KING OF PRUSSIA, Pa. & SAN DIEGO--(BUSINESS WIRE)--Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) and Arcturus Therapeutics (Nasdaq: ARCT) today announce Nature Communications has published results from an integrated phase 1/2/3a/3b study evaluating the safety, immunogenicity, and efficacy of ARCT-154, a novel self-amplifying (sa-mRNA) COVID-19 vaccine and the world’s first approved sa-mRNA COVID-19 vaccine..

普鲁士国王，宾夕法尼亚州和圣地亚哥--（商业新闻短讯）--全球生物技术领导者CSL（ASX:CSL；USOTC:CSLLY）和Arcturus Therapeutics（Nasdaq:ARCT）今天宣布，Nature Communications发布了一项综合性1/2/3a/3b期研究的结果，该研究评估了ARCT-154的安全性，免疫原性和有效性，ARCT-154是一种新型自扩增（sa mRNA）COVID-19疫苗，也是世界上第一种经批准的sa mRNA COVID-19疫苗。。

The results demonstrate that two 5 μg doses of ARCT-154, sa-mRNA vaccine, were well-tolerated, immunogenic and provided significant protection against multiple strains of COVID-19. The efficacy of ARCT-154 against severe COVID-19 was 100 percent in healthy persons aged 18-59 and more than 90 percent in persons at risk of severe consequences of the disease due to co-morbidities or older age..

结果表明，两种5μg剂量的ARCT-154（sa mRNA疫苗）具有良好的耐受性，免疫原性，并对多种COVID-19菌株具有显着的保护作用。在18-59岁的健康人群中，ARCT-154对严重新型冠状病毒肺炎的疗效为100%，在因合并症或年龄较大而有严重后果风险的人群中，ARCT-154的疗效超过90%。。

“The results published in Nature Communications demonstrate the efficacy and tolerability of ARCT-154 and add to a growing body of evidence that our sa-mRNA vaccine has the potential to provide significant protection against the pervasive virus, reinforcing our promise to protect public health,” said Jon Edelman, M.D., Senior Vice President, Vaccines Innovation Unit, CSL..

CSL疫苗创新部高级副总裁Jon Edelman医学博士说：“发表在《自然通讯》上的研究结果证明了ARCT-154的有效性和耐受性，并增加了越来越多的证据表明，我们的sa mRNA疫苗有可能对普遍存在的病毒提供显着的保护，增强了我们保护公共健康的承诺。”。。

“We are thrilled that the results of the ARCT-154 study have been published in the highly respected journal Nature Communications,” said Pad Chivukula, Ph.D., Chief Scientific Officer of Arcturus Therapeutics. “These data and the approval in Japan highlight the strength of our and CSL’s commitment to delivering innovative technology that protects the public from COVID-19.”.

Arcturus Therapeutics首席科学官Pad Chivukula博士说：“我们很高兴ARCT-154研究的结果已经发表在备受尊敬的《自然通讯》杂志上。”。“这些数据和日本的批准突显了我们和CSL致力于提供创新技术以保护公众免受新型冠状病毒肺炎的力量。”。

The Nature Communications article titled, “Safety, immunogenicity and efficacy of the self-amplifying mRNA ARCT-154 COVID-19 vaccine: pooled phase 1, 2, 3a and 3b randomized, controlled trials” was published online.

《自然通讯》的一篇文章题为“自扩增mRNA ARCT-154 COVID-19疫苗的安全性，免疫原性和有效性：合并的1、2、3a和3b期随机对照试验”在线发表。

About the study

关于这项研究

During the observer-blind, randomized, controlled phase 1, 2, 3a and 3b integrated study, adults ≥18 years old receive two 5 μg doses of ARCT-154 or saline placebo 28 days apart. Phase 2/3a/3b participants were stratified by age (< 60 or ≥ 60 years of age) and by risk of severe COVID-19 prior to being randomized 3:1 (phase 1/2/3a) or 1:1 (phase 3b) to vaccine or placebo groups.

在观察者盲，随机，对照的1,2,3a和3b期综合研究中，年龄≥18岁的成年人间隔28天接受两次5μg剂量的ARCT-154或生理盐水安慰剂。在被随机分配到3:1（1/2/3a期）或1:1（3b期）之前，2/3a/3b期参与者按年龄（<60岁或≥60岁）和严重新型冠状病毒肺炎的风险进行分层。疫苗或安慰剂组。

The primary endpoints were vaccine efficacy up to 2 months after dose 2, reactogenicity within up to 7 days of each dose, safety within up to 28 days after each dose, and immunogenicity measured 28 days after each dose. From August 15 to January 12, 2023, 1,001 participants were randomized (748 ARCT-154 and 253 placebo) in the integrated phase 1/2/3a study, and 16,100 participants (8,056 ARCT-154 and 8,044 placebo) in the phase 3b study..

