有关更多信息，请访问www.ArcturusRx.com。此外，请通过Twitter和LinkedIn与我们联系。。

Forward Looking Statements

前瞻性声明

This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, are forward-looking statements, including those regarding the continued clinical development of ARCT-032, the likelihood of sharing and timing for sharing interim Phase 1b data, likelihood of success (including safety and efficacy) of ARCT-032, the Company’s strategy and future operations, and the impact of general business and economic conditions.

本新闻稿包含前瞻性声明，涉及1995年《私人证券诉讼改革法案》提供的安全港的重大风险和不确定性。除本新闻稿中包含的历史事实声明外，任何声明均为前瞻性声明，包括有关ARCT-032的持续临床开发，共享1b期中期数据的可能性和时间，ARCT-032成功的可能性（包括安全性和有效性），公司战略和未来运营，以及一般商业和经济状况的影响。

Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. These statements are only current predictions or expectations, and are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements, including those discussed under the heading 'Risk Factors' in Arcturus’ most recent Annual Report on Form 10-K, and in subsequent filings with, or submissions to, the SEC, which are available on the SEC’s website at www.sec.gov.

大角星可能无法实际实现上述任何前瞻性声明中披露的计划、意图或期望或预测，您不应过度依赖此类前瞻性声明。这些声明仅是当前的预测或预期，并受到已知和未知风险、不确定性以及其他因素的影响，这些因素可能导致我们或我们行业的实际结果、活动水平、绩效或成就与前瞻性声明的预期大不相同，包括Arcturus最新年度报告（表10-K）中“风险因素”标题下讨论的内容，以及随后向美国证券交易委员会提交的文件或提交给美国证券交易委员会的文件，这些文件可在美国证券交易委员会网站www.SEC.gov上查阅。

Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise..

除非法律另有要求，大角星不打算或有义务更新或修订任何前瞻性声明，这些声明仅在其发布之日起发表，无论是由于新信息、未来事件或情况或其他原因。。