MORRISVILLE, NC., July 8, 2024 /PRNewswire/ -- Immorna Biotherapeutics, Inc. (Immorna), a clinical stage biotechnology company developing both self-replicating and conventional mRNA-based therapeutics and vaccines, announced today that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for JCXH-211 intravenous (IV), a novel, first-in-class self-replicating mRNA (srRNA) encoding the engineered human interleukin (IL)-12 protein..

北卡罗来纳州莫里斯维尔，2024年7月8日/PRNewswire/--Immorna Biotherapeutics，Inc.（Immorna），一家开发自我复制和常规基于mRNA的治疗和疫苗的临床阶段生物技术公司，今天宣布，美国食品和药物管理局（FDA）已批准其JCXH-211静脉注射（IV）的研究性新药（IND）申请，JCXH-211静脉注射（IV）是一种编码工程化人白细胞介素（IL）-12蛋白的新型一流自我复制mRNA（srRNA）。。

The IND clearance allows Immorna to initiate a Phase 1/2, multi-center, open-label, dose escalation and expansion study of JCXH-211 administered intravenously in patients with malignant solid tumors. The goal of the study is to assess safety and tolerability, determine the recommended Phase 2 dose (RP2D) for JCXH-211 IV in combination with checkpoint inhibitor (CPI), and assess preliminary efficacy of the combination at the RP2D..

IND清除率允许Immorna启动恶性实体瘤患者静脉注射JCXH-211的1/2期，多中心，开放标签，剂量递增和扩展研究。该研究的目的是评估安全性和耐受性，确定JCXH-211 IV联合检查点抑制剂（CPI）的推荐2期剂量（RP2D），并评估该组合在RP2D的初步疗效。。

'We are excited to have achieved this important milestone for one of our key assets,' said NgocDiep Le, M.D., Ph.D., President and Global Chief Medical Officer of Immorna. Based on data from our preclinical studies, clinical data from our JCXH-211 intratumoral administration (IT) trial, and the candidate drug's mechanism of action, we believe JCXH-211 IV in combination with CPI has the potential to work synergistically to enhance anti-tumor effect.

Immorna总裁兼全球首席医疗官、医学博士NgocDiep Le说，我们很高兴能够为我们的关键资产之一实现这一重要里程碑。根据我们临床前研究的数据，JCXH-211肿瘤内给药（IT）试验的临床数据以及候选药物的作用机制，我们认为JCXH-211 IV与CPI联合使用有可能协同作用以增强抗肿瘤作用。

We look forward to working with the investigators and patients to bring this potential novel therapy to patients who are in dire need of new and effective treatments.'.

我们期待着与研究人员和患者合作，为急需新的有效治疗方法的患者带来这种潜在的新疗法。”。

JCXH-211 is a first-in-class lipid nanoparticle (LNP) encapsulated srRNA, using Immorna's proprietary technology, encoding the engineered human IL-12 protein. In multiple preclinical animal and patient-derived xenograft (PDX) models, the anti-viral innate response triggered by RNA replicon together with the potent anti-cancer immunity stimulated by IL-12 conferred JCXH-211 superior tumor-eradicating potency, which was better than similar preclinical candidates employing conventional (non-replicating) mRNA.

JCXH-211是一流的脂质纳米颗粒（LNP）封装的sRNA，使用Immorna的专有技术，编码工程化的人IL-12蛋白。在多种临床前动物和患者来源的异种移植物（PDX）模型中，由RNA复制子触发的抗病毒先天反应以及由IL-12刺激的有效抗癌免疫赋予JCXH-211优异的肿瘤根除效力，这优于使用常规（非复制）mRNA的类似临床前候选物。

Interim data from a Phase 1 trial of JCXH-211 IT monotherapy has demonstrated excellent safety, tolerability and significant anti-tumor biological activities, including abscopal effect..