主要终点是第2次剂量后2个月内的疫苗效力，每次剂量后7天内的反应原性，每次剂量后28天内的安全性以及每次剂量后28天测量的免疫原性。从2023年8月15日到1月12日，在综合1/2/3a期研究中，1001名参与者被随机分配（748名ARCT-154和253名安慰剂），在3b期研究中有16100名参与者（8056名ARCT-154和8044名安慰剂）。。

In the phase 1/2/3a studies, ARCT-154 was safe and well tolerated. Most solicited adverse events were mild or moderate and resolved quickly, and rates of related or severe unsolicited adverse events were similar in the ARCT-154 and placebo groups. The phase 3b study confirmed these observations.

在1/2/3a期研究中，ARCT-154安全且耐受性良好。大多数征求的不良事件是轻度或中度的，并且很快得到解决，ARCT-154组和安慰剂组的相关或严重未经请求的不良事件发生率相似。3b期研究证实了这些观察结果。

Four weeks after the second ARCT-154 dose in phase 3b, the neutralizing antibody seroconversion rate was 94.1% (95% CI: 92.1–95.8). There were 640 confirmed, protocol-defined COVID-19 cases, mainly of the Delta variant, that were determined to be eligible for analysis, including 43 severe cases and 10 deaths attributed to COVID-19.

在第3b期第二次ARCT-154剂量后四周，中和抗体血清转化率为94.1%（95%CI:92.1-95.8）。有640例确诊的协议定义的新型冠状病毒肺炎病例，主要是Delta变异，被确定有资格进行分析，包括43例严重病例和10例因新型冠状病毒肺炎而死亡。

ARCT-154 absolute efficacy was 56.6% (95% CI: 48.7–63.3) against any COVID-19, 95.3% (80.5–98.9) against severe COVID-19 and 86.5% (-7.4–98.3) against death due to COVID-19. Efficacy against severe COVID-19 was 100% in healthy 18-59-year-olds and 91.9% (37.9–98.9) in participants in that age group with underlying co-morbidities, which put them at risk for severe disease.

ARCT-154对任何新型冠状病毒肺炎的绝对疗效分别为56.6%（95%可信区间：48.7-63.3）、95.3%（80.5-98.9）和86.5%（-7.4-98.3）。在健康的18-59岁人群中，对严重新型冠状病毒肺炎的疗效为100%，在该年龄组中，有潜在合并症的参与者中，对严重新型冠状病毒肺炎的疗效为91.9%（37.9-98.9），这使他们面临严重疾病的风险。

In adults aged 60 years or older, efficacy was 54.3% (28.2–70.9) against COVID-19 of any severity and 94.4% (58.2–99.3) against severe COVID-19..

在60岁或以上的成年人中，对任何严重程度的新型冠状病毒肺炎的疗效分别为54.3%（28.2-70.9）和94.4%（58.2-99.3）。。

The study was co-funded by Vinbiocare Biotechnology Joint Stock Company in Hanoi, Vietnam, and Arcturus Therapeutics.

这项研究由越南河内的Vinbiocare生物技术股份有限公司和Arcturus Therapeutics共同资助。

About sa-mRNA

关于sa mRNA

mRNA vaccines help protect against infectious diseases by providing a blueprint for cells in the body to make a protein to help our immune systems recognize and fight the disease. Unlike standard mRNA vaccines, self-amplifying mRNA vaccines instruct the body to make more mRNA and protein to boost the immune response..

mRNA疫苗通过为体内细胞提供蓝图，使其产生蛋白质，帮助我们的免疫系统识别和抗击疾病，从而有助于预防传染病。与标准的mRNA疫苗不同，自我扩增的mRNA疫苗指导身体产生更多的mRNA和蛋白质来增强免疫反应。。

About CSL

关于CSL

CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies.

CSL（ASX:CSL；USOTC:CSLLY）是一家全球生物技术公司，拥有一系列充满活力的救生药物，包括治疗血友病和免疫缺陷的药物，预防流感的疫苗以及缺铁和肾脏病的疗法。自1916年成立以来，我们一直致力于使用最新技术拯救生命。

Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

如今，CSL（包括我们的三大业务：CSL Behring、CSL Sequirus和CSL Vifor）为100多个国家的患者提供救生产品，拥有32000名员工。我们独特的商业实力、研发重点和卓越运营相结合，使我们能够识别、开发和提供创新，使我们的患者能够过上最充实的生活。

For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit www.CSL.com..

有关生物技术前景的鼓舞人心的故事，请访问CSLBehring.com/Vita，并在Twitter.com/CSL上关注我们。有关CSL的更多信息，请访问www.CSL.com。。

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 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 (Kostaive®) 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（Kostaive®），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 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、中国和其他国家有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。。