JCXH-211 IT单药治疗1期临床试验的中期数据显示出优异的安全性，耐受性和显着的抗肿瘤生物活性，包括远隔效应。。

IL-12 is a naturally occurring cytokine that plays a key role in the body's immune response against cancer. Despite consistently showing potent antitumor activity in preclinical studies, recombinant IL-12 protein treatment at tolerable doses in humans failed to provide clinical benefit. One key challenge for IL-12 protein treatment is the non-overlap of tolerability window and therapeutic window.

IL-12是一种天然存在的细胞因子，在人体对抗癌症的免疫反应中起着关键作用。尽管在临床前研究中一直显示出有效的抗肿瘤活性，但在人类可耐受剂量下的重组IL-12蛋白治疗未能提供临床益处。IL-12蛋白治疗的一个关键挑战是耐受性窗口和治疗窗口的不重叠。

Recombinant IL-12 is unstable in vivo and have a very short half-life, and frequent intravenous administration of recombinant human IL-12 protein was challenging due to unacceptable toxicities. Thanks to the intrinsic feature of our srRNA technology, JCXH-211 IV enables a prolonged expression of IL-12 preferentially in tumor tissues rather than normal tissues, leading to modulation of tumor microenvironment and activation of antitumor immune responses, while minimizing systemic toxicity.

重组IL-12在体内不稳定，半衰期很短，由于不可接受的毒性，频繁静脉注射重组人IL-12蛋白具有挑战性。由于我们的srRNA技术的内在特征，JCXH-211 IV能够优先在肿瘤组织而不是正常组织中延长IL-12的表达，从而调节肿瘤微环境和激活抗肿瘤免疫应答，同时最大程度地降低全身毒性。

JCXH-211-IV demonstrated excellent safety profile in nonclinical studies using rodents and non-human primates. If successful, JCXH-211 IV may become another lifesaving yet easily accessible therapy for cancer patients who have progressed on or are resistant to currently available treatments..

JCXH-211-IV在使用啮齿动物和非人灵长类动物的非临床研究中表现出优异的安全性。如果成功，JCXH-211 IV可能会成为另一种挽救生命但易于获得的治疗方法，适用于已进展或对现有治疗方法有抵抗力的癌症患者。。

More information can be found at www.immorna.com.

有关更多信息，请访问www.immorna.com。

About Immorna

关于Immora

Immorna is a rapidly expanding biotechnology company, focusing on the development of RNA-based therapeutics and vaccines. Immorna is utilizing multiple RNA platforms, including conventional, self-replicating and circular RNAs.

Immorna是一家快速发展的生物技术公司，专注于基于RNA的疗法和疫苗的开发。Immorna正在利用多种RNA平台，包括常规，自我复制和环状RNA。

Since its founding in 2019, Immorna has built a robust CMC platform for RNA synthesis, purification, and analytical testing that is well suited for clinical and commercial development. In addition, with its state-of-the-art screening tools, Immorna has developed an arsenal of RNA delivery vehicles, including polymers and lipid nanoparticles featuring multiple proprietary ionizable cationic lipids suitable for intramuscular, intravenous and tissue-targeting delivery..

自2019年成立以来，Immorna已经建立了一个强大的CMC平台，用于RNA合成，纯化和分析测试，非常适合临床和商业开发。此外，凭借其最先进的筛选工具，Immorna开发了一系列RNA递送载体，包括聚合物和脂质纳米颗粒，其具有多种专有的可电离阳离子脂质，适用于肌内，静脉内和组织靶向递送。。

Immorna has a growing intellectual property portfolio and a diverse RNA development pipeline spanning cancer immunotherapy, infectious diseases, rare genetic diseases, and medical cosmetology, and quickly advancing its oncology drug and infectious disease vaccine candidates into clinical stages.

Immorna拥有不断增长的知识产权组合和多样化的RNA开发渠道，涵盖癌症免疫疗法，传染病，罕见遗传病和医学美容，并迅速将其肿瘤药物和传染病疫苗候选药物推进临床阶段。